- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04724421
Expanded Access Protocol for Adults and Pediatric Patients With Sickle Cell Disease Who Have No Alternative Treatment Options
Study Overview
Detailed Description
This EAP may continue until such time that voxelotor is commercially available or the Sponsor discontinues the voxelotor EAP.
Please note that for US sites, enrollment is open only for 6months to < 4 years of age.
Study Type
Expanded Access Type
- Treatment IND/Protocol
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
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Rio de Janeiro, Brazil, 20211-030
- Available
- HEMORIO - Hematologia Laboratorial
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São Paulo, Brazil, 01232-010
- Available
- ESHO Empresa de Serviços Hospitalares S.A/ Hospital Samaritano de Higienópolis
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Pernambuco
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Recife, Pernambuco, Brazil, 50070-460
- Available
- Multihemo Servicos Medicos S/A
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SAO Paulo
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Ribeirão Preto, SAO Paulo, Brazil, 14048-900
- Available
- Hospital Das Clínicas da Faculdade de Medicina de Ribeirão Preto - USP
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Arizona
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Phoenix, Arizona, United States, 85016
- Available
- Phoenix Children's Hospital
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Principal Investigator:
- Sanjay Shah, MD
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Contact:
- Erica Seiber
- Phone Number: 602-933-0171
- Email: esieber@phoenixchildrens.com
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California
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San Diego, California, United States, 92123
- Available
- Rady Children's Hospital San Diego
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Delaware
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Wilmington, Delaware, United States, 19803
- Available
- Nemours/Alfred I. DuPont Hospital for Children
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District of Columbia
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Washington, District of Columbia, United States, 20010
- Available
- Children's National Medical Center
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Contact:
- Stephanie Margulies
- Phone Number: 202-476-2281
- Email: Stefanie.Margulies@childrensnational.org
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Principal Investigator:
- Deepika Darbari, MD
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Washington, District of Columbia, United States, 20010
- Available
- Children's National Hospital / Children's National Health System
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Florida
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Jacksonville, Florida, United States, 32207
- Available
- Baptist Medical Center/Wolfson Children's Hospital
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Jacksonville, Florida, United States, 32207
- Available
- Nemours Children's Health, Jacksonville
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Orlando, Florida, United States, 32827
- Available
- Nemours Children's Hospital
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Contact:
- Erin Young
- Email: Erin.Young@nemours.org
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Contact:
- Krystle Sullivan
- Email: Krystle.Sullivan@nemours.org
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Principal Investigator:
- Dorothea Douglas, MD
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Georgia
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Atlanta, Georgia, United States, 30342
- Available
- Children's Healthcare of Atlanta Scottish Rite
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Augusta, Georgia, United States, 30912
- Available
- Augusta University
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Contact:
- Krystle Stone
- Phone Number: 706-721-3626
- Email: Kstone6@augusta.edu
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Principal Investigator:
- Nnenna Badamosi, MD
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Augusta, Georgia, United States, 30912
- Available
- AU Medical Center Clinical Research Pharmacy
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Augusta, Georgia, United States, 30912
- Available
- Children's Hospital of Georgia
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Savannah, Georgia, United States, 31404
- Available
- Memorial Health University Medical Center
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Contact:
- Theresa Ryan- Knapp, RN
- Phone Number: 912-350-5645
- Email: Theresa.ryan@hcahealthcare.com
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Principal Investigator:
- Andrew Pendelton, MD
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Louisiana
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Metairie, Louisiana, United States, 70001
- Available
- Tulane Lakeside
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New Orleans, Louisiana, United States, 70112
- Available
- Tulane University Hospitals and Clinics
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New Orleans, Louisiana, United States, 70118
- Available
- Children's Hospital
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New Orleans, Louisiana, United States, 70118
- Available
- Childrens Hospital of NOLA
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Michigan
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Ann Arbor, Michigan, United States, 48109
- Available
- University of Michigan Hospitals
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New Jersey
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New Brunswick, New Jersey, United States, 08901
- Available
- Rutgers Cancer Institute of New Jersey
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Newark, New Jersey, United States, 07112
- Available
- Newark Beth Israel Medical Center & Children's Hospital of New Jersey
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New York
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Bronx, New York, United States, 10457
- Available
- BronxCare Health System
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Contact:
- Diane Strome
- Phone Number: 718-960-1020
- Email: DStrom@bronxleb.org
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Contact:
- Ana Maria Emeh
- Phone Number: 718-960-4498
- Email: anaemeh@bronxleb.org
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Principal Investigator:
- Howard Grodman, MD
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Bronx, New York, United States, 10461
- Available
- Jacobi Medical Center
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North Carolina
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Greenville, North Carolina, United States, 27834
- Available
- East Carolina University Brody School of Medicine(ECU)
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Greenville, North Carolina, United States, 27834
- Available
- ECU Physicians, Brody Outpatient Clinic
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Ohio
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Cleveland, Ohio, United States, 44106
- Available
- University Hospitals Cleveland Medical Center
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South Carolina
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Charleston, South Carolina, United States, 29425
- Available
- Medical University of South Carolina
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Contact:
- Earleisha Mack
- Phone Number: 843-792-2957
- Email: feldere@musc.edu
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Principal Investigator:
- Shayla Bergmann, MD
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Charleston, South Carolina, United States, 29425
- Available
- Medical University of South Carolina: Investigational Drug Services Pharmacy
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Charleston, South Carolina, United States, 29425
- Available
- Medical University of South Carolina: Shawn Jenkins Women's and Children's Hospital
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Greenville, South Carolina, United States, 29605
- Available
- Prisma Health - Upstate
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Tennessee
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Memphis, Tennessee, United States, 38105
- Available
- St. Jude Children's Research Hospital
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Contact:
- Jeremie Estepp, MD
- Phone Number: 866-278-5833
- Email: Jeremie.estepp@stjude.org
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Principal Investigator:
- Jeremie Estepp, MD
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Texas
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Fort Worth, Texas, United States, 76104
- Available
- Cook Children's Health Care System
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Virginia
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Ashburn, Virginia, United States, 20148
- Available
- Pediatric Specialists of Virginia (Inova Ashburn HealthPlex)
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Fairfax, Virginia, United States, 22031
- Available
- Pediatric Specialists of Virginia (Schar Cancer Institute)
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Description
Inclusion Criteria: GBT440-041
- Documented diagnosis of sickle cell disease of any genotype
- Ineligible or unable to participate in actively recruiting clinical studies of voxelotor
- Baseline hemoglobin (Hb) ≤10.5 g/dL
- No alternative treatment options in the judgement of the treating Investigator
- Participants who, if female of childbearing potential (post-menarche), and are sexually active, agree to use highly effective methods of contraception from study start to 30 days after the last dose of voxelotor
- Written informed parental/guardian consent and participant assent (if applicable) has been obtained per Institutional Review Board (IRB) policy and requirements, consistent with International Council for Harmonisation (ICH) guidelines
Exclusion Criteria: GBT440-041
- Receiving chronic red blood cell (RBC) transfusion therapy for primary or secondary stroke prevention
- Hepatic dysfunction characterized by alanine aminotransferase (ALT) >4 × the upper limit of normal (ULN) for age
- Severe renal dysfunction (estimated glomerular filtration rate [eGFR] <30 mL/min/1.73 m2 by Schwartz formula)
Clinically significant bacterial, fungal, parasitic, or viral infection that requires therapy:
- Patients with acute bacterial infection requiring antibiotic use should delay screening/enrollment until the course of antibiotic therapy has been completed.
- Patients with known active hepatitis A, B, or C or who are known to be Human Immunodeficiency Virus (HIV) positive
- Any condition affecting drug absorption, such as major surgery involving the stomach or small intestine (prior cholecystectomy is acceptable)
- Female who is pregnant or breastfeeding
- Participated in another clinical trial of an investigational drug or medical device, within 30 days or 5 half-lives of the investigational drug (whichever is longer) prior to informed consent for the EAP, or is currently participating in another trial of an investigational drug or medical device
- Medical, psychological, or behavioral conditions, that, in the opinion of the Investigator, may preclude informed consent, safe participation, or compliance with the protocol procedures
- Use of herbal medications (eg, St. John's wort), sensitive cytochrome P450 (CYP) 3A4 substrates with a narrow therapeutic index, or strong CYP3A4 inducers
- Active symptomatic COVID-19 infection
Inclusion Criteria: C5341057
- Documented diagnosis of SCD of any genotype confirmed by laboratory genetic testing
- Aged 12 years and older
- Haemoglobin ≥5.5 and ≤ 10.5 g/dL, based on a test performed according to local standard of care
- Patient cannot be treated satisfactorily with any authorised medicinal product
- Patient or their legal representative has provided written informed consent to participate in the voxelotor EAP for the treatment of SCD
- Patient or their legal representative has provided written informed consent on data processing and protection
Exclusion Criteria: C5341057
- Patients with clinically significant bacterial, fungal, parasitic or viral infection which requires therapy.
- History of serious drug hypersensitivity reaction to voxelotor or excipients
- Participated in another clinical trial of an investigational agent (or medical device) within 30 days of participation in EAP.
- Medical, psychological, or behavioral condition that, in the opinion of the study doctor, would confound or interfere with evaluation of safety and/or effectiveness of the study drug, prevent compliance with the study protocol; preclude informed consent; or render the participant unable/unlikely to comply with the study procedures
Study Plan
How is the study designed?
Collaborators and Investigators
Publications and helpful links
Study record dates
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
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- C534 Expanded Access
- GBT440-041 (Other Identifier: Pfizer)
- C5341025 (Other Identifier: Alias Study Number)
- C5341057 (Other Identifier: Pfizer)
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.
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