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

Phase 2

Recruiting

• Conditions: Beta Thalassemia Major Anemia; Myelodysplastic Syndrome
• Interventions: Drug: SP-420

• Registration Number: NCT05693909
• Lead Sponsor: Pharmacosmos A/S

Brief 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.

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

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2022-12-22
• Study First Submitted QC: 2023-01-20
• Study First Posted: 2023-01-23
• Study Start: 2023-09-04
• Status Verified: 2025-09
• Last Update Submitted: 2025-09-19
• Last Update Posted: 2025-09-24
• Primary Completion: 2027-10
• Study Completion: 2028-05-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 90

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
Cohort 1a-1b	SP-420	SP-420 for 48 weeks
Cohort 3a-3b	SP-420	SP-420 for 48 weeks
Cohort 2a-2b	SP-420	SP-420 for 48 weeks
1c-1d	SP-420	SP-420 for 48 weeks
2c-2d	SP-420	SP-420 for 48 weeks
3c-3d	SP-420	SP-420 for 48 weeks

Primary Outcome Measures

Name	Time	Method
To establish dose-response relationship of SP-420 for 24 weeks in the treatment of subjects with transfusion-dependent β-thalassemia	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	12 weeks

Secondary Outcome Measures

Name	Time	Method
To assess the efficacy of SP-420 in clearing iron from the liver after 24 weeks treatment of subjects with transfusion-dependent β-thalassemia	24 weeks	Change in liver iron concentration (LIC) measured by R2-magnetic resonance imaging (MRI) from baseline to week 24
To assess the efficacy of SP-420 on serum (s-) ferritin	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
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	12 and 48 weeks	Change in LIC measured by R2-MRI from baseline to week 12 and week 48
To assess the safety and tolerability of ascending doses of SP-420	48 weeks	Type and incidence of adverse events (AEs)

Trial Locations

Locations (1)

Pharmacosmos Investigational Site; 🇩🇰 Copenhagen, Denmark