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6 minutes assisted leg and arm cycling test: feasibility, validity, test-retest responsivity and responsivity in patients with neuromuscular and metabolic disorders

2.1 Primary Objectives: 1) To test the feasibility of the six minute assisted leg and arm cycling test in patients aged 6-18 years with Duchenne muscular dystrophy (DMD), Beckers muscular dystrophy (BMD), Limb Girdle muscular dystrophy (LGMD),…

Physical fitness levels of wheelchair-bound children and adolescents with osteogenesis imperfecta: a feasibility and reliability study

To investigate whether children with OI who use a wheelchair (for long distances) can reliably perform a valid shuttle ride test in order to assess the levels of health-related fitness in this group.

Clinical application of simulators when learning to use upper limb prostheses

The objective of this study is to determine the application of the prosthesis simulators and the bimanual transfer effects in the existing rehabilitation programs for patients who will obtain an upper limb prosthesis.At first it needs to be revealed…

The Ehlers-Danlos syndrome and regional anesthesia

Aim of this study is measuring the effects of locoregional anaesthesia in EDS patients and comparing these effects with those in a healthy control group. The results of this study can be used to make a more valid choice for the type of anaesthesia…

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…

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 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…

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…

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.