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Expanded Access Protocol for Pediatric Patients With Sickle Cell Disease Who Have No Alternative Treatment Options

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT04724421
Expanded Access Status : Available
First Posted : January 26, 2021
Last Update Posted : December 29, 2021
Information provided by (Responsible Party):
Global Blood Therapeutics

Brief Summary:
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.

Condition or disease Intervention/treatment
Sickle Cell Disease Drug: Voxelotor

Detailed Description:

Pediatric patients who are eligible for participation in this EAP will be treated with voxelotor, dispersible tablets or powder for oral suspension administered orally once daily (QD) at a weight-based dose, and followed by the treating physicians at the participating sites. Participants will receive standard of care treatment and procedures for management of SCD, including an initial visit and routine visits at least every 12 weeks (± 7 days) for clinical and laboratory assessments per standard of care and re-supply of voxelotor. A safety follow-up visit will be conducted 28 days (± 7 days) after the last dose of investigational product.

This EAP may continue until such time that voxelotor is commercially available for patients age 4 to 11 years, or the Sponsor discontinues the voxelotor EAP.

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Study Type : Expanded Access
Expanded Access Type : Treatment IND/Protocol
  See clinical trials of the intervention/treatment in this expanded access record.
Official Title: An Open-label, Expanded Access Protocol for Pediatric Patients With Sickle Cell Disease Who Have No Alternative Treatment Options

Intervention Details:
  • Drug: Voxelotor
    synthetic small molecule
    Other Name: GBT440

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   4 Years to 11 Years   (Child)
Sexes Eligible for Study:   All

Inclusion Criteria:

  1. Documented diagnosis of sickle cell disease of any genotype
  2. Ineligible or unable to participate in actively recruiting clinical studies of voxelotor
  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

Exclusion Criteria:

  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 drug or medical device, within 30 days or 5 half-lives of the investigational drug (whichever is longer) prior to informed consent for the EAP, or is currently participating in another trial of an investigational drug 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, or strong CYP3A4 inducers
  10. Active symptomatic COVID-19 infection

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT04724421

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Contact: Michelle Xu, MD 650-534-2574

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Sponsors and Collaborators
Global Blood Therapeutics
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Responsible Party: Global Blood Therapeutics Identifier: NCT04724421    
Other Study ID Numbers: GBT440-041
First Posted: January 26, 2021    Key Record Dates
Last Update Posted: December 29, 2021
Last Verified: December 2021
Additional relevant MeSH terms:
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Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hematologic Diseases
Genetic Diseases, Inborn