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Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency

NCT02193867 · Status: TERMINATED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2019-11-18

Study results available
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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.

Conditions

  • Lysosomal Acid Lipase Deficiency

Interventions

DRUG

Sebelipase Alfa

Sebelipase alfa is a recombinant human lysosomal acid lipase. The investigational medicinal product is an enzyme replacement therapy intended for treatment of participants with LAL-D. Dosing occurred once weekly for up to 3 years.

Sponsors & Collaborators

  • Alexion Pharmaceuticals, Inc.

    lead INDUSTRY

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
8 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2014-06-06
Primary Completion
2018-10-30
Completion
2018-10-30

Countries

  • United States
  • Finland
  • Italy
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02193867 on ClinicalTrials.gov