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DAVINCY trial: optimal Duration of (fos)aprepitant prophylaxis for nausea and Vomiting INduced by ChemotherapY in children: a double-blind placebo-controlled crossover randomized phase III trial*

This study has been transitioned to CTIS with ID 2024-517846-32-00 check the CTIS register for the current data. To evaluate the effect of prolonged duration of (fos)aprepitant prophylaxis on the prevention of delayed CINV (complete remission in the…

A 52 week, multi-center, randomized, double-blind placebo-controlled study to assess the clinical efficacy and safety of ligelizumab (QGE031) in decreasing the sensitivity to peanuts in patients with peanut allergy

The purpose of this Phase 3 study is to evaluate the safety and clinical efficacy of ligelizumab 240 mg and 120 mg given subcutaneously (s.c.) every 4 weeks (q4w) to ensure protection against allergic reaction by decreasing the sensitivity to oral…

Peppermint Oil for the treatment of Irritable Bowel Syndrome or Functional Abdominal Pain in Children:
the MINT study

The aim of the Mint study is to investigate the effectiveness of peppermint oil capsules compared to placebo capsules in reducing abdominal pain intensity in these children. In addition, we investigate the effect of peppermint oil capsules compared…

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…

Phase 3, Multicenter, Double-blind, Randomized, Placebo-controlled, Parallel Group Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Apremilast in Children From 5 to Less Than 18 Years of Age With Active Juvenile Psoriatic Arthritis (PEAPOD)

This study has been transitioned to CTIS with ID 2023-503435-17-00 check the CTIS register for the current data. Estimate the efficacy of apremilast compared with placebo in the treatment of Juvenile Psoriatic Arthritis (JPsA) in pediatric subjects…

EPIK-P2: A Phase II double-blind study with an upfront, 16-week randomized, placebo-controlled period, to assess the efficacy, safety and pharmacokinetics of alpelisib (BYL719) in pediatric and adult patients with PIK3CA-related overgrowth spectrum (PROS)

This study has been transitioned to CTIS with ID 2023-508530-34-00 check the CTIS register for the current data. Primary objective:* To demonstrate the efficacy of alpelisib as measured by the proportion of participants randomized to alpelisib with…

A Phase 2 study to assess the efficacy and safety of 2 dosage regimens of oral fidrisertib (IPN60130) for the treatment of fibrodysplasia ossificans progressiva in male and female paediatric and adult participants

This study has been transitioned to CTIS with ID 2024-511469-13-00 check the CTIS register for the current data. Main objective:• Evaluate the efficacy of IPN60130 monotherapy compared with placebo recipients in inhibiting new HO volume in adult and…

Phase 1-3, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION373 in Patients with Alexander Disease

This study has been transitioned to CTIS with ID 2024-510603-11-00 check the CTIS register for the current data. Primary Objective: To evaluate the efficacy of ION373 in improving or stabilizing gross motor function in patients with Alexander…

A phase III, randomized, double-masked, placebo-controlled, parallel-group, multicenter study of the
safety and efficacy of OT-101 (Atropine Sulfate 0.01%) in treating the progression of myopia in
pediatric subjects

To evaluate the efficacy of OT-101 Ophthalmic Solution in treating the progression of myopia in pediatric subjects following 3 years of treatment.To evaluate the safety and tolerability of OT-101 Ophthalmic Solution in pediatric subjects with myopia…

Efficacy and safety of the SQ tree sublingual immunotherapy tablet in children and adolescents (5 through 17 years of age) with moderate to severe allergic rhinitis and/or conjunctivitis induced by pollen from birch and trees belonging to the birch homologous group

To compare the efficacy of the SQ tree SLIT-tablet to placebo in the treatment of moderate to severe allergic rhinitis and/or conjunctivitis induced by pollen from birch and trees belonging to the birch homologous group in children and adolescents (…

A Phase III Multicenter, Randomized, Double-Blind, Double-Dummy Study To Evaluate Safety And Efficacy Of Ocrelizumab In comparison with Fingolimod in Children And Adolescents With Relapsing-Remitting Multiple Sclerosis

This study has been transitioned to CTIS with ID 2023-506516-40-00 check the CTIS register for the current data. The primary efficacy objective for this study is to demonstrate non-inferiority of ocrelizumab compared with fingolimod

A Multicenter, Randomized, Double-Blind, Placebo Controlled, Parallel Group Study to Evaluate the Efficacy, Safety, and Tolerability of Soticlestat as Adjunctive Therapy in Pediatric and Young Adult Subjects With Dravet Syndrome (DS)

Primary Objectives:• To assess the efficacy of soticlestat in reducing convulsive seizure frequency as add-on therapy to SOC as compared with placebo during the full treatment period (titration + maintenance).• To assess the efficacy of soticlestat…

A 2-Stage (Open-Label Followed by Randomized Double-Blind, Placebo-Controlled Stage), Phase 2 Trial of Setmelanotide in Patients with Specific Gene Variants in the Melanocortin-4 Receptor Pathway

Primary:• To evaluate the proportion of patients with obesity with genetic variants in a specific gene in the melanocortin-4 receptor (MC4R) pathway who achieve a clinically meaningful reduction in body weight in response to setmelanotide at the end…

A Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel Group Study to Evaluate the Efficacy, Safety, and Tolerability of Soticlestat as Adjunctive Therapy in Pediatric and Adult Subjects With Lennox-Gastaut Syndrome (LGS)

Primary Objectives:• To assess the efficacy of soticlestat in reducing major motor drop (MMD) seizure frequency as add-on therapy to SOC as compared with placebo during the full treatment period (titration + maintenance).For European Medicines…

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 &lt; 18 years of age with oral ulcers associated with BD through week 12. See…

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…