CompletedPhase 3

An Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of BIIB067 (Tofersen) in Adults With Inherited Amyotrophic Lateral Sclerosis (ALS)

United States176 participantsStarted 2016-01-20

Plain-language summary

The primary objectives of Parts A and B of this study are to evaluate the safety, tolerability, and pharmacokinetics (PK) of ascending doses of tofersen in adults with ALS and a documented superoxide dismutase 1 (SOD1) mutation. The primary objective of Part C of this study is to evaluate the clinical efficacy of tofersen administered to adults with ALS and a confirmed SOD1 mutation. The secondary objective of Parts A and B of this study is to evaluate the effects of tofersen on levels of total SOD1 protein in the cerebrospinal fluid (CSF). The secondary objectives of Part C are to evaluate the safety, tolerability, pharmacodynamic (PD), and biomarker effects of tofersen.

Who can participate

Age range18 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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 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, biological agent, or device within 1 month or 5 half-lives of study agent, whichever is longer. Specifically, no prior treatment …

What they're measuring

Parts A and B: Number of Participants Experiencing Adverse Events (AEs) and Serious Adverse Events (SAEs)

Timeframe: Part A: First dose up to Day 63; Part B: First dose up to Day 289

Parts A and B: Number of Participants With Clinically Significant Laboratory Abnormalities

Timeframe: Part A: Up to Day 57; Part B: Up to Day 169

Parts A and B: Number of Participants With Clinically Significant Vital Sign Abnormalities

Timeframe: Part A: Up to Day 57; Part B: Up to Day 169

Parts A and B: Number of Participants With Clinically Significant Physical Examination Abnormalities

Timeframe: Part A: Up to Day 57; Part B: Up to Day 169

Parts A and B: Number of Participants With Clinically Significant Neurological Examination Abnormalities

Timeframe: Part A: Up to Day 57; Part B: Up to Day 169

Parts A and B: Number of Participants With Clinically Significant 12-lead Electrocardiograms (ECGs) Abnormalities

Timeframe: Part A: Up to Day 57; Part B: Up to Day 169

Parts A and B: PK Parameter of BIIB067 in Plasma: Maximum Observed Concentration (Cmax)

Trial details

NCT IDNCT02623699

SponsorBiogen

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2021-07-16

Results submitted2023-05-17

View on ClinicalTrials.gov More Amyotrophic Lateral Sclerosis trials

Plain-language summary

Who can participate

Age range18 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

Parts A and B: Number of Participants Experiencing Adverse Events (AEs) and Serious Adverse Events (SAEs)

Timeframe: Part A: First dose up to Day 63; Part B: First dose up to Day 289

Parts A and B: Number of Participants With Clinically Significant Laboratory Abnormalities

Timeframe: Part A: Up to Day 57; Part B: Up to Day 169

Parts A and B: Number of Participants With Clinically Significant Vital Sign Abnormalities

Timeframe: Part A: Up to Day 57; Part B: Up to Day 169

Parts A and B: Number of Participants With Clinically Significant Physical Examination Abnormalities

Timeframe: Part A: Up to Day 57; Part B: Up to Day 169

Parts A and B: Number of Participants With Clinically Significant Neurological Examination Abnormalities

Timeframe: Part A: Up to Day 57; Part B: Up to Day 169

Parts A and B: Number of Participants With Clinically Significant 12-lead Electrocardiograms (ECGs) Abnormalities

Timeframe: Part A: Up to Day 57; Part B: Up to Day 169

Parts A and B: PK Parameter of BIIB067 in Plasma: Maximum Observed Concentration (Cmax)