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
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
- 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.