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A Randomized, Placebo-Controlled, Crossover Study to Evaluate the Safety and Efficacy of Amifampridine Phosphate in Ambulatory Patients With Spinal Muscular Atrophy (SMA) Type 3

Trial Profile

A Randomized, Placebo-Controlled, Crossover Study to Evaluate the Safety and Efficacy of Amifampridine Phosphate in Ambulatory Patients With Spinal Muscular Atrophy (SMA) Type 3

Not yet recruiting
Phase of Trial: Phase II

Latest Information Update: 27 Dec 2018

At a glance

  • Drugs Amifampridine (Primary)
  • Indications Spinal muscular atrophy
  • Focus Proof of concept; Therapeutic Use
  • Sponsors Catalyst Pharmaceuticals
  • Most Recent Events

    • 07 Aug 2018 According to a Catalyst Pharmaceutical media release, the company expects to enrol first patients in this trial in the second half of 2018.
    • 27 Nov 2017 New trial record
    • 21 Nov 2017 According to a Catalyst Pharmaceutical media release, if the results from this trial support the safety and efficacy of Firdapse as a treatment for ambulatory patients with SMA Type 3, the company intend to submit an application for orphan drug designation.
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