title: A Randomised, Single-blind, Active Controlled, Multi-centre Trial to Evaluate the Efficacy, Safety, Tolerability, Acceptability and Palatability of PMF104 Compared to a Conventional PEG-electrolyte Solution in Children Aged From 2 to Less Than 6, From 6 to Less Than 12, and Adolescents From 12 to Less Than 18 Years of Age Requiring a Diagnostic Procedure Concerning the Colon

summary: A randomised, single-blind, active controlled, multi-centre trial to evaluate the efficacy, safety, tolerability, acceptability and palatability of PMF104 compared to a conventional PEG-electrolyte solution in paediatric subjects requiring a diagnostic procedure concerning the colon.

lead_sponsor: Alfasigma S.p.A.

location_country: Italy Belgium France

location_facility: University and Pediatric Hospital of Lyon

location_facility: Azienda Ospedaliera Universitaria "G. Martino"

location_facility: Pediatric Gastroenterology, Hepatology and Nutrition Children's University Hospital

location_facility: Ospedale Maggiore "C.A. Pizzardi" AUSL Bologna


type: EudraCT Number

title: A Phase IB Combination Study of Rucaparib (CO-338) and Atezolizumab (MPDL3280A) in Participants With Advanced Gynecologic Cancers and Triple-Negative Breast Cancer

summary: This is a Phase Ib, open-label, non-randomized study in patients with previously treated advanced ovarian or endometrial cancer (Part 1) and platinum-sensitive ovarian cancer or triple-negative breast cancer (TNBC) (Part 2) to investigate the dose, safety, pharmacokinetics, and preliminary efficacy of rucaparib in combination with atezolizumab. The study is conducted in 2 parts: a Dose-Finding Phase (Part 1) and a Dose-Expansion Phase (Part 2)

study_start_date_type: Actual

status: Completed

study_completion_date_type: Actual

study_type: Interventional

design_allocation: Non-Randomized

primary_purpose: Treatment

enrollment_type: Actual

lead_sponsor: Hoffmann-La Roche

location_country:
Spain
Australia
United Kingdom
France

location_facility: University College London Hospitals NHS Foundation Trust - University College Hospital

location_facility: Centre Hospitalier Lyon Sud; Service d'Oncologie Médicale

location_facility: Lancashire Teaching Hospitals NHS Foundation Trust

intervention_type: Drug

lead_sponsor_type: industriel

status_simplified: Completed


title: Proof-of-Performance Study of RABIOPRED Assay as an In Vitro Diagnostic Test to Identify Patients With Rheumatoid Arthritis Who Are Unlikely to Show Response to 1st Treatment With Anti-TNFα and Methotrexate Combination.

acronym: RABIOPRED

summary: RABIOPRED is an in vitro non-invasive blood test, which aims to identify patients with rheumatoid arthritis (RA) who are not likely to respond to anti-TNFα and methotrexate combination therapy.

status: Terminated

study_type: Observational

time_perspective: Prospective

lead_sponsor: TcLand Expression S.A.

sponsor_collaborators:
TcLand Expression S.A.
European Commission

location_country: France Netherlands Turkey Czechia Israel

location_facility:
Tel Aviv Surasky Medical Center
CHU Nice
Leiden University Medical Center
Institute of Rheumatology
Istanbul University
University of Marmara
CHU Strasbourg Hautepierre
CHU-Montpellier

intervention_type: Biological

status_simplified: Unknown


title: A Prospective, Multicenter, Single Arm Real-World Registry Investigating the Clinical Use and Safety of the Lutonix Drug Coated Balloon PTA Catheter for Treatment of Dysfunctional Native and Synthetic AV Fistulae

acronym: AVR

summary: The purpose of this Registry is to enroll patients presenting with clinical and hemodynamic abnormalities in native or synthetic (grafts) arteriovenous (AV) fistulae located in the arm. Subjects will be treated with the Lutonix DCB carrying the CE Mark per current IFU and followed clinically for a minimum of 12 months.

ipd_sharing: No

lead_sponsor: C. R. Bard

location_country:
Germany
Italy
Poland
Portugal
United Kingdom
Singapore
Switzerland
Austria
Saudi Arabia
Greece
Turkey
Taiwan
France

location_facility: Klinika Chirurgii Naczyniowej University Hospital nr 1

location_facility: King Abdullah International Medical Research Center

location_facility: Azienda Ospedaliera Di Pisa - Ospedale Cisanello

location_facility: NephroCare Portugal, S.A. NephroCare Lumia

intervention_type: Device


lead_sponsor: Bioprojet

summary: The purpose of this multicenter double blind study is to assess efficacy and safety of Pitolisant versus placebo in paediatric Narcoleptic patients with or without cataplexy.

location_facility: V.M. Behterev National Medical Research Psychiatry and Neurology Center

location_facility: Scientific-Research Medical Complex "Your Health"

location_facility: N.I. Balaban Crimea Repiblic Clinical Psychiatric Hospital No. 1

location_facility: I.M. Sechenov First Moscow State Medical University

status: Active, not recruiting

title: Double Blind, Multicentre, Randomized, Placebo-controlled Trial to Evaluate Safety and Efficacy of Pitolisant in Children From 6 to Less Than 18 Years With Narcolepsy With/Without Cataplexy, Followed by a Prolonged Open-label Period

source: euctr

design_allocation: Randomized

study_completion_date_type: Anticipated

location_country:
Finland
Italy
Netherlands
Russian Federation
France

status_simplified: Ongoing


title: A Phase 2, Open-label, Multiple Ascending Dose Study to Evaluate the Efficacy, Safety, Tolerability, Immunogenicity, Pharmacokinetics, and Pharmacodynamics of ALXN1210 Administered Intravenously to Patients With Paroxysmal Nocturnal Hemoglobinuria

summary: The primary purpose of this study is to evaluate the safety, tolerability, and efficacy of multiple intravenous (IV) doses of ravulizumab administered to complement inhibitor treatment-naïve participants with PNH.

ReferenceType: derived

ReferenceCitation: Roth A, Rottinghaus ST, Hill A, Bachman ES, Kim JS, Schrezenmeier H, Terriou L, Urbano-Ispizua A, Wells RA, Jang JH, Kulasekararaj AG, Szer J, Aguzzi R, Damokosh AI, Shafner L, Lee JW. Ravulizumab (ALXN1210) in patients with paroxysmal nocturnal hemoglobinuria: results of 2 phase 1b/2 studies. Blood Adv. 2018 Sep 11;2(17):2176-2185. doi: 10.1182/bloodadvances.2018020644.

oa_colors: green other

oa_host_type: publisher;repository

evidence: open (via free article)

host_type: publisher

license_normalized: no license

version: publishedVersion

evidence: oa repository (via OAI-PMH doi match)

host_type: repository

repository_institution: PubMed Central - Europe PMC

repository_normalized: PubMed Central

evidence: oa repository (via pmcid lookup)

repositories_concat: PubMed Central

unpaywall_oa_status: bronze

publisher_dissemination: American Society of Hematology

lead_sponsor: Alexion Pharmaceuticals, Inc.

location_country:
Germany
United Kingdom
Korea, Republic of
Spain
Taiwan
Canada
France


title: A Clinical Study of the CardiAQ™ Transcatheter Mitral Valve Implantation (TMVI) System (Transapical Delivery System)

summary:
The purpose of the study is to evaluate the initial short and long-term safety and performance of the CardiAQ™ Transcatheter Mitral Valve Implantation System using the Transapical Delivery System.

The study will enroll patients with moderate to severe mitral valve regurgitation who are considered high or extreme risk for mortality and morbidity from conventional open heart surgery.

lead_sponsor: Edwards Lifesciences

location_country:
Denmark
Netherlands
United Kingdom
France

location_facility: Centre hospitalier régional universitaire de Lille

location_facility: Centre Hospitalier Universiatier de Toulouse


lead_sponsor: Guerbet

summary:
The main purpose of the study is to evaluate the pharmacokinetics of DOTAREM® in the body of children aged less than 2 years thanks to several blood samples (3 ml in total) taken following the administration of DOTAREM®.

DOTAREM® is a contrast agent commonly used for enhancement of Magnetic Resonance Imaging (MRI) to potentially improve the quality of the images and help the diagnosis. Children aged less than 2 years scheduled to undergo routine gadolinium-enhanced MRI of any body region may take part in the study. In this case they will receive DOTAREM®, a solution injected at the standard dose of 0.2mL/kg (0.1 mmol/kg) of body weight.

location_facility: Department of Molecular and Neurological Clinical and Research Center

location_facility: Uniwersytecki Szpital Dziecięcy w Lublinie

location_facility: Borsod-Abaúj-Zemplén University County Hospital

location_facility: Instytut Pomnik -Centrum Zdrowia Dziecka

title: DOTAREM® Pharmacokinetics, Safety and Efficacy Study in Pediatric Subjects Aged <2 Years (Term Newborn Infants to Toddlers 23 Months of Age Inclusive)

primary_purpose: Diagnostic

location_country: Hungary Poland Austria France


title: Benefit of Dual-chamber Pacing With Closed Loop Stimulation (CLS) in Tilt-induced Cardioinhibitory Reflex Syncope. A Randomized Double-blind Parallel Trial.

acronym: BIOSync CLS

summary: The purpose of this study is to assess whether the Closed Loop Stimulation (CLS) in addition to the DDD pacing is effective in reducing syncopal recurrences. The study hypothesis is that DDD pacing with CLS stimulation is able to prevent syncopal recurrences completely or partially by transforming syncope in pre-syncope.

ReferenceCitation: Brignole M, Russo V, Arabia F, Oliveira M, Pedrote A, Aerts A, Rapacciuolo A, Boveda S, Deharo JC, Maglia G, Nigro G, Giacopelli D, Gargaro A, Tomaino M; BioSync CLS trial Investigators. Cardiac pacing in severe recurrent reflex syncope and tilt-induced asystole. Eur Heart J. 2021 Feb 1;42(5):508-516. doi: 10.1093/eurheartj/ehaa936.

oa_colors: green hybrid

evidence: open (via crossref license)

license: cc-by-nc

license_normalized: cc-by-nc

repository_institution: Hospital Center of Central Lisbon - Repository of the Hospital Center of Lisbon Central

unpaywall_oa_status: hybrid

publisher_dissemination: Oxford University Press

ReferenceCitation: Brignole M, Tomaino M, Aerts A, Ammirati F, Ayala-Paredes FA, Deharo JC, Del Rosso A, Hamdan MH, Lunati M, Moya A, Gargaro A; BIOSync Study Steering Committee. Benefit of dual-chamber pacing with Closed Loop Stimulation in tilt-induced cardio-inhibitory reflex syncope (BIOSync trial): study protocol for a randomized controlled trial. Trials. 2017 May 4;18(1):208. doi: 10.1186/s13063-017-1941-4. Erratum In: Trials. 2017 Jun 8;18(1):269.

oa_colors: green gold

evidence: oa journal (via doaj)

license: cc-by

license_normalized: cc-by

repository_institution: Le Centre pour la Communication Scientifique Directe - HAL - Université Paris Descartes

repository_normalized: HAL

repository_institution: Le Centre pour la Communication Scientifique Directe - HAL - Inria

repository_institution: Le Centre pour la Communication Scientifique Directe - HAL - Archive ouverte HAL

repositories:
HAL
PubMed Central

repositories_concat: HAL;PubMed Central

unpaywall_oa_status: gold

publisher_dissemination: Springer-Nature

lead_sponsor: Biotronik SE & Co. KG

location_country: Italy Portugal Netherlands Spain Canada France

location_facility: Azienda Ospedaliera Pia Fondazione di Culto e Religione Cardinale G. Panico

location_facility: Hospital Universitario Nuestra Senora de la Candelaria

location_facility: Azienda Ospedaliera Universitaria Federico II

location_facility: A.O. Dei Colli - Monaldi - University of Campania "Luigi Vanvitelli"


type: Other Identifier

title: Prospective, Randomized, Multicentre Clinical Study of the Hemoteq Ranger™ Paclitaxel-Coated PTA Balloon Catheter (Ranger DCB) in Comparison to Uncoated PTA Balloons in Femoropopliteal Lesions

acronym: RANGER-SFA

summary: The primary objective of this study is to prove the superior performance of the Ranger™ paclitaxel-coated PTA balloon catheter for angioplasty for femoropopliteal artery lesions when compared to non-coated balloons at six months post-procedure when comparing Late Lumen Loss (LLL). Study statistical hypothesis: The %-mean loss of luminal diameter as assessed by angiography at six months follow-up after treatment of the femoropopliteal artery with Ranger DCB study devices is lower than the %-mean loss of luminal diameter after treatment with uncoated PTA balloon control devices.

ReferenceCitation: Bausback Y, Willfort-Ehringer A, Sievert H, Geist V, Lichtenberg M, Del Giudice C, Sauguet A, Diaz-Cartelle J, Marx C, Strobel A, Schult I, Scheinert D; RANGER SFA Investigators. Six-Month Results From the Initial Randomized Study of the Ranger Paclitaxel-Coated Balloon in the Femoropopliteal Segment. J Endovasc Ther. 2017 Aug;24(4):459-467. doi: 10.1177/1526602817710770. Epub 2017 May 31.

oa_host_type: publisher

evidence: open (via free pdf)

repositories_concat: closed

publisher_dissemination: SAGE Publications

lead_sponsor: Hemoteq AG

collaborators:
CERES GmbH
coreLab Black Forest GmbH

sponsor_collaborators:
Hemoteq AG
CERES GmbH
coreLab Black Forest GmbH

location_country: Germany Austria France

location_facility: Hopital Europeen Georges-Pompidou (HEGP)


title: Minitub: Prospective Registry of Sentinel Node (SN) Positive Melanoma Patients With Minimal SN Tumor Burden Who Undergo Completion Lymph Node Dissection (CLND) or Nodal Observation

acronym: Minitub

summary:
The purpose of this registry is to collect data in order to discover whether melanoma patients with minimal SN tumor burden should undergo a complete lymph node dissection (CLND) or not.

Currently, if a patient has a positive (or metastatic) SN, this patient will be offered a CLND, which is a surgical intervention aiming to remove all lymph nodes from the same nodal basin as the SN. However, if the positive (or metastatic) SN is only minimally involved, some centers and/or countries do not offer a CLND routinely. As a matter of fact, the CLND procedure does not increase survival for patients with a minimal SN tumor burden, but can add prognostic information, potentially useful in the subsequent decision-making process. However, this is a surgical operation for the patient, which might be accompanied by significant side effects. Moreover, only approximately 20% of patients with a metastatic SN have further lymph node metastases in the same basin, which means that about 4 patients out of 5 will not benefit from a CLND. Thus, there is an urgent need to identify which SN positive patients could be safely spared from a CLND. It has been demonstrated that breast cancer patients with minimal SN tumor burden can be safely managed with nodal observation only, without performing a CLND. There is evidence that the same situation exists in melanoma as well, but this needs to be validated and this is why we are conducting this registry.

The results of this registry will be crucial to establish an accepted standard of care (CLND or nodal observation) for melanoma patients with minimal SN tumor burden.

lead_sponsor: European Organisation for Research and Treatment of Cancer - EORTC

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

location_facility: Centre Hospitalier Universitaire Vaudois

location_facility: Istituto Nazionale Tumori IRCCS - Fondazione G. Pascale

location_facility: Cambridge University Hospital NHS - Addenbrookes Hospital

location_facility: Charite - Universitaetsmedizin Berlin - Campus Mitte

location_facility: St Helens & Knowsley NHS Trust - Whiston Hospital

location_facility: Istituto Oncologico Veneto IRCCS - Ospedale Busonera

location_facility: The Netherlands Cancer Institute-Antoni Van Leeuwenhoekziekenhuis

location_facility: Erasmus MC Cancer Institute - location Daniel den Hoed

location_facility: Maria Sklodowska-Curie Memorial Cancer Centre

location_facility: Guy's and St Thomas' NHS - St Thomas Hospital


title: A Phase 2, Single-arm, Open-label, Multicenter Study of the Histone Deacetylase Inhibitor (HDACi) JNJ-26481585 in Subjects With Previously Treated Stage Ib-IVa Cutaneous T-cell Lymphoma

summary: The purpose of this study is to determine the overall cutaneous response rate (participants who achieve a complete response or partial response) based on the modified severity weighted assessment tool criteria.

lead_sponsor: Janssen Research & Development, LLC

location_country:
Germany
Portugal
United Kingdom
United States
Spain
France


title: A Phase II Study of Elacytarabine (CP-4055) Plus Idarubicin as Second Course Remission-Induction Therapy in Patients With Acute Myeloid Leukaemia

summary: The main objective of this study is to assess the biological activity of elacytarabine in combination with idarubicin in patients with acute myeloid leukaemia who has failed the first course of a remission-induction treatment with cytarabine (ara-C). In addition, the correlation between hENT1 (human equilibrative nucleoside transporter 1) and overall survival will be studied.

lead_sponsor: Clavis Pharma

collaborators:
Theradex
Syneos Health

sponsor_collaborators:
Clavis Pharma
Theradex
Syneos Health

location_country:
United States
Norway
Germany
France

location_facility: Charite University Hospital Benjamin Franklin


acronym: CT03 Core

lead_sponsor: Chiesi Farmaceutici S.p.A.

summary: The objectives of the study are to demonstrate that Tobrineb®/Actitob®/Bramitob® is non-inferior to TOBI® in the primary efficacy variable, forced expiratory volume in one second (FEV1) percent of predicted normal, and to compare the safety in participants with cystic fibrosis and chronic infection of the lungs with Pseudomonas aeruginosa.

ReferenceType: result

ReferenceCitation: Mazurek H, Chiron R, Kucerova T, Geidel C, Bolbas K, Chuchalin A, Blanco-Aparicio M, Santoro D, Varoli G, Zibellini M, Cicirello HG, Antipkin YG. Long-term efficacy and safety of aerosolized tobramycin 300 mg/4 ml in cystic fibrosis. Pediatr Pulmonol. 2014 Nov;49(11):1076-89. doi: 10.1002/ppul.22989. Epub 2014 Jan 24.

oa_host_type: closed

publisher_dissemination: Wiley

location_facility: Donetsk Regional Children Clinical Hospital

location_facility: Oddzial Kliniczny Interny Dzieciecej i Alergologii, Wojewodzki Szpital Specjalistyczny

location_facility: HELIOS Klinikum Krefeld, Zentrum für Kinder- und Jugendmedizin

location_facility: Klinika Pneumonologii i Mukowiscydozy, Instytut Gruzlicy i Chorob Pluc w Rabce Zdroj

location_facility: Klinika Pediatrii Instytut Matki I Dziecka

location_facility: Lviv Regional Children Specialized Clinical Hospital

location_facility: Centrum pro cystickou fibrosu, Pediatricka klinika UK 2.LF, Fakultní nemocnice v Motole

location_facility: Dzieciecy Szpital Kliniczny Akademii Medycznej, Klinika Chorob Pluc I Reumatologii

location_facility: Federal State Institution: Scientific Research Pulmonology Institute under the Roszdrav

location_facility: Dnipropetrovsk City Children Clinical Hospital # 2

location_facility: State Medical Institution: Regional Children's Hospital Pulmonology Department

location_facility: Complejo Hospitalario Universitario A Coruña (Hospital Materno-Infantil Teresa Herrera)

location_facility: Zaporizhya Regional Clinical Children Hospital

location_facility: Regional State Medical Institution: Smolensk Regional Children's Clinical Hospital

location_facility: Saint-Petersburg State Medical Institution: City Children's Hospital of Saint Olga

location_facility: Odesa Regional Children Clinical Hospital

location_facility: I Oddzial Chorob Dzieciecych, Wojewodzki Specjalistyczny Szpital Dzieciecy

location_facility: Federal State Institution "Nizhegorodskiy Research Institute of Children's Gastroenterology of Russian Medical Technologies"

location_facility: State Higher Educational Institution: Burdenko Voronezh State Medical Academy under the Roszdrav

location_facility: Institute of Phthysiology and Pulmonology n.a., F.G.Yanovskiy of the Academy of Medical Science of Ukraine

location_facility: State Medical Institution: Republican Childrens Clinical Hospital under the Ministry of Health of the Republic of Tatarstan

location_facility: Minicipal Medical Institution: Children's Clinical Hospital #1

location_facility: Institute of Pediatrics, Obstetrics and Gynecology of the Academy of Medical Science of Ukraine

location_facility: State Higher Educational Institution: Bashkir State Medical University under the Roszdrav

location_facility: Szegedi Tudományegyetem Szent-Györgyi Albert Orvos- és Gyógyszerésztudományi Centrum

location_facility: Specjalistyczny ZOZ nad Matka i Dzieckiem, Poradnia Leczenia Mukowiscydozy

location_facility: Pädiatrische Pneumologie und Allergologie, Mukovizidose-Zentrum, Zentrum für Kinderheilkunde und Jugendmedizin

location_facility: Hospital Universitario Ntra Sra. De la Candelaria

location_facility: State Medical Institution: Filatov Chidren's City Clinical Hospital #13

location_facility: Simferopol Central District Clinical Hospital

location_facility: Klinika Pneumonologii, Alergologii Dzieciecej i Immunologii Klinicznej Szpital Kliniczny Uniwersytetu Medycznego w Poznaniu

location_facility: Hospital Universitario Miguel Servet (Children)

location_facility: Hopital Arnaud de Villeneuve, Clinique des maladies respiratoires

location_facility: Poradnia Mukowiscydozy Wojewodzkiej, Przychodni Specjalistycznej dla Dzieci, Szpitala Wojewodzkiego Nr 2

title: A Multicentre, Multinational, Open-Label, Randomised, Parallel Group Clinical Trial of Tobrineb®/Actitob®/Bramitob® (Tobramycin Solution for Nebulisation, 300mg Twice Daily in 4mL Unit Dose Vials) Compared to TOBI® in the Treatment of Patients With Cystic Fibrosis and Chronic Infection With Pseudomonas Aeruginosa

location_country:
Germany
Poland
Hungary
Spain
Czechia
Ukraine
Russian Federation
France


acronym: CERTAIN 2

lead_sponsor: UCB Pharma

summary: To continue to assess the clinical safety and efficacy of Certolizumab Pegol as add-on therapy with stable-dose Disease Modifying Anti-Rheumatic Drugs (DMARDs)

title: A Phase IIIB, Multi-center, Open Label Follow-up Study to Evaluate the Safety and Efficacy of Certolizumab Pegol Administered Concomitantly With DMARDs in Patients With Active Rheumatoid Arthritis Who Participated in C87076.

location_country: Germany Italy Poland Austria France


title: An Open-label, International, Multi-Center, Phase I/II, Dose-escalation Trial Investigating the Safety of Zalutumumab, a Human Monoclonal Epidermal Growth Factor Receptor Antibody in Combination With Radiotherapy, in Patients With Stage III, IVa or IVb Locally Advanced Squamous Cell Carcinoma of the Head and Neck Ineligible for Platinum Based Chemotherapy

summary: The purpose of this study is to investigate the safety of zalutumumab in combination with radiotherapy as the treatment of patients with head and neck cancer who are not eligible for platinum based chemotherapy.

lead_sponsor: Genmab

location_country:
United Kingdom
Belgium
France

location_facility: Sheffield Teaching Hospitals NHS Foundation Trust

location_facility: Centre Georges-Francois Leclerc Hospital


title: Tolerability, Safety and Treatment Response of Flexible Doses of Paliperidone ER in Acutely Exacerbated Subjects With Schizophrenia

acronym: PERTAIN

summary: The purpose of this study is to evaluate the tolerability, safety and treatment response of flexible doses of paliperidone extended-release (ER; designed to slowly release a drug in the body over an extended period of time) tablets in participants with acute schizophrenia (psychiatric disorder with symptoms of emotional instability, detachment from reality, often with delusions and hallucinations, and withdrawal into the self).

lead_sponsor: Janssen-Cilag International NV

location_country: Germany Lithuania France Poland Romania Croatia Israel


title: A Randomized, Multicenter, Open-label, 6-month Study to Explore the Efficacy and Safety of Enteric-coated Mycophenolate Sodium in Combination With Two Corticosteroid Regimens for the Treatment of Lupus Nephritis Flare

summary: The study will investigate the efficacy and safety of enteric-coated mycophenolate sodium in combination with two different corticosteroid (CS) regimes for the induction of remission of a lupus nephritis flare. Patients will be randomly allocated to standard CS regimen (group I) or to a reduced dose CS regimen (group II)

lead_sponsor: Novartis Pharmaceuticals

location_country:
Germany
Italy
Colombia
Hungary
United Kingdom
Spain
Greece
Taiwan
France

location_facility:
Novartis Investigative Site
Novartis


title: Phase I Study of SRC/ABL Tyrosine Kinase Inhibitor Dasatinib [BMS-354825] in Children and Adolescents With Relapsed or Refractory Leukemia, Protocol ITCC 005

summary: The purpose of this clinical research study was to establish a recommended phase 2 once daily (QD) dose of dasatinib and to assess the efficacy of the investigational drug for relapsed or refractory (resistant to previous treatment) leukemia in children and adolescents. The side effects that this oral investigational drug may have in children, and the levels of the drug in the blood, will be studied at different doses.

ReferenceCitation: Zwaan CM, Rizzari C, Mechinaud F, Lancaster DL, Lehrnbecher T, van der Velden VH, Beverloo BB, den Boer ML, Pieters R, Reinhardt D, Dworzak M, Rosenberg J, Manos G, Agrawal S, Strauss L, Baruchel A, Kearns PR. Dasatinib in children and adolescents with relapsed or refractory leukemia: results of the CA180-018 phase I dose-escalation study of the Innovative Therapies for Children with Cancer Consortium. J Clin Oncol. 2013 Jul 1;31(19):2460-8. doi: 10.1200/JCO.2012.46.8280. Epub 2013 May 28.

publisher_dissemination: American Society of Clinical Oncology (ASCO)

lead_sponsor: Bristol-Myers Squibb

collaborators: Innovative Therapies For Children with Cancer Consortium

location_country:
Germany
Italy
United Kingdom
Netherlands
Austria
France


title: Molecular Mechanisms of Disease Progression and Renoprotective Pharmacotherapy in Children With Chronic Renal Failure

acronym: ESCAPE

summary: In children with chronic kidney disease, progression to end-stage renal failure is associated with high patient morbidity and poor quality of life. In adults, inhibition of the renin angiotensin system (RAS) slows down the rate of renal failure progression. This concept is as yet unproven in children, in whom chronic renal failure (CRF) is more commonly due to hypo/dysplastic malformations than to acquired glomerulopathies as typical for adult chronic kidney disease. The current project aims at assessing the genetic and molecular mechanisms and cardiovascular consequences of progressive CRF and to develop a strategy of pharmacological renoprotection in children.

ReferenceType: background

ReferenceCitation: ESCAPE Trial Group; Wuhl E, Trivelli A, Picca S, Litwin M, Peco-Antic A, Zurowska A, Testa S, Jankauskiene A, Emre S, Caldas-Afonso A, Anarat A, Niaudet P, Mir S, Bakkaloglu A, Enke B, Montini G, Wingen AM, Sallay P, Jeck N, Berg U, Caliskan S, Wygoda S, Hohbach-Hohenfellner K, Dusek J, Urasinski T, Arbeiter K, Neuhaus T, Gellermann J, Drozdz D, Fischbach M, Moller K, Wigger M, Peruzzi L, Mehls O, Schaefer F. Strict blood-pressure control and progression of renal failure in children. N Engl J Med. 2009 Oct 22;361(17):1639-50. doi: 10.1056/NEJMoa0902066.

ReferenceCitation: Tabatabaeifar M, Schlingmann KP, Litwin M, Emre S, Bakkaloglu A, Mehls O, Antignac C, Schaefer F, Weber S; ESCAPE Trial Group. Functional analysis of BMP4 mutations identified in pediatric CAKUT patients. Pediatr Nephrol. 2009 Dec;24(12):2361-8. doi: 10.1007/s00467-009-1287-6. Epub 2009 Aug 14.

ReferenceCitation: Gimpel C, Wuhl E, Arbeiter K, Drozdz D, Trivelli A, Charbit M, Gellermann J, Dusek J, Jankauskiene A, Emre S, Schaefer F; ESCAPE Trial Group. Superior consistency of ambulatory blood pressure monitoring in children: implications for clinical trials. J Hypertens. 2009 Aug;27(8):1568-74. doi: 10.1097/HJH.0b013e32832cb2a8.

ReferenceCitation: Grenda R, Wuhl E, Litwin M, Janas R, Sladowska J, Arbeiter K, Berg U, Caldas-Afonso A, Fischbach M, Mehls O, Sallay P, Schaefer F; ESCAPE Trial group. Urinary excretion of endothelin-1 (ET-1), transforming growth factor- beta1 (TGF- beta1) and vascular endothelial growth factor (VEGF165) in paediatric chronic kidney diseases: results of the ESCAPE trial. Nephrol Dial Transplant. 2007 Dec;22(12):3487-94. doi: 10.1093/ndt/gfm300. Epub 2007 Sep 26.

ReferenceCitation: Chinali M, de Simone G, Matteucci MC, Picca S, Mastrostefano A, Anarat A, Caliskan S, Jeck N, Neuhaus TJ, Peco-Antic A, Peruzzi L, Testa S, Mehls O, Wuhl E, Schaefer F; ESCAPE Trial Group. Reduced systolic myocardial function in children with chronic renal insufficiency. J Am Soc Nephrol. 2007 Feb;18(2):593-8. doi: 10.1681/ASN.2006070691. Epub 2007 Jan 10.

ReferenceCitation: Schonfelder EM, Knuppel T, Tasic V, Miljkovic P, Konrad M, Wuhl E, Antignac C, Bakkaloglu A, Schaefer F, Weber S; ESCAPE Trial Group. Mutations in Uroplakin IIIA are a rare cause of renal hypodysplasia in humans. Am J Kidney Dis. 2006 Jun;47(6):1004-12. doi: 10.1053/j.ajkd.2006.02.177.

ReferenceCitation: Matteucci MC, Wuhl E, Picca S, Mastrostefano A, Rinelli G, Romano C, Rizzoni G, Mehls O, de Simone G, Schaefer F; ESCAPE Trial Group. Left ventricular geometry in children with mild to moderate chronic renal insufficiency. J Am Soc Nephrol. 2006 Jan;17(1):218-26. doi: 10.1681/ASN.2005030276. Epub 2005 Nov 9.

ReferenceCitation: Wuhl E, Mehls O, Schaefer F; ESCAPE Trial Group. Antihypertensive and antiproteinuric efficacy of ramipril in children with chronic renal failure. Kidney Int. 2004 Aug;66(2):768-76. doi: 10.1111/j.1523-1755.2004.00802.x.

ReferenceCitation: Jourdan C, Wuhl E, Litwin M, Fahr K, Trelewicz J, Jobs K, Schenk JP, Grenda R, Mehls O, Troger J, Schaefer F. Normative values for intima-media thickness and distensibility of large arteries in healthy adolescents. J Hypertens. 2005 Sep;23(9):1707-15. doi: 10.1097/01.hjh.0000178834.26353.d5.

ReferenceCitation: Litwin M, Wuhl E, Jourdan C, Trelewicz J, Niemirska A, Fahr K, Jobs K, Grenda R, Wawer ZT, Rajszys P, Troger J, Mehls O, Schaefer F. Altered morphologic properties of large arteries in children with chronic renal failure and after renal transplantation. J Am Soc Nephrol. 2005 May;16(5):1494-500. doi: 10.1681/ASN.2004110932. Epub 2005 Mar 16.

ReferenceCitation: Wuhl E, Hadtstein C, Mehls O, Schaefer F; ESCAPE Trial Group. Ultradian but not circadian blood pressure rhythms correlate with renal dysfunction in children with chronic renal failure. J Am Soc Nephrol. 2005 Mar;16(3):746-54. doi: 10.1681/ASN.2004070537. Epub 2005 Jan 12.

ReferenceCitation: Hadtstein C, Wuhl E, Soergel M, Witte K, Schaefer F; German Study Group for Pediatric Hypertension. Normative values for circadian and ultradian cardiovascular rhythms in childhood. Hypertension. 2004 Mar;43(3):547-54. doi: 10.1161/01.HYP.0000116754.15808.d8. Epub 2004 Jan 26.

ReferenceCitation: Wuhl E, Hadtstein C, Mehls O, Schaefer F; Escape Trial Group. Home, clinic, and ambulatory blood pressure monitoring in children with chronic renal failure. Pediatr Res. 2004 Mar;55(3):492-7. doi: 10.1203/01.PDR.0000106863.90996.76. Epub 2003 Nov 19.

lead_sponsor: Heidelberg University

sponsor_collaborators:
Heidelberg University
European Commission
Boehringer Ingelheim
Baxter Healthcare Corporation
Aventis Pharmaceuticals

location_country:
Germany
Italy
Lithuania
Poland
Portugal
Hungary
Sweden
Switzerland
Turkey
Serbia
Czech Republic
France

location_facility: Istanbul University, Cerrahpasa Medical Faculty, Dept, of Pediatrics

location_facility: Hopital Necker, Division of Pediatric Nephrology

location_facility: University Hospital Motol, 1st Department of Pediatrics

location_facility: Hospital S. Joao-Faculade de Medicina do Porto, Dept. of Pediatrics

location_facility: Azienda Ospedaliera, Istitui Clinici di Perfezionamento, Servizio die Emodialisi Pediatrica

location_facility: Humboldt University Berlin, Charité Children's Hospital, Department of Pediatric Nephrology

location_facility: Philipps University Marburg, Dept. of Pediatrics

location_facility: Urban Hospital St. Georg, Department of Pediatrics, Pediatric Nephrology Unit

location_facility: Faculty of Medicine Belgrade, University Children's Hospital, Nephrology Unit

location_facility: Ege University Medical Faculty, Dept. of Pediatric Nephrology

location_facility: Vilnius University Children's Hospital, Pediatric Department, Nephrology Unit

location_facility: Hacettepe University, Faculty of Medicine, Pediatric Nephrology and Rheumatology

location_facility: Karolinska Institute, Huddinge University Hospital, Dept. of Pediatrics

location_facility: Cukurova University School of Medicine, Dept. of Pediatric Nephrology

location_facility: Ospedale Pediatrico Bambino Gesù, Division of Nephrology and Dialysis

location_facility: Azienda Ospedaliera die Padova. U.O. Nefrologia Dialisi e Trapianto - Dipartimento di Pediatria

location_facility: Semmelweis University Budapest, 1st Department of Pediatrics

location_facility: University of Istanbul, Istanbul Medical Faculty, Dept. of Pediatrics

location_facility: University Hospital Hamburg-Eppendorf, University Children's Hospital, Dept. of Pediatric Nephrology

location_facility: Clinic of Pediatrics, Pomeranian Academy of Medicine

location_facility: Jagellonian University Medical College, Department of Pediatric Nephrology

location_facility: Medical University of Gdansk, Pediatric Nephrology Department

location_facility: University Hospital Essen, Department of Pediatrics, Pediatric Nephrology Unit

location_facility: Ospedale Infantile Regina Margherita, U.O.A. Nefrologia, Dialisi, Trapianto

location_facility: Hannover Medical School, Children's Hospital Div. II, Pediatric Nephrology

location_facility: Division of Pediatric Nephrology, Children's Hospital, University of Heidelberg

location_facility: University Children's Hospital, Dept. of Nephrology

location_facility: Johannes Gutenberg University Mainz, Department of Pediatrics,

location_facility: Children's Memorial Health Hospital, Nephrology and Kidney Transplantation Department

lead_sponsor_type: academique


title: A Multi-center, Phase 2, Open-Label Study of Fabrazyme (Recombinant Human a-Galactosidase A) Replacement Therapy in Pediatric Patients With Fabry Disease

summary: People with Fabry disease have an alteration in their genetic material (DNA) which causes a deficiency of the a-galactosidase A enzyme. This enzyme helps to break down and remove certain types of fatty substances called "glycolipids". These glycolipids are normally present within the body in most cells. In people with Fabry disease, glycolipids build up in various tissues such as the liver, kidney, skin, and blood vessels because a-galactosidase A is not present, or is present in small quantities. The build up of glycolipid levels (also referred to as "globotriaosylceramide" or "GL-3") in these tissues is thought to cause the clinical symptoms that are common to Fabry disease. Symptoms commonly appear during childhood with pain in the hands and feet. This study explored the safety, efficacy and pharmacokinetics of Fabrazyme in pediatric patients aged between 7 and 15 years.

ReferenceCitation: Wraith JE, Tylki-Szymanska A, Guffon N, Lien YH, Tsimaratos M, Vellodi A, Germain DP. Safety and efficacy of enzyme replacement therapy with agalsidase beta: an international, open-label study in pediatric patients with Fabry disease. J Pediatr. 2008 Apr;152(4):563-70, 570.e1. doi: 10.1016/j.jpeds.2007.09.007. Epub 2007 Dec 3.

lead_sponsor: Genzyme, a Sanofi Company

location_country:
United States
United Kingdom
Poland
France

location_facility: Great Ormond Street Hospital for Sick Children


title: A Randomized Phase III Study to Assess Intensification of the Conditioning Regimen for Allogenic Stem Cell Transplantation (ALLO-SCT) for Leukemia or Myelodysplastic Syndrome With a High Risk of Relapse

summary:
RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining chemotherapy with peripheral stem cell transplantation may allow the doctor to give higher doses of chemotherapy drugs and kill more cancer cells.

PURPOSE: Randomized phase III trial to compare the effectiveness of idarubicin plus peripheral stem cell transplantation using the patient's own or donated stem cells in treating patients with leukemia or myelodysplastic syndrome.

status: Unknown status

enrollment_type: Anticipated

sponsor_collaborators: European Organisation for Research and Treatment of Cancer - EORTC

location_country: Germany Italy Netherlands Belgium France

location_facility:
Eberhard Karls Universitaet
Institut Jules Bordet
University Medical Center Nijmegen
Leiden University Medical Center
Hotel Dieu de Paris
Institut Gustave Roussy
Hopital Edouard Herriot
Azienda Policlinico Umberto Primo
Ospedale San Eugenio


title: A Randomized, Open-label, Multicentric, Two-arm Pivotal Trial of SonoCloud-9 Combined With Carboplatin (CBDCA) vs Standard of Care Lomustine (CCNU) or Temozolomide (TMZ) in Patients Undergoing Planned Resection for First Recurrence Glioblastoma.

acronym: SONOBIRD

summary: The brain is protected from any toxic or inflammatory molecule by the blood-brain barrier (BBB). This physical barrier is located at the level of the blood vessel walls. Because of these barrier properties, the blood vessels are also impermeable to the passage of therapeutic molecules from the blood to the brain. The development of effective treatments against glioblastoma is thus limited due to the BBB that prevents most drugs injected in the bloodstream from getting into brain tissue where the tumour is seated. The SonoCloud-9 (SC9) is an investigational device using ultrasound technology and specially developed to open the BBB in the area of and surrounding the tumour. The transient opening of the BBB allows more drugs to reach the brain tumour tissue. Carboplatin is a chemotherapy that is approved to treat different cancer types alone or in combination with other drugs, and has been used in the treatment of glioblastoma. Despite its proven efficacy in the laboratory on glioblastoma cells, carboplatin does not readily cross the BBB in humans. A clinical trial has shown that in combination with the SonoCloud-9, more carboplatin can reach the brain tumour tissue. The objective of the proposed trial is to show that the association - carboplatin with the SonoCloud-9 - will increase efficacy of the drug in patients with recurrent glioblastoma.

study_start_date_type: Anticipated

status: Not yet recruiting

ReferenceCitation: Carpentier A, Canney M, Vignot A, Reina V, Beccaria K, Horodyckid C, Karachi C, Leclercq D, Lafon C, Chapelon JY, Capelle L, Cornu P, Sanson M, Hoang-Xuan K, Delattre JY, Idbaih A. Clinical trial of blood-brain barrier disruption by pulsed ultrasound. Sci Transl Med. 2016 Jun 15;8(343):343re2. doi: 10.1126/scitranslmed.aaf6086.

ReferenceCitation: Sonabend AM, Gould A, Amidei C, Ward R, Schmidt KA, Zhang DY, Gomez C, Bebawy JF, Liu BP, Bouchoux G, Desseaux C, Helenowski IB, Lukas RV, Dixit K, Kumthekar P, Arrieta VA, Lesniak MS, Carpentier A, Zhang H, Muzzio M, Canney M, Stupp R. Repeated blood-brain barrier opening with an implantable ultrasound device for delivery of albumin-bound paclitaxel in patients with recurrent glioblastoma: a phase 1 trial. Lancet Oncol. 2023 May;24(5):509-522. doi: 10.1016/S1470-2045(23)00112-2.

lead_sponsor: CarThera

location_country:
Germany
Italy
Belgium
United States
Spain
France

location_facility:
Hospital Universitario 12 de Octubre
Universitair Ziekenhuis Leuven
Hôpital de La Timone
Northwestern University
Neurochirurgie uniklinik Köln
Istituto Oncologico Veneto
Hôpital Neurologique Pierre Wertheimer
Hôpital de la Pitié-Salpêtrière


type: Registry Identifier

title: A Phase I, Non-randomized, Open-label, Multi-center Dose Escalation Trial of BI 764532 Combined With Ezabenlimab in Patients With Small Cell Lung Carcinoma and Other Neuroendocrine Neoplasms Expressing DLL3

summary:
This study is open to adults with small cell lung cancer and other neuroendocrine tumours that are positive for the tumour marker Delta-like 3 (DLL3). The study is in people with advanced cancer for whom previous treatment was not successful or no standard treatment exists.

The purpose of this study is to find out the highest dose of BI 764532 that people can tolerate when taken together with another medicine called ezabenlimab. BI 764532 and ezabenlimab are antibodies that may help the immune system fight cancer. Participants get BI 764532 and ezabenlimab as infusions into a vein.

If there is benefit for the participants and if they can tolerate it, the treatment is given for a maximum of 3 years. During this time, participants visit the study site about every week. The visits also depend on the response to the treatment. At the study visits, the doctors check the health of the participants, take necessary laboratory tests, and note any health problems that could have been caused by the study treatment.

status: Recruiting

ipd_sharing: Yes

ipd_sharing_description:
Once the criteria in section "Time Frame" are fulfilled, researchers can use the following link https://www.mystudywindow.com/msw/datasharing to request access to the clinical study documents regarding this study, and upon a signed "Document Sharing Agreement".

Furthermore, researchers can request access to the clinical study data, for this and other listed studies, after the submission of a research proposal and according to the terms outlined in the website.

lead_sponsor: Boehringer Ingelheim

location_country: Germany Japan Belgium France

location_facility: Universitätsklinikum Carl Gustav Carus Dresden


title: EPOS Discoid Meniscus (DiMe) Project: a Prospective Multicentric Cohort Protocol

acronym: DiMe

summary: The aims of this Discoid Meniscus (DiMe) project are to analyze DM tears characteristics in the pediatric European population to describe current treatment options in symptomatic DM, and to evaluate clinical outcomes. To reach these objectives, a prospective database including symptomatic DM and variables associated with DM characteristics, type of tears, treatment, imaging, and clinical follow-up will be implemented. Data collection platform will provide future studies to understand the best treatment option for skeletally immature patient with symptomatic DM according to their history and lesion characteristics.

ReferenceCitation: Turati M, Anghilieri FM, Accadbled F, Piatti M, Di Benedetto P, Moltrasio F, Zatti G, Zanchi N, Bigoni M. Discoid meniscus in human fetuses: A systematic review. Knee. 2021 Jun;30:205-213. doi: 10.1016/j.knee.2021.04.006. Epub 2021 May 1.

ReferenceCitation: Ahn JH, Lee SH, Yoo JC, Lee YS, Ha HC. Arthroscopic partial meniscectomy with repair of the peripheral tear for symptomatic discoid lateral meniscus in children: results of minimum 2 years of follow-up. Arthroscopy. 2008 Aug;24(8):888-98. doi: 10.1016/j.arthro.2008.03.002. Epub 2008 May 5.

ReferenceCitation: Wasser L, Knorr J, Accadbled F, Abid A, Sales De Gauzy J. Arthroscopic treatment of discoid meniscus in children: clinical and MRI results. Orthop Traumatol Surg Res. 2011 May;97(3):297-303. doi: 10.1016/j.otsr.2010.11.009. Epub 2011 Mar 23.

ReferenceCitation: Kinugasa K, Hamada M, Yonetani Y, Matsuo T, Mae T, Nakata K, Horibe S. Discoid lateral meniscal repair without saucerization for adolescents with peripheral longitudinal tear. Knee. 2019 Jun;26(3):803-808. doi: 10.1016/j.knee.2019.03.007. Epub 2019 May 7.

ReferenceCitation: Matsuo T, Kinugasa K, Sakata K, Ohori T, Mae T, Hamada M. Post-operative deformation and extrusion of the discoid lateral meniscus following a partial meniscectomy with repair. Knee Surg Sports Traumatol Arthrosc. 2017 Feb;25(2):390-396. doi: 10.1007/s00167-016-4393-6. Epub 2016 Dec 23.

ReferenceCitation: Nepple JJ, Milewski MD, Shea KG. Research in Osteochondritis Dissecans of the Knee: 2016 Update. J Knee Surg. 2016 Oct;29(7):533-538. doi: 10.1055/s-0036-1586723. Epub 2016 Aug 17. Erratum In: J Knee Surg. 2016 Nov;29(8):696.

ReferenceCitation: Moksnes H, Engebretsen L, Seil R. The ESSKA paediatric anterior cruciate ligament monitoring initiative. Knee Surg Sports Traumatol Arthrosc. 2016 Mar;24(3):680-7. doi: 10.1007/s00167-015-3746-x. Epub 2015 Aug 7.

ReferenceCitation: Scott EJ, Westermann R, Glass NA, Hettrich C, Wolf BR, Bollier MJ. Performance of the PROMIS in Patients After Anterior Cruciate Ligament Reconstruction. Orthop J Sports Med. 2018 May 25;6(5):2325967118774509. doi: 10.1177/2325967118774509. eCollection 2018 May.

ReferenceCitation: Grant-Beuttler M, Jennings J, McCauley C, Dulay R, Grossnickle K, Kill K, Hay J. Development of an Electronic Version of The Children's Self-Perceptions of Adequacy in and Predilection for Physical Activity (CSAPPA) Scale. Pediatr Exerc Sci. 2017 Feb;29(1):153-160. doi: 10.1123/pes.2016-0115. Epub 2016 Oct 21.

ReferenceCitation: Briggs KK, Lysholm J, Tegner Y, Rodkey WG, Kocher MS, Steadman JR. The reliability, validity, and responsiveness of the Lysholm score and Tegner activity scale for anterior cruciate ligament injuries of the knee: 25 years later. Am J Sports Med. 2009 May;37(5):890-7. doi: 10.1177/0363546508330143. Epub 2009 Mar 4.

ReferenceCitation: Dietvorst M, Reijman M, van Groningen B, van der Steen MC, Janssen RPA. PROMs in paediatric knee ligament injury: use the Pedi-IKDC and avoid using adult PROMs. Knee Surg Sports Traumatol Arthrosc. 2019 Jun;27(6):1965-1973. doi: 10.1007/s00167-017-4687-3. Epub 2017 Sep 19.

ReferenceCitation: Outerbridge RE. The etiology of chondromalacia patellae. 1961. Clin Orthop Relat Res. 2001 Aug;(389):5-8. doi: 10.1097/00003086-200108000-00002. No abstract available.

ReferenceCitation: CholeS Study Group, West Midlands Research Collaborative. Population-based cohort study of outcomes following cholecystectomy for benign gallbladder diseases. Br J Surg. 2016 Nov;103(12):1704-1715. doi: 10.1002/bjs.10287. Epub 2016 Aug 26. Erratum In: Br J Surg. 2018 Aug;105(9):1222.

ReferenceCitation: Courtney A, O'Connell R, Rattay T, Kim B, Cutress RI, Kirwan CC, Gandhi A, Fairbrother P, Sharma N, Cartlidge CWJ, Horgan K, McIntosh SA, Leff DR, Vidya R, Potter S, Holcombe C, Copson E, Coles CE, Dave RV. The B-MaP-C study: Breast cancer management pathways during the COVID-19 pandemic. Study protocol. Int J Surg Protoc. 2020;24:1-5. doi: 10.1016/j.isjp.2020.07.003. Epub 2020 Jul 29.

lead_sponsor: University of Milano Bicocca

location_country:
Finland
Italy
Portugal
United Kingdom
Switzerland
Belgium
France

location_facility: SHEFFIELD CHILDREN'S NHS NHS Foundation Trust,

location_facility: Centre hospitalier universitaire vaudois (CHUV)

location_facility: Department of Pediatric Orthopaedics, Hospital Pediátrico de -Coimbra

location_facility: Department of Orthopaedic Surgery, Hôpital des Enfants, CHU de Toulouse


title: A Participant and Investigator-blinded, Randomized, Placebo-controlled Phase 2a Study to Investigate the Pharmacokinetics, Pharmacodynamics, Safety and Tolerability of TIN816 in Patients With Sepsis-associated Acute Kidney Injury

summary: The purpose of this study is to characterize the pharmacokinetic (PK) and pharmacodynamic (PD) profile and to evaluate the safety and tolerability of TIN816 in hospitalized adult participants in an intensive care setting with a diagnosis of sepsis-associated acute kidney injury (SA-AKI).

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: Germany Hungary Belgium Spain France

intervention_type: Other


title: A Phase 3 Study to Evaluate the Safety and Pharmacokinetics of Berotralstat Prophylaxis in Children With Hereditary Angioedema Who Are 2 to < 12 Years of Age

acronym: APeX-P

summary: The purpose of this study is to evaluate the pharmacokinetics (PK) and safety of berotralstat to determine the appropriate weight-based dose for pediatric participants 2 to < 12 years old for prophylactic treatment to prevent attacks of hereditary angioedema (HAE).

lead_sponsor: BioCryst Pharmaceuticals

location_country:
Germany
France
Italy
Poland
United Kingdom
Romania
Austria
Spain
Canada
Israel

location_facility:
Investigative Site #3
Investigative Site #2
Investigative Site #1


acronym: WISARD

lead_sponsor: NEAT ID Foundation

summary:
HIV-1 infected subjects that experience virological failure while on non nucleoside reverse-transcriptase inhibitors (NNRTIs), including those with the K103N mutation, are usually switched to a boosted PI-based regimen or other antiretroviral (ARV) combinations. The same is true for subjects who need to start antiretroviral therapy and have acquired virus that is already resistant to antiretrovirals. These "second line" combinations are often associated with numerous issues that can have a potential impact on the quality of life (QoL) of these patients. Therefore a simpler and better tolerated alternative second line treatment option would be a useful tool for the clinical management of these patients.

The aim of this study is to assess the efficacy and tolerability of a dual combined therapy of Dolutegravir (DTG) 50 mg OD + Rilpivirine (RPV) 25 mg OD in virologically suppressed participants with previous virological failure with NNRTIs and having the clinically significant mutation K103N. The secondary objective of the study is to assess whether a simplification of the treatment in terms of pill burden, long term metabolic toxicity and potential for drug interactions improves the QOL of the participants. The study will also evaluate DTG & RPV concentrations in the blood plus changes in cell associated virus.

In order to compare the first line treatment (boosted PI and/or other antiretroviral combinations) and the DTG+RPV combination, two thirds of study participants will be switched to DTG+RPV immediately and receive DTG+RPV for 96 weeks. The other third will be switched after 48 weeks of continuing on their first line treatment and receive DTG+RPV for 48 weeks. All participants will then be followed up for a further 30 days. Participants will be recruited from sites across Europe, and randomised onto either arm of the study. After randomisation, participants will attend approximately 10 visits over the course of two years.

sponsor_collaborators:
NEAT ID Foundation
ViiV Healthcare

location_facility: ASST FBF SACCO- I Division of Infectious Diseases 3

location_facility: Hospital General Universitario de Alicante

location_facility: North Bristol NHS Trust, Southmead Hospital

location_facility: ASST GOM Niguarda Milano, Dep. Infectious Disease

location_facility: ASST FBF SACCO- I Division of Infectious Diseases 1

location_facility: Infectious Diseases Unit Hospital de la Santa Creu i Sant Pau

title: An Open-Label, Multi-Centre, Randomised, Switch Study to Evaluate the Virological Efficacy Over 96 Weeks Of 2-Drug Therapy With DTG/RPV FDC in Antiretroviral Treatment-Experienced HIV-1 Infected Subjects Virologically Suppressed With NNRTIs Resistance Mutation K103N

location_country:
Germany
Italy
United Kingdom
Belgium
Spain
France


title: An Open-label, Single-arm, Multicenter Pilot Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Efficacy and Safety of Pegcetacoplan in Patients With Transplant-associated Thrombotic Microangiopathy (TA-TMA) After Hematopoietic Stem Cell Transplantation (HSCT)

summary: The purpose of the study is to assess PK, Pharmacodynamics (PD), Efficacy and safety of pegcetacoplan in patients with TA-TMA after HSCT.

lead_sponsor: Swedish Orphan Biovitrum

sponsor_collaborators:
Swedish Orphan Biovitrum
Apellis Pharmaceuticals, Inc.

location_country:
Italy
United States
Spain
Greece
France

location_facility:
Flore Sicre de Fontbrune, MD
Antonio Risitano, MD
Rafael Francisco Duarte Palomino, MD
Panagiotis Tsirigotis, MD
Giovanni Grillo, MD
Elisabetta Terruzi, MD
Simona Sica, MD
Jerome Cornillon, MD
Ryotaro Nakamura, MD
Hassan AlKhateeb, MD
Ioannis Baltadakis, MD
Luca Castagna, MD
Ioanna Sakellari, MD
Michael Loschi, MD


title: PROton Versus Photon Therapy for Esophageal Cancer - a Trimodality Strategy (PROTECT) A Multicenter International Randomized Phase III Study of Neoadjuvant Proton Versus Photon Chemoradiotherapy in Locally Advanced Esophageal Cancer

acronym: PROTECT

summary: The PROTECT trial will test the hypothesis that proton (PT) -enabled radiation dose reductions to sensitive, normal tissues will result in lower rates of treatment-related pulmonary complications in esophageal cancer compared to standard photon therapy (XT).

lead_sponsor: University of Aarhus

collaborators: CNAO National Center of Oncological Hadrontherapy

collaborators: Agenzia Nazionale per i Servizi Sanitari Regionali

collaborators: Paul Scherrer Institut, Center for Proton Therapy

location_country:
Germany
Italy
United Kingdom
Denmark
Netherlands
Switzerland
Belgium
France

location_facility: Azienda Provinciale Per I Servizi Sanitari (APSS)

location_facility: Centro Nazionale di Adroterapia Oncologica (CNAO)

location_facility: University College London Hospital (UCLH)

location_facility: Stichting Maastricht Radiation Oncology (MAASTRO)

intervention_type: Radiation


title: A Multicenter, Randomized, Double-blind, Placebo-controlled Trial With a Single Arm run-in Period to Evaluate the Safety and Efficacy of Sodium Thiosulfate (BYON5667) Eye Drops to Reduce Ocular Toxicity in Cancer Patients Treated With SYD985

summary: This multicenter, randomized, double-blind, placebo-controlled trial with a single arm run-in period is to evaluate the safety and efficacy of sodium thiosulfate (BYON5667) eye drops to reduce ocular toxicity in cancer patients treated with the antibody-drug conjugate (ADC) SYD985

primary_purpose: Prevention

lead_sponsor: Byondis B.V.

location_country: Spain Belgium France

location_facility: ICO I'Hospitalet - Hospital Duran i Reynals


title: A Randomized, Controlled, Pilot Study to Evaluate Safety and Feasibility of Pressure-controlled Intermittent Coronary Sinus Occlusion (PiCSO) Therapy in Patients With ST Elevation Inferior Wall Myocardial Infarction Presenting With TIMI 0 or 1 and Symptom Duration ≤ 12 Hours Treated Adjunct to Percutaneous Coronary Intervention (PCI) Compared to Standard PCI.

acronym: PiCSO-AMI-V

summary: The objective of this study is to assess safety and feasibility of Pressure-controlled intermittent Coronary Sinus Occlusion (PiCSO) therapy in patients with extensive ST elevation inferior wall myocardial infarction presenting with TIMI 0 or 1 and symptom duration ≤ 12 hours undergoing percutaneous coronary intervention (PCI) compared to standard PCI.

lead_sponsor: Miracor Medical SA

location_country:
United Kingdom
Denmark
Switzerland
Latvia
France

location_facility: CHU Hôpiteaux de Bordeaux, Hôpital Haut Lévéque

location_facility: Centre Hospitalier Universitaire de Toulouse

location_facility: Pauls Stradins Clinical University Hospital

location_facility: EOC Ospedale Regionale di Lugano - Civico

location_facility: Centre Hospitalier Régional Universitaire de Lille


summary: The main purpose of this phase 2 study is to assess the efficacy and safety of CMK389 in patients with atopic dermatitis.

title: A Randomized, Subject and Investigator Blinded, Placebo-controlled Multicenter Study to Assess the Efficacy and Safety of CMK389 in Patients With Moderate to Severe Atopic Dermatitis

location_country: Germany Poland Hungary Spain Czechia France


title: Retrospective Chart Review Study of Patients With PIK3CA-Related Overgrowth Spectrum (PROS) Who Have Received Alpelisib as Part of a Compassionate Use Program (EPIK-P1)

summary:
The study was a site-based retrospective non-interventional medical chart review of pediatric and adult male and female patients with PIK3CA-Related Overgrowth Spectrum (PROS) who initiated alpelisib at least 24 weeks before the cut-off date at a MAP site. The study cut-off date was 09-Mar-2020.

Patient-level data were abstracted from medical charts of all eligible patients at all participating sites. Study completion date refers to the last date data was extracted.

Information from patients treated with alpelisib was used to describe the efficacy and safety of alpelisib in PROS patients.

time_perspective: Retrospective

location_country:
Ireland
Australia
United States
Spain
France

location_facility:
Boston Childrens Hospital
Novartis Investigative Site


title: Elbow Outcomes Clinical Study (WELBOW)

acronym: WELBOW

summary: WELBOW study is an international, ambispective and prospective, single arm, multicenter, observational, non-comparative, Post-Market Clinical Follow-up (PMCF).

time_perspective: Other

lead_sponsor: Stryker Trauma GmbH

location_country:
France
United Kingdom
Belgium
Canada
Sweden

location_facility:
Linköping University
AZ Herentals
Lawson Health Research Institute
Rouen University Hospital
Wrightington Hospital
CHU Tours
Sjukhuset i Varberg Hospital
Clinique du parc


lead_sponsor: Sanofi Pasteur, a Sanofi Company

summary:
Primary Objective:

To demonstrate that high-dose quadrivalent influenza vaccine (QIV-HD) induces an immune response that is superior to the responses induced by standard-dose quadrivalent influenza vaccine (QIV-SD) for all 4 virus strains 28 days post-vaccination in participants 60 to 64 years of age and in participants 65 years of age and older.

Secondary Objective:

Immunogenicity: To further describe the immune response induced by QIV-HD and QIV-SD in all participants by age group, in pooled age groups, and by vaccine group (QIV-HD; QIV-SD).
Safety: To describe the safety profile of all participants by age group, in pooled age groups, and by vaccine group (QIV-HD; QIV-SD).

location_facility:
Investigational Site Number 2760003
Investigational Site Number 0560002
Investigational Site Number 3800001
Investigational Site Number 2760004
Investigational Site Number 6160003
Investigational Site Number 2760001
Investigational Site Number 6160002
Investigational Site Number 6160001
Investigational Site Number 2500004
Investigational Site Number 5280001
Investigational Site Number 2760002
Investigational Site Number 6160004
Investigational Site Number 2500003
Investigational Site Number 0560001
Investigational Site Number 2760005
Investigational Site Number 3800003
Investigational Site Number 2500001

title: Immunogenicity and Safety of a High-Dose Quadrivalent Influenza Vaccine Administered by the Intramuscular Route in Subjects 60 Years of Age and Older

ipd_sharing_description: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org

location_country: Germany Italy Poland Netherlands Belgium France


title: High-Density (HD) Wave Mapping in Subjects With Atrial Fibrillation as a Predictor of Recurrence After a Single Ablation Procedure Using a PVI-Only Strategy

acronym: WAVE-MAP AF

summary: The aim is to use the GRID to characterize the atrial substrate and develop a model for predicting recurrence rates after a single procedure using a PVI only approach and a contact catheter.

ipd_sharing: Undecided

lead_sponsor: Abbott Medical Devices

location_country:
Germany
Italy
Portugal
United Kingdom
Netherlands
Austria
Spain
Czechia
France

location_facility: Complexo Hospitalario Universitario de Santiago

location_facility: Azienda Ospedaliero Universitaria Pisana

intervention_type: Procedure


title: A Randomized, Double-Blind (Sponsor-unblinded), Placebo-Controlled, Adaptive Trial to Investigate the Antiviral Effect, Safety, Tolerability and Pharmacokinetics of GSK3640254 in HIV-1 Infected Treatment-Naïve Adults

summary: Infection with HIV-1 continues to be a serious health threat throughout the world. Chronic exposure to combination anti-retroviral therapy identified anti-retroviral associated long-term toxicities. Hence, there is a need to prevent these co-morbidities. GSK3640254 is a next-generation HIV-1 Maturation Inhibitor (MI) which may be effective for HIV-1 infection. This study will evaluate the antiviral effect, safety, tolerability and pharmacokinetics/ pharmacodynamics of GSK3640254 in HIV-1 infected treatment-naive adults. This study will consists of two parts; Part 1 and Part 2. Part 1 will evaluate two active doses of GSK3640254, 200 milligrams (mg) (Cohort 1) and 10 mg (Cohort 2) along with placebo to match GSK3640254 Mesylate salt. Part 2 will evaluate three active doses of GSK3640254. Dose level 1 of GSK3640254 that can provide at least 30 percent of the maximum effect (Cohort 1), dose level 2 of GSK3640254 that can provide at least 75 percent of the maximum effect (Cohort 2) and dose level 3 of GSK3640254 that can provide at least 90 percent of the maximum effect (Cohort 3). These doses are anticipated to be 5 mg, 40 mg and 100 mg respectively, but could be modified based on data obtained in Part 1. Subjects will also receive placebo to match GSK3640254 Mesylate salt in Part 2 of the study. All doses will be administered after a moderate fat meal. This study will consist of Screening period (up to 14 days), Treatment period (Day 1- Day 10), post-dose Follow-up (Day 11- Day 17) and final Follow-up (Day 18-24). A total of approximately 34 subjects will be enrolled, of which, 14 subjects will be randomized in Part 1 and 20 in Part 2 of the study. Six subjects will be enrolled in each of the active dose cohorts and 2 subjects will be enrolled in each of the placebo cohorts.

ReferenceCitation: Spinner CD, Felizarta F, Rizzardini G, Philibert P, Mitha E, Domingo P, Stephan CJ, DeGrosky M, Bainbridge V, Zhan J, Dumitrescu TP, Jeffrey JL, Xu J, Halliday F, Gan J, Johnson M, Gartland M, Joshi SR, Lataillade M. Phase IIa Proof-of-Concept Evaluation of the Antiviral Efficacy, Safety, Tolerability, and Pharmacokinetics of the Next-Generation Maturation Inhibitor GSK3640254. Clin Infect Dis. 2022 Sep 14;75(5):786-794. doi: 10.1093/cid/ciab1065.

license: cc-by-nc-nd

license_normalized: cc-by-nc-nd

ipd_sharing_description: IPD for this study will be made available via the Clinical Study Data Request site.

lead_sponsor: ViiV Healthcare

location_country:
Germany
Italy
United States
Spain
South Africa
France


title: A Multicentre, Open-label, Three-arm Randomised Phase II Trial Assessing the Safety and Efficacy of the HSP90 Inhibitor Ganetespib in Combination With Carboplatin Followed by Maintenance Treatment With Niraparib Versus Ganetespib Plus Carboplatin Followed by Ganetespib and Niraparib Versus Carboplatin in Combination With Standard Chemotherapy Followed by Niraparib Maintenance Treatment in Platinum-sensitive Ovarian Cancer Patients

acronym: EUDARIO

summary:
This study will be performed in women with platinum-sensitive, high-grade serous, high-grade endometrioid, undifferentiated epithelial ovarian cancer, carcinosarcoma, fallopian tube or primary peritoneal cancer (proven by central histo-pathological review).

A total of 120 subjects will be randomized (1:1:1) to three different treatment arms: (A) Standard arm (arm A): Carboplatin (AUC5 d1, q3w i.v.) in combination with Paclitaxel (175 mg/m² d1, q3w i.v.) or Carboplatin (AUC4 d1, q3w i.v.) in combination with Gemcitabine (1000 mg/m² d1, d8, q3w i.v.) followed by maintenance therapy with Niraparib (200/ 300 mg oral daily, q4w) // (B) First experimental arm (arm B): Ganetespib (150 mg/m2, d1, q3w) in combination with Carboplatin (AUC5 d1, q3w i.v.) followed by maintenance treatment with Niraparib (200/ 300 mg oral daily, q4w) // (C) Second experimental arm (arm C): Ganetespib (150 mg/m² d1, q3w i.v.) plus Carboplatin (AUC5 d1, q3w i.v.) followed by Ganetespib (100 mg/m² d1, d8, d15, d22, q4w i.v.) and Niraparib (200 mg oral daily, q4w).

Chemotherapy treatment will be given for 6 cycles, maintenance treatment with Ganetespib will be given for a maximum of 9 months or until disease progression, maintenance treatment with Niraparib can continue until disease progression.

lead_sponsor: Universitaire Ziekenhuizen KU Leuven

sponsor_collaborators:
Universitaire Ziekenhuizen KU Leuven
European Commission

location_country: Germany Italy Austria Belgium France

location_facility: Kliniken Essen Mitte, Evang. Huyssens- Stiftung/Knappschaft GmbH

location_facility: Fondazione IRCCS Istituto Nazionale dei tumori Milano

location_facility: Centre de lutte contre le cancer Francois Baclesse


title: First Randomized Study of Pressure-controlled Intermittent Coronary Sinus Occlusion (PiCSO) in Acute Myocardial Infarction

acronym: PiCSO-AMI-I

summary: The objective of this study is to assess efficacy and safety of Pressure-controlled intermittent Coronary Sinus Occlusion (PiCSO) therapy started post flow restoration but prior to stenting during percutaneous coronary intervention (PCI) compared to standard PCI in the setting of acute ST-segment elevation anterior myocardial infarction (STEMI).

location_country:
Germany
United Kingdom
Denmark
Switzerland
Latvia
France


title: An International Multicentre, Open-Label First in Human Phase I/II Study to Evaluate the Safety, Tolerability, Biodistribution and Antitumour Activity of 177Lu-3BP-227 for the Treatment of Subjects With Solid Tumours Expressing Neurotensin Receptor 1

summary:
This study was conducted to advance new treatment for patients with metastatic or locally advanced cancers expressing Neurotensin receptor 1 (NTSR1). This study was the first time the investigational drug called 177Lu-3BP-227 was administered to patients under controlled conditions of a clinical study.

The purpose of this study was to evaluate how safe the investigational drug is as well to verify how well it is tolerated by patients after several intravenous administrations. In addition, the effect of the study drug on tumoral lesions and how it distributes throughout the body and at which rate it is removed from the body was evaluated. Since 177Lu-3BP-227 is a radio-labelled drug, it also measured how the emitted radiation is distributed throughout the body (dosimetry).

The study consisted of a phase I dose escalation part. The study originally planned to include a phase II study however due to early termination (not due to safety concerns) the study did not progress to phase II and was stopped during phase I. For the phase I dose escalation part, it was anticipated that approximately 30 subjects will be included, in up to six escalation steps. No expansion cohorts were implemented.

lead_sponsor: Ipsen

location_country:
Switzerland
Netherlands
Belgium
United States
France

location_facility: The University of Texas MD Anderson Cancer Center


title: Adjuvant Immunotherapy in Patients With Resected Gastric Cancer Following Preoperative Chemotherapy With High Risk for Recurrence (N+ and/or R1): an Open Label Randomized Controlled Phase-2-study

acronym: VESTIGE

summary:
The primary objective of the trial is to investigate if nivolumab plus ipilimumab given as adjuvant treatment improve disease free survival (DFS) in patients with stage Ib-IVa gastric and esophagogastric junction adenocarcinoma and high risk of recurrence (defined by ypN1-3 and/or R1 status) following neoadjuvant chemotherapy and resection.

Other study objectives:

To investigate the safety and effect of adjuvant immunotherapy on long term oncologic outcomes and quality of life of patients in the study
To correlate nutritional status assessment on outcomes and quality of life of patients

ReferenceCitation: Smyth E, Knodler M, Giraut A, Mauer M, Nilsson M, Van Grieken N, Wagner AD, Moehler M, Lordick F. VESTIGE: Adjuvant Immunotherapy in Patients With Resected Esophageal, Gastroesophageal Junction and Gastric Cancer Following Preoperative Chemotherapy With High Risk for Recurrence (N+ and/or R1): An Open Label Randomized Controlled Phase-2-Study. Front Oncol. 2020 Jan 30;9:1320. doi: 10.3389/fonc.2019.01320. eCollection 2019.

repository_institution: Université de Lausanne - SERVAL

evidence: oa repository (via OAI-PMH title and first author match)

repository_normalized: qucosa

repository_institution: University of Cambridge - Apollo

repositories_institution:
PubMed Central - Europe PMC
University of Cambridge - Apollo
Université de Lausanne - SERVAL

publisher_dissemination: Frontiers

lead_sponsor: European Organisation for Research and Treatment of Cancer - EORTC

location_country:
Germany
Italy
France
Poland
Norway
United Kingdom
Spain
Czechia
Israel

location_facility: Institut Catalan d'Oncologia - ICO Badalona

location_facility: Istituto Nazionale Tumori IRCCS "Fondazione G. Pascale"

location_facility: Universitaets Krankenhaus Eppendorf - Universitaetsklinikum Hamburg-Eppendorf KE - University Cancer Center

location_facility: Oslo University Hospital - Ullevaal Hospital

location_facility: Azienda Ospedaliera a Papa Giovanni XXIII

location_facility: Universitaetsklinikum Tuebingen-Uni Kliniken Berg

location_facility: University College London Hospitals NHS Foundation Trust

location_facility: Maria Sklodowska-Curie Memorial Cancer Centre

location_facility: Rambam Health Care Campus, Oncology Institute

location_facility: Klinikum Rechts der isar Der Technische Universitaet Muenchen - Klinikum Rechts Der Isar

location_facility: Hospital Clinico Universitario de Valencia


title: A Modular, Multi-arm, Multi-part, First Time in Patient Study to Evaluate the Safety and Tolerability of OMO-1, Alone and in Combination With Anti-cancer Treatments, in Patients With Locally Advanced, Unresectable or Metastatic Solid Malignancies

summary: This is a modular, first time in patient, open-label, multicentre study of OMO-1, administered orally, alone and in combination with anti-cancer treatments, in patients with locally advanced, unresectable or metastatic solid malignancies.

lead_sponsor: Octimet Oncology N.V.

location_country:
United Kingdom
Netherlands
Belgium
United States
France

location_facility: UMCU Universitair Medisch Centrum Utrecht

location_facility: The Newcastle Upon Tyne Hospitals NHS Foundation Trust

location_facility: University of Oxford, Department of Oncology


acronym: EASYKID

summary: The purpose of the protocol is to demonstrate that Eziclen®/Izinova®, an osmotic sulphate-based laxative preparation given on the day before colonoscopy has non-inferior efficacy to Klean-Prep® (polyethylene glycol (PEG)-electrolytes) on colon cleansing in adolescents aged 12 to 17 years (inclusive) with a body weight >40 kg, scheduled to undergo a colonoscopy for a routinely accepted diagnostic indication.

location_facility: Uniwersytecki Szpital Dziecięcy w Krakowie

location_facility: Uniwersytecki Szpital Dziecięcy w Lublinie

location_facility: Uniwersytecki Dziecięcy Szpital Kliniczny im. L. Zamenhofa w Białymstoku

location_facility: Instytut "Pomnik - Centrum Zdrowia Dziecka"

location_facility: ORN Santobono-Pausilipon Padiglione Santobono

location_facility: Samodzielny Publiczny Szpital Kliniczny Nr 6 Śląskiego

location_facility: Universitätsklinikum Leipzig, Klinik und Poliklinik für Kinder- und Jugendmedizin

location_facility: Samodzielny Publiczny Szpital Kliniczny Nr 1 we Wrocławiu

title: Efficacy, Safety and Tolerability of a Bowel Cleansing Preparation (Eziclen/Izinova®) in Paediatric Subjects Undergoing Colonoscopy: a Phase III, Multicentre, Randomised, Comparative Study Versus Klean-Prep® (PEG-Electrolytes), Administered on the Day Before Colonoscopy, Investigator-blinded, Non-inferiority in Adolescents of 12 to 17 Years of Age (Inclusive) >40 kg

primary_purpose: Other

location_country: Germany Italy Poland Netherlands Czechia France


lead_sponsor: Novartis Pharmaceuticals

summary: This is Proof-of-Concept (POC) study to assess the preliminary antitumor activity and safety and tolerablity using ceritinib (LDK378) in the treatment of life threatening tumors that are characterized by ALK genetic alteration (and/or overexpression in some diseases).

title: A Phase II, Open Label, Multi-center, Multi-arm Study of Ceritinib in Patients With Advanced Solid Tumors and Hematological Malignancies Characterized by Genetic Abnormalities of Anaplastic Lymphoma Kinase (ALK)

location_country:
France
Italy
Denmark
Korea, Republic of
Spain
Thailand
Czechia
Israel


lead_sponsor: Janssen Research & Development, LLC

summary: The purpose of this study is to evaluate the long-term safety and tolerability of JNJ-54861911 in participants in the early Alzheimer's disease (AD [progressive brain disease that slowly destroys memory and thinking skills, and eventually even the ability to carry out the simplest tasks]) spectrum that have completed a Phase 1b or Phase 2 clinical trial with JNJ-54861911, who are willing to continue their assigned treatment.

ReferenceCitation: Novak G, Streffer JR, Timmers M, Henley D, Brashear HR, Bogert J, Russu A, Janssens L, Tesseur I, Tritsmans L, Van Nueten L, Engelborghs S. Long-term safety and tolerability of atabecestat (JNJ-54861911), an oral BACE1 inhibitor, in early Alzheimer's disease spectrum patients: a randomized, double-blind, placebo-controlled study and a two-period extension study. Alzheimers Res Ther. 2020 May 14;12(1):58. doi: 10.1186/s13195-020-00614-5.

repository_institution: Vrije Universiteit Brussel - VUBIR

repository_normalized: vubissmart

repository_institution: University of Antwerp - Institutional Repository University of Antwerp

repository_normalized: irua

repository_institution: Research Square - Research Square

repository_normalized: Research Square

version: submittedVersion

repositories:
PubMed Central
Research Square
irua
vubissmart

repositories_pmh: europepmc.org irua vubissmart

title: A Randomized, Two-Period, Double-Blind Placebo-Controlled and Open-Label, Multicenter Extension Study to Determine the Long-Term Safety and Tolerability of JNJ-54861911 in Subjects in the Early Alzheimer's Disease Spectrum

location_country: Germany Sweden Netherlands Belgium Spain France


title: Bifurcation Lesion Analysis and STenting / BLAST

acronym: BLAST

summary: The purpose of this research study is to determine if Intravascular Ultrasound (IVUS) grayscale and VH (Virtual Histology)-IVUS pre-stenting can assess more accurately the location, amount, and type of a blockage than angiogram alone. Additionally, this study is to determine if IVUS grayscale and VH-IVUS guidance will result in improved acute and long term procedural outcome vs. bifurcation stenting with only angiographic guidance in native coronary arteries.

lead_sponsor: Volcano Corporation

location_country:
Italy
Poland
United Kingdom
Denmark
Netherlands
United States
Latvia
France

location_facility: Kardiologisk laboratorium/B-lab Skejby Sygehus

location_facility: Saint Luke's Hospital-Mid America Heart Institute

location_facility: Azienda Sanitaria Ospedaliera Molinette San Giovanni Battista al Torino Departp Emodinamica Universitaria

location_facility: Pauls Stradins Clinical University Hospital

location_facility: St. Thomas Hospital Cardiothoracic Centre


title: A Phase 2a Randomized, Double-blind, Placebo-Controlled, Parallel-Group, Multi-center Study Investigating the Safety and Tolerability of JNJ-54861911 in Subjects With Early Alzheimer's Disease

summary: The purpose of this study is to evaluate the long-term safety and tolerability of JNJ-54861911 during 6 months of treatment in participants with early (predementia) alzheimer's disease (AD [degenerative disease of the brain characterized by the insidious onset of dementia, impairment of memory, judgment, attention span, and problem solving skills are followed by severe apraxias and a global loss of cognitive abilities]).

repositories_institution: University of Antwerp - Institutional Repository University of Antwerp


title: Phase II Study of Cabozantinib in Patients With Gastrointestinal Stromal Tumor (GIST) Who Progressed During Neoadjuvant, Adjuvant or Palliative Therapy With Imatinib and Sunitinib

summary:
The study is a multi-center, multi-national, open label, single arm Phase II study of single-agent cabozantinib. The objective of the study is to assess the safety and activity of cabozantinib in patients with metastatic GIST who have previously progressed on imatinib and sunitinib and have not been exposed yet to other KIT- or PDGFR-directed tyrosine kinase inhibitors.

Patient will receive cabozantinib until they experience no further benefit from the treatment, becoming intolerant to the drug or wishing to discontinue the treatment. Treatment beyond RECIST 1.1 progression is allowed in patients deriving clinical benefit upon investigator's discretion, provided no other criteria for treatment withdrawal are met.

ReferenceCitation: Kyriazoglou A, Jespers P, Vandecavaye V, Mir O, Kasper B, Papai Z, Blay JY, Italiano A, Zaffaroni F, Litiere S, Nzokirantevye A, Schoffski P. Exploratory analysis of tumor imaging in a Phase 2 trial with cabozantinib in gastrointestinal stromal tumor: lessons learned from study EORTC STBSG 1317 'CaboGIST'. Acta Oncol. 2022 Jun;61(6):663-668. doi: 10.1080/0284186X.2022.2068967. Epub 2022 Apr 28.

publisher_dissemination: Informa

ReferenceCitation: Schoffski P, Mir O, Kasper B, Papai Z, Blay JY, Italiano A, Benson C, Kopeckova K, Ali N, Dileo P, LeCesne A, Menge F, Cousin S, Wardelmann E, Wozniak A, Marreaud S, Litiere S, Zaffaroni F, Nzokirantevye A, Vanden Bempt I, Gelderblom H. Activity and safety of the multi-target tyrosine kinase inhibitor cabozantinib in patients with metastatic gastrointestinal stromal tumour after treatment with imatinib and sunitinib: European Organisation for Research and Treatment of Cancer phase II trial 1317 'CaboGIST'. Eur J Cancer. 2020 Jul;134:62-74. doi: 10.1016/j.ejca.2020.04.021. Epub 2020 May 26.

publisher_dissemination: Elsevier

location_country:
Germany
Hungary
United Kingdom
Belgium
Czechia
France

location_facility: University College London Hospitals NHS Foundation Trust - University College Hospital (622)

location_facility: Military Hospital - State Health Centre (3769)

location_facility: Clatterbridge Centre for Oncology NHS Trust - Clatterbridge Cancer Centre NHS Foundation Trust (659)

location_facility: Royal Marsden Hospital - Chelsea, London (613)


title: A Two-part, Multicentre, International Phase I and II Trial Assessing the Safety and Efficacy of the Hsp90 Inhibitor Ganetespib in Combination With Paclitaxel Weekly in Women With High-grade Serous, High-grade Endometrioid, or Undifferentiated, Platinum-resistant Epithelial Ovarian, Fallopian Tube or Primary Peritoneal Cancer

summary: Epithelial ovarian cancer (EOC) is the most lethal gynaecological malignancy causing 41900 deaths annually in Europe. The predominance of aggressive Type II tumours, which are characterised by a high frequency of p53 mutations, and primary or acquired resistance to platinum-based chemotherapy profoundly contribute to the high mortality rate. With current standard therapy the median overall survival of metastatic platinum-resistant (Pt-R) ovarian cancer patients is only 14 month. There is a pressing need for more effective, innovative treatment strategies to particularly improve survival in this subgroup of EOC patients. This is a drug strategy targeting a central driver of tumour aggressiveness and metastatic ability, namely mutant p53, via an innovative new Hsp90 (heat shock protein 90) inhibition mechanism. The most advanced, second-generation Hsp90 inhibitor will be used, Ganetespib. The first part (Phase I) of the GANNET53 trial will test the safety of Ganetespib in a new combination with standard chemotherapy (Paclitaxel weekly) in Pt-R EOC patients. The second part (randomised Phase II) will examine the efficacy of Ganetespib in combination with standard chemotherapy versus standard chemotherapy alone in EOC patients with Pt-R tumours.

lead_sponsor: Medical University Innsbruck

sponsor_collaborators:
Medical University Innsbruck
European Commission

location_country: Germany Austria Belgium France

location_facility: Medical University Innsbruck, Department for Gynaecology and Obstetrics

location_facility: University Hospital Carl Gustav Carus Dresden, Department of Gynaecology and Obstetrics

location_facility: Assistance Publique - Hôpitaux de Paris Medical Oncology Department

location_facility: Centre de lutte contre le cancer Francois Baclesse

location_facility: Universitätsklinikum Hamburg-Eppendorf Dept. of Gynecology and Gynecologic Oncology

location_facility: Kliniken Essen Mitte, Evang. Huyssens-Stiftung / Knappschaft GmbH Department of Gynaecologic Oncology

location_facility: Universitätsmedizin Berlin Charité, Dept. for Gynecology

location_facility: Katholieke Universiteit Leuven, Dept. of Gynaecologic Oncology


summary: Soft tissue sarcomas (STS) are a rare group of malignant heterogenous tumors (> 50 histological subtypes, including liposarcoma, the commonest subtype of STS) with distinct genetic, pathological and clinical profiles, and varying patterns of tumor spread. The optimal cytotoxic treatment for this group of patients remains uncertain. Single agents which are most effective include doxorubicin and ifosfamide, but objective response rates and progression-free survival times remain modest. There is clearly a need to improve treatment options for liposarcoma. Eribulin, a antimicrotubule agent that targets the protein tubulin in cells, interfering with cancer cell division and growth , has demonstrated activity in STS. Therefore, it is reasonable to explore whether other anti-microtubule agent like cabazitaxel have a role in STS. Cabazitaxel has been shown to be a relatively safe, effective and tolerated. This drug has been approved by FDA for prostate cancer. The main objective of this trial is to determine whether cabazitaxel demonstrate sufficient antitumor activity for liposarcoma.

location_facility: Assistance Publique - Hopitaux de Marseille - Hôpital de La Timone (287)

location_facility: Hopitaux Universitaires Bordet-Erasme - Institut Jules Bordet (101)

location_facility: Istituto Oncologico Veneto IRCCS - Ospedale Busonera (3908)

location_facility: Fondazione IRCCS Istituto Nazionale dei Tumori (704)

location_facility: CHU de Dijon - Centre Georges-Francois-Leclerc (229)

title: Phase II Trial of Cabazitaxel in Metastatic or Inoperable Locally Advanced Dedifferentiated Liposarcoma

location_country:
Italy
United Kingdom
Belgium
France


summary: The purpose of this study is to examine the safety and efficacy of decitabine in sequential administration with cytarabine in children with relapsed or refractory acute myeloid leukemia (AML).

title: Phase 1-2 Safety and Efficacy Study of DACOGEN in Sequential Administration With Cytarabine in Children With Relapsed or Refractory Acute Myeloid Leukemia

location_country:
Germany
United Kingdom
Denmark
Netherlands
Belgium
Spain
France


title: A Prospective, Open Label, Multicenter, Partially Randomized, Safety Study of One Cycle of Promethera HepaStem in Urea Cycle Disorders (UCD) and Crigler-Najjar Syndrome (CN) Paediatric Patients.

summary:
The purpose of this study is to assess the safety and to appraise the efficacy of one cycle of Hepastem (Heterologous Human Adult Liver-derived Progenitor Cells, HHALPC) infusions in paediatric patients suffering from CN or UCD.

The study duration: 12 months starting from the day of treatment: 6 months active surveillance and 6 months observation post-infusion.

ReferenceCitation: Coppin LCF, Smets F, Ambroise J, Sokal EEM, Stephenne X. Infusion-related thrombogenesis by liver-derived mesenchymal stem cells controlled by anticoagulant drugs in 11 patients with liver-based metabolic disorders. Stem Cell Res Ther. 2020 Feb 7;11(1):51. doi: 10.1186/s13287-020-1572-7.

lead_sponsor: Cellaion SA

location_country:
France
Italy
United Kingdom
Belgium
Israel

location_facility: Rambam Medical Center, Meyer Children's Hospital

location_facility: Schneider Children's Medical Center of israel

location_facility: Ospedale Pediatrico Bambino Gesu di Roma


title: Virtue® Male Sling European Study

summary: This study is a prospective, single arm, non-randomized, multi-center clinical study that will be conducted in Europe. It is designed to assess efficacy and safety of the Virtue® Male Sling in post-prostatectomy urinary incontinence.

lead_sponsor: Coloplast A/S

location_country:
Germany
Ireland
Italy
United Kingdom
Netherlands
Belgium
Spain
France

location_facility: Fundacio Puigvert - Universitat Autonoma de Barcelona

location_facility: Centre Hospitalier Universitaire Henri Mondor


lead_sponsor: AB Science

summary: The study objective is to compare the efficacy and the safety of masitinib at 6 mg/kg/day versus placebo in the treatment of patients with Severe Persistent Asthma treated with oral corticosteroids.

location_facility: University of Debrecen Medical and Health Science Center

title: A Prospective, Multicenter, Randomised, Double-blind, Placebo-controlled, 2-parallel Groups, Phase 3 Study to Compare the Efficacy and the Safety of Masitinib at 6 mg/kg/Day Versus Placebo in the Treatment of Patients With Severe Persistent Asthma Treated With Oral Corticosteroids

location_country: India Hungary Spain Czechia Bulgaria France


title: HepZero:Heparin Free Dialysis With Evodial: A Prospective Multicenter, Open, Randomized, Controlled Clinical Study With Parallel Groups

summary: The study hypothesis is that with Evodial in patients requiring heparin free dialysis, the heparin free treatment can be performed easily (without saline flushes or blood predilution) and is at least not inferior and maybe superior to the standard care heparin free treatment in terms of clotting.

ReferenceCitation: Kim YG. Anticoagulation during haemodialysis in patients at high-risk of bleeding. Nephrology (Carlton). 2003 Oct;8 Suppl:S23-7. doi: 10.1046/j.1440-1797.8.s.3.x.

ReferenceCitation: European Best Practice Guidelines Expert Group on Hemodialysis, European Renal Association. Section V. Chronic intermittent haemodialysis and prevention of clotting in the extracorporal system. Nephrol Dial Transplant. 2002;17 Suppl 7:63-71. doi: 10.1093/ndt/17.suppl_7.63. No abstract available.

ReferenceCitation: Chanard J, Lavaud S, Maheut H, Kazes I, Vitry F, Rieu P. The clinical evaluation of low-dose heparin in haemodialysis: a prospective study using the heparin-coated AN69 ST membrane. Nephrol Dial Transplant. 2008 Jun;23(6):2003-9. doi: 10.1093/ndt/gfm888. Epub 2007 Dec 21.

ReferenceCitation: Lohr JW, Schwab SJ. Minimizing hemorrhagic complications in dialysis patients. J Am Soc Nephrol. 1991 Nov;2(5):961-75. doi: 10.1681/ASN.V25961.

ReferenceCitation: Yixiong Z, Jianping N, Yanchao L, Siyuan D. Low dose of argatroban saline flushes anticoagulation in hemodialysis patients with high risk of bleeding. Clin Appl Thromb Hemost. 2010 Aug;16(4):440-5. doi: 10.1177/1076029609334628. Epub 2009 Oct 14.

ReferenceCitation: Laville M, Dorval M, Fort Ros J, Fay R, Cridlig J, Nortier JL, Juillard L, Debska-Slizien A, Fernandez Lorente L, Thibaudin D, Franssen C, Schulz M, Moureau F, Loughraieb N, Rossignol P. Results of the HepZero study comparing heparin-grafted membrane and standard care show that heparin-grafted dialyzer is safe and easy to use for heparin-free dialysis. Kidney Int. 2014 Dec;86(6):1260-7. doi: 10.1038/ki.2014.225. Epub 2014 Jul 9.

license: publisher-specific-oa

license_normalized: publisher-specific

ReferenceCitation: Rossignol P, Dorval M, Fay R, Ros JF, Loughraieb N, Moureau F, Laville M. Rationale and design of the HepZero study: a prospective, multicenter, international, open, randomized, controlled clinical study with parallel groups comparing heparin-free dialysis with heparin-coated dialysis membrane (Evodial) versus standard care: study protocol for a randomized controlled trial. Trials. 2013 Jun 1;14:163. doi: 10.1186/1745-6215-14-163.

repository_institution: Le Centre pour la Communication Scientifique Directe - HAL - Hospices Civils de Lyon

repositories_institution: Le Centre pour la Communication Scientifique Directe - HAL - Inria

repositories_institution: Le Centre pour la Communication Scientifique Directe - HAL - Université Paris Descartes

publisher_dissemination: Springer

lead_sponsor: Baxter Healthcare Corporation

collaborators: Statistical Analysis : CIC CHU Brabois Nancy

location_country:
Poland
United Kingdom
Netherlands
Belgium
Spain
Canada
France

location_facility: Hospital Universitary German Trias i Pujol


title: An Extension Protocol for Participants of Genzyme-Sponsored Prospective, Randomized, Open-Label, Parallel-Group, Multicenter Study of MACI® for the Treatment of Symptomatic Articular Cartilage Defects of the Femoral Condyle Including the Trochlea

acronym: Extension

summary: To examine the 5-year efficacy and safety of MACI implant, compared with arthroscopic microfracture, in participants who received study treatment in the SUMMIT study for treatment of symptomatic articular cartilage defects of the knee.

lead_sponsor: Vericel Corporation

location_country:
Poland
Norway
United Kingdom
Sweden
Netherlands
Czechia
France

location_facility: Hôpital d'instruction des Armées Robert Picqué

location_facility: Regional Centre of Knee Surgery & Arthroscopy, Regional Hospital of Traumatologic Surgery

location_facility: Orthopaedic & Traumatology of Motor Organs, Independent Public Clinic Hospital

location_facility: University Medical Centre of Utrecht, Department of Orthopaedics

location_facility: University of Oxford, Nuffield Dept. of Orthopaedic Surgery

location_facility: University Hospital Na Bulovce- Department of Orthopaedic Surgery Postgraduate Medical Institute


title: An Open, Non-comparative, Post-marketing, Multi-center Study to Evaluate Efficacy and Safety of SolestaTM for the Treatment of Fecal Incontinence

summary: The study will evaluate the safety and efficacy of Solesta as an injectable bulking agent in the treatment of fecal incontinence for a total of 24 months after completed treatment.

lead_sponsor: Galderma R&D

location_country: Finland Italy Germany Norway Spain Canada France

location_facility: Klinik für Allgemein- und Vizeralchirurgie

location_facility: CHU de Rouen 1 rue Germont service de chirurgie et digestive

location_facility: University of Bari - Coloproctological Unit of Bari Policinico di Bari Piazza

location_facility: Univ. degli Studi di Padova Hospital Clinica Chirurgica


title: A Phase 2, Randomized, Open-Label Trial of GS-9256 Plus GS-9190 Alone and in Combination With Ribavirin for 28 Days in Treatment Naïve Subjects With Chronic Genotype 1 Hepatitis C Virus Infection (Protocol No. GS-US-196-0112)

summary:
This a phase 2, randomized, open-label trial of GS-9256 plus GS-9190, two oral anti HCV drugs, for 28 days with and without ribavirin (RIBA) and with pegylated interferon (PEG)/RIBA in adults with chronic Hepatitis C virus (HCV). In Part A, approximately thirty (30) subjects 18-70 years of age who meet study entry criteria will be randomized (in other words, selected at random, like flipping a coin) to one of the two treatment groups (GS-9256 plus GS-9190 or GS-9256 plus GS-9190 plus RIBA). In Part B, an additional fifteen (15) subjects will receive 75 mg GS-9256 BID plus 40 mg GS-9190 BID in combination with PEG/RIBA. After the 28-day treatment period, subjects will receive PEG/RIBA as standard of care (SOC).

Following randomization, subjects will return for a Baseline (Day 1) visit, at which time study medication will be dispensed and subjects will enter a 28 day treatment phase. During the treatment phase, subjects will receive oral study drugs twice daily for 28 days and PEG once weekly for Part B. Subjects then receive PEG/RIBA as local SOC starting on Day 28 (not provided as part of the study). Following completion of the 28-day treatment phase, subjects will be followed for approximately 72 weeks.

lead_sponsor: Gilead Sciences

location_country:
Germany
United Kingdom
Belgium
France


title: A Prospective, Unmasked Evaluation of the iStent in Patients With Primary Open-Angle Glaucoma

acronym: SY

summary: Prospective, unmasked, evaluation of the iStent in patients that have primary open-angle glaucoma (OAG). Stent implantation in one eye will be used for analysis, with medication added at 6 months, as required.

ReferenceCitation: Voskanyan L, Garcia-Feijoo J, Belda JI, Fea A, Junemann A, Baudouin C; Synergy Study Group. Prospective, unmasked evaluation of the iStent(R) inject system for open-angle glaucoma: synergy trial. Adv Ther. 2014 Feb;31(2):189-201. doi: 10.1007/s12325-014-0095-y. Epub 2014 Jan 23.

evidence: open (via page says license)

lead_sponsor: Glaukos Corporation

location_country: Germany Italy Austria Spain Armenia France


title: A Phase III Study to Compare SonoVue Guided Prostate Biopsy With Systematic Biopsy in the Detection of Prostate Malignant Lesions in Patients With Suspected Prostate Cancer

summary: This is a phase III European multicenter, open label, prospective study to assess the diagnostic accuracy of the use of SonoVue contrast agent to guide prostate biopsies in comparison with the current practice of ultrasound guided systematic biopsy. The trial will involve 15-20 European Centers.

lead_sponsor: Bracco Diagnostics, Inc

sponsor_collaborators:
Bracco Diagnostics, Inc
Bracco Imaging S.p.A.

location_country:
Germany
Italy
United Kingdom
Netherlands
Austria
Belgium
France

location_facility: Imperial College NHS Trust - Charing Cross

location_facility: Orsola-Malpighi Hospital, Urology and Radiology Unit "Malpighi"


title: Multicenter, Randomized, Double-blind, Placebo-controlled, Phase IIa Study to Evaluate the Efficacy, Safety, and Tolerability of ACT-128800, an S1P1 Receptor Agonist, Administered for 6 Weeks to Subjects With Moderate to Severe Chronic Plaque Psoriasis

summary: This study will assess the efficacy, safety, and tolerability of ACT-128800 in subjects with moderate to severe plaque psoriasis.

lead_sponsor: Actelion

location_country: Germany Hungary Austria Serbia France

location_facility: Landeskrankenhaus - Universitätsklilnikum Graz, Allgemeine Dermatologie

location_facility: University Clinic Münster Clinic and Policlinic for skin diseases

location_facility: Clinical Centre of Nis Clinic of Dermato-venerology

location_facility: Klinik für Dermatologie Venerologie und Allergologie

location_facility: Semmelweis Universtity Dept. dermato-venerology and skin oncology

location_facility: CHU de Nice-Hôpital de l'Archet 2 / Service de dermatologie

location_facility: Veszprem County Csolnoky Ferenc Hospital Dermatology Department

location_facility: Department of Dermatology University Hospital Johannes Gutenberg-University Mainz

location_facility: UNIVERSITY OF DEBRECEN Department of dermatology

location_facility: Dermatologie, Hôpital Purpan Universtité Paul Sabatier

location_facility: Departement de Pneumologie/ Unité Immunologie Clinique Allergie- Centre Hospitalier Lyon sud

location_facility: University of Szeged Department of dermatology and allergology


title: A Phase 2B, Randomised, Double-blind, Placebo-controlled, Parallel Group, Multi-centre Study to Investigate the Efficacy, Mechanism of Action, Pharmacokinetics, Safety and Tolerability of the mGluR5 Negative Allosteric Modulator ADX10059 as Monotherapy in Patients With Gastroesophageal Reflux Disease (GERD)

summary: Evaluation of the effect of ADX10059 on symptom control in patients with gastroesophageal reflux.

ReferenceCitation: Zerbib F, Bruley des Varannes S, Roman S, Tutuian R, Galmiche JP, Mion F, Tack J, Malfertheiner P, Keywood C. Randomised clinical trial: effects of monotherapy with ADX10059, a mGluR5 inhibitor, on symptoms and reflux events in patients with gastro-oesophageal reflux disease. Aliment Pharmacol Ther. 2011 Apr;33(8):911-21. doi: 10.1111/j.1365-2036.2011.04596.x. Epub 2011 Feb 14.

lead_sponsor: Addex Pharma S.A.

location_facility:
Magdeburg 13
Nantes
Madgeburg 12
Berlin
Dresden
Leuven
Wien
Gorlitz
Lyon
Bordeaux
Leipzig


title: A Phase I/II, Open Label, Multi-centre Study to Assess the Safety, Tolerability, Pharmacokinetics and Efficacy of AZD1152 in Patients With Acute Myeloid Leukaemia.

summary: The purpose of this study is to assess safety and tolerability of multiple ascending doses of AZD1152 and to assess effect of AZD1152 on the rate of complete remission in patients with relapsed acute myeloid leukaemia.

ReferenceCitation: Lowenberg B, Muus P, Ossenkoppele G, Rousselot P, Cahn JY, Ifrah N, Martinelli G, Amadori S, Berman E, Sonneveld P, Jongen-Lavrencic M, Rigaudeau S, Stockman P, Goudie A, Faderl S, Jabbour E, Kantarjian H. Phase 1/2 study to assess the safety, efficacy, and pharmacokinetics of barasertib (AZD1152) in patients with advanced acute myeloid leukemia. Blood. 2011 Dec 1;118(23):6030-6. doi: 10.1182/blood-2011-07-366930. Epub 2011 Oct 5.

lead_sponsor: AstraZeneca

location_country:
United States
Italy
Netherlands
France


title: An Open Label, Multicenter Study to Assess the Pharmacokinetics, Tolerability, and Safety of a Pediatric Formulation of Bosentan in Children With Idiopathic or Familial Pulmonary Arterial Hypertension

summary: The aim of the study is to demonstrate that the exposure to bosentan in children with idiopathic pulmonary arterial hypertension (PAH) or familial pulmonary arterial hypertension, using a pediatric formulation, is similar to that in adults with PAH and to evaluate the tolerability and safety of a pediatric formulation of bosentan in this patient population.

ReferenceCitation: Beghetti M, Haworth SG, Bonnet D, Barst RJ, Acar P, Fraisse A, Ivy DD, Jais X, Schulze-Neick I, Galie N, Morganti A, Dingemanse J, Kusic-Pajic A, Berger RM. Pharmacokinetic and clinical profile of a novel formulation of bosentan in children with pulmonary arterial hypertension: the FUTURE-1 study. Br J Clin Pharmacol. 2009 Dec;68(6):948-55. doi: 10.1111/j.1365-2125.2009.03532.x.

location_country:
Germany
Italy
United Kingdom
Netherlands
Switzerland
United States
France

location_facility: The Children's Hospital Cardiac Care Center