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

Biogen

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

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

Available
EA Type
Treatment IND/Protocol
Infantile-onset Spinal Muscular Atrophy

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

Eligibility 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

Eligibility Gender: 
All
Countries: 
China, Colombia, Korea, Republic of, Mexico, Poland, Portugal, Taiwan, Turkey, United Kingdom
Contacts: 

US Biogen Clinical Trial Center
866-633-4636
clinicaltrials@biogen.com

Global Biogen Clinical Trial Center
clinicaltrials@biogen.com

Medical Director
Study Director
Biogen

EA Number: 
Keywords: 
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
MeSH Terms: 
Atrophy, Muscular Atrophy, Muscular Atrophy, Spinal
Disclaimer:
Expanded Access program information for companies listed in the Expanded Access Company Directory is pulled daily from ClinicalTrials.gov, a resource provided by the National Institutes of Health in cooperation with the U.S. Food and Drug Administration. Click here for more information about ClinicalTrials.gov.