Open-Label Extension of Voxelotor

Sickle Cell Disease Clinical Trial

Official title:

An Open-Label Extension Study of Voxelotor Administered Orally to Participants With Sickle Cell Disease Who Have Participated in Voxelotor Clinical Trials

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04188509
• Other study ID #: GBT440-038
• Secondary ID: C53410232019-003
• Status: Recruiting
• Phase: Phase 3
• Start date: November 18, 2019
• Est. completion date: March 4, 2028

Study information

• Verified date: May 2024
• Source: Pfizer
• Contact: Pfizer CT.gov Call Center
• Phone: 1-800-718-1021
• Email: ClinicalTrials.gov_Inquiries[@]pfizer.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

Open-label extension study of voxelotor for participants with Sickle Cell Disease who have participated in voxelotor clinical trials.

Description:

Open-label extension (OLE) study of voxelotor for participants with Sickle Cell Disease who have participated in voxelotor clinical trials. Up to approximately 600 participants with sickle cell disease (SCD), will be enrolled at approximately 70 clinical sites globally. All participants will receive voxelotor once daily, administered orally as tablets, dispersible tablets, or powder for oral suspension formulation. The objective of this OLE is to assess the safety of, and SCD-related complications of, long-term treatment with voxelotor, in participants who have completed treatment in a Global Blood Therapeutics (GBT)-sponsored voxelotor clinical study.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 300
• Est. completion date: March 4, 2028
• Est. primary completion date: March 4, 2028
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Months to 18 Years

Eligibility

Inclusion Criteria:

• Male or female participant with SCD, who participated and received study drug in a GBT-sponsored voxelotor clinical study.

Note: Participants who discontinued study drug due to an AE, but who remained on study, may be eligible for treatment in this study provided the AE does not pose a risk for treatment with voxelotor.

Note: Participants who discontinued Study GBT440-032 as the result of an abnormal transcranial Doppler (TCD) flow velocity assessment (= 200 cm/sec) are eligible for treatment in this study.

• Participant has provided written consent/assent (for pediatric participants, both the consent of the participant's legal representative or legal guardian and the participant's assent [where applicable] must be obtained).

Exclusion Criteria:

• Female participant who is breastfeeding or pregnant

• Participant withdrew consent from a GBT-sponsored voxelotor clinical study

• Known hypersensitivity to voxelotor or any other components of the study drug

• Use of St. John's wort, sensitive cytochrome P450 (CYP) 3A4 substrates with a narrow therapeutic index, or moderate or strong CYP3A4 inducers within 30 days of Day 1

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• Sickle Cell Disease

Intervention

Drug:
• Voxelotor
All participants will receive voxelotor once daily (QD), administered orally as tablets, dispersible tablets, or a powder for oral suspension formulation (powder formulation packaged as stick packs).

Locations

Country	Name	City	State
Egypt	Alexandria University Hospital - Clinical Research Center	Alexandria
Egypt	Ain Shams University Hospital - Clinical Research Center (MASRI-CRC)	Cairo
Egypt	Ain Shams University Hospital - Clinical Research Center (MASRI-CRC)	Cairo
Egypt	Cairo University Hospital, Abu El Rish Hospital	Cairo
Egypt	Zagazig University Hospital	Zagazig	Alsharkia
Lebanon	American University of Beirut - Medical Center	Beirut
Lebanon	Nini Hospital	Tripoli
Nigeria	University of Calabar Teaching Hospital	Calabar	Cross River
Nigeria	College of Medicine, University of Ibadan	Ibadan	OYO State
Nigeria	University College Hospital Paediatric Haematology and Oncology Unit	Ibadan	OYO State
Nigeria	Barau Dikko Teaching Hospital/Kaduna State University	Kaduna
Nigeria	Aminu Kano Teaching Hospital	Kano
Nigeria	Lagos University Teaching Hospital	Lagos
United Kingdom	Barts Health NHS Trust , The Royal London Hospital	London
United Kingdom	Guy's & St Thomas NHS Trust, St Thomas' Hospital	London
United Kingdom	Guy'S and St Thomas' Nhs Foundation Trust	London
United Kingdom	University College Hospital NHS Foundation Trust	London	Greater London
United States	Children's Healthcare of Atlanta Scottish Rite	Atlanta	Georgia
United States	Ann & Robert H. Lurie Children's Hospital of Chicago	Chicago	Illinois
United States	ECU Physicians, Brody Outpatient Clinic	Greenville	North Carolina
United States	Children's Mercy Hospital	Kansas City	Missouri
United States	UPMC Children's Hospital of Pittsburgh	Pittsburgh	Pennsylvania
United States	Children's National Medical Center	Washington	District of Columbia

Sponsors (1)

Lead Sponsor	Collaborator
Pfizer

Countries where clinical trial is conducted

United States,  Egypt,  Lebanon,  Nigeria,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	TEAEs and SAEs	Treatment Emergent Adverse Events and Serious Adverse Events	Throughout entire study
Primary	Sickle Cell Disease-Related Complications	Frequency of SCD-related complications	Throughout entire study

External Links

•   To obtain contact information for a study center near you, click here.