Expanded Access Protocol for Pediatric 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. 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: NCT04724421|
Expanded Access Status : Available
First Posted : January 26, 2021
Last Update Posted : December 29, 2021
|Condition or disease||Intervention/treatment|
|Sickle Cell Disease||Drug: Voxelotor|
Pediatric patients who are eligible for participation in this EAP will be treated with voxelotor, dispersible tablets or powder for oral suspension administered orally once daily (QD) at a weight-based dose, and followed by the treating physicians at the participating sites. Participants will receive standard of care treatment and procedures for management of SCD, including an initial visit and routine visits at least every 12 weeks (± 7 days) for clinical and laboratory assessments per standard of care and re-supply of voxelotor. A safety follow-up visit will be conducted 28 days (± 7 days) after the last dose of investigational product.
This EAP may continue until such time that voxelotor is commercially available for patients age 4 to 11 years, or the Sponsor discontinues the voxelotor EAP.
|Study Type :||Expanded Access|
|Expanded Access Type :||Treatment IND/Protocol|
|See clinical trials of the intervention/treatment in this expanded access record.|
|Official Title:||An Open-label, Expanded Access Protocol for Pediatric Patients With Sickle Cell Disease Who Have No Alternative Treatment Options|
- Drug: Voxelotor
synthetic small moleculeOther 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): NCT04724421
|Contact: Michelle Xu, MDfirstname.lastname@example.org|