SP-420 to remove excess iron in adults with transfusion-dependent beta thalassemia or low‑risk MDS

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• Pharmacosmos Investigational Site
  Copenhagen, Denmark Recruiting

Study contacts

• Study coordinator: Pharmacosmos Clinical and non-clinical Department
• Email: info@pharmacosmos.com
• Phone: +45 5948 5959

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.