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

Study Identifier:
AAAI7400
ClinicalTrials.gov Identifier:
NCT01645787
EudraCT Identifier:
N/A
EU CT Number:
N/A
Sponsor:
Columbia University
Study Complete

Study Details

Medical Condition
  • All Spinal Muscular Atrophies
Study Drug
  • Drug: 4-aminopyridine
  • Drug: Placebo
Date
Jun 2012 - Sep 2015
Patient Requirements
Sex: Female & Male
Age: 18 - 50 Years
Requirements Information
Inclusion and Exclusion 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

Protocol 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.

Trial Locations

Location
Status
Location
Columbia University Medical Center
New York, New York, United States, 10032
Status
N/A

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