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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

Study Focus:
This study will test the safety, tolerability, and pharmacokinetics of single doses of ISIS 333611 administered as 12-hour intrathecal infusions.
Amyotrophic Lateral Sclerosis (ALS), Familial ALS
Study Category:
Drug Trial
Study Status:
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. Read more here: http://www.ncbi.n...icles/PMC3712285/
Phase I
Interventional Trial
with active agents/drugs & a placebo
Funding Source:
Isis Pharmaceuticals, Inc.
Study Chair(s)/Principal Investigator(s):
Merit Cudkowicz MD, MSc, (Massachusetts General Hospital)
Timothy Miller MD, PhD, (Washington University) ID:
NEALS Affiliated?
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.
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).
# of Subjects:
4 cohorts of 8 patients each, total 32 patients
Enrollment Start Date:
Enrollment End Date:
Posting Last Modified Date:
Date Study Added to
Eligibility Criteria
Male & Female
Minimum Age:
Min Vital Capacity (% predicted normal):
Time since Symptom Onset:
Not applicable
Time since Diagnosis:
Not applicable
Can participants use Riluzole?
Coordinating Center
Contact Information
Katy Mahoney
Massachusetts General Hospital
149 13th Street
Charlestown, Massachusetts 02129
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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