ow dose iron chelation as TReatment of Oxidative stress in Sickle cell disease; TROS study

Phase 2

Recruiting

Conditions: Hereditary blooddisorder
Sicklecell anaemia
10018902

Registration Number: NL-OMON49517

Lead Sponsor: Academisch Medisch Centrum

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Recruiting

Sex: Not specified

Target Recruitment: 36

Inclusion Criteria

1. Sickle cell disease diagnosis: HbSS, or HbSβ0-thalassemia genotype
2. Age 18-65 years
3. Willing and able to provide written informed consent

Exclusion Criteria

1. Blood transfusion in the preceding four months
2. Already using iron chelation due to iron overload
3. Ferritin levels of <50 µg/L and/or transferrin saturation of < 0.20.
4. LDH of < 300 U/L
5. Pregnancy or the desire to get pregnant in the following 6 months
6. Impaired renal function of GFR < 60 ml/min/1,73m2 (CKD-EPI).
7. Known allergic reaction to deferasirox.
8. Other somatic or cognitive condition disturbing adherence to study treatment

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>The main endpoints of this study are safety and efficacy.<br /><br>-We will evaluate safety by analysis of adverse events, medication use and<br /><br>physical and laboratory examinations.<br /><br>-The primary efficacy endpoint will be the effect of deferasirox on sickling of<br /><br>red blood cells, measured as changes in Point of Sickling (PoS), as quantified<br /><br>by Oxygenscan. </p><br>

Secondary Outcome Measures

Name	Time	Method
<p>- To evaluate RBC degradation as expressed by phosphatidylserine (PS) exposure<br /><br>on the outer surface of RBC membrane and markers of hemolysis (cell-free heme,<br /><br>lactate dehydrogenase (LDH), bilirubin, reticulocytes and hemoglobin<br /><br>- To evaluate the effect of deferasirox on RBC HbS percentage<br /><br>- Effect of deferasirox on levels of non-transferrin bound iron (NTBI) and<br /><br>labile plasma iron (LPI)<br /><br>- To evaluate the effect of deferasirox on oxidative stress as expressed by<br /><br>intracellular metabolomics and by plasma levels of AGEs<br /><br>- To evaluate the effect of deferasirox on clinical characteristics such as<br /><br>fatigue and pain</p><br>