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

ConditionsBeta Thalassemia Major AnemiaMyelodysplastic Syndrome
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorPharmacosmos A/S

About this trial

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

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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

Disqualifiers

β-thalassemia with the structural Hb variants HbS and HbC

Current MDS

Current biliary disorder

Historic or ongoing clinically significant kidney disease

Trial design

Design model

Sequential

Treatments tested in this trial

  • SP-420

    Drug

    Capsules for oral intake

Treatment groups

90 Participants
are divided into 6 treatment groups

6

Treatment groups

See each treatment group below.

Group A: Cohort 1a-1bExperimental treatment 1 intervention
Group B: Cohort 2a-2bExperimental treatment 1 intervention
Group C: Cohort 3a-3bExperimental treatment 1 intervention
Group D: 1c-1dExperimental treatment 1 intervention
Group E: 2c-2dExperimental treatment 1 intervention
Group F: 3c-3dExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

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
2

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

Secondary outcomes

1

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

Time frame
24 weeks
2

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

Time frame
12 and 48 weeks
3

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

Time frame
up to 48 weeks
4

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

Type and incidence of adverse events (AEs)

Time frame
48 weeks

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Pharmacosmos A/S

Lead sponsor

ICON plc

Collaborator

Source ClinicalTrials.gov
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