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:
2025-12-24 @ 3:55 PM
Ignite Modification Date:
2025-12-24 @ 3:55 PM
NCT ID:
NCT05529992
Eligibility Criteria:
Inclusion:
* Has a documented, confirmed diagnosis of type 1 Gaucher disease based on the following, as determined by the investigator:
1. Decreased glucocerebrosidase (GCB) activity level that is ≤30% of normal or
2. Decreased GCB activity level that is \>30% of normal, but with confirmation of genetic mutation test
* Is at least 2 years of age, inclusive, at screening
* Is naive to treatment for Gaucher disease (Has not received treatment for Gaucher disease \[investigational or approved products\] within the 12 months prior to screening) OR Is receiving or has recently received Imiglucerase ERT (Has received Imiglucerase treatment within the 12 months prior to screening and not within the 14 days prior to screening)
* Has Gaucher disease-related hematological abnormalities, defined as
1. Hemoglobin levels of ≥1 g/dL below the lower limit of normal for their age and gender AND/OR
2. A platelet count of \<90 × 10\^9/L below the lower limit of normal for their age and gender
* Has Gaucher disease-related viscera abnormalities, defined as the following:
* Participant has at least moderate splenomegaly, assessed by palpation (2 to 3 cm below the left costal margin), or by abdominal radiology scan (magnetic resonance imaging \[MRI\] or computed tomography \[CT\] scan, with spleen volume \>5 times normal) AND/OR
* Participant has hepatomegaly, assessed by palpation or by abdominal radiology scan (MRI or CT scan); Participants who have undergone splenectomy must have satisfied these criteria for this study.
Exclusion:
* Has type 2 or 3 Gaucher disease or is suspected of having type 3 Gaucher disease as assessed by the investigator
* Has had a splenectomy or an active, clinically significant spleen infarction within the 12 months prior to screening
* Has received treatment with any investigational drug or device within 30 days prior to screening, or within 5 half-lives of that investigational product, whichever is greater; such treatment during the study will not be permitted
* Is currently receiving red blood cell growth factor (eg, erythropoietin), chronic systemic corticosteroids, or has been on such treatment within the 6 months prior to screening
* Presents with non-Gaucher disease related exacerbated anemia at screening
* Has experienced a severe (grade 3 or higher) infusion-related hypersensitivity reaction (anaphylactic or anaphylactoid reaction) to any ERT (approved or investigational)
Healthy Volunteers:
False
Sex:
ALL
Minimum Age:
2 Years
Study:
NCT05529992
Study Brief:
Protocol Section:
NCT05529992