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
2 other identifiers
expanded_access
N/A
1 country
18
Brief Summary
The intent of this open-label, multicenter expanded access program (EAP) is to provide early access to voxelotor prior to market authorization
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
18 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
May 7, 2019
CompletedFirst Posted
Study publicly available on registry
May 9, 2019
CompletedJuly 18, 2023
July 1, 2023
May 7, 2019
July 14, 2023
Conditions
Interventions
Eligibility Criteria
You may qualify if:
- Documented diagnosis of sickle cell disease of any genotype confirmed by laboratory testing during screening
- Ineligible or unable to participate in actively recruiting clinical studies of voxelotor
- Hemoglobin (Hb) ≤ 10.5 g/dL during screening
- No alternative treatment options in the judgment of the treating investigator
- 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
- 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.
You may not qualify if:
- Female who is breast feeding or pregnant
- Hepatic dysfunction characterized by alanine aminotransferase (ALT) \>4 × ULN
- 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
- Renal dialysis
- Non-SCD conditions or complications of SCD for which survival beyond 3 months is unlikely, in the opinion of the treating Investigator
- Any condition affecting drug absorption, such as major surgery involving the stomach or small intestine (prior cholecystectomy is acceptable)
- 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)
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Pfizerlead
Study Sites (18)
Alabama Oncology
Birmingham, Alabama, 35211, United States
University of South Alabama
Mobile, Alabama, 36693, United States
Children's Healthcare of Atlanta
Atlanta, Georgia, 30342, United States
Augusta University
Augusta, Georgia, 30912, United States
Our Lady of the Lake Physician Group
Baton Rouge, Louisiana, 70809, United States
University Medical Center New Orleans
New Orleans, Louisiana, 70112, United States
The John Hopkins Hospital
Baltimore, Maryland, 21287, United States
Rutgers Cancer Institute of New Jersey
New Brunswick, New Jersey, 08901-1914, United States
Queens Hospital Center
Jamaica, New York, 11432, United States
Montefiore Medical Center PRIME
The Bronx, New York, 10461, United States
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27514, United States
Duke Department of Pediatrics
Durham, North Carolina, 27710, United States
The Ohio State University Wexner Medical Center
Columbus, Ohio, 43210, United States
Thomas Jefferson University Hospital
Philadelphia, Pennsylvania, 19107, United States
University of Pittsburgh Medical Center Health System
Pittsburgh, Pennsylvania, 15232, United States
Cook Children's Medical Center
Fort Worth, Texas, 76104, United States
Texas Children's Hospital
Houston, Texas, 77030, United States
VCU Health
Richmond, Virginia, 23298, United States
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Pfizer CT.gov Call Center
Pfizer
Study Design
- Study Type
- expanded access
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
May 7, 2019
First Posted
May 9, 2019
Last Updated
July 18, 2023
Record last verified: 2023-07