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

Status: Completed
Location: See location...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2/Phase 3

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.

Participation Requirements
Sex: All
Minimum Age: 18
Maximum Age: 50
Healthy Volunteers: No

• Aged 18 to 50 years at the time of enrollment

• Have genetically confirmed SMA 3 (homozygous absence of SMN1 exon 7)

• Ability to walk at least 25 meters without assistance

• Be free of major orthopedic deformities (i.e. scoliosis, contractures)

• Normal Cystatin C clearance (> 80 ml/min)

United States
New York
Columbia University Medical Center
New York
Time Frame
Start Date: June 2012
Completion Date: September 2015
Target number of participants: 11
Active Comparator: 4-aminopyridine (Ampyra)
10 mg tab/ 1 tab twice daily
Placebo Comparator: Sugar pill
Placebo 1 tab /twice daily
Leads: Columbia University

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