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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)…

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…

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…

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.

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.

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:The primary objective of this study is to evaluate the effect of SRP-4045 and SRP-4053 (combined-active group) compared to placebo onambulation, endurance, and muscle function at Week 96, as measured by the 6-minute walk test (6MWT…

A Phase 2, Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study of Palovarotene in Subjects with Multiple Osteochondromas

Primary Objective• To evaluate the efficacy of two dosage regimens of palovarotene compared with placebo in preventing new osteochondromas (OCs) in subjects with multiple osteochondromas (MO) due to exostosin 1 (Ext1) or exostosin 2 (Ext2) mutations…

A Phase IIb Randomized, Double-blind, Parallel Group, Placebo- and Active-controlled Study with Double-Blind Extension to Assess the Efficacy and Safety of Vamorolone in Ambulant Boys with Duchenne Muscular Dystrophy (DMD)

Primary objectives: 1. To compare the efficacy of vamorolone administered orally at daily doses of 6.0 mg/kg over a 24-week treatment period vs. placebo in ambulant boys ages 4 to <7 years with DMD; and2. To evaluate the safety and…

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…

A Phase 3 Randomized, Double-Blind, Parallel-Group, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Daily Subcutaneous Injections of Elamipretide in Subjects with Primary Mitochondrial Disease Resulting from Pathogenic Nuclear DNA Mutations (nPMD)

The primary objective of this study is to evaluate the effect of single daily SC administration of elamipretide for 48 weeks on the distance walked (in meters) on the 6-minute walk test.The secondary objectives of this study are:- To evaluate the…

A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of High Doses of Eteplirsen, Preceded by an Open-Label Dose Escalation, in Patients with Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping

This study has been transitioned to CTIS with ID 2024-511492-15-00 check the CTIS register for the current data. Part 1:to evaluate the safety and tolerability of 2 doses (100 milligrams/kilogram [mg/kg] and 200 mg/kg) of eteplirsen in approximately…

Active Monitoring versus an Abduction Device for treatment of Infants with Centered Dysplastic Hips, a Comprehensive Cohort Study (TReatment with Active Monitoring (TRAM)-Trial)

The aim of this study is to compare treatment with an abduction device to active monitoring of infants with centered dysplastic hips (Graf type IIa- or IIb or IIc) during the first year of life.

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

To evaluate the efficacy and safety of pamrevlumab versus placebo in combination with systemic corticosteroids in subjects with non-ambulatory Duchenne muscular dystrophy (age 12 years and older).

Phase 2 Active Treatment Study to Evaluate the Efficacy and Safety of SRK 015 in Patients with Later onset Spinal Muscular Atrophy (TOPAZ)

Objectives for both the Treatment Period and Extension Period:Primary Objectives: - To assess safety and tolerability of the study medicine in patients with later onset (e.g., Type 2 and Type 3) spinal muscular atrophy (SMA)- To assess the efficacy…

Saracatinib trial TO Prevent FOP

This study has been transitioned to CTIS with ID 2024-515186-33-00 check the CTIS register for the current data. The three key outcome measurements and challenges of this proof of concept study are to achieve a decrease in ongoing HO bone formation…

DEnosumab for the treatment of FIbrous Dysplasia/McCune-Albright Syndrome in adults (DeFiD): a randomized double-blind placebo-controlled trial

This study has been transitioned to CTIS with ID 2024-511090-30-00 check the CTIS register for the current data. To investigate whether 3 monthly Dmab will improve the clinical, radiological and biochemical manifestations of FD bone lesions.