Studies
| Study First Submitted Date | 2021-01-21 |
| Study First Posted Date | 2021-01-26 |
| Last Update Posted Date | 2023-08-02 |
| Verification Month Year | July 2023 |
| Verification Date | 2023-07-31 |
| Last Update Posted Date | 2023-08-02 |
Detailed Descriptions
| Sequence: | 20664475 |
| Description | Pediatric patients who are eligible for participation in this EAP will be treated with voxelotor, dispersible tablets or powder for oral suspension (Cohort A only) 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 aged 6 months to 11 years, or the Sponsor discontinues the voxelotor EAP. |
Facilities
| Sequence: | 199462137 | Sequence: | 199462138 | Sequence: | 199462139 | Sequence: | 199462140 | Sequence: | 199462141 | Sequence: | 199462142 | Sequence: | 199462143 | Sequence: | 199462144 | Sequence: | 199462145 | Sequence: | 199462146 | Sequence: | 199462147 | Sequence: | 199462148 | Sequence: | 199462149 | Sequence: | 199462150 | Sequence: | 199462151 | Sequence: | 199462152 | Sequence: | 199462153 | Sequence: | 199462154 | Sequence: | 199462155 | Sequence: | 199462156 | Sequence: | 199462157 | Sequence: | 199462158 | Sequence: | 199462159 | Sequence: | 199462160 | Sequence: | 199462161 | Sequence: | 199462162 | Sequence: | 199462163 | Sequence: | 199462164 | Sequence: | 199462165 | Sequence: | 199462166 |
| Status | Available | Status | Available | Status | Available | Status | Available | Status | Available | Status | Available | Status | Available | Status | Available | Status | Available | Status | Available | Status | Available | Status | Available | Status | Available | Status | Available | Status | Available | Status | Available | Status | Available | Status | Available | Status | Available | Status | Available | Status | Available | Status | Available | Status | Available | Status | Available | Status | Available | Status | Available | Status | Available | Status | Available | Status | Available | Status | Available |
| Name | Phoenix Children's Hospital | Name | Rady Children's Hospital San Diego | Name | Nemours/Alfred I. duPont Hospital for Children | Name | Children's National Hospital / Children's National Health System | Name | Children's National Medical Center | Name | Baptist Medical Center/Wolfson Children's Hospital | Name | Nemours Children's Health, Jacksonville | Name | Nemours Children's Hospital | Name | Children's Healthcare of Atlanta Scottish Rite | Name | AU Medical Center Clinical Research Pharmacy | Name | Augusta University | Name | Children's Hospital of Georgia | Name | Memorial Health University Medical Center | Name | Tulane Lakeside | Name | Tulane University Hospitals and Clinics | Name | Children's Hospital | Name | Childrens Hospital of NOLA | Name | University Of Michigan Hospitals | Name | Newark Beth Israel Medical Center & Children's Hospital of New Jersey | Name | BronxCare Health System | Name | Jacobi Medical Center | Name | East Carolina University Brody School of Medicine(ECU) | Name | University Hospitals Cleveland Medical Center | Name | Medical University of South Carolina: Investigational Drug Services Pharmacy | Name | Medical University of South Carolina | Name | Prisma Health – Upstate | Name | St. Jude Children's Research Hospital | Name | Cook Children's Health Care System | Name | Pediatric Specialists of Virginia (Inova Ashburn HealthPlex) | Name | Pediatric Specialists of Virginia (Schar Cancer Institute) |
| City | Phoenix | City | San Diego | City | Wilmington | City | Washington | City | Washington | City | Jacksonville | City | Jacksonville | City | Orlando | City | Atlanta | City | Augusta | City | Augusta | City | Augusta | City | Savannah | City | Metairie | City | New Orleans | City | New Orleans | City | New Orleans | City | Ann Arbor | City | Newark | City | Bronx | City | Bronx | City | Greenville | City | Cleveland | City | Charleston | City | Charleston | City | Greenville | City | Memphis | City | Fort Worth | City | Ashburn | City | Fairfax |
| State | Arizona | State | California | State | Delaware | State | District of Columbia | State | District of Columbia | State | Florida | State | Florida | State | Florida | State | Georgia | State | Georgia | State | Georgia | State | Georgia | State | Georgia | State | Louisiana | State | Louisiana | State | Louisiana | State | Louisiana | State | Michigan | State | New Jersey | State | New York | State | New York | State | North Carolina | State | Ohio | State | South Carolina | State | South Carolina | State | South Carolina | State | Tennessee | State | Texas | State | Virginia | State | Virginia |
| Zip | 85016 | Zip | 92123 | Zip | 19803 | Zip | 20010 | Zip | 20010 | Zip | 32207 | Zip | 32207 | Zip | 32827 | Zip | 30342 | Zip | 30912 | Zip | 30912 | Zip | 30912 | Zip | 31404 | Zip | 70001 | Zip | 70112 | Zip | 70118 | Zip | 70118 | Zip | 48109 | Zip | 07112 | Zip | 10457 | Zip | 10461 | Zip | 27834 | Zip | 44106 | Zip | 29425 | Zip | 29425 | Zip | 29605 | Zip | 38105 | Zip | 76104 | Zip | 20148 | Zip | 22031 |
| Country | United States | Country | United States | Country | United States | Country | United States | Country | United States | Country | United States | Country | United States | Country | United States | Country | United States | Country | United States | Country | United States | Country | United States | Country | United States | Country | United States | Country | United States | Country | United States | Country | United States | Country | United States | Country | United States | Country | United States | Country | United States | Country | United States | Country | United States | Country | United States | Country | United States | Country | United States | Country | United States | Country | United States | Country | United States | Country | United States |
Conditions
| Sequence: | 52021975 |
| Name | Sickle Cell Disease |
| Downcase Name | sickle cell disease |
Id Information
| Sequence: | 40041619 | Sequence: | 40041620 |
| Id Source | org_study_id | Id Source | secondary_id |
| Id Value | GBT440-041 | Id Value | C5341025 |
| Id Type | Other Identifier | ||
| Id Type Description | Alias Study Number | ||
Countries
| Sequence: | 42438366 |
| Name | United States |
| Removed | False |
Interventions
| Sequence: | 52334526 |
| Intervention Type | Drug |
| Name | Voxelotor |
| Description | synthetic small molecule |
Browse Conditions
| Sequence: | 192894809 | Sequence: | 192894810 | Sequence: | 192894811 | Sequence: | 192894812 | Sequence: | 192894813 | Sequence: | 192894814 | Sequence: | 192894815 |
| Mesh Term | Anemia, Sickle Cell | Mesh Term | Anemia, Hemolytic, Congenital | Mesh Term | Anemia, Hemolytic | Mesh Term | Anemia | Mesh Term | Hematologic Diseases | Mesh Term | Hemoglobinopathies | Mesh Term | Genetic Diseases, Inborn |
| Downcase Mesh Term | anemia, sickle cell | Downcase Mesh Term | anemia, hemolytic, congenital | Downcase Mesh Term | anemia, hemolytic | Downcase Mesh Term | anemia | Downcase Mesh Term | hematologic diseases | Downcase Mesh Term | hemoglobinopathies | Downcase Mesh Term | genetic diseases, inborn |
| Mesh Type | mesh-list | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor |
Sponsors
| Sequence: | 48180090 |
| Agency Class | INDUSTRY |
| Lead Or Collaborator | lead |
| Name | Pfizer |
Overall Officials
| Sequence: | 29199433 |
| Role | Study Director |
| Name | Pfizer CT.gov Call Center |
| Affiliation | Pfizer |
Central Contacts
| Sequence: | 11975937 |
| Contact Type | primary |
| Name | Pfizer CT.gov Call Center |
| Phone | 1-800-718-1021 |
| ClinicalTrials.gov_Inquiries@pfizer.com | |
| Role | Contact |
Eligibilities
| Sequence: | 30677969 |
| Gender | All |
| Minimum Age | 6 Months |
| Maximum Age | 11 Years |
| Healthy Volunteers | No |
| Criteria | Inclusion Criteria: Documented diagnosis of sickle cell disease of any genotype Ineligible or unable to participate in actively recruiting clinical studies of voxelotor Baseline hemoglobin (Hb) ≤10.5 g/dL No alternative treatment options in the judgement of the treating Investigator 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 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: Receiving chronic red blood cell (RBC) transfusion therapy for primary or secondary stroke prevention Hepatic dysfunction characterized by alanine aminotransferase (ALT) >4 × the upper limit of normal (ULN) for age Severe renal dysfunction (estimated glomerular filtration rate [eGFR] <30 mL/min/1.73 m2 by Schwartz formula) Clinically significant bacterial, fungal, parasitic, or viral infection that requires 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 Any condition affecting drug absorption, such as major surgery involving the stomach or small intestine (prior cholecystectomy is acceptable) Female who is pregnant or breastfeeding 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 Medical, psychological, or behavioral conditions, that, in the opinion of the Investigator, may preclude informed consent, safe participation, or compliance with the protocol procedures Use of herbal medications (eg, St. John's wort), sensitive cytochrome P450 (CYP) 3A4 substrates with a narrow therapeutic index, or strong CYP3A4 inducers Active symptomatic COVID-19 infection |
| Adult | False |
| Child | True |
| Older Adult | False |
Calculated Values
| Sequence: | 253872329 |
| Number Of Facilities | 30 |
| Registered In Calendar Year | 2021 |
| Were Results Reported | False |
| Has Us Facility | True |
| Has Single Facility | False |
| Minimum Age Num | 6 |
| Maximum Age Num | 11 |
| Minimum Age Unit | Months |
| Maximum Age Unit | Years |
Intervention Other Names
| Sequence: | 26591170 |
| Intervention Id | 52334526 |
| Name | GBT440 |
Links
| Sequence: | 4374590 |
| Url | https://pmiform.com/clinical-trial-info-request?StudyID=GBT440-041 |
| Description | To obtain contact information for a study center near you, click here. |
Responsible Parties
| Sequence: | 28791244 |
| Responsible Party Type | Sponsor |