Open-Label Extension of Voxelotor

Phase 3

Terminated

• Conditions: Sickle Cell Disease
• Interventions: Drug: Voxelotor

• Registration Number: NCT04188509
• Lead Sponsor: Pfizer

Brief Summary

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

Detailed 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.

Study Timeline

• Study Start: 2019-11-18
• Study First Submitted: 2019-12-03
• Study First Submitted QC: 2019-12-03
• Study First Posted: 2019-12-06
• Primary Completion: 2024-10-30
• Status Verified: 2024-11
• Study Completion: 2024-11-01
• Last Update Submitted: 2024-11-12
• Last Update Posted: 2024-11-14

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 162

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Voxelotor	Voxelotor	All participants will receive voxelotor once daily (QD), administered orally as tablets, dispersible tablets, or a stick pack formulation (powder blend formulation packaged as stick packs). Participants aged ≥ 12 years and/or ≥ 40 kgs will receive a voxelotor dose of 1500 mg QD. Participants aged \< 12 years and \< 40 kgs will receive weight based dosing of voxelotor. The participant's weight at study entry will be used to determine the starting voxelotor dose in this study. Participants may receive study drug as long they continue to receive clinical benefit that outweighs risk as determined by the investigator and/or until the participant has access to voxelotor from an alternative source.

Primary Outcome Measures

Name	Time	Method
Sickle Cell Disease-Related Complications	Throughout entire study	Frequency of SCD-related complications
TEAEs and SAEs	Throughout entire study	Treatment Emergent Adverse Events and Serious Adverse Events

Trial Locations

Locations (22)

Children's National Medical Center; 🇺🇸 Washington, District of Columbia, 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

Children's Mercy Hospital; 🇺🇸 Kansas City, Missouri, United States

ECU Physicians, Brody Outpatient Clinic; 🇺🇸 Greenville, North Carolina, United States

UPMC Children's Hospital of Pittsburgh; 🇺🇸 Pittsburgh, Pennsylvania, United States

Zagazig University Hospital; 🇪🇬 Zagazig, Alsharkia, Egypt

Alexandria University Hospital - Clinical Research Center; 🇪🇬 Alexandria, Egypt

Cairo University Hospital, Abu El Rish Hospital; 🇪🇬 Cairo, Egypt

Ain Shams University Hospital - Clinical Research Center (MASRI-CRC); 🇪🇬 Cairo, Egypt

American University of Beirut - Medical Center; 🇱🇧 Beirut, Lebanon

Nini Hospital; 🇱🇧 Tripoli, Lebanon

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, Nigeria

University College Hospital NHS Foundation Trust; 🇬🇧 London, Greater London, 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