Actively Recruiting

Phase 2
Age: 18Years +
All Genders
NCT05693909

A Trial Testing SP-420 in Subjects With Transfusion-dependent β-thalassemia or Low-risk Myelodysplastic Syndromes

Led by Pharmacosmos A/S · Updated on 2025-09-24

90

Participants Needed

1

Research Sites

243 weeks

Total Duration

On this page

Sponsors

P

Pharmacosmos A/S

Lead Sponsor

I

ICON plc

Collaborating Sponsor

AI-Summary

What this Trial Is About

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

CONDITIONS

Official Title

A Trial Testing SP-420 in Subjects With Transfusion-dependent β-thalassemia or Low-risk Myelodysplastic Syndromes

Who Can Participate

Age: 18Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Women and men aged 18 years or older
  • Diagnosis of transfusion-dependent beta-thalassemia including HbE/beta-thalassemia requiring iron chelation therapy or low-risk myelodysplastic syndrome
  • On a stable dose of iron chelation therapy for at least 4 weeks before screening (for beta-thalassemia group)
  • Weight of 35 kg or more at screening
  • Evidence of transfusion iron overload
  • Treated and followed for at least 6 months in specialized centers (beta-thalassemia) or experienced medical facilities (MDS)
  • Very low, low, or intermediate risk MDS according to IPSS-R (for MDS group)
Not Eligible

You will not qualify if you...

  • Beta-thalassemia with structural hemoglobin variants HbS and HbC
  • Current myelodysplastic syndrome (for beta-thalassemia group)
  • Therapy-related MDS or MDS with known bone marrow fibrosis (for MDS group)
  • Diagnosis of decompensated liver cirrhosis
  • Clinically significant kidney disease, either historic or ongoing
  • Unable to undergo trial assessments including MRI (e.g., due to claustrophobia)
  • Pregnant or nursing women
  • Men unwilling to use effective barrier contraception during the entire study period
  • Uncontrolled ischemic heart disease, arrhythmia, hypertension, dyslipidaemia, or diabetes
  • Major surgery within 8 weeks prior to screening

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 1 location

1

Pharmacosmos Investigational Site

Copenhagen, Denmark

Actively Recruiting

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Research Team

P

Pharmacosmos Clinical and non-clinical Department

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

RANDOMIZED

Model

SEQUENTIAL

Primary Purpose

TREATMENT

Number of Arms

6

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