Status:
UNKNOWN
Hemolysis Related Complications in SCD. A Phase II Study With Voxelotor
Lead Sponsor:
Assistance Publique - Hôpitaux de Paris
Collaborating Sponsors:
Pfizer
Conditions:
Sickle Cell Disease
Eligibility:
All Genders
18+ years
Phase:
PHASE2
Brief 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, ...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
March 22 2023
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
March 22 2025
Estimated Enrollment :
30 Patients enrolled
Trial Details
Trial ID
NCT05199766
Start Date
March 22 2023
End Date
March 22 2025
Last Update
September 21 2023
Active Locations (1)
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1
Hospital Henri Mondor
Créteil, France, 94010