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

• Status: Completed
• Trial end date: 2015-09-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 2/Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Columbia University
• Treatments: 4-Aminopyridine

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