Safety Study of Transvenous Limb Perfusion in Human Muscular Dystrophy

NCT00873782 | Completed Phase 1 Results DMC

• 1 other identifier: U54AR056953
• Study Type: interventional
• Target: 16
• Locations: 1 country
• Sites: 1

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.

Conditions

Muscular Dystrophies; Duchenne Muscular Dystrophy; Becker Muscular Dystrophy; Limb-Girdle Muscular Dystrophy

Keywords

Limb Perfusion; Gene Therapy

Outcome Measures

Primary Outcomes (1)

• Muscle, Nerve, or Vascular Damage

  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

  Measured within 2 weeks after limb perfusion procedure

Study Arms (1)

1

EXPERIMENTAL

Participants will undergo retrograde high pressure transvenous limb perfusion with normal saline.

Other: Retrograde high pressure transvenous perfusion with normal saline

Interventions

Retrograde high pressure transvenous perfusion with normal saline OTHER

Dose escalation of saline volume, infusion rate, and tourniquet pressure, as determined in a stepwise manner and by careful monitoring

1

Eligibility Criteria

• Age: 21 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• University of North Carolina, Chapel Hill: lead
• National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS): collaborator
• University of North Carolina: collaborator

Study Sites (1)

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, 27599, United States

Location

MeSH Terms

Conditions

Muscular Dystrophies; Muscular Dystrophy, Duchenne; Muscular Dystrophies, Limb-Girdle

Interventions

Saline Solution

Condition Hierarchy (Ancestors)

Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, X-Linked

Intervention Hierarchy (Ancestors)

Crystalloid Solutions; Isotonic Solutions; Solutions; Pharmaceutical Preparations

Results Point of Contact

• Title: William J. Powers, MD
• Organization: University of North Carolina at Chapel HIll

powersw@neurology.unc.edu

919 966 8178

Study Officials

• William J. Powers, MD

  University of North Carolina

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor of Neurology

Study Record Dates

• First Submitted: April 1, 2009
• First Posted: April 2, 2009
• Study Start: March 1, 2009
• Primary Completion: February 1, 2014
• Study Completion: February 1, 2014
• Last Updated: March 9, 2015
• Results First Posted: March 9, 2015

Record last verified: 2015-01

Locations

US (1)