Search for a trial

A phase 2/3 Adaptive, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of VX-147 in Adult and Pediatric Subjects With APOL1-mediated Proteinuric Kidney Disease

• To evaluate the efficacy of VX-147 to reduce proteinuria• To evaluate the efficacy of VX 147 on renal function as measured by eGFR slope

A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients with Glycogen Storage Disease Type Ia

This study has been transitioned to CTIS with ID 2023-508750-25-00 check the CTIS register for the current data. Primary: To evaluate the efficacy of DTX401 to reduce or eliminate dependence on exogenous glucose replacement therapy needed to…

A double-blind, randomised, placebo-controlled, parallel group trial to evaluate the efficacy and safety of empagliflozin and linagliptin over 26 weeks, with a double-blind active treatment safety extension period up to 52 weeks, in children and adolescents with type 2 diabetes mellitus

DINAMOTM (main study)The objective of this study is to assess the efficacy and safety of anempagliflozin dosing regimen and one dose of linagliptin versus placebo after 26 weeks of treatment in children and adolescents with type 2 diabetes mellitus…

A Phase 3, Multicenter, Randomized, Placebo-controlled, Double-blind Study to Evaluate the Efficacy and Safety of Lanadelumab for Prevention Against Acute Attacks of Non-histaminergic Angioedema with Normal C1-Inhibitor (C1-INH)

Primary: To evaluate the efficacy of repeated subcutaneous (SC) administrations of lanadelumab in preventing angioedema attacks in adolescents (12 to <18 years of age) and adults with non-histaminergic angioedema with normal C1-INH.Secondary…

Treatment of chemo-refractory viral infections after allogeneic
stem cell transplantation with multispecific T cells against CMV, EBV and
AdV: A phase III, prospective, multicentre clinical trial

This study has been transitioned to CTIS with ID 2024-512321-84-00 check the CTIS register for the current data. Primary objectives:Evaluation of efficacy of multispecific T-cell transfer in patients with chemo-refractory viral infections after…

A Randomized, Double-blind, Placebo-controlled Phase 2a Study to Evaluate a Range of
Dose Levels and Vaccination Intervals of Ad26.COV2.S in Healthy Adults Aged 18 to 55
Years Inclusive and Adults Aged 65 Years and Older and to Evaluate 2 Dose Levels of Ad26.COV2.S in Healthy Adolescents Aged 12 to 17 Years Inclusive

The primary purpose of this study is to assess humoral immune responses of 3 dose levels of Ad26.COV2.S administered intramuscularly (IM) as a 2-dose schedule (56 days apart); Ad26.COV2.S administered IM as a single vaccination; safety and…

A Phase 3, Multicenter, Double-blind, Randomized, Placebo-controlled, Parallel Group Study, Followed by an Active Treatment Phase to Evaluate the Efficacy and Safety of Apremilast in Children From 2 to Less Than 18 Years of Age With Active Oral Ulcers Associated With Behçet*s Disease (BEAN)

The main objective of study 20190530 is to estimate the efficacy of Apremilast compared to placebo in the treatment of oral ulcers in pediatric subjects from 2 to < 18 years of age with oral ulcers associated with BD through week 12. See…

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, 12-Week Study to Assess the Efficacy and Safety of Etrasimod in Subjects with Moderately to Severely Active Ulcerative Colitis

The primary objective is to assess the efficacy of etrasimod when administered for 12 weeks on clinical remission in subjects with moderately to severely active ulcerative colitis (UC). The secondary objective is to assess the efficacy of etrasimod…

Happy to write: A first study about the influence of writing on your mood

The aim of the study is to investigate the effectiveness of a brief manipulation aimed at improving the body image of adolescents with an eating disorder.

A Randomized, Double-Blind, Placebo-Controlled Study Followed by an Open Label Treatment Period to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents with Heterozygous Familial Hypercholesterolemia

Primary goal: To evaluate the efficacy of alirocumab administered every 2 weeks (Q2W) versus placebo after 24 weeks of double-blind (DB) treatment on low-density lipoprotein cholesterol (LDL-C) levels in children with heterozygous familial…

Multicentre, Randomised, Placebo-Controlled Trial of Mebeverine in Children with Irritable Bowel Syndrome (IBS) or Functional Abdominal Pain- Not Otherwise Specified (FAP-NOS)

The primary objective of this study is to determine the effect of mebeverine on abdominal pain intensity and frequency in children with irritable bowel syndrome or functional abdominal pain - not otherwise specified.

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

This study has been transitioned to CTIS with ID 2024-513483-26-00 check the CTIS register for the current data. To assesses the efficacy of Tinlarebant in slowing the rate of growth of atrophic lesion(s) in adolescent subjects with STGD1.

A Multi-Center, Randomized, Double-Blind, Placebo-Controlled Phase III Study to Assess the Efficacy, Safety, and Tolerability of PXT3003 in Charcot-Marie-Tooth type 1A (CMT1A)

Primary:To evaluate the efficacy of treatment with PXT3003 (a fixed-dose combination of (RS)-baclofen, naltrexone hydrochloride, and D-sorbitol) compared to placebo in subjects with CMT1A.Secondary: To evaluate the safety and tolerability of PXT3003…

Efficacy and safety of oral semaglutide versus placebo both in combination with metformin and/or basal insulin in children and adolescents with type 2 diabetes

This study has been transitioned to CTIS with ID 2023-506923-27-00 check the CTIS register for the current data. Primary objectiveTo confirm superiority of oral semaglutide at the maximum tolerated dose* (3 mg, 7 mg or 14 mg) versus placebo on…

Two part (double-blind) inclisiran versus placebo [Year 1] followed by open-label inclisiran [Year 2] randomized multicentre study to evaluate safety, tolerability, and efficacy of inclisiran in adolescents (12 to less than 18 years) with homozygous familial hypercholesterolemia and elevated LDL-cholesterol (ORION-13)

Study CKJX839C12302 (ORION-13) is a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in adolescents (aged 12 to <18 years) with HoFH and LDL-C >130 mg/dL (3.4 mmol/L). The use of inclisiran…

A 6-month multicenter, randomized, double-blind, placebo-controlled study to evaluate the
efficacy, safety and PK/PD of an age-and body weight-adjusted oral finerenone regimen, in
addition to an ACEI or ARB, for the treatment of children, 6 months to <18 years of age, with
chronic kidney disease and proteinuria

This study has been transitioned to CTIS with ID 2023-504884-17-00 check the CTIS register for the current data. The main goal of this study is to find out whether finerenone in combination with either an ACE inhibitor or an ARB can reduce the…

A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study of Adeno-Associated
Virus Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase
(OTC) in Patients with Late-Onset OTC Deficiency

This study has been transitioned to CTIS with ID 2024-514337-38-00 check the CTIS register for the current data. PrimaryTo evaluate the efficacy of DTX301 on the improvement of OTC function by maintaining safe plasma ammonia levelsSecondaryTo…

Pediatric Options for Migraine Relief: A Randomized, Double-Blind, Placebo-Controlled Study of Lasmiditan for Acute Treatment of Migraine: PIONEER-PEDS1

This study has been transitioned to CTIS with ID 2023-506253-38-00 check the CTIS register for the current data. Primary- To test the hypothesis that lasmiditan high dose is superior to placebo in the acute treatment of a migraine attack in…

A Phase 2/3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Mitapivat in Subjects With Sickle Cell Disease

This study has been transitioned to CTIS with ID 2024-515202-84-00 check the CTIS register for the current data. Phase 2:Primary ObjectivesTo determine the recommended Phase3 dose of mitapivat by evaluating the effect of 2 dose levels of mitapivat…

Two part (double-blind inclisiran versus placebo [Year 1] followed by open-label inclisiran [Year 2]) randomized multicenter study to evaluate safety, tolerability, and efficacy of inclisiran in adolescents (12 to less than 18 years) with heterozygous familial hypercholesterolemia and elevated LDL-cholesterol (ORION-16)

Study CKJX839C12301 (ORION-16) is a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in adolescents (aged 12 to &lt;18 years) with HeFH and LDL-C &gt;130 mg/dL (3.4 mmol/L). The use of inclisiran…