Sickle Cell DiseaseNCT04724421
Expanded Access Protocol for Adults and Pediatric Patients With Sickle Cell Disease Who Have No Alternative Treatment Options
A study testing Voxelotor in people with Sickle Cell Disease, run by Pfizer at 37 sites in 2 countries.
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… Read more about this study
- Who is it for?
- Ages 6 months to 11 years
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- Voxelotor
- Type of treatment
- Pill or oral medicine
- How many people
- Not given
- Where
- 37 sitesin 2 countries
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 months to 11 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
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- Documented diagnosis of SCD of any genotype confirmed by laboratory genetic testing
- Aged 12 years and older
- Haemoglobin ≥5.5 and ≤ 10.5 g/dL, based on a test performed according to local standard of care
- Patient cannot be treated satisfactorily with any authorised medicinal product
- Patient or their legal representative has provided written informed consent to participate in the voxelotor EAP for the treatment of SCD
- Patient or their legal representative has provided written informed consent on data processing and protection
Who cannot join
- 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
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- 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
- Patients with clinically significant bacterial, fungal, parasitic or viral infection which requires therapy.
- History of serious drug hypersensitivity reaction to voxelotor or excipients
- Participated in another clinical trial of an investigational agent (or medical device) within 30 days of participation in EAP.
- 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
Trial locations and contacts
37 sites in 2 countries.
37 sites in 2 countries
United States33 sites
University Of Michigan Hospitals
Ann Arbor, Michigan, 48109
Pediatric Specialists of Virginia (Inova Ashburn HealthPlex)
Ashburn, Virginia, 20148
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
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
University Hospitals Cleveland Medical Center
Cleveland, Ohio, 44106
Pediatric Specialists of Virginia (Schar Cancer Institute)
Fairfax, Virginia, 22031
Cook Children's Health Care System
Fort Worth, Texas, 76104
East Carolina University Brody School of Medicine(ECU)
Greenville, North Carolina, 27834
ECU Physicians, Brody Outpatient Clinic
Greenville, North Carolina, 27834
Prisma Health - Upstate
Greenville, South Carolina, 29605
Baptist Medical Center/Wolfson Children's Hospital
Jacksonville, Florida, 32207
Nemours Children's Health, Jacksonville
Jacksonville, Florida, 32207
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105
Tulane Lakeside
Metairie, Louisiana, 70001
Rutgers Cancer Institute of New Jersey
New Brunswick, New Jersey, 08901
Tulane University Hospitals and Clinics
New Orleans, Louisiana, 70112
Children's Hospital
New Orleans, Louisiana, 70118
Childrens Hospital of NOLA
New Orleans, Louisiana, 70118
Newark Beth Israel Medical Center & Children's Hospital of New Jersey
Newark, New Jersey, 07112
Nemours Children's Hospital
Orlando, Florida, 32827
Phoenix Children's Hospital
Phoenix, Arizona, 85016
Rady Children's Hospital San Diego
San Diego, California, 92123
Memorial Health University Medical Center
Savannah, Georgia, 31404
BronxCare Health System
The Bronx, New York, 10457
Jacobi Medical Center
The Bronx, New York, 10461
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
Nemours/Alfred I. duPont Hospital for Children
Wilmington, Delaware, 19803
Brazil4 sites
Multihemo Serviços Médicos S/A
Recife, Pernambuco, 50070-460
Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto - USP
Ribeirão Preto, São Paulo, 14048-900
HEMORIO - Hematologia Laboratorial
Rio de Janeiro, 20211-030
ESHO Empresa de Serviços Hospitalares S.A/ Hospital Samaritano de Higienópolis
São Paulo, 01232-010
About this study
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
Read the 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.
- Conditions
- Sickle Cell Disease
- Being tested
- Voxelotor
- Keywords
- Voxelotor
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VoxelotorDrug
Also called GBT440
tablet or powder for oral suspension
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Jan 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- More trials run by PfizerThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- Pfizer
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Pfizer CT.gov Call Center, Study director, Pfizer
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04724421
- Sponsor's protocol ID
- C534 Expanded Access
- Other IDs
- C5341025, GBT440-041, C5341057
- Status last verified
- Nov 2024
- First submitted
- 21 Jan 2021
- Last updated
- 20 Nov 2024
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Nov 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Expanded Access Protocol for Adults and Pediatric Patients With Sickle Cell Disease Who Have No Alternative Treatment Options
- ClinicalTrials.gov ID
- NCT04724421
- Acronym / sponsor ID
- C534 Expanded Access
- Conditions
- Sickle Cell Disease
- Keywords
- Voxelotor
- Study type
- Expanded access
- Recruitment status
- No Longer Available
- Lead sponsor
- PfizerIndustry (pharma, biotech, devices)
- First posted
- 26 Jan 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
Who may qualify
- Ages
- 6 months to 11 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (12 listed)
- 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
Key exclusion criteria (16 listed)
- 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:
- Any condition affecting drug absorption, such as major surgery involving the stomach or small intestine (prior cholecystectomy is acceptable)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| VoxelotorAlso: GBT440tablet or powder for oral suspension | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Nov 2024.
- To obtain contact information for a study center near you, click here.Link provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Pfizer CT.gov Call Center study director
Pfizer
37 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Voxelotor in people with Sickle Cell Disease, run by Pfizer at 37 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 16 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 20 Nov 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 37 sites in United States, Brazil. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Nov 2024.