Expanded Access to Voxelotor for Patients With Sickle Cell Disease Who Have No Alternative Treatment Options
|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. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT03943615|
Expanded Access Status : Approved for marketing
First Posted : May 9, 2019
Last Update Posted : December 19, 2019
|Condition or disease||Intervention/treatment|
|Sickle Cell Disease||Drug: Voxelotor|
The purpose of this program is to provide early access to voxelotor for patients with sickle cell disease who have exhausted alternative treatment options and are ineligible for actively-recruiting clinical trials of voxelotor.
This EAP may continue until such time that voxelotor is commercially available or the Sponsor discontinues the voxelotor EAP. Discontinuation may occur earlier if an individual participant or physician or Sponsor Medical Monitor decides to discontinue treatment.
|Study Type :||Expanded Access|
|Expanded Access Type :||Individual Patients|
|See clinical trials of the intervention/treatment in this expanded access record.|
|Official Title:||An Open-Label, Expanded Access Protocol for Patient With Sickle Cell Disease Who Have No Alternative Treatment Options|
- Drug: Voxelotor
Other Name: GBT440
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): NCT03943615
|Study Director:||Jonathan Sorof, MD||Global Blood Therapeutics, Inc.|