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The efficacy and safety of rilzabrutinib in participants aged 10 to 65 years with sickle-cell disease
EU CTIS ID: 2024-518645-17-00
What this study is testing
To assess the efficacy of rilzabrutinib for the prevention of clinical vaso-occlusive crisis (VOC) (acute painful crisis in sickle-cell disease [SCD] patients).
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Participants who have been diagnosed with SCD.
- - Participants who have had between ≥2 and ≤10 episodes of documented clinical VOC within 12 months of the screening events.
- Participants who are either not on hydroxyurea and/or L-glutamine at the Screening Visit and does not plan to receive them during the course of the study or has received HU and/or L-glutamine for a minimum of 6 months. Participants on hydroxyurea and/or L-glutamine must have been on a stable weight-based dose level (mg/kg) for at least 3 months prior to the Screening Visit, with the intent to continue at the same weight-based dose level for the duration of the study, except for safety reasons.
- Participants with Eastern Cooperative Oncology Group (ECOG) performance status grade 2 or lower.
- Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
- For participants ≥10 to <18 years of age: the parent(s)/legal guardian(s) must provide written informed consent prior to any study-related procedures being performed.
You likely can't join if
- Participants with medical history of lymphoma, leukemia, or any malignancy within the past 5 years except for basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of metastatic disease for the past 3 years
- Clinically relevant cardiac abnormality, in the opinion of the Investigator or electrocardiogram (ECG) findings.
- Participants with history of stroke, or history of abnormal transcranial doppler.
- Participants with uncontrolled or active HBV and/or HCV infection including those receiving antiviral therapy at the time of screening.
- HIV infection.
- A history of active or latent tuberculosis (TB)
See the full eligibility criteria
- Participants who have been diagnosed with SCD.
- - Participants who have had between ≥2 and ≤10 episodes of documented clinical VOC within 12 months of the screening events.
- Participants who are either not on hydroxyurea and/or L-glutamine at the Screening Visit and does not plan to receive them during the course of the study or has received HU and/or L-glutamine for a minimum of 6 months. Participants on hydroxyurea and/or L-glutamine must have been on a stable weight-based dose level (mg/kg) for at least 3 months prior to the Screening Visit, with the intent to continue at the same weight-based dose level for the duration of the study, except for safety reasons.
- Participants with Eastern Cooperative Oncology Group (ECOG) performance status grade 2 or lower.
- Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
- For participants ≥10 to <18 years of age: the parent(s)/legal guardian(s) must provide written informed consent prior to any study-related procedures being performed.
- Participants with medical history of lymphoma, leukemia, or any malignancy within the past 5 years except for basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of metastatic disease for the past 3 years
- Clinically relevant cardiac abnormality, in the opinion of the Investigator or electrocardiogram (ECG) findings.
- Participants with history of stroke, or history of abnormal transcranial doppler.
- Participants with uncontrolled or active HBV and/or HCV infection including those receiving antiviral therapy at the time of screening.
- HIV infection.
- A history of active or latent tuberculosis (TB)
- Positive COVID-19 molecular test.
- Participant is taking or has received crizanlizumab (ADAKVEO®) within 90 days and/or voxelotor (OXBRYTA®) within 30 days prior to the Screening visit.
The study team makes the final eligibility decision.
Where it's taking place
- Kenya
- Tanzania, United Republic of
- United Kingdom
- Oman
- Brazil
- United States
- Israel
- Turkey
- Saudi Arabia
- Ghana
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years, 18-64 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Kenya; Tanzania, United Republic of; United Kingdom; Oman; Brazil; United States and 4 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.