Search for a trial

Motorized arm support augmenting upper extremity function of people with Duchenne Muscular Dystrophy

The primary objectives of the different substudies are: 1) to identify the level and behavior of passive forces in the arm2) compare different weight and joint impedance compensation strategies of the motorized arm support3) evaluate the overall…

A Phase 3 Global, Randomized, Double-Blind, Placebo-Controlled, 48-Week, ParallelGroup Study of the Efficacy and Safety of Losmapimod in Treating Patients with Facioscapulohumeral Muscular Dystrophy (REACH)

This study has been transitioned to CTIS with ID 2024-512737-33-00 check the CTIS register for the current data. Primary Objective:Part A:To evaluate the efficacy of losmapimod for the treatment of FSHD by demonstrating slowing of disease…

A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of INCB000928 in Participants With Fibrodysplasia Ossificans Progressiva (PROGRESS)

This study has been transitioned to CTIS with ID 2023-504129-38-00 check the CTIS register for the current data. Primary:To determine the efficacy of INCB000928 for the prevention of new HO in participants with FOP.Key Secondary:To further evaluate…

Effectiveness of early use of the mechanical insufflation- exsufflation compared to air stacking to reduce respiratory tract infections in patients with Duchenne muscular dystrophy.

The aim of this study is to determine whether MI-E leads to a lower number of RTIs as compared to AS combined with MAC in DMD patients with a PECF< 270 l/min over a period of two years.

An Operationally Seamless, Randomized Phase 2/3 Study Consisting of a Phase 2 Single Blind, Dose-Evaluation Phase and a Phase 3 Double-Blind, Placebo-controlled Phase to Assess the Efficacy and Safety of Setrusumab in Subjects with Osteogenesis Imperfecta

This study has been transitioned to CTIS with ID 2024-510919-29-00 check the CTIS register for the current data. OBJECTIVES - Phase 2 (not to be conducted in NL):Phase 2 Primary Objective:- Identify a setrusumab dosing strategy in subjects with OI…

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…

A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of
Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe
Disease

This study has been transitioned to CTIS with ID 2023-505170-15-00 check the CTIS register for the current data. The objective of the study is to assess the long-term safety and efficacy of intravenous ATB200 co-administration with oral AT2221 in…

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

To evaluate the efficacy and safety of pamrevlumab versus placebo in combination with systemic corticosteroids administered every two weeks in ambulatory subjects with Duchenne muscular dystrophy (age 6 to <12 years).

A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy

Primary Objective:Double-blind period : Evaluate the effect of SRP-4045 and SRP-4053 (combined-active group) compared to placebo on ambulation, endurance, and muscle function, as measured by the 6MWTSecondary Objectives:- Double-blind period:…

A Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys with Duchenne Muscular Dystrophy.

Primary* To compare the efficacy of RO7239361 (BMS-986089) to placebo in ambulatory boys with Duchenne Muscular Dystrophy.Secondary* To compare the efficacy of RO7239361 (BMS-986089) to placebo using the following tests:- 4 stair climb velocity (…

A randomized, placebo-controlled study to assess the safety, tolerability, pharmacokinetics, and effects on heterotopic bone formation of REGN2477 in patients with Fibrodysplasia Ossificans Progressiva

Primary Objective:The primary safety objective of the study is to assess the safety and tolerability of REGN2477 in male and female patients with fibrodysplasia ossificans progressiva (FOP).The primary efficacy objective of the study is to assess…

An Open-Label Pilot Study of Losmapimod to Evaluate the Safety, Tolerability, and Changes in Biomarker and Clinical Outcome Assessments in Subjects With Facioscapulohumeral Muscular Dystrophy 1 (FSHD1) with Extension

This study has been transitioned to CTIS with ID 2024-512736-29-00 check the CTIS register for the current data. Primary Objective1. To evaluate the safety and tolerability of long-term dosing of losmapimod tablets in subjects with FSHD1Secondary…

Inspiratory Muscle Training in Myotonic Dystrophy type 1: a pilot study.

Primary Objective: To study in detail whether a 12-week home based personalized IMT training scheme using the POWERbreathe KHP2 in DM1 patients could result in improvement of maximum inspiratory pressure (PImax) and endurance capacity. We postulate…

Effects of Exercise in Children with a chronic illness: Inflammatory Bowel Disease

The study focuses on children and young people with IBD from 6-18 years old. These children have lower levels of physical activity and a limited endurance compared to healthy children. Through this research we want to improve the endurance of…

A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late Onset Pompe Disease Compared With Alglucosidase Alfa/Placebo.

The objective of the study is to asses the efficacy and safety of intravenous ATB200 co-administration with oral AT2221 by evaluating the changes in key clinical outcome measures (eg, motor, respiratory, fatigue) in adult subjects with late-onset…

A Multicenter, Open-Label Extension Study of WVE-210201 in Patients previously enrolled in WVE-DMDX51-001

Primary objective:• To evaluate the safety and tolerability of WVE-210201Secondary objectives:• To evaluate the effect of WVE-210201 on dystrophin production• To evaluate the concentration of WVE-210201 in plasma following treatment with WVE-210201…

A Randomized, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of DYNE-101 Administered to Participants with Myotonic Dystrophy Type 1

This study has been transitioned to CTIS with ID 2023-510353-42-00 check the CTIS register for the current data. The purpose of this first-in-human study is to evaluate the safety, tolerability, efficacy, pharmacokinetics (PK), and pharmacodynamics…

Inspiratory muscle training in patients with nemaline myopathy

The primary objective is to determine the effect of a 8-week inspiratory muscle training program on respiratory muscle function in nemaline myopathy patients. The secondary objective is to determine respiratory muscle function in nemaline myopathy…

A Prospective Study in Subjects with Late-onset Pompe Disease who are Currently Being Treated with Enzyme Replacement Therapy

The purpose of the study is to evaluate changes in key clinical outcome measures (eg, motor, respiratory, fatigue) in adult subjects with late-onset Pompe disease (LOPD) receiving standard-of-care enzyme replacement therapy (ERT). Additionally,…

Patient-specific cutting blocks to improve tibial tubercle transfer surgery for patients with patellar instability

The primary goal of the current research is to determine the accuracy of the procedure using patient-specific cutting guide. Secondary endpoints are surgical time needed and the guides* fit onto the patients* tibia.