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
- 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.