Beta Thalassemia Major Anemia, Myelodysplastic Syndrome
Conditions
Brief 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
Interventions
DRUGSP-420
Capsules for oral intake
Sponsors
ICON plc
Pharmacosmos A/S
Study design
Allocation
RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| To establish dose-response relationship of SP-420 for 24 weeks in the treatment of subjects with transfusion-dependent β-thalassemia | 24 weeks |
| To assess the safety and tolerability of ascending doses of SP-420 after 12 weeks treatment of subjects with transfusion-dependent low-risk myelodysplastic syndrome | 12 weeks |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| To assess the efficacy of SP-420 in clearing iron from the liver after 12 and 48 weeks treatment of subjects with transfusion-dependent β-thalassemia | 12 and 48 weeks | Change in LIC measured by R2-MRI from baseline to week 12 and week 48 |
| To assess the efficacy of SP-420 in clearing iron from the liver after 24 weeks treatment of subjects with transfusion-dependent β-thalassemia | 24 weeks | Change in liver iron concentration (LIC) measured by R2-magnetic resonance imaging (MRI) from baseline to week 24 |
| To assess the efficacy of SP-420 on serum (s-) ferritin | up to 48 weeks | Change in s-ferritin from baseline to weeks 1, 2, 3, 4, 6, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, and 48 |
| To assess the safety and tolerability of ascending doses of SP-420 | 48 weeks | Type and incidence of adverse events (AEs) |
Countries
Denmark
Contacts
Primary ContactPharmacosmos Clinical and non-clinical Department
Outcome results
None listed