TerminatedPhase 2

Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency

United States, Finland, Italy10 participantsStarted 2014-06-06

Plain-language summary

This was an open-label, repeat-dose, study of sebelipase alfa in infants with rapidly progressive lysosomal acid lipase deficiency (LAL-D). Eligible participants received once-weekly infusions of sebelipase alfa for up to 3 years.

Who can participate

Age range8 Months

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Participant's parent or legal guardian (if applicable) consent to participation in the study
✓. 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
✓. 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:

Exclusion criteria

✕. Clinically important concurrent disease
✕. Participant was \> 8 months of age at the time of first dosing
✕. Participant received an investigational medicinal product other than sebelipase alfa within 14 days prior to the first dose of sebelipase alfa in this study
✕. Myeloablative preparation, or other systemic pre-transplant conditioning, for hematopoietic stem cell or liver transplantation
✕. Previous hematopoietic stem cell or liver transplant
✕. Known hypersensitivity to eggs

What they're measuring

Participants Experiencing Severe Treatment-emergent Adverse Events (TEAEs)

Timeframe: Screening through Month 37

Trial details

NCT IDNCT02193867

SponsorAlexion Pharmaceuticals, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2018-10-30

Results submitted2019-07-26

View on ClinicalTrials.gov More Lysosomal Acid Lipase Deficiency trials