Treatment for complications in sickle cell disease using Voxelotor
HEMolyse and Organ Damage imPROvement in Sickle Cell Disease by VoxElotor. An Open-label One Stage Phase II Design
This study is testing if a new oral medication called Voxelotor can help adults with sickle cell disease who have complications from hemolysis by improving their hemoglobin levels and overall health.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 30 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Assistance Publique - Hôpitaux de Paris Academic / other |
| Drugs / interventions | Crizanlizumab |
| Locations | 1 site (Créteil) |
| Trial ID | NCT05199766 on ClinicalTrials.gov |
What this trial studies
This clinical trial investigates the effects of Voxelotor, an oral medication, on patients with sickle cell disease who experience hemolysis-related complications. The study aims to maintain hemoglobin in its oxygenated form to prevent the polymerization of sickle hemoglobin, which leads to severe anemia and inflammation. Participants will receive a daily dose of 1500 mg of Voxelotor for 48 weeks to assess its efficacy in reducing hemolysis and improving overall health outcomes. The trial is designed for adults with specific eligibility criteria related to their hemoglobin levels and previous treatments.
Who should consider this trial
Good fit: Ideal candidates for this study are adults aged 18 and older with sickle cell disease and hemoglobin levels below 9 g/dL.
Not a fit: Patients who do not have sickle cell disease or those who are not currently receiving treatment for their condition may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly improve the quality of life for patients with sickle cell disease by reducing hemolysis and associated complications.
How similar studies have performed: Previous studies have shown promise in using similar approaches to manage hemolysis in sickle cell disease, indicating potential for success with Voxelotor.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * 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. Exclusion Criteria: -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 * 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
Where this trial is running
Créteil
- Hospital Henri Mondor — Créteil, France (Recruiting)
Study contacts
- Study coordinator: Gonzalo De Luna, MD
- Email: gonzalo.deluna@aphp.fr
- Phone: 0149812443
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.