Clinical Trial: Safety Study of Transvenous Limb Perfusion in Human Muscular Dystrophy

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: Safety and Feasibility of Transvenous Limb Perfusion With Normal Saline in Human Muscular Dystrophy

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 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.

Detailed Summary:

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 the legs and pelvis and which can lead to cardiomyopathy, deformities, respiratory failure, and permanent disability. Limb-girdle muscular dystrophy, another type, is also characterized by progressive muscle weakness, first affecting the muscles around the shoulder girdle and hips and possibly affecting other muscles as the disorder progresses. Complications of limb-girdle muscular dystrophy can include abnormal heart rhythms, joint contractures, difficulties with activities of daily living, significant loss of mobility, and permanent disability. There is no cure for muscular dystrophies. Current treatments, such as steroids, mobility aids, physical therapy, and respiratory care, can decrease some of the complications, but there is a clear need for a curative therapy.

The genetic basis of muscular dystrophies is well understood, which makes gene therapy a potential treatment option in the future. A key step in developing gene therapy involves first ensuring safe delivery of genetic material into a single limb of a patient before using active treatment in the patient's entire body. High-pressure, high-volume transvenous limb perfusion, in which fluid is forced under high pressure into arm and leg muscles through the veins, has shown the greatest potential as a delivery method. The purpose of this study is to determine the safety and feasibility of this method with normal saline in people who have Becker's muscular dystrophy or limb-girdle muscular dystrophy.

Participation in this study will last up to 4 weeks. At an initial baseline visit, participants will undergo water emersion measurements of their arm and leg, nerve testing
Sponsor: University of North Carolina, Chapel Hill

Current Primary Outcome: Muscle, Nerve, or Vascular Damage [ Time Frame: Measured within 2 weeks after limb perfusion procedure ]

Number of Participants with all of the following three:

  1. Unchanged Doppler ultrasound to assess venous and arterial damage pre-and post perfusion based on report
  2. Without clinically significant changes in electrodiagnostic testing using standard neurographic techniques pre-and post perfusion:>1 mSec change in baseline distal motor latency; <75% baseline compound muscle action potential amplitude, <75% baseline conduction velocity, sensory nerve action potential
  3. Without clinically significant changes in Quantitative muscle testing (QMT) strength assessments pre-and post perfusion:< 85% baseline


Original Primary Outcome: Muscle, Nerve, or Vascular Damage [ Time Frame: Measured 2 weeks after limb perfusion procedure ]

Current Secondary Outcome:

Original Secondary Outcome:

Information By: University of North Carolina, Chapel Hill

Dates:
Date Received: April 1, 2009
Date Started: March 2009
Date Completion:
Last Updated: February 27, 2015
Last Verified: January 2015