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Sponsors and Collaborators: |
Emory University ALS Association Food and Drug Administration (FDA) Massachusetts General Hospital |
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Information provided by: | Emory University |
ClinicalTrials.gov Identifier: | NCT00706147 |
The purpose of this study will be to demonstrate the safety, tolerability, and efficacy of arimoclomol in subjects with SOD1 positive familial ALS.
Study hypotheses: Arimoclomol, taken at a dose of 100 mg three times daily will reduce by at least 30% the rate of progression of disease. In addition, it will be safe and well tolerated in subjects with SOD1 positive familial ALS.
Condition | Intervention | Phase |
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Familial Form of Amyotrophic Lateral Sclerosis (fALS) SOD1 Positive ALS |
Drug: Arimoclomol |
Phase II Phase III |
Study Type: | Interventional |
Study Design: | Treatment, Randomized, Double Blind (Subject, Caregiver, Investigator), Placebo Control, Parallel Assignment, Safety/Efficacy Study |
Official Title: | Phase II/III Randomized, Placebo-Controlled Trial of Arimoclomol in SOD1 Positive Familial Amyotrophic Lateral Sclerosis (ALS) |
Estimated Enrollment: | 80 |
Study Start Date: | January 2009 |
Estimated Study Completion Date: | December 2012 |
Estimated Primary Completion Date: | December 2012 (Final data collection date for primary outcome measure) |
Arms | Assigned Interventions |
---|---|
1: Placebo Comparator |
Drug: Arimoclomol
Drug: Placebo capsule given three times per day Drug: Arimoclomol capsule given three times per day |
2: Active Comparator |
Drug: Arimoclomol
Drug: Placebo capsule given three times per day Drug: Arimoclomol capsule given three times per day |
Using a seamless, adaptive, phase II/III design, we 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 to Emory or MGH for final eligibility determination, baseline evaluation and will then be randomized 1:1 to receive either placebo or arimoclomol at a dose of 100mg t.i.d. Participants will then be evaluated again in person at Emory or MGH 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. An Emory study coordinator will perform an in-person visit at Month-12. A final evaluation will be performed via telephone at Month -13 (30 days after the last dose of study medication).
Ages Eligible for Study: | 18 Years and older |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Presence of any of the following clinical conditions:
Screening laboratory values:
Contact: Cathy Raiser, MSN, APRN-BC | (404) 712-8578 | craiser@emory.edu |
United States, Georgia | |
Emory University | Recruiting |
Atlanta, Georgia, United States, 30033 | |
Contact: Cathy Raiser, MSN, APRN-BC craiser@emory.edu | |
Principal Investigator: Michael Benatar, MBChB, DPhil |
Principal Investigator: | Michael Benatar, MBChB, DPhil | Emory University |
Principal Investigator: | Merit Cudkowicz, MD, MSc | Massachusetts General Hospital |
Responsible Party: | Emory University School of Medicine ( Michael Benatar MBChB, DPhil ) |
Study ID Numbers: | Arimoclomol in SOD1 fALS |
Study First Received: | June 24, 2008 |
Last Updated: | January 7, 2009 |
ClinicalTrials.gov Identifier: | NCT00706147 |
Health Authority: | United States: Food and Drug Administration |
Lou Gehrig's Disease Motor Neuron Disease Amyotrophic Lateral Sclerosis (ALS) Familial ALS |
Neuromuscular disease SOD1 mutation Superoxide dismutase |
Amyotrophic lateral sclerosis Spinal Cord Diseases Neuromuscular Diseases Amyotrophic Lateral Sclerosis Central Nervous System Diseases Lou Gehrig's disease |
Sclerosis Degenerative motor system disease Neurodegenerative Diseases Motor neuron disease Motor Neuron Disease |
Pathologic Processes Nervous System Diseases |