A Phase 1 Multiple-Ascending-Dose Study to Assess the Safety Tolerability and Pharmacokinetics of BIIB105 Administered Intrathecally to Adults With Amyotrophic Lateral Sclerosis With or Without Poly-CAG Expansion in the Ataxin-2 Gene

This is a first in human, phase 1 trial of BIIB105, which is a novel ASO, in patients with Amyotrophic lateral sclerosis (ALS) and ALS associated with ataxin-2 (ATXN2) poly-CAG expansion (polyQ-ALS). BIIB105 will be administered via fluoro-guided LP up to 5 times over approximately 3 months. Subjects will undergo blood draws, physical examination, pulmonary and strength testing and will complete questionnaires. PolyQ-ALS (Cohort D2) must complete a Natural History Run-in Period prior to dosing.

You may be eligible for this study if you meet the following criteria:

• Conditions:
  Medical Research
• Age: - 99 Years
• Gender: All