- Conditions
- Lysosomal Acid Lipase Deficiency
- Interventions
- There is no intervention in the study
- Other
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Industry
- Eligibility
- 2 Years to 16 Years
- Enrollment
- 22 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2017
- U.S. locations
- 2
- States / cities
- Atlanta, Georgia • Houston, Texas
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Showing 1–8
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- Conditions
- Lysosomal Acid Lipase Deficiency
- Interventions
- Sebelipase Alfa
- Drug
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Industry
- Eligibility
- 8 Months and older
- Enrollment
- 31 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2017
- U.S. locations
- 3
- States / cities
- Chicago, Illinois • Shreveport, Louisiana • Cincinnati, Ohio
- Conditions
- MPS I, MPS II, MPS IVA, MPS VI, Mps VII, Gaucher Disease, Type 2, Gaucher Disease, Type 3, Pompe Disease Infantile-Onset, Wolman Disease
- Interventions
- Aldurazyme (laronidase)
- Drug
- Lead sponsor
- University of California, San Francisco
- Other
- Eligibility
- 18 Years to 50 Years · Female only
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2032
- U.S. locations
- 1
- States / cities
- San Francisco, California
- Conditions
- Cholesterol Ester Storage Disease (CESD), Lysosomal Acid Lipase Deficiency, LAL-Deficiency
- Interventions
- sebelipase alfa
- Drug
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 8 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2017
- U.S. locations
- 5
- States / cities
- Eureka, California • Sacramento, California • San Francisco, California + 2 more
- Conditions
- Lysosomal Acid Lipase Deficiency
- Interventions
- Sebelipase Alfa
- Drug
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Industry
- Eligibility
- Up to 8 Months
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2018
- U.S. locations
- 1
- States / cities
- Phoenix, Arizona
- Conditions
- Cholesterol Ester Storage Disease(CESD), Lysosomal Acid Lipase Deficiency, LAL-Deficiency
- Interventions
- Sebelipase alfa 0.35 mg/kg, Sebelipase alfa 1 mg/kg, Sebelipase alfa 3 mg/kg
- Drug
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2012
- U.S. locations
- 3
- States / cities
- Stanford, California • New York, New York • Pittsburgh, Pennsylvania
- Conditions
- Lysosomal Acid Lipase Deficiency, Wolman Disease
- Interventions
- Sebelipase alfa (SBC-102)
- Drug
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Industry
- Eligibility
- Up to 24 Months
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2018
- U.S. locations
- 1
- States / cities
- Irvine, California
- Conditions
- Lysosomal Acid Lipase Deficiency
- Interventions
- Sebelipase Alfa, Placebo
- Drug
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Industry
- Eligibility
- 4 Years and older
- Enrollment
- 66 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2018
- U.S. locations
- 11
- States / cities
- Tucson, Arizona • Palo Alto, California • San Francisco, California + 8 more