Expanded Access to Voxelotor for Patients With Sickle Cell Disease Who Have No Alternative Treatment Options

An Open-Label, Expanded Access Protocol for Patients With Sickle Cell Disease Who Have No Alternative Treatment Options

The intent of this open-label, multicenter expanded access program (EAP) is to provide early access to voxelotor prior to market authorization

Study Overview

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

Detailed Description

The purpose of this program is to provide early access to voxelotor for patients with sickle cell disease who have exhausted alternative treatment options and are ineligible for actively-recruiting clinical trials of voxelotor.

This EAP may continue until such time that voxelotor is commercially available or the Sponsor discontinues the voxelotor EAP. Discontinuation may occur earlier if an individual participant or physician or Sponsor Medical Monitor decides to discontinue treatment.

• Study Type: Expanded Access
• Expanded Access Type: Individual Patients

Contacts and Locations

Study Locations

• United States
  • Alabama
    • Birmingham, Alabama, United States, 35211
      • Alabama Oncology
    • Mobile, Alabama, United States, 36693
      • University of South Alabama
  • Georgia
    • Atlanta, Georgia, United States, 30342
      • Children's Healthcare of Atlanta
    • Augusta, Georgia, United States, 30912
      • Augusta University
  • Louisiana
    • Baton Rouge, Louisiana, United States, 70809
      • Our Lady of the Lake Physician Group
    • New Orleans, Louisiana, United States, 70112
      • University Medical Center New Orleans
  • Maryland
    • Baltimore, Maryland, United States, 21287
      • The John Hopkins Hospital
  • New Jersey
    • New Brunswick, New Jersey, United States, 08901-1914
      • Rutgers Cancer Institute of New Jersey
  • New York
    • Jamaica, New York, United States, 11432
      • Queens Hospital Center
    • The Bronx, New York, United States, 10461
      • Montefiore Medical Center Prime
  • North Carolina
    • Chapel Hill, North Carolina, United States, 27514
      • University of North Carolina At Chapel Hill
    • Durham, North Carolina, United States, 27710
      • Duke Department of Pediatrics
  • Ohio
    • Columbus, Ohio, United States, 43210
      • The Ohio State University Wexner Medical Center
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19107
      • Thomas Jefferson University Hospital
    • Pittsburgh, Pennsylvania, United States, 15232
      • University of Pittsburgh Medical Center Health System
  • Texas
    • Fort Worth, Texas, United States, 76104
      • Cook Children's Medical Center
    • Houston, Texas, United States, 77030
      • Texas Children's Hospital
  • Virginia
    • Richmond, Virginia, United States, 23298
      • Vcu Health

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 12 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria

1. Documented diagnosis of sickle cell disease of any genotype confirmed by laboratory testing during screening
2. Ineligible or unable to participate in actively recruiting clinical studies of voxelotor
3. Hemoglobin (Hb) ≤ 10.5 g/dL during screening
4. No alternative treatment options in the judgment of the treating investigator
5. Participants, who if female and of child bearing potential, agree to use highly effective methods of contraception from study start to 30 days after the last dose of voxelotor, and who if male, agree to use barrier methods of contraception from study start to 30 days after the last dose of voxelotor
6. Patient has provided documented informed consent (the informed consent form [ICF] must be reviewed and signed by each participant),or for participants under 18 years of age, signed parental/legal guardian consent and participant assent.

Exclusion Criteria

1. Female who is breast feeding or pregnant
2. Hepatic dysfunction characterized by alanine aminotransferase (ALT) >4 × ULN

3. Patients with clinically significant bacterial, fungal, parasitic, or viral infection which require 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
4. Renal dialysis
5. Non-SCD conditions or complications of SCD for which survival beyond 3 months is unlikely, in the opinion of the treating Investigator
6. Any condition affecting drug absorption, such as major surgery involving the stomach or small intestine (prior cholecystectomy is acceptable)
7. Participated in another clinical trial of an investigational agent (or medical device) within 30 days or 5 half-lives of date of informed consent, whichever is longer, or is currently participating in another trial of an investigational agent (or medical device)
8. Medical, psychological, or behavioral conditions, which, in the opinion of the Investigator, may preclude safe participation, interfere with compliance or preclude informed consent

The Sponsor Medical Monitor makes the sole and final enrollment determination for any patient.

Study Plan

How is the study designed?

Collaborators and Investigators

• Sponsor: 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: May 7, 2019
• First Submitted That Met QC Criteria: May 7, 2019
• First Posted (Actual): May 9, 2019

Study Record Updates

• Last Update Posted (Actual): July 18, 2023
• Last Update Submitted That Met QC Criteria: July 14, 2023
• Last Verified: July 1, 2023

More Information

Terms related to this study

• 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: GBT440-037; C5341044 (Other Identifier: Alias Study Number)