NCT03626012

A Study to Assess the Safety, Tolerability, and Pharmacokinetics of BIIB078 in Adults With C9ORF72-Associated Amyotrophic Lateral Sclerosis

A Phase 1 Multiple-Ascending-Dose Study to Assess the Safety, Tolerability, and Pharmacokinetics of BIIB078 Administered Intrathecally to Adults With C9ORF72-Associated Amyotrophic Lateral Sclerosis

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 106 (actual)
• Sponsor: Biogen · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The primary objective of this study is to evaluate the safety and tolerability of BIIB078 in adults with C9ORF72-Amyotrophic Lateral Sclerosis (ALS). The secondary objectives of this study are to evaluate the pharmacokinetic profile of BIIB078 and to evaluate the effects of BIIB078 on clinical function. As the first-in-human study, the study enrolls a small number of participants in each cohort. Every participant in a cohort is treated with the same dose or placebo. The study is designed to evaluate and confirm the safety of each dose before enrolling and exposing new participants to a higher dose in the next cohort.

Conditions

• Amyotrophic Lateral Sclerosis

Interventions

Type	Name	Description
DRUG	BIIB078	Administered as specified in the treatment arm.
DRUG	Placebo	Administered as specified in the treatment arm.

Timeline

• Start date: 2018-09-10
• Primary completion: 2021-11-17
• Completion: 2021-11-17
• First posted: 2018-08-10
• Last updated: 2022-01-14

Locations

21 sites across 6 countries: United States, Canada, Ireland, Netherlands, Switzerland, United Kingdom

Regulatory

• FDA-regulated drug study