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An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, Biological Activity, and Systemic Exposure of ATYR1940 in Adult Patients with Facioscapulohumeral Muscular Dystrophy (FSHD)

The objectives of this study are to:• Evaluate the safety, tolerability, and immunogenicity of long-term treatment with intravenous (IV) ATYR1940 in adult patients with facioscapulohumeral muscular dystrophy (FSHD) previously enrolled in clinical…

Phase 2 Study, Multicenter, Open-Label Extension (OLE) Study in Rheumatoid Arthritis Subjects Who Have Completed a Preceding Phase 2 Randomized Controlled Trial (RCT) with ABT-494

To evaluate the long-term safety, tolerability, and efficacy of ABT-494 in RA subjects who have completed Study M13-550 or Study M13-537 Phase 2 RCT with ABT-494.

Multicenter, open-label, single arm study to evaluate long-term safety, tolerability, and effectiveness of 10mg/kg olesoxime in patients with SMA

Main objective: The main objective of this open-label, single arm study is to further characterize the safety, tolerability and effectiveness profile of olesoxime in SMAThe primary safety objective for this study is as follows:To evaluate the safety…

A prospective randomised controlled study to evaluate ankle dorsiflexion after cast immobiliation and percutaneous gastrocnemius lengthening, for neurologic healthy children (6-18) with an symptomatic equinus contracture.

By making this design study we wish to contribute to profound research on percutaneous gastrocnemius lengthening for for children with an equinus contracture and prevent publication bias for this open-labelled randomized trial.

Treatment trial with Losartan of a patient with stiff skin syndrome.

1. The effect of Losartan on the joint excursions and the Hardening of the skin in a patient with SSS.2. Gain insight in the pathogenesis of SSS3. Gain insight in the functional effect of Losartan in SSS.

Clinical application of simulators when learning to use upper limb prostheses in children

The objective of this study is to establish the effects of transfer of learning in children with upper limb deficiencies after practising with an upper limb prosthetic simulator.In children, transfer of learning effects have hardly been studied. The…

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.

Arm training in boys with Duchenne Muscular Dystrophy

To determine whether 3D arm training with arm-support is effective in delaying the loss of arm functions in daily activities from an early age onwards in boys with DMD.

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…

advancement of the mandible by bilateral sagital spit osteotomy and distraction osteogenesis; a randomised clinical trial

To evaluate the two procedures for lengthening of the mandible.

A Phase III Open-Label, Single-Group, Extension Study to
Obtain Long-Term Safety and Tolerability Data of
Idebenone in the Treatment of Friedreich*s Ataxia Patients

- To gather long-term data on the safety and tolerability of idebenone inFriedreich*s Ataxia- To explore the effect of idebenone after longer term administration onneurological symptoms and signs as assessed by the InternationalCooperative Ataxia…

The Congenital Hand Anomalies: a study of function and daily use

Objective of the study is to evaluate the long term results of the interventions of the above mentioned deformities.

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…

Effectivity of splinting therapy for correction of anatomy in children with congenital hand deformities

The objectives of this study are to gain more insight in the efficacy of splinting therapy for correction of the anatomy and answering the followig questions:1.What are the effects of conservative splinting therapy in children with congenital hand…

The feasibilty and energy expenditure of adults with a spastic bilateral cerebral palsy playing Wii-Sports.

The aim of this study is to determine the feasibility and energy expenditure of adults with a spastic bilateral CP playing Wii-Sports.

Interventions with Music in Pectus Excavatum Treatment

This research will investigate a non-pharmacological, low-cost, durable and safe method during and after pectus excavatum surgery, to alleviate postoperative pain and thereby reduce the dose of analgesics used. Our secondary aim is to investigate…

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…

An open-label study to investigate the safety, tolerability, and Pharmacokinetics/Pharmacodynamics of risdiplam (RO7034067) in adult and pediatric patients with spinal muscular atrophy

This study has been transitioned to CTIS with ID 2023-506739-14-00 check the CTIS register for the current data. Primary ObjectivesThe primary objectives of this study are as follows:• To evaluate the safety and tolerability of risdiplam.• To…

A Phase 1, open-label, single-dose, single-center, mass balance study of IPN60130 following a single 60-mg oral dose of IPN60130 containing 9 µCi of [14C]-IPN60130 in healthy male participants

In this study we will investigate how quickly and to what extent the new compound IPN60130 is absorbed, transported, and eliminated from the body (this is called pharmacokinetics). IPN60130 is radioactively labelled with carbon-14 (14C). In this way…