View clinical trials related to Duchenne Muscular Dystrophy.
Filter by:The purpose of this study is to see whether PRO044 is safe and effective to use as medication for Duchenne Muscular Dystrophy (DMD) patients with a mutation around location 44 in the DNA for the dystrophin protein.
The European Home Mechanical Ventilation Registry (EHMVR) will enable a thorough evaluation of HMV by documenting the characteristics of HMV patients and their treatment. This will facilitate a prospective, observational study to identify the primary indications for HMV, describe patterns of HMV use in European countries, and characterize changes in the initiation and utilization of HMV over time. The registry will target all adult individuals who have an indication for HMV. In the EHMVR, patient data from routine clinical care will be documented using an electronic case report form (eCRF). The eCRF will record: patient demographic data; diagnostic information (including primary diagnosis, 6-minute walk time, the presence of depression, and quality of life); blood gases; ventilation treatment (including type of ventilator, modes and settings, interfaces used); follow-up data (including failure rates, side effects, technical issues). An initial Pilot Phase will be launched with the aim to enrol at least 200 patients over a 6-month period to determine the feasibility of the registry. Steering committee members and their institutions will be the main participants in the Pilot Phase. After completion of the Pilot Phase, the registry will be expanded across Europe with the goal of enrolling approximately 10,000 patients over 5 years.
This is a first-in-human, multiple-dose 2-part study to assess the safety, tolerability, efficacy, and pharmacokinetics of SRP-4053 in Duchenne muscular dystrophy (DMD) patients with deletions amenable to exon 53 skipping.
This is a Phase 2 randomized, 2-period, double-blind, placebo-controlled, multiple ascending dose study to evaluate the safety, efficacy, PK and PD of PF-06252616 administered to ambulatory boys diagnosed with Duchenne Muscular Dystrophy. Three intravenous (IV) dose levels will be investigated in a within subject dose escalating fashion. Subjects will be randomly assigned to 1 of 3 sequence groups for approximately 96 weeks (2 periods of 48 weeks each). In period 1, two of the sequence groups will receive PF-06252616 and one sequence group will receive placebo. In period 2, the placebo group will switch to PF-06252616 and the two remaining sequence groups will either receive placebo or PF-06252616. Efficacy will be based on an observed mean change from baseline on function (4 stair climb) of PF-06252616 as compared to the placebo at the end of period 1. Period 2 provides an opportunity to evaluate PK. Subjects will receive monthly IV infused doses of either PF-06252616 or placebo and will undergo safety evaluations (Laboratory, cardiac monitoring, physical exams, x-ray, MRI), functional evaluations (pulmonary function testing, 4 stair climb, range of motion, strength testing, Northstar Ambulatory Assessment, upper limb functional testing and the six minute walk test), pharmacokinetic testing and pharmacodynamic testing to evaluate changes in muscle volume (MRI).
This is an open label, long-term extension study in approximately 24 male DMD subjects consisting of children (ages 4-12, inclusive) and adolescents (ages 13-16, inclusive) who participated in the MP-104-CL-005 PK study.
Duchenne muscular dystrophy (DMD) is a genetic disorder caused by an absence of dystrophin and characterized by progressive muscle degeneration. There is no cure for DMD at present but, there are several strategies under-researched for treatment of DMD such as steroid treatment, gene theraphy, exon skipping, stop codon read through and gene repair, cell theraphy and theraphy with drug that help to produce utrophin protein. The aim of this study is investigate the eficacy of human umblical cord mesenchymal stem cells on DMD and understanding if wild type gene can be transfered to the patient.
Study to characterize the single-state and steady-state dosing of oral deflazacort in pediatric and adolescents subjects.
The objective of this study is to evaluate the safety and pharmacokinetic of TAS-205 in patients with Duchenne Muscular Dystrophy.
The purpose of this study was to study the effect of stem cell therapy in patients with Duchenne Muscular Dystrophy.
This Phase I/II of the clinical trial is to investigate whether the transplantation of normal myoblasts throughout one muscle (the extensor carpi radialis) of the patients is safe and will improve the strength of that muscle. During this Phase I/II, the patients will be transplanted with myoblasts grown from the muscle biopsy of a donor and kept frozen in liquid nitrogen. Thirty million myoblasts will be injected per cm cube in a progressively higher surface of the radialis (i.e., 3, 6 and 9 cm2). The contralateral muscle will be injected with saline to serve as a control. The strength of both muscles will be measured at 3 months post transplantation to verify whether the myoblast transplantation improved the strength of the muscle. If there is no significant strength improvement, the protocol will be terminated immediately for that patient. If there is a significant strength improvement, the patient will be maintained under immunosuppression until 6 months post transplant and his strength will be re-evaluated.