NCT01645787

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

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

• Status: Completed
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 11 (actual)
• Sponsor: Columbia University · Academic / Other
• Sex: All
• Age: 18 Years – 50 Years
• Healthy volunteers: Not accepted

Summary

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.

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.

Conditions

• Spinal Muscular Atrophy

Interventions

Type	Name	Description
DRUG	4-aminopyridine	10 mg/twice daily
DRUG	Placebo	Crossover study involving one trial with sugar pill (placebo)

Timeline

• Start date: 2012-06-01
• Primary completion: 2015-09-01
• Completion: 2015-09-01
• First posted: 2012-07-20
• Last updated: 2024-09-03
• Results posted: 2024-09-03

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study