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

Last updated: June 7, 2024
Sponsor: Biogen
Overall Status: Planned

Phase

N/A

Condition

Amyotrophic Lateral Sclerosis (Als)

Myasthenia Gravis (Chronic Weakness)

Scar Tissue

Treatment

Tofersen

Clinical Study ID

NCT04972487
233AS001
  • Ages > 18
  • All Genders

Study Summary

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.

Eligibility Criteria

Inclusion

Key Inclusion Criteria:

  • Medically able to undergo the program procedures, as determined by the treatinghealthcare professional (HCP).

  • Weakness attributable to ALS and associated with a mutation in the SOD1 gene (SOD1-ALS).

Exclusion

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 Design

Treatment Group(s): 1
Primary Treatment: Tofersen
Phase:
Study Start date:
Estimated Completion Date:

Study 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.

Connect with a study center

  • Research Site

    Anchorage, Alaska 99508
    United States

    Site Not Available

  • Research Site

    Los Angeles, California 90033
    United States

    Site Not Available

  • Research Site

    Orange, California 92868
    United States

    Site Not Available

  • Research Site

    San Diego, California 92103
    United States

    Site Not Available

  • Research Site

    San Francisco, California 94114
    United States

    Site Not Available

  • Research Site

    Chicago, Illinois 60611
    United States

    Site Not Available

  • Research Site

    Kansas City, Kansas 66160
    United States

    Site Not Available

  • Research Site

    Boston, Massachusetts 02114
    United States

    Site Not Available

  • Research Site

    Worcester, Massachusetts 01655
    United States

    Site Not Available

  • Research Site

    Ann Arbor, Michigan 48109
    United States

    Site Not Available

  • Research Site

    Detroit, Michigan 48202
    United States

    Site Not Available

  • Research Site

    Rochester, Minnesota 55905
    United States

    Site Not Available

  • Research Site

    Saint Louis, Missouri 63110
    United States

    Site Not Available

  • Research Site

    Lebanon, New Hampshire 03766
    United States

    Site Not Available

  • Research Site

    Amherst, New York 14226
    United States

    Site Not Available

  • Research Site

    New York, New York 10032
    United States

    Site Not Available

  • Research Site

    Syracuse, New York 13210
    United States

    Site Not Available

  • Research Site

    Philadelphia, Pennsylvania 19104
    United States

    Site Not Available

  • Research Site

    Austin, Texas 78759
    United States

    Site Not Available

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