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

Expanded access is currently available for this treatment.
Verified August 2017 by Biogen
Information provided by (Responsible Party):
Biogen Identifier:
First received: August 10, 2016
Last updated: August 1, 2017
Last verified: August 2017
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.

Condition Intervention
Infantile-onset Spinal Muscular Atrophy Drug: nusinersen

Study Type: Expanded Access     What is Expanded Access?
Available for Treatment IND/Protocol
Official Title: Expanded Access Program (EAP) to Provide Nusinersen to Patients With Infantile-onset Spinal Muscular Atrophy (SMA)

Resource links provided by NLM:

Further study details as provided by Biogen:

Intervention Details:
    Drug: nusinersen
    Administered by intrathecal injection
    Other Names:
    • ISIS 396443
    • BIIB058
    • Spinraza
Detailed Description:

Availability of nusinersen at an existing clinical trial site will depend on territory eligibility. Program opening date will depend on regulatory requirements and center-specific factors. Participating sites will be added as they apply for the EAP. A doctor must decide whether the potential benefit outweighs the risk of receiving an investigational therapy based on the individual patient's medical history and program eligibility criteria.

The Expanded Access Program in the United States is now closed to new patient enrollment as nusinersen has received FDA approval.


Ages Eligible for Study:   Child, Adult, Senior
Sexes Eligible for Study:   All

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

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT02865109

Contact: Biogen 866-633-4636

  Show 39 Study Locations
Sponsors and Collaborators
Study Director: Medical Director Biogen
  More Information

Responsible Party: Biogen Identifier: NCT02865109     History of Changes
Other Study ID Numbers: 232-SM-901
Study First Received: August 10, 2016
Last Updated: August 1, 2017

Keywords provided by Biogen:
Spinal Muscular Atrophy
ISIS 396443

Additional relevant MeSH terms:
Muscular Atrophy
Muscular Atrophy, Spinal
Pathological Conditions, Anatomical
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Spinal Cord Diseases
Central Nervous System Diseases
Motor Neuron Disease
Neurodegenerative Diseases
Neuromuscular Diseases processed this record on August 17, 2017