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RecruitingLast updated: 27 November 2025

THAL-01: Testing increasing doses of an oral drug called SP-420 in people with Transfusion-dependent β-thalassemia or Low-risk Myelodysplastic SyndromesAn 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

Trial purpose

Medical clipboardCancer treatment

Tumor type

Blood Cancers Haematological

Age

People18+

Trial acronym

THAL-01

Clinical summary

Summary

The goal of this clinical trial is to learn about a medication called SP-420's ability to remove iron from organs in people with transfusion-dependent β-thalassemia or transfusion-dependent low-risk myelodysplastic syndrome (MDS).

The main questions this trial will answer are:

  • How efficient is SP-420 in cleaning iron from the liver?
  • How safe and tolerable are the increasing doses of SP-420?

Participants in this study will:

  • Take the oral capsule SP-420 medication three times weekly
  • Attend up to 20 site visits
  • Undergo MRI scans

Conditions

This trial is treating people with Transfusion-dependent β-thalassemia or Low-risk Myelodysplastic Syndromes

Eligibility

Inclusion

Thalassemia cohorts:

  • 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

Myelodysplastic Syndromes Cohorts:

  • 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

Thalassemia cohorts:

  • 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:

  • 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

Inclusion

  • You are able to swallow medication by mouth.
  • You have had a certain type of treatment or surgical procedure.
  • You have the type of cancer, symptoms, or health risks that this clinical trial is focused on.

Exclusion

  • You have certain types of non-cancer medical conditions.
  • You have had certain treatments, surgical procedures or drugs.
Message

Clinical trials have complex eligibility criteria, and other criteria may apply for this trial. Ask your doctor about whether this trial could be right for you.

Participating hospitals

Recruiting hospitals

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More information

Trial Identifiers

Information on this page is partially produced from ClinicalTrials.gov *. View further details about this trial on the registry via the links below:

Trial sponsor

Pharmacosmos A/S

Scientific 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

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