A Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of BIIB067 Administered to Adult Subjects with Amyotrophic Lateral Sclerosis and Confirmed Superoxide Dismutase 1 Mutation - VALOR (Part C)

Biogen Idec Research Limited0 sites183 target enrollmentJuly 10, 2020

ConditionsAmyotrophic Lateral Sclerosis (ALS)MedDRA version: 20.0Level: PTClassification code 10077024Term: Familial amyotrophic lateral sclerosisSystem Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 21.1Level: PTClassification code 10002026Term: Amyotrophic lateral sclerosisSystem Organ Class: 10029205 - Nervous system disorders Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Overview

Phase: Phase 1
Intervention: Not specified
Conditions: Amyotrophic Lateral Sclerosis (ALS)
Sponsor: Biogen Idec Research Limited
Enrollment: 183
Status: Active, not recruiting
Last Updated: 4 years ago

Overview Investigators Eligibility Criteria Outcomes Similar Trials

Overview

Brief Summary

No summary available.

Registry

who.int

Start Date

July 10, 2020

End Date

TBD

Last Updated

4 years ago

Study Type

Interventional clinical trial of medicinal product

Sex

All

Investigators

Sponsor

Biogen Idec Research Limited

Eligibility Criteria

Inclusion Criteria

•Key Inclusion Criteria: Part A and B
•\- Weakness attributable to ALS and documented SOD1 mutation at Screening Visit 2\.
•\- A forced vital capacity (FVC) \=50% of predicted value as adjusted for sex, age, and height (from the sitting position). Participants with stable FVC \<50% but \=45%, whose FVC has not declined by more than 5% in the last 6 months may be considered for inclusion, at the discretion of the Investigator.
•\- If taking riluzole, participant must be on a stable dose for \=30 days prior to Day 1 and expected to remain at that dose until the final study visit.
•\- Medically able to undergo the study procedures, and to adhere to the visit schedule at the time of study entry, as determined by the Investigator.
•Key Inclusion Criteria: Part C
•\- Weakness attributable to ALS and confirmed SOD1 mutation at Screening Visit.
•\- If taking riluzole, participant must be on a stable dose for \=30 days prior to Day 1 and expected to remain at that dose until the final study visit.
•\- If taking edaravone, participant must have initiated edaravone \=60 days (2 treatment cycles) prior to Day 1 and expected to remain at that dose until the final study visit, unless the Investigator determines that edaravone should be discontinued for medical reasons, in which case it may not be restarted during the study. Edaravone may not be administered on dosing days of this
•\- Medically able to undergo the study procedures and to adhere to the visit schedule at the time of study entry, as determined by the Investigator.

Exclusion Criteria

•Key Exclusion Criteria: Part A and B
•\- History of, or positive test result for human immunodeficiency virus.
•\- History of, or positive test result at Screening, for hepatitis C virus antibody.
•\- Current hepatitis B infection (defined as positive for hepatitis B surface antigen \[HBsAg] and/or hepatitis B core antibody \[HBcAb]). Participants with immunity to hepatitis B from previous natural infection (defined as negative HBsAg, positive hepatitis B surface antibody immunoglobulin G, and positive HBcAb) or vaccination (defined as positive anti\-HBs) are eligible to participate in the study.
•\- Treatment with another investigational drug (including investigational drugs for ALS through compassionate use programs), biological agent, or device within 1 month or 5 half\-lives of study agent, whichever is longer. Specifically, no prior treatment with small interfering ribonucleic acid, stem cell therapy, or gene therapy is allowed.
•\- Current enrollment in any other interventional study.
•\- Current or recent (within 1 month) use, or anticipated need, in the opinion of the Investigator, of copper (II) (diacetyl\-bis (N4\-methylthiosemicarbazone)) or pyrimethamine.
•\- Current or anticipated need, in the opinion of the Investigator, of a diaphragm pacing system (DPS) during the study period.
•Key Exclusion Criteria: Part C
•\- History of or positive test result for human immunodeficiency virus.