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Safety Study of Transvenous Limb Perfusion in Human Muscular Dystrophy

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ClinicalTrials.gov Identifier: NCT00873782
Recruitment Status : Completed
First Posted : April 2, 2009
Results First Posted : March 9, 2015
Last Update Posted : March 9, 2015
Sponsor:
Collaborators:
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
University of North Carolina
Information provided by (Responsible Party):
William Powers, University of North Carolina, Chapel Hill

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Muscular Dystrophies
Duchenne Muscular Dystrophy
Becker Muscular Dystrophy
Limb-Girdle Muscular Dystrophy
Intervention Other: Retrograde high pressure transvenous perfusion with normal saline
Enrollment 16

Recruitment Details  
Pre-assignment Details  
Arm/Group Title High Pressure Transvenous Limb Perfusion
Hide Arm/Group Description Participants will undergo retrograde high pressure transvenous limb perfusion of an arm or a leg with normal saline through an 18g or 20g peripheral catheter with a tourniquet on the upper part of the limb: The volume of saline as % of perfused limb volume was escalated beginning at 5%. Safety was determined by Doppler ultrasound to assess venous and arterial damage electrodiagnostic neurographic testing quantitative muscle testing strength assessments, the Action Research Arm Test (ARAT), basic metabolic panel (Na+, K+, Cl-, CO2, BUN, and creatinine), serum creatine kinase [CK], plasma and urine myoglobin. Some subjects underwent MRI to assess whether saline went subcutaneously or intramuscularly.
Period Title: Overall Study
Started 16
Completed 16
Not Completed 0
Arm/Group Title High Pressure Transvenous Limb Perfusion
Hide Arm/Group Description Participants will undergo retrograde high pressure transvenous limb perfusion of an arm or a leg with normal saline through an 18g or 20g peripheral catheter with a tourniquet on the upper part of the limb: The volume of saline as % of perfused limb volume was escalated beginning at 5%. Safety was determined by Doppler ultrasound to assess venous and arterial damage electrodiagnostic neurographic testing quantitative muscle testing strength assessments, the Action Research Arm Test (ARAT), basic metabolic panel (Na+, K+, Cl-, CO2, BUN, and creatinine), serum creatine kinase [CK], plasma and urine myoglobin. Some subjects underwent MRI to assess whether saline went subcutaneously or intramuscularly.
Overall Number of Baseline Participants 16
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 16 participants
<=18 years
0
   0.0%
Between 18 and 65 years
16
 100.0%
>=65 years
0
   0.0%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 16 participants
31  (8)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 16 participants
Female
7
  43.8%
Male
9
  56.3%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 16 participants
16
1.Primary Outcome
Title Muscle, Nerve, or Vascular Damage
Hide Description

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
Time Frame Measured within 2 weeks after limb perfusion procedure
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title High Pressure Transvenous Limb Perfusion
Hide Arm/Group Description:
Participants will undergo retrograde high pressure transvenous limb perfusion of an arm or a leg with normal saline through an 18g or 20g peripheral catheter with a tourniquet on the upper part of the limb: The volume of saline as % of perfused limb volume was escalated beginning at 5%. Safety was determined by Doppler ultrasound to assess venous and arterial damage electrodiagnostic neurographic testing quantitative muscle testing strength assessments, the Action Research Arm Test (ARAT), basic metabolic panel (Na+, K+, Cl-, CO2, BUN, and creatinine), serum creatine kinase [CK], plasma and urine myoglobin. Some subjects underwent MRI to assess whether saline went subcutaneously or intramuscularly.
Overall Number of Participants Analyzed 16
Measure Type: Number
Unit of Measure: participants
16
Time Frame 1 month
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title High Pressure Transvenous Limb Perfusion
Hide Arm/Group Description Participants will undergo retrograde high pressure transvenous limb perfusion of an arm or a leg with normal saline through an 18g or 20g peripheral catheter with a tourniquet on the upper part of the limb: The volume of saline as % of perfused limb volume was escalated beginning at 5%. Safety was determined by Doppler ultrasound to assess venous and arterial damage electrodiagnostic neurographic testing quantitative muscle testing strength assessments, the Action Research Arm Test (ARAT), basic metabolic panel (Na+, K+, Cl-, CO2, BUN, and creatinine), serum creatine kinase [CK], plasma and urine myoglobin. Some subjects underwent MRI to assess whether saline went subcutaneously or intramuscularly.
All-Cause Mortality
High Pressure Transvenous Limb Perfusion
Affected / at Risk (%)
Total   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
High Pressure Transvenous Limb Perfusion
Affected / at Risk (%)
Total   0/16 (0.00%) 
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
High Pressure Transvenous Limb Perfusion
Affected / at Risk (%)
Total   12/16 (75.00%) 
Musculoskeletal and connective tissue disorders   
Ankle pain   2/16 (12.50%) 
Elevated Muscle Compartment pressures   10/16 (62.50%) 
Nervous system disorders   
Reduced Compound Muscle Action Potentials   1/16 (6.25%) 
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Name/Title: William J. Powers, MD
Organization: University of North Carolina at Chapel HIll
Phone: 919 966 8178
Responsible Party: William Powers, University of North Carolina, Chapel Hill
ClinicalTrials.gov Identifier: NCT00873782     History of Changes
Other Study ID Numbers: U54AR056953 ( U.S. NIH Grant/Contract )
U54AR056953 ( U.S. NIH Grant/Contract )
First Submitted: April 1, 2009
First Posted: April 2, 2009
Results First Submitted: January 22, 2015
Results First Posted: March 9, 2015
Last Update Posted: March 9, 2015