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


18 United States sites


> 12 Years

Study type

Expanded Access







About the study

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

participation requirements

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.

participation restrictions

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


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