Clinical trial NCT02865109: Expanded Access Program (EAP) for Nusinersen in Participants With Infantile-onset (Consistent With Type 1) Spinal Muscular Atrophy (SMA)
Official title: Expanded Access Program (EAP) to Provide Nusinersen to Patients With Infantile-onset Spinal Muscular Atrophy (SMA)
Study type: EXPANDED_ACCESS
Overall status: NO_LONGER_AVAILABLE
Conditions: Infantile-onset Spinal Muscular Atrophy
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
Lead sponsor: Biogen
Sponsor class: INDUSTRY
First posted: 2016-08-12
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT

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.

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

Primary outcomes:
Secondary outcomes:
Other outcomes:
Locations:
city: Medellin
country: Colombia
facility: Hospital Pablo Tobon Uribe
city: Grafton
country: New Zealand
facility: Auckland City Hospital
state: Auckland
city: Grafton
country: New Zealand
facility: Auckland District Health Board ADHB
state: Auckland
city: Kayseri
country: Turkey
facility: Erciyes University Hospital
state: Anatolia
city: Ankara
country: Turkey
facility: Hacettepe University
state: Central Anatolia
city: Kadıköy
country: Turkey
facility: Marmara Uni. Research & Educational Hospital
state: Istanbul
city: Istanbul
country: Turkey
facility: Medipol University Hospital
state: Marmara
Study officials:
affiliation: Biogen
name: Medical Director
role: STUDY_DIRECTOR
References: