acronym: EINSTEIN Jr

title: Multicenter, Open-label, Active-controlled, Randomized Study to Evaluate the Efficacy and Safety of an age-and Body Weight-adjusted Rivaroxaban Regimen Compared to Standard of Care in Children With Acute Venous Thromboembolism

lead_sponsor: Bayer

ReferenceCitation: Palumbo J, Lensing AWA, Brandão LR, Hooimeijer HL, Kenet G, van Ommen H, Pap ÁF, Majumder M, Kubitza D, Thelen K, Willmann S, Prins MH, Monagle P, Male C. Anticoagulation in pediatric cancer-associated venous thromboembolism: a subgroup analysis of EINSTEIN-Jr. Blood Adv. 2022 Aug 25. pii: bloodadvances.2022008160. doi: 10.1182/bloodadvances.2022008160. [Epub ahead of print]

ReferenceCitation: Connor P, Sanchez van Kammen M, Lensing AWA, Chalmers E, Kallay K, Hege K, Simioni P, Biss T, Bajolle F, Bonnet D, Grunt S, Kumar R, Lvova O, Bhat R, Van Damme A, Palumbo J, Santamaria A, Saracco P, Payne J, Baird S, Godder K, Labarque V, Male C, Martinelli I, Morales Soto M, Motwani J, Shah S, Hooimeijer HL, Prins MH, Kubitza D, Smith WT, Berkowitz SD, Pap AF, Majumder M, Monagle P, Coutinho JM. Safety and efficacy of rivaroxaban in pediatric cerebral venous thrombosis (EINSTEIN-Jr CVT). Blood Adv. 2020 Dec 22;4(24):6250-6258. doi: 10.1182/bloodadvances.2020003244.

ReferenceCitation: Thom K, Lensing AWA, Nurmeev I, Bajolle F, Bonnet D, Kenet G, Massicotte MP, Karakas Z, Palumbo JS, Saracco P, Amedro P, Chain J, Chan AK, Ikeyama T, Lam JCM, Gauger C, Pap ÁF, Majumder M, Kubitza D, Smith WT, Berkowitz SD, Prins MH, Monagle P, Young G, Male C. Safety and efficacy of anticoagulant therapy in pediatric catheter-related venous thrombosis (EINSTEIN-Jr CVC-VTE). Blood Adv. 2020 Oct 13;4(19):4632-4639. doi: 10.1182/bloodadvances.2020002637.

ReferenceCitation: Male C, Lensing AWA, Palumbo JS, Kumar R, Nurmeev I, Hege K, Bonnet D, Connor P, Hooimeijer HL, Torres M, Chan AKC, Kenet G, Holzhauer S, Santamaría A, Amedro P, Chalmers E, Simioni P, Bhat RV, Yee DL, Lvova O, Beyer-Westendorf J, Biss TT, Martinelli I, Saracco P, Peters M, Kállay K, Gauger CA, Massicotte MP, Young G, Pap AF, Majumder M, Smith WT, Heubach JF, Berkowitz SD, Thelen K, Kubitza D, Crowther M, Prins MH, Monagle P; EINSTEIN-Jr Phase 3 Investigators. Rivaroxaban compared with standard anticoagulants for the treatment of acute venous thromboembolism in children: a randomised, controlled, phase 3 trial. Lancet Haematol. 2020 Jan;7(1):e18-e27. doi: 10.1016/S2352-3026(19)30219-4. Epub 2019 Nov 5.

ReferenceCitation: Monagle P, Lensing AWA, Thelen K, Martinelli I, Male C, Santamaría A, Samochatova E, Kumar R, Holzhauer S, Saracco P, Simioni P, Robertson J, Grangl G, Halton J, Connor P, Young G, Molinari AC, Nowak-Göttl U, Kenet G, Kapsa S, Willmann S, Pap AF, Becka M, Twomey T, Beyer-Westendorf J, Prins MH, Kubitza D; EINSTEIN-Jr Phase 2 Investigators. Bodyweight-adjusted rivaroxaban for children with venous thromboembolism (EINSTEIN-Jr): results from three multicentre, single-arm, phase 2 studies. Lancet Haematol. 2019 Oct;6(10):e500-e509. doi: 10.1016/S2352-3026(19)30161-9. Epub 2019 Aug 13.

ReferenceCitation: Lensing AWA, Male C, Young G, Kubitza D, Kenet G, Patricia Massicotte M, Chan A, Molinari AC, Nowak-Goettl U, Pap ÁF, Adalbo I, Smith WT, Mason A, Thelen K, Berkowitz SD, Crowther M, Schmidt S, Price V, Prins MH, Monagle P. Rivaroxaban versus standard anticoagulation for acute venous thromboembolism in childhood. Design of the EINSTEIN-Jr phase III study. Thromb J. 2018 Dec 21;16:34. doi: 10.1186/s12959-018-0188-y. eCollection 2018.

location_country:
Spain
Italy
United Kingdom
United States
Belgium
Japan
Hong Kong
Portugal
Austria
Australia
Russian Federation
Argentina
Israel
Turkey
Mexico
Singapore
Sweden
Slovakia
Brazil
Canada
Switzerland
Hungary
China
Germany
Ireland
Finland
France
Netherlands

summary: The purpose of this study is to evaluate comparative efficacy and safety of rivaroxaban to standard of care in children with acute venous thromboembolism.


type: EudraCT Number

title: An Open-label Extension Study of DS-5565 for 52 Weeks in Pain Associated With Fibromyalgia

summary: This is an open-label study of DS-5565 in subjects who either completed participation in a preceding Phase 3 study of DS-5565 in fibromyalgia (FM); i.e. DS5565-A-E309 (NCT02146430), DS5565-A-E310 (NCT02187471), or DS5565-A-E311 (NCT02187159) or are de novo subjects. Eligible subjects will be assigned to receive open-label DS-5565 for 52 weeks. All subjects will receive DS-5565 15 mg once daily (QD) for the first three weeks of the treatment period. After three weeks, subjects may be titrated to 15 mg twice daily (BID) based on protocol-specified criteria.

study_start_date_type: Actual

status: Completed

study_completion_date_type: Actual

study_type: Interventional

primary_purpose: Treatment

enrollment_type: Actual

ReferenceType: derived

ReferenceCitation: Arnold LM, Whitaker S, Hsu C, Jacobs D, Merante D. Efficacy and safety of mirogabalin for the treatment of fibromyalgia: results from three 13-week randomized, double-blind, placebo- and active-controlled, parallel-group studies and a 52-week open-label extension study. Curr Med Res Opin. 2019 Oct;35(10):1825-1835. doi: 10.1080/03007995.2019.1629757. Epub 2019 Jul 9.

ipd_sharing: Yes

ipd_sharing_description: De-identified individual participant data (IPD) and applicable supporting clinical trial documents may be available upon request at https://vivli.org/. In cases where clinical trial data and supporting documents are provided pursuant to our company policies and procedures, Daiichi Sankyo will continue to protect the privacy of our clinical trial participants. Details on data sharing criteria and the procedure for requesting access can be found at this web address: https://vivli.org/ourmember/daiichi-sankyo/

lead_sponsor: Daiichi Sankyo, Inc.

location_country:
Serbia
Spain
United Kingdom
Lithuania
Ukraine
United States
Romania
South Africa
Czechia
Poland
Portugal
Slovenia
Austria
Australia
Russian Federation
Puerto Rico
Norway
Slovakia
Canada
Hungary
Germany
Bulgaria
Denmark
Latvia
Estonia
New Zealand
Finland
France
Chile

intervention_type: Drug

lead_sponsor_type: industriel

status_simplified: Completed


title: A Phase 2, Dose-Ranging, Randomized, Double-Blind, Placebo-Controlled Study of GS-4997 in Subjects With Pulmonary Arterial Hypertension

acronym: ARROW

summary: The primary objective of this study is to evaluate the effect of selonsertib (GS-4997) on pulmonary vascular resistance (PVR), as measured by right heart catheterization (RHC) in adults with pulmonary arterial hypertension (PAH). The study will consist of a 24-week placebo-controlled treatment period and a long-term selonsertib treatment period. Participants completing the 24-week placebo-controlled period will be eligible to receive active treatment with selonsertib in the long-term treatment period.

design_allocation: Randomized

ReferenceCitation: Rosenkranz S, Feldman J, McLaughlin VV, Rischard F, Lange TJ, White RJ, Peacock AJ, Gerhardt F, Ebrahimi R, Brooks G, Satler C, Frantz RP; ARROW Study Group. Selonsertib in adults with pulmonary arterial hypertension (ARROW): a randomised, double-blind, placebo-controlled, phase 2 trial. Lancet Respir Med. 2022 Jan;10(1):35-46. doi: 10.1016/S2213-2600(21)00032-1. Epub 2021 Aug 20.

lead_sponsor: Gilead Sciences

location_country:
Spain
Italy
United Kingdom
Netherlands
Canada
United States
France
Germany

location_facility: Washington University School of Medicine

location_facility: Vancouver General Hospital, The Lung Centre // Vancouver Coastal Health, Vancouver General Hospital

location_facility: Universitatsklinikul Giessen und Marburg GmbH

location_facility: Hopital Cardiologique-CHRU Lille, Service de cardiologie

location_facility: Klinik und Poliklinik fur Innere Medizin II, Universitatsklinikum Regensburg

location_facility: Victoria Hospital - London Health Sciences Centre

location_facility: LA Biomedical Research Institute Harbor-UCLA Medical Center

location_facility: Scottish Pulmonary Vascular Unit, Golden Jubilee National Hospital

location_facility: Mary M. Parkes Center // University of Rochester Medical Center

location_facility: University of South Alabama Medical Center

location_facility: CHU de Bicetre, Service de Pneumologie-Reanimation Respiratoire

location_facility: Hospital Universitario Doce (12) de Octubre

location_facility: VA Greater Los Angeles Healthcare System

location_facility: University of Colorado Cardiac and Vascular Center, Anschutz Inpatient Pavillion

location_facility: CHU de Grenoble Clinique Universitaire de Pneumologie

location_facility: Universita "Sapienza"-Azienda Policlinico Umberto 1

location_facility: Klinik III fur Innere Medizin, Herzzentrum Uniklinik Koln

location_facility: Clinical Research Facility, Royal Hallamshrie Hospital

location_facility: Institut Universitaire de caridologie et de pneumologie de Quebee (IUCPQ)

location_facility: University of Arizona Clinical and Translational Science (CATS) Research Center


title: Pilot Testing of SAGIT in Patients With Acromegaly in Clinical Practice

acronym: SAGIT Pilot

summary:
The purpose of this pilot study is to test SAGIT (Signs and symptoms - Associated comorbidities - GH concentration level - IGF-1 - Tumour). SAGIT is a Clinician-Reported Outcomes (ClinROs) tool developed to describe patients with acromegaly. This study will determine the potential use of a finalised operational version for patient classification in clinical practice and studies.

In addition, this study intends to carry out a qualitative evaluation of the acceptability of SAGIT by the practicing endocrinologist in terms of relevance, ease of use, applicability and usefulness of the tool in practice.

study_type: Observational

time_perspective: Cross-Sectional

ReferenceCitation: Giustina A, Bevan JS, Bronstein MD, Casanueva FF, Chanson P, Petersenn S, Thanh XM, Sert C, Houchard A, Guillemin I, Melmed S; SAGIT Investigator Group. SAGIT®: clinician-reported outcome instrument for managing acromegaly in clinical practice--development and results from a pilot study. Pituitary. 2016 Feb;19(1):39-49. doi: 10.1007/s11102-015-0681-2.

lead_sponsor: Ipsen

location_country:
Brazil
Spain
Italy
United Kingdom
United States
France
Germany

location_facility: NW Pituitary Center and Neurological Surgery

location_facility: Azienda Ospedaliera Città della Salute e della Scienza

location_facility: Universitary Hospital Virgen de la Vitoria

location_facility: Praxisgemeinschaft an der Kaisereiche, Wilhelm-Hauff-Straße 21

location_facility: Azienda Ospedaliera Spedali Civil di Brescia

intervention_type: Other


status: Terminated

title: A Phase 2, Multicenter, Single-arm Study of Moxetumomab Pasudotox in Pediatric Subjects With Relapsed or Refractory Pediatric Acute Lymphoblastic Leukemia (pALL) or Lymphoblastic Lymphoma of B-cell Origin

lead_sponsor: MedImmune LLC

source: euctr

ReferenceType: background

ReferenceCitation: Mussai F, Campana D, Bhojwani D, Stetler-Stevenson M, Steinberg SM, Wayne AS, Pastan I. Cytotoxicity of the anti-CD22 immunotoxin HA22 (CAT-8015) against paediatric acute lymphoblastic leukaemia. Br J Haematol. 2010 Aug;150(3):352-8. doi: 10.1111/j.1365-2141.2010.08251.x. Epub 2010 Jun 7.

location_country:
Spain
Italy
United Kingdom
Australia
Canada
United States
France
Netherlands

summary: The primary objective of this study is to evaluate the efficacy of moxetumomab pasudotox in pediatric participants with relapsed or refractory B-cell acute lymphoblastic leukemia (ALL) or B-cell lymphoblastic lymphoma.

status_simplified: Unknown


title: A Combined Phase IIa / IIb Study of the Efficacy, Safety, and Tolerability of Repeated Topical Doses of Regorafenib Eye Drops, in Treatment-naïve Subjects With Neovascular Age Related Macular Degeneration

acronym: DREAM

summary:
Part A (Phase IIa):

Primary objectives:

The study part A is designed to investigate whether the use of regorafenib eye drops can help patients with neovascular (wet) Age-Related Macular Degeneration (wAMD) to see better after 4 weeks and 12 weeks after inclusion into this study.

Secondary objectives:

The study will also evaluate the safety and tolerability of the regorafenib eye drops.

Part B (Phase IIb):

Primary objectives:

The study part B is designed to investigate:

how often the regorafenib eye drops need to be given per day
whether the use of regorafenib eye drops can help patients with neovascular (wet) Age-Related Macular Degeneration (wAMD) to see better after 4 weeks and 12 weeks after inclusion into this study.

Secondary objectives:

The study will also evaluate how the different dosings of regorafenib eye drops affect patients vision, the safety and the tolerability.

location_country:
Hong Kong
Spain
Italy
Austria
Australia
Canada
Czech Republic
Switzerland
Hungary
Korea, Republic of
United States
Chile
France
Israel
Colombia
Japan
Germany
Slovakia

intervention_type: Procedure


title: An Open-label, Multicenter, Phase IIIb Study Assessing the Long-term Efficacy and Safety of AOP2014 and Standard First Line Treatment (BAT) in Patients With Polycythemia Vera Who Previously Participated in the PROUD-PV Study

acronym: CONTI-PV

summary:
Polycythemia Vera (PV) is a disease of bone marrow stem cells that manifests in a drastic increase of red blood cells and frequently also of white blood cells. The "thickening" of the blood in relation with a modified function of the cells has several consequences like increased blood pressure, pruritus of the skin, fatigue, disturbed blood circulation in the brain as well as fingers and toes and an increased risk of arterial and venous thrombosis (thrombosis is the formation of a blood clot in a vessel); like stroke, cardiac infarction, deep vein thrombosis in the legs. In case of a strong increase of platelets there is an additional risk of bleedings. As the disease progresses the size of spleen and liver increased in most cases and the bone marrow shows signs of fibrosis. In some cases of PV a progression at a later time point to a leukemia (increased formation of white blood cells) can occur.

The aim of this study is to show that the study drug AOP2014 (pegylated proline interferon alpha-2b) has the long term efficacy and safety in controlling the disease. A comparison arm is receiving best available therapy as selected by the investigator. Response to the treatment is measured by several blood parameters as well as size of the spleen.

Interferon-alpha has been shown to be effective in controlling the blood parameters by immunologically influencing the blood building cells. This can lead to a suppression of the disease-causing stem cells and help healthy stem cells to proliferate. Through this mechanism it is possible that Interferon-alpha can avoid long-term damaging effects of the disease.

primary_purpose: Other

lead_sponsor: AOP Orphan Pharmaceuticals AG

location_country:
Poland
Spain
Austria
Russian Federation
Ukraine
Hungary
France
Romania
Germany
Bulgaria
Slovakia
Czechia

location_facility: Dnipropetrovsk City Multispecialty Clinical Hospital #4

location_facility: Multiprofile Hospital for Active Treatment - Hristo Botev, Vratsa, First Department of Internal Medicine

location_facility: University Hospital Carl Gustav Carus, Medical Clinic and Polyclinic I

location_facility: National Research Center for Radiation Medicine, Institute of Clinical Radiology

location_facility: Cherkasy Regional Oncology Center, Regional Treatment and Diagnostics Hematology Center

location_facility: Samara Kalinin Regional Clinical Hospital

location_facility: Bekes County Pandy Kalman Hospital, 1st Department of Medicine, Hematology

location_facility: Emergency Clinical County Hospital Brasov

location_facility: Nicolaus Copernicus Municipal Specialist Hospital

location_facility: Institute of Hematology and Transfusion Medicine

location_facility: Independent Public Teaching Hospital No.1 in Lublin

location_facility: Multiprofile Hospital for Active Treatment "Sveta Marina", Varna

location_facility: Specialized Hospital for Active Treatment of Hematological Diseases

location_facility: Aachen University Hospital, Medical Clinic IV

location_facility: Fryderyk Chopin Provincial Specialized Hospital

location_facility: O.F. Herbachevskyi Regional Clinical Hospital

location_facility: University of Szeged, Albert Szent-Gyorgyi Clinical Center, Koranyi fasor 6

location_facility: University Multiprofile Hospital for Active Treatment "Sveti Georgi", Plovdiv

location_facility: University Hospital Bonn, Center for Internal Medicine, Medical Clinic and Outpatient Clinic III

location_facility: University Hospital with Outpatient Clinic F.D. Roosevelt

location_facility: Institute of Blood Pathology and Transfusion Medicine

location_facility: St Istvan and St Laszlo Hospital of Budapest

location_facility: Saint Cyril and Metod University Hospital Bratislava


title: A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study of ISIS 304801 Administered Subcutaneously to Patients With Familial Chylomicronemia Syndrome (FCS)

summary: The purpose of this study is to evaluate the efficacy and safety of volanesorsen given for 52 weeks in participants with Familial Chylomicronemia Syndrome

ReferenceType: result

ReferenceCitation: Witztum JL, Gaudet D, Freedman SD, Alexander VJ, Digenio A, Williams KR, Yang Q, Hughes SG, Geary RS, Arca M, Stroes ESG, Bergeron J, Soran H, Civeira F, Hemphill L, Tsimikas S, Blom DJ, O'Dea L, Bruckert E. Volanesorsen and Triglyceride Levels in Familial Chylomicronemia Syndrome. N Engl J Med. 2019 Aug 8;381(6):531-542. doi: 10.1056/NEJMoa1715944.

ReferenceCitation: Hegele RA, Berberich AJ, Ban MR, Wang J, Digenio A, Alexander VJ, D'Erasmo L, Arca M, Jones A, Bruckert E, Stroes ES, Bergeron J, Civeira F, Witztum JL, Gaudet D. Clinical and biochemical features of different molecular etiologies of familial chylomicronemia. J Clin Lipidol. 2018 Jul - Aug;12(4):920-927.e4. doi: 10.1016/j.jacl.2018.03.093. Epub 2018 Apr 4.

ReferenceCitation: Digenio A, Dunbar RL, Alexander VJ, Hompesch M, Morrow L, Lee RG, Graham MJ, Hughes SG, Yu R, Singleton W, Baker BF, Bhanot S, Crooke RM. Antisense-Mediated Lowering of Plasma Apolipoprotein C-III by Volanesorsen Improves Dyslipidemia and Insulin Sensitivity in Type 2 Diabetes. Diabetes Care. 2016 Aug;39(8):1408-15. doi: 10.2337/dc16-0126. Epub 2016 Jun 6.

ipd_sharing: No

lead_sponsor: Ionis Pharmaceuticals, Inc.

location_country:
Brazil
Spain
Italy
United Kingdom
Netherlands
Canada
Hungary
United States
France
Israel
South Africa
Germany


title: A Phase 1b Study of ABT-199 (GDC-0199) in Combination With Azacitidine or Decitabine in Treatment-Naive Subjects With Acute Myelogenous Leukemia Who Are Greater Than or Equal to 60 Years of Age and Who Are Not Eligible for Standard Induction Therapy

summary: This is a Phase 1b, open-label, non-randomized, multicenter study to evaluate the safety and pharmacokinetics of orally administered venetoclax (ABT-199) combined with decitabine or azacitidine and the preliminary efficacy of these combinations. In addition, there is a drug-drug interaction (DDI) sub-study only at a single site, to assess the pharmacokinetics and safety of venetoclax (ABT-199) in combination with posaconazole.

design_allocation: Non-Randomized

ReferenceCitation: Badawi M, Chen X, Marroum P, Suleiman AA, Mensing S, Koenigsdorfer A, Schiele JT, Palenski T, Samineni D, Hoffman D, Menon R, Salem AH. Bioavailability Evaluation of Venetoclax Lower-Strength Tablets and Oral Powder Formulations to Establish Interchangeability with the 100 mg Tablet. Clin Drug Investig. 2022 Aug;42(8):657-668. doi: 10.1007/s40261-022-01172-4. Epub 2022 Jul 13.

ReferenceCitation: Konopleva M, Thirman MJ, Pratz KW, Garcia JS, Recher C, Pullarkat V, Kantarjian HM, DiNardo CD, Dail M, Duan Y, Chyla B, Potluri J, Miller CL, Wei AH. Impact of FLT3 Mutation on Outcomes after Venetoclax and Azacitidine for Patients with Treatment-Naïve Acute Myeloid Leukemia. Clin Cancer Res. 2022 Jul 1;28(13):2744-2752. doi: 10.1158/1078-0432.CCR-21-3405.

ReferenceCitation: Pollyea DA, DiNardo CD, Arellano ML, Pigneux A, Fiedler W, Konopleva M, Rizzieri DA, Smith BD, Shinagawa A, Lemoli RM, Dail M, Duan Y, Chyla B, Potluri J, Miller CL, Kantarjian HM. Impact of Venetoclax and Azacitidine in Treatment-Naïve Patients with Acute Myeloid Leukemia and IDH1/2 Mutations. Clin Cancer Res. 2022 Jul 1;28(13):2753-2761. doi: 10.1158/1078-0432.CCR-21-3467.

ReferenceCitation: DiNardo CD, Pratz K, Pullarkat V, Jonas BA, Arellano M, Becker PS, Frankfurt O, Konopleva M, Wei AH, Kantarjian HM, Xu T, Hong WJ, Chyla B, Potluri J, Pollyea DA, Letai A. Venetoclax combined with decitabine or azacitidine in treatment-naive, elderly patients with acute myeloid leukemia. Blood. 2019 Jan 3;133(1):7-17. doi: 10.1182/blood-2018-08-868752. Epub 2018 Oct 25.

ReferenceCitation: DiNardo CD, Pratz KW, Letai A, Jonas BA, Wei AH, Thirman M, Arellano M, Frattini MG, Kantarjian H, Popovic R, Chyla B, Xu T, Dunbar M, Agarwal SK, Humerickhouse R, Mabry M, Potluri J, Konopleva M, Pollyea DA. Safety and preliminary efficacy of venetoclax with decitabine or azacitidine in elderly patients with previously untreated acute myeloid leukaemia: a non-randomised, open-label, phase 1b study. Lancet Oncol. 2018 Feb;19(2):216-228. doi: 10.1016/S1470-2045(18)30010-X. Epub 2018 Jan 12.

ReferenceCitation: Agarwal SK, DiNardo CD, Potluri J, Dunbar M, Kantarjian HM, Humerickhouse RA, Wong SL, Menon RM, Konopleva MY, Salem AH. Management of Venetoclax-Posaconazole Interaction in Acute Myeloid Leukemia Patients: Evaluation of Dose Adjustments. Clin Ther. 2017 Feb;39(2):359-367. doi: 10.1016/j.clinthera.2017.01.003. Epub 2017 Feb 1.

lead_sponsor: AbbVie

location_country:
Australia
United States
Germany
France

location_facility: Universitaetsklinikum Leipzig /ID# 130346

location_facility: The University of Chicago Medical Center /ID# 128742

location_facility: Duplicate_Hopital Universitaire Purpan /ID# 134389

location_facility: University of Texas MD Anderson Cancer Center /ID# 127860

location_facility: Emory Midtown Infectious Disease Clinic /ID# 129715

location_facility: Duplicate_Klinikum Rechts der Isar /ID# 130347

location_facility: Columbia University Medical Center /ID# 130289

location_facility: University of California, Davis Comprehensive Cancer Center /ID# 129719

location_facility: University of Texas MD Anderson Cancer Center /ID# 141581

location_facility: Dana-Farber Cancer Institute /ID# 127857

location_facility: Northwestern University Feinberg School of Medicine /ID# 128741

location_facility: Univ of Colorado Cancer Center /ID# 127859

location_facility: Universitaetsklinikum Carl Gustav Carus an der TU Dresden /ID# 130342


title: A Randomized, Double Blind Placebo-controlled Study to Assess the Safety, Tolerability, Pharmacokinetics, and Preliminary Pharmacodynamics of Single and Multiple Ascending Doses of QBW251 in Healthy Subjects and Multiple Doses in Cystic Fibrosis Patients

lead_sponsor: Novartis Pharmaceuticals

ReferenceCitation: Kazani S, Rowlands DJ, Bottoli I, Milojevic J, Alcantara J, Jones I, Kulmatycki K, Machineni S, Mostovy L, Nicholls I, Nick JA, Rowe SM, Simmonds NJ, Vegesna R, Verheijen J, Danahay H, Gosling M, Ayalavajjala PS, Salman M, Strieter R. Safety and efficacy of the cystic fibrosis transmembrane conductance regulator potentiator icenticaftor (QBW251). J Cyst Fibros. 2021 Mar;20(2):250-256. doi: 10.1016/j.jcf.2020.11.002. Epub 2020 Dec 6.

location_country:
Romania
United Kingdom
Ireland
United States
France
Belgium
Germany

summary: This study is designed to assess the safety, tolerability, pharmacokinetics and preliminary pharmacodynamics (proof of concept) of QBW251 in healthy subjects and cystic fibrosis patients following single and multiple doses. This first-in-human and proof of concept study will consist of 4 parts, with Parts 1 and 2 in healthy volunteers and Parts 3 and 4 in cystic fibrosis patients.


title: 10-day Decitabine Versus Conventional Chemotherapy ("3+7") Followed by Allografting in AML Patients ≥ 60 Years: a Randomized Phase III Study of the EORTC Leukemia Group, CELG, GIMEMA and German MDS Study Group

summary:
Older patients with acute myeloid leukemia (AML) have a small (< 10%) chance of long-term survival. Despite the treatment of elderly AML patients with intensive chemotherapy, the survival has not been improved during the last decades.

The purpose of this study is to determine whether frontline therapy with a 10-day decitabine schedule provides a better survival than standard intensive combination chemotherapy in elderly AML patients (>= 60 years).

status: Active, not recruiting

study_completion_date_type: Anticipated

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

location_country: Croatia Portugal Italy Netherlands Lithuania France Belgium Germany Bulgaria

location_facility: Amedeo Avogadro University of Eastern Piedmont-Ospedale Maggiore della Carita

location_facility: National Specialized Hospital for Active Treatment of Haematological Diseases

location_facility: Assistance Publique - Hôpitaux de Paris - Hôpital Saint Antoine

location_facility: Azienda Ospedaliero - Universitaria Policlinico di Modena

location_facility: Azienda Ospedaliero-Universitaria "Santa Maria della Misericordia" di Udine

location_facility: Klinikum Der Phillips - Universität Marburg

location_facility: Instituto Regina Elena / Instituti Fisioterapici Ospitalieri

location_facility: Azienda Ospedaliero Universitaria - Ospedali Riuniti

location_facility: Azienda Ospedallera Universitaria - Policlinico Tor Vergata

location_facility: Universitaetklinikum Halle - Martin Luther Universitaet - Universitaetsklinikum Halle (Saale)

location_facility: Helios Kliniken - Helios Klinikum Berlin-Buch

location_facility: Universitätsklinikum Aachen AÖR - Medizinische Fakultät der RWTH

location_facility: AUSL Romagna - Ospedale Infermi di Rimini

location_facility: Azienda Ospedaliera Città della Salute e della Scienza di Torino - Ospedale Molinette

location_facility: Radboud University Medical Center Nijmegen

location_facility: AUSL Romagna - Ospedale Santa Maria dell Croci

location_facility: Universita Degli Studi Di Bari - Policlinico

location_facility: Universita Degli Studi Di Roma La Sapienza - Ospedale Sant'Andrea

location_facility: Clinica Ematologica dell'Universita di Roma La Sapienza

location_facility: Vilnius University Hospital Santariskiu Clinics

location_facility: Universitaet Rostock - Medizinische Fakultaet

location_facility: Universitaetsklinikum Tuebingen-Uni Kliniken Berg

status_simplified: Ongoing


type: Other Identifier

type: Registry Identifier

title: An Open-label Single-arm Multicentre Non-controlled Phase 3 a Trial Investigating Safety and Efficacy of Nonacog Beta Pegol (N9-GP) in Prophylaxis and Treatment of Bleeding Episodes in Previously Untreated Patients With Haemophilia B (FIX Activity Below or Equal to 2 Percent)

summary: This trial is conducted globally. The aim of the trial is to investigate the safety and efficacy of nonacog beta pegol (N9-GP) in previously untreated patients with Haemophilia B.

enrollment_type: Anticipated

ReferenceCitation: Chan AK, Alamelu J, Barnes C, Chuansumrit A, Garly ML, Meldgaard RM, Young G. Nonacog beta pegol (N9-GP) in hemophilia B: First report on safety and efficacy in previously untreated and minimally treated patients. Res Pract Thromb Haemost. 2020 Jul 29;4(7):1101-1113. doi: 10.1002/rth2.12412. eCollection 2020 Oct.

lead_sponsor: Novo Nordisk A/S

location_country:
Taiwan
Spain
Italy
Austria
Australia
Netherlands
Argentina
Canada
Thailand
United Kingdom
Algeria
United States
France
Israel
Japan
Malaysia


title: An Open-label Single-arm Multicentre Non-controlled Phase 3a Trial Investigating Safety and Efficacy of N8-GP in Prophylaxis and Treatment of Bleeding Episodes in Previously Untreated Paediatric Patients With Severe Haemophilia A

location_country:
Taiwan
Spain
Greece
Austria
Australia
Italy
Argentina
Thailand
Algeria
United States
France
Romania
Japan
Germany

summary: This trial is conducted globally. The aim of the trial is to investigate the safety and efficacy of turoctocog alfa pegol (N8-GP) in previously untreated patients (PUPs) with haemophilia A.


title: A Randomized, Double-Blind, Phase 2 Study of Ruxolitinib or Placebo in Combination With Capecitabine in Subjects With Advanced or Metastatic HER2-Negative Breast Cancer

summary: This was a randomized, double-blind, placebo-controlled phase 2 clinical trial comparing the overall survival of women with advanced or metastatic HER2-negative breast cancer who received treatment with capecitabine in combination with ruxolitinib versus those who received treatment with capecitabine alone.

lead_sponsor: Incyte Corporation

location_country:
Portugal
Spain
Italy
United Kingdom
United States
France


title: A Phase II, Multicenter, Single-Arm Study of Atezolizumab in Patients With Locally Advanced or Metastatic Urothelial Bladder Cancer

lead_sponsor: Hoffmann-La Roche

ReferenceCitation: Holm JS, Funt SA, Borch A, Munk KK, Bjerregaard AM, Reading JL, Maher C, Regazzi A, Wong P, Al-Ahmadie H, Iyer G, Tamhane T, Bentzen AK, Herschend NO, De Wolf S, Snyder A, Merghoub T, Wolchok JD, Nielsen M, Rosenberg JE, Bajorin DF, Hadrup SR. Neoantigen-specific CD8 T cell responses in the peripheral blood following PD-L1 blockade might predict therapy outcome in metastatic urothelial carcinoma. Nat Commun. 2022 Apr 11;13(1):1935. doi: 10.1038/s41467-022-29342-0.

ReferenceCitation: Shemesh CS, Chan P, Legrand FA, Shames DS, Das Thakur M, Shi J, Bailey L, Vadhavkar S, He X, Zhang W, Bruno R. Pan-cancer population pharmacokinetics and exposure-safety and -efficacy analyses of atezolizumab in patients with high tumor mutational burden. Pharmacol Res Perspect. 2020 Dec;8(6):e00685. doi: 10.1002/prp2.685.

ReferenceCitation: Teo MY, Seier K, Ostrovnaya I, Regazzi AM, Kania BE, Moran MM, Cipolla CK, Bluth MJ, Chaim J, Al-Ahmadie H, Snyder A, Carlo MI, Solit DB, Berger MF, Funt S, Wolchok JD, Iyer G, Bajorin DF, Callahan MK, Rosenberg JE. Alterations in DNA Damage Response and Repair Genes as Potential Marker of Clinical Benefit From PD-1/PD-L1 Blockade in Advanced Urothelial Cancers. J Clin Oncol. 2018 Jun 10;36(17):1685-1694. doi: 10.1200/JCO.2017.75.7740. Epub 2018 Feb 28.

ReferenceCitation: Perez-Gracia JL, Loriot Y, Rosenberg JE, Powles T, Necchi A, Hussain SA, Morales-Barrera R, Retz MM, Niegisch G, Durán I, Théodore C, Grande E, Shen X, Wang J, Nelson B, Derleth CL, van der Heijden MS. Atezolizumab in Platinum-treated Locally Advanced or Metastatic Urothelial Carcinoma: Outcomes by Prior Number of Regimens. Eur Urol. 2018 Mar;73(3):462-468. doi: 10.1016/j.eururo.2017.11.023. Epub 2017 Dec 20. Erratum in: Eur Urol. 2019 Mar;75(3):e82-e83.

ReferenceCitation: Necchi A, Joseph RW, Loriot Y, Hoffman-Censits J, Perez-Gracia JL, Petrylak DP, Derleth CL, Tayama D, Zhu Q, Ding B, Kaiser C, Rosenberg JE. Atezolizumab in platinum-treated locally advanced or metastatic urothelial carcinoma: post-progression outcomes from the phase II IMvigor210 study. Ann Oncol. 2017 Dec 1;28(12):3044-3050. doi: 10.1093/annonc/mdx518.

ReferenceCitation: Snyder A, Nathanson T, Funt SA, Ahuja A, Buros Novik J, Hellmann MD, Chang E, Aksoy BA, Al-Ahmadie H, Yusko E, Vignali M, Benzeno S, Boyd M, Moran M, Iyer G, Robins HS, Mardis ER, Merghoub T, Hammerbacher J, Rosenberg JE, Bajorin DF. Contribution of systemic and somatic factors to clinical response and resistance to PD-L1 blockade in urothelial cancer: An exploratory multi-omic analysis. PLoS Med. 2017 May 26;14(5):e1002309. doi: 10.1371/journal.pmed.1002309. eCollection 2017 May.

ReferenceCitation: Balar AV, Galsky MD, Rosenberg JE, Powles T, Petrylak DP, Bellmunt J, Loriot Y, Necchi A, Hoffman-Censits J, Perez-Gracia JL, Dawson NA, van der Heijden MS, Dreicer R, Srinivas S, Retz MM, Joseph RW, Drakaki A, Vaishampayan UN, Sridhar SS, Quinn DI, Durán I, Shaffer DR, Eigl BJ, Grivas PD, Yu EY, Li S, Kadel EE 3rd, Boyd Z, Bourgon R, Hegde PS, Mariathasan S, Thåström A, Abidoye OO, Fine GD, Bajorin DF; IMvigor210 Study Group. Atezolizumab as first-line treatment in cisplatin-ineligible patients with locally advanced and metastatic urothelial carcinoma: a single-arm, multicentre, phase 2 trial. Lancet. 2017 Jan 7;389(10064):67-76. doi: 10.1016/S0140-6736(16)32455-2. Epub 2016 Dec 8. Erratum in: Lancet. 2017 Aug 26;390(10097):848.

ReferenceCitation: Rosenberg JE, Hoffman-Censits J, Powles T, van der Heijden MS, Balar AV, Necchi A, Dawson N, O'Donnell PH, Balmanoukian A, Loriot Y, Srinivas S, Retz MM, Grivas P, Joseph RW, Galsky MD, Fleming MT, Petrylak DP, Perez-Gracia JL, Burris HA, Castellano D, Canil C, Bellmunt J, Bajorin D, Nickles D, Bourgon R, Frampton GM, Cui N, Mariathasan S, Abidoye O, Fine GD, Dreicer R. Atezolizumab in patients with locally advanced and metastatic urothelial carcinoma who have progressed following treatment with platinum-based chemotherapy: a single-arm, multicentre, phase 2 trial. Lancet. 2016 May 7;387(10031):1909-20. doi: 10.1016/S0140-6736(16)00561-4. Epub 2016 Mar 4.

location_facility: The Angeles Clinic and Research Institute - W LA Office

location_facility: University Hospital Birmingham The Cancer Centre, Queen Elizabeth Hospital

location_facility: Hospital General Universitario Gregorio Marañon; Servicio de Oncologia

location_facility: University of Texas Health Science Center at San Antonio

location_facility: Institut Gustave Roussy; Oncologie Medicale

location_facility: Hospital Universitario Virgen del Rocio; Servicio de Oncologia

location_facility: Beth Israel Deaconess Medical Center, Harvard Medical School; Department of Medicine

location_facility: University Health Network; Princess Margaret Hospital; Medical Oncology Dept

location_facility: Universitätsklinikum Freiburg; Chirurgische Klinik; Abteilung Urologie

location_facility: Irccs Istituto Nazionale Dei Tumori (Int);S.C. Medicina Oncologica 2

location_facility: Klinikum rechts der Isar der TU München; Urologische Klinik und Poliklinik

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

location_facility: Institut Catala d Oncologia Hospital Duran i Reynals

location_facility: McGill University; Sir Mortimer B Davis Jewish General Hospital; Oncology

location_facility: Dana Farber Cancer Inst. ; Dept. of Medical Oncology

location_facility: Mount Sinai School of Medicine - Tisch Cancer Institute

location_facility: Hospital Ramon y Cajal; Servicio de Oncologia

location_facility: Kimmel Cancer Center Thomas Jefferson University

location_facility: University of Michigan Comprehensive Cancer Center

location_facility: Clinica Universitaria de Navarra; Servicio de Oncologia

location_facility: Charité - Universitätsmedizin Berlin; CC 8: Chirurgische Medizin; Klinik für Urologie

location_facility: Indiana University Health; Goshen Center for Cancer Care

location_facility: Virginia Oncology Associates - Lake Wright Cancer Center

location_facility: Willamette Valley Cancer Ctr - 520 Country Club

location_facility: The Ottawa Hospital Cancer Centre; Oncology

location_facility: University of Chicago; Hematology/Oncology

location_facility: Kaiser Permanente; Oncology Clinical Trials

location_facility: The Clatterbridge Cancer Centre NHS Foundation Trust

location_facility: Universitätsklinikum Hamburg-Eppendorf Onkologisches Zentrum Medizinische Klinik II

location_facility: Royal Marsden Hospital; Dept of Medical Oncology

location_facility: Universitätsklinikum Düsseldorf; Urologische Klinik

location_facility: Azienda USL8 Arezzo-Presidio Ospedaliero 1 San Donato;U.O.C. Oncologia

location_facility: Sarah Cannon Cancer Center - Tennessee Oncology, Pllc

location_facility: Hospital Universitario 12 de Octubre; Servicio de Oncologia

location_facility: Urology Cancer Center & GU Research Network

location_facility: Georgetown University Medical Center Lombardi Cancer Center

location_facility: The Netherlands Cancer Institute - Antoni Van Leeuwenhoekziekenhuis

summary: This Phase II, single-arm study is designed to evaluate the effect of atezolizumab treatment in participants with locally advanced or metastatic urothelial bladder cancer. Participants will be enrolled into 1 of 2 cohorts. Cohort 1 will consist of participants who are treatment-naïve and ineligible for cisplatin-containing chemotherapy. The results of Cohort 1 are reported separately (NCT02951767). Cohort 2 (reported here) will contain participants who have progressed during or following a prior platinum-based chemotherapy regimen. Participants in both cohorts will be given a 1200 milligrams (mg) intravenous (IV) dose of atezolizumab on Day 1 of 21-day cycles. Treatment of participants in Cohort 1 will continue until disease progression per Response Evaluation Criteria in Solid Tumors Version 1.1 (RECIST v1.1) or unmanageable toxicity. Treatment of participants in Cohort 2 will continue until loss of clinical benefit or unmanageable toxicity.


acronym: CheckMate 141: CHECKpoint pathway and nivoluMAb clinical Trial Evaluation 141

title: An Open Label, Randomized Phase 3 Clinical Trial of Nivolumab vs Therapy of Investigator's Choice in Recurrent or Metastatic Platinum-refractory Squamous Cell Carcinoma of the Head and Neck (SCCHN)

lead_sponsor: Bristol-Myers Squibb

ReferenceCitation: Yen CJ, Kiyota N, Hanai N, Takahashi S, Yokota T, Iwae S, Shimizu Y, Hong RL, Goto M, Kang JH, Li WSK, Ferris RL, Gillison M, Endo T, Jayaprakash V, Tahara M. Two-year follow-up of a randomized phase III clinical trial of nivolumab vs. the investigator's choice of therapy in the Asian population for recurrent or metastatic squamous cell carcinoma of the head and neck (CheckMate 141). Head Neck. 2020 Oct;42(10):2852-2862. doi: 10.1002/hed.26331. Epub 2020 Jun 24.

ReferenceCitation: Saba NF, Blumenschein G Jr, Guigay J, Licitra L, Fayette J, Harrington KJ, Kiyota N, Gillison ML, Ferris RL, Jayaprakash V, Li L, Brossart P. Nivolumab versus investigator's choice in patients with recurrent or metastatic squamous cell carcinoma of the head and neck: Efficacy and safety in CheckMate 141 by age. Oral Oncol. 2019 Sep;96:7-14. doi: 10.1016/j.oraloncology.2019.06.017. Epub 2019 Jul 3.

ReferenceCitation: Cocks K, Contente M, Simpson S, DeRosa M, Taylor FC, Shaw JW. A Q-TWiST Analysis Comparing Nivolumab and Therapy of Investigator's Choice in Patients with Recurrent/Metastatic Platinum-Refractory Squamous Cell Carcinoma of the Head and Neck. Pharmacoeconomics. 2019 Aug;37(8):1041-1047. doi: 10.1007/s40273-019-00798-1.

ReferenceCitation: Pai SI, Faivre S, Licitra L, Machiels JP, Vermorken JB, Bruzzi P, Gruenwald V, Giglio RE, Leemans CR, Seiwert TY, Soulieres D. Comparative analysis of the phase III clinical trials of anti-PD1 monotherapy in head and neck squamous cell carcinoma patients (CheckMate 141 and KEYNOTE 040). J Immunother Cancer. 2019 Apr 3;7(1):96. doi: 10.1186/s40425-019-0578-0.

ReferenceCitation: Ferris RL, Blumenschein G Jr, Fayette J, Guigay J, Colevas AD, Licitra L, Harrington KJ, Kasper S, Vokes EE, Even C, Worden F, Saba NF, Docampo LCI, Haddad R, Rordorf T, Kiyota N, Tahara M, Lynch M, Jayaprakash V, Li L, Gillison ML. Nivolumab vs investigator's choice in recurrent or metastatic squamous cell carcinoma of the head and neck: 2-year long-term survival update of CheckMate 141 with analyses by tumor PD-L1 expression. Oral Oncol. 2018 Jun;81:45-51. doi: 10.1016/j.oraloncology.2018.04.008. Epub 2018 Apr 17.

ReferenceCitation: Kiyota N, Hasegawa Y, Takahashi S, Yokota T, Yen CJ, Iwae S, Shimizu Y, Hong RL, Goto M, Kang JH, Sum Kenneth Li W, Ferris RL, Gillison M, Namba Y, Monga M, Lynch M, Tahara M. A randomized, open-label, Phase III clinical trial of nivolumab vs. therapy of investigator's choice in recurrent squamous cell carcinoma of the head and neck: A subanalysis of Asian patients versus the global population in checkmate 141. Oral Oncol. 2017 Oct;73:138-146. doi: 10.1016/j.oraloncology.2017.07.023. Epub 2017 Sep 1.

ReferenceCitation: Harrington KJ, Ferris RL, Blumenschein G Jr, Colevas AD, Fayette J, Licitra L, Kasper S, Even C, Vokes EE, Worden F, Saba NF, Kiyota N, Haddad R, Tahara M, Grünwald V, Shaw JW, Monga M, Lynch M, Taylor F, DeRosa M, Morrissey L, Cocks K, Gillison ML, Guigay J. Nivolumab versus standard, single-agent therapy of investigator's choice in recurrent or metastatic squamous cell carcinoma of the head and neck (CheckMate 141): health-related quality-of-life results from a randomised, phase 3 trial. Lancet Oncol. 2017 Aug;18(8):1104-1115. doi: 10.1016/S1470-2045(17)30421-7. Epub 2017 Jun 23.

ReferenceCitation: Ferris RL, Blumenschein G Jr, Fayette J, Guigay J, Colevas AD, Licitra L, Harrington K, Kasper S, Vokes EE, Even C, Worden F, Saba NF, Iglesias Docampo LC, Haddad R, Rordorf T, Kiyota N, Tahara M, Monga M, Lynch M, Geese WJ, Kopit J, Shaw JW, Gillison ML. Nivolumab for Recurrent Squamous-Cell Carcinoma of the Head and Neck. N Engl J Med. 2016 Nov 10;375(19):1856-1867. Epub 2016 Oct 8.

location_country:
Taiwan
Brazil
Hong Kong
Spain
Italy
United Kingdom
Netherlands
Argentina
Canada
Switzerland
Korea, Republic of
United States
France
Japan
Germany

location_facility: Ist.Scient. Romagnolo Per Lo Studio E Cura Tumori

summary: The purpose of this study is to find out whether Nivolumab will significantly improve overall survival as compared to therapy of investigator's choice in patients with recurrent or metastatic head and neck carcinoma.


title: A Retrospective, Non-interventional, Epidemiologic Study of the Natural History of Patients With Juvenile-onset Hypophosphatasia (HPP)

summary: The purpose of this study is to characterize the natural history of HPP in patients with Juvenile-onset HPP.

time_perspective: Retrospective

lead_sponsor: Alexion Pharmaceuticals

location_country:
United Kingdom
Russian Federation
Australia
Canada
United States
France
Turkey
Netherlands


intervention_type: Biological

title: BOTOX® in the Treatment of Urinary Incontinence Due to Overactive Bladder in Patients 12 to 17 Years of Age

lead_sponsor: Allergan

ipd_sharing_description: AbbVie is committed to responsible data sharing regarding the clinical trials we sponsor. This includes access to anonymized, individual and trial-level data (analysis data sets), as well as other information (e.g., protocols and clinical study reports), as long as the trials are not part of an ongoing or planned regulatory submission. This includes requests for clinical trial data for unlicensed products and indications.

location_country:
Poland
United Kingdom
Italy
Australia
Netherlands
Canada
United States
France
South Africa
Belgium
Norway
Germany
Czechia

location_facility: Urologische Gemeinschaftspraxis /ID# 234978

location_facility: Universitair Ziekenhuis Leuven /ID# 237218

location_facility: Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wrocławiu /ID# 238166

location_facility: Medical Concierge Centrum Medyczne /ID# 235200

location_facility: Radboud Universitair Medisch Centrum /ID# 237043

location_facility: Maastricht Universitair Medisch Centrum /ID# 237678

location_facility: Universitaetsklinikum Schleswig-Holstein Campus Luebeck /ID# 234288

location_facility: Arkansas Children's Hospital /ID# 237787

location_facility: Yale New Haven Hospital - Yale School of Medicine /ID# 238222

location_facility: London Health Sciences Center /ID# 234304

location_facility: Sheffield Children's NHS Foundation Trust /ID# 237854

location_facility: Hôpital de la Mère et de l'Enfant /ID# 235227

location_facility: Royal Berkshire NHS Foundation Trust /ID# 236915

location_facility: Orlando Health-Arnold Palmer Hospital for Children Pediatric Urology /ID# 235283

location_facility: Universitair Ziekenhuis Antwerpen /ID# 237997

location_facility: The Children's Hospital at Westmead /ID# 234337

location_facility: Specjalistyczny Gabinet Lekarski /ID# 235257

location_facility: Children's Hospital Wisconsin - Milwaukee Campus /ID# 237544

location_facility: Manchester University NHS Foundation Trust /ID# 234380

location_facility: Alder Hey Children's NHS Foundation Trust /ID# 237279

location_facility: AOU Universita degli Studi della Campania Luigi Vanvitelli /ID# 237308

location_facility: Hôpitaux Pédiatriques de Nice CHU-LENVAL /ID# 235278

location_facility: Children's Hospital Colorado /ID# 237621

location_facility: Norfolk and Norwich University Hospitals NHS Foundation Trust /ID# 234819

location_facility: Associated Urologist of North Carolina /ID# 235437

location_facility: Evangelisches Krankenhaus Bielefeld /ID# 235234

location_facility: NHS Greater Glasgow and Clyde /ID# 237430

location_facility: Duplicate_CHU Bordeaux-Hopital Pellegrin /ID# 237392

summary: This study will evaluate the safety and efficacy of onabotulinumtoxinA (BOTOX®) for the treatment of urinary incontinence due to overactive bladder in pediatric patients between the ages of 12 to 17 years who have not been adequately managed with anticholinergics.


title: An Evaluation of the Efficacy and Safety of Tapentadol Oral Solution in the Treatment of Post-operative Acute Pain Requiring Opioid Treatment in Pediatric Subjects Aged From Birth to Less Than 18 Years Old

lead_sponsor: Grünenthal GmbH

ReferenceCitation: Schwartz GJ, Feld LG, Langford DJ. A simple estimate of glomerular filtration rate in full-term infants during the first year of life. J Pediatr. 1984 Jun;104(6):849-54.

ipd_sharing_description: Information available on the Grünenthal Web Site

location_country:
Croatia
Poland
Spain
United Kingdom
Hungary
France
United States
Germany
Bulgaria
Czechia

summary: The purpose of the study was to evaluate the efficacy of tapentadol oral solution, based on the total amount of supplemental opioid analgesic used over 12 hours or 24 hours after initiation of investigational medicinal product (IMP) in children and adolescents who had undergone surgery that would produce moderate to severe pain during opioid treatment.


title: MERIT-2 : Long Term, Multicenter, Single-arm, Open-label Extension Study of the MERIT-1 Study, to Assess the Safety, Tolerabilty and Efficacy of Macitentan in Subjects With Inoperable Chronic Thromboembolic Pulmonary Hypertension (CTEPH)

lead_sponsor: Actelion

location_country:
Poland
United Kingdom
Russian Federation
Mexico
Lithuania
Thailand
Switzerland
Ukraine
Hungary
France
Turkey
China
Belgium
Germany
Czechia

summary: Long-term study to evaluate if macitentan is safe, tolerable and efficient enough to be used for treatment of inoperable chronic thromboembolic pulmonary hypertension (CTEPH)


acronym: Not Applicable

title: Effect of Calcipotriol Plus Betamethasone Dipropionate Gel on the HPA Axis and Calcium Metabolism in Adolescent Subjects (Aged 12 to 16 Years, 11 Months) With Scalp and Body Psoriasis

lead_sponsor: LEO Pharma

location_country:
Poland
United Kingdom
Canada
United States
France
Romania
Germany

location_facility: Specjalistyczny Ofrodek Leczniczo Clinic

location_facility: Spitalul Clinic Municipal de Urgenta Timisoara

location_facility: Cabinet Medical Individual Tatu G. Alin Laurentiu

location_facility: Policlinica de Diagnostic Rapid S.A. Clinic

location_facility: Spitalul Clinic Judetean de Urgenta Cluj-Napoca

location_facility: Clinical Research Center, Morsani Center for Advanced Healthcare

location_facility: Dermatology and Laser Center of Charleston, PA

summary: An international, multi-centre, prospective, non-controlled, open, single-group, 8-week trial in adolescent subjects (aged 12 to 16 years, 11 months) with scalp and body psoriasis.


title: A Phase 1B, Multi-Center, Open-Label Study of Novel Combinations of CC-122, CC-223, CC-292, and Rituximab in Diffuse Large B-Cell Lymphoma and Follicular Lymphoma

summary: First study, at multiple clinical centers, exploring the effects of different combinations of compounds (CC-122, CC-223 ,CC-292 and rituximab) to treat Diffuse Large B Cell Lymphoma (DLBCL) and Follicular Lymphoma

lead_sponsor: Celgene

location_country:
United States
Italy
Canada
France

location_facility: Azienda Sanitaria Ospedaliera San Giovanni Battista Molinette

location_facility: Rocky Mountain Cancer Centers, LLP [Boulder-COBO]

location_facility: H. Lee Moffitt Cancer Center and Research Institute

location_facility: University of Wisconsin Osteoporosis Clinical Center and Research Program

location_facility: Institut Bergonie Centre Regional de Lutte Contre Le Cancer de Bordeaux Et Sud Ouest


title: A Multicenter, Long-term Extension Study to Further Evaluate the Safety and Tolerability of Telotristat Etiprate (LX1606)

acronym: TELEPATH

summary: The primary objective of this study is to evaluate the long-term safety and tolerability of orally administered telotristat etiprate.

ReferenceCitation: Hörsch D, Anthony L, Gross DJ, Valle JW, Welin S, Benavent M, Caplin M, Pavel M, Bergsland E, Öberg K, Kassler-Taub KB, Binder P, Banks P, Lapuerta P, Kulke MH. Long-Term Treatment with Telotristat Ethyl in Patients with Carcinoid Syndrome Symptoms: Results from the TELEPATH Study. Neuroendocrinology. 2022;112(3):298-310. doi: 10.1159/000516958. Epub 2021 May 3.

lead_sponsor: Lexicon Pharmaceuticals

location_country:
Spain
Italy
United Kingdom
Australia
Netherlands
Canada
Sweden
United States
France
Israel
Belgium
Germany

location_facility:
Lexicon Investgational Site
Lexicon Investigational Site


title: A Three Arm Randomized, Open-label Phase II Study of Radium-223 Dichloride 50 kBq/kg (55 kBq/kg After Implementation of NIST Update) Versus 80 kBq/kg (88 kBq/kg After Implementation of NIST Update), and Versus 50 kBq/kg (55 kBq/kg After Implementation of NIST Update) in an Extended Dosing Schedule in Subjects With Castration-resistant Prostate Cancer Metastatic to the Bone

summary: This study will assess different doses and regimens of radium-223 dichloride on the incidence of symptomatic skeletal events. Eligible subjects must have castration resistant prostate cancer with 2 or more skeletal metastases documented within 8 weeks of randomization. Subjects will be randomized to one of 3 treatment arms in a 1:1:1 fashion: a standard regimen of radium-223 dichloride of 50 kBq/kg (55 kBq/kg after implementation of NIST update) injections every month for 6 months, a high dose regimen of 80 kBq/kg (88 kBq/kg after implementation of NIST update)injections every month for 6 months or an extended duration regimen of 50 kBq/kg (55 kBq/kg after implementation of NIST update) injections every month for 12 months. Following the treatment phase, subjects will be followed up every 12 weeks for a minimum of 2 years, at which point they will enter a long term follow-up period during which they are seen every 6 months for up to 7 years after the last dose of radium dichloride. Symptomatic skeletal event and safety endpoints will be assessed at each clinic visit. Pain and analgesic use data will be collected every 4 weeks through Week 48. Additionally, radiological assessments including MRI/CT of the abdomen and pelvis and chest CT, as well as technetium-99 bone scans will be performed at Weeks 8, 16, and 24 and continue every 12 weeks thereafter until disease progression is documented in either the bone or in soft tissue. Radiological imaging will be evaluated by blinded central review.

ReferenceCitation: Sternberg CN, Saad F, Graff JN, Peer A, Vaishampayan UN, Leung E, Rosenbaum E, Gurney H, Epstein RJ, Davis ID, Wu B, Trandafir L, Wagner VJ, Hussain M. A randomised phase II trial of three dosing regimens of radium-223 in patients with bone metastatic castration-resistant prostate cancer. Ann Oncol. 2020 Feb;31(2):257-265. doi: 10.1016/j.annonc.2019.10.025. Epub 2019 Dec 23.

lead_sponsor: Bayer

location_country:
Taiwan
Brazil
Spain
Italy
United Kingdom
Australia
Canada
Sweden
Korea, Republic of
United States
Chile
France
Israel
Germany
Czechia

location_facility: IBCC - Instituto Brasileiro de Controle do Cancer

location_facility: Irmandade Santa Casa de Misericordia de Porto Alegre

location_facility: Instituto do Câncer do Estado de São Paulo


title: Prospective, Randomized, Placebo-controlled, Double-blind, Multicenter, Parallel-group, 24-week Study to Assess the Efficacy, Safety and Tolerability of Macitentan in Subjects With Inoperable Chronic Thromboembolic Pulmonary Hypertension

summary: Study to evaluate if macitentan is efficient, safe and tolerable enough to be used for treatment of inoperable chronic thromboembolic pulmonary hypertension (CTEPH).

ReferenceCitation: Ghofrani HA, Simonneau G, D'Armini AM, Fedullo P, Howard LS, Jaïs X, Jenkins DP, Jing ZC, Madani MM, Martin N, Mayer E, Papadakis K, Richard D, Kim NH; MERIT study investigators. Macitentan for the treatment of inoperable chronic thromboembolic pulmonary hypertension (MERIT-1): results from the multicentre, phase 2, randomised, double-blind, placebo-controlled study. Lancet Respir Med. 2017 Oct;5(10):785-794. doi: 10.1016/S2213-2600(17)30305-3. Epub 2017 Sep 11.

location_country:
Poland
United Kingdom
Russian Federation
Mexico
Lithuania
Thailand
Switzerland
Ukraine
Hungary
Korea, Republic of
France
Turkey
China
Belgium
Germany
Czechia

location_facility: Severance Hospital, YonSei University Health System

location_facility: Cardiovascular institute & Fuwai Hospital- Thrombus Center

location_facility: Wojewódzki Szpital Specjalistyczny w Lublinie im. Stefana Kardynała Wyszyńskiego SPZOZ Oddział Kardiologii - Pododdział Intensywnego Nadzoru Kardiologicznego

location_facility: Siriraj Hospital, Division of Respiratory Disease and Tuberculosis

location_facility: Lviv Regional Clinical Hospital, Cardiosurgery Department

location_facility: State Institute of Phthisiology and Pulmonology n.a. F.G. Yanovskiy of AMS Ukraine

location_facility: E.N.Meshalkin Novosibirsk State Research Institute Of Circulation Pathology Rosmedtechnology

location_facility: Centre for PPH, Charles University , II Interni klinika1.LF a VFN

location_facility: The General Hospital of Shenyang Military Region,Congenital Heart Disease Department

location_facility: Hôpital Européen Georges Pompidou Service de Pneumologie, soins intensifs et endoscopies bronchiques

location_facility: Missionsärztliche Klinik gGmbH Akademisches Lehrkrankenhaus der Julius-Maximilians-Universität

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

location_facility: Istanbul University İstanbul Faculty Medicine Pulmonology Department

location_facility: Instituto Nacional de Cardiologia (INC) Ignacio Chavez

location_facility: Sheffield Teaching Hospitals NHS Foundation Trust Royal Hallamshire Hospital, Pulmonary Vascular Medicine

location_facility: Lietuvos Sveikatos Mokslų Universiteto Ligoninės Kauno Klinikos Pulmonologijos-Imunologijos Klinika

location_facility: Federal State Budgetary Institution "Scientific Research Institute of Systemic Problems of Cardiovascular Diseases", Siberian branch of RAMS

location_facility: ngShanghai Pulmonary Hospital, Department of Pulmonary Circulation

location_facility: Papworth Hospital NHS Trust, Pulmonary Vascular Diseases Unit

location_facility: Wojewódzki Szpital Specjalistyczny we Wrocawiu

location_facility: Debreceni Egyetem Orvos- és Egészségtudományi Centrum, Kardiológiai Klinika

location_facility: Federal State Budgetary Institution "Russian Cardiology Scientific and Production Complex" of the Ministry of Health Care of the Russian Federation

location_facility: University Hospital Gasthuisberg / Interne Geneeskunde - I.G. Pneumologie

location_facility: Beijing Chao-Yang Hospital-Department of Respiration

location_facility: The first affiliated hospital of guangzhou medical university-respiratory department

location_facility: Federal State Budgetary Institution "Research Institute for Cardiology" of Siberian Branch under the Russian Academy of Medical Sciences / Cardiovascular Surgery Department

location_facility: King Chulalongkorn Memorial Hospital, Division of Respiratory and Respiratory Critical Care Medicine

location_facility: MAHARAJ NAKORN CHIANG MAI HOSPITAL, Department of Internal Medicine

location_facility: Thoraxklinik am Universitätsklinikum Heidelberg


title: A Phase IIb, Randomized, Multi-Center, Double-Blind, Dose-Ranging Study to Evaluate the Efficacy and Safety of Clazakizumab in Subjects With Moderate to Severe Active Rheumatoid Arthritis Who Have Experienced an Inadequate Response to TNF Inhibitors

summary: The primary purpose of this study is to identify an appropriate dose of study medication.

lead_sponsor: CSL Behring

location_country:
Italy
Argentina
Canada
Hungary
United States
France
South Africa
Mexico
Japan

location_facility: Cincinnati Rheumatic Disease Study Group

location_facility: Paramount Medical Research & Consulting, Llc

location_facility: Valerius Med Group & Res Ctr Of Greater Long Beach, Inc.

location_facility: Rheumatology Associates Of North Alabama, P.C.

location_facility: Arthritis & Rheumatology Center Of Oklahoma Pllc

location_facility: Dr. Latha Naik Medical Professional Corporation

location_facility: Cividino Medicine Professional Corporation

location_facility: Joint And Muscle Medical Care And Research Institute (Jmmcri)


acronym: RESPITE

title: An Open-label, International, Multicenter, Single-arm, Uncontrolled, Phase IIIb Study of Riociguat in Patients With Pulmonary Arterial Hypertension (PAH) Who Demonstrate an Insufficient Response to Treatment With Phosphodiesterase-5 Inhibitors (PDE-5i)

ReferenceCitation: Hoeper MM, Klinger JR, Benza RL, Simonneau G, Langleben D, Naeije R, Corris PA. Rationale and study design of RESPITE: An open-label, phase 3b study of riociguat in patients with pulmonary arterial hypertension who demonstrate an insufficient response to treatment with phosphodiesterase-5 inhibitors. Respir Med. 2017 Jan;122 Suppl 1:S18-S22. doi: 10.1016/j.rmed.2016.11.001. Epub 2016 Nov 5.

ReferenceCitation: Hoeper MM, Simonneau G, Corris PA, Ghofrani HA, Klinger JR, Langleben D, Naeije R, Jansa P, Rosenkranz S, Scelsi L, Grünig E, Vizza CD, Chang M, Colorado P, Meier C, Busse D, Benza RL. RESPITE: switching to riociguat in pulmonary arterial hypertension patients with inadequate response to phosphodiesterase-5 inhibitors. Eur Respir J. 2017 Sep 9;50(3). pii: 1602425. doi: 10.1183/13993003.02425-2016. Print 2017 Sep.

location_country:
United Kingdom
Italy
Canada
Switzerland
France
United States
Belgium
Germany
Czechia

summary:
BAY63-2521 Riociguat leads to the relaxation of smooth muscle cells in pulmonary arteria and may also inhibit abnormal remodeling of lung blood vessels. In patients with pulmonary arterial hypertension Riociguat showed to reduce the pulmonary blood pressure and improved the right heart function without unacceptable side effects. Here dose of Riociguat will be adjusted over 8 weeks then a Maintenance Phase of 16 weeks follows. Patients with Pulmonary Arterial Hypertension treated with stable doses of Phosphodiesterase Type-5 Inhibitors (Eg Sildenafil, Tadalafil) not appropriately responding to therapy will be included. Based on previous evidence and on the different modes of action an improvement of exercise capacity, heart function and quality of life may be expected if PDE5i treatment is transitioned to riociguat.

Where Riociguat is pending market approval or reimbursement once the treatment phase is completed drug can be made available for another 18 months (Extended Drug Supply Phase - EDSP) under study conditions. Patients may also transition at the end of the maintenance period or any time during the EDSP to any program that is intended to provide riociguat until drug approval/reimbursement, e.g. a long-term extension study, compassionate use or named patient program. Study termination is also possible at any time.


title: A Phase 2 Single Arm, Multicenter Trial to Evaluate the Efficacy of the BiTE Antibody Blinatumomab in Adult Subjects With Relapsed/Refractory Philadelphia Positive B-precursor Acute Lymphoblastic Leukemia (Alcantara Study)

summary: The primary objective is to evaluate the rate of complete remission/complete remission with partial hematological recovery (CRh*) in adults with relapsed/refractory Philadelphia chromosome positive (Ph+) B-precursor acute lymphoblastic leukemia (ALL) who receive blinatumomab.

ReferenceCitation: Martinelli G, Boissel N, Chevallier P, Ottmann O, Gökbuget N, Topp MS, Fielding AK, Rambaldi A, Ritchie EK, Papayannidis C, Sterling LR, Benjamin J, Stein A. Complete Hematologic and Molecular Response in Adult Patients With Relapsed/Refractory Philadelphia Chromosome-Positive B-Precursor Acute Lymphoblastic Leukemia Following Treatment With Blinatumomab: Results From a Phase II, Single-Arm, Multicenter Study. J Clin Oncol. 2017 Jun 1;35(16):1795-1802. doi: 10.1200/JCO.2016.69.3531. Epub 2017 Mar 29. Erratum in: J Clin Oncol. 2017 Aug 10;35(23):2722. J Clin Oncol. 2017 Aug 20;35(24):2856.

ReferenceCitation: Kuchimanchi M, Zhu M, Clements JD, Doshi S. Exposure-response analysis of blinatumomab in patients with relapsed/refractory acute lymphoblastic leukaemia and comparison with standard of care chemotherapy. Br J Clin Pharmacol. 2019 Apr;85(4):807-817. doi: 10.1111/bcp.13864. Epub 2019 Feb 18.

ReferenceCitation: Kantarjian HM, Zugmaier G, Brüggemann M, Wood BL, Horst HA, Zeng Y, Martinelli G. Impact of Blinatumomab Treatment on Bone Marrow Function in Patients with Relapsed/Refractory B-Cell Precursor Acute Lymphoblastic Leukemia. Cancers (Basel). 2021 Nov 9;13(22). pii: 5607. doi: 10.3390/cancers13225607.

ReferenceCitation: Clements JD, Zhu M, Kuchimanchi M, Terminello B, Doshi S. Population Pharmacokinetics of Blinatumomab in Pediatric and Adult Patients with Hematological Malignancies. Clin Pharmacokinet. 2020 Apr;59(4):463-474. doi: 10.1007/s40262-019-00823-8.

ReferenceCitation: Martinelli G, Boissel N, Chevallier P, Ottmann O, Gökbuget N, Rambaldi A, Ritchie EK, Papayannidis C, Tuglus CA, Morris JD, Stein A. Long-term follow-up of blinatumomab in patients with relapsed/refractory Philadelphia chromosome-positive B-cell precursor acute lymphoblastic leukaemia: Final analysis of ALCANTARA study. Eur J Cancer. 2021 Mar;146:107-114. doi: 10.1016/j.ejca.2020.12.022. Epub 2021 Feb 13.

ReferenceCitation: Topp MS, Stein AS, Gokbuget N, Horst HA, Boissel N, Martinelli G, Kantarjian H, Bruggemann M, Chen Y, Zugmaier G. Blinatumomab as first salvage versus second or later salvage in adults with relapsed/refractory B-cell precursor acute lymphoblastic leukemia: Results of a pooled analysis. Cancer Med. 2021 Apr;10(8):2601-2610. doi: 10.1002/cam4.3731. Epub 2021 Mar 18.

ReferenceCitation: Shi Z, Zhu Y, Zhang J, Chen B. Monoclonal antibodies: new chance in the management of B-cell acute lymphoblastic leukemia. Hematology. 2022 Dec;27(1):642-652. doi: 10.1080/16078454.2022.2074704. Review.

lead_sponsor: Amgen

location_country:
United Kingdom
Italy
United States
France
Germany


title: A Phase 2, Open-label, Randomized Study to Evaluate the Safety and Efficacy of Momelotinib in Subjects With Polycythemia Vera or Essential Thrombocythemia

summary: This open-label study is to determine the safety and efficacy of momelotinib in participants with either polycythemia vera (PV) or essential thrombocythemia (ET) who have not yet received treatment with a Janus kinase (JAK) inhibitor.

ReferenceCitation: Verstovsek S, Courby S, Griesshammer M, Mesa RA, Brachmann CB, Kawashima J, Maltzman JD, Shao L, Xin Y, Huang D, Bajel A. A phase 2 study of momelotinib, a potent JAK1 and JAK2 inhibitor, in patients with polycythemia vera or essential thrombocythemia. Leuk Res. 2017 Sep;60:11-17. doi: 10.1016/j.leukres.2017.05.002. Epub 2017 May 30.

lead_sponsor: Sierra Oncology, Inc.

location_country:
Australia
Canada
United States
France
Germany


acronym: PROgECT

title: A Phase 2a Open-Label Study to Evaluate Prediction of Response to Golimumab Using a Transcriptomic Profile in Subjects With Moderately to Severely Active Ulcerative Colitis

lead_sponsor: Janssen Research & Development, LLC

ReferenceCitation: Li K, Strauss R, Marano C, Greenbaum LE, Friedman JR, Peyrin-Biroulet L, Brodmerkel C, De Hertogh G. A Simplified Definition of Histologic Improvement in Ulcerative Colitis and its Association With Disease Outcomes up to 30 Weeks from Initiation of Therapy: Post Hoc Analysis of Three Clinical Trials. J Crohns Colitis. 2019 Aug 14;13(8):1025-1035. doi: 10.1093/ecco-jcc/jjz022.

ReferenceCitation: Telesco SE, Brodmerkel C, Zhang H, Kim LL, Johanns J, Mazumder A, Li K, Baribaud F, Curran M, Strauss R, Paxson B, Plevy S, Davison T, Knight L, Dibben S, Schreiber S, Sandborn W, Rutgeerts P, Siegel CA, Reinisch W, Greenbaum LE. Gene Expression Signature for Prediction of Golimumab Response in a Phase 2a Open-Label Trial of Patients With Ulcerative Colitis. Gastroenterology. 2018 Oct;155(4):1008-1011.e8. doi: 10.1053/j.gastro.2018.06.077. Epub 2018 Jul 4.

location_country:
Poland
Russian Federation
Netherlands
Canada
Czech Republic
Ukraine
Hungary
United States
France
Belgium
Germany
Bulgaria

summary: The purpose of this study is to evaluate the accuracy of a subset of the length-109 probe set panel (a genetic test) in predicting response to golimumab treatment in participants with moderately to severely active ulcerative colitis (UC).


title: A Double-blind, Randomized, Multicenter Study to Evaluate the Safety and Efficacy of Evolocumab, Compared With Ezetimibe, in Hypercholesterolemic Subjects Unable to Tolerate an Effective Dose of a HMG-CoA Reductase Inhibitor Due to Muscle Related Side Effects

summary: The primary objective of this study was to evaluate the effect of 24 weeks of evolocumab administered subcutaneously (SC) every month, compared with ezetimibe, on low-density lipoprotein cholesterol (LDL-C) levels in adults with high cholesterol who are unable to tolerate an effective dose of a statin due to muscle-related side effects (MRSE).

ReferenceCitation: Cho L, Dent R, Stroes ESG, Stein EA, Sullivan D, Ruzza A, Flower A, Somaratne R, Rosenson RS. Persistent Safety and Efficacy of Evolocumab in Patients with Statin Intolerance: a Subset Analysis of the OSLER Open-Label Extension Studies. Cardiovasc Drugs Ther. 2018 Aug;32(4):365-372. doi: 10.1007/s10557-018-6817-7.

ReferenceCitation: Schmidt AF, Carter JL, Pearce LS, Wilkins JT, Overington JP, Hingorani AD, Casas JP. PCSK9 monoclonal antibodies for the primary and secondary prevention of cardiovascular disease. Cochrane Database Syst Rev. 2020 Oct 20;10:CD011748. doi: 10.1002/14651858.CD011748.pub3.

ReferenceCitation: Nissen SE, Stroes E, Dent-Acosta RE, Rosenson RS, Lehman SJ, Sattar N, Preiss D, Bruckert E, Ceška R, Lepor N, Ballantyne CM, Gouni-Berthold I, Elliott M, Brennan DM, Wasserman SM, Somaratne R, Scott R, Stein EA; GAUSS-3 Investigators. Efficacy and Tolerability of Evolocumab vs Ezetimibe in Patients With Muscle-Related Statin Intolerance: The GAUSS-3 Randomized Clinical Trial. JAMA. 2016 Apr 19;315(15):1580-90. doi: 10.1001/jama.2016.3608.

location_country:
Denmark
United Kingdom
Italy
Australia
Netherlands
Canada
New Zealand
United States
France
South Africa
Norway
Germany
Czechia


title: A Phase II, Randomized Study of Atezolizumab (Anti-PD-L1 Antibody) Administered as Monotherapy or in Combination With Bevacizumab Versus Sunitinib in Patients With Untreated Advanced Renal Cell Carcinoma

ReferenceCitation: Pal SK, McDermott DF, Atkins MB, Escudier B, Rini BI, Motzer RJ, Fong L, Joseph RW, Oudard S, Ravaud A, Bracarda S, Suárez C, Lam ET, Choueiri TK, Ding B, Quach C, Hashimoto K, Schiff C, Piault-Louis E, Powles T. Patient-reported outcomes in a phase 2 study comparing atezolizumab alone or with bevacizumab vs sunitinib in previously untreated metastatic renal cell carcinoma. BJU Int. 2020 Jul;126(1):73-82. doi: 10.1111/bju.15058. Epub 2020 Apr 24.

location_country:
Poland
Spain
Italy
United Kingdom
United States
France
Romania
Germany
Czechia

location_facility: Klinikum d.Universität München Campus Großhadern

location_facility: Hospital Clinic de Barcelona. Unidad de Nuevas Terapias;Oncology Department

location_facility: Hopital Europeen Georges Pompidou; Service D'Oncologie Medicale

location_facility: City of Hope Comprehensive Cancer Center

location_facility: Institut Gustave Roussy; Departement Oncologie Medicale

location_facility: Irccs Istituto Nazionale Dei Tumori (Int);S.C. Medicina Oncologica 2

location_facility: Yale Uni School of Medicine; Section of Medical Oncology

location_facility: Cleveland Clinic Foundation; Taussig Cancer Center

location_facility: HonorHealth Research Institute - Bisgrove

location_facility: Christie Hospital Nhs Trust; Medical Oncology

location_facility: Texas Oncology-Baylor Sammons Cancer Center

location_facility: Klinikum rechts der Isar der TU München; Klinikapotheke

location_facility: Prof. Dr. I. Chiricuta Institute of Oncology

location_facility: Tennessee Oncology PLLC - Nashville (20th Ave)

location_facility: Hosp de Madrid Norte Sanchinarro; Centro Integral; Onco Clara Campal

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

location_facility: Azienda Ospedaliera Universitaria Senese, U.O.C. Immunoterapia Oncologica

location_facility: Rocky Mountain Cancer Ctr - Denver (Williams)

summary: This multicenter, randomized, open-label study will evaluate the efficacy, safety and tolerability of atezolizumab as monotherapy or in combination with bevacizumab versus sunitinib in participants with histologically confirmed, inoperable, locally advanced or metastatic renal cell carcinoma who have not received prior systemic therapy either in the adjuvant or metastatic setting.


title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study Evaluating the Efficacy and Safety of Idelalisib in Combination With Bendamustine and Rituximab for Previously Untreated Chronic Lymphocytic Leukemia

summary:
The primary objective of this study is to evaluate the progression-free survival in participants with previously untreated chronic lymphocytic leukemia (CLL) who would otherwise be suitable for bendamustine and rituximab treatment as standard of care.

An increased rate of deaths and serious adverse events (SAEs) among participants with front-line CLL and early-line indolent non-Hodgkin lymphoma (iNHL) treated with idelalisib in combination with standard therapies was observed by the independent data monitoring committee (DMC) during regular review of 3 Gilead Phase 3 studies. Gilead reviewed the unblinded data and terminated this study in agreement with the DMC recommendation and in consultation with the US Food and Drug Administration (FDA).

ipd_sharing_description: Qualified external researchers may request IPD for this study after study completion. For more information, please visit our website at http://www.gilead.com/research/disclosure-and-transparency.

lead_sponsor: Gilead Sciences

location_country:
Croatia
Poland
Romania
Spain
Italy
United Kingdom
Australia
Canada
Hungary
United States
France
Belgium
Czechia

location_facility: East Kent Hospitals University NHS Foundation Trust

location_facility: Royal Wolverhampton Hospital NHS Trust, New Cross Hospital

location_facility: Haematology and Bone Marrow Transplant Unit, Royal Adelaide Hospital

location_facility: St. Jude Heritage Healthcare Virginia K. Crosson Cancer Center

location_facility: University of Debrecen HSC Institute of internal Medicine, Department of Hematology

location_facility: Barwon Health, University Hospital Geelong

location_facility: Dolnoslakie Centrum Transplantacji Komorkowych z Krajowym Bankiem Dawcow Szpiku

location_facility: Emergency County Clinical Hospital Brasov

location_facility: CHU de Québec - Hôpital de l'Enfant-Jésus

location_facility: Royal Liverpool & Broadgreen Univ. Hospitals NHS Trust

location_facility: Samodzielny Publiczny Szpital Kliniczny nr 1 we Wroclawiu Klinika i Katedra Hematologii,Nowotworow Krwi i Transplantacji Szpiku

location_facility: Centrum Onkologii-Instytut Marii Sklodowskiej -Curie klinika Nowotworow Ukladu Chlonnego

location_facility: Hematology /Oncology Associates of Northern New Jersey

location_facility: Wojewodzki Szpital Specjalistyczny im. M. Kopernika Klinika Hematologii

location_facility: Franciscan Physician Network Oncology & Hematology

location_facility: Azienda Ospedaliero Universitaria Policlinico di Modena

location_facility: Siouxland Hematology-Oncology Associates, LLP

location_facility: Szegedi Tudomanyegyetem AOK - Szent-Gyorgyi Albert Klinikai Kozpont, II. Belgyógyászati Klinika

location_facility: Centralny Szpital Kliniczny MSW w Warszawie Klinika Onkologii i Hematologii

location_facility: Kaposi Mor Oktato Korhaz, Intezeti Gyogyszertar,

location_facility: Flinders Medical Centre, Department of Haematology, Level 6

location_facility: University Hospital Southampton NHS Trust

location_facility: Texas Oncology-Baylor Charles A. Sammons Cancer Center


title: A Multicenter, Single-arm, Open-label, Postmarketing Safety Study to Evaluate the Risk of Seizure Among Subjects With Metastatic Castration-Resistant Prostate Cancer (mCRPC) Treated With Enzalutamide Who Are at Potential Increased Risk of Seizure

acronym: UPWARD

summary: The objective of this study was to evaluate the incidence of seizures and monitor the safety of enzalutamide treatment in participants with metastatic castration-resistant prostate cancer (mCRPC) known to have risk factor(s) for seizure.

ipd_sharing_description: Access to anonymized individual participant level data collected during the study, in addition to study-related supporting documentation, is planned for studies conducted with approved product indications and formulations, as well as compounds terminated during development. Studies conducted with product indications or formulations that remain active in development are assessed after study completion to determine if Individual Participant Data can be shared. Conditions and exceptions are described under the Sponsor Specific Details for Astellas on www.clinicalstudydatarequest.com.

lead_sponsor: Astellas Pharma Global Development, Inc.

location_country:
Taiwan
Spain
Italy
United Kingdom
Australia
New Zealand
Argentina
Canada
Finland
Sweden
Hungary
Korea, Republic of
United States
Chile
France
Israel
Belgium
Singapore
Germany
Czechia

location_facility:
Site AU61001
Site BE32001
Site IT39005
Site SG65002
Site BE32004
Site ES34003
Site CZ42004
Site IL97201
Site KR82003
Site KR82004
Site US10014
Site IT39003
Site AU61004
Site IL97206
Site ES34007
Site CL56004
Site ES34006
Site FI35801
Site DE49003
Site KR82001
Site IT39002
Site ES34004
Site CL56003
Site IL97204
Site IT39001
Site HU36002
Site US10008
Site DE49001
Site IL97207
Site KR82007
Site NZ64001
Site GB44002
Site CL56001
Site US10005
Site FR33002
Site AU61002
Site AR54006
Site CZ42002
Site CL56002
Site IL97203
Site IL97208
Site FR33005
Site AU61011
Site CA15001
Site FI35803
Site IL97202
Site FR33004
Site US10039
Site US10026
Site FI35802
Site KR82006
Site CA15010
Site ES34001
Site AU61005
Site AR54001
Site US10025
Site IL97205
Site SE46002
Site BE32003
Site AU61012
Site TW88601
Site AR54004
Site DE49009
Site AR54003
Site US10001
Site TW88603
Site AR54005
Site US10016
Site CA15004
Site AU61007
Site CA15005
Site AR54002
Site ES34005
Site SE46001
Site CA15014
Site US10024


title: A Randomised, Double-Blind, Placebo-Controlled, Multicentre Phase II Study to Compare the Efficacy, Safety and Tolerability of Olaparib Versus Placebo When Given in Addition to Abiraterone Treatment in Patients With Metastatic Castrate-Resistant Prostate Cancer Who Have Received Prior Chemotherapy Containing Docetaxel

summary: This is a 2-part study in patients with metastatic CRPC. Part A is an open-label safety run-in study to assess the safety, tolerability and pharmacokinetics (PK) of olaparib when given in addition to abiraterone 1000 mg once daily. Part B is a randomised, double-blind, placebo controlled comparison of the efficacy, safety and tolerability of the dose of olaparib selected from Part A when given in addition to abiraterone, versus placebo given in addition to abiraterone. Abiraterone is indicated in combination with prednisone or prednisolone for the treatment of patients with metastatic CRPC. Prednisone or prednisolone 5 mg twice daily (bid) will be administered with the abiraterone in this study.

ReferenceCitation: Clarke N, Wiechno P, Alekseev B, Sala N, Jones R, Kocak I, Chiuri VE, Jassem J, Fléchon A, Redfern C, Goessl C, Burgents J, Kozarski R, Hodgson D, Learoyd M, Saad F. Olaparib combined with abiraterone in patients with metastatic castration-resistant prostate cancer: a randomised, double-blind, placebo-controlled, phase 2 trial. Lancet Oncol. 2018 Jul;19(7):975-986. doi: 10.1016/S1470-2045(18)30365-6. Epub 2018 Jun 4.

ReferenceCitation: Saad F, Thiery-Vuillemin A, Wiechno P, Alekseev B, Sala N, Jones R, Kocak I, Chiuri VE, Jassem J, Fléchon A, Redfern C, Kang J, Burgents J, Gresty C, Degboe A, Clarke NW. Patient-reported outcomes with olaparib plus abiraterone versus placebo plus abiraterone for metastatic castration-resistant prostate cancer: a randomised, double-blind, phase 2 trial. Lancet Oncol. 2022 Oct;23(10):1297-1307. doi: 10.1016/S1470-2045(22)00498-3. Epub 2022 Sep 2. Erratum in: Lancet Oncol. 2022 Oct;23(10):e446.

ipd_sharing_description:
Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal. All request will be evaluated as per the AZ disclosure commitment: https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.

Yes, indicates that AZ are accepting requests for IPD, but this does not mean all requests will be shared.

lead_sponsor: AstraZeneca

location_country:
Poland
Spain
Italy
Russian Federation
United Kingdom
Canada
France
United States
Belgium
Netherlands
Czechia


title: A Randomized, Double-blind, Placebo-controlled, Parallel-Group, 26-Week, Phase 3 Study of 2 Doses of EVP-6124 or Placebo in Subjects With Mild to Moderate Alzheimer's Disease Currently or Previously Receiving an Acetylcholinesterase Inhibitor Medication

summary: The purpose of this study is to evaluate the safety and efficacy of 2 fixed doses of EVP-6124 compared to placebo for 26 weeks in subjects with mild to moderate Alzheimer's disease currently receiving stable treatment or previously treated with an acetylcholinesterase inhibitor.

lead_sponsor: FORUM Pharmaceuticals Inc

location_country:
Spain
Italy
United Kingdom
Australia
Netherlands
Argentina
Czech Republic
Canada
Korea, Republic of
United States
France
South Africa
Mexico
Germany


title: A Phase 2b, Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel Group, Dose-response Study Evaluating the Efficacy and Safety of JNJ-54781532 in Subjects With Moderately to Severely Active Ulcerative Colitis

summary: The purpose of the study is to evaluate dose response of JNJ-54781532 in participants with moderately to severely active ulcerative colitis (UC).

ReferenceCitation: Sands BE, Sandborn WJ, Feagan BG, Lichtenstein GR, Zhang H, Strauss R, Szapary P, Johanns J, Panes J, Vermeire S, O'Brien CD, Yang Z, Bertelsen K, Marano C; Peficitinib-UC Study Group. Peficitinib, an Oral Janus Kinase Inhibitor, in Moderate-to-severe Ulcerative Colitis: Results From a Randomised, Phase 2 Study. J Crohns Colitis. 2018 Nov 9;12(10):1158-1169. doi: 10.1093/ecco-jcc/jjy085.

location_country:
Poland
Romania
Russian Federation
Australia
Netherlands
Canada
Ukraine
Hungary
United States
France
Israel
Belgium
Germany
Bulgaria


title: A Randomized, Open-label, Multicenter, Controlled, Parallel Arm, Phase III Study Assessing the Efficacy and Safety of AOP2014 vs. Hydroxyurea in Patients With Polycythemia Vera

acronym: PROUD-PV

summary: Phase III study to compare the efficacy and safety of the novel monopegylated interferon alpha 2b AOP2014 versus Hydroxyurea (the current licensed therapy for this disease). One year treatment of patients with polycythemia vera. Objective is to demonstrate non-inferiority of AOP2014 vs. HU in terms of disease response rate in both HU naïve and currently treated patients, diagnosed with Polycythemia Vera. Response is measured as normalisation of key lab parameters as well as normalized spleen size.

ReferenceCitation: Verger E, Soret-Dulphy J, Maslah N, Roy L, Rey J, Ghrieb Z, Kralovics R, Gisslinger H, Grohmann-Izay B, Klade C, Chomienne C, Giraudier S, Cassinat B, Kiladjian JJ. Ropeginterferon alpha-2b targets JAK2V617F-positive polycythemia vera cells in vitro and in vivo. Blood Cancer J. 2018 Oct 4;8(10):94. doi: 10.1038/s41408-018-0133-0.

lead_sponsor: AOP Orphan Pharmaceuticals AG

location_country:
Poland
Romania
Spain
Italy
Austria
Russian Federation
Czech Republic
Ukraine
Hungary
France
Belgium
Germany
Bulgaria
Slovakia

location_facility: Dnipropetrovsk City Multispecialty Clinical Hospital #4

location_facility: Multiprofile Hospital for Active Treatment - Hristo Botev, Vratsa, First Department of Internal Medicine

location_facility: University Hospital Carl Gustav Carus, Medical Clinic and Polyclinic I

location_facility: National Research Center for Radiation Medicine, Institute of Clinical Radiology

location_facility: First Pavlov State Medical University of St. Petersburg

location_facility: Samara Kalinin Regional Clinical Hospital

location_facility: Bekes County Pandy Kalman Hospital, 1st Department of Medicine, Hematology

location_facility: Institute of Hematology and Blood Transfusion

location_facility: Cherkasy Regional Oncology Center, Regional Treatment and Diagnostics Hematology Center

location_facility: Hospital Clinic i Provincial de Barcelona

location_facility: Andrzej Mielecki Independent Public Clinical Hospital of Medical University of Silesia in Katowice

location_facility: Emergency Clinical County Hospital Brasov

location_facility: "Prof. Dr. Ion Chiricuta" Institute of Oncology

location_facility: Multiprofile Hospital for Active Treatment "Sveta Marina"

location_facility: Nicolaus Copernicus Municipal Specialist Hospital

location_facility: Institute of Hematology and Transfusion Medicine

location_facility: Independent Public Teaching Hospital No.1 in Lublin

location_facility: Specialized Hospital for Active Treatment of Hematological Diseases

location_facility: Aachen University Hospital, Medical Clinic IV

location_facility: Fryderyk Chopin Provincial Specialized Hospital

location_facility: O.F. Herbachevskyi Regional Clinical Hospital

location_facility: University of Szeged, Albert Szent-Gyorgyi Clinical Center, Koranyi fasor 6

location_facility: University Multiprofile Hospital for Active Treatment "Sveti Georgi"

location_facility: University Hospital Bonn, Center for Internal Medicine, Medical Clinic and Outpatient Clinic III

location_facility: University Hospital with Outpatient Clinic F.D. Roosevelt

location_facility: Institute of Blood Pathology and Transfusion Medicine

location_facility: St Istvan and St Laszlo Hospital of Budapest

location_facility: Saint Cyril and Metod University Hospital Bratislava


title: A Multicenter, Phase 2, Randomized, Open Label, Active-controlled, Parallel-group Study Investigating the Safety, Tolerability, and Efficacy of Different Dose Levels of ACP-001 Administered Once Weekly Versus Standard Daily rhGH Replacement Therapy in Pre-pubertal Children With Growth Hormone Deficiency (GHD)

lead_sponsor: Ascendis Pharma A/S

ReferenceCitation: Chatelain P, Malievskiy O, Radziuk K, Senatorova G, Abdou MO, Vlachopapadopoulou E, Skorodok Y, Peterkova V, Leff JA, Beckert M; TransCon GH Working Group. A Randomized Phase 2 Study of Long-Acting TransCon GH vs Daily GH in Childhood GH Deficiency. J Clin Endocrinol Metab. 2017 May 1;102(5):1673-1682. doi: 10.1210/jc.2016-3776.

location_country:
Poland
Belarus
Slovenia
Greece
Russian Federation
Czech Republic
Ukraine
Hungary
France
Turkey
Romania
Germany
Bulgaria
Egypt

location_facility: Louis Turcanu Emergency Hospital for Children Timisoara

location_facility: Donetsk Regional Children Clinical Hospital

location_facility: Ukrainian Children Specialized Clinical Hospital

location_facility: St. Spiridon County Clinic Emergency Hospital

location_facility: Children's Hospital of Athens "P. A. Kyriakou"

location_facility: Children's Memorial Health Institute Warsaw

location_facility: Institute of Endocrinology and Metabolism

location_facility: St. Petersburg State Pediatric Medical Academy

location_facility: University Children's Hospital Magdeburg

location_facility: University Multiprofile Hospital for Active Treatment (UMHAT) "Sv. Marina"

summary: A six month study of ACP-001, a long-acting growth hormone product, versus standard human growth hormone therapy. ACP-001 will be given once-a-week, standard human growth hormone (hGH) will be given on a daily basis. The primary aim is to demonstrate safety, pharmacokinetics and pharmacodynamics over a period of six months. A secondary objective is the comparison of height velocity (HV) of the ACP-001 treated groups to the daily hGH treatment group.


title: Phase Ib/II Multicenter Study of Buparlisib Plus Carboplatin or Lomustine in Patients With Recurrent Glioblastoma Multiforme

summary:
This is a multi-center, phase Ib/ II study (two parts) with patients that had recurrent glioblastoma multiforme. The first part (phase Ib) was to investigate the maximum tolerated dose/Recommended phase ll dose (MTD/RP2D) of once daily buparlisib in combination with every-three-week carboplatin or buparlisib once daily in combination with every-six-week lomustine (CCNU) using a Bayesian model. Once MTD/ RP2D is established in either of the 2 arms, the corresponding phase II portion of the study was to start. Phase II was to assess the treatment effect of buparlisib in combination with carboplatin in terms of Progression Free Survival (PFS) and was to compare the treatment effect of buparlisib with lomustine versus lomustine plus placebo in terms of PFS.

A preliminary assessment for both combinations (buparlisib plus carboplatin or lomustine) demonstrated that there was not enough antitumor activity compared to historical data with single agent carboplatin or lomustine. Based on the overall safety profile, and preliminary anti-tumor activity observed in this study, Novartis decided that no additional patients would be enrolled into this study. As a consequence, the Phase II part of the study was not conducted.

ReferenceCitation: Rosenthal M, Clement PM, Campone M, Gil-Gil MJ, DeGroot J, Chinot O, Idbaih A, Gan H, Raizer J, Wen PY, Pineda E, Donnet V, Mills D, El-Hashimy M, Mason W. Buparlisib plus carboplatin or lomustine in patients with recurrent glioblastoma: a phase Ib/II, open-label, multicentre, randomised study. ESMO Open. 2020 Jul;5(4). pii: e000672. doi: 10.1136/esmoopen-2020-000672.

lead_sponsor: Novartis Pharmaceuticals

location_country:
Spain
Australia
Canada
United States
France
Belgium

location_facility: MD Anderson Cancer Center/University of Texas

location_facility: Barrow Neurological Insitute St. Joseph's Hospital


acronym: Dolutegravir Womens Study

title: A Phase IIIb, Randomized, Open-label Study of the Safety and Efficacy of Dolutegravir/Abacavir/Lamivudine Once Daily Compared to Atazanavir and Ritonavir Plus Tenofovir/Emtricitabine Once Daily in HIV-1 Infected Antiretroviral Therapy Naïve Women

lead_sponsor: ViiV Healthcare

ReferenceCitation: Orrell C, Hagins DP, Belonosova E, Porteiro N, Walmsley S, Falcó V, Man CY, Aylott A, Buchanan AM, Wynne B, Vavro C, Aboud M, Smith KY; ARIA study team. Fixed-dose combination dolutegravir, abacavir, and lamivudine versus ritonavir-boosted atazanavir plus tenofovir disoproxil fumarate and emtricitabine in previously untreated women with HIV-1 infection (ARIA): week 48 results from a randomised, open-label, non-inferiority, phase 3b study. Lancet HIV. 2017 Dec;4(12):e536-e546. doi: 10.1016/S2352-3018(17)30095-4. Epub 2017 Jul 17. Erratum in: Lancet HIV. 2017 Dec;4(12 ):e535.

location_country:
Portugal
Spain
Italy
Russian Federation
United Kingdom
Argentina
Canada
Thailand
Puerto Rico
United States
France
South Africa
Mexico

summary: This study is designed to demonstrate the non-inferior antiviral activity of DTG/ABC/3TC fixed dose combination (FDC) once daily (OD) compared to atazanavir plus ritonavir (ATV+RTV) and tenofovir disoproxil fumarate/emtricitabine fixed dose combination (TDF/FTC FDC) OD in HIV-1 infected, ART-naïve women over 48 weeks. This study will also characterize the safety and tolerability of DTG/ABC/3TC FDC compared to ATV+RTV+TDF/FTC FDC. Sufficient number of subjects will be screened in order to ensure a total of approximately 474 subjects will be randomized (237 in each study arm)


title: A Randomised, Double-blind, Double-dummy, Active-controlled Study Evaluating the Efficacy, Safety and Tolerability of Twice-daily Aclidinium Bromide/Formoterol Fumarate Compared With Twice-daily Salmeterol/Fluticasone Propionate for 24 Weeks Treatment in Symptomatic Patients With Chronic Obstructive Pulmonary Disease (COPD)

summary: The purpose of the study is to compare the efficacy, safety and tolerability of aclidinium bromide/formoterol fumarate and salmeterol/fluticasone propionate in patients with chronic obstructive pulmonary disease (COPD)

ReferenceCitation: Vogelmeier C, Paggiaro PL, Dorca J, Sliwinski P, Mallet M, Kirsten AM, Beier J, Seoane B, Segarra RM, Leselbaum A. Efficacy and safety of aclidinium/formoterol versus salmeterol/fluticasone: a phase 3 COPD study. Eur Respir J. 2016 Oct;48(4):1030-1039. doi: 10.1183/13993003.00216-2016. Epub 2016 Aug 4.

location_country:
Poland
Spain
Italy
Austria
Netherlands
United Kingdom
Canada
Czech Republic
Lithuania
Hungary
France
South Africa
Germany
Bulgaria


title: An Open-label, Multicenter, Multinational, Ascending Dose Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Repeated Biweekly Infusions of neoGAA in naïve and Alglucosidase Alfa Treated Late-onset Pompe Disease Patients.

summary:
Primary Objective:

To evaluate the safety and tolerability of neoGAA in treatment naïve and alglucosidase alfa treated late-onset Pompe disease patients.

Secondary Objective:

To evaluate the pharmacokinetics, pharmacodynamics of neoGAA in treatment naïve and alglucosidase alfa treated late-onset Pompe disease patients.

To evaluate the effect of neoGAA on exploratory efficacy endpoints in treatment naïve and alglucosidase alfa treated late-onset Pompe disease patients.

lead_sponsor: Genzyme, a Sanofi Company

location_country:
Denmark
United Kingdom
Netherlands
United States
France
Belgium
Germany

location_facility:
Investigational Site Number 250003
Investigational Site Number 056001
Investigational Site Number 840002
Investigational Site Number 840001
Investigational Site Number 250002
Investigational Site Number 208001
Investigational Site Number 276002
Investigational Site Number 840010
Investigational Site Number 840006
Investigational Site Number 826003
Investigational Site Number 840003
Investigational Site Number 276003
Investigational Site Number 276001
Investigational Site Number 840008
Investigational Site Number 528001
Investigational Site Number 250001
Investigational Site Number 840009


title: A Randomized, Phase III, Multicenter, Double-Blind, Placebo-Controlled Study Evaluating the Efficacy and Safety of Onartuzumab in Combination With Erlotinib as First-Line Treatment for Patients With MET-Positive Unresectable Stage IIIb or IV Non-Small Cell Lung Cancer (NSCLC) Carrying an Activating EGFR Mutation

lead_sponsor: Hoffmann-La Roche

location_country:
Taiwan
Spain
Korea, Republic of
United States
France
Malaysia
Japan
Germany

summary: This multicenter, randomized, double-blind, placebo-controlled study will evaluate the safety and efficacy of onartuzumab in combination with erlotinib in patients with previously untreated, unresectable stage IIIB or IV non-small cell lung cancer identified to carry and activating EGFR mutation and MET-positive. Patients will be randomized to receive either onartuzumab 15 mg/kg intravenously every 3 weeks in combination with erlotinib 150 mg orally daily or placebo in combination with erlotinib. Anticipated time on study treatment is until disease progression or unacceptable toxicity occurs.


title: A Blind Randomized Multicenter Study of Accelerated Fractionated Chemo-radiotherapy With or Without the Hypoxic Cell Radiosensitizer Nimorazole (Nimoral), Using a 15-gene Signature for Hypoxia in the Treatment of Squamous Cell Carcinoma of the Head and Neck.

summary:
The drug nimorazole belongs to a class of chemicals known as 5-nitroimidazoles. Drugs from this class are used against infection. In addition, nimorazole makes tumor cells more sensitive to radiotherapy.

Therefore, the investigators want to find out whether the addition of nimorazole to the standard treatment with radiotherapy in combination with chemotherapy with cisplatin shows activity against your type of head and neck cancer and is safe.

Furthermore the investigators will investigate if a specific examination done with your tumor tissue will help to predict whether the treatment will work or not.

To find out if the activity observed with this treatment is not caused by chance alone, the investigators need to obtain data from patients who receive this treatment and from patients who receive other treatments.

The data from these two groups of patients will be compared to see which treatment is better.

Participants will be split into 2 groups. Each group will receive different treatments. The treatment each group receives is determined by chance using a computer program. This works like flipping a coin and is called randomization. This helps to make sure that groups of patients are similar when the study starts. Neither you, your study doctor, nor the study staff can influence in which group you will be placed or which treatment you will receive.

If allocated to group 1, Patient will receive radiotherapy in combination with chemotherapy with cisplatin and nimorazole as a pill. This is considered the 'experimental' treatment.

If allocated to group 2, patient will receive radiotherapy in combination with chemotherapy with cisplatin and a so called 'placebo' as a pill. The placebo is a dummy treatment. It looks like the real one, but it is not. It contains no active ingredient/medicine.

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

location_country: Poland Australia Netherlands Switzerland France Belgium Germany

location_facility: Charite - Universitaetsmedizin Berlin - Campus Virchow-Klinikum

location_facility: Hopitaux Universitaires Bordet-Erasme - Institut Jules Bordet

location_facility: Radboud University Medical Center Nijmegen

location_facility: Princess Alexandra Hospital - University Of Queensland

location_facility: Hôpitaux universitaires de Genève - HUG - site de Cluse-Roseraie

location_facility: Maria Sklodowska-Curie Memorial Cancer Centre

location_facility: Ludwig-Maximilians-Universitaet Muenchen - Klinikum der Universitaet Muenchen - Campus Grosshadern

intervention_type: Radiation


title: A Phase Ib/II, Multicenter Study of the Combination of LEE011 and BYL719 With Letrozole in Adult Patients With Advanced ER+ Breast Cancer

summary:
The purpose of this trial is to inform the future clinical development of the two investigational agents in ER+ breast cancer, LEE011 (CDK4/6 inhibitor) and BYL719 (PI3K-alpha inhibitor).

This is a multi-center, open-label Phase Ib study. The Phase Ib dose escalation will estimate the MTD and/or RP2D for three regimens: two double combinations, LEE011 with letrozole and BYL719 with letrozole, followed by triple combinations of LEE011 + BYL719 with letrozole (Arms 3 and 4).

The Phase Ib dose escalation part will be followed by Phase Ib dose expansions to further characterize the safety, tolerability, PK and preliminary clinical anti-tumor activity of the combinations. Optional crossover for patients who have progressed while on dose escalation or dose expansion with doublet treatment on Arms 1 or 2 to be treated with the triplet combination (Arm 3) after the determination of the RP2D for Arm 3; is no longer permitted after protocol amendment 6.

Approximately 270 adult women with ER+/HER2- locally advanced or metastatic breast cancer will be enrolled.

location_country:
Spain
Italy
United Kingdom
Australia
Switzerland
Korea, Republic of
United States
France

location_facility: H Lee Moffitt Cancer Center and Research Institute H. Lee Moffitt Cancer Center

location_facility: University of California at San Diego, Moores Cancer Ctr Dept. of Moores Cancer Center

location_facility: Vanderbilt University Medical Center Vanderbilt - Thompson Ln

location_facility: Cancer Therapy and Research Center UT Health Science Center Dept of Onc

location_facility: Northwest Medical Specialties Northwest Medical Specialties