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 : No longer available

First Posted : August 12, 2016

Last Update Posted : April 5, 2021

View this study on the modernized ClinicalTrials.gov

Sponsor:

Information provided by (Responsible Party):

Biogen

Study Description

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 Design

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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)

Resource links provided by the National Library of Medicine

MedlinePlus Genetics related topics: Spinal muscular atrophy

MedlinePlus related topics: Spinal Muscular Atrophy

Drug Information available for: Nusinersen

Genetic and Rare Diseases Information Center resources: Spinal Muscular Atrophy Amyotrophic Lateral Sclerosis

U.S. FDA: Expanded Access (Compassionate Use)

U.S. FDA Resources

Interventions

Intervention Details:

Drug: Nusinersen
Administered by intrathecal injection
Other Names:
- ISIS 396443
- BIIB058
- Spinraza

Eligibility Criteria

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

Contacts and Locations

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

Locations

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

Sponsors and Collaborators

Biogen

Investigators

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Study Director:	Medical Director	Biogen

More Information

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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 5, 2021
Last Verified:	March 2021

Keywords provided by Biogen:


Spinal Muscular Atrophy SMA SMN SMNRx ISIS-SMNRx ISIS-SMN Rx ISIS 396443 ISIS-396443 ENDEAR	IONIS-SMNRx IONIS-SMN Rx EMBRACE NURTURE IONIS BIOGEN SHINE Nusinersen

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

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