Not medical advice. This website is not medical advice, a diagnosis, a match to a trial, or a recommendation to join a study. Listings are plain-English rewrites of public ClinicalTrials.gov records. Whether a site is still enrolling, and whether you qualify, can only be confirmed by the study team and your clinician. Source registry: ClinicalTrials.gov. Editorial rules: methodology.
Phase 3: testing Rilzabrutinib for Sickle Cell Disease
This is a multicenter, randomly assigned, neither you nor the study doctors know who gets the real treatment, compared with a dummy treatment, parallel-group, flexible-adaptive, group-sequential study (Part A), followed by an open-label LTE period (Part B) to investigate the how well it works, and safety of rilzabrutinib in participants with sickle-cell disease (SCD). Study details include: * Study duration: a 52-week neither you nor the study doctors know who gets the real treatment period (Part A), followed by an open-label LTE period (Part B).
Phase 3: this treatment already looked useful in smaller studies. Now it is tested in a large group, often against standard care or a dummy treatment, to see if it should be approved.
Phase labels are FDA/ICH categories applied to the registry Phase field. They are not a finding that this study succeeded.
What the registry says happens
If you join, the listed intervention(s) are Rilzabrutinib, or Placebo. The registry lists allocation as randomized. This is a multicenter, randomly assigned, neither you nor the study doctors know who gets the real treatment, compared with a dummy treatment, parallel-group, flexible-adaptive, group-sequential study (Part A), followed by an open-label LTE period (Part B) to investigate the how well it works, and safety of rilzabrutinib in participants with sickle-cell disease (SCD).
They plan to enroll about 192 people at 63 sites. Taking part typically means taking a study drug (or dummy pill) plus check-ins.
Official title (unrewritten)
A 52-week, Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group, Flexible-adaptive, Group Sequential Study to Evaluate the Efficacy and Safety of Rilzabrutinib in Participants Aged 10 to 65 Years With Sickle-cell Disease
Interventions: Rilzabrutinib, Placebo
Primary outcome (registry wording): Annualized rate of clinical VOC
Who it is for (from inclusion criteria)
Ages 10 Years to 65 Years
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 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
Who the registry excludes
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 infection and/or HCV infection including those receiving antiviral therapy at the time of screening
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 above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial
Public study contact (from the registry)
Trial Transparency email recommended (Toll free for US & Canada)
This study is not a treatment protocol and no experimental treatments are involved. Study participants may be seen as needed for clinical, translational and basic research studies, or as medically indicated.
This study will collect representative blood samples from healthy children and adults and from children and adults who have unique red blood cell features that are related to sickle cell disease. Sickle cell disease is a blood disease that limits the ability of red blood cells to carry oxygen throughout the body.
Background: Some people with the same disorder on a genetic level have more complications than others. Researchers want to look for a link between the PKLR gene and sickle cell disease (SCD) symptoms.
The sponsor posted status “Recruiting” on ClinicalTrials.gov at last ingest. Sites can pause independently. Confirm with the official record and the listed contact.
What phase is NCT06975865?
Phase 3. Phase labels are FDA/ICH categories applied to the registry Phase field. They are not a finding that this study succeeded.
Can PlainTrials tell me if I qualify for NCT06975865?
No. The inclusion and exclusion lists on this page are readable extracts of the public eligibility block. Only the study team can screen you.