A Trial Testing SP-420 in Subjects With Transfusion-dependent β-thalassemia or Low-risk Myelodysplastic Syndromes
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
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
Treatments tested in this trial
- SP-420
Treatment groups
90 Participants
are divided into 6 treatment groups6
Treatment groupsSee each treatment group below.
Sponsors and collaborators
Pharmacosmos A/S
Lead sponsor
ICON plc
Collaborator
Source ClinicalTrials.gov