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No Longer Available No phase listed Expanded access

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

ClinicalTrials.gov ID: NCT04724421

Public ClinicalTrials.gov record NCT04724421. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.

ClinicalTrials.gov public records Last synced Sep 2, 2026, 9:38 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Brief summary

Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.

The objective of this Expanded Access Protocol (EAP) is to provide voxelotor for the treatment of sickle cell disease (SCD): The study GBT440-041 is the EAP for pediatric patients aged 6 months to 11 years who have no alternative treatment options and are ineligible to participate in a clinical trial of voxelotor and the study C5341057 is the EAP for adults/adolescents 12 years of age and older for patients who cannot satisfactorily be treated with an authorized medicinal product

Study identification

NCT ID
NCT04724421
Recruitment status
No Longer Available
Study type
Expanded access
Phase
Not listed
Lead sponsor
Pfizer
Industry
Enrollment
Not listed

Conditions and interventions

Interventions

Drug

Eligibility (public fields only)

Age range
6 Months to 11 Years
Sex
All
Healthy volunteers
Healthy volunteers not accepted

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date
Not listed
Primary completion
Not listed
Completion
Not listed
Last update posted
Nov 19, 2024

United States locations

U.S. sites
33
U.S. states
16
U.S. cities
23
Facility City State ZIP Site status
Phoenix Children's Hospital Phoenix Arizona 85016
Rady Children's Hospital San Diego San Diego California 92123
Nemours/Alfred I. duPont Hospital for Children Wilmington Delaware 19803
Children's National Hospital / Children's National Health System Washington D.C. District of Columbia 20010
Children's National Medical Center Washington D.C. District of Columbia 20010
Baptist Medical Center/Wolfson Children's Hospital Jacksonville Florida 32207
Nemours Children's Health, Jacksonville Jacksonville Florida 32207
Nemours Children's Hospital Orlando Florida 32827
Children's Healthcare of Atlanta Scottish Rite Atlanta Georgia 30342
AU Medical Center Clinical Research Pharmacy Augusta Georgia 30912
Augusta University Augusta Georgia 30912
Children's Hospital of Georgia Augusta Georgia 30912
Memorial Health University Medical Center Savannah Georgia 31404
Tulane Lakeside Metairie Louisiana 70001
Tulane University Hospitals and Clinics New Orleans Louisiana 70112
Children's Hospital New Orleans Louisiana 70118
Childrens Hospital of NOLA New Orleans Louisiana 70118
University Of Michigan Hospitals Ann Arbor Michigan 48109
Rutgers Cancer Institute of New Jersey New Brunswick New Jersey 08901
Newark Beth Israel Medical Center & Children's Hospital of New Jersey Newark New Jersey 07112
BronxCare Health System The Bronx New York 10457
Jacobi Medical Center The Bronx New York 10461
East Carolina University Brody School of Medicine(ECU) Greenville North Carolina 27834
ECU Physicians, Brody Outpatient Clinic Greenville North Carolina 27834
University Hospitals Cleveland Medical Center Cleveland Ohio 44106
Medical University of South Carolina: Investigational Drug Services Pharmacy Charleston South Carolina 29425
Medical University of South Carolina: Shawn Jenkins Women's and Children's Hospital Charleston South Carolina 29425
Medical University of South Carolina Charleston South Carolina 29425
Prisma Health - Upstate Greenville South Carolina 29605
St. Jude Children's Research Hospital Memphis Tennessee 38105
Cook Children's Health Care System Fort Worth Texas 76104
Pediatric Specialists of Virginia (Inova Ashburn HealthPlex) Ashburn Virginia 20148
Pediatric Specialists of Virginia (Schar Cancer Institute) Fairfax Virginia 22031

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

Non-U.S. locations

This page focuses on the U.S. directory. The official record also lists 4 non-U.S. sites.

About this trial record page

What this page shows
Public field values for ClinicalTrials.gov record NCT04724421, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
What this page does not do
No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
Where the data comes from
Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
Last refresh
Last update posted Nov 19, 2024 · Synced Sep 2, 2026

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Open the official record

The complete protocol, eligibility criteria, and contact information for NCT04724421 live on ClinicalTrials.gov.

View official ClinicalTrials.gov record →