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

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

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.

No longer available
Individual Patients
Sickle Cell Disease

Drug: Voxelotor

Other Name: GBT440

Eligibility Criteria

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

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

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

Eligibility Gender
Eligibility Age
Minimum: 12 Years ~ Maximum: N/A
United States

Alabama Oncology
Birmingham, Alabama, United States

University of South Alabama
Mobile, Alabama, United States

Children's Healthcare of Atlanta
Atlanta, Georgia, United States

Augusta University
Augusta, Georgia, United States

Our Lady of the Lake Physician Group
Baton Rouge, Louisiana, United States

University Medical Center New Orleans
New Orleans, Louisiana, United States

The John Hopkins Hospital
Baltimore, Maryland, United States

Rutgers Cancer Institute of New Jersey
New Brunswick, New Jersey, United States

Montefiore Medical Center PRIME
Bronx, New York, United States

Queens Hospital Center
Jamaica, New York, United States

University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, United States

Duke Department of Pediatrics
Durham, North Carolina, United States

The Ohio State University Wexner Medical Center
Columbus, Ohio, United States

Thomas Jefferson University Hospital
Philadelphia, Pennsylvania, United States

University of Pittsburgh Medical Center Health System
Pittsburgh, Pennsylvania, United States

Cook Children's Medical Center
Fort Worth, Texas, United States

Texas Children's Hospital
Houston, Texas, United States

VCU Health
Richmond, Virginia, United States

Pfizer Call Center, Study Director

NCT Number
MeSH Terms
Anemia, Sickle Cell