Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.

COMPLETED Phase 3

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

NCT02623699 · View on ClinicalTrials.gov ↗

Study 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.

Interventions

  • DRUG Placebo
  • DRUG Tofersen

Study Locations (20)

Florida

  • Mayo Clinic in Florida — Jacksonville
  • University of Miami School of Medicine — Miami
  • Bioclinica Research — Orlando

California

  • University of California San Diego Medical Center — La Jolla
  • California Pacific Medical Center — San Francisco

Arizona

  • Barrow Neurological Institute — Phoenix

Georgia

  • Emory University Hospital — Atlanta

Illinois

  • Northwestern University Feinberg School of Medicine — Chicago

Maryland

  • Johns Hopkins University — Baltimore

Massachusetts

  • Massachusetts General Hospital — Boston

Michigan

  • Henry Ford Hospital — Detroit

Trial Details

FieldValue
Enrollment Target 176 participants
Start Date 2016-01-20
Est. Completion 2021-07-16
Phase Phase 3

Sponsor

Biogen

74 total trials

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Full Details on ClinicalTrials.gov ↗

What the Registry Record Tells You About NCT02623699

The ClinicalTrials.gov registry entry for NCT02623699 describes a study currently listed as completed. It is categorized as Phase 3, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 176 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Biogen, which has 74 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.

The record links to 1 condition, with Amyotrophic Lateral Sclerosis appearing as the primary indexed condition, and to 2 interventions — of which Placebo is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.

Geographic footprint matters for practical reasons: NCT02623699 reports 20 study locations spanning 17 distinct geographic areas — top geographies include Florida, California, Arizona. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.

Frequently Asked Questions

What is clinical trial NCT02623699 about?

NCT02623699 is a clinical study titled "An Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of BIIB067 (Tofersen) in Adults With Inherited Amyotrophic Lateral Sclerosis (ALS)". 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 clinic...

What is the current status of trial NCT02623699?

This trial is currently completed. It is a Phase 3 study. The enrollment target is 176 participants. The study started on 2016-01-20. Estimated completion is 2021-07-16.

What conditions does trial NCT02623699 study?

This clinical trial studies the following conditions: Amyotrophic Lateral Sclerosis. These conditions were identified from the trial registry and reflect the primary focus areas of the research.

What interventions are being tested in trial NCT02623699?

The interventions under investigation include: Placebo (DRUG), Tofersen (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.

Who is sponsoring clinical trial NCT02623699?

This trial is sponsored by Biogen, which has 74 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.

Where is trial NCT02623699 being conducted?

This trial has 20 study locations across Arizona, California, Florida, Georgia, Illinois. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.

Related

Data sourced from official U.S. government datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial