Beta Thalassemia Major AnemiaPhase 2NCT05693909
A Trial Testing SP-420 in Subjects With Transfusion-dependent β-thalassemia or Low-risk Myelodysplastic Syndromes
A Phase 2 study testing SP-420 in people with Beta Thalassemia Major Anemia and Myelodysplastic Syndrome, run by Pharmacosmos A/S at 1 site in Denmark.
Trial summary
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 more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Beta Thalassemia Major Anemia, Myelodysplastic Syndrome
- What is being tested?
- SP-420
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 90 participants
- Where
- 1 siteDenmark
- Study dates
- 4 Sep 2023to May 2028
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
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- 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 join
- 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
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- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Denmark1 site, 1 recruiting
- Recruiting
Pharmacosmos Investigational Site
Copenhagen
About this study
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
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
- Being tested
- SP-420
- Medical subject headings
- Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SP-420Drug
Capsules for oral intake
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1a-1b | Experimental | SP-420 for 48 weeks Receives: Drug: SP-420 |
| Cohort 2a-2b | Experimental | SP-420 for 48 weeks Receives: Drug: SP-420 |
| Cohort 3a-3b | Experimental | SP-420 for 48 weeks Receives: Drug: SP-420 |
| 1c-1d | Experimental | SP-420 for 48 weeks Receives: Drug: SP-420 |
| 2c-2d | Experimental | SP-420 for 48 weeks Receives: Drug: SP-420 |
| 3c-3d | Experimental | SP-420 for 48 weeks Receives: Drug: SP-420 |
What is being measured
The primary outcome is the main question the study is designed to answer.
To establish dose-response relationship of SP-420 for 24 weeks in the treatment of subjects with transfusion-dependent β-thalassemia
Measured: 24 weeks
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
Measured: 12 weeks
Secondary outcomes (4)
To assess the efficacy of SP-420 in clearing iron from the liver after 24 weeks treatment of subjects with transfusion-dependent β-thalassemia
Change in liver iron concentration (LIC) measured by R2-magnetic resonance imaging (MRI) from baseline to week 24
Measured: 24 weeks
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
Change in LIC measured by R2-MRI from baseline to week 12 and week 48
Measured: 12 and 48 weeks
To assess the efficacy of SP-420 on serum (s-) ferritin
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
Measured: up to 48 weeks
To assess the safety and tolerability of ascending doses of SP-420
Type and incidence of adverse events (AEs)
Measured: 48 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Jan 2023
- Study start4 Sep 2023
- Primary completionOct 2027 (estimated)
- Study completionMay 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Beta Thalassemia Major AnemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 90 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Pharmacosmos A/S
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- ICON plc
- Responsible party
- Sponsor
- Investigators
- Pharmacosmos Clinical and non-clinical Department, Study director, Pharmacosmos A/S
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05693909
- Sponsor's protocol ID
- P-SP420-THAL-01
- Status last verified
- Sep 2025
- First submitted
- 22 Dec 2022
- Last updated
- 24 Sep 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT05693909
- Acronym / sponsor ID
- P-SP420-THAL-01
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 90 participants (estimated)
- Lead sponsor
- Pharmacosmos A/SIndustry (pharma, biotech, devices)
- Collaborators
- ICON plc
- Study start
- 4 Sep 2023 (actual)
- Primary completion
- Oct 2027 (estimated)
- Study completion
- May 2028 (estimated)
- First posted
- 23 Jan 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 6
| Arm | Type | Receives |
|---|---|---|
| Cohort 1a-1b | Experimental | Drug: SP-420SP-420 for 48 weeks |
| Cohort 2a-2b | Experimental | Drug: SP-420SP-420 for 48 weeks |
| Cohort 3a-3b | Experimental | Drug: SP-420SP-420 for 48 weeks |
| 1c-1d | Experimental | Drug: SP-420SP-420 for 48 weeks |
| 2c-2d | Experimental | Drug: SP-420SP-420 for 48 weeks |
| 3c-3d | Experimental | Drug: SP-420SP-420 for 48 weeks |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- 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
Key exclusion criteria (18 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SP-420Capsules for oral intake | Drug | 1c-1d, 2c-2d, 3c-3d, Cohort 1a-1b, Cohort 2a-2b, Cohort 3a-3b |
Primary outcome
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
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
4 secondary outcomes
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
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
To assess the efficacy of SP-420 on serum (s-) ferritin
Time frame: up to 48 weeks
To assess the safety and tolerability of ascending doses of SP-420
Time frame: 48 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Sep 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Pharmacosmos Clinical and non-clinical Department study director
Pharmacosmos A/S
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing SP-420 in people with Beta Thalassemia Major Anemia and Myelodysplastic Syndrome, run by Pharmacosmos A/S at 1 site in Denmark.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 24 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Denmark. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Pharmacosmos Clinical and non-clinical Department (+45 5948 5959, info@pharmacosmos.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Sep 2025; AzTrials last refreshed its copy on 1 Nov 2026.