Status:
COMPLETED
Safety Study of Transvenous Limb Perfusion in Human Muscular Dystrophy
Lead Sponsor:
University of North Carolina, Chapel Hill
Collaborating Sponsors:
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
University of North Carolina
Conditions:
Muscular Dystrophies
Duchenne Muscular Dystrophy
Eligibility:
All Genders
21+ years
Phase:
PHASE1
Brief 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 ...
Detailed Description
There are many types of muscular dystrophies, all of which are progressive, degenerative genetic disorders. One type is Becker's muscular dystrophy, which involves slowly worsening muscle weakness of ...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
March 1 2009
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
February 1 2014
Estimated Enrollment :
16 Patients enrolled
Trial Details
Trial ID
NCT00873782
Start Date
March 1 2009
End Date
February 1 2014
Last Update
March 9 2015
Active Locations (1)
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1
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, United States, 27599