SP-420 to remove excess iron in adults with transfusion-dependent beta thalassemia or low‑risk MDS
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
This will test whether SP-420 can clear excess liver iron in adults who receive regular transfusions for beta thalassemia or transfusion‑dependent low‑risk myelodysplastic syndrome.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 90 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Pharmacosmos A/S Industry-sponsored |
| Locations | 1 site (Copenhagen) |
| Trial ID | NCT05693909 on ClinicalTrials.gov |
What this trial studies
This is a Phase 2 interventional, dose-escalation study of SP-420 given three times weekly to adults with transfusion-dependent β-thalassemia or transfusion-dependent low‑risk MDS. Participants will attend up to 20 site visits and undergo MRI scans to measure liver iron while receiving ascending doses to characterize safety and tolerability. Eligible patients must have transfusion-related iron overload and be on a stable iron chelation regimen before screening. The trial is sponsored by Pharmacosmos and conducted at a Pharmacosmos investigational site in Copenhagen.
Who should consider this trial
Good fit: Adults (≥18 years) with transfusion-dependent β-thalassemia or transfusion-dependent low‑risk MDS who have documented transfusion iron overload, are stable on iron chelation, weigh ≥35 kg, and can attend study visits and MRI scans.
Not a fit: People without transfusion-dependent iron overload, those with significant kidney or biliary disease, thalassemia with structural HbS/HbC variants, pregnant or nursing women, or anyone unable to undergo MRI are unlikely to benefit or be eligible.
Why it matters
Potential benefit: If successful, SP-420 could more effectively remove liver iron and help reduce organ damage and complications from long-term transfusions.
How similar studies have performed: Established iron chelators have reduced organ iron in other trials, but SP-420 is a novel agent with limited prior clinical data.
Eligibility criteria
Show full inclusion / exclusion 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 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
Where this trial is running
Copenhagen
- Pharmacosmos Investigational Site — Copenhagen, Denmark (Recruiting)
Study contacts
- Study coordinator: Pharmacosmos Clinical and non-clinical Department
- Email: info@pharmacosmos.com
- Phone: +45 5948 5959
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.