A Trial Testing SP-420 in Subjects With Transfusion-dependent β-thalassemia or Low-risk Myelodysp… (NCT05693909) | Clinical Trial Compass
RecruitingPhase 2
A Trial Testing SP-420 in Subjects With Transfusion-dependent β-thalassemia or Low-risk Myelodysplastic Syndromes
Denmark90 participantsStarted 2023-09-04
Plain-language 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
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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
* Uncontrol…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
To establish dose-response relationship of SP-420 for 24 weeks in the treatment of subjects with transfusion-dependent β-thalassemia
Timeframe: 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