A Phase 2, Open Label, Multicenter Study to Evaluate the Safety, Tolerability, Efficacy, and Pharmacokinetics of Sebelipase Alfa in Infants with Rapidly Progressive Lysosomal Acid Lipase Deficiency

Alexion Pharmaceuticals Inc0 sites10 target enrollmentStarted: April 24, 2014Last updated: February 28, 2019

Trial Snapshot

No summary available.

•A subject must meet all of the following criteria to be eligible to participate in
•this study:
•1. Subject’s parent or legal guardian (if applicable) consents to participation in the study.
•2. Confirmation of documented decreased LAL activty relative to the normal range of the lab performing the assay or a documented result of molecular genetic testing confirming a diagnosis of LAL deficiency.
•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:
•a) Marked abdominal distension and hepatomegaly
•b) Failure to thrive as evidenced by:
•i) Weight for height is 2 or more SD below the mean for gender and age
•ii) Weight curve has crossed downward by more than 2 major percentile lines on the WHO growth curves (1st, 3rd, 5th, 10th,
•25th, 50th, 75th, 90th, 95th, 97th, 99th) after having previously achieved a stable pattern of growth
•c) Disturbance of coagulation (e.g., requirement for FFP; two values of PT >15 sec or PTT > 40 sec)
•d) Severe anemia (e.g., requirement for blood transfusion or hemoglobin < 8 g/dL)
•e) Sibling with rapidly progressive course of LAL Deficiency
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 10
•F.1.2 Adults (18-64 years) no
•F.1.2.1 Number of subjects for this age range
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

•A subject who meets any of the following criteria will be ineligible to
•participate in this study:
•1. Clinically important concurrent disease or co-morbidities which, in the opinion of the Investigator and Sponsor, would interfere with study participation, including, but not restricted to:
•a) Additional severe congenital abnormality
•b) Presence of severe infection that requires treatment with parenteral anti-infective treatment within the past 14 days
•c) Previous history of circulatory collapse requiring inotropic support for more than 48 hours
•d) Congestive heart failure
•e) Acute or chronic renal failure
•f) Other extenuating circumstances such as life-threatening under nutrition or rapidly progressive liver disease
•2. Subject will be > 8 months of age at the time of first dosing.
•3. Subject has 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.

Sponsor