Tests SP-420 for removing iron from organs in certain blood disorders
Tests SP-420 to remove excess iron in adults with thalassemia or myelodysplastic syndrome.
Key facts
- Who runs it
- Pharmacosmos A/S
- Where
- Aarhus N, Copenhagen Oe, and Odense C, DenmarkSee the 3 sitesAlso in 3 other countries, 25 sites
- Trial code
- NCT05693909
- EU 2023-507396-21-00
- EudraCT 2022-002395-36
- U1111-1277-5903
- Registry title
- A Trial Testing SP-420 in Subjects With Transfusion-dependent β-thalassemia or Low-risk Myelodysplastic Syndromes
- Status
- Recruiting
- Phase
- Phase 2
- People taking part
- 90 people, planned
- Started
- 4 Sept 2023
- Expected to end
- May 2028
- Type of study
- Interventional, 6 groups
- Treatments tested
- SP-420
- Record updated
- 24 September 2025
Who can take part
AI summary
From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.
The trial is looking for adults with transfusion-dependent β-thalassemia or low-risk myelodysplastic syndrome who have too much iron in the body from blood transfusions.
- Age
- 18 to 64 years
- Sex
- Women and men can join.
- Disease stage
- Transfusion-dependent β-thalassemia (including HbE/β-thalassemia) on iron chelation, or very low, low or intermediate risk myelodysplastic syndrome (IPSS-R). Both groups must have iron overload from transfusions.
- Earlier treatment
- Thalassemia group: a stable dose of iron chelation for at least 4 weeks before screening. Both groups: treated and followed for at least the past 6 months.
Main reasons for not taking part
- Weight under 35kg (77 lb (35 kg))
- Pregnant or nursing women
- Clinically significant kidney disease, past or current
- Unable to have MRI scans, for example because of claustrophobia
- Thalassemia group: structural Hb variants HbS and HbC, current MDS or a current biliary disorder; MDS group: therapy-related MDS, bone marrow fibrosis or decompensated liver cirrhosis
The registry criteria, word for word
Show all 29 criteriaShow less
Who can take part
- 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
- 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
Who cannot take part
- Therapy-related MDS or MDS with a known bone marrow fibrosis
- Major surgery within 8 weeks prior to screening
- Current biliary disorder
- Historic or ongoing clinically significant kidney disease
- Men who do not agree to practice effective barrier contraception during the entire period; Myelodysplastic Syndromes Cohorts:
- Clinically significant kidney disease, either historic or ongoing
- Uncontrolled ischemic heart disease or uncontrolled arrythmia
- Uncontrolled hypertension
- Uncontrolled Diabetes
- β-thalassemia with the structural Hb variants HbS and HbC
- Current MDS
- Unable to undergo trial assessments including MRI e.g. due to claustrophobia in MRI scanner
- Pregnant or nursing women
- Diagnosis of decompensated liver cirrhosis
- Uncontrolled dyslipidaemia
- Pregnant or nursing women
- Men who do not agree to practice effective barrier contraception during the entire period
Other criteria
- Thalassemia cohorts:
Check if you might fit
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 18 and 64 years old?
Where in Denmark
Also in 3 other countries, 25 sites
- Region MidtjyllandAarhus N, DenmarkTaking people
- RigshospitaletCopenhagen Oe, DenmarkTaking people
- Odense University HospitalOdense C, DenmarkTaking people
About the trial
What the trial wants to find out
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.
Read full original descriptionShow less
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
The 6 groups in the trial
- Cohort 1a-1bTreatment being tested
SP-420 for 48 weeks
Treatment: SP-420
- Cohort 2a-2bTreatment being tested
SP-420 for 48 weeks
Treatment: SP-420
- Cohort 3a-3bTreatment being tested
SP-420 for 48 weeks
Treatment: SP-420
- 1c-1dTreatment being tested
SP-420 for 48 weeks
Treatment: SP-420
- 2c-2dTreatment being tested
SP-420 for 48 weeks
Treatment: SP-420
- 3c-3dTreatment being tested
SP-420 for 48 weeks
Treatment: SP-420
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Contact the study team: see the registry record → Contacts and Locations
Print a page for my doctorData and sources
- Registry record
- Listed on ClinicalTrials.gov and on the EU's official list of trials (CTIS).
- Official 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
- Plain-language title
- Tests SP-420 for removing iron from organs in certain blood disordersGenerated from the registry record. The registry title is the official one.
- AI summary
- From the trial's eligibility criteria. Read by machine on 12 November 2026.
- Fit questions
- Built from NCT05693909, who can take part
- Record updated
- 24 September 2025