| City: |
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Boston |
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State:
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MA |
| Zip Code: |
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02114 |
| Conditions: |
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Amyotrophic Lateral Sclerosis 1 - Acquired Amyotrophic Lateral Sclerosis - Amyotrophic Lateral Sclerosis |
| Purpose: |
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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 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.
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| Study summary: |
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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).
Participants who complete ths study will be eligible to receive open-label Arimoclomol. |
| Criteria: |
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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.
- Not taking riluzole, or on a stable dose for at least thirty days prior to the
baseline visit.
- 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. |
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| Study is available at: |
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Massachusetts General Hospital
Boston, MA 02114
United States
Primary Contact:
Nazem Atassi, MD, MSc
Email: natassi@partners.org
Phone: 617-643-0842
Secondary Contact:
Margaret Walker, PhD
Email: mwalker@med.miami.edu
Phone: 305-951-9326 |
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If you are interested in this clinical trial please use the contact information above. If you would like to get additional information about this clinical trial please visit ClinicalTrials.gov.
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| Data Source: |
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ClinicalTrials.gov |
| Date Processed: |
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March 22, 2011 |
Modifications to
this listing: |
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above to view all information about this clinical trial. |
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