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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…

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.

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),…

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.

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.

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…

Follow You intervention study: Effectiveness of a multidisciplinary training program on daily physical functioning in children and young adolescents with a hereditary connective tissue disorder: a feasibility study

1) Improving the (physical) daily functioning of children with HCTD2) Testing feasibility of intervention for child / parents and therapists

Feasibility study of an orthosis for treatment of a Clubfoot

Primary ObjectiveThe primary objective of this study is to examine the feasibility of using the clubfoot brace. This will be done by recording the effect of the brace on the clubfoot deformity by determining the improvement of the Dimeglio and…

Open label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD patients who have been previously treated in one of the GIVINOSTAT studies.

This study has been transitioned to CTIS with ID 2023-504520-26-00 check the CTIS register for the current data. Primary objective:To assess the long term safety and tolerability of GIVINOSTAT in patients with DMD following core protocols program…

A Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, then Dose Expansion, in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment

This study has been transitioned to CTIS with ID 2023-509935-23-00 check the CTIS register for the current data. Part A:To evaluate the safety and tolerability of multiple ascending doses of SRP-5051 (4 mg/kg, 10 mg/kg, 20 mg/kg, and 30 mg/kg),…

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 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…

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…