- Conditions
- Calciphylaxis
- Interventions
- INZ-701
- Drug
- Lead sponsor
- Inozyme Pharma
- Industry
- Eligibility
- 18 Years to 69 Years
- Enrollment
- 11 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024
- U.S. locations
- 2
- States / cities
- Coral Springs, Florida • Hollywood, Florida
Search Results
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Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results are retrieved from ClinicalTrials.gov and synchronized into the directory. Search pages remain noindex by default.
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Showing 1–24
of 133
matching trials from the live ClinicalTrials.gov search.
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- Conditions
- Mucopolysaccharidosis VI
- Interventions
- N-acetylgalactosamine 4-sulfatase
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- Not listed
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2000 – 2005
- U.S. locations
- 1
- States / cities
- Novato, California
- Conditions
- Idiopathic Short Stature
- Interventions
- Vosoritide Injection, Human Growth Hormone, Placebo
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 3 Years to 11 Years
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2036
- U.S. locations
- 35
- States / cities
- California City, California • Los Angeles, California • Oakland, California + 28 more
- Conditions
- CLN2 Disease
- Interventions
- BMN190, recombinant human tripeptidyl peptidase-1 (rhTPP1)
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 2 Years and older
- U.S. locations
- 2
- States / cities
- Orange, California • Columbus, Ohio
- Conditions
- Morquio A Syndrome, Mucopolysaccharidosis IV A
- Interventions
- Not listed
- Lead sponsor
- Nadia Ali, PhD
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2018
- U.S. locations
- 1
- States / cities
- Decatur, Georgia
- Conditions
- Sickle Cell Disease
- Interventions
- Sapropterin Dihydrochloride
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 15 Years and older
- Enrollment
- 32 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2009
- U.S. locations
- 12
- States / cities
- Washington D.C., District of Columbia • Augusta, Georgia • Savannah, Georgia + 9 more
- Conditions
- Batten Disease, CLN2, Neuronal Ceroid-Lipofuscinoses
- Interventions
- CLN2 Treatment
- Other
- Lead sponsor
- Jessica Scherr
- Other
- Eligibility
- Not listed
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2025
- U.S. locations
- 1
- States / cities
- Columbus, Ohio
- Conditions
- Mucopolysaccharidosis IV Type A, Morquio A Syndrome, MPS IVA
- Interventions
- Vimizim® (elosulfase alfa)
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- Not listed
- Enrollment
- 418 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2024
- U.S. locations
- 26
- States / cities
- Phoenix, Arizona • Little Rock, Arkansas • Los Angeles, California + 22 more
- Conditions
- Achondroplasia
- Interventions
- BMN 111, Placebo
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- Up to 59 Months
- Enrollment
- 75 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2022
- U.S. locations
- 9
- States / cities
- Oakland, California • Torrance, California • Wilmington, Delaware + 6 more
- Conditions
- Mucopolysaccharidosis IVA, Morquio A Syndrome, MPS IVA
- Interventions
- BMN 110
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- Not listed
- U.S. locations
- 24
- States / cities
- Birmingham, Alabama • Little Rock, Arkansas • Oakland, California + 21 more
- Conditions
- Achondroplasia
- Interventions
- BMN 333, Vosoritide Injection [Voxzogo]
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 2 Years to 17 Years
- Enrollment
- 160 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2029
- U.S. locations
- 9
- States / cities
- Oakland, California • Wilmington, Delaware • Chicago, Illinois + 6 more
- Conditions
- Hypertension
- Interventions
- 6R-BH4 (sapropterin dihydrochloride)
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 84 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2008
- U.S. locations
- 24
- States / cities
- Hoover, Alabama • Huntsville, Alabama • Mobile, Alabama + 21 more
- Conditions
- Phenylketonuria (PKU)
- Interventions
- Pegvaliase, Diet Only
- Drug · Other
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 12 Years to 17 Years
- Enrollment
- 55 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2027
- U.S. locations
- 13
- States / cities
- Phoenix, Arizona • Little Rock, Arkansas • Aurora, Colorado + 10 more
- Conditions
- Pompe Disease
- Interventions
- Thoracic MRI, Spirometry, Inspiratory Load Compensation, Maximal Inspiratory Pressure, Resting Breathing Pattern, Respiratory Muscle Endurance Test
- Other
- Lead sponsor
- University of Florida
- Other
- Eligibility
- 20 Years to 65 Years
- Enrollment
- 14 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2015 – 2016
- U.S. locations
- 1
- States / cities
- Gainesville, Florida
- Conditions
- Mucopolysaccharidosis VI, Maroteaux-Lamy Syndrome
- Interventions
- Naglazyme
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- Up to 1 Year
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2009
- U.S. locations
- 2
- States / cities
- Los Angeles, California • Oakland, California
- Conditions
- Phenylketonuria
- Interventions
- Sapropterin
- Drug
- Lead sponsor
- University of Missouri-Columbia
- Other
- Eligibility
- 6 Years and older
- Enrollment
- 20 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2009 – 2011
- U.S. locations
- 1
- States / cities
- Columbia, Missouri
- Conditions
- Phenylketonuria
- Interventions
- saproterin dihydrochloride, Diet treatment
- Drug · Other
- Lead sponsor
- University Hospitals Cleveland Medical Center
- Other
- Eligibility
- 2 Years to 60 Years
- Enrollment
- 7 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2019
- U.S. locations
- 1
- States / cities
- Cleveland, Ohio
- Conditions
- Hypochondroplasia
- Interventions
- Vosoritide, Placebo
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 3 Years to 17 Years
- Enrollment
- 80 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2026
- U.S. locations
- 4
- States / cities
- Wilmington, Delaware • Washington D.C., District of Columbia • Chicago, Illinois + 1 more
- Conditions
- Phenylketonurias
- Interventions
- Pegvaliase-Pqpz
- Drug
- Lead sponsor
- University of Missouri-Columbia
- Other
- Eligibility
- 18 Years to 55 Years
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2025
- U.S. locations
- 2
- States / cities
- Boston, Massachusetts • Columbia, Missouri
- Conditions
- Morquio Syndrome A, Maroteaux Lamy Syndrome, MPS IVA, MPS VI
- Interventions
- Not listed
- Lead sponsor
- Greenwood Genetic Center
- Other
- Eligibility
- Up to 18 Years
- Enrollment
- 17 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2015
- U.S. locations
- 1
- States / cities
- Greenville, South Carolina
- Conditions
- Phenylketonuria (PKU)
- Interventions
- BMN165 20mg/day, BMN165 40mg/day, Placebo
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 18 Years to 70 Years
- Enrollment
- 215 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2019
- U.S. locations
- 29
- States / cities
- La Jolla, California • Oakland, California • Aurora, Colorado + 26 more
- Conditions
- Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2
- Interventions
- Cerliponase Alfa, Administration Kit
- Drug · Device
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- Not listed
- Enrollment
- 35 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2030
- U.S. locations
- 16
- States / cities
- Little Rock, Arkansas • Orange, California • Aurora, Colorado + 12 more
- Conditions
- Phenylketonuria
- Interventions
- Sapropterin (Kuvan)
- Drug
- Lead sponsor
- Washington University School of Medicine
- Other
- Eligibility
- 6 Years to 50 Years
- Enrollment
- 45 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2008 – 2018
- U.S. locations
- 1
- States / cities
- St Louis, Missouri
- Conditions
- Claudication, Intermittent, Peripheral Artery Disease, Peripheral Vascular Diseases
- Interventions
- Tetrahydrobiopterin 10 mg/kg, Tetrahydrobiopterin 20 mg/kg, L-Ascorbate, L-Arginine
- Drug
- Lead sponsor
- Louis Messina
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2027
- U.S. locations
- 1
- States / cities
- Worcester, Massachusetts