CompletedPhase 3

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

United States, Australia, Belgium176 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 range

18 Years

Sex

ALL

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 …

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

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

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 range

18 Years

Sex

ALL

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