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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. Identifier: NCT02865109
Expanded Access Status : Available
First Posted : August 12, 2016
Last Update Posted : April 29, 2020
Information provided by (Responsible Party):

Tracking Information
First Submitted Date August 10, 2016
First Posted Date August 12, 2016
Last Update Posted Date April 29, 2020
Descriptive Information
Brief Title Expanded Access Program (EAP) for Nusinersen in Participants With Infantile-onset (Consistent With Type 1) 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.

Study Type Expanded Access
Expanded Access Type Treatment IND/Protocol
Intervention Drug: Nusinersen
Administered by intrathecal injection
Other Names:
  • ISIS 396443
  • BIIB058
  • Spinraza
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Recruitment Information
Expanded Access Status Available
Contact: US Biogen Clinical Trial Center 866-633-4636
Contact: Global Biogen Clinical Trial Center
Listed Location Countries China,   Colombia,   Mexico,   New Zealand,   Taiwan,   Turkey
Removed Location Countries Australia,   Austria,   Belgium,   Canada,   Denmark,   Finland,   France,   Germany,   Greece,   Ireland,   Israel,   Italy,   Korea, Republic of,   Netherlands,   Norway,   Poland,   Portugal,   Slovenia,   Spain,   Sweden,   Switzerland,   United Kingdom,   United States
Administrative Information
NCT Number NCT02865109
Responsible Party Biogen
Study Sponsor Biogen
Collaborators Not Provided
Study Director: Medical Director Biogen
PRS Account Biogen
Verification Date April 2020