HEMolyse and Organ damage imPROvement in sickle cell disease by VoxElotor

Korábbi kezelés

• Patients in chronic transfusion program or transfused < 3 months before enrolment
• Prior drug hypersensitivity to Voxelotor or excipients
• Transplant patients.

Más betegség

• Auto immune disease or infection not controlled or cancer
• VIH, HBV, HCV current infection
• Known allergy or hypersensitivity to imaging contrast product
• 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
• Patient unable to understand the purpose and conditions of the study and unable to give consent

Laborértékek

• Patient with severe organ involvement: hepatic (TP <50%), renal (eGFR<30 ml / ml/1.73m2 according to CKD/EPI or cardiac (LVEF <45%)

Egyéb

• Chronic use of NSAIDs (more than 10 days by month)
• Ongoing therapeutic study
• Pregnancy
• Breast feeding patients
• Homeless patient
• Patient deprived of liberty by judicial or administrative decision or patient under guardianship

• 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), inhibitor/angiotensin receptor blocker (ARB), glutamine or crizanlizumab 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.