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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02865109
Expanded Access Status : Available
First Posted : August 12, 2016
Last Update Posted : April 29, 2020
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.

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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
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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

Locations
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China, Taiwan, Province OF China
Kaohsiung Medical University Hospital Available
Kaohsiung, Taiwan, Province OF China, China, 807
National Taiwan University Hospital Available
Taipei, Taiwan, Province OF China, China, 100
Colombia
Hospital Pablo Tobon Uribe Available
Medellin, Colombia, 050036
Mexico
Hospital Infantil de Mexico Federico Gomez Available
Mexico city, Mexico, 06720
New Zealand
Auckland City Hospital Available
Grafton, Auckland, New Zealand, 1023
Auckland District Health Board ADHB Available
Grafton, Auckland, New Zealand, 1023
Taiwan
Taipei Veterans General Hospital Available
Taipei city, Beitou District, Taiwan, 11217
Kaohsiung Medical University Hospital Available
Kaohsiung, Taiwan, 807
National Taiwan University Hospital Available
Taipei, Taiwan, 114
Turkey
Erciyes University Hospital Available
Kayseri, Anatolia, Turkey, 38000
Hacettepe University Available
Ankara, Central Anatolia, Turkey, 06100
Marmara Uni. Research & Educational Hospital Available
Kadıköy, Istanbul, Turkey, 34722
Medipol University Hospital Available
Istanbul, Marmara, Turkey, 34214
Sponsors and Collaborators
Biogen
Investigators
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Study Director: Medical Director Biogen
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Responsible Party: Biogen
ClinicalTrials.gov Identifier: NCT02865109    
Other Study ID Numbers: 232-SM-901
First Posted: August 12, 2016    Key Record Dates
Last Update Posted: April 29, 2020
Last Verified: April 2020
Keywords provided by Biogen:
ISIS 396443
ISIS-396443
Nusinersen
Spinal Muscular Atrophy
SMA
SMN
SMNRx
ISIS-SMNRx
ISIS-SMN Rx
ENDEAR
IONIS-SMNRx
IONIS-SMN Rx
EMBRACE
NURTURE
IONIS
BIOGEN
SHINE
Additional relevant MeSH terms:
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Muscular Atrophy
Muscular Atrophy, Spinal
Atrophy
Pathological Conditions, Anatomical
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Spinal Cord Diseases
Central Nervous System Diseases
Motor Neuron Disease
Neurodegenerative Diseases
Neuromuscular Diseases