acronym: GETGOAL-L

title: A Randomized, Double-blind, Placebo-controlled, 2-arm Parallel-group, Multicenter Study With a 24-week Main Treatment Period and an Extension Assessing the Efficacy and Safety of AVE0010 in Patients With Type 2 Diabetes Insufficiently Controlled With Basal Insulin

lead_sponsor: Sanofi

ReferenceCitation: Riddle MC, Aronson R, Home P, Marre M, Niemoeller E, Miossec P, Ping L, Ye J, Rosenstock J. Adding once-daily lixisenatide for type 2 diabetes inadequately controlled by established basal insulin: a 24-week, randomized, placebo-controlled comparison (GetGoal-L). Diabetes Care. 2013 Sep;36(9):2489-96. doi: 10.2337/dc12-2454. Epub 2013 Apr 29.

ReferenceCitation: Davidson JA, Stager W, Paranjape S, Berria R, Leiter LA. Achieving postprandial glucose control with lixisenatide improves glycemic control in patients with type 2 diabetes on basal insulin: a post-hoc analysis of pooled data. Clin Diabetes Endocrinol. 2020 Jan 14;6:2. doi: 10.1186/s40842-019-0088-5. eCollection 2020.

ReferenceCitation: Charbonnel B, Bertolini M, Tinahones FJ, Domingo MP, Davies M. Lixisenatide plus basal insulin in patients with type 2 diabetes mellitus: a meta-analysis. J Diabetes Complications. 2014 Nov-Dec;28(6):880-6. doi: 10.1016/j.jdiacomp.2014.07.007. Epub 2014 Jul 18.

location_country:
Brazil
United Kingdom
Italy
Russian Federation
Canada
Korea, Republic of
United States
Chile
France
India
Mexico
Puerto Rico
Germany
Turkey
Egypt

summary:
The purpose of this study is to evaluate the benefits and risks of lixisenatide (AVE0010), in comparison to placebo, as an add-on treatment to basal insulin with or without metformin over a period of 24 weeks of treatment, followed by an extension.

The primary objective is to assess the effects of lixisenatide when added to basal insulin on glycemic control in terms of glycosylated hemoglobin (HbA1c) reduction at Week 24.

The secondary objectives are to assess the effects of lixisenatide when added to basal insulin on body weight, 2-hour postprandial plasma glucose (PPG) after standardized meal challenge test, percentage of patients reaching HbA1c less than 7 percent (%), percentage of patients reaching HbA1c less than or equal to 6.5%, fasting plasma glucose (FPG), change in 7-point self-monitored plasma glucose (SMPG) profiles, change in basal insulin and total insulin doses; to evaluate safety, tolerability, pharmacokinetics (PK) and anti-lixisenatide antibody development.


title: A Multi-center, Randomized, Double-blind Clinical Trial to Evaluate the Safety and Tolerability of 24 Weeks Treatment With (50 mg qd) Versus Placebo in Patients With Type 2 Diabetes and Moderate or Severe Renal Insufficiency

summary: This clinical trial is designed to provide additional information on the safety and tolerability of vildagliptin (50 mg once daily (qd)) when used in patients with type 2 diabetes mellitus (T2DM) and moderate or severe renal insufficiency.

status: Completed

study_type: Interventional

design_allocation: Randomized

primary_purpose: Treatment

enrollment_type: Actual

lead_sponsor: Novartis

location_country:
Spain
Russian Federation
Australia
Finland
Argentina
Canada
Sweden
Costa Rica
France
India
Norway
Germany

intervention_type: Drug

lead_sponsor_type: industriel

status_simplified: Completed


acronym: EGN

title: A Two Year Multicentre, Randomized Two Arm Study Of Genotropin Treatment In Very Young Children Born Small For Gestational Age: Early Growth And Neurodevelopment(Egn)

study_completion_date_type: Actual

lead_sponsor: Pfizer

type: EudraCT Number

type: Other Identifier

source: euctr

ReferenceType: derived

ReferenceCitation: De Schepper J, Vanderfaeillie J, Mullis PE, Rooman R, Robertson A, Dilleen M, Gomez R, Wollmann HA. A 2-year multicentre, open-label, randomized, controlled study of growth hormone (Genotropin®) treatment in very young children born small for gestational age: Early Growth and Neurodevelopment (EGN) Study. Clin Endocrinol (Oxf). 2016 Mar;84(3):353-60. doi: 10.1111/cen.12968. Epub 2015 Nov 25.

location_country:
Spain
Italy
Netherlands
Czech Republic
Switzerland
Sweden
France
Belgium
Germany

location_facility: Universitair Ziekenhuis Antwerpen / Pediatrie

location_facility: CHU Toulouse, Hôpital des Enfants, Service Endocrinologie

location_facility: Dipartimento Materno Infantile, UO di Pediatria e Neonatologia

location_facility: Dipartimento di Medicina Pediatrica, UO di Endocrinologia e Diabetologia

location_facility: Fakultni nemocnice Ostrava, Detska klinika

location_facility: Drottning Silvias Barn och Ungdomssjukhus

location_facility: Universitaetsklinikum Erlangen, Kinder- und Jugendklinik

location_facility: Centro di Endocrinologia Pediatrica, Dipartimento di Pediatria

location_facility: Policlinico Universitario, Istituto di Clinica Pediatrica

summary: To demonstrate the effect on height and psychomotor development of Growth Hormone treatment in very young children starting at an age of 2 years.


title: Open-label Extension Study to Evaluate the Long-term Safety, Tolerability and Pharmacodynamics of AT1001 in Patients With Fabry Disease

summary: Study to evaluate the long-term safety, tolerability, and pharmacodynamics (PD) of migalastat hydrochloride (HCl) (migalastat) in participants with Fabry disease

study_start_date_type: Actual

status: Terminated

lead_sponsor: Amicus Therapeutics

location_country:
Brazil
United Kingdom
Australia
United States
France

status_simplified: Unknown


title: A Randomized, Open-label, Multi-center Trial Investigating Zalutumumab, a Human Monoclonal Anti-EGF Receptor Antibody, in Combination With Chemo-Radiation in Stage IIIA-IIIB Non Small Cell Lung Cancer Patients

summary: The purpose of the study is to investigate if treatment with zalutumumab in combination with chemotherapy and radiotherapy (chemo-radiation) will lead to a prolonged life in patients with lung cancer compared to patients treated with chemo-radiation alone.

lead_sponsor: Genmab

location_country:
United Kingdom
United States
France
Belgium
Netherlands

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

location_facility: CHU Sart-Tilman Domaine Universitaire du Sart-Tilman

intervention_type: Biological


title: A Multicenter, Randomized, Double-Blind, Placebo-Controlled,Parallel-Group Study to Evaluate the Safety and Efficacy of CC-5013 in the Treatment of Adolescents and Adults With Moderately Severe Crohn's Disease

summary: A Multicenter, Randomized, Double-Blind, Placebo-Controlled,Parallel-Group Study to Evaluate the Safety and Efficacy of CC-5013 in the Treatment of Adolescents and Adults with Moderately Severe Crohn's Disease

ReferenceType: background

ReferenceCitation: Mansfield JC, Parkes M, Hawthorne AB, Forbes A, Probert CS, Perowne RC, Cooper A, Zeldis JB, Manning DC, Hawkey CJ. A randomized, double-blind, placebo-controlled trial of lenalidomide in the treatment of moderately severe active Crohn's disease. Aliment Pharmacol Ther. 2007 Aug 1;26(3):421-30.

lead_sponsor: Celgene Corporation

location_country:
United States
Israel
United Kingdom
France

location_facility: Washington Hospital Center Physicians Office Building

location_facility: Department of Gastroenterology and Hepatology

location_facility: Department of Medicine A, Liver & Gastroenterology Units


title: A Randomized, Double-Blind, Parallel-Group, Placebo-Controlled, Dose-Response, Multicentre, Multinational Study Evaluating the Efficacy and Safety of AVE2268 Administered Either Twice Daily (Breakfast and Lunch) at a Dose of 300, 600 and 1200 mg or Once Daily (Breakfast) at a Dose of 1200 mg, in Patients With Type 2 Diabetes Treated With Metformin and Not Adequately Controlled

summary:
Dose-ranging study of AVE2268 in the management of patients with type 2 diabetes mellitus also receiving metformin.

Its main objectives will be to assess the effects of several doses of AVE2268 on Mean Plasma Glucose. Its secondary objectives will be to assess the effects of AVE2268 on plasma glucose (fasting and post-prandial), and also the safety and tolerability of AVE2268.

location_country:
Denmark
Poland
Italy
Australia
Netherlands
Argentina
France
Chile
South Africa
Belgium
Germany


title: A Multicenter, Double-blind, Randomized, Active-controlled, Parallel Group, Noninferiority Study Comparing 75mg Risedronate Dosed on 2 Consecutive Days Monthly With 5mg Daily Risedronate in the Treatment of Postmenopausal Osteoporosis as Assessed Over 24 Months.

summary:
To confirm the non-inferiority of 75 mg risedronate tablets taken on 2 consecutive days per month as compared to 5 mg risedronate tablets taken daily in increasing bone mass in lumbar spine in postmenopausal women with osteoporosis.

To confirm the efficacy of 75 mg risedronate tablets taken on 2 consecutive days per month in postmenopausal women with osteoporosis in increasing bone mass in proximal femur, femoral neck and femoral trochanter and decreasing bone resorption.

To confirm general safety of 75 mg risedronate tablets taken on 2 consecutive days per month as compared to 5 mg risedronate taken daily.

location_country:
Poland
United Kingdom
Australia
Lebanon
Argentina
Czech Republic
Canada
United States
France
Turkey
South Africa


title: Healthy Endothelial Accelerated Lining Inhibits Neointimal Growth. A Clinical, Multi-center, Prospective, Non-Randomized Study

summary:
This is a multi-center, prospective, non-randomized study. Approximately 90 patients from up to 16 centers will be entered in the study. Patients will be followed clinically for up to 5 years post-procedure. All patients will have a repeat angiography at 6 months follow-up.

The primary objective of this study is to evaluate the safety and effectiveness of the Genous Bio-engineered R stentTM in conjunction with optimal statin therapy (80mg of atorvastatin), in the treatment of elective patients with up to two de novo native coronary artery lesions. The Genous stent received CE mark for the intended indication in August 2005

design_allocation: Non-Randomized

ReferenceCitation: den Dekker WK, Houtgraaf JH, Onuma Y, Benit E, de Winter RJ, Wijns W, Grisold M, Verheye S, Silber S, Teiger E, Rowland SM, Ligtenberg E, Hill J, Wiemer M, den Heijer P, Rensing BJ, Channon KM, Serruys PW, Duckers HJ. Final results of the HEALING IIB trial to evaluate a bio-engineered CD34 antibody coated stent (Genous™Stent) designed to promote vascular healing by capture of circulating endothelial progenitor cells in CAD patients. Atherosclerosis. 2011 Nov;219(1):245-52. doi: 10.1016/j.atherosclerosis.2011.06.032. Epub 2011 Jun 25.

lead_sponsor: OrbusNeich

location_country:
United Kingdom
Austria
Netherlands
France
Belgium
Germany

location_facility: Herz- und Diabeteszentrum Nordrhein-Westfalen

intervention_type: Device


title: A Double-Blind, Multi-Center, Multinational, Randomized Withdrawal Study Evaluating the Efficacy and Safety of SR58611A (350mg q12) Versus Placebo in the Prevention of Depression Relapse up to 1 Year in Patients With Major Depressive Disorder Improved After 12 Weeks of Open Treatment With SR58611A (350mg q12)

acronym: CALYPSO

summary: The purpose of the study is to assess whether treatment with SR58611A can prevent relapse of depressive symptoms in patients with major depressive disorder. Relapse will be assessed using the MADRS scale.Patients who demonstrate improvement in depressive symptoms at the end of the initial 12-week open-label treatment period with SR58611A are randomized to continue SR58611A or switch to placebo under double blind conditions for up to 52 weeks of additional treatment. The secondary objective is to evaluate the safety of SR58611A in patients with MDD.

location_country:
Croatia
Poland
Romania
Serbia
Russian Federation
Argentina
Czech Republic
Finland
France
South Africa
Mexico
Bulgaria
Slovakia


title: A Multicenter, Randomized, Placebo-Controlled, Double-Blind, Parallel-Group, Fixed-Dose Study Evaluating the Effect of One Dose of Rimonabant (20 mg/Day) on Glycemic Control in Type 2 Diabetic Patients Inadequately Controlled With Insulin

summary:
Primary: Effect on HbA1c over 48 weeks in insulin-treated patients with type 2 diabetes

Secondary: Effect on glucose, total daily insulin dose, body weight, waist circumference, HDL-cholesterol, triglycerides - Safety, tolerability

location_country:
United Kingdom
Italy
Russian Federation
Australia
Netherlands
Argentina
Canada
United States
Chile
France
South Africa
Germany


title: A Multicenter, Open-Label, Randomized, Phase III Trial Comparing Immediate Adjuvant Hormonal Therapy (ELIGARD®- Leuprolide Acetate) in Combination With TAXOTERE® (Docetaxel) Administered Every Three Weeks Versus Hormonal Therapy Alone Versus Deferred Therapy Followed by the Same Therapeutic Options in Patients With Prostate Cancer at High Risk of Relapse After Radical Prostatectomy

summary:
This is a prospective, multicenter, open-label, randomized phase III study in participants at high risk of recurrent prostate cancer after radical prostatectomy. The study will investigate

Treatment with docetaxel (TAXOTERE®) every three weeks (q3w) plus leuprolide acetate (ELIGARD®) versus leuprolide acetate alone (ELIGARD®)
Immediate treatment following prostatectomy versus deferred treatment at the time of relapse

Using a 2x2 factorial design participants will therefore be randomized to

Immediate adjuvant treatment with docetaxel plus leuprolide acetate (chemotherapy and hormonal therapy)
Immediate adjuvant treatment with leuprolide acetate alone (hormonal therapy)
Deferred treatment with docetaxel plus leuprolide acetate (chemotherapy and hormonal therapy)
Deferred treatment with leuprolide acetate alone (hormonal therapy)

Primary Objective:

The primary objective of the study is to compare progression-free survival using a 2x2 factorial design

Secondary Objectives:

To compare the 5-year overall, cancer-specific and metastasis-free survival after systemic treatment between the groups
To compare the safety and tolerability between Docetaxel in combination with leuprolide acetate and leuprolide acetate alone.
To evaluate quality of life as measured by the FACT-P questionnaire.

Originally, 1696 participants were planned in the study (with 424 participants randomized to each arm). However, only a total of 211 participants completed the randomization procedure as of 26 September 2007. Thus, sanofi-aventis, in accordance with the Steering Committee, decided to stop the participant recruitment as of 26 September 2007. Participants who had already signed their Informed Consent (IC) before September 26, 2007 were allowed to enter the randomization if they met eligibility criteria. The final revised number of planned participants to be randomly assigned to the 4 treatment arms was 250, and 228 participants were actually randomized.

The final sample size did not allow all the statistical analyses to be conducted on efficacy data. Therefore, the protocol was amended to reflect the change in the plans for statistical analysis. The study was underpowered to serve as the basis for drawing conclusions regarding efficacy and quality of life (QoL) endpoints.

location_country:
Poland
Brazil
United Kingdom
Italy
Austria
Australia
Netherlands
Canada
Russian Federation
United States
France
India
Israel
Mexico
South Africa
Germany
Turkey


title: A Randomized, Double-blind, Placebo-controlled Phase II/III Trial to Evaluate the Efficacy and Safety of 2 Doses of Oral Liarozole (75 mg od and 150 mg od) Given During 12 Weeks in Lamellar Ichthyosis

summary: Lamellar ichthyosis is a congenital disease of the skin with a generalized scaling. The primary activity of liarozole is considered to be the inhibition of the degradation of a substance called retinoic acid, which is the principal endogenous regulator of growth and differentiation of epithelial tissues in mammals. The current study intends to evaluate the efficacy and safety in patients with lamellar ichthyosis.

ReferenceCitation: Van Wauwe JP, Coene MC, Goossens J, Cools W, Monbaliu J. Effects of cytochrome P-450 inhibitors on the in vivo metabolism of all-trans-retinoic acid in rats. J Pharmacol Exp Ther. 1990 Jan;252(1):365-9.

ReferenceCitation: Van Wauwe J, Van Nyen G, Coene MC, Stoppie P, Cools W, Goossens J, Borghgraef P, Janssen PA. Liarozole, an inhibitor of retinoic acid metabolism, exerts retinoid-mimetic effects in vivo. J Pharmacol Exp Ther. 1992 May;261(2):773-9.

ReferenceCitation: Van Wauwe J, Coene MC, Cools W, Goossens J, Lauwers W, Le Jeune L, Van Hove C, Van Nyen G. Liarozole fumarate inhibits the metabolism of 4-keto-all-trans-retinoic acid. Biochem Pharmacol. 1994 Feb 11;47(4):737-41.

ReferenceCitation: Kang S, Duell EA, Kim KJ, Voorhees JJ. Liarozole inhibits human epidermal retinoic acid 4-hydroxylase activity and differentially augments human skin responses to retinoic acid and retinol in vivo. J Invest Dermatol. 1996 Aug;107(2):183-7.

ReferenceCitation: Dockx P, Decree J, Degreef H. Inhibition of the metabolism of endogenous retinoic acid as treatment for severe psoriasis: an open study with oral liarozole. Br J Dermatol. 1995 Sep;133(3):426-32.

ReferenceCitation: Berth-Jones J, Todd G, Hutchinson PE, Thestrup-Pedersen K, Vanhoutte FP. Treatment of psoriasis with oral liarozole: a dose-ranging study. Br J Dermatol. 2000 Dec;143(6):1170-6.

ReferenceCitation: Bhushan M, Burden AD, McElhone K, James R, Vanhoutte FP, Griffiths CE. Oral liarozole in the treatment of palmoplantar pustular psoriasis: a randomized, double-blind, placebo-controlled study. Br J Dermatol. 2001 Oct;145(4):546-53.

ReferenceCitation: Lucker GP, Heremans AM, Boegheim PJ, van de Kerkhof PC, Steijlen PM. Oral treatment of ichthyosis by the cytochrome P-450 inhibitor liarozole. Br J Dermatol. 1997 Jan;136(1):71-5.

ReferenceCitation: Vahlquist A, Blockhuys S, Steijlen P, van Rossem K, Didona B, Blanco D, Traupe H. Oral liarozole in the treatment of patients with moderate/severe lamellar ichthyosis: results of a randomized, double-blind, multinational, placebo-controlled phase II/III trial. Br J Dermatol. 2014 Jan;170(1):173-81. doi: 10.1111/bjd.12626.

lead_sponsor: Stiefel, a GSK Company

location_country:
Dominican Republic
Italy
Netherlands
Canada
Sweden
France
Belgium
Norway
Germany

location_facility: Fondazione Policlinico Mangiagalli e Regina Elena

location_facility: Academisch Ziekenhuis Vrije Universiteit Brussel


title: An Open-Label, Multi-Centre, Extension Study, Evaluating the Long-Term Safety and Tolerability of Different Three-Month Degarelix Dosing Regimens in Patients With Prostate Cancer

summary: The purpose of this extension study was to collect long-term safety and tolerability information to support a marketing authorisation application for a three-month dosage regimen of degarelix.

lead_sponsor: Ferring Pharmaceuticals

location_country:
Montenegro
Romania
Serbia
United Kingdom
Russian Federation
Netherlands
Finland
France
Belgium
Germany

location_facility: Clinical Center of Serbia, Institute of Urology and Nephrology

location_facility: Pavlov State Medical University, Urology Department

location_facility: Mount Vernon Cancer Centre, Marie Cuire Research Wing

location_facility: Gemeinschaftspraxis Dres Effert und Benedic

location_facility: Fédération d'Urologie et Néphrologie, BP69 Hôpital Pasteur

location_facility: Clinical Center Novi Sad, Clinic of Urology

location_facility: Level 7, Urology Research Unit, Derriford Hospital

location_facility: Ward 13, NHS Forth Valley Acute Operating Division, Falkirk and District Royal Infirmary, Majors Loans

location_facility: "Centrul Medical Privat" Prof. Dr. Ioiart Ioan"

location_facility: Pavlov State Medical University, Outpatient Diagnostic Center affiliated with the Urology Department

location_facility: Clinical Hospital "Prof. Dr. Theodor Burghele", Urology Department

location_facility: Helsinki University Hospital, Maria Hospital, Building 11


title: An Open-Label, Multicenter Study Evaluating the Long-Term Safety and Efficacy of SR121463B in Patients With Syndrome of Inappropriate Antidiuretic Hormone Secretion

acronym: SIADH Safety

summary:
To determine the long-term safety and tolerability of SR121463B in patients with syndrome of inappropriate antidiuretic secretion (SIADH). To assess the long-term efficacy of SR121463B in maintaining normal levels of serum sodium in patients with SIADH.

Long term safety is evaluated further in an open label extension study with flexible doses of satavaptan.

location_country:
Brazil
Hong Kong
Russian Federation
Australia
Canada
Switzerland
Hungary
United States
France
Belgium
Germany

location_facility:
Sanofi-Aventis Administrative Office
sanofi-aventis Canada


title: Evaluation of Two Doses of SR31747A (75 mg and 125 mg) in Non-Metastatic Androgen-Independent Prostate Cancer. Randomized, Double-Blind, Placebo Controlled Phase II Study

acronym: ODYSSEY

summary: To evaluate the efficacy of SR31747 given at 75 or 125mg per day versus placebo in androgen prostate cancer patient without distant metastases with Time to Clinical progression as main objective and PSA parameters, Tumor response, survival , safety,Tumor-related symptoms deterioration Quality of Life, PK analysis as secondary objectives

location_country:
Poland
Portugal
Spain
Italy
United Kingdom
Australia
Mexico
Canada
Czech Republic
France
Chile
Belgium
Netherlands

location_facility: sanofi-aventis Australia & New Zealand administrative office


title: Phase III Randomized Trial of Concomitant Radiation, Cisplatin, and Tirapazamine Versus Concomitant Radiation and Cisplatin in Patients With Advanced Head and Neck Cancer

acronym: TRACE

summary: The trial will compare the efficacy and safety of concomitant chemoradiation with tirapazamine, cisplatin and radiation versus cisplatin and radiation.

location_country:
Poland
Taiwan
Hong Kong
Spain
Italy
Australia
New Zealand
Argentina
Canada
Switzerland
Hungary
United States
Chile
France
Singapore
South Africa
Germany

location_facility: sanofi-aventis New Zealand administrative office


title: Extension to a Multi-Center, Randomized, Crossover, Open Label, Dose Finding Study to Compare the Safety, Efficacy, and Pharmacokinetics/Pharmacodynamics (PK/PD) Relationship of Multiple Doses of Pasireotide (SOM230) (200, 400, and 600 μg Bid) and Doses of Open Label Sandostatin® (SMS) (100 μg Tid) in Acromegalic Patients

summary: Acromegaly is a rare, serious condition characterized by chronic hypersecretion of growth hormone (GH), generally caused by a GH-secreting pituitary adenoma. The study assessed the long-term safety and efficacy of pasireotide in participants with acromegaly.

ReferenceType: result

ReferenceCitation: Petersenn S, Farrall AJ, De Block C, Melmed S, Schopohl J, Caron P, Cuneo R, Kleinberg D, Colao A, Ruffin M, Hermosillo Reséndiz K, Hughes G, Hu K, Barkan A. Long-term efficacy and safety of subcutaneous pasireotide in acromegaly: results from an open-ended, multicenter, Phase II extension study. Pituitary. 2014 Apr;17(2):132-40. doi: 10.1007/s11102-013-0478-0. Erratum in: Pituitary. 2014 Apr;17(2):141. Block, Christophe [corrected to De Block, Christophe].

lead_sponsor: Novartis Pharmaceuticals

location_country:
Italy
Australia
Switzerland
United States
France
Belgium
Germany

location_facility: Cedars Sinai Medical Center Dept. of Pituitary Ctr.

location_facility: University of Michigan Health System StudyCoordinatorCSOM230B2201E1


summary: This study will evaluate the safety and efficacy of an intravitreal implant of dexamethasone for the treatment of macular edema associated with retinal vein occlusion.

ReferenceCitation: Haller JA, Bandello F, Belfort R Jr, Blumenkranz MS, Gillies M, Heier J, Loewenstein A, Yoon YH, Jacques ML, Jiao J, Li XY, Whitcup SM; OZURDEX GENEVA Study Group. Randomized, sham-controlled trial of dexamethasone intravitreal implant in patients with macular edema due to retinal vein occlusion. Ophthalmology. 2010 Jun;117(6):1134-1146.e3. doi: 10.1016/j.ophtha.2010.03.032. Epub 2010 Apr 24.

lead_sponsor: Allergan

location_country:
Taiwan
Portugal
Austria
Australia
Canada
United States
France
Israel
South Africa
Mexico
Philippines
Germany
Czechia

intervention_type: Other


title: A Single Dose, Randomized, 2-Period Crossover Study in Patients With Hemophilia A to Evaluate the Pharmacokinetics of ReFacto (B-domain Deleted Recombinant Human Factor VIII (BDDrFVIII) and Advante (a Full-length Recombinant Factor VIII (FLrFVIII)

summary: The purpose of the study is to compare the pharmacokinetic parameters of ReFacto and Advate, using the chromogenetic substrate assay to measure plasma Factor VIII activity in plasma.

status: Withdrawn

lead_sponsor: Wyeth is now a wholly owned subsidiary of Pfizer

location_country:
United Kingdom
Italy
Netherlands
New Zealand
United States
France
Belgium
Germany


title: Randomized, Double-Blind, Placebo-Controlled Study of the Safety, Efficacy and Pharmacokinetics of Myozyme in Patients With Late-Onset Pompe Disease.

summary: Pompe disease (also known as glycogen storage disease Type II) is caused by a deficiency of a critical enzyme in the body called acid alpha-glucosidase (GAA). Normally, GAA is used by the body's cells to break down glycogen (a stored form of sugar) within specialized structures called lysosomes. In patients with Pompe disease, an excessive amount of glycogen accumulates and is stored in various tissues, especially heart and skeletal muscle, which prevents their normal function. The overall objective is to evaluate the safety, efficacy, and pharmacokinetics (PK) of alglucosidase alfa treatment in patients with late-onset Pompe disease as compared to placebo.

ReferenceCitation: Forsha D, Li JS, Smith PB, van der Ploeg AT, Kishnani P, Pasquali SK; Late-Onset Treatment Study Investigators. Cardiovascular abnormalities in late-onset Pompe disease and response to enzyme replacement therapy. Genet Med. 2011 Jul;13(7):625-31. doi: 10.1097/GIM.0b013e3182142966.

ReferenceCitation: van der Ploeg AT, Clemens PR, Corzo D, Escolar DM, Florence J, Groeneveld GJ, Herson S, Kishnani PS, Laforet P, Lake SL, Lange DJ, Leshner RT, Mayhew JE, Morgan C, Nozaki K, Park DJ, Pestronk A, Rosenbloom B, Skrinar A, van Capelle CI, van der Beek NA, Wasserstein M, Zivkovic SA. A randomized study of alglucosidase alfa in late-onset Pompe's disease. N Engl J Med. 2010 Apr 15;362(15):1396-406. doi: 10.1056/NEJMoa0909859.

lead_sponsor: Genzyme, a Sanofi Company

location_country:
Netherlands
United States
France

location_facility: University of Pittsburgh, Dept. of Neurology


status: Unknown status

intervention_type: Radiation

title: Randomized Phase III Study Of Neoadjuvant Chemotherapy Followed By Surgery Vs. Concomitant Radiotherapy And Chemotherapy In FIGO Ib2, IIa>4 cm or IIb Cervical Cancer

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

enrollment_type: Anticipated

location_country:
Portugal
Spain
Italy
Austria
United Kingdom
France
Belgium
Netherlands

location_facility: Queen Elizabeth The Queen Mother Hospital

location_facility: Universitair Medisch Centrum - Academisch Ziekenhuis

location_facility: Academisch Medisch Centrum at University of Amsterdam

location_facility: Hospitais da Universidade de Coimbra (HUC)

location_facility: Universitair Medisch Centrum St. Radboud - Nijmegen

location_facility: NHS Greater Glasgow and Clyde - Beatson West of Scotland Cancer Centre

location_facility: Istituto Nazionale Per Lo Studio E La Cura Dei Tumori

location_facility: Erasmus MC - Daniel den Hoed Cancer Center

location_facility: Centre Hospitalier Regional de la Citadelle

summary:
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Giving chemotherapy drugs before surgery may shrink the tumor so that it can be removed during surgery. Radiation therapy uses high-energy x-rays to kill tumor cells. Combining radiation therapy with chemotherapy may kill more tumor cells. It is not yet known whether chemotherapy is more effective followed by surgery or combined with radiation therapy in treating cervical cancer.

PURPOSE: Randomized phase III trial to compare the effectiveness of chemotherapy followed by radical hysterectomy with that of chemotherapy plus radiation therapy in treating patients who have stage IB or stage II cervical cancer.


title: A Randomized Phase II Study Of CMF Alone And In Combination With Anti c-erbB2 Antibody (Herceptin) In Women With c-erbB2 Positive Metastatic Breast Cancer

summary:
RATIONALE: Drugs used in chemotherapy such as cyclophosphamide, methotrexate, and fluorouracil use different ways to stop tumor cells from dividing so they stop growing or die. Monoclonal antibodies, such as trastuzumab, can locate tumor cells and either kill them or deliver tumor-killing substances to them without harming normal cells. Combining chemotherapy with trastuzumab may kill more tumor cells.

PURPOSE: Phase II trial to study the effectiveness of combining combination chemotherapy with trastuzumab in treating women who have metastatic breast cancer.

ReferenceCitation: Neskovic-Konstantinovic Z, Nooij M, Khaled H, et al.: Safety and efficacy of combined trastuzumab and CMF therapy in women with metastatic breast cancer: EORTC protocol 10995. [Abstract] J Clin Oncol 25 (Suppl 18): A-1040, 2007.

ReferenceCitation: Tryfonidis K, Marreaud S, Khaled H, De Valk B, Vermorken J, Welnicka-Jaskiewicz M, Aalders K, Bartlett JMS, Biganzoli L, Bogaerts J, Cameron D; EORTC- Breast Cancer Group. Cardiac safety, efficacy, and correlation of serial serum HER2-extracellular domain shed antigen measurement with the outcome of the combined trastuzumab plus CMF in women with HER2-positive metastatic breast cancer: results from the EORTC 10995 phase II study. Breast Cancer Res Treat. 2017 Jun;163(3):507-515. doi: 10.1007/s10549-017-4203-y. Epub 2017 Mar 21.

location_country:
Denmark
Poland
Serbia
United Kingdom
France
South Africa
Belgium
Netherlands
Egypt

location_facility: Herlev Hospital - University Hospital of Copenhagen

location_facility: Nijmegen Cancer Center at Radboud University Medical Center

location_facility: Institute of Oncology and Radiology of Serbia

location_facility: Centre de Lutte Contre le Cancer Georges-Francois Leclerc


title: International Collaborative Treatment Protocol for Infants Under One Year With Acute Lymphoblastic Leukemia

summary:
RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining more than one drug may kill more cancer cells. It is not yet known which combination chemotherapy regimen is most effective for treating infants with acute lymphoblastic leukemia.

PURPOSE: Randomized phase III trial to compare the effectiveness of different combination chemotherapy regimens in treating infants who have newly diagnosed acute lymphoblastic leukemia.

ReferenceCitation: Hempel G, Relling MV, de Rossi G, Stary J, De Lorenzo P, Valsecchi MG, Barisone E, Boos J, Pieters R. Pharmacokinetics of daunorubicin and daunorubicinol in infants with leukemia treated in the interfant 99 protocol. Pediatr Blood Cancer. 2010 Mar;54(3):355-60. doi: 10.1002/pbc.22266.

ReferenceCitation: Mann G, Attarbaschi A, Schrappe M, De Lorenzo P, Peters C, Hann I, De Rossi G, Felice M, Lausen B, Leblanc T, Szczepanski T, Ferster A, Janka-Schaub G, Rubnitz J, Silverman LB, Stary J, Campbell M, Li CK, Suppiah R, Biondi A, Vora A, Valsecchi MG, Pieters R; Interfant-99 Study Group. Improved outcome with hematopoietic stem cell transplantation in a poor prognostic subgroup of infants with mixed-lineage-leukemia (MLL)-rearranged acute lymphoblastic leukemia: results from the Interfant-99 Study. Blood. 2010 Oct 14;116(15):2644-50. doi: 10.1182/blood-2010-03-273532. Epub 2010 Jun 30.

ReferenceCitation: Lönnerholm G, Valsecchi MG, De Lorenzo P, Schrappe M, Hovi L, Campbell M, Mann G, Janka-Schaub G, Li CK, Stary J, Hann I, Pieters R; Interfant-99 study group. Pharmacokinetics of high-dose methotrexate in infants treated for acute lymphoblastic leukemia. Pediatr Blood Cancer. 2009 May;52(5):596-601. doi: 10.1002/pbc.21925.

ReferenceCitation: van der Linden MH, Valsecchi MG, De Lorenzo P, Möricke A, Janka G, Leblanc TM, Felice M, Biondi A, Campbell M, Hann I, Rubnitz JE, Stary J, Szczepanski T, Vora A, Ferster A, Hovi L, Silverman LB, Pieters R. Outcome of congenital acute lymphoblastic leukemia treated on the Interfant-99 protocol. Blood. 2009 Oct 29;114(18):3764-8. doi: 10.1182/blood-2009-02-204214. Epub 2009 Aug 5.

ReferenceCitation: Van der Velden VH, Corral L, Valsecchi MG, Jansen MW, De Lorenzo P, Cazzaniga G, Panzer-Grümayer ER, Schrappe M, Schrauder A, Meyer C, Marschalek R, Nigro LL, Metzler M, Basso G, Mann G, Den Boer ML, Biondi A, Pieters R, Van Dongen JJ; Interfant-99 Study Group. Prognostic significance of minimal residual disease in infants with acute lymphoblastic leukemia treated within the Interfant-99 protocol. Leukemia. 2009 Jun;23(6):1073-9. doi: 10.1038/leu.2009.17. Epub 2009 Feb 12.

ReferenceCitation: Pieters R, Schrappe M, De Lorenzo P, Hann I, De Rossi G, Felice M, Hovi L, LeBlanc T, Szczepanski T, Ferster A, Janka G, Rubnitz J, Silverman L, Stary J, Campbell M, Li CK, Mann G, Suppiah R, Biondi A, Vora A, Valsecchi MG. A treatment protocol for infants younger than 1 year with acute lymphoblastic leukaemia (Interfant-99): an observational study and a multicentre randomised trial. Lancet. 2007 Jul 21;370(9583):240-250. doi: 10.1016/S0140-6736(07)61126-X.

ReferenceCitation: Pieters R, Schrappe M, de Lorenzo P, et al.: Outcome of infants less than one year of age with acute lymphoblastic leukemia treated with the Interfant-99 protocol. [Abstract] Blood 108 (11): A-145, 2006.

lead_sponsor: Dutch Childhood Oncology Group

location_country:
United Kingdom
Ireland
Austria
Italy
Netherlands
Czech Republic
Sweden
France
United States
Belgium
Germany

location_facility: Our Lady's Hospital for Sick Children Crumlin

location_facility: Central Manchester and Manchester Children's University Hospitals NHS Trust

location_facility: Nuovo Ospedale San Gerardo at University of Milano-Bicocca

location_facility: Royal Liverpool Children's Hospital, Alder Hey

location_facility: Institute of Child Health at University of Bristol

location_facility: Royal Marsden NHS Foundation Trust - Surrey

location_facility: Hopital Universitaire Des Enfants Reine Fabiola

location_facility: Sir James Spence Institute of Child Health

location_facility: Dana-Farber/Harvard Cancer Center at Dana Farber Cancer Institute

location_facility: Southampton University Hospital NHS Trust

location_facility: University Medical Center Hamburg - Eppendorf

location_facility: Great Ormond Street Hospital for Children NHS Trust

location_facility: Addenbrooke's Hospital at Cambridge University Hospitals NHS Foundation Trust

location_facility: Leeds Cancer Centre at St. James's University Hospital

location_facility: Royal Belfast Hospital for Sick Children


title: Neuropsychological Evaluation of Psychiatric and Neurological Patients

summary:
This study will allow researchers to use various types of tests to evaluate cognitive and sensory functions. These tests, referred to as "batteries" will evaluate attention, executive functions, general intellectual functioning, language, memory, motor functions, orientation, personality, selected sensory and perceptual functions, vigilance (alertness), and visual-spatial functions. Children and adult patient will receive different test batteries.

The goals of this research study are to;

Create descriptions based on the performance of each patient on the test batteries. Then use this information to relate patient behavior to their neurophysiological, neuroradiological, and biochemical descriptions.
Define subgroups of patients based on their neurobehavior in order to decrease the variability of psychiatric diagnoses, treatments, and prognoses.

study_type: Observational

ReferenceCitation: Johnstone EC, Owens DG, Bydder GM, Colter N, Crow TJ, Frith CD. The spectrum of structural brain changes in schizophrenia: age of onset as a predictor of cognitive and clinical impairments and their cerebral correlates. Psychol Med. 1989 Feb;19(1):91-103.

lead_sponsor: National Institute of Mental Health (NIMH)

location_country:
Russian Federation
United States
France

location_facility: National Institutes of Health Clinical Center, 9000 Rockville Pike


title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Deucravacitinib in Participants With Active Systemic Lupus Erythematosus (SLE) (POETYK SLE-1)

acronym: POETYK SLE-1

summary: The purpose of this study is to evaluate the effectiveness and safety of deucravacitinib compared with placebo in an active moderate to severe Systemic Lupus Erythematosus (SLE) population.

study_start_date_type: Anticipated

status: Not yet recruiting

study_completion_date_type: Anticipated

ipd_sharing: Yes

ipd_sharing_description:
BMS will provide access to individual anonymized participant data upon request from qualified researchers, and subject to certain criteria.

Additional information regarding Bristol Myer Squibb's data sharing policy and process can be found at:

https://www.bms.com/researchers-and-partners/clinical-trials-and-research/disclosurecommitment.html

lead_sponsor: Bristol-Myers Squibb

location_country:
Poland
Brazil
Hong Kong
Portugal
Ireland
Italy
Mexico
Romania
Argentina
Canada
Korea, Republic of
United States
France
China
Peru
Colombia
Germany

location_facility: Indiana University Health University Hospital

location_facility: Private Practice - Dr. Chandrakant V. Mehta

location_facility: Advanced Rheumatology of Houston - Woodlands

location_facility: Dallas Fort Worth Clinical Research Associates - North Riverside Drive

location_facility: Arthritis & Rheumatology Research Institute

location_facility: University of Florida College of Medicine

location_facility: University of Miami Miller School of Medicine

status_simplified: Ongoing


title: Universal Rare Gene Study: A Registry and Natural History Study of Retinal Dystrophies Associated With Rare Disease-Causing Genetic Variants

acronym: Uni-Rare

summary:
This is an international, multicenter study with two components:

Registry

A standardized genetic screening and a prospective, standardized, cross-sectional clinical data collection
Enrollment is open to all genes on the RD Rare Gene List

Natural History Study

A prospective, standardized, longitudinal Natural History Study
Enrollment opens gene-by-gene, based on funding and within-gene Registry enrollment The study objectives are as follows.

Registry Objectives

Genotype Characterization
Cross-Sectional Phenotype Characterization (within gene)
Establish a Link to My Retina Tracker Registry (MRTR)
Ancillary Exploratory Studies - Pooling of Genes

Natural History Study Objectives

Natural History (within gene)
Structure-Function Relationship (within gene)
Risk Factors for Progression (within gene)
Ancillary Exploratory Studies - Pooling of Genes

time_perspective: Cross-Sectional

ipd_sharing_description: A de-identified database is placed in the public domain on the FFB/Jaeb public website after the completion of each protocol and publication of the manuscript.

lead_sponsor: Jaeb Center for Health Research

location_country:
United Kingdom
Finland
Canada
United States
Israel
France
Netherlands

location_facility: Harvard Univ., Massachusetts Eye and Ear Infirmary

location_facility: University of Michigan, Kellogg Eye Center

location_facility: Johns Hopkins University, Wilmer Eye Institute

location_facility: University of Utah, John Moran Eye Center

location_facility: University of Arkansas, Jones Eye Institute

location_facility: Hadassah-Hebrew University Medical Center

location_facility: University of Miami, Bascom Palmer Eye Institute

location_facility: University of Toronto, Hospital for Sick Children

location_facility: Medical College of Wisconsin Eye Institute

location_facility: Univ. of California San Diego, Jacobs Retina Center

location_facility: Baylor College of Medicine, Alkek Eye Center

location_facility: Oregon Health & Science Univ., Casey Eye Institute


type: Registry Identifier

title: A Randomized, Double-blind, Placebo-controlled Clinical Study to Evaluate Mavacamten in Adults With Symptomatic Non-obstructive Hypertrophic Cardiomyopathy

summary: The purpose of this study is to evaluate the safety, tolerability, and efficacy of mavacamten compared with placebo in participants with symptomatic non-obstructive hypertrophic cardiomyopathy (nHCM).

ipd_sharing_description: BMS will provide access to individual anonymized participant data upon request from qualified researchers, and subject to certain criteria. Additional information regarding Bristol Myers Squibb's data sharing policy and process can be found at: https://www.bms.com/researchers-and-partners/clinical-trials-and-research/disclosurecommitment.html

location_country:
Denmark
Poland
Brazil
Portugal
Spain
United Kingdom
Austria
Australia
Netherlands
Canada
Hungary
United States
France
Israel
Norway
Belgium
Japan
Germany

location_facility: Washington University School of Medicine

location_facility: North Shore University Hospital-(Manhasset)

location_facility: University of Texas Southwestern Medical Center

location_facility: Indiana University School of Medicine-Indianapolis


title: Avalus European Registry - an Observational Study to Evaluate Safety and Efficacy in a Real World Population

acronym: Avalus

summary: This is a prospective, observational, single-arm, multi-center registry of patients undergoing bioprosthetic aortic valve replacement with the Avalus valve. This prospective registry aims to examine the outcome and performance of surgical aortic valve replacement with the Avalus pericardial bioprosthesis.

status: Recruiting

time_perspective: Prospective

ipd_sharing: No

lead_sponsor: Universitaire Ziekenhuizen KU Leuven

location_country: Denmark Spain Italy Netherlands Finland France Belgium Germany

location_facility:
CHU Nantes
Osp. S.Orsola Malpighi
Policlinico San Donato S.P.A
A. Sanitaria O. Ordine Mauriziano
UZ Gent
Kuopio University Hospital
UMC Maastricht
UCL St Luc
UZ Leuven
Rigshospitalet
European Hospital
Fondazione Poliambulanza
Villa Maria Cecilia
Hospital Rambla
ZOL
Universität Herzzentrum
AZ Maria Middelares
Hospital Clinico De Valladolid
Schüchtermann-Klinik

lead_sponsor_type: academique


title: A Multi-center, Randomized, Double-blind, Placebo-controlled, Parallel Group, Phase 2 Study to Evaluate Clinical Efficacy and Safety of Deucravacitinib (BMS-986165) in Participants With Alopecia Areata

summary: The purpose of this study is to evaluate the efficacy of Deucravacitinib versus placebo at Week 24 and safety and tolerability of Deucravacitinib versus placebo in adults with Alopecia Areata.

location_country:
Poland
Australia
Canada
United States
France
Japan

location_facility: Jordan Valley Dermatology and Research Center

location_facility: Houston Skin Associates - (Dermatological Association of Texas)

location_facility: Midwest Center for Dermatology and Cosmetic Surgery - Clinto

location_facility: University of North Carolina Medical Center

location_facility: DermResearch Inc. Clinical Research Company

location_facility: Progressive Clinical Research - San Antonio

location_facility: University of California, Irvine (UCI) Health - UC Irvine Medical Center

location_facility: Dermatology Institute & Skin Care Center (DISCC)


title: An Open-Label Multicenter Study to Evaluate the Pharmacokinetics, Pharmacodynamics, and Safety of Inebilizumab in Pediatric Subjects With Neuromyelitis Optica Spectrum Disorder

summary: A Phase 2, open-label, multicenter study to evaluate the pharmacokinetics (PK), pharmacodynamics (PD), and safety of inebilizumab in eligible pediatric participants 2 to < 18 years of age with recently active neuromyelitis optica spectrum disorder (NMOSD) who are seropositive for autoantibodies against aquaporin-4 (AQP4-immunoglobulin [Ig]G).

lead_sponsor: Horizon Therapeutics Ireland DAC

location_country:
Poland
Brazil
Serbia
Spain
United Kingdom
Argentina
Canada
United States
France
Sweden

location_facility: Centre Hospitalier Universitaire de Bicêtre

location_facility: Great Ormond Street Hospital for Children

location_facility: Clinic for Neurology and Psychiatry for Children and Youth

location_facility: Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo

location_facility: Hospital de Pediatría S.A.M.I.C.- Prof. Dr. Juan P. Garrahan

location_facility: UC San Diego Altman Clinical and Translational Research Institute (ACTRI) Building

location_facility: Hospital Sao Lucas Da Pontificia Universidade Catolica Do Rio Grande Do Sul (PUCRS)


title: Long-Term Open-Label Study to Assess the Safety and Efficacy of Vatiquinone in Patients With Friedreich Ataxia

summary: The primary objective of this study is to assess the long-term safety of vatiquinone in participants with Friedreich ataxia (FA) previously exposed to vatiquinone in Study PTC743-NEU-003-FA (NCT04577352) or Study PTC743-NEU-005-FA.

lead_sponsor: PTC Therapeutics

location_country:
Brazil
Spain
Italy
Australia
New Zealand
Canada
United States
France
Germany

location_facility: CBR Neurogenetic Research Clinic, University of Auckland

location_facility: Hospital Sant Joan de Déu Barcelona Unidad de Enfermedades Neuromusculares

location_facility: Hôpital Pitié-Salpêtrière, Institut du Cerveau (Paris Brain Institute)

location_facility: Department of Neurology and Hertie-Institute for Clinical Brain Research German Center of Neurodegenerative Diseases (DZNE)

location_facility: University of Campinas (UNICAMP) - School of Medical Sciences, Dept of Neurology

location_facility: Centre de Recherche du Centre Hospitalier de l'Université de Montreal (CRCHUM)


title: An 18-month, Open-label, Single-arm Safety Extension Study of an age-and Bodyweight-adjusted Oral Finerenone Regimen, in Addition to an ACEI or ARB, for the Treatment of Children and Young Adults From 1 to 18 Years of Age With Chronic Kidney Disease and Proteinuria

acronym: FIONA OLE

summary:
Researchers are looking for a better way to treat children who have chronic kidney disease (CKD), which is long-term kidney disease, and proteinuria, a condition in which a person´s kidneys leak protein into the urine.

The kidneys filter waste and fluid from the blood to form urine. In children with CKD, the kidney´s filters do not work as well as they should. This can lead to accumulation of waste and fluid in the body and proteinuria. CKD can lead to other medical problems, such as high blood pressure, also known as hypertension. Vice versa, hypertension and proteinuria can also contribute to worsening of CKD. Therefore, the treatment of CKD aims to control blood pressure and proteinuria. There are treatments available for doctors to prescribe to children with CKD and hypertension and/or proteinuria. These include "angiotensin-converting enzyme inhibitors" (ACEI) and "angiotensin receptor blockers" (ARB). Both ACEI and ARB can help improve kidney function by reducing the activity of the renin-angiotensin-aldosterone system (RAAS). The RAAS is a system that works with the kidneys to control blood pressure and the balance of fluid and electrolytes in the blood. In people with CKD, the RAAS is often too active, which can impair the ability of the kidneys to work properly and cause hypertension and proteinuria. However, ACEI or ARB treatment alone does not work for all patients with CKD as they only target the angiotensin part of the renin-angiotensin-aldosterone system.

The study treatment, finerenone, is expected to help control RAAS overactivation together with an ACEI or ARB.

So, the researchers in this study want to learn more about whether finerenone given in addition to either an ACEI or ARB can help their kidney function.

The main purpose of this study is to learn how safe the treatment is when used of finerenone in addition to an ACEI or ARB in long-term.

To see how safe the treatment is, the study team will collect information on medical problems which are also known as "treatment emergent adverse events" (TEAEs). And they will also collect levels of an electrolyte called potassium in the blood by taking blood samples, and measure blood pressure during the study.

The secondary purpose of this study is to learn how well long-term use of finerenone can reduce the amount of protein in the participants' urine and benefit kidney function when taken with standard of care.

To see how the treatment works, the study team will collect participants' urine samples to assess urinary albumin-to-creatinine ratio (UACR) and urinary protein-to-creatinine ratio (UPCR), which are important assessments for calculating the level of protein in the urine. Researchers will also collect blood samples to analyze serum creatinine and calculate estimated glomerular filtration rate (eGFR). A significant decline in eGFR indicates worsening kidney function.

The study will include participants who had previously participated in FIONA study (NCT05196035). The participants will be aged from 1 year up to 18 years.

The participants will be in the study for approximately 19 months. They will take study treatment for up to 18 months and will be follow up for 1 month. During this period, at least 12 visits are planned for patients who newly start finerenone, and at least 8 visits for patients who already received finerenone.

In the visit, the study team will:

have their blood pressure, heart rate, temperature, height and weight measured
have blood and urine samples taken
have physical examinations
have their heart examined by an electrocardiogram and echocardiography (a sonogram of the heart)
answer questions about their medication and whether they have any adverse events, or have their parents or guardian's answer
answer questions about how they are feeling, or have their parents or guardian's answer
answer question about how they like the study medication, or have their parents or guardian's answer The doctors will keep track of any adverse events. An adverse event is any medical problem that a participant has during a study. Doctors keep track of all adverse events that happen in studies, even if they do not think the adverse events might be related to the study treatments.

The doctors will check the participants' health about 30 days after the participants take their last treatment.

ipd_sharing_description:
Availability of this study's data will later be determined according to Bayer's commitment to the EFPIA/PhRMA "Principles for responsible clinical trial data sharing". This pertains to scope, timepoint and process of data access. As such, Bayer commits to sharing upon request from qualified researchers patient-level clinical trial data, study-level clinical trial data, and protocols from clinical trials in patients for medicines and indications approved in the US and EU as necessary for conducting legitimate research. This applies to data on new medicines and indications that have been approved by the EU and US regulatory agencies on or after January 01, 2014.

Interested researchers can use www.vivli.org to request access to anonymized patient-level data and supporting documents from clinical studies to conduct research. Information on the Bayer criteria for listing studies and other relevant information is provided in the member section of the portal.

lead_sponsor: Bayer

location_country:
Spain
Italy
United Kingdom
Lithuania
Belgium
Czechia
Poland
Portugal
Austria
Australia
Israel
Turkey
Sweden
Canada
Switzerland
Hungary
Germany
Denmark
Greece
Finland
Korea, Republic of
France
Netherlands


title: A Phase 1 Study of VAC85135, a Neoantigen Vaccine Regimen, Concurrently Administered With Ipilimumab for the Treatment of Myeloproliferative Neoplasms

summary: The purpose of this study is to evaluate the safety of VAC85135 administered with ipilimumab for the treatment of myeloproliferative neoplasms (MPNs).

ipd_sharing_description: The data sharing policy of the Janssen Pharmaceutical Companies of Johnson & Johnson is available at www.janssen.com/clinical-trials/transparency. As noted on this site, requests for access to the study data can be submitted through Yale Open Data Access (YODA) Project site at yoda.yale.edu

lead_sponsor: Janssen Research & Development, LLC

location_country:
United Kingdom
Spain
Canada
United States
France

location_facility: University Health Network (UHN) Princess Margaret Cancer Centre

location_facility: The Christie NHS Foundation Trust - Christie Hospital

location_facility: Vanderbilt University Medical Center (VUMC) - Vanderbilt-Ingram Cancer Center (VICC)


title: Microbiome-based Stratification of Individuals at Risk of HIV-1 Acquisition, Chronic Clinical Complications, Antimicrobial Drug Resistance, and Unresponsiveness to Therapeutic HIV-1 Vaccination

acronym: MISTRAL

summary:
MISTRAL (Microbiome-based stratification of individuals at risk of HIV-1 acquisition, chronic clinical complications, antimicrobial drug resistance, and unresponsiveness to therapeutic HIV-1 vaccination) is a 5-year EU Horizon 2020 project, running from 1/1/2020 - 31/12/2024.

The project is led by Fundacio Privada Institut de Recerca de la Sida-Caixa CAIXA in Barcelona and aims to explore the gut microbiota in relation to HIV-1, seeking microbiome biomarkers to support development of interventions that mitigate infection and enhance response to vaccines and therapies.

If successful, MISTRAL will benefit millions of human beings living with, or at risk of acquiring HIV-1 infection, and will produce novel concepts and technical innovations applicable to other human diseases. By doing that, MISTRAL will help to unlock the full clinical potential of the human microbiome to stratify patient outcomes and will irreversibly bring microbiome science closer to clinical practice

ReferenceCitation: Li SX, Armstrong A, Neff CP, Shaffer M, Lozupone CA, Palmer BE. Complexities of Gut Microbiome Dysbiosis in the Context of HIV Infection and Antiretroviral Therapy. Clin Pharmacol Ther. 2016 Jun;99(6):600-11. doi: 10.1002/cpt.363. Epub 2016 Apr 16. Review.

ReferenceCitation: Herrera S, Martínez-Sanz J, Serrano-Villar S. HIV, Cancer, and the Microbiota: Common Pathways Influencing Different Diseases. Front Immunol. 2019 Jun 27;10:1466. doi: 10.3389/fimmu.2019.01466. eCollection 2019. Review.

ReferenceCitation: Hsu DC, Sereti I, Ananworanich J. Serious Non-AIDS events: Immunopathogenesis and interventional strategies. AIDS Res Ther. 2013 Dec 13;10(1):29. doi: 10.1186/1742-6405-10-29.

lead_sponsor: Rigshospitalet, Denmark

location_country:
Denmark
Croatia
Poland
Luxembourg
Spain
Greece
Italy
United Kingdom
Hungary
France
Israel
Belgium
Germany
Czechia

location_facility: University Hospital Bonn, Klinisches StudieZentrum Immun.

location_facility: University Hospital of Infectious Diseases

location_facility: Hosp. Universitari Germans Trias i Pujol, Hospital de dia VIH

location_facility: Wojewodzki Szpital Specjalistyczny im. WI. Bieganskiego

location_facility: Hospital Clinic - IDIBAPS. University of Barcelona, C/Villarroel, 170

location_facility: University College London, Mortimer Market Centre

location_facility: Royal Sussex County Hospital, HIV/GUM Research


title: A Randomized, Open-label, Phase 3 Study of Sacituzumab Govitecan Versus Treatment of Physician's Choice in Patients With Previously Untreated, Locally Advanced, Inoperable or Metastatic Triple-Negative Breast Cancer Whose Tumors Do Not Express PD-L1 or in Patients Previously Treated With Anti-PD-(L)1 Agents in the Early Setting Whose Tumors Do Express PD-L1

summary: The primary objective of this study is to compare the progression-free survival (PFS) between sacituzumab govitecan-hziy (SG) versus treatment of physician's choice (TPC) in participants with previously untreated, locally advanced, inoperable or metastatic triple-negative breast cancer whose tumors do not express programmed cell death ligand 1 (PD-L1) or in participants previously treated with anti-programmed cell death (ligand or protein) 1 (Anti-PD-(L)1) Agents in the early setting whose tumors do express PD-L1.

lead_sponsor: Gilead Sciences

location_country:
Spain
Switzerland
Puerto Rico
United States
France

location_facility: Kantonsspital Baselland - Standort Liestal

location_facility: Northwest Georgia Oncology Northwest Georgia Oncology Centers, PCCenters, PC

location_facility: Florida Cancer Specialists - West Palm Beach

location_facility: Florida Cancer Specialists - North Region Research Office

location_facility: Eastern Connecticut Hematology and Oncology Associates

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

location_facility: Hematology Oncology Clinic - Baton Rouge

location_facility: Hematology Oncology Associates of Central New York


acronym: DELTA TEEN

title: A Phase 3 Clinical Trial to Evaluate Efficacy and Safety of Twice-daily Applications of Delgocitinib Cream 20 mg/g Compared With Cream Vehicle for a 16-week Treatment Period in Adolescents 12-17 Years of Age With Moderate to Severe Chronic Hand Eczema

lead_sponsor: LEO Pharma

location_country:
Poland
Spain
United Kingdom
Australia
Canada
France
Belgium

summary:
The purpose of this trial is to test if delgocitinib cream is effective at treating chronic hand eczema (CHE) and what side effects it may have, in children aged 12-17. There will be a range of assessments that rate the severity and extent of CHE symptoms, general health and quality of life.

Delgocitinib is a cream that suppresses specific processes in the body's response to diseases like CHE, such as inflammation.

The trial will last up to 22 weeks and has a 1-4 week screening period, a 16 week treatment period and a 2 week follow up period. During the treatment period each child will use either delgocitinib cream or a cream vehicle twice a day. Which cream each child receives is chosen randomly by a computer. The cream vehicle is made of the same ingredients as the delgocitinib cream except for the active medical ingredient. There will be 8 visits with the trial doctor.


title: FINE-REAL: A Non-interventional Study Providing Insights Into the Use of Finerenone in a Routine Clinical Setting

summary:
This is an observational study in people with chronic kidney disease (CKD) and type 2 diabetes (T2D) who will be receiving finerenone.

Kidneys filter extra water and waste out of the blood and make urine. CKD is a long-term, progressive, decrease in the kidneys' ability filter the blood properly. In people with T2D, the body does not make enough of a hormone called insulin, or does not use insulin well enough, resulting in high blood sugar levels that can cause damage to the kidneys. As a result, CKD can occur as a complication of T2D.

Finerenone works by blocking certain proteins, called mineralocorticoid receptors. An increased stimulation of these proteins is thought to damage the kidneys and the heart. By lowering their stimulation, finerenone reduces the risk of kidney disease progressively getting worse.

Finerenone is available and approved for doctors to prescribe to people with CKD and T2D. Since it has only recently become available for these patients, there is a need for more information about the use of finerenone in the real-world setting.

The main purpose of the study is to learn more about treatment patterns in people with CKD and T2D who will start finerenone treatment as decided and prescribed by their doctor as part of their routine medical care.

To answer this question, the researchers will collect data on:

Clinical characteristics (e.g., history of CKD and T2D, blood pressure, heart health) of the participants
Reasons for starting finerenone
Reasons for stopping finerenone early
How long participants have been taking finerenone (planned by their doctor compared to actual time it was taken)
Dosing of finerenone
Other medications used while taking finerenone

The researchers will also collect data on medical problems (called adverse events) that the participants may have during the study. All adverse events are collected, even if they might not be related to the study treatment.

Hyperkalemia, a medical term used to describe a potassium level in the blood that is higher than normal, is of special interest when finerenone is combined with some medications commonly taken to control blood pressure. Researchers want to know how often higher potassium levels occur, and when it leads to:

Stopping of finerenone treatment
Dialysis (a medical procedure to filter the blood of extra water and waste)
Care in a hospital

All data will come from medical records or from interviews study doctors will have with the participants during visits that take place during routine medical care.

Participants in the US will be invited to provide voluntary blood and urine samples that could be analyzed later to better understand possible changes in protein or nucleic acid levels over time.

Each participant will be in the study for 12 months. This time participating in the study may be shorter if their finerenone treatment is stopped early or the study comes to an end as planned in November 2025.

location_country:
Luxembourg
Saudi Arabia
Italy
Spain
United States
Belgium
Slovenia
Russian Federation
Argentina
Mexico
Singapore
Brazil
Canada
Switzerland
China
Germany
Denmark
Taiwan
Thailand
France
Colombia
Netherlands

location_facility: Academy of Diabetes Thyroid and Endocrine

location_facility: University of Maryland Midtown Professionals Group Healthcare

location_facility: Keck School of Medicine of University of Southern California

location_facility: Jackson Medical Mall University Home Dialysis Clinic

location_facility: Kidney and Hypertension Center of Wabash Valley LLC

location_facility: University of Iowa Preventive Intervention Clinic

location_facility: Nephrology Associates Northern Illinois and Indiana

location_facility: MultiCare Endocrinology Specialists Tacoma

location_facility: University of Central Florida College of Medicine


title: A Phase 1b/2, Multicenter, Open-label Basket Study Evaluating the Safety and Efficacy of Bemarituzumab Monotherapy in Solid Tumors With FGFR2b Overexpression (FORTITUDE-301)

summary: The primary objectives of this study are to observe the safety and tolerability of bemarituzumab and to evaluate preliminary antitumor activity.

ipd_sharing_description: De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request.

lead_sponsor: Amgen

location_country:
Poland
Spain
Greece
Austria
Australia
Mexico
Canada
United Kingdom
Switzerland
Hungary
Korea, Republic of
United States
France
Belgium
Japan
Czechia

location_facility: US Oncology Regulatory Affairs Corporate Office

location_facility: Universite Catholique de Louvain Cliniques Universitaires Saint Luc

location_facility: Universitair Ziekenhuis Leuven - Campus Gasthuisberg

location_facility: Severance Hospital Yonsei University Health System

location_facility: Hospital Clinico Universitario Virgen de la Victoria

location_facility: US Oncology Research Investigational Products Center

location_facility: Centro de Infusion e Investigacion Oncologia de Saltillo SC

location_facility: Hospital Clinico Universitario de Santiago

location_facility: The Cancer Institute Hospital of Japanese Foundation for Cancer Research


title: A Randomized, Double-blind, Placebo-Controlled Phase 2 Study With Open-label Extension to Assess the Efficacy and Safety of Namilumab in Subjects With Chronic Pulmonary Sarcoidosis

acronym: RESOLVE-Lung

summary: This is a randomized, double-blind, placebo-controlled study with an open-label extension (OLE).

lead_sponsor: Kinevant Sciences GmbH

location_country:
Netherlands
United States
France


acronym: GYROS

summary: The Gyrate Atrophy Ocular and Systemic Study characterizes the natural history of ornithine levels and retinal degeneration (RD) associated with disease-causing OAT variants in the presence of standard care dietary treatment regimens over 4 years. The research goal is to understand the impact of OAT mutations on plasma ornithine levels and retinal degeneration.

ipd_sharing_description: After the study is completed, the de-identified, archived data will be transmitted to and stored at the FFB Consortium Coordinating Center, under the supervision of Allison Ayala, for use by other researchers including those outside of the study.

location_country:
Brazil
United Kingdom
Finland
Canada
United States
France
Israel
Germany

location_facility: Harvard Univ., Massachusetts Eye and Ear Infirmary

location_facility: Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts, INSERM-DGOS CIC1423

location_facility: Johns Hopkins University, Wilmer Eye Institute

location_facility: Center for Advanced Retinal and Ocular Therapeutic

location_facility: Hadassah-Hebrew University Medical Center

location_facility: University of Tuebingen, Centre for Ophthalmology

location_facility: University of Toronto, Hospital for Sick Children


title: Non-Arteritic Anterior Ischemic Optic Neuropathy Risk Factors: New Perspectives

acronym: NARROW

summary: The purpose of the study is to use new diagnostic methods (OCT and OCT-A) to shed light on risk factors for the development of NA-AION. The risk factors we are focusing on are comorbidities along with anatomical and vascular characteristics of the optic nerve.

location_country:
Denmark
United Kingdom
Australia
Iran, Islamic Republic of
Canada
New Zealand
United States
France
Israel

location_facility:
John A. Moran Eye Center
Sydney Eye Hospital
Capital and Coast DHB
UCSF Medical Center
University og Colorado
Farabi Eye Hospital
University of Cambridge
University of Calgary
Moorfield's Eye Hospital
Aarhus University Hospital
Massachusetts Eye and Ear
Lawson Health Research Institute
Aalborg University Hospital
Bordeaux University Hospital
Odense University Hospital
Zealand University Hospital
Sheba Medical Center
King's College Hospital
Research St. Joseph's
Stanford Medicine


title: Endovascular Therapy vs Medical Management of PosterIor Cerebral Artery Occlusion Stroke

acronym: PLATO

summary:
For this retrospective study, the investigators will collect and analyze data of patients who presented with posterior artery occlusion and underwent mechanical thrombectomy (the type of endovascular stroke treatment) and intravenous thrombolysis (the type of non-endovascular stroke treatment).

The electronic health records will be queried for the demographic, medical history, and outcomes data of all patients with posterior cerebral artery occlusion who underwent mechanical thrombectomy, intravenous thrombolysis (IVT), or medical management.

time_perspective: Retrospective

ReferenceCitation: Strambo D, Bartolini B, Beaud V, Marto JP, Sirimarco G, Dunet V, Saliou G, Nannoni S, Michel P. Thrombectomy and Thrombolysis of Isolated Posterior Cerebral Artery Occlusion: Cognitive, Visual, and Disability Outcomes. Stroke. 2020 Jan;51(1):254-261. doi: 10.1161/STROKEAHA.119.026907. Epub 2019 Nov 13.

ReferenceCitation: Meyer L, Stracke CP, Jungi N, Wallocha M, Broocks G, Sporns PB, Maegerlein C, Dorn F, Zimmermann H, Naziri W, Abdullayev N, Kabbasch C, Behme D, Jamous A, Maus V, Fischer S, Möhlenbruch M, Weyland CS, Langner S, Meila D, Miszczuk M, Siebert E, Lowens S, Krause LU, Yeo LLL, Tan BY, Anil G, Gory B, Galván J, Arteaga MS, Navia P, Raz E, Shapiro M, Arnberg F, Zelenák K, Martinez-Galdamez M, Fischer U, Kastrup A, Roth C, Papanagiotou P, Kemmling A, Gralla J, Psychogios MN, Andersson T, Chapot R, Fiehler J, Kaesmacher J, Hanning U. Thrombectomy for Primary Distal Posterior Cerebral Artery Occlusion Stroke: The TOPMOST Study. JAMA Neurol. 2021 Apr 1;78(4):434-444. doi: 10.1001/jamaneurol.2021.0001.

ReferenceCitation: Herweh C, Abdalkader M, Nguyen TN, Puetz V, Schöne D, Kaiser D, Chen CH, Jeng JS, Möhlenbruch MA, Ringleb PA, Nagel S. Mechanical Thrombectomy in Isolated Occlusion of the Proximal Posterior Cerebral Artery. Front Neurol. 2021 Jul 29;12:697348. doi: 10.3389/fneur.2021.697348. eCollection 2021.

ReferenceCitation: Cunha B, Baptista M, Pamplona J, Carvalho R, da Câmara CP, Alves M, Papoila AL, Nunes AP, Reis J, Fragata I. Acute Treatment of Isolated Posterior Cerebral Artery Occlusion: Single Center Experience. J Stroke Cerebrovasc Dis. 2022 Feb;31(2):106239. doi: 10.1016/j.jstrokecerebrovasdis.2021.106239. Epub 2021 Dec 12.

ReferenceCitation: Miszczuk M, Bauknecht HC, Kleine JF, Kabbasch C, Liebig T, Bohner G, Siebert E. Mechanical thrombectomy of acute distal posterior cerebral artery occlusions. J Clin Neurosci. 2021 Jun;88:57-62. doi: 10.1016/j.jocn.2021.03.027. Epub 2021 Apr 1.

ReferenceCitation: Clarençon F, Baronnet F, Shotar E, Degos V, Rolla-Bigliani C, Bartolini B, Veznedaroglu E, Budzik R, English J, Baxter B, Liebeskind DS, Krajina A, Gupta R, Miralbes S, Lüttich A, Nogueira RG, Samson Y, Alamowitch S, Sourour NA. Should posterior cerebral artery occlusions be recanalized? Insights from the Trevo Registry. Eur J Neurol. 2020 May;27(5):787-792. doi: 10.1111/ene.14154. Epub 2020 Feb 21.

ReferenceCitation: Baig AA, Monteiro A, Waqas M, Cappuzzo JM, Siddiqi M, Doane J, Dossani RH, Almayman F, Khawar WI, Davies JM, Snyder KV, Levy EI, Siddiqui AH. Acute isolated posterior cerebral artery stroke treated with mechanical thrombectomy: A single-center experience and review of the literature. Interv Neuroradiol. 2022 Jan 10:15910199211070949. doi: 10.1177/15910199211070949. [Epub ahead of print]

ReferenceCitation: Maus V, Rogozinski S, Borggrefe J, Barnikol UB, Saklak M, Mpotsaris A. Clinical presentation of posterior cerebral artery occlusions - Clinical rationale for a more aggressive therapeutic strategy? eNeurologicalSci. 2021 Sep 4;25:100368. doi: 10.1016/j.ensci.2021.100368. eCollection 2021 Dec.

ReferenceCitation: Brouwer J, Ergezen S, Mulder MJHL, Lycklama A Nijeholt GJ, van Es ACGM, van der Lugt A, Dippel DWJ, Majoie CBLM, Roos YBWEM, Coutinho JM, Emmer BJ; MR CLEAN Registry investigators. Endovascular treatment for isolated posterior cerebral artery occlusion stroke in the MR CLEAN registry. J Neurointerv Surg. 2022 Mar 15. pii: neurintsurg-2021-018505. doi: 10.1136/neurintsurg-2021-018505. [Epub ahead of print]

ipd_sharing: Undecided

lead_sponsor: Boston Medical Center

location_country:
Portugal
Spain
Italy
Netherlands
Finland
Switzerland
United States
France
Germany

location_facility: University Medical Center Hamburg-Eppendorf

location_facility: Centro Hospitalar Universitário de Lisboa Central

location_facility: IRCCS Istituto delle Scienze Neurologiche di Bologna


title: An Open-label Trial of the Long-term Safety and Tolerability of Nintedanib Per os, on Top of Standard of Care, Over at Least 2 Years, in Children and Adolescents With Clinically Significant Fibrosing Interstitial Lung Disease (InPedILD®-ON)

summary:
This study is open to children and adolescents with interstitial lung disease (ILD) that causes lung fibrosis. This is a study for people who took part in a previous study (study 1199-0337, InPedILD®) and for people who are between 6 and 17 years old and have fibrosing ILD.

This study tests a medicine called nintedanib. Nintedanib is already used to treat different types of lung fibrosis in adults. The purpose of the study is to find out how well long-term treatment with nintedanib is tolerated in children and adolescents.

All participants take nintedanib capsules twice a day. Participants are in the study for at least 2 years or until nintedanib or other treatment options become available outside of this study.

During the first 2 years, they visit the study site between 9 and 11 times. Afterwards, they visit the study site every 3 months.

The doctors collect information on any health problems of the participants.

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

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

lead_sponsor: Boehringer Ingelheim

location_country:
Poland
Portugal
Spain
Greece
Italy
Mexico
Netherlands
Argentina
Canada
Finland
United Kingdom
United States
France
Belgium
Norway
Germany
Czechia

location_facility: Independent Public Teaching Children's Hospital

location_facility: Centro Hospitalar Universitário de Lisboa Norte, EPE - Hospital Santa Maria

location_facility: General Hospital of Thessaloniki "Ippokrateio"

location_facility: Oslo Universitetssykehus HF, Rikshospitalet

location_facility: Children's Hospital of Pittsburgh of University of Pittsburgh Medical Center

location_facility: Hamburger Zentrum für Kinder- und Jugendrheumatologie

location_facility: Teaching Hospital Motol, Oncology Clinic


title: A Phase 2, Multicenter, Randomized, Open-label Study of DS-7300a, a B7-H3 Antibody Drug Conjugate (ADC), in Subjects With Pretreated Extensive-stage Small Cell Lung Cancer (ES-SCLC)

summary: This study intends to define the recommended Phase 2 dose of ifinatamab deruxtecan (I-DXd) based on the efficacy, safety, and pharmacokinetics (PK) results observed in participants with Extensive-stage Small Cell Lung Cancer (ES-SCLC) who received at least 1 prior line of platinum-based chemotherapy and a maximum 3 prior lines of therapy and to investigate I-DXd anti-tumor activity in this population.

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:
Taiwan
United States
France
China
Japan

location_facility: Millennium Research & Clinical Development

location_facility: Cancer and Hematology Centers of Western Michigan

location_facility: West China Hospital of Sichuan University

location_facility: Union Hospital of Tongji Medical College, Huazhong University of Science and Technology

location_facility: The Cancer Institute Hospital of Japanese Foundation For Cancer Research


title: A Phase 1/2, Multi-Center, Open-Label Study to Evaluate the Safety, Tolerability, and Preliminary Anti-tumor Activity of TNG908 in Patients With MTAP-deleted Advanced or Metastatic Solid Tumors

summary: This is a first in human study in patients with advanced or metastatic solid tumors known to have an MTAP deletion. The first part of the study is an open-label, dose escalation and the second part is an open label dose expansion in specific MTAP-deleted tumor types. The study drug, TNG908, is a selective PRMT5 inhibitor administered orally. The study is planned to treat up to 170 participants.

lead_sponsor: Tango Therapeutics, Inc.

location_country:
United States
France

location_facility: The University of Texas MD Anderson Cancer Center


title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Multicenter Study of Itolizumab in Combination With Corticosteroids for the Initial Treatment of Acute Graft Versus Host Disease

summary: This is a multi-center study to compare the efficacy and safety of itolizumab versus placebo as first-line therapy for subjects with Grade III-IV aGVHD or Grade II with LGI involvement, in combination with corticosteroids

lead_sponsor: Equillium

location_country:
Spain
Italy
Australia
Canada
United States
France

location_facility: University of Pittsburgh Medical Center (UPMC) - Hillman Cancer Center

location_facility: University Hospitals Cleveland Medical Center

location_facility: Azienda Ospedaliera - Universitaria Integrata di Verona - Ospedale Borgo Roma

location_facility: University of California, Los Angeles (UCLA) - Medical Center

location_facility: McGill University Health Center - Research Institute

location_facility: Sarah Cannon Research Institute, LLC (SCRI)

location_facility: University of Pennsylvania Abramson Cancer Center


title: Phase 3, Multicenter, Randomized, Double-Masked, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Tinlarebant in the Treatment of Stargardt Disease in Adolescent Subjects

summary: The primary objective of this trial is to assesses the efficacy of tinlarebant in slowing the rate of growth of atrophic lesion(s) in adolescent subjects with STGD1

lead_sponsor: Belite Bio, Inc

location_country:
Taiwan
Hong Kong
United Kingdom
Australia
Switzerland
United States
France
China
Belgium
Germany

location_facility: Columbia University Irving Medical Center, Harkness Eye Institute

location_facility: Eye and ENT Hospital of Fudan University

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

location_facility: Beijing Tongren Hospital - Capital Medical University


title: A Randomized, Open-label, Phase 3 Trial of Dato-DXd Plus Pembrolizumab vs Pembrolizumab Alone in Treatment-naïve Subjects With Advanced or Metastatic PD-L1 High (TPS ≥50%) Non-small Cell Lung Cancer Without Actionable Genomic Alterations (TROPION-Lung08)

summary: This study is designed to assess the efficacy and safety of datopotamab deruxtecan (Dato-DXd) in combination with pembrolizumab versus pembrolizumab alone in participants with advanced or metastatic non-small cell lung cancer (NSCLC).

location_country:
Poland
Taiwan
Spain
Greece
Italy
Australia
Switzerland
Hungary
Korea, Republic of
United States
France
China
Belgium
Japan

location_facility: National Cheng Kung University Hospital Nckuh

location_facility: Tokyo Metropolitan Cancer and Infectious Diseases Center Komagome Hospital

location_facility: Sotiria General Hosptial of Chest Diseases

location_facility: Onze-Lieve-Vrouwziekenhuis Olvz - Campus Aalst

location_facility: Seoul National University Bundang Hospital

location_facility: Az Maria Middelares - Campus Maria Middelares

location_facility: Fejer Megyei Szent Gyorgy Egyetemi Oktato Korhaz

location_facility: Hospital Universitario Fundacion Jimenez Diaz

location_facility: National Hospital Organization Kyushu Cancer Center

location_facility: The Catholic Univ. of Korea, Seoul St. Mary'S Hospital

location_facility: Centre Hospitalier Universitaire de Montpellier

location_facility: Veszprem Megyei Tudogyogyintezet Farkasgyepu

location_facility: Semmelweis University Department of Pulmonology

location_facility: The Oncology Institute of Hope and Innovation

location_facility: The First Hospital of China Medical University

location_facility: The First Affiliated Hospital of College of Medicine Zhejiang University

location_facility: The Second People's Hospital of Neijiang

location_facility: Hospital Universitari i Politècnic La Fe

location_facility: The First Affiliated Hospital of Nanchang University

location_facility: Chang Gung Memorial Hospital Cgmh - Kaohsiung Branch

location_facility: Kyungpook National University Chilgok Hospital

location_facility: Union Hospital Affiliated With Tongji Medical College Huazhong University of Science and Technology

location_facility: Harbin Medical University Cancer Hospital


title: ENCORAFENIB/BINIMETINIB MASTER PROTOCOL: AN OPEN-LABEL CONTINUATION STUDY FOR PARTICIPANTS CONTINUING FROM ENCORAFENIB/BINIMETINIB CLINICAL STUDIES

summary:
The purpose of this clinical trial (called the FLOTILLA study) is to provide continued access to the study medicines, as well as safety follow-up, for participants in prior clinical trials of encorafenib and/or binimetinib.

All participants in prior encorafenib and/or binimetinib studies may participate the FLOTILLA study if they are still benefiting from the use of the study medicines. This will be determined by the study doctor. People may not participate in the FLOTILLA study if they have not enrolled in a prior study of encorafenib or binimetinib, or if they have stopped receiving the study treatment in a prior study.

Participants in the FLOTILLA study will receive encorafenib and/or binimetinib at the same dose and frequency as in their prior study, for up to about 5 years.

ipd_sharing_description: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

lead_sponsor: Pfizer

location_country:
Spain
Italy
Australia
United States
France

location_facility: Rockefeller Outpatient Pavilion (53rd Street)

location_facility: Istituto Nazionale Tumori IRCCS Fondazione Pascale

location_facility: St. Joseph's Hospital and Medical Center


title: The Oxford Risk Factors And Non-Invasive Imaging Study

acronym: ORFAN

summary: ORFAN is a prospective, multi-centre, multi-ethnic cohort observational study collecting CT scans, biological material and outcomes data, to develop and validate novel biomarkers of cardiometabolic and other disease risk.

time_perspective: Other

lead_sponsor: University of Oxford

location_country:
Romania
United Kingdom
Greece
Italy
Australia
Netherlands
Hungary
Korea, Republic of
United States
France
China
Belgium
Japan
Germany
United Arab Emirates

location_facility: Oxford University Hospitals NHS Foundation Trust

location_facility: Cleveland Clinic Heart and Vascular Institute

location_facility: University Hosptials of Leicester NHS Trust

location_facility: Sandwell and West Birmingham Hospitals NHS Trust

location_facility: Guy's and St Thomas' NHS Foundation Trust

location_facility: University of Medicine and Pharmacy of Targu Mures

location_facility: University Hospitals Birmingham NHS Trust

location_facility: University Hospital of Manchester NHS Foundation Trust

location_facility: Royal United Hospitals Bath NHS Foundation Trust

location_facility: MTA-SE Cardiovascular Imaging Research Group

location_facility: Heart and Lung Centre, New Cross Hospital

location_facility: NIH National Heart, Lunch, and Blood Institute

location_facility: TEDA International Cardiovascular Hospital

location_facility: Centro Cardiologico Monzino IRCCS, University of Milan

location_facility: University of Edinburgh, Royal Infirmary


title: Phase 3, Double-Blind, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Apitegromab (SRK-015) in Patients With Later-Onset Spinal Muscular Atrophy Receiving Background Nusinersen or Risdiplam Therapy

acronym: SAPPHIRE

summary: This Phase 3 trial (Study SRK-015-003) is being conducted in patients ≥2 years old at Screening, who were previously diagnosed with later-onset spinal muscular atrophy (SMA) (i.e., Type 2 and Type 3 SMA) and are receiving an approved survival motor neuron (SMN) upregulator therapy (i.e., either nusinersen or risdiplam), to confirm the efficacy and safety of apitegromab as an adjunctive therapy to nusinersen and evaluate the efficacy and safety of apitegromab as an adjunctive therapy to risdiplam.

lead_sponsor: Scholar Rock, Inc.

location_country:
Poland
Spain
Italy
United Kingdom
Netherlands
United States
France
Belgium
Germany

location_facility: Centro Clinico Nemo Pediatrico Policlinico A. Gemelli-Università Cattolica Sacro Cuore

location_facility: Washington University School of Medicine in St. Louis

location_facility: Hospital Universitario y Politécnico La Fe

location_facility: University of Wisconsin School of Medicine and Public Health

location_facility: Gillette Children's Specialty Healthcare

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

location_facility: Foundation I.R.C.C.S. Carlo Besta Neurological Institute

location_facility: Instytut Pomnik - Centrum Zdrowia Dziecka: CZD Warszawa

location_facility: Ann & Robert H. Lurie Children's Hospital of Chicago

location_facility: Columbia University, SMA Clinical Research Center


title: Single Blind Randomized Controlled Trial to Assess the Safety and Efficacy of High Dose Pulse Intravenous Corticosteroid Therapy to Treat Patients With Complicated/Fulminant Acute Myocarditis

acronym: MYTHS

summary: This is a phase III, multi-center international, single blind randomized controlled trial to test the efficacy of pulsed intravenous (IV) methylprednisolone versus standard therapy on top of maximal support in patients with Acute myocarditis (AM).

ReferenceCitation: Ammirati E, Frigerio M, Adler ED, Basso C, Birnie DH, Brambatti M, Friedrich MG, Klingel K, Lehtonen J, Moslehi JJ, Pedrotti P, Rimoldi OE, Schultheiss HP, Tschöpe C, Cooper LT Jr, Camici PG. Management of Acute Myocarditis and Chronic Inflammatory Cardiomyopathy: An Expert Consensus Document. Circ Heart Fail. 2020 Nov;13(11):e007405. doi: 10.1161/CIRCHEARTFAILURE.120.007405. Epub 2020 Nov 12. Review.

ReferenceCitation: Kociol RD, Cooper LT, Fang JC, Moslehi JJ, Pang PS, Sabe MA, Shah RV, Sims DB, Thiene G, Vardeny O; American Heart Association Heart Failure and Transplantation Committee of the Council on Clinical Cardiology. Recognition and Initial Management of Fulminant Myocarditis: A Scientific Statement From the American Heart Association. Circulation. 2020 Feb 11;141(6):e69-e92. doi: 10.1161/CIR.0000000000000745. Epub 2020 Jan 6.

ReferenceCitation: Ammirati E, Cipriani M, Moro C, Raineri C, Pini D, Sormani P, Mantovani R, Varrenti M, Pedrotti P, Conca C, Mafrici A, Grosu A, Briguglia D, Guglielmetto S, Perego GB, Colombo S, Caico SI, Giannattasio C, Maestroni A, Carubelli V, Metra M, Lombardi C, Campodonico J, Agostoni P, Peretto G, Scelsi L, Turco A, Di Tano G, Campana C, Belloni A, Morandi F, Mortara A, Cirò A, Senni M, Gavazzi A, Frigerio M, Oliva F, Camici PG; Registro Lombardo delle Miocarditi. Clinical Presentation and Outcome in a Contemporary Cohort of Patients With Acute Myocarditis: Multicenter Lombardy Registry. Circulation. 2018 Sep 11;138(11):1088-1099. doi: 10.1161/CIRCULATIONAHA.118.035319.

ReferenceCitation: Ammirati E, Veronese G, Brambatti M, Merlo M, Cipriani M, Potena L, Sormani P, Aoki T, Sugimura K, Sawamura A, Okumura T, Pinney S, Hong K, Shah P, Braun Ö, Van de Heyning CM, Montero S, Petrella D, Huang F, Schmidt M, Raineri C, Lala A, Varrenti M, Foà A, Leone O, Gentile P, Artico J, Agostini V, Patel R, Garascia A, Van Craenenbroeck EM, Hirose K, Isotani A, Murohara T, Arita Y, Sionis A, Fabris E, Hashem S, Garcia-Hernando V, Oliva F, Greenberg B, Shimokawa H, Sinagra G, Adler ED, Frigerio M, Camici PG. Fulminant Versus Acute Nonfulminant Myocarditis in Patients With Left Ventricular Systolic Dysfunction. J Am Coll Cardiol. 2019 Jul 23;74(3):299-311. doi: 10.1016/j.jacc.2019.04.063.

ReferenceCitation: Chen H, Liu J, Yang M. Corticosteroids for viral myocarditis. Cochrane Database Syst Rev. 2006 Oct 18;(4):CD004471. Review. Update in: Cochrane Database Syst Rev. 2013;10:CD004471.

ReferenceCitation: Mason JW, O'Connell JB, Herskowitz A, Rose NR, McManus BM, Billingham ME, Moon TE. A clinical trial of immunosuppressive therapy for myocarditis. The Myocarditis Treatment Trial Investigators. N Engl J Med. 1995 Aug 3;333(5):269-75.

ReferenceCitation: Ammirati E, Cipriani M, Lilliu M, Sormani P, Varrenti M, Raineri C, Petrella D, Garascia A, Pedrotti P, Roghi A, Bonacina E, Moreo A, Bottiroli M, Gagliardone MP, Mondino M, Ghio S, Totaro R, Turazza FM, Russo CF, Oliva F, Camici PG, Frigerio M. Survival and Left Ventricular Function Changes in Fulminant Versus Nonfulminant Acute Myocarditis. Circulation. 2017 Aug 8;136(6):529-545. doi: 10.1161/CIRCULATIONAHA.117.026386. Epub 2017 Jun 2.

lead_sponsor: Niguarda Hospital

location_country:
Spain
Italy
Finland
United States
France
Belgium
Japan
Sweden

location_facility: Presidio Ospedaliero Universitario "Santa Maria della Misericordia"

location_facility: Heart and Lung Center, Helsinki University Hospital

location_facility: Hospital Universitario Puerta de Hierro Majadahonda

location_facility: Complexo Hospitalario Universitario A Coruña (CHUAC)

location_facility: Nagoya University Graduate School of Medicine

location_facility: Fondazione Policlinico Universitario Agostino Gemelli IRCCS

location_facility: Azienda Ospedaliero Universitaria di Parma

location_facility: Azienda Ospedaliera Specialistica dei Colli - Ospedale Monaldi

location_facility: AOU Ospedali Riuniti Umberto I°-Lancisi-Salesi di Ancona

location_facility: Lund University and Skåne University Hospital

location_facility: National Cerebral and Cardiovascular Center

location_facility: Hospices Civils de Lyon, Lyon 08, Rhône-Alpes

location_facility: Hospital General Universitario Gregorio Marañón in Madrid

location_facility: Azienda Ospedaliera Universitaria Senese, Policlinico Santa Maria alle Scotte

location_facility: Azienda Ospedaliera Universitaria Città della Salute e della Scienza di Torino

location_facility: Hospital Universitario Virgen de la Victoria

location_facility: ASST Grande Ospedale Metropolitano Niguarda

location_facility: Hospital Universitario Virgen de la Arrixaca


acronym: ORBIT

title: An Operationally Seamless, Randomized Phase 2/3 Study Consisting of a Phase 2 Single-Blind, Dose-Evaluation Phase and a Phase 3 Double-Blind, Placebo-Controlled Phase to Assess the Efficacy and Safety of Setrusumab in Subjects With Osteogenesis Imperfecta

lead_sponsor: Ultragenyx Pharmaceutical Inc

location_country:
Denmark
Poland
Portugal
United Kingdom
Italy
Australia
Netherlands
Argentina
Canada
United States
France
Turkey
Germany

location_facility: Washington University School of Medicine

location_facility: Nemours/ Alfred i. duPoint Hospital for Children

location_facility: University of Toronto- The Hospital for Sick Children (SickKids)

location_facility: New Mexico Clinical Research & Osteoporosis Center, Inc.

location_facility: Sheffield Children's NHS Foundation Trust

location_facility: The Royal Hospital for Children NHS Greater Glasgow

location_facility: Azienda Ospedaliera Universitaria Policlinico Umberto I

location_facility: Azienda Ospedaliera Universitaria Integrata Verona

location_facility: Uniwersytet Medyczny w Lodzi - Klinika Endokrynologii i Chorob Metabolicznych

location_facility: Nationwide Children's Hospital- Ohio State University College of Medicine

location_facility: Shriners Hospitals for Children - Chicago

location_facility: Shriners Hospitals for Children - Canada

summary: The primary objectives of the study are to identify a setrusumab dosing strategy in participants with OI and to evaluate the effect of setrusumab vs placebo on reduction in fracture rate.


title: A Phase 1/2 Study of the Highly Selective ROS1 Inhibitor NVL-520 in Patients With Advanced NSCLC and Other Solid Tumors (ARROS-1)

summary:
Phase 1/2, dose escalation and expansion study designed to evaluate the safety and tolerability of NVL-520, determine the recommended phase 2 dose (RP2D), and evaluate the antitumor activity in patients with advanced ROS1-positive (ROS1+) NSCLC and other advanced ROS1-positive solid tumors.

Phase 1 will determine the RP2D and, if applicable, the maximum tolerated dose (MTD) of NVL-520 in patients with advanced ROS1-positive solid tumors.

Phase 2 will determine the objective response rate (ORR) as assessed by Blinded Independent Central Review (BICR) of NVL-520 at the RP2D. Secondary objectives will include the duration of response (DOR), time to response (TTR), progression-free survival (PFS), overall survival (OS), and clinical benefit rate (CBR) of NVL-520 in patients with advanced ROS1-positive NSCLC and other solid tumors.

lead_sponsor: Nuvalent Inc.

location_country:
Spain
Australia
United States
France
Netherlands

location_facility: NEXT Oncology - Virginia Cancer Specialists

location_facility: UOMI Cancer Center - Clinica Tres Torres


primary_purpose: Other

acronym: UNITED

title: A Phase 3, Multicenter, Open-label, Basket, Long-term Extension Study of Ustekinumab in Pediatric Clinical Study Participants (2 to <18 Years of Age)

lead_sponsor: Janssen Research & Development, LLC

location_country:
Poland
Hungary
United States
France
Belgium
Japan
Germany

location_facility: Uniwersytecki Szpital Dzieciecy w Krakowie

location_facility: Charité - Universitätsmedizin Berlin, Campus Virchow Klinikum

location_facility: Szabolcs-Szatmar-Bereg Megyei Korhazak es Egyetemi Oktatokorhaz, Josa Andras Oktatokorhaz

location_facility: Children's Center For Digestive Healthcare, Llc

location_facility: National Center for Child Health and Development

location_facility: Borsod-Abauj-Zemplen Megyei Korhaz es Egyetemi Oktato Korhaz

summary: The purpose of this study is to collect long-term safety data of subcutaneous (SC) ustekinumab


title: A Phase 3 Study to Evaluate the Efficacy and Safety of ARO-APOC3 in Adults With Familial Chylomicronemia Syndrome

acronym: PALISADE

summary: The purpose or AROAPOC3-3001 is to evaluate the efficacy and safety of ARO-APOC3 in adult participants with familial chylomicronemia syndrome (FCS). Participants who have met all protocol eligibility criteria will be randomized to receive 4 total doses of ARO-APOC3 or matching placebo administered subcutaneously.

lead_sponsor: Arrowhead Pharmaceuticals

location_country:
Poland
Austria
Australia
Mexico
Argentina
Canada
New Zealand
United States
France
Belgium
Japan
Germany

location_facility: Washington University School of Medicine, Division of Endocrinology, Metabolism and Lipid Research

location_facility: Instituto de Diabetes Obesidad y Nutrición S.C.

location_facility: New York University Langone Medical Center

location_facility: Centro Especializado en Diabetes Obesidad y Prevención de entermedades Cardiovasculares (CEDOPEC)

location_facility: National Institute of Medical Sciences and Nutrition - Salvador Zubiran (INCMNSZ)

location_facility: Clinique des Maladies Lipidiques de Quebec Inc.

location_facility: Institute de Recherches Cliniques de Montreal

location_facility: Centre Hospitalier Universitaire (CHU) de Liege


title: A Phase-II Study Comparing Non-Myeloablative Allogeneic Hematopoietic Stem Cell Transplantation Following Autologous Transplantation to Autologous Transplantation Alone in Multiple Myeloma

summary: The purpose of this study is to determine whether autologous transplantation (using the patient's own stem cells from the blood), followed by non-myeloablative (i.e. less intense) allogeneic transplantation (where the blood stem cells from a sibling donor are used for the transplantation) improves the outcome in patients with newly diagnosed multiple myeloma.

lead_sponsor: European Society for Blood and Marrow Transplantation

location_country:
Denmark
United Kingdom
Italy
Austria
Finland
Canada
Sweden
France
Turkey
Norway
Germany

location_facility:
CHU Lapeyronie
Uni. Modena, Policlinico
Medizinische Universität Wien
Tampere University Hospital
University Hospital
University of Milano
IRCCS, Casa Sollievo della Sofferenza
University of Leipzig
Sahlgrenska University Hospital
Rigshospitalet
Huddinge University Hospital
Royal Marsden Hospital
University of Heidelberg
Ospedale Ferrarotto
Ospedale di Careggi
Helsinki University Central Hospital
Turku University
Rikshospitalet
Herlev Hospital
University Faculty of Medicine

intervention_type: Procedure


title: Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients With Symptomatic Generalized Myasthenia Gravis

acronym: NIMBLE

summary:
The primary objective is:

To evaluate the effect of pozelimab + cemdisiran on daily functioning that is impacted by signs and symptoms in patients with symptomatic generalized myasthenia gravis (gMG)

The secondary objectives of the study are:

To evaluate the effect of pozelimab + cemdisiran (ie, combination) and cemdisiran monotherapy on:

Clinician-assessed signs of myasthenia gravis (MG) and muscle strength
Daily functioning that is impacted by signs and symptoms in patients with symptomatic gMG (cemdisiran monotherapy only).
Proportion of patients with improvements in daily function that is impacted by signs and symptoms of MG
Proportion of patients that have improvements in clinician-assessed signs of MG and muscle strength
Health related quality of life
Proportion of patients with minimal MG symptoms
Patient- and clinician-reported signs and symptoms of MG
To evaluate the safety and tolerability of pozelimab + cemdisiran and cemdisiran monotherapy
To assess the concentration of total pozelimab in serum
To assess the concentrations of cemdisiran and its metabolites in plasma
To assess the immunogenicity of pozelimab
To assess the concentration of total C5 in plasma
To assess the immunogenicity of cemdisiran
To study the effect of pozelimab + cemdisiran and cemdisiran monotherapy on complement activation

ipd_sharing_description: All Individual Patient Data (IPD) that underlie publicly available results will be considered for sharing

lead_sponsor: Regeneron Pharmaceuticals

location_country:
Poland
Taiwan
Spain
Italy
Australia
Canada
Korea, Republic of
United States
France
Turkey
Belgium
Japan
Czechia

location_facility: Clinical Research Center Spólka z Ograniczona Odpowiedzialnoscia, Medic-R Spólka Komandytowa

location_facility: University Of Cincinnati Gardner Neuroscience institute

location_facility: Prairie Education and Research Cooperative

location_facility: Centrum Neurologii Klinicznej, Krakowska Akademia Neurologii

location_facility: Fondazione IRCCS Di Rilievo Nazionale Istituto Nazionale Neurologico Carlo Besta

location_facility: Weill Cornell Medicine - Peripheral Neuropathy Center

location_facility: Northwest Neurology Ltd. - Clinedge - PPDS

location_facility: Chang Gung Medical Foundation Kaohsiung Chang Gung Memorial Hospital

location_facility: Kyungpook National University Chilgok Hospital

location_facility: Colorado Springs Neurological Associates