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

Study point of contact

Michelle Xu, MD
[email protected]
Julia Martinisi
[email protected]


12 United States sites


4 to 11 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 treatment with voxelotor prior to market authorization for pediatric patients age 4
to 11 years with sickle cell disease (SCD) who have no alternative treatment options and are
ineligible to participate in clinical trials of voxelotor.

participation requirements

1. Documented diagnosis of sickle cell disease of any genotype

2. Ineligible or unable to participate in actively recruiting clinical studies of

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

participation restrictions

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 product (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 product (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, strong CYP3A4 inhibitors, fluconazole, or moderate or strong CYP3A4 inducers


  • Phoenix, Arizona, United States, Phoenix Children's Hospital, 85016 [Available]
  • Washington, District of Columbia, United States, Children's National Medical Center, 20010 [Available]
  • Atlanta, Georgia, United States, Children's Healthcare of Atlanta, 30322 [Available]
  • Augusta, Georgia, United States, Augusta University, 30912 [Available]
  • Savannah, Georgia, United States, Memorial Health University Medical Center, 31404 [Available]
  • New Orleans, Louisiana, United States, Children's National Medical Center, 20010 [Available]
  • Newark, New Jersey, United States, Newark Beth Israel Medical Center, 07112 [Available]
  • Bronx, New York, United States, NYC Health and Hospitals, 10461 [Available]
  • Cleveland, Ohio, United States, UH Rainbow Babies & Children's Hospital, 44106 [Available]
  • Charleston, South Carolina, United States, Medical University of South Carolina, 29425 [Available]
  • Greenville, South Carolina, United States, Prisma Health Upstate, 29605 [Available]
  • Fort Worth, Texas, United States, Cook Children's Medical Center, 76104 [Available]