Expanded Access Program (EAP) for Nusinersen in Participants With Infantile-onset (Consistent With Type 1) Spinal Muscular Atrophy (SMA)

April 1, 2021 updated by: Biogen

Expanded Access Program (EAP) to Provide Nusinersen to Patients With Infantile-onset Spinal Muscular Atrophy (SMA)

To provide access to nusinersen to eligible patients with Infantile-onset Spinal Muscular Atrophy (SMA) (consistent with Type 1) to address a high-unmet medical need.

Study Overview

Status

No longer available

Conditions

Intervention / Treatment

Detailed Description

The nusinersen expanded access program (EAP) is available at approved treatment centers in select territories.

A doctor must decide whether nusinersen treatment is appropriate for each patient, based on the patient's medical history and program eligibility criteria. A full list of participating treatment centers is provided in the 'Contacts and Locations' section of this listing, and is regularly updated.

Following local approval and official reimbursement of nusinersen in each territory, the EAP will close and patients will transfer to commercially available drug.

Study Type

Expanded Access

Expanded Access Type

  • Treatment IND/Protocol

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

  • Colombia
      • Medellin, Colombia, 050036
        • Hospital Pablo Tobon Uribe
  • New Zealand
    • Auckland
      • Grafton, Auckland, New Zealand, 1023
        • Auckland City Hospital
      • Grafton, Auckland, New Zealand, 1023
        • Auckland District Health Board ADHB
  • Turkey
    • Anatolia
      • Kayseri, Anatolia, Turkey, 38000
        • Erciyes University Hospital
    • Central Anatolia
      • Ankara, Central Anatolia, Turkey, 06100
        • Hacettepe University
    • Istanbul
      • Kadıköy, Istanbul, Turkey, 34722
        • Marmara Uni. Research & Educational Hospital
    • Marmara
      • Istanbul, Marmara, Turkey, 34214
        • Medipol University Hospital

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

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Key Inclusion Criteria:

  • Genetic documentation of 5q SMA homozygous gene deletion, homozygous mutation, or compound heterozygote.
  • Onset of clinical signs and symptoms at ≤ 6 months (180 days) of age, consistent with infantile onset, Type I SMA
  • Patient whose care in the opinion of the treating physician meets, and is expected to continue to meet, the guidelines set out in the 2007 Consensus Statement for Standard of Care in SMA

Key Exclusion Criteria:

  • Patient is qualified to participate in an ongoing clinical trial with nusinersen
  • Participation in a prior nusinersen study
  • Previous exposure to nusinersen
  • History of brain or spinal cord disease that would interfere with the LP procedures or CSF circulation
  • Presence of implanted shunt for the drainage of CSF or implanted CNS catheter
  • Previous or current participation in a clinical trial with an investigational gene therapy for SMA
  • Participation in a study with an investigational therapy for SMA within 6 months or five half-lives of the investigational drug, whichever is the longer, prior to the first dose of nusinersen.

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 Major Dates

Study Start

December 7, 2022

Primary Completion

December 7, 2022

Study Completion

December 7, 2022

Study Registration Dates

First Submitted

August 10, 2016

First Submitted That Met QC Criteria

August 10, 2016

First Posted (Estimated)

August 12, 2016

Study Record Updates

Last Update Posted (Actual)

April 5, 2021

Last Update Submitted That Met QC Criteria

April 1, 2021

Last Verified

March 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 232-SM-901

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

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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