Sickle Cell DiseasePhase 2HEMOPROVENCT05199766
Hemolysis Related Complications in SCD. A Phase II Study With Voxelotor
A Phase 2 study testing Voxelotor 1500 mg oral per day (GBT440) for 48 weeks in patients with sickle-cell disease in people with Sickle Cell Disease, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
Trial summary
Intro: Sickle cell disease is a genetic disorder caused by a mutation of the β hemoglobin called HbS, which causes red blood cell (RBC) abnormalities responsible for hemolysis, mainly intravascular, leading to chronic anemia. Intravascular hemolysis is responsible for severe inflammation and endothelial dysfunction. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- Voxelotor 1500 mg oral per day (GBT440) for 48 weeks in patients with sickle-cell disease
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteFrance
- Study dates
- 22 Mar 2023to 22 Mar 2025
- Phase
- Phase 2
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- SS or S-β0 major sickle cell syndrome
- Hemoglobin level < 9 g/dL
- Aged 18 years or older
- Stable dose for at least 3 months if treated with HU, EPO, angiotensin-converting enzyme (ACE) or inhibitor/angiotensin receptor blocker (ARB) therapy; at least after 6 months after initiating HU treatment
- Patient with social security
- Female patient must have a negative serum pregnancy test (betaHCGat inclusion W0-V1D1) or evidence of post-menopausal status
- Effective methods of birth control (e.g., condom, spermicidal gel, oral contraceptive, indwelling intrauterine device, hormonal implant/patch, injections, approved cervical ring) or abstinence from screening through 4 weeks after last Voxelotor dose.
Who cannot join
- -If patient does not have any of the following treatments (HU, Crizanlizumab) he will then be excluded if: Patient meets, at screening, Hydroxyurea/ Crizanlizumab indications of treatment (recurrent painful vaso-occlusive crises, including acute chest syndrome), even if these treatments are inappropriate (e.g. hematologic toxicity antecedent) or if the patient refuses these treatments
- Patients in chronic transfusion program or transfused < 3 months before enrolment
- Patient with severe organ involvement: hepatic (TP <50%), renal (eGFR<30 ml / ml/1.73m2 according to CKD/EPI or cardiac (LVEF <45%)
- Transplant patients.
- Pregnancy.
- Breast feeding patients
Show all 15Show fewer
- Homeless patient
- Patient deprived of liberty by judicial or administrative decision or patient under guardianship
- Patient unable to understand the purpose and conditions of the study and unable to give consent
- Chronic use of NSAIDs (more than 10 days by month)
- Auto immune disease or infection not controlled or cancer
- VIH, HBV, HCV current infection
- Prior drug hypersensitivity to Voxelotor or excipients
- Known allergy or hypersensitivity to imaging contrast product
- Ongoing therapeutic study
Trial locations and contacts
1 site in 1 country.
France1 site, 1 recruiting
- Recruiting
Hospital Henri Mondor
Créteil, 94010
Gonzalo De Luna, MD, 0149812443 ext. +33, gonzalo.deluna@aphp.fr
Gonzalo De Luna, MD
About this study
Official title: HEMolyse and Organ Damage imPROvement in Sickle Cell Disease by VoxElotor. An Open-label One Stage Phase II Design
Intro:
Sickle cell disease is a genetic disorder caused by a mutation of the β hemoglobin called HbS, which causes red blood cell (RBC) abnormalities responsible for hemolysis, mainly intravascular, leading to chronic anemia. Intravascular hemolysis is responsible for severe inflammation and endothelial dysfunction.
Read more
Maintaining hemoglobin in its oxygenated R-conformation is one of the strategies for inhibiting the polymerization of HbS. Previous experimental therapeutic approaches having this effect have been discontinued due to poor pharmaceutical properties or toxicity. Nevertheless, they proved the validity of the concept by demonstrating an increase in oxyhemoglobin and a decrease in biomarkers of hemolysis.
Voxelotor binds to the α chain of globin and maintains Hb in its R conformation, thereby inhibiting the polymerization of HbS while increasing the affinity of Hb for oxygen.
Because of its mechanism of action affecting anemia and hemolysis, Voxelotor is a promising treatment for the prevention and treatment of renal and cerebral arterial disease.
Hypothesis/Objective :
Investigator hypothesis is that the treatment by Voxelotor (GBT440) will improve intra vascular hemolysis and will increase the total mass of hemoglobin with beneficial effects on organ function.
The primary objective of the study is to evaluate the biological activity of Voxelotor on the reduction of intra vascular hemolysis measured by plasma hemoglobin.
The secondary objectives of the study will aim at characterizing the effects of GBT 440 Voxelotor on:
- Intra vascular hemolysis measured by plasma Heme
- Total hemoglobin mass (MHb)
- RBCs lifespan
- Blood volumes (plasma volume (PV), red blood cell mass (RBCM), total blood volume (BV))
- Blood viscosity
- Cerebral perfusion
- Cerebrovascular vaso-reactivity
- Cognitive function (MoCA)
- Six minute walk test
- Renal perfusion and iron deposits in renal cortex
- Measurement of Glomerular filtration rate Estimation of glomerular filtration rate (CKD/EPI equation)
- Urine albumin/creatinine ratio
- Ability to decrease or stop erythropoietin in patients under EPO treatment
- Safety (VOC, ACS, Priapism) and tolerability of voxelotor
- RBC properties
Method:
This is an open-label, single-arm, single-stage phase II trial in patients treated with Voxelotor 1500 mg daily for 48 weeks. Assessments will be done during the study at week 0, week 6, week 12, week 24, week 36 and week 48.
- Conditions
- Sickle Cell Disease
- Being tested
- Voxelotor 1500 mg oral per day (GBT440) for 48 weeks in patients with sickle-cell disease
- Keywords
- Sickle Cell Disease, Hemolysis, Plasma Hemoglobin
- Medical subject headings
- Anemia, Sickle Cell, Hemolysis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Voxelotor 1500 mg oral per day (GBT440) for 48 weeks in patients with sickle-cell diseaseDrug
This is an open-label, single-arm, single-stage phase II trial of voxelotor in patients with sickle-cell disease. Voxelotor 500mg tablets. Voxelotor will be administered as 500mg tablets orally once daily. The participant should always take all 3 tablets in a row at the same time each day, unless the study doctor has instructed them to adjust the dose.
| Study group | Role | What they receive |
|---|---|---|
| Voxelotor 1500 mg oral per day (GBT440) for 48 weeks | Experimental | Voxelotor 1500 mg oral per day (GBT440) for 48 weeks, in case of discontinuation due to patient's wishes and/or adverse event above grade 2 : 1000mg during 14 days then complete discontinuation if no resolution. In case of sudden discontinuation a therapeutic phlebotomy will be authorized. Receives: Drug: Voxelotor 1500 mg oral per day (GBT440) for 48 weeks in patients with sickle-cell disease |
What is being measured
The primary outcome is the main question the study is designed to answer.
Evaluation of the biological activity of voxelotor on the change of intra vascular hemolysis measured by decrease of plasma hemoglobin.
Change of Intravascular hemolysis, as defined by a ≥20% decrease of plasma Hemoglobin (µmol/l)
Measured: Change from Baseline at Week 48
Secondary outcomes (16)
Intra vascular hemolysis measured by plasma Heme
Absolute and relative (%) changes in plasma Hemoglobin (µmol/l) and free plasma Heme (µmol/l)
Measured: Week 0, Week 24, Week 48
Total hemoglobin mass (MHb)
Measurement of total hemoglobin mass based on the CO rebreathing technique (g of Hb / kg), or a stable evolution (i.e. decrease ≤ 10%) in patients initially under EPO therapy and who decreased or discontinued EPO during the study period.
Measured: Week 0, Week 24, Week 48
RBCs lifespan
RBC lifespan by measurement of alveolar CO (in days).
Measured: Week 0, Week 24, Week 48
Change of blood volumes (plasma volume (PV) and total blood volume (BV))
Blood volumes by CO rebreathing method (Total blood volume (L), Plasma Volume (L) )
Measured: Week 0, Week 24, Week 48
Change of red blood cell mass (RBCM)
RBC mass (g)
Measured: Week 0, Week 24, Week 48
Change of Total Mass of Hemoglobin
Total Mass of Hemoglobin (g of Hb)
Measured: Week 0, Week 24, Week 48
Change of blood viscosity
Blood viscosity (cP)
Measured: Week 0, Week 24, Week 48
Cerebral perfusion
Cerebral perfusion measured by MRI
Measured: Week 0, Week 24, Week 48
Change of cerebrovascular vaso-reactivity measured by transcranial Doppler
Transcranial Doppler (Breath holding test)
Measured: Week 0, Week 24, Week 48
Change of cerebrovascular vaso-reactivity measured by Near Infra Red Spectroscopy
Cerebral vaso-reactivity measured by Near Infra Red Spectroscopy
Measured: Week 0, Week 24, Week 48
Cognitive function (MoCA)
Cognitive performance measured by MoCA Improvement in the 6 minutes walk test on : Time spent under Sp02 88 and 90%, Borg Rating of Perceived Exertion (RPE), distance.
Measured: Week 0, Week 24, Week 48
Measurement of renal perfusion and amount of deoxyhemoglobin
Amount of deoxyhemoglobin by MRI
Measured: Week 0, Week 24, Week 48
Study of iron deposits in renal cortex
Iron deposits in renal cortex measured by MRI
Measured: Week 0, Week 24, Week 48
Measurement of Glomerular filtration rate
Estimation of glomerular filtration rate (CKD/EPI equation)
Measured: Week 0, Week 24, Week 48
Ability to decrease or stop erythropoietin in patients under EPO treatment
Concomitant treatment observation: decrease / interruption of EPO dose
Measured: From Week 0 to Week48
Incidence of Treatment-Adverse Events VOC, ACS and Priapism
Presence/Absence of adverse Events VOC, ACS, Priapism
Measured: From Week 0 to Week48
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Jan 2022
- Study start22 Mar 2023
- Primary completion22 Mar 2024 (estimated)
- Study completion22 Mar 2025 (estimated)
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 DiseaseStudies of the same condition, open and under way.
- More trials run by Assistance Publique - Hôpitaux de ParisThe 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
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- This is an open-label, single-arm, single-stage phase II trial in patients treated with Voxelotor 1500 mg daily for 48 weeks. Assessments will be done during the study at week 0, week 6, week 12, week 24, week 36 and week 48.
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Collaborators
- Pfizer
- Responsible party
- Sponsor
- Investigators
- Pierre-Andre Natella, PHD, Study director, Assistance Publique - Hôpitaux de Paris
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05199766
- Sponsor's protocol ID
- APHP200750
- Other IDs
- 2020-005424-11
- Acronym
- HEMOPROVE
- Status last verified
- Sep 2023
- First submitted
- 6 Dec 2021
- Last updated
- 21 Sep 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: APHP IS DATA'S OWNER, PLEASE CONTACT BOARD
Publications
Liu W, Kassasseya C, Papamanolis L, Nguyen-Peyre KA, Garreau M, Eckert S, De Luna G, d'Humieres T, de Pierrefeu V, Arnaud C, Bekeziz N, Metteil MPG, Provost C, Gerbeau JF, Verlhac S, Bartolucci P, Vignon-Clementel I. Vascular Geometry Drives Stroke Risk in Sickle Cell Disease. Am J Hematol. 2026 Mar;101(3):477-486. doi: 10.1002/ajh.70184. Epub 2026 Jan 28.
PubMed 41603130
Study at a glance
From the registry record, last updated by the sponsor on 21 Sep 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- HEMolyse and Organ Damage imPROvement in Sickle Cell Disease by VoxElotor. An Open-label One Stage Phase II Design
- ClinicalTrials.gov ID
- NCT05199766
- Acronym / sponsor ID
- HEMOPROVE / APHP200750
- Conditions
- Sickle Cell Disease
- Keywords
- Sickle Cell Disease, Hemolysis, Plasma Hemoglobin
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Collaborators
- Pfizer
- Study start
- 22 Mar 2023 (actual)
- Primary completion
- 22 Mar 2024 (estimated)
- Study completion
- 22 Mar 2025 (estimated)
- First posted
- 20 Jan 2022 (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.
Intro:
Sickle cell disease is a genetic disorder caused by a mutation of the β hemoglobin called HbS, which causes red blood cell (RBC) abnormalities responsible for hemolysis, mainly intravascular, leading to chronic anemia. Intravascular hemolysis is responsible for severe inflammation and endothelial dysfunction.
Maintaining hemoglobin in its oxygenated R-conformation is one of the strategies for inhibiting the polymerization of HbS. Previous experimental therapeutic approaches having this effect have been discontinued due to poor pharmaceutical properties or toxicity. Nevertheless, they proved the validity of the concept by demonstrating an increase in oxyhemoglobin and a decrease in biomarkers of hemolysis.
Voxelotor binds to the α chain of globin and maintains Hb in its R conformation, thereby inhibiting the polymerization of HbS while increasing the affinity of Hb for oxygen.
Because of its mechanism of action affecting anemia and hemolysis, Voxelotor is a promising treatment for the prevention and treatment of renal and cerebral arterial disease.
Hypothesis/Objective :
Investigator hypothesis is that the treatment by Voxelotor (GBT440) will improve intra vascular hemolysis and will increase the total mass of hemoglobin with beneficial effects on organ function.
The primary objective of the study is to evaluate the biological activity of Voxelotor on the reduction of intra vascular hemolysis measured by plasma hemoglobin.
The secondary objectives of the study will aim at characterizing the effects of GBT 440 Voxelotor on:
- Intra vascular hemolysis measured by plasma Heme
- Total hemoglobin mass (MHb)
- RBCs lifespan
- Blood volumes (plasma volume (PV), red blood cell mass (RBCM), total blood volume (BV))
- Blood viscosity
- Cerebral perfusion
- Cerebrovascular vaso-reactivity
- Cognitive function (MoCA)
- Six minute walk test
- Renal perfusion and iron deposits in renal cortex
- Measurement of Glomerular filtration rate Estimation of glomerular filtration rate (CKD/EPI equation)
- Urine albumin/creatinine ratio
- Ability to decrease or stop erythropoietin in patients under EPO treatment
- Safety (VOC, ACS, Priapism) and tolerability of voxelotor
- RBC properties
Method:
This is an open-label, single-arm, single-stage phase II trial in patients treated with Voxelotor 1500 mg daily for 48 weeks. Assessments will be done during the study at week 0, week 6, week 12, week 24, week 36 and week 48.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- This is an open-label, single-arm, single-stage phase II trial in patients treated with Voxelotor 1500 mg daily for 48 weeks. Assessments will be done during the study at week 0, week 6, week 12, week 24, week 36 and week 48.
| Arm | Type | Receives |
|---|---|---|
| Voxelotor 1500 mg oral per day (GBT440) for 48 weeks | Experimental | Drug: Voxelotor 1500 mg oral per day (GBT440) for 48 weeks in patients with sickle-cell diseaseVoxelotor 1500 mg oral per day (GBT440) for 48 weeks, in case of discontinuation due to patient's wishes and/or adverse event above grade 2 : 1000mg during 14 days then complete discontinuation if no resolution. In case of sudden discontinuation a therapeutic phlebotomy will be authorized. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- SS or S-β0 major sickle cell syndrome
- Hemoglobin level < 9 g/dL
- Aged 18 years or older
- Stable dose for at least 3 months if treated with HU, EPO, angiotensin-converting enzyme (ACE) or inhibitor/angiotensin receptor blocker (ARB) therapy; at least after 6 months after initiating HU treatment
- Patient with social security
Key exclusion criteria (15 listed)
- -If patient does not have any of the following treatments (HU, Crizanlizumab) he will then be excluded if: Patient meets, at screening, Hydroxyurea/ Crizanlizumab indications of treatment (recurrent painful vaso-occlusive crises, including acute chest syndrome), even if these treatments are inappropriate (e.g. hematologic toxicity antecedent) or if the patient refuses these treatments
- Patients in chronic transfusion program or transfused < 3 months before enrolment
- Patient with severe organ involvement: hepatic (TP <50%), renal (eGFR<30 ml / ml/1.73m2 according to CKD/EPI or cardiac (LVEF <45%)
- Transplant patients.
- Pregnancy.
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 |
|---|---|---|
| Voxelotor 1500 mg oral per day (GBT440) for 48 weeks in patients with sickle-cell diseaseThis is an open-label, single-arm, single-stage phase II trial of voxelotor in patients with sickle-cell disease. Voxelotor 500mg tablets. Voxelotor will be administered as 500mg tablets orally once daily. The participant should always take all 3 tablets in a row at the same time each day, unless the study doctor has instructed them to adjust the dose. | Drug | Voxelotor 1500 mg oral per day (GBT440) for 48 weeks |
Primary outcome
Evaluation of the biological activity of voxelotor on the change of intra vascular hemolysis measured by decrease of plasma hemoglobin.
Time frame: Change from Baseline at Week 48
Change of Intravascular hemolysis, as defined by a ≥20% decrease of plasma Hemoglobin (µmol/l)
16 secondary outcomes
Intra vascular hemolysis measured by plasma Heme
Time frame: Week 0, Week 24, Week 48
Total hemoglobin mass (MHb)
Time frame: Week 0, Week 24, Week 48
RBCs lifespan
Time frame: Week 0, Week 24, Week 48
Change of blood volumes (plasma volume (PV) and total blood volume (BV))
Time frame: Week 0, Week 24, Week 48
Change of red blood cell mass (RBCM)
Time frame: Week 0, Week 24, Week 48
Change of Total Mass of Hemoglobin
Time frame: Week 0, Week 24, Week 48
Change of blood viscosity
Time frame: Week 0, Week 24, Week 48
Cerebral perfusion
Time frame: Week 0, Week 24, Week 48
Change of cerebrovascular vaso-reactivity measured by transcranial Doppler
Time frame: Week 0, Week 24, Week 48
Change of cerebrovascular vaso-reactivity measured by Near Infra Red Spectroscopy
Time frame: Week 0, Week 24, Week 48
Cognitive function (MoCA)
Time frame: Week 0, Week 24, Week 48
Measurement of renal perfusion and amount of deoxyhemoglobin
Time frame: Week 0, Week 24, Week 48
Study of iron deposits in renal cortex
Time frame: Week 0, Week 24, Week 48
Measurement of Glomerular filtration rate
Time frame: Week 0, Week 24, Week 48
Ability to decrease or stop erythropoietin in patients under EPO treatment
Time frame: From Week 0 to Week48
Incidence of Treatment-Adverse Events VOC, ACS and Priapism
Time frame: From Week 0 to Week48
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Sep 2023.
- Derived from this study: PubMed 41603130Liu W, Kassasseya C, Papamanolis L, Nguyen-Peyre KA, Garreau M, Eckert S, De Luna G, d'Humieres T, de Pierrefeu V, Arnaud C, Bekeziz N, Metteil MPG, Provost C, Gerbeau JF, Verlhac S, Bartolucci P, Vignon-Clementel I. Vascular Geometry Drives Stroke Risk in Sickle Cell Disease. Am J Hematol. 2026 Mar;101(3):477-486. doi: 10.1002/ajh.70184. Epub 2026 Jan 28.
- Individual participant dataSharing planned: No. APHP IS DATA'S OWNER, PLEASE CONTACT BOARD
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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.
Pierre-Andre Natella, PHD study director
Assistance Publique - Hôpitaux de Paris
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Voxelotor 1500 mg oral per day (GBT440) for 48 weeks in patients with sickle-cell disease in people with Sickle Cell Disease, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
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 7 inclusion and 15 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 21 Sep 2023. 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 France. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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?
Contact Gonzalo De Luna, MD (0149812443 ext. +33, gonzalo.deluna@aphp.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Evaluation of the biological activity of voxelotor on the change of intra vascular hemolysis measured by decrease of plasma hemoglobin. (time frame: Change from Baseline at Week 48). 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 NCT05199766, 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 21 Sep 2023.