Expanded Access Protocol for Adults and Pediatric Patients With Sickle Cell Disease Who Have No Alternative Treatment Options
NCT ID: NCT04724421
Last Updated: 2024-11-20
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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NO_LONGER_AVAILABLE
EXPANDED_ACCESS
Brief Summary
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Detailed Description
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Please note that for US sites, enrollment is open only for 6months to \< 4 years of age.
Conditions
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Interventions
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Voxelotor
tablet or powder for oral suspension
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
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
1. Documented diagnosis of SCD of any genotype confirmed by laboratory genetic testing
2. Aged 12 years and older
3. Haemoglobin ≥5.5 and ≤ 10.5 g/dL, based on a test performed according to local standard of care
4. Patient cannot be treated satisfactorily with any authorised medicinal product
5. Patient or their legal representative has provided written informed consent to participate in the voxelotor EAP for the treatment of SCD
6. Patient or their legal representative has provided written informed consent on data processing and protection
Exclusion Criteria
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
1. Patients with clinically significant bacterial, fungal, parasitic or viral infection which requires therapy.
2. History of serious drug hypersensitivity reaction to voxelotor or excipients
3. Participated in another clinical trial of an investigational agent (or medical device) within 30 days of participation in EAP.
4. Medical, psychological, or behavioral condition that, in the opinion of the study doctor, would confound or interfere with evaluation of safety and/or effectiveness of the study drug, prevent compliance with the study protocol; preclude informed consent; or render the participant unable/unlikely to comply with the study procedures
6 Months
11 Years
ALL
No
Sponsors
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Pfizer
INDUSTRY
Responsible Party
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Principal Investigators
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Pfizer CT.gov Call Center
Role: STUDY_DIRECTOR
Pfizer
Locations
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Phoenix Children's Hospital
Phoenix, Arizona, United States
Rady Children's Hospital San Diego
San Diego, California, United States
Nemours/Alfred I. duPont Hospital for Children
Wilmington, Delaware, United States
Children's National Hospital / Children's National Health System
Washington D.C., District of Columbia, United States
Children's National Medical Center
Washington D.C., District of Columbia, United States
Baptist Medical Center/Wolfson Children's Hospital
Jacksonville, Florida, United States
Nemours Children's Health, Jacksonville
Jacksonville, Florida, United States
Nemours Children's Hospital
Orlando, Florida, United States
Children's Healthcare of Atlanta Scottish Rite
Atlanta, Georgia, United States
AU Medical Center Clinical Research Pharmacy
Augusta, Georgia, United States
Augusta University
Augusta, Georgia, United States
Children's Hospital of Georgia
Augusta, Georgia, United States
Memorial Health University Medical Center
Savannah, Georgia, United States
Tulane Lakeside
Metairie, Louisiana, United States
Tulane University Hospitals and Clinics
New Orleans, Louisiana, United States
Children's Hospital
New Orleans, Louisiana, United States
Childrens Hospital of NOLA
New Orleans, Louisiana, United States
University Of Michigan Hospitals
Ann Arbor, Michigan, United States
Rutgers Cancer Institute of New Jersey
New Brunswick, New Jersey, United States
Newark Beth Israel Medical Center & Children's Hospital of New Jersey
Newark, New Jersey, United States
BronxCare Health System
The Bronx, New York, United States
Jacobi Medical Center
The Bronx, New York, United States
East Carolina University Brody School of Medicine(ECU)
Greenville, North Carolina, United States
ECU Physicians, Brody Outpatient Clinic
Greenville, North Carolina, United States
University Hospitals Cleveland Medical Center
Cleveland, Ohio, United States
Medical University of South Carolina: Investigational Drug Services Pharmacy
Charleston, South Carolina, United States
Medical University of South Carolina: Shawn Jenkins Women's and Children's Hospital
Charleston, South Carolina, United States
Medical University of South Carolina
Charleston, South Carolina, United States
Prisma Health - Upstate
Greenville, South Carolina, United States
St. Jude Children's Research Hospital
Memphis, Tennessee, United States
Cook Children's Health Care System
Fort Worth, Texas, United States
Pediatric Specialists of Virginia (Inova Ashburn HealthPlex)
Ashburn, Virginia, United States
Pediatric Specialists of Virginia (Schar Cancer Institute)
Fairfax, Virginia, United States
Multihemo Serviços Médicos S/A
Recife, Pernambuco, Brazil
Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto - USP
Ribeirão Preto, São Paulo, Brazil
HEMORIO - Hematologia Laboratorial
Rio de Janeiro, , Brazil
ESHO Empresa de Serviços Hospitalares S.A/ Hospital Samaritano de Higienópolis
São Paulo, , Brazil
Countries
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Related Links
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To obtain contact information for a study center near you, click here.
Other Identifiers
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C5341025
Identifier Type: OTHER
Identifier Source: secondary_id
GBT440-041
Identifier Type: OTHER
Identifier Source: secondary_id
C5341057
Identifier Type: OTHER
Identifier Source: secondary_id
C534 Expanded Access
Identifier Type: -
Identifier Source: org_study_id
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