Phase 1
N=16
Safety Study of Transvenous Limb Perfusion in Human Muscular Dystrophy
Muscular Dystrophies · Duchenne Muscular Dystrophy · Becker Muscular Dystrophy · Limb-Girdle Muscular Dystrophy
Bottom Line
View on ClinicalTrials.gov: NCT00873782 ↗Enrolled (actual)
16
Serious AEs
0.0%
Results posted
Mar 2015
Primary outcome: Primary: Muscle, Nerve, or Vascular Damage — 16 participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 1
- Interventions
- Retrograde high pressure transvenous perfusion with normal saline (Other)
- Age
- Adult, Older Adult · 21+ yrs
- Sex
- All
- Sponsor
- University of North Carolina, Chapel Hill
- Primary completion
- Feb 2014
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Muscle, Nerve, or Vascular Damage |
16 | — |
Summary
Muscular dystrophies are inherited disorders in which the skeletal and heart muscles become progressively weaker, sometimes leading to permanent disability. Current treatments aim to control symptoms as much as possible, but there is no cure. Gene therapy, in which defective genes causing the disorder are corrected, is a potential treatment option and is in the process of being developed for muscular dystrophies. This study will determine the safety and feasibility of a particular delivery method for gene therapy that could be used in the future to treat people with muscular dystrophies. Only normal saline, and no active treatment, will be used in this study.
Eligibility Criteria
Inclusion Criteria
- Diagnosis of Duchenne or Becker muscular dystrophy, as defined by progressive weakness with onset before the age of 21, X-linked inheritance, and reduced dystrophin (less than 3%) on muscle biopsy OR mutation in the dystrophin gene
- Diagnosis of limb girdle muscular dystrophy, as defined by progressive weakness with onset before the age of 21, normal dystrophin on muscle biopsy OR proven mutation associated with one of the types of limb girdle dystrophy
- Older than 21 years of age and preferably younger 30 years of age
- Able to stand, independently or with assistance
- Able to communicate with pertinent staff
- Able to understand and willingly comply with the requirements of the study
Exclusion Criteria
- Confirmed diagnosis of any other muscle disease
- Previous compartment syndrome requiring surgical decompression
- Previous venous or arterial thrombosis other than superficial venous thrombosis associated with intravenous catheter
- Coagulopathy, including known diagnosis of bleeding diathesis, history of excessive bleeding on multiple occasions, or taking anticoagulant or platelet inhibitory medications
- Systemic arterial or venous disease (e.g., Raynaud's, aortic coarctation or aneurysm)
- Previous injury to selected limb with residual effect other than superficial scarring
- Previous vascular surgery to selected limb
- Previous compressive neuropathy (e.g., carpal tunnel syndrome in arm, peroneal palsy in leg)
- Complex regional pain syndrome or other neurological cause of limb pain
- Previous clinical diagnosis of congestive heart failure
- Previous echocardiography showing ejection fraction less than 40% or ventricular dilation
- Previous chest x-ray showing enlarged cardiac silhouette or pulmonary edema
- History of rhabdomyolysis with worsening renal function
- Creatinine greater than 1.7 mg/dL
- Resting hypoxemia with SaO2 less than 90% on room air
- Other significant heart, lung, or kidney disease that would compromise the body's capacity to handle a fluid load
- Previous forced vital capacity less than 75% of age and height adjusted norm, in the absence of acute reversible pulmonary disease
- Sickle cell disease (sickle cell anemia [SS] or sickle hemoglobin C disease [SC])
- Pregnant
- Non-English speaker
Data sourced from ClinicalTrials.gov (NCT00873782). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.