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

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ClinicalTrials.gov Identifier: NCT04972487
Expanded Access Status : Available
First Posted : July 22, 2021
Last Update Posted : July 22, 2021
Sponsor:
Information provided by (Responsible Party):
Biogen

Brief Summary:
The objective is to provide access to tofersen to participants with SOD1-ALS with the most rapidly progressive disease.

Condition or disease Intervention/treatment
Superoxide Dismutase 1-Amyotropic Lateral Sclerosis Drug: Tofersen

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Study Type : Expanded Access
Expanded Access Type : Individual Patients
  See clinical trials of the intervention/treatment in this expanded access record.
Official Title: Expanded Access Program (EAP) for Tofersen in Participants With Superoxide Dismutase 1-Amyotropic Lateral Sclerosis (SOD1-ALS)



Intervention Details:
  • Drug: Tofersen
    Administered as intrathecal bolus injection
    Other Name: BIIB067

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
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).
  • Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS-R) decline faster than 2.0 points per month (points/month) at most recent assessment, with slope calculated as: [(48) - (ALSFRS-R score at entry)] / [months since symptom onset].

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


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04972487


Contacts
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Contact: US Biogen Clinical Trial Center 866-633-4636 clinicaltrials@biogen.com
Contact: Global Biogen Clinical Trial Center clinicaltrials@biogen.com

Sponsors and Collaborators
Biogen
Investigators
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Study Director: Medical Director Biogen
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Responsible Party: Biogen
ClinicalTrials.gov Identifier: NCT04972487    
Other Study ID Numbers: Expanded Access for Tofersen
First Posted: July 22, 2021    Key Record Dates
Last Update Posted: July 22, 2021
Last Verified: July 2021
Additional relevant MeSH terms:
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Motor Neuron Disease
Sclerosis
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases