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

Sickle Cell Disease Clinical Trial

Official title:

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

Clinical Trial Details — Status: Available

Administrative data

• NCT number: NCT04724421
• Other study ID #: C534 Expanded Access
• Secondary ID: C5341025GBT440-0
• Status: Available

Study information

• Verified date: April 2024
• Source: Pfizer
• Contact: Pfizer CT.gov Call Center
• Phone: 1-800-718-1021
• Email: ClinicalTrials.gov_Inquiries[@]pfizer.com
• Is FDA regulated: No
• Study type: Expanded Access

Clinical Trial 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

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.

Recruitment information / eligibility

• Status: Available
• Enrollment: 0
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Months to 11 Years

Eligibility

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

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• Sickle Cell Disease

Intervention

Drug:
• Voxelotor
tablet or powder for oral suspension

Locations

Country	Name	City	State
Brazil	Multihemo Serviços Médicos S/A	Recife	Pernambuco
Brazil	Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto - USP	Ribeirão Preto	SAO Paulo
Brazil	HEMORIO - Hematologia Laboratorial	Rio de Janeiro
Brazil	ESHO Empresa de Serviços Hospitalares S.A/ Hospital Samaritano de Higienópolis	São Paulo
United States	University Of Michigan Hospitals	Ann Arbor	Michigan
United States	Pediatric Specialists of Virginia (Inova Ashburn HealthPlex)	Ashburn	Virginia
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	BronxCare Health System	Bronx	New York
United States	Jacobi Medical Center	Bronx	New York
United States	Medical University of South Carolina	Charleston	South Carolina
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	University Hospitals Cleveland Medical Center	Cleveland	Ohio
United States	Pediatric Specialists of Virginia (Schar Cancer Institute)	Fairfax	Virginia
United States	Cook Children's Health Care System	Fort Worth	Texas
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	Prisma Health - Upstate	Greenville	South Carolina
United States	Baptist Medical Center/Wolfson Children's Hospital	Jacksonville	Florida
United States	Nemours Children's Health, Jacksonville	Jacksonville	Florida
United States	St. Jude Children's Research Hospital	Memphis	Tennessee
United States	Tulane Lakeside	Metairie	Louisiana
United States	Rutgers Cancer Institute of New Jersey	New Brunswick	New Jersey
United States	Children's Hospital	New Orleans	Louisiana
United States	Childrens Hospital of NOLA	New Orleans	Louisiana
United States	Tulane University Hospitals and Clinics	New Orleans	Louisiana
United States	Newark Beth Israel Medical Center & Children's Hospital of New Jersey	Newark	New Jersey
United States	Nemours Children's Hospital	Orlando	Florida
United States	Phoenix Children's Hospital	Phoenix	Arizona
United States	Rady Children's Hospital San Diego	San Diego	California
United States	Memorial Health University Medical Center	Savannah	Georgia
United States	Children's National Hospital / Children's National Health System	Washington	District of Columbia
United States	Children's National Medical Center	Washington	District of Columbia
United States	Nemours/Alfred I. duPont Hospital for Children	Wilmington	Delaware

Sponsors (1)

Lead Sponsor	Collaborator
Pfizer

Countries where clinical trial is conducted

United States,  Brazil, 

External Links

•   To obtain contact information for a study center near you, click here.