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A Phase 1, Double-Blind, Placebo-Controlled, Dose-Escalation Study of the Safety, Tolerability, & Pharmacokinetics of ISIS333611 Administered Intrathecally to Patients w Familial Amyotrophic Lateral Sclerosis Due to Superoxide Dismutase 1 Gene Mutation |
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Study Focus:
This study will test the safety, tolerability, and pharmacokinetics of single doses of ISIS 333611 administered as 12-hour intrathecal infusions. |
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Disease:
Amyotrophic Lateral Sclerosis (ALS), Familial ALS |
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Study Category:
Drug Trial |
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Study Status:
Closed |
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Study Results:
A total of 32 doses were given to 21 individuals with SOD1 ALS (7 subjects were dosed 2 times and 2 subjects were dosed 3 times). Results indicate that ISIS 333611 was well tolerated. No safety or tolerability concerns related to ISIS 333611 were identified. Drug levels in the spinal fluid and blood corresponded well with the predicted levels based on preclinical testing. |
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Phase:
Phase I |
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Type:
Interventional Trial
with active agents/drugs & a placebo |
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Funding Source:
Isis Pharmaceuticals, Inc. |
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Study Chair(s)/Principal Investigator(s):
Merit Cudkowicz MD, MSc, (Massachusetts General Hospital)
Timothy Miller MD, PhD, (Washington University) |
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Clinicaltrials.gov ID:
NCT01041222 |
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NEALS Affiliated?
Yes
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Study Summary:
This study will test the safety, tolerability, and pharmacokinetics of single doses of ISIS 333611 administered as 12-hour intrathecal infusions. Four dose levels (0.15, 0.5, 1.5 and 3 mg) will be evaluated sequentially. The volume of the infusion is 0.25 mL/12 hours. Each dose level will be studied in a cohort of 8 patients where 6 are randomized to active treatment with ISIS 333611 and 2 are randomized to placebo. |
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Participant Duration:
Total enrollment time for this study is about 8 weeks. The screening visit will last approximately 2-4 hours. During the treatment phase of the study, you will be admitted to the hospital for the temporary intrathecal infusion procedure for about two days. After the treatment phase, there is a 4 week follow up period with two in-clinic visits (each visit is about 2-3 hours) and one telephone contact/visit (about 30 minutes). |
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# of Subjects:
4 cohorts of 8 patients each, total 32 patients |
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Enrollment Start Date:
01/01/2010 |
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Enrollment End Date:
12/01/2011 |
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Posting Last Modified Date:
12/01/2011 |
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Date Study Added to alscortium.org:
11/02/2011 |
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| Eligibility Criteria |
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Gender:
Male & Female |
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Minimum Age:
18 |
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Min Vital Capacity (% predicted normal):
50% |
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Time since Symptom Onset:
Not applicable |
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Time since Diagnosis:
Not applicable |
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Can participants use Riluzole?
Yes |
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Coordinating Center
Contact Information |
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| Katy Mahoney |
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| kmahoney17@partners.org |
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| 617-643-7434 |
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| Massachusetts General Hospital |
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149 13th Street
Charlestown, Massachusetts 02129
UNITED STATES |
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Other Eligibility Criteria:
If taking riluzole, patients must be on stable dosage for at least 30 days prior to starting the study and expect to remain at that dosage until the end of the study. |
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Site Contact Information (Please click to show/hide) |
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| California |
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Center for Neurologic Study
Lucie Barbie
lbarbie@sandiegotrials.com
858-455-5463
La Jolla, California 92037
UNITED STATES |
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| Maryland |
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Johns Hopkins University
Richard Kimball
rkimbal1@jhmi.edu
410-955-8511
Baltimore, Maryland 21287
UNITED STATES |
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| Massachusetts |
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Massachusetts General Hospital
Melanie Majkut
mmajkut@partners.org
617-643-5581
Charlestown, Massachusetts 02129
UNITED STATES |
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| Missouri |
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Washington University School of Medicine
Charlie Wulf
wulfc@neuro.wustl.edu
314-362-6980
St Louis , Missouri 63110
UNITED STATES |
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| Texas |
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Methodist Hospital Research Institute
Sharon L. Halton
SLHalton@tmhs.org
713-441-3420
Houston, Texas 77030
UNITED STATES |
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Familial ALS- ALS cases are inherited from 2 or more family members, affects 5-10% of ALS cases. Sporadic ALS- disease occurs at random with no clearly associated risk factors; most common form of ALS, affects approximately 90-95% ALS cases. Interventional trial- study where exposure, for example a drug, is assigned; used to determine the effectiveness of a treatment or intervention. With drugs and a placebo- One group gets the active treatment, the other gets the placebo. Everything else is held the same between the two groups, so that any difference in their outcome can be attributed to the active treatment. Observational study- study in which patients are observed and where no treatment is given; researcher has no control over the experiment because they are done in a more natural setting than an interventional trial. Trial Phase- objectives are different for each phase of a trial. - Phase I, drug is given to a small number of people to see if it is safe.
- Phase II begins to look at effectiveness of the drug.
- Phase III is considered the definitive test of whether a drug is effective, held at multiple research sites and much larger than the previous phases.
Vital Capacity- the maximum amount of air that can be expelled from the lungs after a maximum drawing of air into the lungs, measured by a spirometer and then scored as a percentage. |
