A Trial Testing SP-420 in Subjects With Transfusion-dependent β-thalassemia or Low-risk Myelodysplastic Syndromes
An Open-label, Dose-escalation, Dose-finding, and Proof-of-concept Trial of SP-420 in Subjects With Transfusion-dependent β-thalassemia or Low-risk Myelodysplastic Syndromes
Pharmacosmos A/S
90 participants
Sep 4, 2023
INTERVENTIONAL
Conditions
Summary
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
Inclusion Criteria12
- Thalassemia cohorts:
- 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
- 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 Criteria18
- 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:
- 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
Interested in this trial?
Get notified about updates and connect with the research team.
Interventions
Capsules for oral intake
Locations(1)
View Full Details on ClinicalTrials.gov
For the most up-to-date information, visit the official listing.
NCT05693909