Spinal Muscular Atrophy Clinical Trial
Official title:
Columbia SMA Project: 4-AP as a Potential SMA Therapeutic Agent and Biological Mechanisms of Action
Verified date | May 2016 |
Source | Columbia University |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
The purpose of this study is to assess whether 4-AP (Dalfampridine-ER, Ampyra) improves walking ability and endurance in adult patients with Spinal muscular atrophy (SMA) Type 3 compared to placebo and whether the duration of treatment affects outcome.
Status | Completed |
Enrollment | 11 |
Est. completion date | September 2015 |
Est. primary completion date | September 2015 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 18 Years to 50 Years |
Eligibility |
Inclusion Criteria: 1. Aged 18 to 50 years at the time of enrollment 2. Have genetically confirmed SMA 3 (homozygous absence of SMN1 exon 7) 3. Ability to walk at least 25 meters without assistance 4. Be free of major orthopedic deformities (i.e. scoliosis, contractures) 5. Normal Cystatin C clearance (> 80 ml/min) Exclusion Criteria: 1. Patients with a history of seizures 2. Patients with any renal impairment 3. Inability to comply with the study procedures 4. Unstable medical illness 5. Any ventilatory assistance 6. Taking experimental medication for SMA other than under this protocol 7. Pregnancy or lactation 8. Menstruating women, not sterilized or not using effective birth control 9. Planning to undergo scoliosis surgery within the next 10 months 10. Inability to give informed consent |
Country | Name | City | State |
---|---|---|---|
United States | Columbia University Medical Center | New York | New York |
Lead Sponsor | Collaborator |
---|---|
Columbia University |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Six Minute Walk Test (6MWT) with Kinematic Evaluation of Gait | 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. | Up to 21 Weeks | |
Secondary | Hammersmith Functional Motor Scale, Expanded (HFMSE) | 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. | Up to 21 Weeks | |
Secondary | Manual Muscle Testing (MMT)/Hand Held Dynamometer (HHD) | 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. | Up to 21 Weeks | |
Secondary | Change in Motor Unit Number Estimation (MUNE)/Nerve Conduction Study (NCS) | 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. | Baseline, Week 2 and Week 5 |
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