Back to resultsExpanded Access Program for Tofersen in Participants With Superoxide Dismutase 1-Amyotropic Lateral Sclerosis
Primary Purpose
Superoxide Dismutase 1-Amyotropic Lateral Sclerosis
Status
Approved for marketing
Phase
Locations
United States
Study Type
Expanded Access
Intervention
Tofersen
Sponsored by
About this trial
This is an expanded access trial for Superoxide Dismutase 1-Amyotropic Lateral Sclerosis
Eligibility Criteria
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
Sites / Locations
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Outcomes
Primary Outcome Measures
Secondary Outcome Measures
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT04972487
Brief Title
Expanded Access Program for Tofersen in Participants With Superoxide Dismutase 1-Amyotropic Lateral Sclerosis
Official Title
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
Study Type
Expanded Access
2. Study Status
Record Verification Date
May 2023
Overall Recruitment Status
Approved for marketing
Study Start Date
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Primary Completion Date
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Study Completion Date
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3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Biogen
4. Oversight
5. Study Description
Brief 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.
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Superoxide Dismutase 1-Amyotropic Lateral Sclerosis
7. Study Design
8. Arms, Groups, and Interventions
Intervention Type
Drug
Intervention Name(s)
Tofersen
Other Intervention Name(s)
BIIB067, QALSODY
Intervention Description
Administered as intrathecal bolus injection
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Director
Organizational Affiliation
Biogen
Official's Role
Study Director
Facility Information:
Facility Name
Research Site
City
Anchorage
State/Province
Alaska
ZIP/Postal Code
99508
Country
United States
Facility Name
Research Site
City
Los Angeles
State/Province
California
ZIP/Postal Code
90033
Country
United States
Facility Name
Research Site
City
Orange
State/Province
California
ZIP/Postal Code
92868
Country
United States
Facility Name
Research Site
City
San Diego
State/Province
California
ZIP/Postal Code
92103
Country
United States
Facility Name
Research Site
City
San Francisco
State/Province
California
ZIP/Postal Code
94114
Country
United States
Facility Name
Research Site
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Facility Name
Research Site
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
Country
United States
Facility Name
Research Site
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States
Facility Name
Research Site
City
Worcester
State/Province
Massachusetts
ZIP/Postal Code
01655
Country
United States
Facility Name
Research Site
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48109
Country
United States
Facility Name
Research Site
City
Detroit
State/Province
Michigan
ZIP/Postal Code
48202
Country
United States
Facility Name
Research Site
City
Rochester
State/Province
Minnesota
ZIP/Postal Code
55905
Country
United States
Facility Name
Research Site
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Facility Name
Research Site
City
Lebanon
State/Province
New Hampshire
ZIP/Postal Code
03766
Country
United States
Facility Name
Research Site
City
Amherst
State/Province
New York
ZIP/Postal Code
14226
Country
United States
Facility Name
Research Site
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
Facility Name
Research Site
City
Syracuse
State/Province
New York
ZIP/Postal Code
13210
Country
United States
Facility Name
Research Site
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
Research Site
City
Austin
State/Province
Texas
ZIP/Postal Code
78759
Country
United States
12. IPD Sharing Statement
Learn more about this trial
Expanded Access Program for Tofersen in Participants With Superoxide Dismutase 1-Amyotropic Lateral Sclerosis
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