Expanded Access Protocol for Adults and Pediatric Patients With Sickle Cell Disease Who Have No Alternative Treatment Options

The objective of this Expanded Access Protocol (EAP) is to provide voxelotor for the treatment of sickle cell disease (SCD): The study GBT440-041 is the EAP for pediatric patients aged 6 months to 11 years who have no alternative treatment options and are ineligible to participate in a clinical trial of voxelotor and the study C5341057 is the EAP for adults/adolescents 12 years of age and older for patients who cannot satisfactorily be treated with an authorized medicinal product

Study Overview

• Status: No longer available
• Conditions: Sickle Cell Disease
• Intervention / Treatment: Drug: Voxelotor

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
• Expanded Access Type: Treatment IND/Protocol

Contacts and Locations

Study Locations

• Brazil
  • Rio de Janeiro, Brazil, 20211-030
    • HEMORIO - Hematologia Laboratorial
  • São Paulo, Brazil, 01232-010
    • ESHO Empresa de Serviços Hospitalares S.A/ Hospital Samaritano de Higienópolis
  • Pernambuco
    • Recife, Pernambuco, Brazil, 50070-460
      • Multihemo Servicos Medicos S/A
  • São Paulo
    • Ribeirão Preto, São Paulo, Brazil, 14048-900
      • Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto - USP
• United States
  • Arizona
    • Phoenix, Arizona, United States, 85016
      • Phoenix Children's Hospital
  • California
    • San Diego, California, United States, 92123
      • Rady Children's Hospital San Diego
  • Delaware
    • Wilmington, Delaware, United States, 19803
      • Nemours/Alfred I. duPont Hospital for Children
  • District of Columbia
    • Washington D.C., District of Columbia, United States, 20010
      • Children's National Medical Center
    • Washington D.C., District of Columbia, United States, 20010
      • Children's National Hospital / Children's National Health System
  • Florida
    • Jacksonville, Florida, United States, 32207
      • Baptist Medical Center/Wolfson Children's Hospital
    • Jacksonville, Florida, United States, 32207
      • Nemours Children's Health, Jacksonville
    • Orlando, Florida, United States, 32827
      • Nemours Children's Hospital
  • Georgia
    • Atlanta, Georgia, United States, 30342
      • Children's Healthcare of Atlanta Scottish Rite
    • Augusta, Georgia, United States, 30912
      • Augusta University
    • Augusta, Georgia, United States, 30912
      • Children's Hospital of Georgia
    • Augusta, Georgia, United States, 30912
      • AU Medical Center Clinical Research Pharmacy
    • Savannah, Georgia, United States, 31404
      • Memorial Health University Medical Center
  • Louisiana
    • Metairie, Louisiana, United States, 70001
      • Tulane Lakeside
    • New Orleans, Louisiana, United States, 70118
      • Children's Hospital
    • New Orleans, Louisiana, United States, 70112
      • Tulane University Hospitals and Clinics
    • New Orleans, Louisiana, United States, 70118
      • Childrens Hospital of NOLA
  • Michigan
    • Ann Arbor, Michigan, United States, 48109
      • University of Michigan Hospitals
  • New Jersey
    • New Brunswick, New Jersey, United States, 08901
      • Rutgers Cancer Institute of New Jersey
    • Newark, New Jersey, United States, 07112
      • Newark Beth Israel Medical Center & Children's Hospital of New Jersey
  • New York
    • The Bronx, New York, United States, 10461
      • Jacobi Medical Center
    • The Bronx, New York, United States, 10457
      • BronxCare Health System
  • North Carolina
    • Greenville, North Carolina, United States, 27834
      • East Carolina University Brody School of Medicine(ECU)
    • Greenville, North Carolina, United States, 27834
      • ECU Physicians, Brody Outpatient Clinic
  • Ohio
    • Cleveland, Ohio, United States, 44106
      • University Hospitals Cleveland Medical Center
  • South Carolina
    • Charleston, South Carolina, United States, 29425
      • Medical University of South Carolina
    • Charleston, South Carolina, United States, 29425
      • Medical University of South Carolina: Shawn Jenkins Women's and Children's Hospital
    • Charleston, South Carolina, United States, 29425
      • Medical University of South Carolina: Investigational Drug Services Pharmacy
    • Greenville, South Carolina, United States, 29605
      • Prisma Health - Upstate
  • Tennessee
    • Memphis, Tennessee, United States, 38105
      • St. Jude Children's Research Hospital
  • Texas
    • Fort Worth, Texas, United States, 76104
      • Cook Children's Health Care System
  • Virginia
    • Ashburn, Virginia, United States, 20148
      • Pediatric Specialists of Virginia (Inova Ashburn HealthPlex)
    • Fairfax, Virginia, United States, 22031
      • Pediatric Specialists of Virginia (Schar Cancer Institute)

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 4 years to 11 years (Child)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria: GBT440-041

1. Documented diagnosis of sickle cell disease of any genotype
2. Ineligible or unable to participate in actively recruiting clinical studies of voxelotor
3. Baseline hemoglobin (Hb) ≤10.5 g/dL
4. No alternative treatment options in the judgement of the treating Investigator
5. 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
6. 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

1. Receiving chronic red blood cell (RBC) transfusion therapy for primary or secondary stroke prevention
2. Hepatic dysfunction characterized by alanine aminotransferase (ALT) >4 × the upper limit of normal (ULN) for age
3. Severe renal dysfunction (estimated glomerular filtration rate [eGFR] <30 mL/min/1.73 m2 by Schwartz formula)

4. Clinically significant bacterial, fungal, parasitic, or viral infection that requires therapy:

   1. Patients with acute bacterial infection requiring antibiotic use should delay screening/enrollment until the course of antibiotic therapy has been completed.
   2. Patients with known active hepatitis A, B, or C or who are known to be Human Immunodeficiency Virus (HIV) positive
5. Any condition affecting drug absorption, such as major surgery involving the stomach or small intestine (prior cholecystectomy is acceptable)
6. Female who is pregnant or breastfeeding
7. 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
8. Medical, psychological, or behavioral conditions, that, in the opinion of the Investigator, may preclude informed consent, safe participation, or compliance with the protocol procedures
9. Use of herbal medications (eg, St. John's wort), sensitive cytochrome P450 (CYP) 3A4 substrates with a narrow therapeutic index, or strong CYP3A4 inducers
10. Active symptomatic COVID-19 infection

Inclusion Criteria: C5341057

1. Documented diagnosis of SCD of any genotype confirmed by laboratory genetic testing
2. Aged 12 years and older
3. Haemoglobin ≥5.5 and ≤ 10.5 g/dL, based on a test performed according to local standard of care
4. Patient cannot be treated satisfactorily with any authorised medicinal product
5. Patient or their legal representative has provided written informed consent to participate in the voxelotor EAP for the treatment of SCD
6. Patient or their legal representative has provided written informed consent on data processing and protection

Exclusion Criteria: C5341057

1. Patients with clinically significant bacterial, fungal, parasitic or viral infection which requires therapy.
2. History of serious drug hypersensitivity reaction to voxelotor or excipients
3. Participated in another clinical trial of an investigational agent (or medical device) within 30 days of participation in EAP.
4. 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

• Sponsor: Pfizer
• Collaborators: Pfizer
• Investigators: Study Director: Pfizer CT.gov Call Center, Pfizer

Publications and helpful links

Helpful Links

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

Study record dates

Study Registration Dates

• First Submitted: January 21, 2021
• First Submitted That Met QC Criteria: January 21, 2021
• First Posted (Actual): January 26, 2021

Study Record Updates

• Last Update Posted (Actual): November 20, 2024
• Last Update Submitted That Met QC Criteria: November 15, 2024
• Last Verified: November 1, 2024

More Information

Terms related to this study

• Keywords: Voxelotor
• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Hematologic Diseases; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hemoglobinopathies; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Hemic and Lymphatic Diseases; Anemia, Sickle Cell; voxelotor
• Other Study ID Numbers: C534 Expanded Access; GBT440-041 (Other Identifier: Pfizer); C5341025 (Other Identifier: Alias Study Number); C5341057 (Other Identifier: Pfizer)