- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00706147
Phase II/III Randomized, Placebo-controlled Trial of Arimoclomol in SOD1 Positive Familial Amyotrophic Lateral Sclerosis
Phase II/III Randomized, Placebo-Controlled Trial of Arimoclomol in SOD1 Positive Familial Amyotrophic Lateral Sclerosis (ALS)
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). This type of ALS is HEREDITARY (runs in families), and at least one other person in the family must have had ALS.
Study hypotheses: Arimoclomol, taken at a dose of 200 mg three times daily will improve survival as defined by time to death, tracheostomy or permanent assisted ventilation. In addition, it will be safe and well tolerated in subjects with SOD1 positive familial ALS.
Funding Source - FDA-OOPD
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
Study Type
Enrollment (Actual)
Phase
- Phase 2
- Phase 3
Contacts and Locations
Study Locations
-
-
Florida
-
Miami, Florida, United States, 33136
- University of Miami Miller School of Medicine
-
-
Massachusetts
-
Boston, Massachusetts, United States, 02114
- Massachusetts General Hospital
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
- 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 a gene known to be associated with ALS, such as 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).
- Age 18 years or older; male or female.
- 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.
- Willing to remain on a stable dose of Riluzole or to remain off Riluzole for the duration of the trial.
- Identifiable local medical doctor to assist with urgent care of any medical complications that may arise.
- Absence of any of the exclusion criteria.
Exclusion Criteria:
- History of known sensitivity or intolerability to Arimoclomol or to any other related compound.
- Exposure to any investigational drug within 30 days of the screening visit.
Presence of any of the following clinical conditions:
- Substance abuse within the past year.
- Unstable cardiac, pulmonary, renal, hepatic, endocrine, hematologic, or active infectious disease.
- AIDS or AIDS-related complex.
- Unstable psychiatric illness defined as psychosis (hallucinations or delusions), untreated major depression within 90 days of the screening visit.
- Positive pregnancy test at screening visit.
Screening laboratory values:
- Creatinine greater than 1.5.
- Alanine aminotransferase (ALT) or Aspartate aminotransferase (AST). greater than 3.0 times the upper limit of normal.
- Total bilirubin greater than 2.0 times the upper limit of normal.
- White blood cell (WBC) count less than 3,500/mm3.
- Platelet concentration less than 100,000/ul.
- Hematocrit level less than 33 for female or less than 35 for male.
- Female patients who are breast-feeding.
Study Plan
How is the study designed?
Design Details
- Primary Purpose: TREATMENT
- Allocation: RANDOMIZED
- Interventional Model: PARALLEL
- Masking: TRIPLE
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
ACTIVE_COMPARATOR: 2
|
Drug: Arimoclomol capsules given three times per day
Other Names:
|
PLACEBO_COMPARATOR: 1
|
Drug: Placebo capsules given three times per day
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Time to death, tracheostomy or permanent assisted ventilation will be the primary outcome measure.
Time Frame: 12 months
|
12 months
|
Secondary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Rate of decline of ALSFRS-R (ALS functional rating scale-revised) over a period of up to 12 months.
Time Frame: 12 months
|
12 months
|
Disease progression as measured by the rate of decline of FEV6.
Time Frame: 12 months
|
12 months
|
Safety and tolerability of arimoclomol will be evaluated by using vital signs and weight, clinical laboratory measures, physical examination, report of adverse events, and the proportion of subjects completing the study on assigned treatment.
Time Frame: 12 months
|
12 months
|
Collaborators and Investigators
Sponsor
Collaborators
Publications and helpful links
Study record dates
Study Major Dates
Study Start
Primary Completion (ACTUAL)
Study Completion (ACTUAL)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (ESTIMATE)
Study Record Updates
Last Update Posted (ACTUAL)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- 20100758
- 3517 (OTHER_GRANT: OOPD)
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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