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Iron lowering treatment to reduce tissue damage in sickle cell disease

Phase 1
Conditions
Sickle cell disease (an hereditary hemoglobinopathy)
Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Registration Number
EUCTR2020-004995-17-NL
Lead Sponsor
AMsterdam UMC-AMC
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
Authorised-recruitment may be ongoing or finished
Sex
All
Target Recruitment
30
Inclusion Criteria

1. Sickle cell disease diagnosis: HbSS, or HbSß0-thalassemia genotype
2. Age 18-65 years
3. Willing and able to provide written informed consent
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 30
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

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 clinical trial of medicinal product
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Secondary Outcome Measures
NameTimeMethod
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