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Recruiting NCT05693909

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

Phase II Interventional Beta Thalassemia Major Anemia Myelodysplastic Syndrome

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: SP-420.
Who it may be relevant to
Registry conditions: Beta Thalassemia Major Anemia, Myelodysplastic Syndrome. Basic parameters: from 18 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Denmark
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

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

Overview

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

Interventions

  • Drug SP-420
    Capsules for oral intake

Primary outcome measures

  • 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]
Secondary outcome measures (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 [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]

Eligibility criteria

Thalassemia cohorts:

Inclusion criteria

  • 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

Exclusion criteria

  • 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:

Inclusion criteria

  • 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

Exclusion criteria

  • 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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Randomized
Model
Sequential
Masking
Open label
Primary purpose
Treatment

Study locations

Denmark · 1 center
  • Pharmacosmos Investigational Site — Copenhagen

Identifiers

NCT: NCT05693909 · P-SP420-THAL-01

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗