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.|
|ClinicalTrials.gov Identifier: NCT02865109|
Expanded Access Status : Available
First Posted : August 12, 2016
Last Update Posted : June 19, 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 :||Expanded Access|
|Expanded Access Type :||Treatment IND/Protocol|
|See clinical trials of the intervention/treatment in this expanded access record.|
|Official Title:||Expanded Access Program (EAP) to Provide Nusinersen to Patients With Infantile-onset Spinal Muscular Atrophy (SMA)|
- Drug: nusinersen
Administered by intrathecal injectionOther Names:
- 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
Show 35 Study Locations
|Study Director:||Medical Director||Biogen|