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Study on the Effectiveness of Decitabine and Tetrahydrouridine in Patients with Sickle Cell Disease Not Eligible for Hydroxyurea Treatment

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What is this study about?

This clinical trial is focused on studying the effects of a new treatment for sickle cell disease, a condition that affects the red blood cells and can cause pain and other serious health problems. The treatment being tested is a combination of two substances, decitabine and tetrahydrouridine, which are taken as a capsule. The study will compare this treatment to a placebo to see how well it works in improving hemoglobin levels in patients who cannot use another medication called hydroxycarbamide.

The purpose of the study is to evaluate the effectiveness and safety of the decitabine-tetrahydrouridine combination. Participants will take the medication orally over a period of time, and their health will be monitored regularly. The study will last for several weeks, during which changes in hemoglobin and other health markers will be observed. The goal is to determine if the new treatment can help improve the condition of people with sickle cell disease.

Throughout the study, participants will undergo various assessments to track their progress. These assessments will include measuring the concentration of the medication in the blood and observing any changes in the number of painful episodes or other complications related to sickle cell disease. The study aims to provide valuable information on whether this new treatment can offer a better option for managing sickle cell disease in patients who are not eligible for existing treatments.

1 joining the study

Upon joining the study, eligibility is confirmed based on specific criteria such as age, diagnosis of sickle cell disease, and recent medical history.

The study is designed for individuals who are not eligible for hydroxyurea treatment.

2 initial assessment

An initial assessment is conducted to establish baseline health metrics, including haemoglobin levels and reticulocyte count.

This assessment helps in measuring changes throughout the study.

3 medication administration

Participants receive either the Decitabine/Tetrahydrouridine combination or a placebo.

The medication is administered orally in the form of hard capsules.

The dosage is 5 mg of Decitabine and 250 mg of Tetrahydrouridine.

4 treatment period

The treatment period lasts for 24 weeks.

During this time, regular monitoring of haemoglobin levels and other health indicators is conducted.

5 follow-up assessments

Follow-up assessments occur at specified intervals to evaluate the maximum concentration of Decitabine and Tetrahydrouridine in the blood.

Other health metrics, such as foetal haemoglobin levels and the number of vaso-occlusive crises, are also monitored.

6 end of treatment evaluation

At the end of the 24-week treatment period, a comprehensive evaluation is conducted.

This includes measuring changes in haemoglobin levels and assessing any adverse events.

7 extended monitoring

Participants are monitored for an additional 24 weeks to track long-term effects and any further health changes.

The study concludes with a final assessment to gather all necessary data.

Who Can Join the Study?

  • Must be 18 years or older at the time of signing the consent form.
  • Must have a confirmed diagnosis of sickle cell disease. This includes different types like HbSS, HbSC, HbSβ0 thalassemia, HbSβ+ thalassemia, or other variants.
  • Must have had 2 to 10 episodes of a condition called VOCs (vaso-occlusive crises) in the last 12 months before the screening visit. VOCs are painful episodes that occur in sickle cell disease.
  • Must have a hemoglobin level between 5.0 g/dL and 10.5 g/dL at the first visit. Hemoglobin is a protein in red blood cells that carries oxygen.
  • Must have an absolute reticulocyte count above the ULN (Upper Limit of Normal) at the first visit. Reticulocytes are young red blood cells, and this count helps assess bone marrow activity.
  • Must have a body weight between 40 kg and 125 kg (inclusive).

Who Cannot Join the Study?

  • Patients who are eligible for treatment with hydroxyurea (HU) cannot participate. Hydroxyurea is a medication used to treat sickle cell disease.
  • Patients who do not have sickle cell disease cannot participate. Sickle cell disease is a group of inherited red blood cell disorders.
  • Patients who are not within the specified age range for the study cannot participate. The study is for certain age groups only.
  • Patients who are part of a vulnerable population cannot participate. A vulnerable population includes groups that may need special protection.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
CHU Grenoble Alpes La Tronche France

Other Sites

Site Name City Country Status
Centro Ricerche Cliniche Di Verona S.r.l. Verona Italy
Nosokomeio Paidon I Agia Sofia Athens Greece
General Hospital Of Larissa Koutlibaneio And Triantafylleio Larissa Greece
Azienda Ospedaliera di Padova Padua Italy
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico Milan Italy
Ente Ospedaliero Ospedali Galliera Di Genova Genoa Italy
Centre Hospitalier Lyon Sud Pierre Benite France
Fuetjkenf Pufv Lf Iygskrhvdygaa Bozpippur Dno Hqmdhkpm Uluwwwbjrjfqp Lf Plh Madrid Spain

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

Trial status

Country Status Recruitment Start
France France
Not recruiting
07.07.2022
Greece Greece
Not recruiting
07.07.2022
Italy Italy
Not recruiting
07.07.2022
Spain Spain
Not recruiting
07.07.2022

Trial locations

Investigated drugs:

Decitabine-Tetrahydrouridine (NDec) is a combination medication being studied for its potential to improve hemoglobin levels in patients with sickle cell disease. This medication is taken orally and is being evaluated for its effectiveness in patients who are not eligible for treatment with hydroxyurea. The goal of the trial is to determine if this combination can provide a beneficial effect on hemoglobin levels, which is a critical factor in managing sickle cell disease.

Investigated diseases:

Sickle Cell Disease – Sickle cell disease is a genetic disorder that affects the red blood cells, causing them to become rigid and shaped like a sickle or crescent. These abnormally shaped cells can block blood flow in small blood vessels, leading to pain and potential organ damage. The disease is characterized by episodes of pain, known as vaso-occlusive crises, and can also cause anemia due to the rapid breakdown of the sickle cells. Over time, sickle cell disease can lead to complications such as acute chest syndrome, stroke, and increased risk of infections. The severity and frequency of symptoms can vary widely among individuals. The condition is inherited in an autosomal recessive pattern, meaning both copies of the gene in each cell have mutations.

Trial ID:
2023-508506-22-00
Protocol code:
NN7533-4470
Trial Phase:
Therapeutic exploratory (Phase II)

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