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 21, 2019
|Condition or disease||Intervention/treatment|
|Infantile-onset Spinal Muscular Atrophy||Drug: Nusinersen|
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|
|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
|Contact: US Biogen Clinical Trial Centeremail@example.com|
|Contact: Global Biogen Clinical Trial Centerfirstname.lastname@example.org|
|Study Director:||Medical Director||Biogen|