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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.

The evaluation of the effects of a prebiotic mouth rinse on the oral ecosystem.

This study aims to evaluate the changes in microbial composition of the oral microbiome (main objective), changes in proportion of the red fluorescing plaque (RFP) and in gingival bleeding on marginal probing (BOMP) (secondary objectives) after…

*The MEtformin-LIfestyle in Antipsychotic users trial (MELIA): optimizing the use of metformin in the management of antipsychotic-induced weight gain.*

Objective: We aim to optimize the application of an existing drug, metformin, for a new indication, AiWG, by showing that metformin in combination with lifestyle interventions reduces AiWG compared to placebo in combination with lifestyle…

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.

Respiratory muscle training in patients with Spinal Muscular Atrophy

The aim of this study is to assess the feasibility and efficacy of respiratory muscle training (RMT) in patients with SMA and respiratory muscle weakness.

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, 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

A Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study Comparing the Efficacy, Safety, and Tolerability of Subcutaneous Administration of Fremanezumab Versus Placebo for the Preventive Treatment of Episodic Migraine in Pediatric Patients 6 to 17 Years of Age

Main objective:To evaluate the efficacy of fremanezumab as compared to placebo for the preventive treatment of episodic migraine (EM)Secondary objectives:-To evaluate the safety and tolerability of fremanezumab in the preventive treatment of…

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of Mavorixafor in patients with WHIM Syndrome with Open-Label Extension

This study has been transitioned to CTIS with ID 2024-518461-10-00 check the CTIS register for the current data. • Randomized Placebo-Controlled Period:Primary:- to demonstrate the efficacy of mavorixafor in patients with Warts,…

A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination with Systemic Corticosteroids in Subjects with Non-ambulatory Duchenne Muscular Dystrophy (DMD)

To evaluate the efficacy and safety of pamrevlumab versus placebo in combination with systemic corticosteroids in subjects with non-ambulatory Duchenne muscular dystrophy (age 12 years and older).

A Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study Comparing the Efficacy, Safety, and Tolerability of Subcutaneous Administration of Fremanezumab Versus Placebo for the Preventive Treatment of Chronic Migraine in Pediatric Patients 6 to 17 Years of Age

Main objective:The primary objective of the trial is to evaluate the efficacy of test investigational medicinal product (IMP) as compared to placebo IMP for the preventive treatment of chronic migraine (CM). Secondary objectives:To evaluate the…

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…

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…

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…