Clinical trial NCT04724421: Expanded Access Protocol for Adults and Pediatric Patients With Sickle Cell Disease Who Have No Alternative Treatment Options
Official title: Expanded Access Protocol for Adults and Pediatric Patients With Sickle Cell Disease Who Have No Alternative Treatment Options
Study type: EXPANDED_ACCESS
Overall status: NO_LONGER_AVAILABLE
Conditions: Sickle Cell Disease
Keywords: Voxelotor
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2021-01-26
Sex: ALL
Minimum age (years): 6
Maximum age (years): 11
Standard ages: CHILD
Accepts healthy volunteers: no

Brief summary: 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

Detailed description: This EAP may continue until such time that voxelotor is commercially available or the Sponsor discontinues the voxelotor EAP.

Please note that for US sites, enrollment is open only for 6months to \< 4 years of age.

Eligibility criteria: Inclusion Criteria: GBT440-041

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: GBT440-041

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

Inclusion Criteria: C5341057

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: C5341057

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

Primary outcomes:
Secondary outcomes:
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Phoenix Children's Hospital
state: Arizona
city: San Diego
country: United States
facility: Rady Children's Hospital San Diego
state: California
city: Wilmington
country: United States
facility: Nemours/Alfred I. duPont Hospital for Children
state: Delaware
city: Washington
country: United States
facility: Children's National Hospital / Children's National Health System
state: District of Columbia
city: Washington
country: United States
facility: Children's National Medical Center
state: District of Columbia
city: Jacksonville
country: United States
facility: Baptist Medical Center/Wolfson Children's Hospital
state: Florida
city: Jacksonville
country: United States
facility: Nemours Children's Health, Jacksonville
state: Florida
city: Orlando
country: United States
facility: Nemours Children's Hospital
state: Florida
city: Atlanta
country: United States
facility: Children's Healthcare of Atlanta Scottish Rite
state: Georgia
city: Augusta
country: United States
facility: AU Medical Center Clinical Research Pharmacy
state: Georgia
city: Augusta
country: United States
facility: Augusta University
state: Georgia
city: Augusta
country: United States
facility: Children's Hospital of Georgia
state: Georgia
city: Savannah
country: United States
facility: Memorial Health University Medical Center
state: Georgia
city: Metairie
country: United States
facility: Tulane Lakeside
state: Louisiana
city: New Orleans
country: United States
facility: Tulane University Hospitals and Clinics
state: Louisiana
city: New Orleans
country: United States
facility: Children's Hospital
state: Louisiana
city: New Orleans
country: United States
facility: Childrens Hospital of NOLA
state: Louisiana
city: Ann Arbor
country: United States
facility: University Of Michigan Hospitals
state: Michigan
city: New Brunswick
country: United States
facility: Rutgers Cancer Institute of New Jersey
state: New Jersey
city: Newark
country: United States
facility: Newark Beth Israel Medical Center & Children's Hospital of New Jersey
state: New Jersey
city: Bronx
country: United States
facility: BronxCare Health System
state: New York
city: Bronx
country: United States
facility: Jacobi Medical Center
state: New York
city: Greenville
country: United States
facility: East Carolina University Brody School of Medicine(ECU)
state: North Carolina
city: Greenville
country: United States
facility: ECU Physicians, Brody Outpatient Clinic
state: North Carolina
city: Cleveland
country: United States
facility: University Hospitals Cleveland Medical Center
state: Ohio
city: Charleston
country: United States
facility: Medical University of South Carolina: Investigational Drug Services Pharmacy
state: South Carolina
city: Charleston
country: United States
facility: Medical University of South Carolina: Shawn Jenkins Women's and Children's Hospital
state: South Carolina
city: Charleston
country: United States
facility: Medical University of South Carolina
state: South Carolina
city: Greenville
country: United States
facility: Prisma Health - Upstate
state: South Carolina
city: Memphis
country: United States
facility: St. Jude Children's Research Hospital
state: Tennessee
city: Fort Worth
country: United States
facility: Cook Children's Health Care System
state: Texas
city: Ashburn
country: United States
facility: Pediatric Specialists of Virginia (Inova Ashburn HealthPlex)
state: Virginia
city: Fairfax
country: United States
facility: Pediatric Specialists of Virginia (Schar Cancer Institute)
state: Virginia
city: Recife
country: Brazil
facility: Multihemo Serviços Médicos S/A
state: Pernambuco
city: Ribeirão Preto
country: Brazil
facility: Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto - USP
state: SAO Paulo
city: Rio de Janeiro
country: Brazil
facility: HEMORIO - Hematologia Laboratorial
city: São Paulo
country: Brazil
facility: ESHO Empresa de Serviços Hospitalares S.A/ Hospital Samaritano de Higienópolis
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References: