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NCT Number: NCT05693909

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

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

Recruiting

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Key information

Conditions

Beta Thalassemia Major Anemia Bone Marrow Diseases Hematologic Diseases Hemic and Lymphatic Diseases Myelodysplastic Syndrome Myelodysplastic Syndromes

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Pharmacosmos Investigational Site

Copenhagen, Denmark

Location status: Recruiting

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

SP-420

Drug

Capsules for oral intake

Primary outcomes

  1. To establish dose-response relationship of SP-420 for 24 weeks in the treatment of subjects with transfusion-dependent β-thalassemia

    Time frame: 24 weeks

  2. 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

    Time frame: 12 weeks

Secondary outcomes

  1. To assess the efficacy of SP-420 in clearing iron from the liver after 24 weeks treatment of subjects with transfusion-dependent β-thalassemia

    Time frame: 24 weeks

    Change in liver iron concentration (LIC) measured by R2-magnetic resonance imaging (MRI) from baseline to week 24

  2. 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

    Time frame: 12 and 48 weeks

    Change in LIC measured by R2-MRI from baseline to week 12 and week 48

  3. To assess the efficacy of SP-420 on serum (s-) ferritin

    Time frame: 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

  4. To assess the safety and tolerability of ascending doses of SP-420

    Time frame: 48 weeks

    Type and incidence of adverse events (AEs)

Study contacts

Contact information is provided by the study sponsor or research team.

Pharmacosmos Clinical and non-clinical Department

CONTACT

[email protected]

+45 5948 5959

Sponsors and collaborators

Lead sponsor

Pharmacosmos A/S

Industry

Collaborators

  • ICON plc

Registry information

Official study title

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

Important dates

Study start
2023
Primary completion
2027
Study completion
2028
First posted
Jan 23, 2023
Registry last updated
Sep 24, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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