title: An Open-label, Prospective Multicenter Study to Assess the Pharmacokinetics, Tolerability, Safety and Efficacy of the Pediatric Formulation of Bosentan Two Versus Three Times a Day in Children With Pulmonary Arterial Hypertension

acronym: FUTURE 3

summary: The primary objective of AC-052-373 was to assess the pharmacokinetic (PK) profile of two dosing regimens of the pediatric formulation of bosentan in children with pulmonary arterial hypertension (PAH) <12 years of age.

lead_sponsor: Actelion

location_country:
Poland
Belarus
Serbia
Italy
Russian Federation
Australia
Mexico
Netherlands
Spain
Ukraine
Hungary
United States
France
China
India
Israel
South Africa
Germany
Czechia

location_facility: State Educational Institution of Higher Professional Education "Saint Petersburg State Pediatric Medical Academy of Roszdrav" - Site 3801

location_facility: Children's National Medical Center - Site 9104

location_facility: Shanghai Children's Medical Center - Site 5102

location_facility: Schneider Children's Medical Center- Institute of pediatric cardiology - Site 7101

location_facility: Federal State Institution "Federal center of Heart, Blood and Endocrinology named after V.A.Almazov Rosmedtekhnologies" - Site 3802

location_facility: Deutsches Herzzentrum Kinderkardiologie - Site 1401

location_facility: Shanghai Pulmonary Hospital, Department of Pulmonary Circulation - Site 5101

location_facility: Hospital Universitatario Vall d'Hebron, Neumologia - Site 1907

location_facility: Universitätsklinikum Bonn Abteilung für Kinderkardiologie - Site 1404

location_facility: RAMS Institution, Research Institute for complex issues of cardiovascular diseases, Siberian branch of the Russian Academy of Medical Sciences - Site 3805

location_facility: Beijing Anzhen Hospital, Capital Medical University- Department of Pediatric Cardiology - Site 5103

location_facility: Clinical Diagnostic Center - Pediatric Cardiovascular and ANES and Intensive Care Department - Site 4103

location_facility: Columbia University Medical Center Children's Hospital of New York Presbyterian - Site 9101

location_facility: The Republican Scientific-Practical Center "Cardiology" - Site 3001

location_facility: Division of Paediatric Cardiology, Steve Biko Academic Hospital - Site 6002

location_facility: Universitair Medish Centrum Groningen, Kindercardiologie - Site 1601

location_facility: Ospedale Pediatrico "Bambino Gesù" - Dipartimento Medico Chirurgico di Cardiologia Pediatrica - Site 1502

location_facility: Paediatric Cardiology Albert Luthuli Central Hospital - Site 6003

location_facility: Moscow Scientific Research Institute for Pediatrics and Childrens Surgery of Rosmedtechnologies - Site 3804

location_facility: Hospital Universitario La Paz - Paediatric Cardiology Department - Site 1906

location_facility: Royal Children's Hospital Melbourne, Cardiology - Site 5001

location_facility: Indraprashta Apollo Hospitals, Pediatric Cardiology - Site 5303

location_facility: Department of Paediatric Cardiology University of the Free State - Site 6001

location_facility: Cardiovascular Institute and Fuwai Hospital

location_facility: Gusak Ins Urgent and Recovery SUR AMS - Cardiovascular Rehabilitation Pediatric Department - Site 4101

location_facility: Instytut Centrum Zdrowia Matki Polki Klinika Kardiologii ICMP w Lodz - Site 3602

location_facility: Justus-Liebig-Universität Giessen, Kinderherzzentrum - Site 1403

location_facility: Università Degli Studi di Padova - Dipartimento di Pediatria - Servizio di Cardiologia Pediatrica - Site 1501

location_facility: West China 2nd university Hospital-Center of interventional diagnosis and therapy for Children's cardiovascular disease - Site 5104

location_facility: CHU de Toulouse - Hôpital des Enfants, Service de Cardiologie Pédiatrique - Site 2202

location_facility: Instytut Pomnik - Centrum Zdrowia Dziecka Klinika Kardiologii Dziecięcej - Site 3601

location_facility: Gover INS - Scientific Practical Cardiovascular Pediatric Center - MOH Ukraine - Site 4102

location_facility: Fakultní nemocnice v Motole, dětské kardiocentrum - Site 3301

location_facility: Texas Children's Hospital - Department of Cardiology - Site 9107

location_facility: Scientific Center of Cardiovascular Surgery named after A.N.Bakulev of the RAMS - Site 3803

location_facility: Institut za zdravstvenu zaštitu majke i deteta Srbije "Dr Vukan Čupić", Služba za ispitivanje i lečenje bolesti srca i krvnih sudova - Site 3902

location_facility: Instituto Nacional de Cardiologia (INC) Ignacio Chavez - Site 8401

location_facility: Wojewódzki Szpital Specjalistyczny we Wrocławiu Oddział Kardiologii Dziecięcej z pododdziałem Intensywnego Nadzoru Kardiologicznego - Site 3605

location_facility: CARE Hospitals, Cardiology Dep. Hyderabad - Site 5302

location_facility: Szegedi Tudományegyetem ÁOK Szent-Györgyi Albert Klinikai Központ, Gyermekgyógyászati Klinika és Gyermekegészségügyi Központ - Site 3402

location_facility: Univerzitetska dečja klinika, Služba za kardiologiju - Site 3901

location_facility: Uniwersyteckie Centrum Kliniczne Klinika Kardiologii Dziecięcej i Wad Wrodzonych Serca - Site 3604

location_facility: Hopital Necker-Enfants Malades, Service de Cardiologie Pédiatrique - Site 2201

location_facility: Gottsegen György Országos Kardiológiai Intézet, Gyermekszív Központ, Gyermek Kardiológiai osztály - Site 3401

location_facility: Unidad de Investigacion Clinica en Medicina, SC (UDICEM) - Site 8402


title: EnSite™ HD Grid Catheter Mapping System for Advanced High Density Three-Dimensional Mapping in Non-Paroxysmal Atrial Fibrillation and Atrial Tachycardia

summary: In this clinical investigation, the safety, feasibility and performance of the novel EnSite™ HD Grid Catheter mapping system for advanced high-density three-dimensional mapping will be studied in patients undergoing catheter ablation procedures for the treatment of non-paroxysmal atrial fibrillation (AF) or left atrial tachycardia (AT).

study_start_date_type: Actual

status: Completed

study_completion_date_type: Actual

study_type: Interventional

primary_purpose: Diagnostic

enrollment_type: Actual

lead_sponsor: Abbott Medical Devices

location_country:
Hong Kong
Italy
Australia
France
Germany

location_facility: Herzzentrum Dresden GmbH Universitatsklinik

intervention_type: Device

lead_sponsor_type: industriel

status_simplified: Completed


type: EudraCT Number

title: Randomized, Double-Masked, Sham-Controlled Clinical Trial to Evaluate the Efficacy of a Single Intravitreal Injection of GS010 in Subjects Affected for More Than 6 Months and To 12 Months by LHON Due to the G11778A Mutation in the ND4 Gene

acronym: REVERSE

summary: The goal of this clinical trial is to assess the effectiveness of GS010, a gene therapy, in improving the visual outcome in participants with Leber Hereditary Optic Neuropathy (LHON) due to the G11778A ND4 mitochondrial mutation when vision loss is present for more than six months and up to one year.

design_allocation: Randomized

primary_purpose: Treatment

ReferenceType: derived

ReferenceCitation: Newman NJ, Yu-Wai-Man P, Carelli V, Biousse V, Moster ML, Vignal-Clermont C, Sergott RC, Klopstock T, Sadun AA, Girmens JF, La Morgia C, DeBusk AA, Jurkute N, Priglinger C, Karanjia R, Josse C, Salzmann J, Montestruc F, Roux M, Taiel M, Sahel JA. Intravitreal Gene Therapy vs. Natural History in Patients With Leber Hereditary Optic Neuropathy Carrying the m.11778G>A ND4 Mutation: Systematic Review and Indirect Comparison. Front Neurol. 2021 May 24;12:662838. doi: 10.3389/fneur.2021.662838. eCollection 2021.

ipd_sharing: No

lead_sponsor: GenSight Biologics

location_country:
United Kingdom
Italy
United States
France
Germany

location_facility: Department of Neurology, University of Munich, Friedrich-Baur-Institute

location_facility: Neuro Ophthalmologic Associates, Wills Eye Hospital, Thomas Jefferson University

location_facility: Centre National Hospitalier d'Ophtalmologie des Quinze-Vingt

location_facility: Moorfields Eye Hospital NHS Foundation Trust

location_facility: IRCCS Istituto delle Scienze Neurologiche di Bologna, UOC Clinica Neurologica, Ospedale Bellaria

location_facility: Doheny Eye Center, University of California, Los Angeles

location_facility: Department of Ophthalmology, Emory University School of Medicine

intervention_type: Biological


title: Study of GSK2302024A Antigen-Specific Cancer Immunotherapeutic Combined With Standard Neoadjuvant Treatment in Patients With WT1-positive Primary Invasive Breast Cancer

acronym: INDUCT

summary: The purpose of this study is to evaluate the safety, immunogenicity and clinical activity of a new WT1 anti-cancer immunotherapy in patients with WT1-positive Stage II or III breast cancer. The treatment will be given before surgery in combination with standard therapy.

status: Terminated

ReferenceCitation: Higgins M, Curigliano G, Dieras V, Kuemmel S, Kunz G, Fasching PA, Campone M, Bachelot T, Krivorotko P, Chan S, Ferro A, Schwartzberg L, Gillet M, De Sousa Alves PM, Wascotte V, Lehmann FF, Goss P. Safety and immunogenicity of neoadjuvant treatment using WT1-immunotherapeutic in combination with standard therapy in patients with WT1-positive Stage II/III breast cancer: a randomized Phase I study. Breast Cancer Res Treat. 2017 Apr;162(3):479-488. doi: 10.1007/s10549-017-4130-y. Epub 2017 Feb 7.

ipd_sharing: Yes

ipd_sharing_description: IPD for this study is available via the Clinical Study Data Request site (click on the link provided below).

lead_sponsor: GlaxoSmithKline

location_country:
United Kingdom
Italy
Russian Federation
United States
France
Belgium
Germany

status_simplified: Unknown


intervention_type: Drug

title: An Open-label, Multicenter, Multinational Extension Study of the Long-term Safety, Pharmacodynamics, and Exploratory Efficacy of GZ/SAR402671 in Adult Male Patients Diagnosed With Fabry Disease

lead_sponsor: Genzyme, a Sanofi Company

type: Other Identifier

source: euctr

location_country:
Poland
United Kingdom
Russian Federation
United States
France

location_facility:
Investigational Site Number 643002
Investigational Site Number 840002
Investigational Site Number 840001
Investigational Site Number 826002
Investigational Site Number 840003
Investigational Site Number 250001
Investigational Site Number 616001

summary:
Primary Objective:

To assess the long-term safety of GZ/SAR402671 in adult male participants with Fabry disease who previously completed study ACT13739 (NCT02489344).

Secondary Objective:

To assess the long-term effect of GZ/SAR402671 on pharmacodynamic and exploratory efficacy endpoints in adult male participants with Fabry disease who previously completed study ACT13739.


title: A Phase Ib/II, Multicenter, Open-label Study of EGF816 in Combination With INC280 in Adult Patients With EGFR Mutated Non-small Cell Lung Cancer.

lead_sponsor: Novartis Pharmaceuticals

ReferenceCitation: Jia Y, Juarez J, Li J, Manuia M, Niederst MJ, Tompkins C, Timple N, Vaillancourt MT, Pferdekamper AC, Lockerman EL, Li C, Anderson J, Costa C, Liao D, Murphy E, DiDonato M, Bursulaya B, Lelais G, Barretina J, McNeill M, Epple R, Marsilje TH, Pathan N, Engelman JA, Michellys PY, McNamara P, Harris J, Bender S, Kasibhatla S. EGF816 Exerts Anticancer Effects in Non-Small Cell Lung Cancer by Irreversibly and Selectively Targeting Primary and Acquired Activating Mutations in the EGF Receptor. Cancer Res. 2016 Mar 15;76(6):1591-602. doi: 10.1158/0008-5472.CAN-15-2581. Epub 2016 Jan 29.

ipd_sharing_description:
Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

location_country:
Taiwan
Spain
Italy
Australia
Canada
Korea, Republic of
United States
France
Singapore
Norway
Germany

location_facility: Massachusetts General Hospital Mass General

summary: The purpose of this study is to determine the maximum tolerated dose (MTD) / recommended phase 2 dose (RP2D) of nazartinib (EGF816) in combination with capmatinib (INC280) and to estimate the preliminary anti-tumor activity of nazartinib in combination with capmatinib in participants with advanced non-small cell lung cancer (NSCLC) with documented EGFR mutation.


title: A Phase II, Multicenter, Open-label Study of EGF816 in Combination With Nivolumab in Adult Patients With EGFR Mutated Non-small Cell Lung Cancer and of INC280 in Combination With Nivolumab in Adult Patients With cMet Positive Non-small Cell Lung Cancer

summary: To determine the efficacy and safety of nivolumab in combination with EGF816 and of nivolumab in combination with INC280 in previously treated NSCLC patients

design_allocation: Non-Randomized

location_country:
Spain
Italy
Australia
Switzerland
United States
France
Singapore
Germany

location_facility: University of Texas MD Anderson Cancer Center Thoractic Head/Neck Med.Onc(2)


title: Testing the Effect of Intrauterine Slow Release Insemination (SRI) on the Pregnancy Rate in Women Designated for Standard Intrauterine Insemination (IUI)

acronym: EVIE

summary:
Overall Study Design and Plan Description

The study will be conducted on women with fertility difficulties who are designated for intrauterine insemination treatment (IUI). The research sample size is 137 treatment cycles per group (total of 274 treatment cycles). The women will be randomly divided into two groups. Some will undergo the standard bolus IUI treatment and others will be treated with the EVIE Slow Release Insemination method (SRI). Women who fail to conceive in the first treatment will next receive the alternative treatment. This means that a woman treated with the IUI method who does not become pregnant will then be treated with SRI, and vice versa (crossover method). The study will include women treated with Clomiphene Citrate or with other Gonadotropin-based treatment. Every couple treated will undergo at least 2 insemination cycles (unless pregnancy has already been achieved).
Before beginning the hormone treatment, each couple will have the trial procedure explained to them. This explanation will include all the detailed information and instruction about the IUI and the Slow Release Insemination methods. A Patient Information Sheet will be available to them.
Preparation of the spermatozoa for intra uterine insemination will be identical to the method performed currently before IUI.

Approximately two weeks after the insemination treatment, a blood sample will be taken from the woman to check for Beta hCG levels to indicate pregnancy.

Hypothesis:

SRI leads to higher pregnancy rates compared to standard IUI

Primary endpoint:

• Pregnancy rate Effect of controlled Intra-uterine slow release insemination on the pregnancy rate of women designated for intra uterine insemination in comparison to the accepted IUI method.

status: Unknown status

study_completion_date_type: Anticipated

enrollment_type: Anticipated

ReferenceType: background

ReferenceCitation: Muharib NS, Abdel Gadir A, Shaw RW. Slow release intrauterine insemination versus the bolus technique in the treatment of women with cervical mucus hostility. Hum Reprod. 1992 Feb;7(2):227-9.

lead_sponsor: Medical University of Vienna

location_country:
Austria
Germany
France
United Kingdom

location_facility: Dept. Obstetrics and Gynaecology, Medical University of Vienna

location_facility: Klinikum der Johann Wolfgang-Goethe-Universität Klinik für Frauenheilkunde und Geburtshilfe

location_facility: LMU, Klinikum der Universität München Klinik und Poliklinik für Frauenheilkunde und Geburtshilfe

location_facility: Dept. Obstetrics and Gynaekology, Medical Faculty of Aachen

location_facility: Frauenärztliche Gemeinschaftspraxis Dr. Maarten R. van Santen; KriegsstraBe 216

location_facility: Centre for Reproductive and Genetic Health - Eastman Dental Hospital

location_facility: Kinderwunschzentrum der Goldenes Kreuz Privatklinik

intervention_type: Other

lead_sponsor_type: academique


title: A Randomised Phase IIb Trial of Bevacizumab Added to Temozolomide ± Irinotecan for Children With Refractory/Relapsed Neuroblastoma - BEACON-Neuroblastoma Trial

acronym: BEACON

summary:
The purpose of this study is to investigate whether Bevacizumab (an anti-VEGF monoclonal antibody) added to a backbone chemotherapy regimen (Temozolomide, Irinotecan-Temozolomide or Topotecan-Temozolomide) demonstrates activity in children with relapsed or refractory neuroblastoma. Also, to investigate whether the addition of Irinotecan or Topotecan to Temozolomide increases the activity of chemotherapy.The primary objective of the study is the best response (Complete Response or Partial Response) while trial treatment, within 18 or 24 weeks depending on the arm of the trial the participant is randomised to. Secondary endpoints are assessing the side effects, the length of time before progression (Progression Free Survival) and overall survival (OS).

This trial will address two important questions:

does targeting blood vessel development using bevacizumab, (a monoclonal antibody against the Vascular Endothelial Growth Factor (VEGF)), add to the effect on a tumour when used with existing chemotherapy, compared to the effect of the existing chemotherapy alone (temozolomide)? NOTE- This question has been completed.
does the addition of a second chemotherapy drug (irinotecan or topotecan) increase the effect on a tumour compared to the effect of one alone (temozolomide) NOTE - This question has been completed.
does the addition of dinutuximab beta added to a backbone chemotherapy (temozolomide or temozolomide + topotecan) increase the effect of backbone alone.

Patients aged 1-21 years of age with relapsed or refractory high-risk neuroblastoma are randomised to one of two treatment arms: temozolomide-topotecan (TTo) or dinutuximab beta-temozolomide-topotecan (dBTTo). Temozolomide (T), irinotecan-temozolomide (IT), bevacizumab-T (BT), BIT (bevacizumab-IT), bevacizumab-temozolomide-topotecan (BTTo) and dinutuximab beta-temozolomide (dBT) are now closed to recruitment.

status: Active, not recruiting

lead_sponsor: University of Birmingham

location_country:
Denmark
Spain
Ireland
Austria
Italy
United Kingdom
Switzerland
France
Belgium
Netherlands

location_facility: St Anna Children's Hospital and CCRI/Studies and Statistics

location_facility: The Royal Marsden NHS Foundation Trust and Institute of Cancer Research

status_simplified: Ongoing


ipd_sharing: Undecided

title: A Randomized, Blinded, Placebo-controlled, Phase II Trial of LEE011 in Patients With Relapsed, Refractory, Incurable Teratoma With Recent Progression

location_country:
Netherlands
United States
Spain
France

location_facility: Memorial Sloan Kettering Oncology Department.

location_facility: USC Kenneth Norris Comprehensive Cancer Center Oncology Dept

summary:
This was a multi-center, randomized, double blind (investigator and subject), placebo controlled Phase II study to determine the efficacy and safety of treatment with ribociclib versus placebo in subjects with progressive relapsed, refractory incurable teratoma. Eligible subjects were randomized in a 2:1 ratio to ribociclib or placebo.

After discontinuation of study treatment, patients were followed up for safety, disease progression and overall survival.


title: Open-Label, Non Randomized Phase 2 Study With Safety Run-In Evaluating Efficacy and Safety of PQR309 in Patients With Relapsed or Refractory Lymphoma

summary: The main goal of this study is to determine the Maximum Tolerated Dose (MTD) and the Recommended Phase II Dose (RP2D) as well as preliminary antitumor activity of bimiralisib (PQR309) administered orally, as once daily capsules continuously and on intermittent schedule in patients with relapsed or refractory lymphomas.

lead_sponsor: PIQUR Therapeutics AG

location_country:
Bosnia and Herzegovina
Serbia
United Kingdom
United States
Israel
France

location_facility: Institute for Oncology and radiology of Serbia

location_facility: University Clinical Center Republic of Srpska


title: A Phase 2 Study to Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ/SAR402671 in Enzyme Replacement Therapy (ERT) Treatment-naïve Adult Male Patients Diagnosed With Fabry Disease

summary:
Primary Objective:

To assess the safety, pharmacokinetics (PK), pharmacodynamics (PD), and exploratory efficacy of GZ/SAR402671 in enzyme replacement therapy treatment-naïve adult male participants diagnosed with Fabry disease.

location_facility:
Investigational Site Number 643002
Investigational Site Number 840002
Investigational Site Number 840001
Investigational Site Number 826002
Investigational Site Number 826003
Investigational Site Number 840003
Investigational Site Number 250001
Investigational Site Number 616001


title: A Phase III Trial of Risk-stratified, Reduced Intensity Adjuvant Treatment in Patients Undergoing Transoral Surgery for Human Papillomavirus (HPV)-Positive Oropharyngeal Cancer

acronym: PATHOS

summary:
The main objectives of the PATHOS study are:

To assess whether swallowing function can be improved following transoral resection of HPV-positive OPSCC, by reducing the intensity of adjuvant treatment protocols. The aim is to personalise treatment, based on disease biology (HPV status and pathology findings), to optimise patient outcomes.

To demonstrate the non-inferiority of reducing the intensity of adjuvant treatment protocols in terms of overall survival in the reduced intensity treatment arms.

status: Recruiting

ReferenceCitation: Owadally W, Hurt C, Timmins H, Parsons E, Townsend S, Patterson J, Hutcheson K, Powell N, Beasley M, Palaniappan N, Robinson M, Jones TM, Evans M. PATHOS: a phase II/III trial of risk-stratified, reduced intensity adjuvant treatment in patients undergoing transoral surgery for Human papillomavirus (HPV) positive oropharyngeal cancer. BMC Cancer. 2015 Aug 27;15:602. doi: 10.1186/s12885-015-1598-x.

lead_sponsor: Lisette Nixon

location_country:
United Kingdom
Australia
United States
France
Germany

location_facility: Oxford University Hospitals NHS Foundation Trust

location_facility: City Hospitals Sunderland NHS Foundation Trust

location_facility: University Hospitals Bristol NHS Foundation Trust

location_facility: Cardiff and Vale University Local Health Board

location_facility: Derby Teaching Hospitals NHS Foundation Trust

location_facility: Central Manchester University Hospital NHS Foundation Trust

location_facility: Swansea Bay University Local Health Board

location_facility: Cambridge University Hospitals NHS Foundation Trust

location_facility: Guys and St Thomas's NHS Foundation Trust

location_facility: Royal United Hospitals Bath NHS Foundation Trust

location_facility: Newcastle upon Tyne Hospitals NHS Foundation Trust

location_facility: Royal Devon University Health Care NHS Foundation Trust

location_facility: University Hospitals Dorset NHS Foundation

location_facility: University College London Hospitals NHS Foundation Trust

location_facility: HPV Research Group Section of Pathology Cardiff University ,School of Medicine

intervention_type: Radiation


title: A Multi-center, Randomized, Double-blind, Placebo-controlled, Parallel Group, Repeated-dose Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacokinetics of Three Different Dosing Regimens of Inhaled Indacaterol Maleate in Patients With Persistent Asthma

location_country:
Jordan
United Kingdom
United States
France
Netherlands
Germany

location_facility:
Novartis Investigative Site
Novartis Investigator Site

summary: This study assessed the bronchodilator efficacy of three different regimens of indacaterol in patients with asthma


type: Other Grant/Funding Number

title: Canadian Study on the Endovascular Treatment of Unruptured Intracranial Aneurysms Versus Surgical Treatment. A Randomized Comparison of Clinical and Angiographic Results of Intracranial Aneurysms

acronym: CURES

summary:
Purpose:

Phase 1: (Pilot Phase)

To compare the treatment efficacy of surgical clipping and endovascular coiling for unruptured intracranial aneurysms.

To obtain better estimates of morbidity and mortality related to a surgical or endovascular treatment strategy at one year within the context of an RCT.

To show that an RCT comparing the morbidity and mortality of a surgical management strategy to an endovascular management strategy is feasible.

Phase 2:

To compare the results of surgical and endovascular management strategies, in terms of:

Overall mortality and morbidity at 1 and 5 years.
The clinical efficacy and safety of a surgical or endovascular management strategy at 1 and 5 years

Hypotheses: Phase 1 Hypotheses:

Surgical clipping of intradural, saccular, unruptured intracranial aneurysms is superior to endovascular management in terms of a lesser number of patients experiencing treatment failure.
An RCT comparing the clinical outcomes of a surgical versus endovascular management strategy is feasible.

Phase 1 Primary End-points:

• Treatment failure, hereby defined as having occurred when either: the intended initial modality (surgical or endovascular) fails to occlude the aneurysm, a "major" (saccular) angiographic aneurysm recurrence is found, or an intracranial hemorrhagic event occurs during the 1-year follow-up period.

Phase 1 Secondary End-points:

Overall morbidity and mortality at one year.
Occurrence of morbidity (mRS >2) or mortality following treatment.
Occurrence of failure of aneurysm occlusion using the initial intended treatment modality.
Occurrence of a "major" (saccular) angiographic aneurysm recurrence.
Occurrence of an intracranial hemorrhage following treatment.
Peri-treatment hospitalization lasting more than 5 days
Discharge following treatment to a location other than home

Treatment:

Trial feasibility, or the capacity for patient recruitment, would require enrollment of at least 8 patients per actively recruiting center per year.

Phase 2 Hypotheses:

It may be too early to explicitly define the primary hypothesis of Phase 2, however, the intent of Phase 2 can be expressed as:

One management strategy is superior to the other in terms of clinical outcome at five years.
One management strategy is superior to the other in terms of clinical efficacy at five years.

study_type: Observational

time_perspective: Prospective

ReferenceCitation: Naggara O, Darsaut T, Trystram D, Tselikas L, Raymond J. Unruptured intracranial aneurysms: why we must not perpetuate the impasse for another 25 years. Lancet Neurol. 2014 Jun;13(6):537-8. doi: 10.1016/S1474-4422(14)70091-2.

lead_sponsor: Centre hospitalier de l'Université de Montréal (CHUM)

location_country: Canada France Belgium

location_facility: Centre hospitalier de l'Université de Montréal - CHUM

location_facility: Centre hospitalier universitaire de Liège

intervention_type: Procedure


title: Long-term Follow-up of a Multicentre, Randomized, Double-blind, Placebo-controlled Trial (AN004T) Assessing the Efficacy and Tolerability of 2 Dosing Regimens of AllerT in Adult Subjects Allergic to Birch Pollen

summary: Study aiming to evaluate the efficacy of a 2-month pre-seasonal treatment with an AllerT 50 µg or 100 µg maintenance dose administered in previous study AN004T in reducing symptoms of allergic rhinoconjunctivitis during the 2nd following birch pollen season.

lead_sponsor: Anergis

location_country: Denmark Poland Latvia Lithuania Switzerland France Sweden

location_facility: Viktorija Vevere private practice of Allergology

location_facility: Centre Hospitalier Universitaire Vaudois

location_facility: Antakalnio affiliation of the Vilnius City Allergy Center

location_facility: Silesian Piasts University of Medicine in Wrocław

location_facility: SPZOZ Uniwersytecki Szpital Kliniczny - Allergology

location_facility: Center of examination and treatment of allergic diseases


title: Natural History and Outcomes in X-Linked Hypohidrotic Ectodermal Dysplasia

summary: The proposed natural history study will enroll male and female patients, ages 36 months and younger, who have a diagnosis of XLHED based on genetic testing and who have not received an investigational study drug. The study protocol will include collection of all relevant medical history and documentation of clinical outcomes using age-appropriate, minimally invasive technologies. Data will be collected both retrospectively, back to pregnancy assessments that may be available, and prospectively through age 5 yrs.

time_perspective: Other

lead_sponsor: Edimer Pharmaceuticals

location_facility: Azienda Ospedaliera-Polo Universitario "Luigi Sacco"

location_facility: Washington University School of Medicine


title: A Phase Ib/II Study of LEE011 in Combination With Fulvestrant and BYL719 or BKM120 in the Treatment of Postmenopausal Women With Hormone Receptor Positive, HER2 Negative Locally Recurrent or Advanced Metastatic Breast Cancer

summary:
The purpose of this trial is to explore the clinical utility of the three investigational agents in HR+, HER2- breast cancer. LEE011 (CDK4/6 inhibitor), BKM120 (PI3K-pan class I-inhibitor) and BYL719 (PI3K-alpha specific class I inhibitor) in combination with fulvestrant.

This is a multi-center, open-label Phase Ib/II study. The Phase Ib portion of the study is a dose escalation to estimate the MTD and/or RP2D for three regimens: LEE011 with fulvestrant; LEE011 and BKM120 with fulvestrant; LEE011and BYL719 with fulvestrant.

The Phase II portion of the study was planned to be a randomized study to assess the anti-tumor activity as well as safety and tolerability of LEE011 with fulvestrant to LEE011 and BKM120 with fulvestrant, and LEE011 and BYL719 with fulvestrant in patients with ER+/HER2- locally advanced or metastatic breast cancer.

Approximately 216 adult women with ER+/HER2- locally advanced or metastatic breast cancer were planned to be enrolled.

ReferenceCitation: Tolaney SM, Im YH, Calvo E, Lu YS, Hamilton E, Forero-Torres A, Bachelot T, Maur M, Fasolo A, Tiedt R, Nardi L, Stammberger U, Abdelhady AM, Ruan S, Lee SC. Phase Ib Study of Ribociclib plus Fulvestrant and Ribociclib plus Fulvestrant plus PI3K Inhibitor (Alpelisib or Buparlisib) for HR(+) Advanced Breast Cancer. Clin Cancer Res. 2021 Jan 15;27(2):418-428. doi: 10.1158/1078-0432.CCR-20-0645. Epub 2020 Sep 4.

location_country:
Taiwan
Spain
Italy
United Kingdom
Korea, Republic of
United States
France
Singapore

location_facility: Sarah Cannon Research Institute Onc Dept

location_facility: University of Alabama at Birmingham/ Kirklin Clinic Dept Onc


title: A 6-Month, Open-Label, Flexible-Dosage (150 to 200 mg/Day) Extension Study of the Safety and Efficacy of Armodafinil Treatment as Adjunctive Therapy in Adults With Major Depression Associated With Bipolar I Disorder

lead_sponsor: Cephalon

location_country:
Croatia
Poland
Brazil
Serbia
Spain
Italy
Australia
Argentina
Canada
Ukraine
Hungary
United States
France
South Africa
Germany
Bulgaria
Slovakia

location_facility:
Teva Investigational Site 292
Teva Investigational Site 106
Teva Investigational Site 135
Teva Investigational Site 282
Teva Investigational Site 884
Teva Investigational Site 698
Teva Investigational Site 207
Teva Investigational Site 688
Teva Investigational Site 661
Teva Investigational Site 184
Teva Investigational Site 117
Teva Investigational Site 832
Teva Investigational Site 834
Teva Investigational Site 403
Teva Investigational Site 634
Teva Investigational Site 105
Teva Investigational Site 624
Teva Investigational Site 258
Teva Investigational Site 603
Teva Investigational Site 872
Teva Investigational Site 600
Teva Investigational Site 238
Teva Investigational Site 229
Teva Investigational Site 609
Teva Investigational Site 100
Teva Investigational Site 689
Teva Investigational Site 286
Teva Investigational Site 228
Teva Investigational Site 852
Teva Investigational Site 134
Teva Investigational Site 177
Teva Investigational Site 280
Teva Investigational Site 853
Teva Investigational Site 290
Teva Investigational Site 237
Teva Investigational Site 182
Teva Investigational Site 190
Teva Investigational Site 116
Teva Investigational Site 616
Teva Investigational Site 115
Teva Investigational Site 156
Teva Investigational Site 122
Teva Investigational Site 875
Teva Investigational Site 336
Teva Investigational Site 223
Teva Investigational Site 299
Teva Investigational Site 697
Teva Investigational Site 706
Teva Investigational Site 687
Teva Investigational Site 833
Teva Investigational Site 608
Teva Investigational Site 635
Teva Investigational Site 612
Teva Investigational Site 225
Teva Investigational Site 141
Teva Investigational Site 411
Teva Investigational Site 107
Teva Investigational Site 200
Teva Investigational Site 404
Teva Investigational Site 193
Teva Investigational Site 148
Teva Investigational Site 633
Teva Investigational Site 281
Teva Investigational Site 655
Teva Investigational Site 664
Teva Investigational Site 128
Teva Investigational Site 118
Teva Investigational Site 666
Teva Investigational Site 119
Teva Investigational Site 155
Teva Investigational Site 303
Teva Investigational Site 300
Teva Investigational Site 129
Teva Investigational Site 195
Teva Investigational Site 136
Teva Investigational Site 712
Teva Investigational Site 196
Teva Investigational Site 831
Teva Investigational Site 430
Teva Investigational Site 255
Teva Investigational Site 699
Teva Investigational Site 235
Teva Investigational Site 295
Teva Investigational Site 871
Teva Investigational Site 181
Teva Investigational Site 610
Teva Investigational Site 462
Teva Investigational Site 147
Teva Investigational Site 153
Teva Investigational Site 110
Teva Investigational Site 837
Teva Investigational Site 606
Teva Investigational Site 433
Teva Investigational Site 248
Teva Investigational Site 202
Teva Investigational Site 146
Teva Investigational Site 236
Teva Investigational Site 204
Teva Investigational Site 400
Teva Investigational Site 201
Teva Investigational Site 212
Teva Investigational Site 855
Teva Investigational Site 145
Teva Investigational Site 213
Teva Investigational Site 371
Teva Investigational Site 180
Teva Investigational Site 138
Teva Investigational Site 217
Teva Investigational Site 409
Teva Investigational Site 861
Teva Investigational Site 709
Teva Investigational Site 183
Teva Investigational Site 247
Teva Investigational Site 131
Teva Investigational Site 157
Teva Investigational Site 710
Teva Investigational Site 408
Teva Investigational Site 301
Teva Investigational Site 175
Teva Investigational Site 249
Teva Investigational Site 662
Teva Investigational Site 111
Teva Investigational Site 104
Teva Investigational Site 192
Teva Investigational Site 296
Teva Investigational Site 224
Teva Investigational Site 132
Teva Investigational Site 881
Teva Investigational Site 133
Teva Investigational Site 219
Teva Investigational Site 873
Teva Investigational Site 198
Teva Investigational Site 259
Teva Investigational Site 176
Teva Investigational Site 696
Teva Investigational Site 692
Teva Investigational Site 113
Teva Investigational Site 121
Teva Investigational Site 240
Teva Investigational Site 103
Teva Investigational Site 406
Teva Investigational Site 605
Teva Investigational Site 450
Teva Investigational Site 370
Teva Investigational Site 127
Teva Investigational Site 256
Teva Investigational Site 401
Teva Investigational Site 102
Teva Investigational Site 613
Teva Investigational Site 711
Teva Investigational Site 245
Teva Investigational Site 708
Teva Investigational Site 854
Teva Investigational Site 434
Teva Investigational Site 651
Teva Investigational Site 851
Teva Investigational Site 205
Teva Investigational Site 257

summary: The primary objective of this study is to evaluate the safety and tolerability of long term (6 months) armodafinil treatment as adjunctive therapy to mood-stabilizing medications in adults with bipolar I disorder.


acronym: PERSIST

title: An Open-Label, Rollover Study to Evaluate the Long Term Safety and Efficacy of VX-770 in Subjects With Cystic Fibrosis

lead_sponsor: Vertex Pharmaceuticals Incorporated

ReferenceCitation: McKone EF, Borowitz D, Drevinek P, Griese M, Konstan MW, Wainwright C, Ratjen F, Sermet-Gaudelus I, Plant B, Munck A, Jiang Y, Gilmartin G, Davies JC; VX08-770-105 (PERSIST) Study Group. Long-term safety and efficacy of ivacaftor in patients with cystic fibrosis who have the Gly551Asp-CFTR mutation: a phase 3, open-label extension study (PERSIST). Lancet Respir Med. 2014 Nov;2(11):902-910. doi: 10.1016/S2213-2600(14)70218-8. Epub 2014 Oct 9.

location_country:
United Kingdom
Ireland
Australia
Canada
Czech Republic
United States
France
Germany

summary: The primary objective of the study was to evaluate the safety of long-term VX-770 treatment in participants with cystic fibrosis (CF). The secondary objective of the study was to evaluate the efficacy of long-term VX-770 treatment in subjects with CF.


acronym: ACES 263

title: A Multicenter, Randomized, Double-blind Study to Evaluate the Efficacy, Safety and Tolerability of an Oral Aripiprazole/Escitalopram Combination Therapy in Patients With Major Depressive Disorder

lead_sponsor: Otsuka Pharmaceutical Development & Commercialization, Inc.

ipd_sharing_description: Anonymized Individual participant data (IPD) that underlie the results of this study will be shared with researchers to achieve aims pre-specified in a methodologically sound research proposal. Small studies with less than 25 participants are excluded from data sharing.

location_country:
Croatia
Poland
Spain
Canada
Sweden
Hungary
United States
France
India
South Africa
Malaysia

location_facility:
Study Site 2
Study Site 1

summary: This will be a multicenter, randomized, double-blind study designed to assess the efficacy, safety and tolerability of an oral Aripiprazole/Escitalopram combination therapy in participants with MDD who have demonstrated an incomplete response to a prospective trial of Escitalopram, and report a treatment history for the current MDD episode of an inadequate response to at least one and no more than three adequate trials of an approved antidepressant other than Escitalopram. An inadequate response is defined as less than a 50% reduction in depressive symptom severity as assessed by the participant's self-report on the Massachusetts General Hospital Antidepressant Treatment Response Questionnaire (ATRQ) and evaluated by the investigator as part of the participant's medical and psychiatric history. An adequate trial is defined as an antidepressant treatment for at least 6 weeks duration (or at least 3 weeks for combination treatments) at an approved dose as specified in the ATRQ.


primary_purpose: Basic Science

acronym: SHRINK

title: A Study to Evaluate the Overall Effect of Solifenacin 5mg and 10mg on Bladder Wall Thickness and Urinary Nerve Growth Factor in Female Subjects With Overactive Bladder and a Diagnosis of Detrusor Overactivity - A Double-blind, Randomised, Placebo-controlled, Parallel Group, Multi-centre Study.

lead_sponsor: Astellas Pharma Inc

ipd_sharing_description: Details of the IPD sharing plan for this study can be found at www.clinicalstudydatarequest.com.

location_country:
Poland
Romania
Spain
Italy
Austria
Russian Federation
United Kingdom
Canada
Czech Republic
Sweden
Hungary
United States
France
Israel
Turkey
Belgium
Norway
Germany
Bulgaria
Slovakia

summary: The purpose is to see if solifenacin has any effect on bladder wall thickness and urine inflammatory marker measurements after 12 weeks of treatment.


type: Registry Identifier

title: A One Year Open Label, Non-comparative Extension to a Randomized, Multicenter, Phase II Study to Evaluate the Safety, Tolerability, Pharmacokinetics (PK) and Effects on Liver Iron Concentration (LIC) of Repeated Doses of 5-30mg/kg/Day ICL670 Relative to Deferoxamine (DFO) in Sickle Cell Disease (SCD) Patients With Transfusional Hemosideresis (THS) [Amendment 3: Extension Prolonged to 4-years]

summary: The safety, tolerability, effects on liver iron concentration and pharmacokinetics of ICL670 is studied in sickle cell disease patients with transfusional hemosiderosis.

location_country:
United Kingdom
Italy
Canada
United States
France

location_facility: University of Colorado Health Science Center

location_facility: Children's Hospital & Research Center at Oakland

location_facility: Children's Hospital of the King's Daughters

location_facility: University of South Alabama College of Medicine

location_facility: Grady Hospital, Georgia Comprehensive Sickle Cell Center


title: A Phase II, Multicenter, Randomized, Controlled, Open-label Study of the Safety, Efficacy and Pharmacokinetics of ABT-263 in Combination With Dose-intensive Rituximab, or Dose-intensive Rituximab Alone, in Previously Untreated Patients With B-Cell, Chronic Lymphocytic Leukemia (CLL)

summary: This Phase II, randomized, open-label, international, multicenter trial is designed to evaluate the safety and efficacy of rituximab monotherapy when given according to a dose intense regimen and to assess the safety, efficacy, and pharmacokinetics of ABT-263 when combined with dose-intense rituximab in previously untreated patients with B-cell CLL.

lead_sponsor: Genentech, Inc.

location_country:
Poland
Brazil
United Kingdom
Italy
Russian Federation
Australia
Czech Republic
Ukraine
Puerto Rico
United States
France
Israel


title: A Phase 2a, Multicenter, Randomized, Double-Blind, Placebo-Controlled, Study to Evaluate the Safety, Tolerability and Clinical Effect of Laquinimod in Active Lupus Nephritis Patients, in Combination With Standard of Care (Mycophenolate Mofetil and Steroids)

lead_sponsor: Teva Branded Pharmaceutical Products R&D, Inc.

location_country:
United Kingdom
Russian Federation
Canada
United States
France

location_facility:
Teva Investigational Site 1114
Teva Investigational Site 3509
Teva Investigational Site 5003
Teva Investigational Site 1113
Teva Investigational Site 1028
Teva Investigational Site 3410
Teva Investigational Site 1030
Teva Investigational Site 1024
Teva Investigational Site 5005
Teva Investigational Site 5007
Teva Investigational Site 5002
Teva Investigational Site 1115
Teva Investigational Site 1025
Teva Investigational Site 1026
Teva Investigational Site 1021
Teva Investigational Site 1032
Teva Investigational Site 1018
Teva Investigational Site 1020
Teva Investigational Site 3510
Teva Investigational Site 5006
Teva Investigational Site 3412
Teva Investigational Site 1016
Teva Investigational Site 1019
Teva Investigational Site 5001
Teva Investigational Site 1031
Teva Investigational Site 3413
Teva Investigational Site 3409
Teva Investigational Site 3411
Teva Investigational Site 1017
Teva Investigational Site 1022

summary: The study aims to evaluate the safety and clinical effect of daily oral treatment with laquinimod capsules in active lupus nephritis participants. This study will assess Laquinimod doses of 0.5 milligrams (mg)/day and 1 mg/day in combination with standard of care treatment (mycophenolate mofetil [MMF] and corticosteroids). Laquinimod is a novel immunomodulating drug which is currently in advanced stages of development by Teva Pharmaceuticals Ltd. for Multiple Sclerosis.


title: A Phase 1/2 Dose-Escalation Study of XL147 (SAR245408) or XL765 (SAR245409) in Combination With Letrozole in Subjects With Hormone Receptor-Positive and HER2-Negative Breast Cancer Refractory to a Nonsteroidal Aromatase Inhibitor

summary:
Phase 1 of this study will evaluate the maximum tolerated dose (MTD) of XL147 when given in combination with letrozole (Femara) and of XL765 when given in combination with letrozole. After the MTD is established for each combination (Phase 2), subjects will be enrolled to evaluate the preliminary efficacy and safety of these combinations in subjects with breast cancer refractory to a non-steroidal aromatase inhibitor that is ER+/PGR+ and HER2-. Letrozole is used in the treatment of different types of breast cancer, but patients can develop resistance.

Upregulation of PI3K activity is one of the most common characteristics of human cancer cells, including breast tumor cells. Activation of PI3K results in stimulation of AKT and mTOR kinases, resulting in the promotion of tumor cell proliferation and survival. Preclinical and retrospective clinical data suggest that aberrant activation of the PI3K pathway may play a role in aromatase inhibitor resistance in patients with ER+, HER2- breast cancer. XL147 is a potent inhibitor of PI3K, and XL765 is a potent dual inhibitor of PI3K and mTOR; therefore either of these compounds in combination with letrozole warrants clinical investigation.

lead_sponsor: Sanofi

location_country:
United States
Spain
France

location_facility:
Investigational Site Number 1601
Investigational Site Number 1214
Investigational Site Number 3321
Investigational Site Number 3419
Investigational Site Number 1331
Investigational Site Number 5246
Investigational Site Number 1537
Investigational Site Number 3324
Investigational Site Number 3413
Investigational Site Number 3420
Investigational Site Number 1238
Investigational Site Number 1330
Investigational Site Number 1138
Investigational Site Number 1252
Investigational Site Number 1441
Investigational Site Number 3415
Investigational Site Number 5201


title: Medtronic Adapta/Versa/Sensia Long Term Reliability Study

summary: The primary purpose of this study is to assess the long term reliability of the Medtronic Adapta®/Sensia™/Versa™ platform of devices. This study is required by FDA as a condition of approval of nEw3 devices. Patients will be followed for 5 years after implant. This study utilizes data collected from the System Longevity Study (SLS).

lead_sponsor: Medtronic

location_country:
Denmark
Serbia
Italy
Austria
Netherlands
Canada
Switzerland
Sweden
United States
France
Belgium
Germany


title: A Phase 3, Randomized, Multi-Center, Multi-National, Double-Blind Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Once Daily Versus Twice Daily Dosing of Genz-112638 in Patients With Gaucher Disease Type 1 Who Have Demonstrated Clinical Stability on a Twice Daily Dose of Genz-112638

summary: The primary objective of this study was to evaluate the efficacy and safety of once daily (QD) versus twice daily (BID) dosing of eliglustat tartrate (Genz-112638) in participants with Gaucher disease type 1 who had demonstrated clinical stability on BID dosing of eliglustat tartrate (Genz-112638). The secondary objective was to evaluate the pharmacokinetics (PK) of Genz-99067 when eliglustat tartrate (Genz-112638) was administered QD and BID in participants with Gaucher disease type 1 who had demonstrated clinical stability on BID dosing of eliglustat tartrate (Genz-112638).

ReferenceCitation: McEachern KA, Fung J, Komarnitsky S, Siegel CS, Chuang WL, Hutto E, Shayman JA, Grabowski GA, Aerts JM, Cheng SH, Copeland DP, Marshall J. A specific and potent inhibitor of glucosylceramide synthase for substrate inhibition therapy of Gaucher disease. Mol Genet Metab. 2007 Jul;91(3):259-67. Epub 2007 May 16.

ReferenceCitation: Lukina E, Watman N, Arreguin EA, Banikazemi M, Dragosky M, Iastrebner M, Rosenbaum H, Phillips M, Pastores GM, Rosenthal DI, Kaper M, Singh T, Puga AC, Bonate PL, Peterschmitt MJ. A phase 2 study of eliglustat tartrate (Genz-112638), an oral substrate reduction therapy for Gaucher disease type 1. Blood. 2010 Aug 12;116(6):893-9. doi: 10.1182/blood-2010-03-273151. Epub 2010 May 3.

ReferenceCitation: Lukina E, Watman N, Arreguin EA, Dragosky M, Iastrebner M, Rosenbaum H, Phillips M, Pastores GM, Kamath RS, Rosenthal DI, Kaper M, Singh T, Puga AC, Peterschmitt MJ. Improvement in hematological, visceral, and skeletal manifestations of Gaucher disease type 1 with oral eliglustat tartrate (Genz-112638) treatment: 2-year results of a phase 2 study. Blood. 2010 Nov 18;116(20):4095-8. doi: 10.1182/blood-2010-06-293902. Epub 2010 Aug 16. Erratum in: Blood. 2011 May 19;117(20):5551.

ReferenceCitation: Charrow J, Fraga C, Gu X, Ida H, Longo N, Lukina E, Nonino A, Gaemers SJM, Jouvin MH, Li J, Wu Y, Xue Y, Peterschmitt MJ. Once- versus twice-daily dosing of eliglustat in adults with Gaucher disease type 1: The Phase 3, randomized, double-blind EDGE trial. Mol Genet Metab. 2018 Mar;123(3):347-356. doi: 10.1016/j.ymgme.2017.12.001. Epub 2018 Jan 4.

lead_sponsor: Genzyme, a Sanofi Company

location_country:
Croatia
Brazil
Portugal
Serbia
Greece
Austria
Australia
Russian Federation
Canada
Sweden
United States
France
China
India
Romania
Japan
Netherlands

location_facility: Hospital Universitario Walter Cantidio - HUWC

location_facility: Hospital de Clínicas da Universidade Federal do Parana

location_facility: Shanghai Xinhua Hospital Shanghai Xinhua Hospital

location_facility: IGEIM - Institute of Genetic and Inborn Erros of Metabolism

location_facility: University of California, San Diego Medical Center

location_facility: Hôpital Femme Mère Enfant Centre de référence des maladies Héréditaires du métabolisme

location_facility: General Hospital of Athens "G. Gennimatas"

location_facility: Hemocentro de Ribeirão Preto Núcleo de Hemoterapia de Franca

location_facility: St. Petersburg State Medical Pavlov University

location_facility: Hematology Research Center of Russian Academy of Medical Sciences

location_facility: Instituto Tropical de Medicina Reprodutiva e Menopausa - INTRO

location_facility: Cettro - Centro de Tratamento de Oncologia e Hematologia

location_facility: State Medical and Prophylactic Healthcare Institution; Chelyabinsk Regional Clinical Hospital


title: A Multicenter, Multinational, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Aerosolized ALN-RSV01 Plus Standard of Care in Lung Transplant Patients Infected With Respiratory Syncytial Virus (RSV)

summary: To evaluate the efficacy and safety of ALN-RSV01 plus standard of care in RSV infected lung transplant patients

lead_sponsor: Alnylam Pharmaceuticals

location_country:
Austria
Australia
Canada
United States
France
Germany


title: Multicenter Phase 2 Trial of ARQ 197 for Subjects With Relapsed or Refractory Germ Cell Tumors

summary: This is a multicenter, single-arm study for safety and efficacy.

lead_sponsor: Daiichi Sankyo, Inc.

location_country:
United States
United Kingdom
France


title: A Phase II, Open-label, 1:1 Randomized, Controlled Trial Exploring the Efficacy of EMD 1201081 in Combination With Cetuximab in Second-Line Cetuximab-Naïve Subjects With Recurrent or Metastatic Squamous Cell Carcinoma of the Head and Neck (R/M SCCHN)

summary:
The purpose of this study is to determine if EMD 1201081 in combination with cetuximab is more efficient than cetuximab alone to control the cancer.

EMD 1201081 is an immune modulatory oligonucleotide (IMO) containing phosphorothioate oligodeoxynucleotide and acts as an agonist of Toll-like receptor 9 (TLR9).

EMD 1201081 has been studied in six clinical trials in over 170 subjects either as a monotherapy or in combination with chemotherapeutic agents or targeted therapies. Two studies have been conducted in healthy volunteers. In the other five studies, subjects with advanced solid tumors, renal cell carcinoma, non-small cell lung cancer and colorectal cancer have been treated with EMD 1201081. Two studies are still ongoing. Future clinical development of EMD 1201081 will focus on colorectal cancer (CRC) and squamous cell cancer of the head and neck (SCCHN).

In this Phase 2 study, subjects with recurrent or metastatic squamous cell cancer of the head and neck (R/M SCCHN), will be treated with cetuximab plus EMD 1201081 or cetuximab alone. The study will be conducted as a multicenter study in several European Union (EU) member states and the Unites States.

EMD 1201081 in combination with cetuximab will be evaluated for antitumor activity in subjects by examining its effects on accepted clinical endpoints. Progression-free survival (PFS) will be evaluated in subjects treated with EMD 1201081 plus cetuximab compared to cetuximab alone in cetuximab-naïve subjects with R/M SCCHN who have progressed on a cytotoxic therapy.

Cetuximab, approved in colorectal cancer and SCCHN in combination with platinum-based chemotherapy and SCCHN in combination with radiotherapy in the EU, will be provided as investigational medicinal product (IMP) in this study. Commercially available Cetuximab will be provided in the United States.

ReferenceType: result

ReferenceCitation: Ruzsa A, Sen M, Evans M, Lee LW, Hideghety K, Rottey S, Klimak P, Holeckova P, Fayette J, Csoszi T, Erfan J, Forssmann U, Goddemeier T, Bexon A, Nutting C; NA EMD 1201081 Study Group. Phase 2, open-label, 1:1 randomized controlled trial exploring the efficacy of EMD 1201081 in combination with cetuximab in second-line cetuximab-naïve patients with recurrent or metastatic squamous cell carcinoma of the head and neck (R/M SCCHN). Invest New Drugs. 2014 Dec;32(6):1278-84. doi: 10.1007/s10637-014-0117-2. Epub 2014 Jun 4.

lead_sponsor: EMD Serono

location_country:
Poland
United Kingdom
Czech Republic
Hungary
France
United States
Belgium
Slovakia

location_facility: Zala Megyei Kohaz Kulsokorhaz Onkologia Osztaly

location_facility: University of Kentucky, Markey Cancer Center

location_facility: Zaklad Opleki Zdrowotnej MSWIA z Warminsko-Mazurskim Centrum Onkologil, Oddziat Chemioterapli

location_facility: Jasz-Nagykun-Szolnok Megyei Hetenyi Geza Korhaz-Rendelointezet

location_facility: Szegedi Tudomayegyetem Altalanos Orvostudomanyi Kar Onkoterapias Klinika

location_facility: Southampton University Hospitals NHS Trust

location_facility: SPZOZ Centrum Onkologi Liemi Lubelskiej, II Odzial Radioterapiii z pododdzialem Chemioterpii

location_facility: Centrum Onkologi - Instytut im. Marii Sklodowskiej-Curie, Klinika Nowotworow Glowy i Szyi (NCI)

location_facility: Ustav radiacni onkologie Fakultni nemacnice Na Bulovce


acronym: RESTORE

title: The Efficacy and Safety of Erdosteine in the Long-term Therapy of Chronic Obstructive Pulmonary Disease (COPD). A 12-month, Randomised, Double-blind, Placebo-controlled, Parallel Group, Multicenter Study

lead_sponsor: Edmond Pharma

ReferenceCitation: Dal Negro RW, Wedzicha JA, Iversen M, Fontana G, Page C, Cicero AF, Pozzi E, Calverley PMA; RESTORE group; RESTORE study. Effect of erdosteine on the rate and duration of COPD exacerbations: the RESTORE study. Eur Respir J. 2017 Oct 12;50(4). pii: 1700711. doi: 10.1183/13993003.00711-2017. Print 2017 Oct.

location_country:
Denmark
Poland
Romania
United Kingdom
Italy
Czech Republic
France
Belgium
Bulgaria
Slovakia

location_facility: Department of Respiratory Diseases, Copenhagen University Hospital

location_facility: Regional Respiratory Centre, Belfast City Hospital

location_facility: Clinic for emergency Medicine and Intensive care, Pneumology and Physiatry

location_facility: Cardiovascular and Respiratory Studies Department

location_facility: Katedra i Klinika Pneumonologii, Warszawski Uniwersytet Medyczny

location_facility: UOC di Pneumologia- Ospedale Civile di Massa e Carrara

location_facility: Campus Gasthuisberg Dep.t Longfunctiemetingen

location_facility: Spitalul de Pneumoftiziologie "Leon Daniello"

location_facility: Clinic of Pulmonary Disease-Military Medical Academy

location_facility: Katedra Pulmunologii i Alergologii, Klinika Gruźlicy Uniwersytetu Medycznego

location_facility: Ospedale San Carlo Borromeo, Dipartimento Broncocardiopneumologico

location_facility: Spitalul de Pneumoftiziologie "Victor Babes"

location_facility: Aintree Chest Centre, University Hospital Aintree

location_facility: UOC di Pneumologia-Ospedale Mater Salutis

location_facility: Katedra i klinika Pulmonologii, Uniwersytet Medyczny

location_facility: Nemocnica s Poliklinicou "Svateho Jakuba"n.o.

location_facility: Specializovana NemocnicaSvorada Zobor n.o.

location_facility: Ospedale Careggi, Malattie dell'Apparato Respiratorio

location_facility: Service de Pneumologie, Hopital Bois-Guillaume

location_facility: Klinika Pulmonologii II Katedry, Collegium Medicum Uniwersytetu

location_facility: Institutul de Pneumoftiziologie "Marius Nasta"

location_facility: Department of Respiratory Medicine, Faculty Hospital

location_facility: Clinica Pneumologie I, Spitalul Clinic Judetean Mureş

summary: The aim of the study is to evaluate the effect of erdosteine, compared to placebo, on exacerbation rate over a 12-month treatment period in patients with moderate-to-severe COPD. Moreover, the effects of erdosteine on pulmonary function parameters, clinical symptoms and quality of life, and the long-term safety of the drug will be assessed.


title: A Phase 3, Randomized, Controlled, Multi-Center, Open-Label Study to Compare Tivozanib (AV-951) to Sorafenib in Subjects With Advanced Renal Cell Carcinoma (TIVO-1)

summary: This is an open-label, randomized, controlled, multi-national, multi-center, parallel-arm trial comparing tivozanib to sorafenib in subjects with advanced RCC. The study is designed to compare the PFS, OS, ORR, DR, safety and tolerability, and kidney specific symptoms/health outcome measurements of tivozanib and sorafenib.

lead_sponsor: AVEO Pharmaceuticals, Inc.

location_country:
Poland
Serbia
United Kingdom
Italy
Russian Federation
Argentina
Canada
Ukraine
Hungary
United States
Chile
France
India
Romania
Bulgaria
Czechia

location_facility:
Site 498
Site 444
Site 431
Site 185
Site 156
Site 402
Site 454
Site 450
Site 466
Site 463
Site 190
Site 493
Site 403
Site 191
Site 495
Site 467
Site 160
Site 400
Site 440
Site 155
Site 424
Site 465
Site 182
Site 494
Site 161
Site 443
Site 434
Site 157
Site 481
Site 162
Site 433
Site 401
Site 121
Site 152
Site 457
Site 435
Site 154
Site 490
Site 468
Site 158
Site 480
Site 133
Site 187
Site 421
Site 102
Site 484
Site 180
Site 496
Site 172
Site 110
Site 123
Site 404
Site 491
Site 459
Site 492
Site 150
Site 442
Site 456
Site 430
Site 483
Site 170
Site 186
Site 423
Site 455
Site 460
Site 432
Site 159
Site 452
Site 441
Site 464
Site 462
Site 422
Site 497
Site 461
Site 436
Site 151
Site 451
Site 411
Site 184
Site 122
Site 153
Site 130
Site 482
Site 453
Site 173
Site 458


acronym: B-Long

title: B-LONG: An Open-Label, Multicenter Evaluation of the Safety, Pharmacokinetics, and Efficacy of Recombinant, Long-acting Coagulation Factor IX Fc Fusion Protein (rFIXFc) in the Prevention and Treatment of Bleeding in Previously Treated Subjects With Severe Hemophilia B

lead_sponsor: Bioverativ Therapeutics Inc.

ReferenceCitation: Powell JS, Pasi KJ, Ragni MV, Ozelo MC, Valentino LA, Mahlangu JN, Josephson NC, Perry D, Manco-Johnson MJ, Apte S, Baker RI, Chan GC, Novitzky N, Wong RS, Krassova S, Allen G, Jiang H, Innes A, Li S, Cristiano LM, Goyal J, Sommer JM, Dumont JA, Nugent K, Vigliani G, Brennan A, Luk A, Pierce GF; B-LONG Investigators. Phase 3 study of recombinant factor IX Fc fusion protein in hemophilia B. N Engl J Med. 2013 Dec 12;369(24):2313-23. doi: 10.1056/NEJMoa1305074. Epub 2013 Dec 4.

location_country:
Poland
Brazil
Hong Kong
United Kingdom
Italy
Russian Federation
Australia
Canada
Sweden
United States
France
China
India
Belgium
Japan
Germany
South Africa

location_facility:
Ressearch Site
Research site
Research Site

summary: The primary objectives of the study were: to evaluate the safety and tolerability of rFIXFc; to evaluate the efficacy of rFIXFc in all treatment arms; to evaluate the effectiveness of prophylaxis over on-demand (episodic) therapy by comparing the annualized number of bleeding episodes between participants receiving rFIXFc on each prevention (prophylaxis) regimen and participants receiving rFIXFc on an episodic regimen. The secondary objectives of the study were: to evaluate and assess the pharmacokinetic (PK) parameter estimates of rFIXFc and rFIX (BeneFIX®) at baseline in the Sequential PK subgroup as well as rFIXFc at Week 26 (±1 week); to evaluate participants' response to treatment; to evaluate rFIXFc consumption.


acronym: Safety and efficacy of positron emission tomography imaging with BAY 94-9172 (ZK 6013443)

title: An Open-label, Non-randomized Study to Evaluate the Efficacy and Safety of BAY94-9172 (ZK 6013443) Positron Emission Tomography (PET) Imaging for Detection/Exclusion of Cerebral Beta-amyloid When Compared to Postmortem Histopathology

lead_sponsor: Life Molecular Imaging SA

ReferenceCitation: Seibyl J, Catafau AM, Barthel H, Ishii K, Rowe CC, Leverenz JB, Ghetti B, Ironside JW, Takao M, Akatsu H, Murayama S, Bullich S, Mueller A, Koglin N, Schulz-Schaeffer WJ, Hoffmann A, Sabbagh MN, Stephens AW, Sabri O. Impact of Training Method on the Robustness of the Visual Assessment of 18F-Florbetaben PET Scans: Results from a Phase-3 Study. J Nucl Med. 2016 Jun;57(6):900-6. doi: 10.2967/jnumed.115.161927. Epub 2016 Jan 28.

ReferenceCitation: Sabri O, Sabbagh MN, Seibyl J, Barthel H, Akatsu H, Ouchi Y, Senda K, Murayama S, Ishii K, Takao M, Beach TG, Rowe CC, Leverenz JB, Ghetti B, Ironside JW, Catafau AM, Stephens AW, Mueller A, Koglin N, Hoffmann A, Roth K, Reininger C, Schulz-Schaeffer WJ; Florbetaben Phase 3 Study Group. Florbetaben PET imaging to detect amyloid beta plaques in Alzheimer's disease: phase 3 study. Alzheimers Dement. 2015 Aug;11(8):964-74. doi: 10.1016/j.jalz.2015.02.004. Epub 2015 Mar 28.

location_country:
Australia
United States
France
Japan
Germany

summary: To determine the sensitivity and specificity of the visual assessment of tracer uptake in the Florbetaben PET images compared to histological verification of the presence or absence of cerebral β-amyloid in the respective histopathologic post mortem specimens as the standard of truth


acronym: EMERGE

title: Randomized, Controlled Phase 2a/b Study of the Efficacy and Safety of PEG-rIL-29 Administered in Combination With Ribavirin to Treatment-Naive Subjects With Chronic Hepatitis C Virus Infection

lead_sponsor: ZymoGenetics

ReferenceCitation: Wang X, Hruska M, Chan P, Ahmad A, Freeman J, Horga MA, Hillson J, Kansra V, Lopez-Talavera JC. Derivation of Phase 3 dosing for peginterferon lambda-1a in chronic hepatitis C, Part 1: Modeling optimal treatment duration and sustained virologic response rates. J Clin Pharmacol. 2015 Jan;55(1):63-72. doi: 10.1002/jcph.363. Epub 2014 Jul 24.

ReferenceCitation: Hruska M, Wang X, Chan P, Ahmad A, Freeman J, Horga MA, Hillson J, Kansra V, Lopez-Talavera JC. Derivation of Phase 3 dosing for peginterferon lambda-1a in chronic hepatitis C, Part 2: Exposure-response analyses for efficacy and safety variables. J Clin Pharmacol. 2015 Jan;55(1):73-80. doi: 10.1002/jcph.361. Epub 2014 Jul 24.

location_country:
Poland
Spain
Italy
Austria
Australia
Canada
Puerto Rico
United States
France
Romania
Germany

summary: Interleukin 29 (IL-29) is a substance that is produced in the body to help fight viral infections. The purpose of this study is to evaluate the safety and antiviral effects of several different doses of PEG-rIL-29 (a man-made form of IL-29) when it is given in combination with daily oral doses of ribavirin (an antiviral drug) to subjects with hepatitis C infection who have received no prior treatment for this disease.


acronym: TULIPIA

title: Phase II, Open, Randomised, Parallel Group, Noncomparative Multicentre Study to Assess the Efficacy and Safety of Repeated Subcutaneous (S.C.) Administration of Different Doses of BIM 23A760 in Acromegalic Patients

lead_sponsor: Ipsen

location_country:
Poland
Brazil
Romania
Latvia
Mexico
Lithuania
Ukraine
Sweden
France
United States
Belgium
Netherlands
Czechia

location_facility: Academy of Medical Science of Ukraine, Department of Clinical Endocrinology

location_facility: P. Stradins Clinical University Hospital

location_facility: Hospital Universitário Clementino Fraga Filho

location_facility: UIM Endocrinología Experimental, Hospital de Especialidades

location_facility: University Hospital Olomouc, Clinic of Internal Medicine

location_facility: Insituto Nacional de Neurologia y Neurocirugia

location_facility: Administration of Medical Services and Rehabilitation of "ARTEM"

location_facility: "C.I. Parhon" National Institute of Endocrinology

location_facility: Oregon University, Dept. of Endocrinology and Neurosurgery

location_facility: Vilnius University Hospital "Santariskiu Klinikos"

location_facility: Massachusetts General Hospital / Neuroendocrine Unit

location_facility: Centre Hospitalier Universitaire de Liège

location_facility: General University Hospital, Clinic of Internal Medicine,

location_facility: National Medical University n.a. M.I.Pirogov

summary: The purpose of this study is to assess the efficacy of repeated subcutaneous (under the skin) injections at different doses of BIM 23A760 on growth hormone and insulin-like growth factor-1 levels in patients with acromegaly after 6 months of treatment.


acronym: INSPIRE

title: A Randomized, Multicenter, Open-Label Phase 3 Study of Pemetrexed-Cisplatin Chemotherapy Plus Necitumumab (IMC-11F8) Versus Pemetrexed-Cisplatin Chemotherapy Alone in the First-Line Treatment of Patients With Stage IV Nonsquamous Non-Small Cell Lung Cancer (NSCLC)

lead_sponsor: Eli Lilly and Company

ReferenceCitation: Paz-Ares L, Mezger J, Ciuleanu TE, Fischer JR, von Pawel J, Provencio M, Kazarnowicz A, Losonczy G, de Castro G Jr, Szczesna A, Crino L, Reck M, Ramlau R, Ulsperger E, Schumann C, Miziara JE, Lessa ÁE, Dediu M, Bálint B, Depenbrock H, Soldatenkova V, Kurek R, Hirsch FR, Thatcher N, Socinski MA; INSPIRE investigators. Necitumumab plus pemetrexed and cisplatin as first-line therapy in patients with stage IV non-squamous non-small-cell lung cancer (INSPIRE): an open-label, randomised, controlled phase 3 study. Lancet Oncol. 2015 Mar;16(3):328-37. doi: 10.1016/S1470-2045(15)70046-X. Epub 2015 Feb 18.

ipd_sharing_description: Anonymized individual patient level data will be provided in a secure access environment upon approval of a research proposal and assigned data sharing agreement.

location_country:
Croatia
Poland
Brazil
Portugal
Romania
Greece
Austria
Australia
Italy
Canada
Russian Federation
Spain
United Kingdom
Hungary
United States
France
South Africa
Belgium
Germany
Slovakia

location_facility:
Imclone Investigational Site
ImClone Investigational Site

summary: The research study is testing the investigational drug necitumumab in the treatment of advanced non-small cell lung cancer. The aim of this study is to determine if necitumumab, given together with a standard chemotherapy combination consisting of cisplatin and pemetrexed will be more effective in improving participant disease than the standard chemotherapy combination alone.


title: Continued Access to Etravirine (ETR) in Treatment Experienced HIV-1 Infected Subjects

lead_sponsor: Janssen Sciences Ireland UC

location_country:
Brazil
Spain
Argentina
Canada
Thailand
Puerto Rico
United States
France
Panama
South Africa
Romania

summary: The purpose of this study is to provide etravirine (ETR) through this trial until participants can be switched to locally available ETR-based treatment regimens (that is, commercially available and reimbursed, or accessible through another source [example, access program or government program]), or local standard of care, as appropriate.


title: A Phase I, Open-Label, Multicentre Study to Assess the Safety, Tolerability, Pharmacokinetics and Preliminary Anti-tumour Activity of Ascending Doses of AZD4547 in Patients With Advanced Solid Malignancies

summary: This study is primarily designed to assess the safety and tolerability of AZD4547 at increasing doses in patients with advanced solid malignancies and for whom no standard medication options are available. It also assesses the blood levels and action of AZD4547 in the body over a period of time.

lead_sponsor: AstraZeneca

location_country:
Spain
Italy
United Kingdom
Netherlands
United States
France
Germany


title: A Multi-centered, Randomized, Double-blind, Placebo-controlled Clinical Trial to Assess the Efficacy, Safety, and Tolerability of Rasagiline Mesylate 1 mg in Patients With Multiple System Atrophy of the Parkinsonian Subtype (MSA-P)

ReferenceCitation: Poewe W, Seppi K, Fitzer-Attas CJ, Wenning GK, Gilman S, Low PA, Giladi N, Barone P, Sampaio C, Eyal E, Rascol O; Rasagiline-for-MSA investigators. Efficacy of rasagiline in patients with the parkinsonian variant of multiple system atrophy: a randomised, placebo-controlled trial. Lancet Neurol. 2015 Feb;14(2):145-52. doi: 10.1016/S1474-4422(14)70288-1. Epub 2014 Dec 8.

location_country:
Portugal
Spain
Italy
Austria
Netherlands
United Kingdom
Canada
Hungary
United States
France
Israel
Germany

location_facility:
Teva Investigational Site 3401
Teva Investigational Site 3204
Teva Investigational Site 1002
Teva Investigational Site 1001
Teva Investigational Site 5101
Teva Investigational Site 1061
Teva Investigational Site 3304
Teva Investigational Site 8002
Teva Investigational Site 1111
Teva Investigational Site 3603
Teva Investigational Site 1003
Teva Investigational Site 3802
Teva Investigational Site 8004
Teva Investigational Site 1010
Teva Investigational Site 5102
Teva Investigational Site 1008
Teva Investigational Site 3801
Teva Investigational Site 3101
Teva Investigational Site 3402
Teva Investigational Site 3403
Teva Investigational Site 1012
Teva Investigational Site 1004
Teva Investigational Site 1005
Teva Investigational Site 3203
Teva Investigational Site 3004
Teva Investigational Site 3201
Teva Investigational Site 1108
Teva Investigational Site 3205
Teva Investigational Site 3005
Teva Investigational Site 3006
Teva Investigational Site 1011
Teva Investigational Site 1109
Teva Investigational Site 3103
Teva Investigational Site 5103
Teva Investigational Site 1006
Teva Investigational Site 1007
Teva Investigational Site 1009
Teva Investigational Site 8003
Teva Investigational Site 3305
Teva Investigational Site 3202
Teva Investigational Site 3502
Teva Investigational Site 1110
Teva Investigational Site 3206
Teva Investigational Site 3503
Teva Investigational Site 3102
Teva Investigational Site 1014
Teva Investigational Site 1013

summary: To test the clinical effect of rasagiline on subjects with MSA of the parkinsonian subtype.


title: A Randomized, Phase II, Placebo-controlled Study of Ipatasertib (GDC-0068), an Inhibitor to Akt, in Combination With Fluoropyrimidine Plus Oxaliplatin in Patients With Locally Advanced or Metastatic Gastric or Gastroesophageal Junction Adenocarcinoma

summary: This multicenter, randomized, double-blind, placebo-controlled study will evaluate the efficacy of ipatasertib in combination with oxaliplatin, 5-fluorouracil, and leucovorin (modified FOLFOX6 [mFOLFOX6]) chemotherapy in participants with advanced or metastatic gastric or gastroesophageal junction (GEJ) cancer. Participants will be randomized to receive either ipatasertib or placebo orally daily on Days 1 to 7 of each 14-day cycle in combination with mFOLFOX6 on Day 1 of each cycle.

ReferenceCitation: Bang YJ, Kang YK, Ng M, Chung HC, Wainberg ZA, Gendreau S, Chan WY, Xu N, Maslyar D, Meng R, Chau I, Ajani JA. A phase II, randomised study of mFOLFOX6 with or without the Akt inhibitor ipatasertib in patients with locally advanced or metastatic gastric or gastroesophageal junction cancer. Eur J Cancer. 2019 Feb;108:17-24. doi: 10.1016/j.ejca.2018.11.017. Epub 2018 Dec 25.

lead_sponsor: Genentech, Inc.

location_country:
Taiwan
Hong Kong
Spain
Italy
United Kingdom
Korea, Republic of
United States
France
Malaysia
Singapore
Germany

location_facility: University Malaya Medical Centre; Clinical Oncology Unit,

location_facility: IRCCS Istituto Oncologico Veneto (IOV); Oncologia Medica Seconda

location_facility: Azienda Ospedaliero Universitaria Pisana; Uff. Sperim. Clin. U.O. Farmaceutica

location_facility: Institut Gustave Roussy; Departement Oncologie Medicale

location_facility: Queen Mary Hospital; Dept. of Clinical Oncology

location_facility: Charité-Universitätsm. Berlin; Med. Klinik mit Schwerpunkt Hämatologie, Onkologie und Tumorimmunolo.

location_facility: Hospital Univ Vall d'Hebron; Servicio de Oncologia

location_facility: Hospital Universiti Sains Malaysia [Neurology]

location_facility: Univ of Calif, Los Angeles; Hematology/Oncology

location_facility: Medizinische Hochschule Hannover; Zentrum Innere Medizin; Abt. Hämatologie u. Onkologie

location_facility: Yonsei Uni College of Medicine, Severance Hospital; Internal Medicine Dept.

location_facility: HOSPITAL DE MADRID NORTE SANCHINARRO- CENTRO INTEGRAL ONCOLOGICO CLARA CAMPAL; Servicio de Oncologia

location_facility: Universitatsklinik Heidelberg; Universitätshautklinik und Nationales Centrum für Tumorerkrankungen

location_facility: Seoul National University Hosp; Dept Internal Med Hem Onc

location_facility: National Cancer Centre; Medical Oncology

location_facility: Gachon Medical School Gil Medical Centre; Internal Medicine

location_facility: Chang Gung Medical Foundation - Linkou; Division of Hematology- Oncology

location_facility: National Taiwan Uni Hospital; Dept of Oncology

location_facility: Hospital Clinico Universitario de Valencia

location_facility: Mount Vernon Hospital; Centre For Cancer Treatment

location_facility: IRCCS Istituto Nazionale Per La Ricerca Sul Cancro (IST); Oncologia Medica A

location_facility: University of Texas M.D. Anderson Cancer Center

location_facility: Seoul National University Bundang Hospital


title: A Prospective Randomized Clinical Trial on 90Yttrium Trans-arterial Radio-Embolization (TheraSphere®) vs. Standard of Care (Sorafenib) for the Treatment of Advanced Hepatocellular Carcinoma (HCC) With Portal Vein Thrombosis (PVT)

acronym: YES-P

summary: This is a two-arm, open-label, prospective, multi-center, randomized, active-controlled clinical trial to assess efficacy and safety of TheraSphere in comparison to standard of care therapy (sorafenib) in the treatment of participants with inoperable liver cancer and blockage of the portal vein.

lead_sponsor: Boston Scientific Corporation

location_country:
Spain
Italy
United Kingdom
United States
France
Belgium

location_facility: Azienda Ospedaliero-Universitaria di Bologna

location_facility: S.C Radiologia Interventistica, A.O.Ospedale Niguarda Ca Granda

location_facility: Hospital Universitario Puerta de Hierro, Gastroenterology & Hepatology Dept

location_facility: UO Radiologia, Ospedali Riuniti di Bergamo

location_facility: University Hospitals Birmingham NHS Foundation Trust

location_facility: Azienda Ospedaliera Ospedali Riuniti "Villa Sofia-Cervello"

location_facility: Northwestern Medical Faculty Foundation, Div of Hematology/Oncology

location_facility: Hospital Universitari i Politecnic la Fe

location_facility: Hospital Clínic Barcelona. IDIBAPS. CIBEREHD. Liver Unit

location_facility: Fondazione Istituto Nazionale Tumori di Milano


title: An Open-Label, Single-arm, Phase Ib/II Study of AEB071 (a Protein Kinase C Inhibitor) and Everolimus (mTOR Inhibitor) in Patients With CD79-mutant or ABC Subtype Diffuse Large B-Cell Lymphoma

lead_sponsor: Novartis Pharmaceuticals

location_country:
Taiwan
Hong Kong
Italy
Netherlands
Korea, Republic of
United States
France
Germany

location_facility: Washington University School of Medicine Dept of Oncology.

location_facility: Sarah Cannon Research Institute Dept of Onc

location_facility: Memorial Sloan Kettering Cancer Center Onc. Dept.

summary:
Study of the safety and efficacy of AEB071 and EVEROLIMUS in patients with CD79-mutant or ABC subtype Diffuse Large B-Cell Lymphoma.

The trial did not progress into Phase II due to the suboptimal tolerability of the combination treatment of sotrastaurin and everolimus in the Phase Ib part of the study. There were no serious safety concerns associated with this combination.


title: A Phase 2 Open-label, Dose-escalation Study to Evaluate the Safety, Pharmacokinetics, Immunogenicity and Pharmacodynamics/Efficacy of EDI200, an EDA-A1 Replacement Protein, Administered to Male Infants With X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED)

lead_sponsor: Edimer Pharmaceuticals

location_country:
United Kingdom
Italy
United States
France
Germany

location_facility: Azienda Ospedaliera-Polo Universitario "Luigi Sacco"

location_facility: Washington University School of Medicine

summary: This Phase 2 first-in-neonate EDI200 study will enroll treatment-naïve, XLHED-affected male newborns in the first two weeks of life. All subjects will meet entry criteria including documentation of an Ectodysplasin (EDA) mutation associated with XLHED. Following Baseline evaluations, EDI200 dosing will be initiated between day-of-life 2 and 14, with each study subject receiving 2 doses/week for a total of 5 doses. The study will enroll subjects in two cohorts with subjects in cohort 1 dosed at 3 mg/kg/dose, associated with partial efficacy, and cohort 2 dosed at 10 mg/kg/dose where enhanced efficacy was demonstrated in the most relevant preclinical model. Given the challenge of identifying families where the subject is yet to be born, it is expected that cohort size and time for recruitment will be variable.


title: RELIANCE 4-FRONT™ Active Fixation Defibrillation Lead Post Market Clinical Follow-Up (PMCF) Study

summary: The objective of this study is to gather data to establish the chronic safety, performance and effectiveness of the RELIANCE 4-FRONT™ Active Fixation Defibrillation Leads.

location_country:
Denmark
Portugal
Spain
Italy
Austria
United Kingdom
Sweden
France
Israel
China
Germany

location_facility: Hospital Universitario Virgen de la Victoria

location_facility: Azienda Ospedaliero Universitaria Pisana


title: A Phase Ib, Open-label, Multi-center, Two-arm, Dose-finding Study to Assess Safety and Efficacy of the Oral Combination or INC424 (INC424) and BKM120 in Patients With Primary Myelofibrosis (PMF), Postpolycythemia Vera-myelofibrosis (PPV-MF), or Post-essential Thrombocythemia-myelofibrosis (PET-MF)

summary: The purpose of this phase Ib clinical trial is to evaluate the safety of the combination of INC424 and BKM120 in the myelofibrosis population and to establish the maximum tolerated dose and or the Recommended Phase II dose of the combination guided by the Bayesian dose escalation model. INC424 has shown efficacy in myelofibrosis (MF) and is approved in the US and EU for the treatment of MF. BKM120 is a PI3K inhibitor. Preclinical and early clinical experience support inhibition of the PI3K/mTOR pathway in MF as aberrant activation of the pathway has been observed in MF models and may contribute to the pathogenesis of the disease.

location_country:
Spain
Italy
Austria
Australia
United Kingdom
France
Israel
Singapore
Germany


title: A Randomized, Double-blind, Placebo Controlled, Parallel Group Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of QGE031 in the Treatment of Patients With Bullous Pemphigoid With Disease Refractory to Oral Steroid Treatment

location_country:
Taiwan
Austria
United States
France
Japan
Germany

summary: To evaluate the safety and efficacy of QGE031 versus placebo in patients with bullous pemphigoid. Efficacy will be assessed as a reduction of disease activity. How QGE031 is broken down by the body and the impact it has on different blood and tissue markers will also be explored.


title: Prognostic Factors and Therapeutic Effects of Surgical Treatment for Traumatic Spinal Column Injury With Spinal Cord Injury: A Prospective, Observational European Multi-center Study.

acronym: SCI-POEM

summary: Despite many years of research, an incomprehensible amount of scientific efforts worldwide and billions of dollars invested, no effective therapy resulting in major neurological or functional recovery is available to date for traumatic spinal cord injury (tSCI). Although there is increasing experimental evidence from animal models that surgical decompression of the spinal cord improves recovery after tSCI, clinical studies have not shown conclusive data yet. The main explanations for this lack of convincing evidence are relatively small sample sizes in previous studies, their predominantly retrospective nature, suboptimal measurement methods for the assessment of neurological deficits, and inappropriate recording and documentation of potential confounding factors.

lead_sponsor: AOSpine Europe

location_country:
Croatia
Serbia
United Kingdom
Ireland
Austria
Italy
Netherlands
France
Turkey
Romania
Germany
Bulgaria

location_facility: National Institute of Emergency Medicine "N.I. Pirogov"


title: A Phase 1 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Biological Activity of SAR405838 in Patients With Advanced Cancer

summary:
Primary Objectives:

To determine safety and the maximum tolerated dose (MTD) of SAR405838 through the characterization of dose-limiting toxicities (DLTs).
To assess biological activities in patients with dedifferentiated liposarcoma during MTD cohort expansion.

Secondary Objectives:

Pharmacokinetic (PK) profile of SAR405838.
Biomarkers in association with SAR405838.
Anti-tumor activity in response to SAR405838.
Food effect on SAR405838 PK.
Compliance with SAR405838 treatment.
Cytochrome P450 3A4/5 (CYP3A4/5) activity.

ReferenceCitation: de Jonge M, de Weger VA, Dickson MA, Langenberg M, Le Cesne A, Wagner AJ, Hsu K, Zheng W, Macé S, Tuffal G, Thomas K, Schellens JH. A phase I study of SAR405838, a novel human double minute 2 (HDM2) antagonist, in patients with solid tumours. Eur J Cancer. 2017 May;76:144-151. doi: 10.1016/j.ejca.2017.02.005. Epub 2017 Mar 17.

lead_sponsor: Sanofi

location_country:
Netherlands
United States
France

location_facility:
Investigational Site Number 840101
Investigational Site Number 840002
Investigational Site Number 840001
Investigational Site Number 528003
Investigational Site Number 528002
Investigational Site Number 528001
Investigational Site Number 250001


title: An Open-label Extension Study to Assess Efficacy, Safety and Tolerability of Canakinumab and the Efficacy and Safety of Childhood Vaccinations in Patients With Cryopyrin Associated Periodic Syndromes (CAPS)

ReferenceCitation: Brogan PA, Hofer M, Kuemmerle-Deschner JB, Koné-Paut I, Roesler J, Kallinich T, Horneff G, Calvo Penadés I, Sevilla-Perez B, Goffin L, Lauwerys BR, Lachmann HJ, Uziel Y, Wei X, Laxer RM. Rapid and Sustained Long-Term Efficacy and Safety of Canakinumab in Patients With Cryopyrin-Associated Periodic Syndrome Ages Five Years and Younger. Arthritis Rheumatol. 2019 Nov;71(11):1955-1963. doi: 10.1002/art.41004. Epub 2019 Sep 9.

location_country:
Spain
United Kingdom
Canada
Switzerland
France
Belgium
Germany

summary: This trial will provide long-term safety, efficacy and tolerability of ACZ885 in CAPS patients that completed the CACZ885D2307 study


title: A Multi-centre, Randomised, Double-blind, Parallel Group, Placebo Controlled Trial to Evaluate the Efficacy and Safety of Activated Recombinant Factor VII (rFVIIa/NovoSeven®/ Niastase®) in the Treatment of Bleeding in Severely Injured Trauma Subjects

summary: This trial is conducted in Africa, Asia, Europe, Oceania and North America. The aim of this trial is to evaluate the efficacy of activated recombinant human factor VII given in conjunction with standard therapy in the treatment of massive bleeding in subjects with severe blunt and/or penetrating trauma injury.

ReferenceCitation: Rossaint R, Christensen MC, Choong PI, Boffard KD, Riou B, Rizoli S, Kluger Y, Lefering R, Morris S; NovoSeven® Trauma Study Group. Cost-Effectiveness of Recombinant Activated Factor VII as Adjunctive Therapy for Bleeding Control in Severely Injured Trauma Patients in Germany. Eur J Trauma Emerg Surg. 2007 Oct;33(5):528-38. doi: 10.1007/s00068-007-6210-x. Epub 2007 Jul 20.

ReferenceCitation: Boffard KD, Riou B, Warren B, Choong PI, Rizoli S, Rossaint R, Axelsen M, Kluger Y; NovoSeven Trauma Study Group. Recombinant factor VIIa as adjunctive therapy for bleeding control in severely injured trauma patients: two parallel randomized, placebo-controlled, double-blind clinical trials. J Trauma. 2005 Jul;59(1):8-15; discussion 15-8.

ReferenceCitation: Rizoli SB, Boffard KD, Riou B, Warren B, Iau P, Kluger Y, Rossaint R, Tillinger M; NovoSeven Trauma Study Group. Recombinant activated factor VII as an adjunctive therapy for bleeding control in severe trauma patients with coagulopathy: subgroup analysis from two randomized trials. Crit Care. 2006;10(6):R178.

ReferenceCitation: Morris S, Ridley S, Munro V, Christensen MC. Cost effectiveness of recombinant activated factor VII for the control of bleeding in patients with severe blunt trauma injuries in the United Kingdom. Anaesthesia. 2007 Jan;62(1):43-52.

ReferenceCitation: Champion HR, Fingerhut A, Escobar MA, Weiskopf RB. The role of data and safety monitoring in acute trauma resuscitation research. J Am Coll Surg. 2007 Jan;204(1):73-83.

ReferenceCitation: Klitgaard T, Tabanera y Palacios R, Boffard KD, Iau PT, Warren B, Rizoli S, Rossaint R, Kluger Y, Riou B; NovoSeven Trauma Study Group. Pharmacokinetics of recombinant activated factor VII in trauma patients with severe bleeding. Crit Care. 2006;10(4):R104.

ReferenceCitation: Kluger Y, Riou B, Rossaint R, Rizoli SB, Boffard KD, Choong PI, Warren B, Tillinger M. Safety of rFVIIa in hemodynamically unstable polytrauma patients with traumatic brain injury: post hoc analysis of 30 patients from a prospective, randomized, placebo-controlled, double-blind clinical trial. Crit Care. 2007;11(4):R85.

ReferenceCitation: Hsia CC, Chin-Yee IH, McAlister VC. Use of recombinant activated factor VII in patients without hemophilia: a meta-analysis of randomized control trials. Ann Surg. 2008 Jul;248(1):61-8. doi: 10.1097/SLA.0b013e318176c4ec.

ReferenceCitation: Levy JH, Fingerhut A, Brott T, Langbakke IH, Erhardtsen E, Porte RJ. Recombinant factor VIIa in patients with coagulopathy secondary to anticoagulant therapy, cirrhosis, or severe traumatic injury: review of safety profile. Transfusion. 2006 Jun;46(6):919-33.

lead_sponsor: Novo Nordisk A/S

location_country:
United Kingdom
Austria
Australia
Canada
France
Israel
Singapore
South Africa
Germany


title: Observational Post-marketing Study on the Safety and Efficacy of Dotarem® (SECURE Study)

summary: The main purpose of this study is to prospectively assess the general safety profile of Dotarem

ReferenceCitation: Heshmatzadeh Behzadi A, McDonald J. Gadolinium-based contrast agents for imaging of the central nervous system: A multicenter European prospective study. Medicine (Baltimore). 2022 Aug 26;101(34):e30163. doi: 10.1097/MD.0000000000030163.

lead_sponsor: Guerbet

location_country:
Saudi Arabia
Italy
Austria
Spain
United Kingdom
Argentina
France
India
China
Germany

location_facility: Bombay Hospital Institute of Medical Sciences


title: An Open-label, Single Arm, Phase II Trial to Investigate the Safety and Efficacy of Sym004 in Patients With Recurrent and/or Metastatic Squamous Cell Carcinoma of the Head and Neck (SCCHN) Who Have Failed Anti-EGFR Monoclonal Antibody-based Therapy

summary:
The trial is designed as a multi-center, open label Phase 2 trial that investigates the efficacy and safety of Sym004 in subjects with squamous cell cancer of the head and neck (SCCHN). Subjects included must have responded to previous anti-epidermal growth factor receptor (anti-EGFR) monoclonal antibody-based therapy and subsequently become resistant to that therapy. It is believed that Sym004 has the potential to induce tumor responses and provide a superior treatment option to subjects with advanced SCCHN.

Symphogen was the sponsor for planning/conducting and reporting results for this trial.

ReferenceCitation: Machiels JP, Specenier P, Krauß J, Dietz A, Kaminsky MC, Lalami Y, Henke M, Keilholz U, Knecht R, Skartved NJ, Horak ID, Pamperin P, Braun S, Gauler TC. A proof of concept trial of the anti-EGFR antibody mixture Sym004 in patients with squamous cell carcinoma of the head and neck. Cancer Chemother Pharmacol. 2015 Jul;76(1):13-20. doi: 10.1007/s00280-015-2761-4. Epub 2015 May 8.

lead_sponsor: Symphogen A/S

location_country: Germany France Belgium

location_facility: 3303; Centre Alexis Vautrin; Département d'Oncologie Médicale

location_facility: 4906; University Medical Center Hamburg Eppendorf; Department of Otorhinolaryngology and Head and Neck Surgery

location_facility: 3203; Antwerp University Hospital; Department of Medical Oncology

location_facility: 3201; Cliniques Universitaires St-Luc; Centre du Cancer

location_facility: 4905; Charité Campus Benjamin Franklin; Hematology, Oncology and Transfusion Medicine

location_facility: 3202; Jules Bordet Institute; Clinique d'Oncologie Médicale

location_facility: 4907; University Hospital Heidelberg; Nationales Centrum für Tumorerkrankungen (NCT)


summary: This study is conducted in 3 stages. Stage 1 is an open-label, dose-escalation assessment of the safety of AGN-150998 administered as a single intravitreal injection to patients with advanced exudative Age-related Macular Degeneration (AMD). Stage 2 and Stage 3 are randomized, double-masked, comparisons of the safety and treatment effects on retinal edema and best-corrected visual acuity (BCVA) of AGN-150998 and ranibizumab in treatment-naive patients with exudative AMD. Study medication is administered as needed in Stage 2 and with a fixed-dosing schedule in Stage 3. The study objectives are (1) to identify the highest tolerated dose of AGN-150998, (2) to assess the safety and duration of treatment effects on retinal edema and BCVA, and (3) to characterize the systemic pharmacokinetic profile of AGN-150998.

ReferenceCitation: Callanan D, Kunimoto D, Maturi RK, Patel SS, Staurenghi G, Wolf S, Cheetham JK, Hohman TC, Kim K, López FJ, Schneider S. Double-Masked, Randomized, Phase 2 Evaluation of Abicipar Pegol (an Anti-VEGF DARPin Therapeutic) in Neovascular Age-Related Macular Degeneration. J Ocul Pharmacol Ther. 2018 Dec;34(10):700-709. doi: 10.1089/jop.2018.0062. Epub 2018 Nov 9.

lead_sponsor: Allergan

location_country:
Italy
Austria
Australia
Switzerland
United States
France
Israel
Germany


title: A Re-Survey Study of Patients With MPS VI (Maroteaux-Lamy Syndrome) Who Previously Participated in ASB-00-02

summary: There is limited information on the long-term effects of treating patients with MPS VI with Naglazyme® and limited data on the natural history of treated and untreated MPS VI patients. The Re-survey Study ASB-00-03 will assist in understanding the effects of long-term Naglazyme treatment and the natural history of the MPS VI patient population.

ReferenceCitation: Giugliani R, Lampe C, Guffon N, Ketteridge D, Leão-Teles E, Wraith JE, Jones SA, Piscia-Nichols C, Lin P, Quartel A, Harmatz P. Natural history and galsulfase treatment in mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy syndrome)--10-year follow-up of patients who previously participated in an MPS VI Survey Study. Am J Med Genet A. 2014 Aug;164A(8):1953-64. doi: 10.1002/ajmg.a.36584. Epub 2014 Apr 24.

lead_sponsor: BioMarin Pharmaceutical

location_country:
Brazil
Portugal
United Kingdom
Australia
United States
France
Germany

location_facility: Manchester Academic Health Sciences Centre


title: A One-year Open-label, Multicenter Trial to Assess Efficacy, Safety and Tolerability of Canakinumab (ACZ885) and the Efficacy and Safety of Childhood Vaccinations in Patients Aged 4 Years or Younger With Cryopyrin Associated Periodic Syndromes (CAPS)

summary: This trial will assess the safety, efficacy and tolerability of ACZ885 in patients aged 4 years and younger with cryopyrin associated periodic syndromes (CAPS)


title: Early Therapy of Atrial Fibrillation for Stroke Prevention Trial (EAST).

acronym: EAST

summary:
EAST prospectively tests the hypothesis that an early, structured rhythm control therapy based on antiarrhythmic drugs and catheter ablation can prevent atrial fibrillation (AF) related complications in patients with AF when compared to usual care.

Patients will be randomized to early therapy or usual care. In the early therapy group, patients will receive either catheter ablation (usually by pulmonary vein isolation), or adequate antiarrhythmic drug therapy at an early time point. The initial therapy will be selected by the local investigator. Upon AF recurrence, both modalities will be combined.

Usual care will be conducted following the 2010European Society of Cardiology ( ESC )guidelines for AF treatment. Early rhythm control therapy will be guided by Electrocardiogram (ECG) monitoring.

primary_purpose: Prevention

ReferenceCitation: International Landmark EAST Study Inverstigates Treatment Options for Atrial Fibrillation

ReferenceCitation: Eckardt L, Sehner S, Suling A, Borof K, Breithardt G, Crijns H, Goette A, Wegscheider K, Zapf A, Camm J, Metzner A, Kirchhof P. Attaining sinus rhythm mediates improved outcome with early rhythm control therapy of atrial fibrillation: the EAST-AFNET 4 trial. Eur Heart J. 2022 Oct 21;43(40):4127-4144. doi: 10.1093/eurheartj/ehac471.

ReferenceCitation: Rillig A, Borof K, Breithardt G, Camm AJ, Crijns HJGM, Goette A, Kuck KH, Metzner A, Vardas P, Vettorazzi E, Wegscheider K, Zapf A, Kirchhof P. Early Rhythm Control in Patients With Atrial Fibrillation and High Comorbidity Burden. Circulation. 2022 Sep 13;146(11):836-847. doi: 10.1161/CIRCULATIONAHA.122.060274. Epub 2022 Aug 15.

ReferenceCitation: Goette A, Borof K, Breithardt G, Camm AJ, Crijns HJGM, Kuck KH, Wegscheider K, Kirchhof P; EAST-AFNET 4 Investigators. Presenting Pattern of Atrial Fibrillation and Outcomes of Early Rhythm Control Therapy. J Am Coll Cardiol. 2022 Jul 26;80(4):283-295. doi: 10.1016/j.jacc.2022.04.058.

ReferenceCitation: Willems S, Borof K, Brandes A, Breithardt G, Camm AJ, Crijns HJGM, Eckardt L, Gessler N, Goette A, Haegeli LM, Heidbuchel H, Kautzner J, Ng GA, Schnabel RB, Suling A, Szumowski L, Themistoclakis S, Vardas P, van Gelder IC, Wegscheider K, Kirchhof P. Systematic, early rhythm control strategy for atrial fibrillation in patients with or without symptoms: the EAST-AFNET 4 trial. Eur Heart J. 2022 Mar 21;43(12):1219-1230. doi: 10.1093/eurheartj/ehab593.

ReferenceCitation: Rillig A, Magnussen C, Ozga AK, Suling A, Brandes A, Breithardt G, Camm AJ, Crijns HJGM, Eckardt L, Elvan A, Goette A, Gulizia M, Haegeli L, Heidbuchel H, Kuck KH, Ng A, Szumowski L, van Gelder I, Wegscheider K, Kirchhof P. Early Rhythm Control Therapy in Patients With Atrial Fibrillation and Heart Failure. Circulation. 2021 Sep 14;144(11):845-858. doi: 10.1161/CIRCULATIONAHA.121.056323. Epub 2021 Jul 30.

ReferenceCitation: Kirchhof P, Camm AJ, Goette A, Brandes A, Eckardt L, Elvan A, Fetsch T, van Gelder IC, Haase D, Haegeli LM, Hamann F, Heidbüchel H, Hindricks G, Kautzner J, Kuck KH, Mont L, Ng GA, Rekosz J, Schoen N, Schotten U, Suling A, Taggeselle J, Themistoclakis S, Vettorazzi E, Vardas P, Wegscheider K, Willems S, Crijns HJGM, Breithardt G; EAST-AFNET 4 Trial Investigators. Early Rhythm-Control Therapy in Patients with Atrial Fibrillation. N Engl J Med. 2020 Oct 1;383(14):1305-1316. doi: 10.1056/NEJMoa2019422. Epub 2020 Aug 29.

ReferenceCitation: Kirchhof P, Breithardt G, Camm AJ, Crijns HJ, Kuck KH, Vardas P, Wegscheider K. Improving outcomes in patients with atrial fibrillation: rationale and design of the Early treatment of Atrial fibrillation for Stroke prevention Trial. Am Heart J. 2013 Sep;166(3):442-8. doi: 10.1016/j.ahj.2013.05.015. Epub 2013 Jul 30.

ipd_sharing_description:
We will share all data that support published results of the trial. Additional data that have not been published will be withheld until six months after publication of these results.

Data will be made available as required for specific, approved analyses. Data will be provided from locked, cleaned and de-identified study database. Requests will be reviewed by AFNET as per the predefined subanalysis plan.

lead_sponsor: Atrial Fibrillation Network

location_country:
Denmark
Poland
Spain
Italy
United Kingdom
Netherlands
Switzerland
France
Belgium
Germany
Czechia

location_facility:
2 Sites
14 Sites
13 Sites
51 Sites
22 Sites
5 Sites
4 Sites
12 Sites
10 Sites


title: Patient Reported Outcomes in Chronic Myeloid Leukemia. GIMEMA QoL - CML0310.

summary:
Although the impact of disease and treatment related burden on health-related quality of life (HRQOL) in patients with solid tumours has been well studied, with several clinical trials that included HRQOL as an endpoint, the general understanding in patients with Chronic Myeloid Leukaemia (CML) is lacking in comparison.

The literature shows that patients' perspective is unique and should always be measured with methodologically sound instruments that are devised for this purpose.

The main scope of this project is develop to an international validated questionnaire for the purpose of HRQOL assessment; such a tool will then be used to provide important data, from the patients' perspective, to make more informed treatment decisions.

time_perspective: Cross-Sectional

ReferenceCitation: Efficace F, Baccarani M, Breccia M, Saussele S, Abel G, Caocci G, Guilhot F, Cocks K, Naeem A, Sprangers M, Oerlemans S, Chie W, Castagnetti F, Bombaci F, Sharf G, Cardoni A, Noens L, Pallua S, Salvucci M, Nicolatou-Galitis O, Rosti G, Mandelli F. International development of an EORTC questionnaire for assessing health-related quality of life in chronic myeloid leukemia patients: the EORTC QLQ-CML24. Qual Life Res. 2014 Apr;23(3):825-36. doi: 10.1007/s11136-013-0523-5. Epub 2013 Sep 13.

lead_sponsor: Gruppo Italiano Malattie EMatologiche dell'Adulto

location_country:
Taiwan
Greece
Austria
Iraq
Italy
Netherlands
United States
France
Belgium
Germany

location_facility: Centre Hospitalier Universitaire of Poitiers

location_facility: Policlinico S. Orsola - Malpighi, Università di Bologna

location_facility: Istituto Scientifico Romagnolo per lo Studio e la Cura dei Tumori- IRST

location_facility: Dana-Farber Cancer Institute Harvard University

location_facility: Ematologia - Sapienza Università di Roma


title: Phase II Multicenter, Open-label, Clinical and Pharmacokinetic Study of Zalypsis® (PM00104) in Patients With Unresectable Locally Advanced and/or Metastatic Ewing Family of Tumors (EFT) Progressing After at Least One Prior Line of Chemotherapy

summary: This is a phase II Multicenter, Open-label, Clinical and Pharmacokinetic Study of Zalypsis® (PM00104) in Patients with Unresectable Locally Advanced and/or Metastatic Ewing Family of Tumors (EFT) Progressing After at Least One Prior Line of Chemotherapy to determine the antitumor activity of Zalypsis.

lead_sponsor: PharmaMar

location_country:
United States
Italy
France

location_facility:
Istituto Clinico Humanitas
St. Jude Children 's Research Hospital
Centre Léon Bérard
Seattle Cancer Care Alliance
Sarcoma Oncology Center
Istituto Nazionale dei Tumori
Istituto Ortopedici Rizzoli