Phase II/III Randomized, Placebo-controlled Trial of Arimoclomol in SOD1 Positive Familial Amyotrophic Lateral Sclerosis
Study Purpose:
The purpose of this study will be to demonstrate the safety, tolerability, and efficacy of arimoclomol in subjects with SOD1 positive familial Amyotrophic Lateral Sclerosis (ALS).
Disease:
Amyotrophic Lateral Sclerosis (ALS), Familial ALSStudy Type:
Interventional TrialStudy Category:
Drug TrialStudy Status:
Not enrollingPhase:
Phase IIStudy Chair(s)/Principal Investigator(s):
Michael Benatar MBChB, DPhil, (University of Miami)
Merit Cudkowicz MD, MSc, (Massachusetts General Hospital)
Clinicaltrials.gov ID (11 digit #):
NCT00706147Neals Affiliated?
YesCoordinating Center Contact Information
University of Miami.(JavaScript must be enabled to view this email address) / 305-243-0108
.(JavaScript must be enabled to view this email address) 1120 NW 14th Street
Miami, Florida 33136 United States
Full Study Summary:
Using a seamless, adaptive, phase II/III design, the investigators will determine the safety and efficacy of arimoclomol in patients with SOD1 positive familial ALS. Both stage-1 and stage-2 are randomized, double-blind and placebo-controlled in a population of patients with rapidly progressive SOD1 positive familial ALS. Patients with ALS, a history of a relative affected with ALS (i.e. familial ALS) and the presence of a demonstrable mutation in the SOD1 gene that is known to be associated with rapidly progressive disease, will be eligible for inclusion in this study. Potentially eligible subjects will undergo screening via telephone and, if necessary, review of outside medical records. Subjects who meet all eligibility criteria will travel a study site for final eligibility determination, baseline evaluation and will then be randomized 1:1 to receive either placebo or arimoclomol at a dose of 200 mg t.i.d. Participants will then be evaluated again in person at a study site at Month-2. Subsequent telephonic evaluations at Month-3, -4, -5, -6, -8, and -10 will be performed in participants' homes. Safety and tolerability evaluations will be performed at each of these visits. Collection of blood samples for safety laboratory analyses and measurement of blood pressure, heart rate, respiratory rate, temperature and weight will be performed at Months -1, -3, -5, -6, -8, and -10 in the participant's home by a representative of a medical monitoring company. A study coordinator may perform an in-person visit at Month-12, or this visit may occur telephonically. A final evaluation will be performed via telephone at Month -13 (30 days after the last dose of study medication).
Study Sponsor:
FDA, ALSAParticipant Duration:
12 months