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|
Recruitment Status : Available
First Posted : August 12, 2016
Last Update Posted : April 9, 2018
|Condition or disease||Intervention/treatment|
|Infantile-onset Spinal Muscular Atrophy||Drug: nusinersen|
Availability of nusinersen at an existing clinical trial site will depend on territory eligibility. Program opening date will depend on regulatory requirements and center-specific factors. Participating sites will be added as they apply for the EAP. A doctor must decide whether the potential benefit outweighs the risk of receiving an investigational therapy based on the individual patient's medical history and program eligibility criteria.
The Expanded Access Program in the United States is now closed to new patient enrollment as nusinersen has received FDA approval.
|Study Type :||
|Official Title:||Expanded Access Program (EAP) to Provide Nusinersen to Patients With Infantile-onset Spinal Muscular Atrophy (SMA)|
U.S. FDA Resources
- ISIS 396443
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
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|Study Director:||Medical Director||Biogen|