- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04188509
Open-Label Extension of Voxelotor
An Open-Label Extension Study of Voxelotor Administered Orally to Participants With Sickle Cell Disease Who Have Participated in Voxelotor Clinical Trials
Study Overview
Detailed Description
Study Type
Enrollment (Estimated)
Phase
- Phase 3
Expanded Access
Contacts and Locations
Study Contact
- Name: Pfizer CT.gov Call Center
- Phone Number: 1-800-718-1021
- Email: ClinicalTrials.gov_Inquiries@pfizer.com
Study Locations
-
-
-
Alexandria, Egypt, 21131
- Recruiting
- Alexandria University Hospital - Clinical Research Center
-
Cairo, Egypt, 11566
- Recruiting
- Ain Shams University Hospital - Clinical Research Center (MASRI-CRC)
-
-
Alsharkia
-
Zagazig, Alsharkia, Egypt
- Recruiting
- Zagazig University Hospital
-
-
-
-
-
Beirut, Lebanon
- Recruiting
- American University of Beirut - Medical Center
-
Tripoli, Lebanon
- Recruiting
- Nini Hospital
-
-
-
-
-
Kaduna, Nigeria, 800212
- Recruiting
- Barau Dikko Teaching Hospital/Kaduna State University
-
Kano, Nigeria, 700233
- Recruiting
- Aminu Kano Teaching Hospital
-
Lagos, Nigeria, 100254
- Recruiting
- Lagos University Teaching Hospital
-
-
OYO State
-
Ibadan, OYO State, Nigeria, 200281
- Recruiting
- University College Hospital Paediatric Haematology and Oncology Unit
-
Ibadan, OYO State, Nigeria, 200212
- Recruiting
- College of Medicine, University of Ibadan
-
-
-
-
-
London, United Kingdom, SE1 7EH
- Recruiting
- Guy's and St Thomas' NHS Foundation Trust
-
London, United Kingdom, E1 1BB
- Recruiting
- Barts Health NHS Trust , The Royal London Hospital
-
London, United Kingdom, SE1 7EH
- Recruiting
- Guy's & St Thomas NHS Trust, St Thomas' Hospital
-
-
Greater London
-
London, Greater London, United Kingdom, NW1 2PG
- Recruiting
- University College Hospital NHS Foundation Trust
-
-
-
-
District of Columbia
-
Washington, District of Columbia, United States, 20010
- Active, not recruiting
- Children's National Medical Center
-
-
Georgia
-
Atlanta, Georgia, United States, 30342
- Active, not recruiting
- Children's Healthcare of Atlanta Scottish Rite
-
-
Illinois
-
Chicago, Illinois, United States, 60611
- Active, not recruiting
- Ann & Robert H. Lurie Children's Hospital of Chicago
-
-
Missouri
-
Kansas City, Missouri, United States, 64108
- Active, not recruiting
- Children's Mercy Hospital
-
-
North Carolina
-
Greenville, North Carolina, United States, 27834
- Recruiting
- ECU Physicians, Brody Outpatient Clinic
-
-
Pennsylvania
-
Pittsburgh, Pennsylvania, United States, 15224
- Active, not recruiting
- UPMC Children's Hospital of Pittsburgh
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Description
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 Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: 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.
|
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).
Other Names:
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
TEAEs and SAEs
Time Frame: Throughout entire study
|
Treatment Emergent Adverse Events and Serious Adverse Events
|
Throughout entire study
|
Sickle Cell Disease-Related Complications
Time Frame: Throughout entire study
|
Frequency of SCD-related complications
|
Throughout entire study
|
Collaborators and Investigators
Publications and helpful links
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Estimated)
Study Completion (Estimated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- GBT440-038
- C5341023 (Other Identifier: Alias Study Number)
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
IPD Plan Description
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Sickle Cell Disease
-
Klein Buendel, Inc.National Institute on Minority Health and Health Disparities (NIMHD); Hilton...CompletedSickle Cell Disease | Sickle Cell Anemia in Children | Sickle Cell Thalassemia | Sickle Cell SC DiseaseUnited States
-
Nova Laboratories LimitedCompletedSickle Cell Disease | Sickle Cell Hemoglobin C | Sickle Cell-beta-thalassemia | Sickle-Cell; Hemoglobin Disease, ThalassemiaUnited Kingdom, Jamaica
-
SangartCompletedSickle Cell Disease | Anemia, Sickle Cell | Sickle Cell Anemia | Hemoglobin SC Disease | Sickle Cell Disorders | Sickle Cell Hemoglobin C DiseaseUnited Kingdom, France, Jamaica, Lebanon
-
SangartWithdrawnSickle Cell Disease | Anemia, Sickle Cell | Sickle Cell Anemia | Hemoglobin SC Disease | Sickle Cell Disorders | Sickle Cell Hemoglobin C DiseaseFrance, United Kingdom, Netherlands, Turkey, Bahrain, Belgium, Brazil, Lebanon, Qatar
-
University of British ColumbiaCompletedSickle Cell Disease | Beta-Thalassemia | Sickle Cell Trait | Sickle Cell-Beta Thalassemia | Sickle Cell-SS DiseaseCanada, Nepal
-
Sidney Kimmel Cancer Center at Thomas Jefferson...National Heart, Lung, and Blood Institute (NHLBI)TerminatedSickle Cell Anemia | Sickle Cell-hemoglobin C Disease | Sickle Cell-β0-thalassemiaUnited States
-
University of RegensburgRecruitingSickle Cell Disease | Sickle Cell Anemia | Sickle Cell Disorders | HbS Disease | Hemoglobin S Disease | Sickling Disorder Due to Hemoglobin SGermany, Austria
-
Centre Hospitalier Intercommunal CreteilRecruitingSickle-Cell Disease Nos With CrisisFrance
-
HemaQuest Pharmaceuticals Inc.TerminatedSickle Cell Disease | Sickle Cell Anemia | Sickle Cell Disorders | Hemoglobin S Disease | Sickling Disorder Due to Hemoglobin SUnited States, Lebanon, Egypt, Canada, Jamaica
-
HemaQuest Pharmaceuticals Inc.CompletedSickle Cell Disease | Sickle Cell Anemia | Sickle Cell Disorders | Hemoglobin S Disease | Sickling Disorder Due to Hemoglobin SUnited States, Lebanon, Canada, Egypt, Jamaica
Clinical Trials on Voxelotor
-
PfizerPfizerRecruitingSickle Cell DiseaseUnited States
-
University of Illinois at ChicagoGlobal Blood TherapeuticsRecruitingSickle Cell Disease | Sickle Cell NephropathyUnited States
-
PfizerPfizerActive, not recruitingSickle Cell DiseaseUnited States, Lebanon, United Kingdom
-
PfizerPfizerCompletedSickle Cell DiseaseUnited States
-
Amy TangPfizerRecruitingSickle Cell Anemia in ChildrenUnited States
-
PfizerActive, not recruitingSickle Cell DiseaseUnited States, Oman, United Kingdom, France, Egypt, Ghana, Italy, Kenya, Nigeria, Saudi Arabia
-
PfizerNo longer availableSickle Cell DiseaseUnited States
-
PfizerTerminatedSickle Cell DiseaseUnited Kingdom
-
Elizabeth Yang, MD, PhDUniversity of California, San Francisco; Global Blood Therapeutics; PediatrixCompleted
-
Inova Health Care ServicesPfizerRecruitingSickle Cell DiseaseUnited States