Low Dose Iron Chelation as TReatment of Oxidative Damage in Sickle Cell Disease
Status:
Recruiting
Trial end date:
2022-11-20
Target enrollment:
Participant gender:
Summary
Objective:
To study the safety and efficacy of deferasirox as treatment of oxidative stress in adult
subjects with sickle cell disease.
Endpoints:
The investigators will determine whether treatment with iron chelators results in decreased
sickling of RBCs, oxidative stress, neutrophil activation, inflammation, endothelial
activation and hypercoagulability and ultimately reduced disease severity. If the hypothesis
is confirmed in this pilot dose-finding study, a larger randomized controlled clinical trial
will be initiated.
Study design:
This will be an open-label pilot study, including 12 patients per dose group with a maximum
of 3 dose groups. As the antioxidant capacity of deferasirox might be dose-dependent, the
investigators will start with the highest dose of deferasirox (360 mg) deemed adequate for
chronic use without causing iron depletion in adult SCD patients.
Study population:
Adult patients with sickle cell anemia (HbSS) or HbS-β0-thalassemia (HbSβ0-thal) visiting the
outpatient-clinic of the Academic Medical Center, Amsterdam will be asked for inclusion in
the study.
Phase:
Phase 2
Details
Lead Sponsor:
Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)