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NCT01645787 · ClinicalTrials.gov registry record · Phase 2
Short and Long Term Treatment With 4-AP in Ambulatory SMA Patients
A Phase 2 study, sponsored by Columbia University.
- Completed
- Registry status
- Phase 2
- Development phase
- 11
- Enrollment target
NCT01645787: Completed Phase 2 study, sponsored by Columbia University.
NCT01645787 is a Phase 2 study that has completed, run by Columbia University. The registered enrollment target is 11 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (92% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01645787, a Phase 2 study, has completed, sponsored by Columbia University.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 11 participants
- Enrollment target
Study 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.
Primary Outcome
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 chan
Interventions
- DRUG Placebo
- DRUG 4-aminopyridine
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 11 participants |
| Start Date | 2012-06-01 |
| Est. Completion | 2015-09 |
| Phase | Phase 2 |
What the finished NCT01645787 record still lists
NCT01645787 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 11 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (92% lower).
The record links to 0 conditions, and to 2 interventions - of which Placebo is the first listed.
NCT01645787 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT01645787 about?
NCT01645787 is a clinical study titled "Short and Long Term Treatment With 4-AP in Ambulatory SMA Patients". 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.
What is the current status of trial NCT01645787?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 11 participants. The study started on 2012-06-01. Estimated completion is 2015-09.
What interventions are being tested in trial NCT01645787?
The interventions under investigation include: Placebo (DRUG), 4-aminopyridine (DRUG).
Who is sponsoring clinical trial NCT01645787?
This trial is sponsored by Columbia University, which has 958 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT01645787's enrollment target sits among peer trials
11 1967th of 2000 higher than 33 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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