Biogen 245AS101
Research type
Research Study
Full title
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.
IRAS ID
247000
Contact name
Pamela Shaw
Contact email
Sponsor organisation
Biogen Idec Research Limited
Eudract number
2017-000294-36
Clinicaltrials.gov Identifier
N/A, N/A
Duration of Study in the UK
1 years, 11 months, 4 days
Research summary
Research Summary:
This is a Phase 1, double blind, multi-centre clinical research study. This means that it is the first time the study drug, BIIB078, will be tested in humans. The main purpose of this study is to find out about the safety, tolerability, and pharmacokinetics (how the study drug is processed by the body) of the study drug when given as repeated doses in adult patients with C9ORF72-associated amyotrophic lateral sclerosis (ALS), a progressive neurodegenerative disease affecting nerve cells in the brain and spinal cord.
Patients who have been diagnosed with ALS, have the C9ORF72 gene mutation, and are currently showing signs of weakness due to ALS can be invited to be part of this study.
The study drug will be administered “intrathecally”, meaning that the study drug is given to participants by a procedure called a lumbar puncture (LP), using a thin needle that is inserted through the lower back into the fluid-filled space below the end of the spinal cord.
To find out how well the study drug works, it will be compared to placebo. A placebo looks like the study drug but does not contain any active ingredients.
Participants will be assigned to 1 of 4 groups.
• Cohort 1: 5 milligrams (mg) study drug or placebo
• Cohort 2: 20 mg study drug or placebo
• Cohort 3: 60 mg study drug or placebo
• Cohort 4: 120 mg study drug or placebo (this dose level will not be evaluated in the UK)
Participants within each group will be randomised in a 3:1 (active: placebo) ratio overall to receive BIIB078 or placebo.
Approximately 59 participants in about 20 study centres worldwide will take part to ensure approximately 44 participants complete the treatment period. The study will last approximately 40 weeks and participants will visit the study centre up to 13 times.Summary of results:
The results were submitted to EudraCT, however, as this is a Phase 1 study solely conducted in adults, the results are not made public on EudraCT.https://www.biogentriallink.com/NCT03626012
REC name
Yorkshire & The Humber - Leeds East Research Ethics Committee
REC reference
18/YH/0417
Date of REC Opinion
14 Apr 2019
REC opinion
Further Information Favourable Opinion