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 @ 9:54 PM
Ignite Modification Date:
2025-12-24 @ 9:54 PM
NCT ID:
NCT04877132
Eligibility Criteria:
Inclusion Criteria:
All patients (adult and pediatric)
* Unsolicited request for the patient from a site with at least 3 years of experience in administration and safety management of ERT.
* Written informed consent signed by the patient or the patient's parent(s)/guardian(s), where applicable.
* Documented deficiency of acid sphingomyelinase in peripheral leukocytes, lymphocytes, or cultured fibroblasts.
Adult patients
* Age ≥ 18 years.
* Clinically documented advanced disease evidenced by defined thresholds for lung, spleen, liver, and hematologic parameters.
Pediatric patients
\- Age \>3 years and \<18 years or clinical diagnosis consistent with ASMD Type A/B or Type B.
Exclusion Criteria:
All patients (adult and pediatric)
* Active serious intercurrent illness which will preclude enrollment, significant liver disease with etiology other than ASMD, Malignancy with poor prognosis, serious medical or psychiatric condition that may preclude participation, or circumstances that may interfere with compliance in this compassionate use program, requirement for recurrent dose adjustment of anticoagulation treatment over the last 6 months.
* Pregnancy or breastfeeding.
* For female patients of childbearing potential, a positive serum pregnancy (β human chorionic gonadotropin \[HCG\]) test result.
* For female patients of childbearing potential and sexually active male patients, unwillingness to abstain from heterosexual intercourse in accordance with their preferred and usual lifestyle, or to use 2 acceptable, effective contraceptive methods, while participating in this program and for 15 days after the last infusion of olipudase alfa.
* For pediatric patients, clinical diagnosis, or suspicion of infantile onset ASMD. Genotype compatible with ASMD type A.
Sex:
ALL
Minimum Age:
3 Years
Study:
NCT04877132
Study Brief:
Protocol Section:
NCT04877132