Clinical trial NCT04188509: Open-Label Extension of Voxelotor
Official title: An Open-Label Extension Study of Voxelotor Administered Orally to Participants With Sickle Cell Disease Who Have Participated in Voxelotor Clinical Trials
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE3
Conditions: Sickle Cell Disease
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2019-12-06
Enrollment: 162
Sex: ALL
Minimum age (years): 6
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Open-Label Extension study available to eligible participants from GBT-sponsored voxelotor clinical studies.
Primary purpose: TREATMENT

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.

Eligibility criteria: 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

Primary outcomes:
measure: TEAEs and SAEs
timeFrame: Throughout entire study
measure: Sickle Cell Disease-Related Complications
timeFrame: Throughout entire study
Secondary outcomes:
Other outcomes:
Locations:
city: Washington
country: United States
facility: Children's National Medical Center
state: District of Columbia
city: Atlanta
country: United States
facility: Children's Healthcare of Atlanta Scottish Rite
state: Georgia
city: Chicago
country: United States
facility: Ann & Robert H. Lurie Children's Hospital of Chicago
state: Illinois
city: Kansas City
country: United States
facility: Children's Mercy Hospital
state: Missouri
city: Greenville
country: United States
facility: ECU Physicians, Brody Outpatient Clinic
state: North Carolina
city: Pittsburgh
country: United States
facility: UPMC Children's Hospital of Pittsburgh
state: Pennsylvania
city: Zagazig
country: Egypt
facility: Zagazig University Hospital
state: Alsharkia
city: Alexandria
country: Egypt
facility: Alexandria University Hospital - Clinical Research Center
city: Cairo
country: Egypt
facility: Cairo University Hospital, Abu El Rish Hospital
city: Cairo
country: Egypt
facility: Ain Shams University Hospital - Clinical Research Center (MASRI-CRC)
city: Cairo
country: Egypt
facility: Ain Shams University Hospital - Clinical Research Center (MASRI-CRC)
city: Cairo
country: Egypt
facility: Cairo University Hospital, Abu El Rish Hospital
city: Beirut
country: Lebanon
facility: American University of Beirut - Medical Center
city: Tripoli
country: Lebanon
facility: Nini Hospital
city: Calabar
country: Nigeria
facility: University of Calabar Teaching Hospital
state: Cross River
city: Calabar
country: Nigeria
facility: University of Calabar Teaching Hospital
state: Cross River
city: Ibadan
country: Nigeria
facility: College of Medicine, University of Ibadan
state: OYO State
city: Ibadan
country: Nigeria
facility: University College Hospital Paediatric Haematology and Oncology Unit
state: OYO State
city: Kaduna
country: Nigeria
facility: Barau Dikko Teaching Hospital/Kaduna State University
city: Kano
country: Nigeria
facility: Aminu Kano Teaching Hospital
city: Lagos
country: Nigeria
facility: Lagos University Teaching Hospital
city: London
country: United Kingdom
facility: University College Hospital NHS Foundation Trust
state: Greater London
city: London
country: United Kingdom
facility: Barts Health NHS Trust , The Royal London Hospital
city: London
country: United Kingdom
facility: Guy's & St Thomas NHS Trust, St Thomas' Hospital
city: London
country: United Kingdom
facility: Guy'S and St Thomas' Nhs Foundation Trust
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References: