Sickle Cell DiseaseNCT03943615
Expanded Access to Voxelotor for 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 18 sites in United States.
Trial summary
The intent of this open-label, multicenter expanded access program (EAP) is to provide early access to voxelotor prior to market authorization Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- Voxelotor
- How many people
- Not given
- Where
- 18 sitesUnited States
- 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 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Documented diagnosis of sickle cell disease of any genotype confirmed by laboratory testing during screening
- Ineligible or unable to participate in actively recruiting clinical studies of voxelotor
- Hemoglobin (Hb) ≤ 10.5 g/dL during screening
- No alternative treatment options in the judgment of the treating investigator
- Participants, who if female and of child bearing potential, agree to use highly effective methods of contraception from study start to 30 days after the last dose of voxelotor, and who if male, agree to use barrier methods of contraception from study start to 30 days after the last dose of voxelotor
- Patient has provided documented informed consent (the informed consent form [ICF] must be reviewed and signed by each participant),or for participants under 18 years of age, signed parental/legal guardian consent and participant assent.
Who cannot join
- Female who is breast feeding or pregnant
- Hepatic dysfunction characterized by alanine aminotransferase (ALT) >4 × ULN
- Patients with clinically significant bacterial, fungal, parasitic, or viral infection which require 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
- Renal dialysis
Show all 11Show fewer
- Non-SCD conditions or complications of SCD for which survival beyond 3 months is unlikely, in the opinion of the treating Investigator
- Any condition affecting drug absorption, such as major surgery involving the stomach or small intestine (prior cholecystectomy is acceptable)
- Participated in another clinical trial of an investigational agent (or medical device) within 30 days or 5 half-lives of date of informed consent, whichever is longer, or is currently participating in another trial of an investigational agent (or medical device)
- Medical, psychological, or behavioral conditions, which, in the opinion of the Investigator, may preclude safe participation, interfere with compliance or preclude informed consent
- The Sponsor Medical Monitor makes the sole and final enrollment determination for any patient.
Trial locations and contacts
18 sites in 1 country.
18 sites in 1 country
United States18 sites
Children's Healthcare of Atlanta
Atlanta, Georgia, 30342
Augusta University
Augusta, Georgia, 30912
The John Hopkins Hospital
Baltimore, Maryland, 21287
Our Lady of the Lake Physician Group
Baton Rouge, Louisiana, 70809
Alabama Oncology
Birmingham, Alabama, 35211
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27514
The Ohio State University Wexner Medical Center
Columbus, Ohio, 43210
Duke Department of Pediatrics
Durham, North Carolina, 27710
Cook Children's Medical Center
Fort Worth, Texas, 76104
Texas Children's Hospital
Houston, Texas, 77030
Queens Hospital Center
Jamaica, New York, 11432
University of South Alabama
Mobile, Alabama, 36693
Rutgers Cancer Institute of New Jersey
New Brunswick, New Jersey, 08901-1914
University Medical Center New Orleans
New Orleans, Louisiana, 70112
Thomas Jefferson University Hospital
Philadelphia, Pennsylvania, 19107
University of Pittsburgh Medical Center Health System
Pittsburgh, Pennsylvania, 15232
VCU Health
Richmond, Virginia, 23298
Montefiore Medical Center PRIME
The Bronx, New York, 10461
About this study
Official title: An Open-Label, Expanded Access Protocol for Patients With Sickle Cell Disease Who Have No Alternative Treatment Options
The intent of this open-label, multicenter expanded access program (EAP) is to provide early access to voxelotor prior to market authorization
Read the detailed description
The purpose of this program is to provide early access to voxelotor for patients with sickle cell disease who have exhausted alternative treatment options and are ineligible for actively-recruiting clinical trials of voxelotor.
This EAP may continue until such time that voxelotor is commercially available or the Sponsor discontinues the voxelotor EAP. Discontinuation may occur earlier if an individual participant or physician or Sponsor Medical Monitor decides to discontinue treatment.
- Conditions
- Sickle Cell Disease
- Being tested
- 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
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 May 2019
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
- NCT03943615
- Sponsor's protocol ID
- GBT440-037
- Other IDs
- C5341044
- Status last verified
- Jul 2023
- First submitted
- 7 May 2019
- Last updated
- 18 Jul 2023
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Jul 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label, Expanded Access Protocol for Patients With Sickle Cell Disease Who Have No Alternative Treatment Options
- ClinicalTrials.gov ID
- NCT03943615
- Acronym / sponsor ID
- GBT440-037
- Conditions
- Sickle Cell Disease
- Study type
- Expanded access
- Recruitment status
- No Longer Available
- Lead sponsor
- PfizerIndustry (pharma, biotech, devices)
- First posted
- 9 May 2019 (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 intent of this open-label, multicenter expanded access program (EAP) is to provide early access to voxelotor prior to market authorization
Detailed description
The purpose of this program is to provide early access to voxelotor for patients with sickle cell disease who have exhausted alternative treatment options and are ineligible for actively-recruiting clinical trials of voxelotor.
This EAP may continue until such time that voxelotor is commercially available or the Sponsor discontinues the voxelotor EAP. Discontinuation may occur earlier if an individual participant or physician or Sponsor Medical Monitor decides to discontinue treatment.
Study design
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Documented diagnosis of sickle cell disease of any genotype confirmed by laboratory testing during screening
- Ineligible or unable to participate in actively recruiting clinical studies of voxelotor
- Hemoglobin (Hb) ≤ 10.5 g/dL during screening
- No alternative treatment options in the judgment of the treating investigator
- Participants, who if female and of child bearing potential, agree to use highly effective methods of contraception from study start to 30 days after the last dose of voxelotor, and who if male, agree to use barrier methods of contraception from study start to 30 days after the last dose of voxelotor
Key exclusion criteria (11 listed)
- Female who is breast feeding or pregnant
- Hepatic dysfunction characterized by alanine aminotransferase (ALT) >4 × ULN
- Patients with clinically significant bacterial, fungal, parasitic, or viral infection which require therapy:
- Renal dialysis
- Non-SCD conditions or complications of SCD for which survival beyond 3 months is unlikely, in the opinion of the treating Investigator
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: GBT440 | 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 18 Jul 2023.
- 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
18 sites in 1 country: 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 18 sites in United States.
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 6 inclusion and 11 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 18 Jul 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 18 sites in United States. 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 18 Jul 2023.