Sickle Cell AnemiaPhase 4NCT04581356
Voxelotor Sickle Cell Exercise Study
A Phase 4 study testing Voxelotor in people with Sickle Cell Anemia, run by Elizabeth Yang, MD, PhD at 1 site in United States.
Trial summary
This study is a pilot, open-label, single-arm study to evaluate the effect of the sickle cell medication voxelotor on exercise capacity, as measured by cardiopulmonary exercise testing (CPET) in patients 12 years of age and older with sickle cell anemia (SCA). Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Sickle Cell Anemia
- What is being tested?
- Voxelotor
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 14 participants
- Where
- 1 siteUnited States
- Study dates
- 21 Sep 2020to 29 Dec 2021
- Phase
- Phase 4
- 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
- Provision of signed and dated informed consent form
- Stated willingness to comply with all study procedures and availability for the duration of the study
- Male or female, age > 12 years
- In good general health as evidenced by medical history and diagnosed with a genetically severe form of sickle cell anemia (Hgb SS, Hgb S beta 0 thalassemia, Hgb SCHarlem, and others)
- Patients who are on Hydroxyurea need to be on a stable dose for at least 3 months without anticipated change in dosing until the study is completed.
- Ability to take oral medication and willingness to adhere to daily voxelotor and 2 CPETs at scheduled intervals.
- For females of reproductive potential who are sexually active: use of highly effective contraception for at least 1 month prior to screening and agreement to use such a method during study participation and for an additional 30 days after the end of study.
- For males of reproductive potential: use of condoms or other methods to ensure effective contraception with partner
Who cannot join
- Patients on chronic transfusions or who received a transfusion within last 8 weeks
- Patients who had hospitalization for vaso-occlusive crisis or acute chest syndrome within 30 days prior to informed consent/assent.
- Patients who have screening alanine aminotransferase (ALT) > 4X upper limit of normal
- Patients who suffer from physical inactivity attributable to clinically significant musculoskeletal, cardiovascular, or respiratory comorbidities
- Patients already taking commercially available voxelotor
- Prior hypersensitivity to voxelotor or excipients.
- Pregnant patients
Trial locations and contacts
1 site in 1 country.
United States1 site
Pediatric Specialist of Virginia
Fairfax, Virginia, 22031
About this study
Official title: The Effect of Voxelotor on Exercise Capacity of Youths With Sickle Cell Anemia
This study is a pilot, open-label, single-arm study to evaluate the effect of the sickle cell medication voxelotor on exercise capacity, as measured by cardiopulmonary exercise testing (CPET) in patients 12 years of age and older with sickle cell anemia (SCA).
Read the detailed description
This study will assess exercise capacity by cardiopulmonary exercise testing (CPET) before and after 8 weeks of voxelotor therapy.
Patients with genetically severe forms of sickle cell disease, including Hgb SS, Hgb S beta 0 thalassemia, Hgb SC Harlem, etc., age 12 or older, with stable Hgb and Hgb F will be recruited. Enrolled subjects will have study labs drawn, undergo baseline CPET in the exercise lab, then take voxelotor 1500mg daily for 2 months, followed by repeat study labs and a second CPET. Each subject's CPET results before and after voxelotor will be compared, and the study labs before and after voxelotor will be compared. Each subject will be compared to him/herself.
- Conditions
- Sickle Cell Anemia
- 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, Oxbryta
daily voxelotor 1500mg oral medication
| Study group | Role | What they receive |
|---|---|---|
| voxelotor | Experimental | Voxelotor 1500mg daily orally Receives: Drug: Voxelotor |
What is being measured
The primary outcome is the main question the study is designed to answer.
Peak oxygen consumption (VO2)
Change in peak oxygen consumption (VO2) measured in CPET after voxelotor treatment
Measured: 8 weeks
Secondary outcomes (9)
Change in Biochemical markers of red cell sickling: Hemoglobin
Measured before and after treatment with voxelotor.
Measured: 8 weeks
Change in Biochemical markers of red cell sickling: Reticulocyte Count
Measured before and after treatment with voxelotor.
Measured: 8 weeks
Change in Biochemical markers of red cell sickling: Bilirubin
Measured before and after treatment with voxelotor.
Measured: 8 weeks
Change in Biochemical markers of red cell sickling: Lactate Dehydrogenase (LDH)
Measured before and after treatment with voxelotor.
Measured: 8 weeks
Change in Biochemical markers of red cell sickling: Haptoglobin
Measured before and after treatment with voxelotor.
Measured: 8 weeks
Change in Biochemical markers of red cell sickling: % Fetal Hemoglobin expressing cells
Measured before and after treatment with voxelotor.
Measured: 8 weeks
Change in Biochemical markers of red cell sickling: P50 oxygen dissociation
Measured before and after treatment with voxelotor.
Measured: 8 weeks
Change in Biochemical markers of red cell sickling: Point of Sickling (POS)
Measured before and after treatment with voxelotor.
Measured: 8 weeks
Change in Biochemical markers of red cell sickling: Dense Cells
Measured before and after treatment with voxelotor.
Measured: 8 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Oct 2020
- Study start21 Sep 2020
- Primary completion25 Oct 2021
- Study completion29 Dec 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Sickle Cell AnemiaStudies of the same condition, open and under way.
- 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
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- single-arm, open-label, pilot study in which each subject compared to him/herself
- Masking
- None (open label)
- Enrolment
- 14 participants (actual)
Sponsor and investigators
- Lead sponsor
- Elizabeth Yang, MD, PhD
- Sponsor type
- University, hospital or other
- Collaborators
- Pediatrix, University of California, San Francisco, Global Blood Therapeutics
- Responsible party
- Sponsor-investigator, Elizabeth Yang, MD, PhD, Pediatric Specialists of Virginia
- Investigators
- Elizabeth Yang, MD, PhD., Principal investigator, Pediatric Specialists of VirginiaVivian Phan, MS, Study director, Pediatric Specialists of VirginiaKari Wheeler, BSN, RN, Study director, Pediatric Specialists of Virginia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04581356
- Sponsor's protocol ID
- ESR-C006
- Status last verified
- Jun 2022
- First submitted
- 11 Sep 2020
- Last updated
- 15 Jun 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Phan V, Hershenson J, Caldarera L, Larkin SK, Wheeler K, Cortez AL, Dulman R, Briere N, Lewis A, Kuypers FA, Yang E. Effect of voxelotor on cardiopulmonary testing in youths with sickle cell anemia in a pilot study. Pediatr Blood Cancer. 2023 Aug;70(8):e30423. doi: 10.1002/pbc.30423. Epub 2023 May 29.
PubMed 37247190
Study at a glance
From the registry record, last updated by the sponsor on 15 Jun 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Effect of Voxelotor on Exercise Capacity of Youths With Sickle Cell Anemia
- ClinicalTrials.gov ID
- NCT04581356
- Acronym / sponsor ID
- ESR-C006
- Conditions
- Sickle Cell Anemia
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 14 participants (actual)
- Lead sponsor
- Elizabeth Yang, MD, PhDUniversity, hospital or other
- Collaborators
- Pediatrix, University of California, San Francisco, Global Blood Therapeutics
- Study start
- 21 Sep 2020 (actual)
- Primary completion
- 25 Oct 2021 (actual)
- Study completion
- 29 Dec 2021 (actual)
- First posted
- 9 Oct 2020 (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.
This study is a pilot, open-label, single-arm study to evaluate the effect of the sickle cell medication voxelotor on exercise capacity, as measured by cardiopulmonary exercise testing (CPET) in patients 12 years of age and older with sickle cell anemia (SCA).
Detailed description
This study will assess exercise capacity by cardiopulmonary exercise testing (CPET) before and after 8 weeks of voxelotor therapy.
Patients with genetically severe forms of sickle cell disease, including Hgb SS, Hgb S beta 0 thalassemia, Hgb SC Harlem, etc., age 12 or older, with stable Hgb and Hgb F will be recruited. Enrolled subjects will have study labs drawn, undergo baseline CPET in the exercise lab, then take voxelotor 1500mg daily for 2 months, followed by repeat study labs and a second CPET. Each subject's CPET results before and after voxelotor will be compared, and the study labs before and after voxelotor will be compared. Each subject will be compared to him/herself.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- single-arm, open-label, pilot study in which each subject compared to him/herself
| Arm | Type | Receives |
|---|---|---|
| voxelotor | Experimental | Drug: VoxelotorVoxelotor 1500mg daily orally |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (8 listed)
- Provision of signed and dated informed consent form
- Stated willingness to comply with all study procedures and availability for the duration of the study
- Male or female, age > 12 years
- In good general health as evidenced by medical history and diagnosed with a genetically severe form of sickle cell anemia (Hgb SS, Hgb S beta 0 thalassemia, Hgb SCHarlem, and others)
- Patients who are on Hydroxyurea need to be on a stable dose for at least 3 months without anticipated change in dosing until the study is completed.
Key exclusion criteria (7 listed)
- Patients on chronic transfusions or who received a transfusion within last 8 weeks
- Patients who had hospitalization for vaso-occlusive crisis or acute chest syndrome within 30 days prior to informed consent/assent.
- Patients who have screening alanine aminotransferase (ALT) > 4X upper limit of normal
- Patients who suffer from physical inactivity attributable to clinically significant musculoskeletal, cardiovascular, or respiratory comorbidities
- Patients already taking commercially available voxelotor
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, Oxbrytadaily voxelotor 1500mg oral medication | Drug | voxelotor |
Primary outcome
Peak oxygen consumption (VO2)
Time frame: 8 weeks
Change in peak oxygen consumption (VO2) measured in CPET after voxelotor treatment
9 secondary outcomes
Change in Biochemical markers of red cell sickling: Hemoglobin
Time frame: 8 weeks
Change in Biochemical markers of red cell sickling: Reticulocyte Count
Time frame: 8 weeks
Change in Biochemical markers of red cell sickling: Bilirubin
Time frame: 8 weeks
Change in Biochemical markers of red cell sickling: Lactate Dehydrogenase (LDH)
Time frame: 8 weeks
Change in Biochemical markers of red cell sickling: Haptoglobin
Time frame: 8 weeks
Change in Biochemical markers of red cell sickling: % Fetal Hemoglobin expressing cells
Time frame: 8 weeks
Change in Biochemical markers of red cell sickling: P50 oxygen dissociation
Time frame: 8 weeks
Change in Biochemical markers of red cell sickling: Point of Sickling (POS)
Time frame: 8 weeks
Change in Biochemical markers of red cell sickling: Dense Cells
Time frame: 8 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Jun 2022.
- Derived from this study: PubMed 37247190Phan V, Hershenson J, Caldarera L, Larkin SK, Wheeler K, Cortez AL, Dulman R, Briere N, Lewis A, Kuypers FA, Yang E. Effect of voxelotor on cardiopulmonary testing in youths with sickle cell anemia in a pilot study. Pediatr Blood Cancer. 2023 Aug;70(8):e30423. doi: 10.1002/pbc.30423. Epub 2023 May 29.
- Individual participant dataSharing planned: No
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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.
Elizabeth Yang, MD, PhD. principal investigator
Pediatric Specialists of Virginia
Vivian Phan, MS study director
Pediatric Specialists of Virginia
Kari Wheeler, BSN, RN study director
Pediatric Specialists of Virginia
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Voxelotor in people with Sickle Cell Anemia, run by Elizabeth Yang, MD, PhD at 1 site 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 8 inclusion and 7 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: finished, as of the sponsor's last update on 15 Jun 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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.
What does the primary outcome measure?
Peak oxygen consumption (VO2) (time frame: 8 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04581356, which also lists 1 linked publication.
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 15 Jun 2022.