Help us determine the safety and tolerability of an ALS gene therapy
Closed for enrollment
Study Name
Biogen-C9 Phase I Clinical Trial
PI
Michael Benatar, MD, PhD
Study Type
Interventional (Clinical Trial), Phase I, Randomized, Placebo-controlled
Eligible Population
Individuals affected with ALS, and positive for the C9ORF72 gene mutation
Study Purpose/Goals
To investigate the safety and tolerability of the experimental drug BIIB078 in patients with C9orf72-ALS
A Phase I Multiple-Ascending-Dose Study to Assess the Safety, Tolerability, and Pharmacokinetics of BIIB078 Administered Intrathecally to Adults with C9ORF72-Associated Amyotrophic Lateral Sclerosis.
Procedures/Participation
40 weeks of participation:
- Screening: 6 weeks
- Treatment: 12 weeks (3 doses, once every 2 weeks + 2 doses, once every 4 weeks)
- Follow-up: 22 weeks
Procedures:
- Medical history and medication review
- Drug dosing
- Blood and urine collection
- Neurological exam
- Muscle testing
- Electrocardiogram
- Physical Exam
- Neuropsychiatric testing
- Surveys
- Breathing testing
Contact
Join our family - together, we can change the course of this disease.
Join our family
Together, we can change the course of this disease.
ALS Treatment
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