An Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of BIIB067 (Tofersen) in Adults With Inherited Amyotrophic Lateral Sclerosis (ALS)
Public ClinicalTrials.gov record NCT02623699. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
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
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT02623699
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 176 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 18 Years and older
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Jan 20, 2016 (Actual)
- Primary completion
- Jul 16, 2021 (Actual)
- Completion
- Jul 16, 2021 (Actual)
- Last update posted
- Jul 28, 2023
2016 – 2021
United States locations
- U.S. sites
- 20
- U.S. states
- 17
- U.S. cities
- 20
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Barrow Neurological Institute | Phoenix | Arizona | 85013 | — |
| University of California San Diego Medical Center | La Jolla | California | 92093 | — |
| California Pacific Medical Center | San Francisco | California | 94115 | — |
| Mayo Clinic in Florida | Jacksonville | Florida | 32224 | — |
| University of Miami School of Medicine | Miami | Florida | 33136 | — |
| Bioclinica Research | Orlando | Florida | 32806 | — |
| Emory University Hospital | Atlanta | Georgia | 30322 | — |
| Northwestern University Feinberg School of Medicine | Chicago | Illinois | 60611 | — |
| Johns Hopkins University | Baltimore | Maryland | 21287 | — |
| Massachusetts General Hospital | Boston | Massachusetts | 02114 | — |
| Henry Ford Hospital | Detroit | Michigan | 48202 | — |
| Mayo Clinic - Rochester | Rochester | Minnesota | 55905 | — |
| Washington University School of Medicine | St Louis | Missouri | 63110 | — |
| Neurology Associates, P.C. | Lincoln | Nebraska | 68506 | — |
| Columbia University Medical Center | New York | New York | 10032 | — |
| The Cleveland Clinic Foundation | Cleveland | Ohio | 44106 | — |
| Providence ALS Center | Portland | Oregon | 97213 | — |
| University of Pennsylvania | Philadelphia | Pennsylvania | 19104 | — |
| New Orleans Center for Clinical Research/Volunteer Research Group, an AMR Company | Knoxville | Tennessee | 37920 | — |
| Methodist Neurological Institute | Houston | Texas | 77030 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 20 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT02623699, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Jul 28, 2023 · Synced Oct 29, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT02623699 live on ClinicalTrials.gov.