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 @ 1:43 AM
Ignite Modification Date:
2025-12-25 @ 1:43 AM
NCT ID:
NCT02112994
Eligibility Criteria:
Key Inclusion Criteria:
1. Participant was \>8 months of age at the time of dosing.
2. Confirmation of LAL-D diagnosis as determined by the central laboratory or, for participants with prior hematopoietic stem cell transplant or liver transplant, historical enzyme activity or molecular genetic testing confirming a diagnosis of LAL-D.
3. Participants \>8 months but \<4 years of age at Screening had at least 1 of the following documented clinical manifestations of LAL-D:
* Dyslipidemia
* Elevated transaminases
* Impaired growth
* Suspected malabsorption
* Other clinical manifestation of LAL-D
4. Participants ≥4 years of age at Screening had at least 1 of the following documented clinical manifestations of LAL-D:
* Evidence of advanced liver disease
* Histologically confirmed disease recurrence in participants with past liver or hematopoietic transplant
* Persistent dyslipidemia
* Suspected malabsorption
* Other clinical manifestation of LAL-D
Key Exclusion Criteria:
1. Participant had known causes of active liver disease other than LAL-D, which had not been adequately treated.
2. Participant received a hematopoietic stem cell or liver transplant \<2 years from the time of dosing.
3. Participant with co-morbidities other than complications due to LAL-D, which were irreversible or associated with a high mortality risk within 6 months or would interfere with study compliance or data interpretation.
Healthy Volunteers:
False
Sex:
ALL
Minimum Age:
8 Months
Study:
NCT02112994
Study Brief:
Protocol Section:
NCT02112994