- Conditions
- Gaucher Disease
- Interventions
- Plant cell expressed recombinant glucocerebrosidase (prGCD)
- Drug
- Lead sponsor
- Pfizer
- Industry
- Eligibility
- 18 Years and older
- U.S. locations
- 14
- States / cities
- La Jolla, California • Aurora, Colorado • Coral Springs, Florida + 11 more
Search Results
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Showing 1–24
of 119
matching trials from the live ClinicalTrials.gov search.
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- 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
- Conditions
- Gaucher's Disease
- Interventions
- CEREDASE™
- Drug
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
- NIH
- Eligibility
- Not listed
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1993 – 2000
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Mucopolysaccharidosis IVA, Morquio A Syndrome, MPS IVA
- Interventions
- BMN 110
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 5 Years and older
- Enrollment
- 16 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2014
- U.S. locations
- 2
- States / cities
- Oakland, California • Chicago, Illinois
- Conditions
- Mucopolysaccharidosis Type IH, MPS I, Hurler Syndrome
- Interventions
- Laronidase
- Drug
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Other
- Eligibility
- Not listed
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2018
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Hypophosphatasia
- Interventions
- ALXN1850
- Biological
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years to 64 Years
- Enrollment
- 15 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2022
- U.S. locations
- 4
- States / cities
- Las Vegas, Nevada • Columbus, Ohio • Nashville, Tennessee + 1 more
- Conditions
- Short Stature, Idiopathic
- Interventions
- Fresh Frozen Plasma
- Other
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Other
- Eligibility
- 18 Years and older · Female only
- Enrollment
- 1 participant
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015
- U.S. locations
- 1
- States / cities
- Cincinnati, Ohio
- Conditions
- Mucopolysaccharidosis IV A, Morquio A Syndrome, MPS IVA
- Interventions
- BMN 110 - Weekly, BMN 110 - Every Other Week
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 5 Years and older
- Enrollment
- 173 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2016
- U.S. locations
- 10
- States / cities
- Phoenix, Arizona • Oakland, California • Orange, California + 7 more
- Conditions
- Gaucher Disease
- Interventions
- Standard of Care
- Other
- Lead sponsor
- Takeda
- Industry
- Eligibility
- Up to 5 Years
- Enrollment
- 11 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2023
- U.S. locations
- 1
- States / cities
- Fairfax, Virginia
- Conditions
- Cystic Fibrosis, Growth Failure, Exocrine Pancreatic Insufficiency
- Interventions
- Not listed
- Lead sponsor
- Seattle Children's Hospital
- Other
- Eligibility
- Up to 3 Months
- Enrollment
- 231 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2015
- U.S. locations
- 28
- States / cities
- Birmingham, Alabama • Little Rock, Arkansas • Aurora, Colorado + 24 more
- Conditions
- MPS 7, Sly Syndrome, Mucopolysaccharidosis, MPS VII
- Interventions
- UX003, Placebo
- Drug · Other
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- 5 Years to 35 Years
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2016
- U.S. locations
- 4
- States / cities
- Oakland, California • Orange, California • Miami, Florida + 1 more
- Conditions
- Fibrosing Colonopathy
- Interventions
- ULTRESA, PANCREAZE, CREON, ZENPEP, other non-sponsor pancreatic enzyme replacement therapy, PERTZYE, No pancreatic enzyme replacement therapy
- Biological · Other
- Lead sponsor
- AbbVie
- Industry
- Eligibility
- 0 Months to 99 Years
- Enrollment
- 17 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2020
- U.S. locations
- 123
- States / cities
- Birmingham, Alabama • Anchorage, Alaska • Phoenix, Arizona + 102 more
- Conditions
- Mucopolysaccharidosis Type I (MPS I), Mucopolysaccharidosis Type II (MPS II), Mucopolysaccharidosis Type III (MPS III), Mucopolysaccharidosis Type VI (MPS VI), Krabbe Disease
- Interventions
- Not listed
- Lead sponsor
- University of Chicago
- Other
- Eligibility
- 1 Day to 18 Years
- Enrollment
- 19 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2016
- U.S. locations
- 3
- States / cities
- Chicago, Illinois • Minneapolis, Minnesota • Buffalo, New York
- Conditions
- Cystic Fibrosis
- Interventions
- Not listed
- Lead sponsor
- AbbVie
- Industry
- Eligibility
- 2 Years to 99 Years
- Enrollment
- 1,310 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2016
- U.S. locations
- 47
- States / cities
- Anchorage, Alaska • Little Rock, Arkansas • Long Beach, California + 41 more
- Conditions
- Fabry Disease
- Interventions
- Pegunigalsidase alfa
- Biological
- Lead sponsor
- Protalix
- Industry
- Eligibility
- 18 Years to 60 Years
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2020
- U.S. locations
- 7
- States / cities
- Birmingham, Alabama • Atlanta, Georgia • Iowa City, Iowa + 4 more
- Conditions
- Exocrine Pancreatic Insufficiency, Cystic Fibrosis
- Interventions
- Liprotamase Powder for Oral Solution
- Drug
- Lead sponsor
- Anthera Pharmaceuticals
- Industry
- Eligibility
- 28 Days and older
- Enrollment
- 15 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2017
- U.S. locations
- 21
- States / cities
- Little Rock, Arkansas • Orange, California • Aurora, Colorado + 17 more
- Conditions
- Spinal Cord Compression, Mucopolysaccharidosis I, Hurler-Scheie Syndrome, Scheie Syndrome, Lysosomal Storage Disease
- Interventions
- laronidase
- Drug
- Lead sponsor
- Patricia I. Dickson, M.D.
- Individual
- Eligibility
- 8 Years and older
- Enrollment
- 3 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2011
- U.S. locations
- 1
- States / cities
- Torrance, California
- Conditions
- Acid Sphingomyelinase Deficiency, Niemann-Pick Disease
- Interventions
- rhASM
- Drug
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 11 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2009
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Adenosine Deaminase Severe Combined Immunodeficiency
- Interventions
- elapegademase-lvlr
- Biological
- Lead sponsor
- Chiesi Farmaceutici S.p.A.
- Industry
- Eligibility
- 0 Months to 65 Years
- Enrollment
- 32 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2023
- U.S. locations
- 14
- States / cities
- Los Angeles, California • San Diego, California • San Francisco, California + 11 more
- Conditions
- Pancreatic Cancer, Esophageal Cancer, Gastric Cancer
- Interventions
- Nutritional Intervention, Physical Activity Intervention
- Combination Product · Behavioral
- Lead sponsor
- Siobhan Trotter
- Other
- Eligibility
- 25 Years to 80 Years
- Enrollment
- 60 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2019
- U.S. locations
- 1
- States / cities
- New Orleans, Louisiana
- Conditions
- Exocrine Pancreatic Insufficiency, Cystic Fibrosis
- Interventions
- Liprotamase
- Drug
- Lead sponsor
- Anthera Pharmaceuticals
- Industry
- Eligibility
- 7 Years and older
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2018
- U.S. locations
- 3
- States / cities
- Aurora, Colorado • Jackson, Mississippi • Burlington, Vermont
- Conditions
- Late-onset Pompe Disease
- Interventions
- Not listed
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2018
- U.S. locations
- 17
- States / cities
- Irvine, California • Gainesville, Florida • Decatur, Georgia + 14 more
- Conditions
- Pompe Disease (Late-onset)
- Interventions
- Clenbuterol, Placebos
- Drug
- Lead sponsor
- Duke University
- Other
- Eligibility
- 18 Years and older
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2025
- U.S. locations
- 1
- States / cities
- Durham, North Carolina
- Conditions
- Fabry Disease
- Interventions
- Agalsidase alfa
- Drug
- Lead sponsor
- Shire
- Industry
- Eligibility
- 7 Years to 17 Years
- Enrollment
- 17 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2004 – 2011
- U.S. locations
- 13
- States / cities
- Tucson, Arizona • Palm Beach Gardens, Florida • Lake Charles, Louisiana + 9 more