Expanded Access Program for Tofersen in Participants With Superoxide Dismutase 1-Amyotropic Lateral Sclerosis

June 7, 2024 updated by: Biogen

Global Early Access Program to Provide Tofersen To Patients With Amyotrophic Lateral Sclerosis (ALS) Associated With a Mutation in the Superoxide Dismutase 1 (SOD1) Gene

The objective of this early access program (EAP) is to provide access to tofersen to eligible participants with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 (SOD1) gene prior to an alternative access mechanism in order to address a high unmet medical need in this population.

Study Overview

Status

Approved for marketing

Conditions

Intervention / Treatment

Detailed Description

Treating healthcare professionals (HCPs) of participants who meet the inclusion/exclusion criteria may request access by emailing medicineaccess@clinigengroup.com. The current EAP structure does not limit participants to receive access at specific sites. Treating HCPs can submit requests on behalf of participants by emailing Clinigen at the email address provided below. Information on the EAP eligibility criteria, including participants and center criteria, will be provided by Clinigen as part of the enrollment request process.

Study Type

Expanded Access

Expanded Access Type

  • Intermediate-size Population

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Alaska
      • Anchorage, Alaska, United States, 99508
        • Research Site
    • California
      • Los Angeles, California, United States, 90033
        • Research Site
      • Orange, California, United States, 92868
        • Research Site
      • San Diego, California, United States, 92103
        • Research Site
      • San Francisco, California, United States, 94114
        • Research Site
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Research Site
    • Kansas
      • Kansas City, Kansas, United States, 66160
        • Research Site
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • Research Site
      • Worcester, Massachusetts, United States, 01655
        • Research Site
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
        • Research Site
      • Detroit, Michigan, United States, 48202
        • Research Site
    • Minnesota
      • Rochester, Minnesota, United States, 55905
        • Research Site
    • Missouri
      • St Louis, Missouri, United States, 63110
        • Research Site
    • New Hampshire
      • Lebanon, New Hampshire, United States, 03766
        • Research Site
    • New York
      • Amherst, New York, United States, 14226
        • Research Site
      • New York, New York, United States, 10032
        • Research Site
      • Syracuse, New York, United States, 13210
        • Research Site
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Research Site
    • Texas
      • Austin, Texas, United States, 78759
        • Research Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

N/A

Description

Key Inclusion Criteria:

  • Medically able to undergo the program procedures, as determined by the treating healthcare professional (HCP).
  • Weakness attributable to ALS and associated with a mutation in the SOD1 gene (SOD1-ALS).

Key Exclusion Criteria:

  • Previous or current participation in a clinical trial of tofersen.
  • Use of an investigational medicinal product (IMP) for amyotrophic lateral sclerosis (ALS) within 5 half-lives of the IMP before the first dose of tofersen.
  • Participant's primary place of residence is outside of the country of treatment.

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Biogen

Investigators

  • Study Director: Medical Director, Biogen

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

First Submitted

July 15, 2021

First Submitted That Met QC Criteria

July 15, 2021

First Posted (Actual)

July 22, 2021

Study Record Updates

Last Update Posted (Actual)

June 10, 2024

Last Update Submitted That Met QC Criteria

June 7, 2024

Last Verified

June 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 233AS001

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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