Trial Outcomes & Findings for Safety Study of Transvenous Limb Perfusion in Human Muscular Dystrophy (NCT NCT00873782)

Results Overview

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

Recruitment status

COMPLETED

Study phase

PHASE1

Target enrollment

16 participants

Primary outcome timeframe

Measured within 2 weeks after limb perfusion procedure

Results posted on

2015-03-09

Participant Flow

Participant milestones

Participant milestones
Measure	High Pressure Transvenous Limb Perfusion 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 Study STARTED	16
Overall Study COMPLETED	16
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Safety Study of Transvenous Limb Perfusion in Human Muscular Dystrophy

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	High Pressure Transvenous Limb Perfusion n=16 Participants 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.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	16 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	31 years STANDARD_DEVIATION 8 • n=5 Participants
Sex: Female, Male Female	7 Participants n=5 Participants
Sex: Female, Male Male	9 Participants n=5 Participants
Region of Enrollment United States	16 participants n=5 Participants

PRIMARY outcome

Timeframe: 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

Outcome measures

Outcome measures
Measure	High Pressure Transvenous Limb Perfusion n=16 Participants 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.
Muscle, Nerve, or Vascular Damage	16 participants

Adverse Events

High Pressure Transvenous Limb Perfusion

Serious events: 0 serious events

Other events: 12 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	High Pressure Transvenous Limb Perfusion n=16 participants at risk 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.
Musculoskeletal and connective tissue disorders Ankle pain	12.5% 2/16 • 1 month
Musculoskeletal and connective tissue disorders Elevated Muscle Compartment pressures	62.5% 10/16 • 1 month
Nervous system disorders Reduced Compound Muscle Action Potentials	6.2% 1/16 • 1 month

Additional Information

William J. Powers, MD

University of North Carolina at Chapel HIll

Phone: 919 966 8178

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place