Eligibility Module
The Eligibility Module contains detailed information about who can participate in the clinical trial. This includes eligibility criteria, age restrictions, gender requirements, healthy volunteer status, and study population descriptions, helping researchers understand who is eligible to participate in the study.
Eligibility Module path is as follows:
Study -> Protocol Section -> Eligibility Module
Eligibility Module
Ignite Creation Date:
2025-12-25 @ 3:36 AM
Ignite Modification Date:
2025-12-25 @ 3:36 AM
NCT ID:
NCT05407805
Eligibility Criteria:
Inclusion Criteria (All Groups):
\- Confirmed diagnosis of stable SCD (HbS/S or HbS/beta-zero-thalassemia).
Additional Inclusion Criteria (No Disease Modifying Treatment Control Group):
* Have experienced ≥1 episode(s) of medical utilization (MU) VOC within 12 months prior to Screening.
* Data available for number of MU VOC(s) during the 12-month interval prior to Screening and a value for %fetal hemoglobin (HbF) collected subsequent to 1 year of age in the absence of recent transfusion.
Additional Inclusion Criteria (SCD Disease Modifying Treatment Group):
* Have experienced ≥1 episode(s) of MU VOC within 12 months prior to initiation of HU and/or crizanlizumab (whichever was initiated earlier).
* Must be on a stable dose of their SCD treatment regimen ≥8 weeks prior to Day 1 with the intent of remaining on the same dose throughout the study, unless adjustments are medically necessary due to bone marrow suppression, in accordance with published guidelines and/or product specific guidance (eg, package label). Accepted SCD disease modifying treatment regimens include:
* HU alone and/or in combination with crizanlizumab, L-glutamine and/or voxelotor; or
* Crizanlizumab alone and/or in combination with HU, L-glutamine and/or voxelotor.
* Data available for number of MU VOC(s) during the 12-month interval prior to initiation of any SCD disease modifying treatment, as described above, and a value for %HbF collected subsequent to 1 year of age, prior to initiation of any HU treatment, and in the absence of recent transfusion.
Exclusion Criteria (All Groups):
* Evidence or history of ongoing (condition or sequelae) clinically significant hematological (non-SCD), renal, endocrine, pulmonary, gastrointestinal, cardiovascular (including overt stroke but excluding silent cerebral infarct), hepatic (excluding cholelithiasis), psychiatric or neurological disease as assessed from medical records.
* Marked ongoing bone marrow suppression as evidenced by any of the following as per medical record: severe anemia, absolute neutrophil count (ANC) \<1000 mm3 white blood cell (WBC), thrombocytopenia (platelet count \<100,000 mm3) within ≤8 weeks prior to Day 1 enrollment.
* History of hematopoietic stem cell transplant or treatment with gene therapy as assessed from medical records.
* History of simple transfusion within ≤4 weeks prior to Day 1 enrollment as assessed from medical records or participant self-report.
* History of chronic transfusion/exchange transfusion within ≤12 weeks prior to Day 1 enrollment as assessed from medical records or participant self-report and/or plan to initiate such treatment during the 6-month observation period.
Additional Exclusion Criteria (No Disease Modifying Treatment Control Group):
* Participant received HU and/or crizanlizumab at any time within ≤18 months of Day 1 enrollment and treatment(s) was discontinued due to lack of efficacy (no reduction in the frequency of VOCs, documented or perceived) and/or plan to initiate said treatment(s) during the 6-month observation period.
* Participant received voxelotor or L-glutamine within ≤4 weeks of Day 1 enrollment and/or plan to initiate said treatment(s) during the 6-month observation period.
Healthy Volunteers:
False
Sex:
ALL
Minimum Age:
18 Years
Study:
NCT05407805
Study Brief:
Protocol Section:
NCT05407805