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A Phase 3 Study to Evaluate the Efficacy and Safety of Fitusiran in Patients with Hemophilia A or B, with Inhibitory Antibodies to Factor VIII or IX

1.3. Study Design Rationale The ATLAS-INH trial (ALN-AT3SC-003) is a multicenter, multinational, randomized, openlabel Phase 3 study designed to demonstrate the efficacy and safety of fitusiran in patients with haemophilia A or B with inhibitory…

Training immune functions through pharmacotherapeutic conditioning in
juvenile idiopathic arthritis

This study*s objective is to reduce MTX related side effects with pharmacotherapeutic conditioning, by using variable reinforcement principles in patients with JIA. Pharmacotherapeutic conditioning enables to alternate standard MTX dosing with lower…

A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)

Primary Objective:To evaluate the efficacy of VX-445 in triple combination (TC) with tezacaftor (TEZ) andivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous for F508del and aminimal function mutation (F/MF subjects)Secondary…

A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del Mutation (F/F)

Primary Objective:To evaluate the efficacy of VX-445 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are homozygous for the F508del mutation (F/F)Secondary Objectives:* To evaluate the…

A randomised, double-blind, placebo-controlled, parallel-group, multi-centre trial to evaluate the efficacy, safety, and tolerability of tralokinumab monotherapy in adolescent subjects with moderate-to-severe atopic dermatitis who are candidates for systemic therapy.

Primary objectiveTo evaluate the efficacy of subcutaneous (SC) administration of tralokinumab compared with placebo in treating adolescent subjects (age 12 to <18 years) with moderate-to-severe AD.Secondary objectivesTo evaluate the efficacy…

Prospective Multi-Center Evaluation of the Duration of Therapy for Thrombosis in Children

Specific Aim #1: To evaluate the efficacy and safety of shortened-duration (6 weeks total) versus conventional duration (3 months total) anticoagulation for first-episode, provoked, acute venous thrombosis among children in whom thrombus resolution/…

A Phase 3 Study to Evaluate the Efficacy and Safety of Fitusiran in Patients With Hemophilia A or B, Without Inhibitory Antibodies to Factor VIII or IX

The ATLAS-A/B trial (ALN-AT3SC-004) is a multicenter, multinational, randomized, openlabel Phase 3 study designed to demonstrate the efficacy and safety of fitusiran in patients with hemophilia A or B without inhibitory antibodies to FVIII or FIX…

ATLAS-PPX trial: an open-label, multinational, switching study to describe the efficacy and safety of fitusiran prophylaxis in patients with hemophilia A and B with inhibitory antibodies to factor VIII or IX previously receiving bypassing agent prophylaxis.

1.3. Study Design Rationale The ATLAS-PPX trial (ALN-AT3SC-009) is a multicenter, multinational, open-label Phase 3 switching study designed to demonstrate the efficacy and safety of fitusiran in patients with hemophilia A or B, with inhibitory…

A Phase III Open Label Extension Study to Assess the Long-Term Safety
and Efficacy of Idebenone in Patients with Duchenne Muscular Dystrophy
(DMD) who completed the SIDEROS study

Primary:* To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.Secondary:* To describe the long-term evolution of respiratory function in idebenone-treated DMD patients who completed the SIDEROS study,…

A Phase 3 Randomized, Double-blind, Placebo-controlled, Parallel-group Efficacy and Safety Study of SHP647 as Induction Therapy in Subjects With Moderate to Severe Crohn*s Disease (CARMEN CD 305)

Coprimary: The coprimary objectives of this study are to evaluate the efficacy of ontamalimab in subjects with moderate to severe Crohn*s disease (CD) in:* Inducing clinical remission based on 2 item patient reported outcome (PRO) (abdominal pain…

International phase 3 trial in Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) testing imatinib in combination with two different cytotoxic chemotherapy backbones

This study has been transitioned to CTIS with ID 2024-515499-12-00 check the CTIS register for the current data. The primary objective is reducing treatment-related morbidity and mortality without adversely impacting DFS in Ph+ ALL patients,…

Tamoxifen in Duchenne muscular dystrophy - TAMDMD:
A multicentre, randomised, double-blind, placebo-controlled, phase 3 safety and efficacy 48-week trial
The study will be extended to an open label study with the following title (OLE: Open Label Extension):
Tamoxifen in Duchenne muscular dystrophy - TAMDMD:
A 48 week open label extension of a multi centre, randomised, double-blind, placebo-controlled, phase 3 safety and efficacy trial

To test if tamoxifen treatment, compared to placebo, reduces the progression of the disease in 6.5-12 years old ambulant DMD patients by at least 50% (using the MFM D1 subscore as primary clinical endpoint in group A patients).To test if tamoxifen…

A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy in Subjects With Cystic Fibrosis (CF) Who Are Homozygous or Heterozygous for the F508del Mutation

Primair eindpunt:Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy in Subjects With Cystic Fibrosis (CF) Who Are Homozygous or Heterozygous for the F508del Mutation.Secundair eindpunt:Evaluate the long-term efficacy of VX-…

A Phase 3, Multicenter, Randomized, Placebo- and Active Comparator-Controlled Study Evaluating the Efficacy, Safety, and Pharmacokinetics (PK) of Subcutaneously Administered Guselkumab for the Treatment of Chronic Plaque Psoriasis in Pediatric Subjects (>=6 To <18 Years of Age)

Primary ObjectivesThe primary objective of this study is to evaluate the efficacy and safety of guselkumab in pediatric participants aged >=6 through <18 years with chronic plaque psoriasis.Secondary ObjectivesThe secondary objectives…

A Multicenter, Open-Label, Longer-Term Study of AR101 Characterized Oral
Desensitization Immunotherapy in Subjects Who Participated in a Prior AR101 Study

Primary: To describe safety and tolerability during longer-term administration of AR101 and follow-up observation after the last dose of AR101.Secondary: - To assess the level of desensitization achievable through extended maintenance dosing of…

A Two-Part Study of ZX008 in Children and Adults with Lennox-Gastaut Syndrome (LGS); Part 1: A Randomized, Double-blind, Placebo-controlled Trial of Two Fixed Doses of ZX008 (Fenfluramine Hydrochloride) Oral Solution as Adjunctive Therapy for Seizures in Children and Adults with LGS, Followed by Part 2: An Open-label Extension to Assess Long-Term Safety of ZX008 in Children and Adults with LGS.

The primary objective of Part 1 is the primary objective of the entire study.Part 1The primary objective of Part 1 is:* To evaluate the effect of ZX008 0.8 mg/kg/day versus placebo as adjunctive therapy for the treatment of uncontrolled seizures in…

A Phase 3 Randomized, Open-Label Study to Assess the Efficacy, Safety,and Pharmacokinetics of Golimumab Treatment, a Human anti-TNFα Monoclonal Antibody, Administered Subcutaneously in Pediatric Participants with Moderately to Severely Active Ulcerative Colitis

Primary Objectives* To evaluate the efficacy of golimumab in inducing clinical remission as assessed by the Mayo score,in pediatric participants with moderately to severely active ulcerative colitis (UC).* To evaluate the safety profile of golimumab…

An Open Label Extension Study of Voxelotor (GBT440) Administered Orally to Participants with Sickle Cell Disease Who Have Participated in Voxelotor Clinical Trials

The objective of this open-label extension (OLE) study is to assess the effect of long-term safety and treatment effect of Voxelotor in participants who have completed treatment in Study GBT440-031, using the following parameters:- Safety based upon…

A multi-center, randomized, double-blind, active and placebo-controlled study to investigate the efficacy and safety of ligelizumab (QGE031) in the treatment of Chronic Spontaneous Urticaria (CSU) in adolescents and adults inadequately controlled with H1-antihistamines

The purpose of this study is to establish efficacy and safety of ligelizumab in adolescent and adult subjects with CSU who remain symptomatic despite standard of care treatment by demonstrating better efficacy over omalizumab.

Efficacy and safety of bumetanide oral liquid formulation in children and adolescents aged from 7 to less than 18 years old with Autism Spectrum Disorder.
A 6-month randomised, double-blind, placebo controlled multicentre parallel group study to evaluate efficacy and safety of bumetanide 0.5mg twice a day followed by an open label active 6-month treatment period with bumetanide (0.5mg twice a day) and a 6 weeks discontinuation period after treatment stop.

The primary objective is to demonstrate the superiority of bumetanide (0.5mg BID) oral liquid formulation compared to placebo in the improvement of ASD core symptoms after 6 months of treatment in ASD children and adolescents aged from 7 to less…