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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.

A Placebo-Controlled, Randomized, Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Biological Activity of ATYR1940 in Adult Patients with Molecularly Defined Genetic Muscular Dystrophies

Primary objective:Evaluate the safety, tolerability, pharmacokinetics (PK), and immunogenicity of multiple doses of intravenous (IV) ATYR1940 in adults 18 to 65 years of age, inclusive, with FSHDSecondary objective:Explore pharmacodynamic (PD)…

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.

Optimizing the intermanual transfer effects after training with a prosthetic simulator

The objective of this study is to determine the optimal myo-electric prosthesis training for obtaining the largest transfer effect. Here fore we plan to execute three experiments. At first it needs to be revealed which trainingtasks leads to the…

Intermanual transfer using mental imagery in prosthetic training

The objective of this study is to determine the surplus value of mental imagery on myo-electric prosthesis training Here fore we plan to execute two experiments. The goal of the first experiment is to test if intermanual transfer effects of able-…

A Randomized, Double-Blind, Placebo-Controlled, Phase 2 Study to investigate the Safety and Efficacy of ABT-494 Given with Methotrexate (MTX) in Subjects with Moderately to Severely Active Rheumatoid Arthritis (RA) Who Have Had an Inadequate Response or Intolerance to Anti-TNF Biologic Therapy

The primary objective is to compare the safety and efficacy of multiple doses of ABT-494 versus placebo in moderately to severely active RA subjects on stable background MTX therapy with inadequate response or intolerance to anti-TNF biologic…

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…

A phase II, double blind, exploratory, parallel-group, placebo-controlled clinical study to assess two dosing regimens of GSK2402968 for efficacy, safety, tolerability and pharmacokinetics in ambulant subjects with Duchenne muscular dystrophy

Primary objective: • To assess the efficacy of 2 different dosing regimens of subcutaneous GSK2402968 administered over 24 weeks in ambulant subjects with DMD.Secondary objectives:• To assess the safety and tolerability of 2 different dosing…

Reduction of Bone Morbidity using Oral Bisphosphonate in Fibrous Dysplasia of Bone

To evaluate the effect of the bisphosphonate risedronate (together with vitamin D and calcium or vitamin D calicum and phosphate in patients with renal phosphate wasting) on bone pain and the evolution of osteolytic bone lesions in patients with FD.

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…