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

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ClinicalTrials.gov Identifier: NCT02865109
Expanded Access Status : Available
First Posted : August 12, 2016
Last Update Posted : October 24, 2018
Sponsor:
Information provided by (Responsible Party):
Biogen

Brief Summary:
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 or disease Intervention/treatment
Infantile-onset Spinal Muscular Atrophy Drug: Nusinersen

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
  See clinical trials of the intervention/treatment in this expanded access record.
Official Title: Expanded Access Program (EAP) to Provide Nusinersen to Patients With Infantile-onset Spinal Muscular Atrophy (SMA)



Intervention Details:
  • Drug: Nusinersen
    Administered by intrathecal injection
    Other Names:
    • ISIS 396443
    • BIIB058
    • Spinraza

Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Criteria

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


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): NCT02865109


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

  Show 39 Study Locations
Sponsors and Collaborators
Biogen
Investigators
Study Director: Medical Director Biogen

Responsible Party: Biogen
ClinicalTrials.gov Identifier: NCT02865109     History of Changes
Other Study ID Numbers: 232-SM-901
First Posted: August 12, 2016    Key Record Dates
Last Update Posted: October 24, 2018
Last Verified: October 2018

Keywords provided by Biogen:
SMNRx
ISIS-SMNRx
IONIS-SMNRx
Spinal Muscular Atrophy
SMA
SMN
ISIS-SMN Rx
ISIS 396443
ISIS-396443
ENDEAR
IONIS-SMN Rx
EMBRACE
NURTURE
IONIS
BIOGEN
SHINE
Nusinersen

Additional relevant MeSH terms:
Atrophy
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