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

2 1 1

What is this study about?

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.

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 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 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 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 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 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 Study?

  • 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 Study?

  • 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.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Other Sites

Site Name City Country Status
Azienda Ospedaliero Universitaria Di Modena Modena Italy
Geniko Nosokomeio Thessalonikis George Papanikolaou Thessaloniki Greece
Mtz Clinical Research Powered By Pratia Warsaw Poland
Nosokomeio Paidon I Agia Sofia Athens Greece
Ippokratio General Hospital Of Thessaloniki Thessaloniki Greece
Copernicus Podmiot Leczniczy Sp. z o.o. Gdansk Poland
Szpital Kliniczny Ministerstwa Spraw Wewnetrznych I Administracji Z Warminsko-Mazurskim Centrum Onkologii W Olsztynie Olsztyn Poland
General Hospital Of Larissa Koutlibaneio And Triantafylleio Larissa Greece
Hippokration Hospital Athens Greece
Specjalistyczny Szpital Im. Dra Alfreda Sokolowskiego Walbrzych Poland
University General Hospital Of Ioannina Ioannina Greece
Odense University Hospital Odense Denmark
Ospedale San Raffaele S.r.l. Milan Italy
ASST Grande Ospedale Metropolitano Niguarda Milan Italy
Fondazione IRCCS Policlinico San Matteo Pavia Italy
Ente Ospedaliero Ospedali Galliera Di Genova Genoa Italy
IRCCS Ospedale Policlinico San Martino Genoa Italy
Region Midtjylland Aarhus Denmark
ARNAS G. Brotzu Cagliari Italy
Rigshospitalet Copenhagen Denmark
Olympion Therapeftirio General Clinic Of Patras S.A. Patras Greece
Sjzazyl Wqtcjmpwgu W Ogsei Soi z otiq Opole Poland
Ltbph Ggikibi Hbrxfnvi Ov Aftjxe Athens Greece
Aasnwyk Sza z ouwh Poznan Poland
Adeaifk Uqwxo Skvdyeima Lrazlm Dd Bcwljjq Bologna Italy
Ucjrhczpqq Gxgyzzb Hqpdgurq Adzwafs Athens Greece

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Denmark Denmark
Recruiting
11.05.2023
Greece Greece
Recruiting
11.05.2023
Italy Italy
Recruiting
11.05.2023
Poland Poland
Recruiting
11.05.2023

Trial locations

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.

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 ID:
2023-507396-21-00
Protocol code:
P-SP420-THAL-01
NCT ID:
NCT05693909
Trial Phase:
Therapeutic exploratory (Phase II)

Other Trials to Consider

  • Study on the Effects of Exagamglogene Autotemcel for Patients with Transfusion-Dependent Beta-Thalassemia or Severe Sickle Cell Disease

    Recruiting

    3 1 1 1
    Investigated diseases:
    Germany Italy
  • Study on Luspatercept for Improving Brain Oxygenation in Patients with Non-Transfusion Dependent Thalassemia

    Recruiting

    3 1 1 1
    Investigated drugs:
    The Netherlands