Superoxide Dismutase 1-Amyotropic Lateral SclerosisNCT04972487
Expanded Access Program for Tofersen in Participants With Superoxide Dismutase 1-Amyotropic Lateral Sclerosis
A study testing Tofersen in people with Superoxide Dismutase 1-Amyotropic Lateral Sclerosis, run by Biogen at 19 sites in United States.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Superoxide Dismutase 1-Amyotropic Lateral Sclerosis
- What is being tested?
- Tofersen
- Type of treatment
- Injection or infusion
- How many people
- Not given
- Where
- 19 sitesUnited States
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- Adult, Older adult
Who may be able to join
- 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).
Who cannot join
- 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
Trial locations and contacts
19 sites in 1 country.
19 sites in 1 country
United States19 sites
Research Site
Amherst, New York, 14226
Research Site
Anchorage, Alaska, 99508
Research Site
Ann Arbor, Michigan, 48109
Research Site
Austin, Texas, 78759
Research Site
Boston, Massachusetts, 02114
Research Site
Chicago, Illinois, 60611
Research Site
Detroit, Michigan, 48202
Research Site
Kansas City, Kansas, 66160
Research Site
Lebanon, New Hampshire, 03766
Research Site
Los Angeles, California, 90033
Research Site
New York, New York, 10032
Research Site
Orange, California, 92868
Research Site
Philadelphia, Pennsylvania, 19104
Research Site
Rochester, Minnesota, 55905
Research Site
San Diego, California, 92103
Research Site
San Francisco, California, 94114
Research Site
St Louis, Missouri, 63110
Research Site
Syracuse, New York, 13210
Research Site
Worcester, Massachusetts, 01655
About this study
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
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.
Read the 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.
- Being tested
- Tofersen
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TofersenDrug
Also called BIIB067, QALSODY
Administered as intrathecal bolus injection
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jul 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Superoxide Dismutase 1-Amyotropic Lateral SclerosisStudies of the same condition, open and under way.
- More trials run by BiogenThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- Biogen
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Biogen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04972487
- Sponsor's protocol ID
- 233AS001
- Status last verified
- Jun 2024
- First submitted
- 15 Jul 2021
- Last updated
- 10 Jun 2024
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Jun 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT04972487
- Acronym / sponsor ID
- 233AS001
- Study type
- Expanded access
- Recruitment status
- Approved For Marketing
- Lead sponsor
- BiogenIndustry (pharma, biotech, devices)
- First posted
- 22 Jul 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- 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 (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TofersenAlso: BIIB067, QALSODYAdministered as intrathecal bolus injection | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Jun 2024.
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Director study director
Biogen
19 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Tofersen in people with Superoxide Dismutase 1-Amyotropic Lateral Sclerosis, run by Biogen at 19 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 10 Jun 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 19 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Jun 2024.