View clinical trials related to Muscular Dystrophy, Duchenne.
Filter by:The main purpose of this study is to test the safety and tolerability of different, increasing doses of an experimental medication called HT-100 in boys and young men with Duchenne muscular dystrophy (DMD). The study medication, HT-100, is a medicine that may help promote healthy muscle regeneration, diminish inflammation and the resulting damage to muscle, and decrease the scar tissue that forms in the muscles of children with DMD. In this study, pharmacokinetic sampling, or measurements of the amount of HT-100 in the bloodstream will also be taken.
The purpose of the study is to see whether PRO045 is safe and effective to use as medication for Duchenne Muscular Dystrophy (DMD) patients with a mutation around location 45 in the DNA for the dystrophin protein.
This is a phase III, multicenter, open-label, uncontrolled extension study in male subjects with DMD open to eligible US and Canadian subjects who previously participated in the following studies of drisapersen: DMD114876, DMD114044 and DMD114349. Subjects will receive 6mg/kg subcutaneous drisapersen on a weekly basis. For subjects who have previously experienced significant safety or tolerability issues or who experience these during the study, there is the potential of an alternate intermittent dosing arm that will be given as a regimen of 6 mg/kg weekly for 8 weeks followed by 4 weeks off treatment. For subjects who experience or have previously experienced significant safety/tolerability issues, side effects or reactions or intermittent dosing, intravenous dosing will be made available.
To characterize the natural history and progression of Duchenne Muscular Dystrophy (DMD) to help inform the design of future studies, to capture biomarkers of safety and disease progression and to provide comparative data for the development of rare exons for which formal controlled trials are not feasible.
The purpose of this study is to explore long-term safety, tolerability and efficacy of GSK2402968 in DMD subjects who previously participated in either DMD114117 or DMD114044.
Background: We want to learn more about the relationship between the way families function and how children adapt to having a sibling with Duchenne muscular dystrophy (DMD). What we learn will help us design better interventions for families. Objective: - To learn more about how families with an individual with DMD function. - To learn how siblings adapt in families with an individual with DMD. Eligibility: - One parent and one child, age 13-18, from a family where another child has DMD. - The parent and the child must be able to read and write English. Design: - One parent from each family will complete a survey about how family members communicate and relate with each other. The parent will also answer questions about the behavior of the child without DMD. This survey will take you about 40 minutes to complete. - One child from each family, either a boy or a girl, will also complete a survey. This survey asks about how he/she views him/herself. It also asks about how he/she interacts with peers and family members and how he/she behaves. The survey also asks how satisfied he/she is with how his/her family functions. This survey takes about 30 minutes to finish.
To evaluate the long-term safety and tolerability of ACE-031 administration in subjects with Duchenne muscular dystrophy (DMD) who participated in Study A031-03. [Note: This study was terminated based on preliminary safety data. Pending further analysis of safety data and discussion with health authorities, a new ACE-031 trial will be planned.]
This study, supported by Charley's Fund, Inc., is being done to determine if the drug Revatio®(also known as Sildenafil), as compared to placebo (an inactive substance that looks like the study drug, but contains no medication), improves heart function in people with Duchenne Muscular Dystrophy and Becker Muscular Dystrophy (DBMD). In people with DBMD, dystrophin is not present or lacking in heart and muscle. This is associated with abnormalities in an enzyme called "neuronal nitric oxide synthase" or nNOS, and leads to decreases in "cyclic GMP," which is necessary for proper function of those muscles. Revatio blocks an enzyme called phosphodiesterase 5 (PDE5), and helps to restore the normal amounts of cyclic GMP. The purpose of this research is to determine if Revatio is safe for people with DBMD and if it can improve heart function. Hypothesis : PDE5 inhibition, with the use of Revatio, will improve cardiac function in patients with DBMD.
The purpose of this study is to determine if ACE-031 is safe and well-tolerated in boys with Duchenne Muscular Dystrophy (DMD) and to select the optimal doses of ACE-031 in terms of safety and pharmacodynamic (PD) activity for designing future studies. [Note: This study was terminated based on safety data]
Duchenne/Becker muscular dystrophy (DMD/BMD) is a genetic disorder that develops in boys. It is caused by a mutation in the gene for dystrophin, a protein that is important for maintaining normal muscle structure and function. Loss of dystrophin causes muscle fragility that leads to weakness and loss of walking ability during childhood and teenage years. A specific type of mutation, called a nonsense (premature stop codon) mutation is the cause of DMD/BMD in approximately 10-15% of boys with the disease. Ataluren (PTC124) is an orally delivered, investigational drug that has the potential to overcome the effects of the nonsense mutation. This study is a Phase 2a trial that enrolled boys with nonsense mutation DMD/BMD who have lost independent mobility due to the disease. This study evaluated the safety and tolerability of ataluren (PTC124) and also evaluated efficacy outcomes in this participant population.