Research Studies
Study Title
Biogen SOD1 ASO “VALOR” Trial (An Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of BIIB067 in Adults with Inherited ALS)
Enrollment Status
Completed
Principal Investigators
Site-PI: Michael Benatar, MD, PhD
Study Type
Drug Trial
Goals
- Determine the safety and efficacy of the experimental antisense oligonucleotide (ASO) BIIB067 in patients with SOD1-ALS
Enrolling
SOD1 ALS patients
Eligibility
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Individuals affected with ALS, and positive for an SOD1 gene mutation that is associated with rapidly progressive disease
Study Involvement
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Multiple study visits with spinal tap and intrathecal administration of experimental agent. “Loading” comprises 3 administrations, with each dose separated by 2 weeks. “Maintenance” comprises 5 doses, administered every 4 weeks.
Study procedures include:
Study procedures include:
- Medical history and medication review
- Drug dosing
- Blood, urine and CSF collection
- Neurological exam
- Muscle testing
- Electrocardiogram
- Physical Exam
- Surveys
- Breathing testing
- Biomarker procedures (EIM, HHD, MUNIX)
Monthly intrathecal administration (following a blinded induction phase) of BIIB067 during the open label extension phase.
Contact
Primary Funding Source
Biogen
Related Publications
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Related Presentations & Lectures
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