Study on How Well Etavopivat Works for Adolescents and Adults with Sickle Cell Disease

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What is this study about?

This clinical trial is focused on studying sickle cell disease, a genetic condition that affects the red blood cells, causing them to become misshapen and leading to various health complications. The study will evaluate the effectiveness and safety of a treatment called etavopivat, also known by its code name FT-4202. Participants in the study will receive either etavopivat or a placebo, which is a substance with no active medication.

The purpose of the study is to determine if etavopivat is more effective than a placebo in treating adolescents and adults with sickle cell disease. Participants will take part in a series of assessments over the course of the study, which will last for a period of time. These assessments will help researchers understand how well the treatment works and its safety profile. The study will involve regular check-ups and monitoring to track any changes in the participants’ health and symptoms.

Throughout the study, researchers will pay close attention to the number of painful episodes known as vaso-occlusive crises, which are common in sickle cell disease. They will also monitor changes in hemoglobin levels, which is a protein in red blood cells that carries oxygen, and other health indicators. The goal is to see if etavopivat can reduce these painful episodes and improve overall health outcomes for those living with sickle cell disease.

1 randomization and initial assessment

Upon joining the study, participants are randomly assigned to receive either etavopivat or a placebo. This process is double-blind, meaning neither the participant nor the study team knows which treatment is being administered.

An initial assessment is conducted to confirm eligibility, including a review of medical history and a physical examination.

2 treatment phase

Participants take the assigned treatment orally in the form of a tablet. The dosage is 200 mg of etavopivat or a matching placebo.

The treatment is administered daily for the duration of the study.

3 monitoring and follow-up

Regular follow-up visits are scheduled to monitor health and response to the treatment. These visits include blood tests to measure haemoglobin levels and other relevant markers.

Participants are assessed for any vaso-occlusive crises (VOCs), which are painful episodes common in sickle cell disease.

4 evaluation of outcomes

The primary outcome is the number of VOC events requiring medical contact.

Secondary outcomes include changes in haemoglobin levels, fatigue scores, and physical performance as measured by the 6-minute walking test.

5 completion and final assessment

At the end of the study, a final assessment is conducted to evaluate the overall health and any changes experienced during the trial.

Participants are informed of the treatment they received after the study concludes.

Who Can Join the Study?

  • Must be either male or female.
  • Must be at least 12 years old at the time of signing the informed consent.
  • Must have a confirmed diagnosis of sickle cell disease (SCD). This means having a specific type of blood disorder, which should be documented through previous lab tests or a screening test.
  • Must have experienced between 2 to 15 episodes of vaso-occlusive crises (VOCs) in the past year. These are painful events caused by blocked blood flow. The episodes must be recorded in the medical records before joining the study. Memories of events without documentation do not count.
  • Must have a hemoglobin level between 5.0 and 10.0 grams per deciliter (g/dL) at the time of screening. Hemoglobin is a protein in red blood cells that carries oxygen.

Who Cannot Join the Study?

  • Patients who do not have sickle cell disease cannot participate. Sickle cell disease is a condition where red blood cells, which carry oxygen, are shaped like a sickle or crescent instead of being round.
  • Patients who are not adolescents or adults are excluded. This means the study is only for people who are teenagers or older.
  • Patients who are not able to give their own consent or are considered part of a vulnerable group may not be eligible. A vulnerable group might include people who need extra protection, like those with certain disabilities or conditions.

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
Haga Hospital Hague The Netherlands
Centre Hospitalier Universitaire Rouen Rouen France
Centro Ricerche Cliniche Di Verona S.r.l. Verona Italy
Azienda Ospedaliero Universitaria Di Modena Modena Italy
CHU Saint Pierre Brussels Belgium
Hospital General Universitario Gregorio Maranon Madrid Spain
ARNAS Garibaldi Di Catania Catania Italy
Nosokomeio Paidon I Agia Sofia Athens Greece
Fondazione IRCCS San Gerardo Dei Tintori Monza Italy
Azienda Ospedaliera di Padova Padua Italy
Hippokration Hospital Athens Greece
University General Hospital Of Thessaloniki Ahepa Thessaloniki Greece
University General Hospital Of Ioannina Ioannina Greece
Virgen del Rocío University Hospital Sevilla Spain
Azienda Ospedaliero-Universitaria San Luigi Gonzaga Orbassano Italy
Universitair Medisch Centrum Utrecht Utrecht The Netherlands
Centre Hospitalier Lyon Sud Pierre Benite France
Hopital Beaujon Clichy France
Universita Degli Studi Di Brescia Brescia Italy
University Childrens Hospital Queen Fabiola Brussels Belgium
Lffvd Gftmxgv Hhudprsz Ok Azeaec Athens Greece
Afguptj Oovnriecwzn Ohphznmx Rtgspji Vqhuj Sszev Chiejine Palermo Italy
Ahehogzbs Urz Amsterdam The Netherlands
Esvucih Uqmzcsvezcmv Mxwbqhi Cictinp Rewrpnopx (fbaijnw Mtv Rotterdam The Netherlands
Ukbaizckjx Oq Axyyvbi Edegem Belgium
Fjwzmwvhr Pety Lm Icyarrtthjrlg Bmyktpsgc Dhv Hbkyxcqz Uaehjtuxhokaf Lt Pwd Madrid Spain
Hsswjpbw Vjac desmxgfg Barcelona Spain

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

Trial status

Country Status Recruitment Start
Belgium Belgium
Recruiting
16.12.2024
France France
Recruiting
16.12.2024
Greece Greece
Recruiting
16.12.2024
Italy Italy
Recruiting
16.12.2024
Spain Spain
Recruiting
16.12.2024
The Netherlands The Netherlands
Recruiting
16.12.2024

Trial locations

Investigated drugs:

Etavopivat is a medication being studied for its potential to help people with sickle cell disease. Sickle cell disease is a condition where red blood cells become misshapen and can cause pain and other serious health problems. Etavopivat is being tested to see if it can improve the health of these red blood cells, making them more flexible and less likely to cause blockages in blood vessels. This study aims to determine if etavopivat is more effective than a placebo in treating adolescents and adults with 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 crescent or sickle. 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, which can vary in frequency and severity. Over time, sickle cell disease can lead to complications such as anemia, increased risk of infections, and damage to organs like the spleen and liver. The condition is inherited and is most common in individuals of African, Mediterranean, or Middle Eastern descent. Regular medical care and monitoring are essential to manage symptoms and prevent complications.

Trial ID:
2023-509175-16-00
Protocol code:
NN7535-7807
Trial Phase:
Therapeutic confirmatory (Phase III)

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