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Study of SP-420 for Patients with Transfusion-Dependent Alpha or Beta Thalassemia

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a condition known as transfusion-dependent thalassemia, which includes both α-thalassemia and β-thalassemia. These are blood disorders that require regular blood transfusions. The trial will test a new treatment called SP-420, which is taken as a capsule. The purpose of the study is to find the right dose of SP-420 and to see how well it works in removing excess iron from the body over a period of 24 weeks.

Participants in the study will take SP-420 orally, which means they will swallow the capsules. The study will last for up to 48 weeks, during which the amount of iron in the body will be measured at different times. This will help researchers understand how much iron is removed by SP-420. The study will also monitor changes in the amount of iron in the liver using a method called magnetic resonance imaging (MRI), which is a type of scan that provides detailed images of the inside of the body.

Throughout the study, researchers will keep track of any side effects that participants might experience. The main goal is to determine how effective SP-420 is at reducing iron levels in people with transfusion-dependent thalassemia, while also ensuring the treatment is safe. Participants will be closely monitored by healthcare professionals to ensure their well-being during the trial.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the trial

    Upon joining the trial, eligibility is confirmed based on specific criteria such as age, medical condition, and treatment history.

    Participants must be willing to stop their current iron chelation therapy 7 days before starting the trial medication.

  2. Step 2

    Initial assessment

    An initial assessment is conducted to establish baseline measurements, including liver iron concentration (LIC) using R2-MRI.

    This assessment helps in understanding the participant's current health status and iron levels.

  3. Step 3

    Medication administration

    The trial involves taking a medication called SP-420 in the form of capsules.

    The medication is taken orally, and the dosage may be adjusted during the trial to find the most effective dose.

  4. Step 4

    Treatment period

    The treatment period lasts for 24 weeks, during which the participant continues to take SP-420 as directed.

    Regular monitoring is conducted to assess the medication's effects on iron levels and overall health.

  5. Step 5

    Follow-up assessments

    Follow-up assessments are scheduled at various intervals, including weeks 12, 24, and 48, to measure changes in liver iron concentration and total body iron.

    These assessments help in evaluating the effectiveness of the treatment.

  6. Step 6

    Completion of trial

    Upon completing the trial, a final assessment is conducted to determine the total body iron removed and any changes in liver iron concentration.

    Participants may discuss the results and any further steps with their healthcare provider.

Who can join the trial?

8 criteria

  • Women and men who are **18 years or older**.
  • Have **transfusion-dependent α-thalassemia** or **transfusion-dependent β-thalassemia**, including a type called **HbE/β-thalassemia**, and need treatment to remove excess iron from the body. This includes β-thalassemia with changes in the α-globin gene.
  • Have been on a **stable dose of iron chelation therapy** (treatment to remove excess iron) for at least **4 weeks** before the study starts.
  • Weigh **35 kg** (about 77 pounds) or more at the start of the study.
  • Agree to stop their current iron chelation therapy **7 days** (plus or minus 3 days) before the first dose of the study medication, SP-420, and for the entire duration of the study.
  • Have **transfusion iron overload**, which means having a liver iron concentration (LIC) between **5 and 35 mg/g dry weight**, measured by a special MRI scan within 2 weeks before starting the study.
  • Have been treated and monitored for at least the past **6 months** in a specialized center that keeps detailed medical records, including information about blood transfusions and iron chelation treatments.
  • Willing to participate in the study and sign the **informed consent form**, which is a document explaining the study and confirming their agreement to join.

Who cannot join the trial?

10 criteria

  • Individuals who are not dependent on blood transfusions for their condition.
  • Individuals who do not have **α- or β-thalassemia**, which are types of blood disorders.
  • Individuals who are not within the specified age range for the study.
  • Individuals who do not meet the health requirements set by the study.
  • Individuals who are not able to follow the study procedures or instructions.
  • Individuals who are pregnant or breastfeeding, if applicable.
  • Individuals who have other medical conditions that might interfere with the study.
  • Individuals who are taking medications that might affect the study results.
  • Individuals who have participated in another clinical trial recently.
  • Individuals who have a history of allergic reactions to the study medication.
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Investigated drugs

SP-420 is an investigational medication being studied for its potential to treat individuals with transfusion-dependent alpha or beta thalassemia. The trial aims to determine how different doses of SP-420 affect patients over a 24-week period. This medication is being tested to see if it can help reduce the need for blood transfusions in people with these types of thalassemia.

What is already known about the treatment

SP-420 – This medication is administered orally and is currently being studied in clinical trials for its effectiveness in treating transfusion-dependent alpha or beta-thalassemia. It is in the experimental stage, with ongoing research to determine the optimal dosage and its potential benefits. The main therapeutic indication is to reduce the need for blood transfusions in patients with these forms of thalassemia. At the molecular level, SP-420 works by modulating iron metabolism, which is crucial in managing thalassemia symptoms. It is classified pharmacologically as an iron chelator, aiming to balance iron levels in the body.

Investigated diseases

Transfusion-dependent α- or β-thalassemia – This is a genetic blood disorder characterized by the body's inability to produce enough hemoglobin, which is essential for carrying oxygen in the blood. Individuals with this condition often require regular blood transfusions to manage their symptoms and maintain adequate hemoglobin levels. Over time, the frequent transfusions can lead to an accumulation of iron in the body, which can cause complications if not managed properly. The disease is caused by mutations in the genes responsible for producing the alpha or beta chains of hemoglobin. Symptoms can include fatigue, weakness, and pale skin due to anemia. The condition is typically diagnosed in early childhood and requires lifelong management.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IITrial ID2023-507396-21-00Protocol codeP-SP420-THAL-01Estimated enrolment162 patientsSponsorPharmacosmos A/S

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On this site, “treatment” means an investigational medicine being studied in a clinical trial. Its safety and efficacy for the use being studied have not yet been confirmed, some participants may receive a placebo or a comparator medicine, and taking part does not guarantee any health benefit. The decision to take part is made by the doctor at the research site. This site is for information only and does not replace medical advice.

This service is not affiliated with the European Commission, the EMA, or the official CTIS system. Most information comes from publicly available international clinical-trial registries, supplemented by data from academic sites, national regulators and commercial sponsors. On this site, “treatment” and “therapy” mean a medicine being tested in a clinical trial. Its safety and effectiveness in the use being studied are not yet confirmed, some participants may receive a placebo or a comparator, and taking part does not guarantee a health benefit. The doctor at the research site decides who can take part. This site provides information, not medical advice. Certain content and visual elements on this website have been generated or enhanced using artificial intelligence (AI).