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17 studies found for:    "gene therapy" OR "gene transfer" OR "virus delivery" | Muscular Dystrophy
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Rank Status Study
1 Completed Gene Transfer Therapy for Treating Children and Adults With Limb Girdle Muscular Dystrophy Type 2D (LGMD2D)
Condition: Muscular Dystrophies
Interventions: Genetic: rAAV1.tMCK.human-alpha-sarcoglycan- First cohort;   Genetic: Genetic: rAAV1.tMCK.human-alpha-sarcoglycan- Second cohort
2 Active, not recruiting Clinical Intramuscular Gene Transfer of rAAV1.CMV.huFollistatin344 Trial to Patients With Duchenne Muscular Dystrophy
Condition: Duchenne Muscular Dystrophy
Intervention: Biological: rAAV1.CMV.huFollistin344
3 Active, not recruiting Clinical Intramuscular Gene Transfer Trial of rAAVrh74.MCK.Micro-Dystrophin to Patients With Duchenne Muscular Dystrophy
Condition: Duchenne Muscular Dystrophy
Intervention: Biological: rAAVrh74.MCK.micro-Dystrophin
4 Completed Clinical Study of AAV1-gamma-sarcoglycan Gene Therapy for Limb Girdle Muscular Dystrophy Type 2C
Conditions: Limb Girdle Muscular Dystrophy Type 2C;   Gamma-sarcoglycanopathy
Intervention: Biological: AAV1-gamma-sarcoglycan vector injection
5 Withdrawn Gene Transfer Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MCK.GALGT2
Condition: Duchenne Muscular Dystrophy
Interventions: Biological: rAAVrh74.MCK.GALGT2;   Other: PLACEBO (Saline)
6 Active, not recruiting Follistatin Gene Transfer to Patients With Becker Muscular Dystrophy and Sporadic Inclusion Body Myositis
Conditions: Becker Muscular Dystrophy;   Sporadic Inclusion Body Myositis
Intervention: Biological: rAAV1.CMV.huFollistatin344
7 Enrolling by invitation Gene Transfer Clinical Trial for LGMD2D (Alpha-sarcoglycan Deficiency) Using scAAVrh74.tMCK.hSGCA
Condition: Limb Girdle Muscular Dystrophy Type 2D (LGMD2D)
Intervention: Drug: scAAVrh74.tMCK.hSGCA
8 Completed
Has Results
Safety Study of Transvenous Limb Perfusion in Human Muscular Dystrophy
Conditions: Muscular Dystrophies;   Duchenne Muscular Dystrophy;   Becker Muscular Dystrophy;   Limb-Girdle Muscular Dystrophy
Intervention: Other: Retrograde high pressure transvenous perfusion with normal saline
9 Completed Safety Study of Mini-dystrophin Gene to Treat Duchenne Muscular Dystrophy
Condition: Duchenne Muscular Dystrophy
Intervention: Biological: rAAV2.5-CMV-minidystrophin (d3990)
10 Completed A Multicenter Randomized Placebo-Controlled Double-Blind Study to Assess Efficacy and Safety of Glutamine and Creatine Monohydrate in Duchenne Muscular Dystrophy (DMD)
Condition: Muscular Dystrophy, Duchenne
Interventions: Drug: glutamine;   Drug: creatine monohydrate
11 Recruiting rAAVrh74.MHCK7.DYSF.DV for Treatment of Dysferlinopathies
Condition: Dysferlinopathy
Intervention: Drug: rAAVrh74.MHCK7.DYSF.DV
12 Active, not recruiting Genetic Characterization of Individuals With Limb Girdle Muscular Dystrophy
Condition: Limb-Girdle Muscular Dystrophy
Intervention:
13 Completed Six Month Study of Gentamicin in Duchenne Muscular Dystrophy With Stop Codons
Condition: Duchenne Muscular Dystrophy
Intervention: Drug: Gentamicin infusions twice a week for six months
14 Unknown  Endomysial Fibrosis, Muscular Inflammatory Response and Calcium Homeostasis Dysfunction in Duchenne Muscular Dystrophy
Condition: Duchenne Muscular Distrophy (DMD)
Intervention: Other: Muscle biopsy
15 Completed
Has Results
Cardiomyopathy in DMD: Lisinopril vs. Losartan
Conditions: Duchenne Muscular Dystrophy (DMD);   Cardiomyopathy
Interventions: Drug: Losartan;   Drug: Lisinopril
16 Active, not recruiting Historically Controlled Trial of Corticosteroids in Young Boys With Duchenne Muscular Dystrophy
Condition: Duchenne Muscular Dystrophy
Intervention: Drug: Prednisolone
17 Recruiting Confirmatory Study of Eteplirsen in DMD Patients
Condition: Duchenne Muscular Dystrophy (DMD)
Intervention: Drug: eteplirsen

Study has passed its completion date and status has not been verified in more than two years.