Phase 3
Completed N=11
Short and Long Term Treatment With 4-AP in Ambulatory SMA Patients
Source: ClinicalTrials.gov NCT01645787 ↗Enrolled (actual)
11
Serious AEs
0.0%
Results posted
Sep 2024
Primary outcomePrimary: Six Minute Walk Test (6MWT) With Kinematic Evaluation of Gait (Short Term) — 293.05; 299.50 meters
◆ Published Evidence
No publication linked
No peer-reviewed publication reporting this trial's results has been linked yet. This can indicate results are unpublished — a known publication-bias signal. We re-check periodically.
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.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Six Minute Walk Test (6MWT) With Kinematic Evaluation of Gait (Short Term) |
293.05; 299.50 | — |
| PRIMARY Six Minute Walk Test (6MWT) With Kinematic Evaluation of Gait (Long Term) |
299.10; 295.20 | — |
| SECONDARY Hammersmith Functional Motor Scale, Expanded (HFMSE) (Short Term) |
50.84; 50.43 | — |
| SECONDARY Hammersmith Functional Motor Scale, Expanded (HFMSE) (Long Term) |
50.90; 50.30 | — |
| SECONDARY Manual Muscle Testing (MMT) Total Score (Short Term) |
181.86; 182.23 | — |
| SECONDARY Manual Muscle Testing (MMT) Total Score (Long Term) |
177.80; 181.10 | — |
| SECONDARY Motor Unit Number Estimation (MUNE) |
190.86; 194.26 | — |
Eligibility Criteria
Inclusion Criteria
- 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)
Exclusion Criteria
- Patients with a history of seizures
- Patients with any renal impairment
- Inability to comply with the study procedures
- Unstable medical illness
- Any ventilatory assistance
- Taking experimental medication for SMA other than under this protocol
- Pregnancy or lactation
- Menstruating women, not sterilized or not using effective birth control
- Planning to undergo scoliosis surgery within the next 10 months
- Inability to give informed consent
Data sourced from ClinicalTrials.gov (NCT01645787). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.