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).


Amyotrophic Lateral Sclerosis (ALS),  Familial ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Not enrolling


Phase II

Study Chair(s)/Principal Investigator(s):

Michael Benatar MBChB, DPhil, (University of Miami)
Merit Cudkowicz MD, MSc, (Massachusetts General Hospital) ID (11 digit #):


Neals Affiliated?


Coordinating 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:


Participant Duration:

12 months

Estimated Enrollment:


Estimated Study Start Date:


Estimated Study Completion Date:


Posting Last Modified Date:


Date Study Added to

  • More Information

    Estimated Study Completion Date: December 2016


  • Eligibility Criteria


    Female, Male

    Minimum Age:


    Maximum Age:


    Time since Symptom Onset:

    Time since Diagnosis:

    <12 months

    Can participants use Riluzole?


    If taking Riluzole, must be on a stable dose for at least thirty days prior to the baseline visit.

    • Type of ALS that is hereditary (runs in families) only.
    • El Escorial criteria for familial ALS and a family history of a pathogenic mutation in the SOD1 gene
    • Willingness to undergo genetic testing and to learn the results.
    • Demonstrable mutation in the SOD1 gene that is reported to be associated with a rapid rate of disease progression (i.e. A4V, A4T, C6F, C6G, V7E, L8Q, G10V, G41S, H43R, H48Q, D90V, G93A, D101H, D101Y, L106V, I112M, I112T, R115G, L126X, G127X, A145T, V148G, V148I) or possibly associated with rapidly progressive disease (E21G, G37R, L38V, D76Y, L84F, L84V, N86S, D90A het, G93R, I104F, I113T, L144F, L144S).
    • Capable of providing informed consent and complying with trial procedures.
    • Diagnosis within less than 9 months of the anticipated date of the baseline visit AND study participants' subjective evaluation that they expect their physical condition to permit travel to the study site for both the baseline and 2-month study visits.
    • Women must not be able to become pregnant (e.g. post menopausal for at least one year, surgically sterile, or practicing adequate birth control methods) for the duration of the study. Adequate contraception includes: oral contraception, implanted contraception, intrauterine device in place for at least 3 months, or barrier method in conjunction with spermicide.
    • Women of childbearing potential must have a negative pregnancy test at screening visit and be non-lactating.
    • Identifiable local medical doctor to assist with urgent care of any medical complications that may arise.

  • Site Contact Information

    University of Miami
    Michael Benatar, MD / .(JavaScript must be enabled to view this email address) / 305-243-6480
    1120 NW 14th Street
    Miami, Florida 33136
    United States

    Massachusetts General Hospital
    Julia Yasek / .(JavaScript must be enabled to view this email address) / 617-643-7428
    149 13th Street
    Charlestown, Massachusetts 02129
    United States