Overview
The goal of this clinical trial is to learn about SP-420 ability to remove iron from organs in subjects with transfusion-dependent β-thalassemia or transfusion-dependent low-risk myelodysplastic syndrome.
The main questions it aims to answer are:
- How efficient is SP-420 in cleaning iron from the liver?
- How is the safety and tolerability of ascending doses of SP-420?
Participants will:
- Take medication three times weekly
- Attend up to 20 site visits
- Undergo MRI scans
Eligibility
Thalassemia cohorts:
Inclusion criteria:
- Women and men aged 18 years or older
- Transfusion-dependent β-thalassemia including HbE/β-thalassemia requiring iron chelation therapy (β-thalassemia with mutation and/or multiplication of α-globin is allowed)
- On a stable dose of iron chelation for at least 4 weeks prior to screening
- Weight ≥ 35kg at screening
- Transfusion iron overload
- Treated and followed for at least the past 6 months in a specialized centre
Exclusion criteria:
- β-thalassemia with the structural Hb variants HbS and HbC
- Current MDS
- Current biliary disorder
- Historic or ongoing clinically significant kidney disease
- Unable to undergo trial assessments including MRI e.g. due to claustrophobia in MRI scanner
- Pregnant or nursing women
- Men who do not agree to practice effective barrier contraception during the entire period
Myelodysplastic Syndromes Cohorts:
Inclusion criteria:
- Women and men aged 18 years or older
- Very low, low, or intermediate risk Myelodysplastic Syndrome according to IPSS-R
- Weight ≥ 35kg at screening
- Transfusion iron overload
- Treated and followed for at least the past 6 months at medical facilities experienced with MDS
Exclusion criteria:
- Therapy-related MDS or MDS with a known bone marrow fibrosis
- Diagnosis of decompensated liver cirrhosis
- Clinically significant kidney disease, either historic or ongoing
- Uncontrolled ischemic heart disease or uncontrolled arrythmia
- Uncontrolled hypertension
- Uncontrolled dyslipidaemia
- Uncontrolled Diabetes
- Major surgery within 8 weeks prior to screening
- Pregnant or nursing women
- Men who do not agree to practice effective barrier contraception during the entire period
