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 @ 12:52 AM
Ignite Modification Date:
2025-12-25 @ 12:52 AM
NCT ID:
NCT02193867
Eligibility Criteria:
Inclusion Criteria:
1. Participant's parent or legal guardian (if applicable) consent to participation in the study
2. Confirmation of documented decreased LAL activity relative to the normal range of the lab performing the assay or confirmation of LAL-D diagnosis as determined by a Sponsor-approved central laboratory
3. Substantial clinical concerns, in the opinion of Investigator and Sponsor, of rapid disease progression requiring urgent medical intervention including, but not restricted to the following:
* Marked abdominal distension and hepatomegaly
* Failure to thrive
* Disturbance of coagulation
* Severe anemia
* Sibling with rapidly progressive course of LAL-D
Exclusion Criteria:
1. Clinically important concurrent disease
2. Participant was \> 8 months of age at the time of first dosing
3. Participant received an investigational medicinal product other than sebelipase alfa within 14 days prior to the first dose of sebelipase alfa in this study
4. Myeloablative preparation, or other systemic pre-transplant conditioning, for hematopoietic stem cell or liver transplantation
5. Previous hematopoietic stem cell or liver transplant
6. Known hypersensitivity to eggs
Healthy Volunteers:
False
Sex:
ALL
Maximum Age:
8 Months
Study:
NCT02193867
Study Brief:
Protocol Section:
NCT02193867