NCT05693909

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

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
90

participants targeted

Target at P50-P75 for phase_2

Timeline
24mo left

Started Sep 2023

Longer than P75 for phase_2

Geographic Reach
1 country

1 active site

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress57%
Sep 2023May 2028

First Submitted

Initial submission to the registry

December 22, 2022

Completed
1 month until next milestone

First Posted

Study publicly available on registry

January 23, 2023

Completed
7 months until next milestone

Study Start

First participant enrolled

September 4, 2023

Completed
4.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 1, 2027

Expected
7 months until next milestone

Study Completion

Last participant's last visit for all outcomes

May 1, 2028

Last Updated

September 24, 2025

Status Verified

September 1, 2025

Enrollment Period

4.1 years

First QC Date

December 22, 2022

Last Update Submit

September 19, 2025

Conditions

Beta Thalassemia Major AnemiaMyelodysplastic Syndrome

Outcome Measures

Primary Outcomes (2)

  • 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 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

    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

    12 and 48 weeks

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

    up to 48 weeks

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

    48 weeks

Study Arms (6)

Cohort 1a-1b

EXPERIMENTAL

SP-420 for 48 weeks

Drug: SP-420

Cohort 2a-2b

EXPERIMENTAL

SP-420 for 48 weeks

Drug: SP-420

Cohort 3a-3b

EXPERIMENTAL

SP-420 for 48 weeks

Drug: SP-420

1c-1d

EXPERIMENTAL

SP-420 for 48 weeks

Drug: SP-420

2c-2d

EXPERIMENTAL

SP-420 for 48 weeks

Drug: SP-420

3c-3d

EXPERIMENTAL

SP-420 for 48 weeks

Drug: SP-420

Interventions

SP-420DRUG

Capsules for oral intake

1c-1d2c-2d3c-3dCohort 1a-1bCohort 2a-2bCohort 3a-3b

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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:
  • 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
  • Therapy-related MDS or MDS with a known bone marrow fibrosis
  • Diagnosis of decompensated liver cirrhosis
  • +8 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Pharmacosmos Investigational Site

Copenhagen, Denmark

RECRUITING

MeSH Terms

Conditions

Myelodysplastic Syndromes

Interventions

SP-420

Condition Hierarchy (Ancestors)

Bone Marrow DiseasesHematologic DiseasesHemic and Lymphatic Diseases

Study Officials

  • Pharmacosmos Clinical and non-clinical Department

    Pharmacosmos A/S

    STUDY DIRECTOR

Central Study Contacts

Pharmacosmos Clinical and non-clinical Department

CONTACT

+45 5948 5959info@pharmacosmos.com

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SEQUENTIAL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

December 22, 2022

First Posted

January 23, 2023

Study Start

September 4, 2023

Primary Completion (Estimated)

October 1, 2027

Study Completion (Estimated)

May 1, 2028

Last Updated

September 24, 2025

Record last verified: 2025-09

Data Sharing

IPD Sharing
Will not share

Locations

DK(1)