Clincosm LogoSign in Get a demo

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

Sponsor
Global Blood Therapeutics (Industry)
Overall Status
Available
CT.gov ID
NCT04724421
Collaborator
(none)
20
Locations

Study Details

Study Description

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 Phase

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.

Study Design

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

Outcome Measures

Primary Outcome Measures

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    4 Years to 11 Years
    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:

    5. Patients with acute bacterial infection requiring antibiotic use should delay screening/enrollment until the course of antibiotic therapy has been completed.

    6. Patients with known active hepatitis A, B, or C or who are known to be Human Immunodeficiency Virus (HIV) positive

    7. Any condition affecting drug absorption, such as major surgery involving the stomach or small intestine (prior cholecystectomy is acceptable)

    8. Female who is pregnant or breastfeeding

    9. 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

    10. Medical, psychological, or behavioral conditions, that, in the opinion of the Investigator, may preclude informed consent, safe participation, or compliance with the protocol procedures

    11. Use of herbal medications (eg, St. John's wort), sensitive cytochrome P450 (CYP) 3A4 substrates with a narrow therapeutic index, or strong CYP3A4 inducers

    12. Active symptomatic COVID-19 infection

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Phoenix Children's Hospital Phoenix Arizona United States 85016
    2 Rady's Children Hospital San Diego California United States 92123
    3 Nemours Alfred duPont Hospital for Children Wilmington Delaware United States 19803
    4 Children's National Medical Center Washington District of Columbia United States 20010
    5 Nemours Children's Specialty Care Jacksonville Florida United States 32207
    6 Nemours Children's Hospital Orlando Florida United States 32827
    7 Children's Healthcare of Atlanta Atlanta Georgia United States 30322
    8 Augusta University Augusta Georgia United States 30912
    9 Memorial Health University Medical Center Savannah Georgia United States 31404
    10 Tulane University School of Medicine-Sickle Cell Center of Southern Louisiana New Orleans Louisiana United States 20010
    11 University of Michigan Ann Arbor Michigan United States 48109
    12 Newark Beth Israel Medical Center Newark New Jersey United States 07112
    13 BronxCare Health System Bronx New York United States 10457
    14 NYC Health and Hospitals Bronx New York United States 10461
    15 UH Rainbow Babies & Children's Hospital Cleveland Ohio United States 44106
    16 Medical University of South Carolina Charleston South Carolina United States 29425
    17 Prisma Health Upstate Greenville South Carolina United States 29605
    18 St. Jude Children's Research Hospital Memphis Tennessee United States 38105
    19 Cook Children's Medical Center Fort Worth Texas United States 76104
    20 Pediatric Specialists of Virginia Fairfax Virginia United States 22031

    Sponsors and Collaborators

    • Global Blood Therapeutics

    Investigators

    None specified.

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Global Blood Therapeutics
    ClinicalTrials.gov Identifier:
    NCT04724421
    Other Study ID Numbers:
    • GBT440-041
    First Posted:
    Jan 26, 2021
    Last Update Posted:
    Dec 29, 2021
    Last Verified:
    Dec 1, 2021
    Additional relevant MeSH terms:
    Anemia, Sickle Cell

    Study Results

    No Results Posted as of Dec 29, 2021