Study on Rilzabrutinib for Patients Aged 10-65 with Sickle Cell Disease

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What is this study about?

This clinical trial is focused on studying the effects of a medication called rilzabrutinib in patients with sickle-cell disease. Sickle-cell disease is a blood disorder that can cause episodes of pain known as vaso-occlusive crises. The purpose of the study is to evaluate how well rilzabrutinib can prevent these painful episodes in patients aged 10 to 65 years.

Participants in the study will be randomly assigned to receive either rilzabrutinib or a placebo, which is a substance with no active medication. The study will last for 52 weeks, during which time the safety and effectiveness of rilzabrutinib will be closely monitored. The study aims to see if rilzabrutinib can reduce the number of painful episodes and improve the overall health of patients with sickle-cell disease.

Throughout the study, participants will have regular check-ups to monitor their health and any changes in their condition. The study will also track the time it takes for the first painful episode to occur, the number of visits to the doctor due to complications, and any changes in fatigue levels. The goal is to gather comprehensive information on how rilzabrutinib affects patients with sickle-cell disease and to ensure the treatment is safe and effective.

1 joining the study

Upon joining the study, participants will be randomly assigned to receive either the study medication, rilzabrutinib, or a placebo. A placebo is a tablet that looks like the medication but does not contain the active substance.

The study is designed to be ‘double-blind,’ meaning neither the participants nor the researchers will know who is receiving the actual medication or the placebo.

2 medication administration

Participants will take the assigned tablets orally. The exact dosage and frequency will be provided by the study team and must be followed as instructed.

The duration of the medication administration is up to 52 weeks, during which participants will continue to take the tablets as directed.

3 regular check-ups

Participants will attend regular check-ups at the study center. These visits are important to monitor health and any effects of the medication.

During these visits, various assessments will be conducted, including blood tests and other evaluations to ensure safety and measure the effectiveness of the treatment.

4 recording symptoms

Participants will be asked to record any symptoms or health changes in a diary provided by the study team. This includes any episodes of pain related to sickle-cell disease.

The diary helps track the frequency and severity of symptoms, which is crucial for evaluating the treatment’s impact.

5 final evaluation

At the end of the 52-week period, participants will undergo a final evaluation. This will include a comprehensive review of health status and any changes experienced during the study.

The results from this evaluation will contribute to understanding the efficacy and safety of rilzabrutinib in treating sickle-cell disease.

Who Can Join the Study?

Participants must have been diagnosed with sickle-cell disease (SCD).
Participants must have experienced between 2 to 10 episodes of documented acute clinical vaso-occlusive crisis (VOC) within 12 months before the screening visit. VOC is an acute painful crisis that occurs in SCD patients.
Participants should either not be taking hydroxyurea and/or L-glutamine at the screening visit and not plan to take them during the study, or they should have been taking these medications for at least 6 months. If they are on these medications, they must have been on a stable dose for at least 3 months before the screening visit and plan to continue the same dose during the study, unless there are safety concerns.
Participants must have an Eastern Cooperative Oncology Group (ECOG) performance status of grade 2 or lower. This is a scale used to assess how a disease affects a patient’s daily living abilities, with lower numbers indicating better functioning.
Participants must use contraception methods that comply with local regulations for clinical study participants.
For participants aged 10 to under 18 years, a parent or legal guardian must provide written informed consent before any study-related procedures are performed.

Who Cannot Join the Study?

Patients with any other blood and lymphatic diseases cannot participate. These are diseases that affect the blood, bone marrow, lymph, and lymphatic system.
Patients who are not within the specified age range for the study cannot participate. The age range is not specified here, but it is important to check if you meet the age requirements.
Patients who are part of a vulnerable population cannot participate. This means groups of people who might be at a higher risk of harm or exploitation.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country
Azienda Ospedaliero Universitaria Careggi	Florence	Italy
Oncopole Claudius Regaud	Toulouse	France
Hopital Necker Enfants Malades	Paris	France
Azienda Ospedaliera Universitaria Universita’ Degli Studi Della Campania Luigi Vanvitelli	Naples	Italy
Katholieke Universiteit te Leuven	Leuven	Belgium
Université Libre de Bruxelles – Hôpital Erasme	Brussels	Belgium

Other Sites

Site Name	City	Country
Centro Ricerche Cliniche Di Verona S.r.l.	Verona	Italy
Hospital Sant Joan De Deu Barcelona	Esplugues De Llobregat	Spain
Hospital General Universitario Gregorio Maranon	Madrid	Spain
Nosokomeio Paidon I Agia Sofia	Athens	Greece
Robert Bosch Gesellschaft fuer medizinische Forschung mbH	Stuttgart	Germany
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico	Milan	Italy
Azienda Ospedaliero-Universitaria San Luigi Gonzaga	Orbassano	Italy
Ente Ospedaliero Ospedali Galliera Di Genova	Genoa	Italy
General University Hospital Of Patras	Patras	Greece
Ospedale Pediatrico Bambino Gesu’	Rome	Italy
Charite Universitaetsmedizin Berlin KöR	Berlin	Germany
Medical Center – University Of Freiburg	Freiburg Im Breisgau	Germany
Association Hospitaliere De Bruxelles Et De Schaerbeek Centre Hospitalier Universitaire Brugmann	Brussels	Belgium
Centre Hospitalier Regional De La Citadelle	Liege	Belgium
Uferwzzoiomoedfieyfbf Erxmw Abo	Essen	Germany
Ljixe Gdpbttm Hffpulgm Or Afmznl	Athens	Greece
Acrqcqm Otjjxrnahin Oefopkgt Rfsipab Vpcqg Szgzh Cxniuquw	Palermo	Italy
Atnegjfqnk Ptjjczxd Hxkcjhaq Dv Mmkngvtjs	Marseille	France
Eynznss Ujqpxwxgawcm Mtjypyb Cndlzzg Reluqcjlt (osaibnu Mzf	Rotterdam	The Netherlands
Fzpjmmsrb Pmst Lo Ibjxkwyfsznyc Bdtljafts Dqq Hmpxlecf Uraoxrozsfkbx Lp Piu	Madrid	Spain
Hgylznq Hsbol Mixvkg &yplvnr 1 rys Gziiixw Eqmibh	Creteil	France

Trial status

Country	Status	Recruitment Start
Belgium	Recruiting	15.05.2025
France	Recruiting	15.05.2025
Germany	Not yet recruiting	15.05.2025
Greece	Recruiting	15.05.2025
Italy	Recruiting	15.05.2025
Spain	Recruiting	15.05.2025
The Netherlands	Not yet recruiting	15.05.2025

Trial locations

Investigated drugs:

Rilzabrutinib

Rilzabrutinib is a medication being studied for its potential to help people with sickle-cell disease. Sickle-cell disease is a condition where the red blood cells, which carry oxygen around the body, become misshaped and can block blood flow, causing pain and other serious problems. Rilzabrutinib is being tested to see if it can prevent these painful episodes, known as vaso-occlusive crises, by helping to keep the blood flowing smoothly. This medication is taken by mouth and works by targeting specific parts of the immune system that may contribute to these blockages. The goal of the study is to see if rilzabrutinib can reduce the number of painful crises and improve the quality of life for people with sickle-cell disease.

Sickle Cell Disease – Sickle Cell Disease is a genetic blood disorder characterized by the production of abnormal hemoglobin, known as hemoglobin S. This abnormal hemoglobin causes red blood cells to become rigid, sticky, and shaped like a crescent or sickle. These sickle-shaped cells can block blood flow in small vessels, leading to episodes of pain known as vaso-occlusive crises. Over time, the disease can cause damage to organs and tissues due to reduced blood flow and oxygen delivery. The condition can also lead to anemia, as the sickle cells break down more rapidly than normal red blood cells. Sickle Cell Disease is a lifelong condition that varies in severity among individuals.

Trial ID:

2024-518645-17-00

Protocol code:

EFC17872

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study on Rilzabrutinib for Patients Aged 10-65 with Sickle Cell Disease

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