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Short and Long Term Treatment With 4-AP in Ambulatory SMA Patients

Information source: Columbia University
ClinicalTrials.gov processed this data on August 20, 2015
Link to the current ClinicalTrials.gov record.

Condition(s) targeted: Spinal Muscular Atrophy

Intervention: 4-aminopyridine (Drug); Placebo (Drug)

Phase: Phase 2/Phase 3

Status: Active, not recruiting

Sponsored by: Columbia University

Official(s) and/or principal investigator(s):
Claudia A. Chiriboga, MD, MPH, Principal Investigator, Affiliation: Columbia University

Summary

The purpose of this study is to assess whether 4-AP (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.

Clinical Details

Official title: Columbia SMA Project: 4-AP as a Potential SMA Therapeutic Agent and Biological Mechanisms of Action

Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Crossover Assignment, Masking: Double Blind (Subject, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Primary outcome: Six Minute Walk Test (6MWT) with Kinematic Evaluation of Gait

Secondary outcome:

Hammersmith Functional Motor Scale, Expanded (HFMSE)

Manual Muscle Testing (MMT)/Hand Held Dynamometer (HHD)

Change in Motor Unit Number Estimation (MUNE)/Nerve Conduction Study (NCS)

Detailed description: Spinal muscular atrophy (SMA) is a genetically determined neuromuscular disorder that results in muscle weakness and impaired functional mobility. Fatigue is a common symptom in SMA with a resultant impact on physical function and quality of life however the precise mechanisms are unknown. At present there is no treatment for SMA. There is evidence that 4-AP improves function in SMA animal models. In patients with multiple sclerosis, 4-AP was found to improve walking ability and diminish fatigue. The purpose of the study is to determine whether treatment with 4-AP is associated with an increase in walking speed and endurance compared to placebo and whether the duration of treatment affects outcome. The study comprises a short term treatment trial in which participants are treated for 2 weeks with 4-AP and placebo in random sequence followed by a long treatment trial of 6 weeks in which patients are also treated with placebo and 4 AP. The primary outcome measure of the clinical study will be the six minute walk test (6MWT), which has been documented to be a valid and sensitive instrument to identify fatigue among ambulatory SMA patients. We will also assess the effect of 4-AP on muscle and nerve electrical function via electromyography (EMG) during the short term trial. Results of this study may provide support for larger clinical trials.

Eligibility

Minimum age: 18 Years. Maximum age: 50 Years. Gender(s): Both.

Criteria:

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

Locations and Contacts

Columbia University Medical Center, New York, New York 10032, United States
Additional Information

Starting date: June 2012
Last updated: May 13, 2015

Page last updated: August 20, 2015

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