Short and Long Term Treatment With 4-AP in Ambulatory SMA Patients
|ClinicalTrials.gov Identifier: NCT01645787|
Recruitment Status : Completed
First Posted : July 20, 2012
Last Update Posted : May 18, 2016
|Condition or disease||Intervention/treatment||Phase|
|Spinal Muscular Atrophy||Drug: 4-aminopyridine Drug: Placebo||Phase 2 Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||11 participants|
|Intervention Model:||Crossover Assignment|
|Masking:||Triple (Participant, Investigator, Outcomes Assessor)|
|Official Title:||Columbia SMA Project: 4-AP as a Potential SMA Therapeutic Agent and Biological Mechanisms of Action|
|Study Start Date :||June 2012|
|Actual Primary Completion Date :||September 2015|
|Actual Study Completion Date :||September 2015|
U.S. FDA Resources
Active Comparator: 4-aminopyridine (Ampyra)
10 mg tab/ 1 tab twice daily
10 mg/twice daily
Placebo Comparator: Sugar pill
Placebo 1 tab /twice daily
Crossover study involving one trial with sugar pill (placebo)
Other Name: Sugar pill
- Six Minute Walk Test (6MWT) with Kinematic Evaluation of Gait [ Time Frame: Up to 21 Weeks ]The primary outcome measure will be distance walked in the 6MWT. This measure is an objective evaluation of functional capacity which measures the distance a person can walk quickly in six minutes and is most representative of a person's ability because the test intensity is self-selected. The 6MWT can be safely performed in ambulatory SMA patients and correlates with standard SMA outcome measures including timed walking tests. In SMA, the 6MWT may be more sensitive to clinically meaningful changes in patients with type 3 SMA as it is a direct measure of their functional mobility.
- Hammersmith Functional Motor Scale, Expanded (HFMSE) [ Time Frame: Up to 21 Weeks ]Assessments of motor function are clinically relevant and are a good adjunct to tests of walking ability. The HFMSE, a 33-item scale designed for SMA type 2 and 3 patients, and is associated with minimal patient burden requiring only standard equipment and is completed on average in less than 15 minutes. The HFMSE showed good test-retest reliability and is correlated with other clinical and physiological measures in SMA.
- Manual Muscle Testing (MMT)/Hand Held Dynamometer (HHD) [ Time Frame: Up to 21 Weeks ]MMT will involve pushing and pulling against the evaluators hand (MMT) and HHD will involve pushing or pulling as against a handheld measuring device. The purpose of these tests is to measure the strength in different muscles. The MMT involves testing fourteen muscle groups of the arm and leg on both sides of the body. The evaluator will alternate sides between tests. The measuring device will be used on 10 muscle groups on both sides of the body.
- Change in Motor Unit Number Estimation (MUNE)/Nerve Conduction Study (NCS) [ Time Frame: Baseline, Week 2 and Week 5 ]Motor Unit Number Estimation (MUNE) is a noninvasive test that identifies the number of surviving motor units (motor nerve cells and the territory of muscle fibers they control) using electrical muscle stimulation and recording the response. The nerve conduction study involves the administration of modest electrical stimulations (pulsations or throbbing sensations from low level electricity) to a total of 4 nerves in your right arm and leg while recording the response over a muscle innervated by each nerve.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01645787
|United States, New York|
|Columbia University Medical Center|
|New York, New York, United States, 10032|
|Principal Investigator:||Claudia A. Chiriboga, MD, MPH||Columbia University|