A randomized, double-blind, 2-period, 2-treatment, crossover, proof-of-concept study of amifampridine (Firdapse®) in ambulatory patients diagnosed with Spinal Muscular Atrophy (SMA) Type 3

Trial Profile

A randomized, double-blind, 2-period, 2-treatment, crossover, proof-of-concept study of amifampridine (Firdapse®) in ambulatory patients diagnosed with Spinal Muscular Atrophy (SMA) Type 3

Recruiting
Phase of Trial: Phase II

Latest Information Update: 27 Nov 2017

At a glance

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

    • 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.
    • 21 Nov 2017 According to a Catalyst Pharmaceutical media release, the study will be conducted by a team of researchers led by Lorenzo Maggi, MD, and Giovanni Baranello, MD, of the Fondazione Istituto Neurologico Carlo Besta in Milan, Italy and the company anticipates reporting top-line results from the study in the first half of 2019.
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