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A Randomized, Double-Blind, Double Dummy, Parallel Group, Multicenter 24 to 52 Week Variable Length Study to Assess the Efficacy and Safety of Budesonide, Glycopyrronium, and Formoterol Fumarate Metered Dose Inhaler (MDI) Relative to Budesonide and Formoterol Fumarate MDI and Symbicort® Pressurized MDI in Adult and Adolescent Participants with Inadequately Controlled Asthma (KALOS)

To evaluate the effect of triple-therapy wih Budesonide, Glycopyrronium and Formoterol Fumarate (PT010) versus dual therapy with Budesonide and Formoterol Fumarate on asthma exacerbations in adult and adolescent subjects with inadequately controlled…

A Phase 3 Double-blind, Randomized, Placebo-controlled Study of the Safety and Efficacy of Odevixibat (A4250) in Patients with Alagille Syndrome (ASSERT)

Primary Objective:To demonstrate the efficacy of repeated daily doses of 120 µg/kg/day odevixibat in relieving pruritus in patients with ALGS.Secondary Objectives:To assess the impact of odevixibat on serum bile acid levels in patients with ALGS.To…

A Double-Blind, Vehicle-Controlled, Randomized Withdrawal and Treatment Extension Study to Assess the Long-Term Efficacy and Safety of Ruxolitinib Cream in Participants With Vitiligo

Primary Objective:To evaluate the duration of clinical response of ruxolitinib cream in participants with vitiligo.

Effectiveness of methylphenidate in children and adults with Smith Magenis syndrome and attention-deficit/hyperactivity disorder: An N-of-1 series

The primary objective is to determine the efficacy of methylphenidate on ADHD symptoms in Smith Magenis syndrome.The secondary objectives include the efficacy of methylphenidate on emotion dysregulation and specific goals that are important to the…

A PHASE 3, RANDOMIZED, DOUBLE-BLINDED,
PLACEBO-CONTROLLED TRIAL TO EVALUATE THE EFFICACY AND
SAFETY OF A RESPIRATORY SYNCYTIAL VIRUS (RSV) PREFUSION F
SUBUNIT VACCINE IN INFANTS BORN TO WOMEN VACCINATED DURING
PREGNANCY
Short title: A Phase 3 Trial to Evaluate the Efficacy and Safety of RSVpreF in Infants Born to Women Vaccinated During Pregnancy.

to assess the efficacy, safety, and immunogenicity of bivalent RSVpreF or placebo (1:1 randomization) in infants born to healthy women vaccinated during pregnancy, as well as the safety and immunogenicity in the pregnant women.

A phase 3, randomised, double-blind, multi-centre trial to evaluate the efficacy,
safety, and tolerability of brodalumab treatment compared to placebo (blinded) and
ustekinumab (open-label) in adolescent subjects (12-17 years of age) with
moderate-to-severe plaque psoriasis

To determine the efficacy of subcutaneous administration of brodalumab compared with placebo in treating adolescents with moderate-to-severe plaque psoriasis.

Randomized, double-blind, placebo-controlled, two way crossover, single centre study evaluating the acute and chronic effect of clonazepam on cognitive tests and patient-reported outcome measures in patients with ARID1B-related intellectual disability

* To test the hypothesis that clonazepam administration has acute beneficial effects compared to placebo on body sway, adaptive tracking, smooth eye pursuit, tapping frequency and the animal fluency test.* To test the hypothesis that multiple-doses…

A multicenter, double-blind, randomized, placebo-controlled, parallel-arm study to investigate the efficacy and safety of subcutaneous administration of CSL312 (garadacimab) in the prophylactic treatment of hereditary angioedema

The primary objective of this study is to evaluate the efficacy of SC administration of CSL312 as prophylaxis to prevent HAE attacks in subjects with HAE.The secondary objectives of the study are:1. To characterize the clinical efficacy of SC CSL312…

A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of High Doses of Eteplirsen, Preceded by an Open-Label Dose Escalation, in Patients with Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping

This study has been transitioned to CTIS with ID 2024-511492-15-00 check the CTIS register for the current data. Part 1:to evaluate the safety and tolerability of 2 doses (100 milligrams/kilogram [mg/kg] and 200 mg/kg) of eteplirsen in approximately…

A Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy and Safety of Nemolizumab (CD14152) in Subjects with Moderate-to-Severe Atopic Dermatitis

Primary objective: The primary objective is to assess the efficacy and safety of nemolizumab (CD14152) after a 16-week treatment period in adult and adolescent subjects with moderate-to-severe atopic dermatitis (AD) not adequately controlled with…

A Phase 3, Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group, Safety and Efficacy Study of Linaclotide Administered Orally to Children, Ages 6 to 17 Years, With Functional Constipation (FC)

To evaluate the safety and efficacy of 12 weeks of linaclotide therapy in comparison with placebo in pediatric participants aged 6 to 17 years who fulfill modified Rome III Criteria for Child/Adolescent FC

Effectiveness of blood flow restriction training after bone-patellar tendon-bone anterior cruciate ligament reconstruction: a randomized placebo-controlled trial

The purpose of this study is to evaluate the effect of low-load BFR therapy on quadriceps and hamstring strength 8-, 14-, 26-, 39- and 52-weeks after BPTB ACL reconstruction compared to placebo BFR therapy and conventional HLR training.

Do effects of methylphenidate decline after long-term use? A double-blind, placebo-controlled cross-over study of effects of methylphenidate on cognitive functioning and real world behavior in treatment naïve children compared to effects after 9 months of treatment in clinical practice

The main objective of this study is to compare the acute effects of methylphenidate on behavioral and cognitive functioning in 6 to 12 year old treatment naïve children compared to the effects after nine months of treatment with methylphenidate in…

A Phase 3, Double blind, Randomized, Multicenter, Parallel Group, Placebo controlled Sequential Dose Titration Study to Evaluate Efficacy, Safety and Pharmacokinetics of Mirabegron in Pediatric Subjects from 5 to < 18 Years of Age with Overactive Bladder

ObjectivesPrimary* To evaluate the efficacy of mirabegron in children (5 to < 18 years of age) with OABSecondary* To evaluate the efficacy of mirabegron in children (5 to < 18 years of age) with OAB* To evaluate the safety and…

The effects of plant stanol ester supplements on liver inflammation in children with overweight or obesity

To assess the effect of consuming soft chews enriched with plant stanol esters (3 grams/day) vs. control chews for 6 months on ALTconcentrations in children with overweight or (morbid) obesity who are at risk of developing NAFLD.

Vitamins and Nutrients as Supplementation for Irritability (VANTASTIC Study) - A Double-blind Randomized Placebo-Controlled Trial

The main objective of this study is to investigate the possible benefits of micronutrients on irritability as a population trait in children and adolescents between 11 and 17;6 years of age with a high level of irritability. The secondary objectives…

Combining emotion recognition training with oxytocin administration: A psychobiological approach

The primary aim of this study is to examine the effectiveness of a psychobiological intervention combining oxytocin administration with ERT. We hypothesize that the combined intervention will be more effective in improving emotion recognition skills…

Exploratory study into the effect of salt supplementation in Gitelman syndrome

To determine the effect of salt (NaCl) supplementation on (1) physiological parameters (such as serum potassium) and on (2) clinical signs and symptoms and quality of life.

A Phase 3, Double-Blind, Randomized, Vehicle-Controlled, Efficacy and Safety Study of Ruxolitinib Cream Followed by an Extension Period in Participants With Vitiligo

Primary:To evaluate the efficacy of ruxolitinib cream in participants with vitiligo.Secondary:To further assess the efficacy of ruxolitinib cream.To evaluate the safety and tolerability of ruxolitinib cream.To evaluate the ruxolitinib PK in plasma…

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…