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 MuscularAtrophy (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.

No longer available
Treatment IND/Protocol
Infantile-onset Spinal Muscular Atrophy

Drug: Nusinersen

Administered by intrathecal injection
Other Name: ISIS 396443,BIIB058,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
Eligibility Age
Minimum: N/A ~ Maximum: N/A
Countries
Colombia
New Zealand
Turkey
Locations

Hospital Pablo Tobon Uribe
Medellin, Colombia

Auckland City Hospital
Grafton, Auckland, New Zealand

Auckland District Health Board ADHB
Grafton, Auckland, New Zealand

Erciyes University Hospital
Kayseri, Anatolia, Turkey

Hacettepe University
Ankara, Central Anatolia, Turkey

Marmara Uni. Research & Educational Hospital
Kadıköy, Istanbul, Turkey

Medipol University Hospital
Istanbul, Marmara, Turkey

Medical Director, Study Director
Biogen

NCT 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
Muscular Atrophy
Muscular Atrophy, Spinal
Atrophy