- Conditions
- Mucopolysaccharidosis Type II (MPS II)
- Interventions
- RGX-121
- Genetic
- Lead sponsor
- REGENXBIO Inc.
- Industry
- Eligibility
- 4 Months to 5 Years · Male only
- Enrollment
- 48 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2025
- U.S. locations
- 4
- States / cities
- Oakland, California • New Brunswick, New Jersey • Philadelphia, Pennsylvania + 1 more
Search Results
Search by objective public record fields.
Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results come from ClinicalTrials.gov. When the live registry is unavailable, a clearly labeled stored copy may be shown.
These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Showing 25–48
of 55
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Mucopolysaccharidosis II
- Interventions
- No Intervention
- Other
- Lead sponsor
- Denali Therapeutics Inc.
- Industry
- Eligibility
- Up to 30 Years
- Enrollment
- 18 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2024
- U.S. locations
- 3
- States / cities
- Oakland, California • Chapel Hill, North Carolina • Pittsburgh, Pennsylvania
- Conditions
- Mucopolysaccharidosis (MPS)
- Interventions
- Not listed
- Lead sponsor
- Shire
- Industry
- Eligibility
- Up to 7 Years · Male only
- Enrollment
- 159 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2015
- U.S. locations
- 7
- States / cities
- Los Angeles, California • Jacksonville, Florida • Minneapolis, Minnesota + 4 more
- Conditions
- Mucopolysaccharidosis (MPS), Hunter Syndrome
- Interventions
- Not listed
- Lead sponsor
- Shire
- Industry
- Eligibility
- 2 Years to 18 Years · Male only
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2016
- U.S. locations
- 3
- States / cities
- Oakland, California • Chicago, Illinois • Chapel Hill, North Carolina
- 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
- Hunter Syndrome, Mucopolysaccharidosis II (MPS II)
- Interventions
- Idursulfase
- Biological
- Lead sponsor
- Shire
- Industry
- Eligibility
- 5 Years and older · Male only
- Enrollment
- 94 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2004 – 2008
- U.S. locations
- 16
- States / cities
- Phoenix, Arizona • Oakland, California • Denver, Colorado + 13 more
- Conditions
- Hunter Syndrome
- Interventions
- Idursulfase-IT, Elaprase
- Drug
- Lead sponsor
- Shire
- Industry
- Eligibility
- Up to 18 Years · Male only
- Enrollment
- 56 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2024
- U.S. locations
- 3
- States / cities
- Oakland, California • Chicago, Illinois • Chapel Hill, North Carolina
- Conditions
- Hunter Syndrome
- Interventions
- Control, Idursulfase IT (1 mg), Idursulfase IT (10 mg), Idursulfase IT (30 mg)
- Other · Drug
- Lead sponsor
- Shire
- Industry
- Eligibility
- 3 Years to 18 Years · Male only
- Enrollment
- 16 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2012
- U.S. locations
- 1
- States / cities
- Chapel Hill, North Carolina
- Conditions
- Mucopolysaccharidosis, Hurler Syndrome, Hunter Syndrome, Maroteaux-Lamy Syndrome, Sly Syndrome, Alpha Mannosidosis, Fucosidosis, Aspartylglucosaminuria, Adrenoleukodystrophy (ALD), Krabbe Disease, Metachromatic Leukodystrophy (MLD), Sphingolipidoses, Peroxisomal Disorders
- Interventions
- Campath-1H, Cyclophosphamide, Busulfan, Allogeneic stem cell transplantation, Cyclosporine A, Mycophenolate Mofetil
- Drug · Procedure
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Other
- Eligibility
- Up to 21 Years
- Enrollment
- 46 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2017
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Mucopolysaccharidosis II
- Interventions
- JR-141, Idursulfase, JR-141 or Idursulfase
- Drug
- Lead sponsor
- JCR Pharmaceuticals Co., Ltd.
- Industry
- Eligibility
- Not listed
- Enrollment
- 86 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2027
- U.S. locations
- 7
- States / cities
- Phoenix, Arizona • Oakland, California • Chicago, Illinois + 4 more
- Conditions
- Mucopolysaccharidosis II
- Interventions
- iduronate-2-sulfatase enzyme replacement therapy, Placebo
- Biological
- Lead sponsor
- Shire
- Industry
- Eligibility
- 5 Years to 25 Years · Male only
- Enrollment
- 96 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2003 – 2005
- U.S. locations
- 4
- States / cities
- Oakland, California • St Louis, Missouri • Chapel Hill, North Carolina + 1 more
- Conditions
- Hunter Syndrome
- Interventions
- Elaprase for intravenous (IV) infusion
- Drug
- Lead sponsor
- Shire
- Industry
- Eligibility
- Up to 5 Years · Male only
- Enrollment
- 21 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2025
- U.S. locations
- 1
- States / cities
- Chicago, Illinois
- Conditions
- Mucopolysaccharidosis II
- Interventions
- Genetic HMI-203
- Biological
- Lead sponsor
- Homology Medicines, Inc
- Industry
- Eligibility
- 18 Years to 45 Years · Male only
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2029
- U.S. locations
- 5
- States / cities
- Oakland, California • New Haven, Connecticut • Hackensack, New Jersey + 2 more
- Conditions
- Hunter Syndrome
- Interventions
- Idursulfase
- Biological
- Lead sponsor
- Shire
- Industry
- Eligibility
- 5 Years and older · Male only
- Enrollment
- 26 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2013
- U.S. locations
- 2
- States / cities
- Oakland, California • Minneapolis, Minnesota
- Conditions
- Mucopolysaccharidoses
- Interventions
- Not listed
- Lead sponsor
- University of Minnesota
- Other
- Eligibility
- 3 Years to 18 Years
- Enrollment
- 1,147 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2017
- U.S. locations
- 2
- States / cities
- Orange, California • Minneapolis, Minnesota
- Conditions
- Adrenoleukodystrophy, Batten Disease, Mucopolysaccharidosis II, Leukodystrophy, Globoid Cell, Leukodystrophy, Metachromatic, Neimann Pick Disease, Pelizaeus-Merzbacher Disease, Sandhoff Disease, Tay-Sachs Disease, Brain Diseases, Metabolic, Inborn, Alpha-Mannosidosis, Sanfilippo Mucopolysaccharidoses
- Interventions
- DUOC-01
- Biological
- Lead sponsor
- Joanne Kurtzberg, MD
- Other
- Eligibility
- 1 Week to 22 Years
- Enrollment
- 40 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2027
- U.S. locations
- 1
- States / cities
- Durham, North Carolina
- Conditions
- Mucopolysaccharidoses, Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis IV, Mucopolysaccharidosis VI
- Interventions
- Dried blood spot test for MPS
- Other
- Lead sponsor
- National MPS Society
- Other
- Eligibility
- 6 Months to 18 Years
- Enrollment
- 3,000 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2014
- U.S. locations
- 2
- States / cities
- New Brunswick, New Jersey • New York, New York
- Conditions
- Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis VI
- Interventions
- Adalimumab Injection [Humira], Saline Solution for Injection
- Drug
- Lead sponsor
- Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
- Other
- Eligibility
- 5 Years and older
- Enrollment
- 14 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2027
- U.S. locations
- 1
- States / cities
- Torrance, California
- Conditions
- Non Malignant Disorders, Immunodeficiencies, Congenital Marrow Failures, Hemoglobinopathies, Inborn Errors of Metabolism, Sickle Cell, Thalassemia, Lysosomal Storage Disease
- Interventions
- Unrelated Umbilical Cord Blood Transplant, Reduced Intensity Conditioning
- Biological · Drug
- Lead sponsor
- Duke University
- Other
- Eligibility
- Up to 21 Years
- Enrollment
- 22 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2014
- U.S. locations
- 1
- States / cities
- Durham, North Carolina
- Conditions
- Inherited Metabolic Diseases, Lysosomal Storage Disorders, Peroxisomal Storage Diseases, Inborn Errors of Metabolism, Mucopolysaccharidosis
- Interventions
- ALD-101
- Biological
- Lead sponsor
- Aldagen
- Industry
- Eligibility
- Up to 16 Years
- Enrollment
- 40 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2011
- U.S. locations
- 3
- States / cities
- Los Angeles, California • New York, New York • Durham, North Carolina
- Conditions
- Mucopolysaccharidosis II
- Interventions
- JR-141
- Drug
- Lead sponsor
- JCR Pharmaceuticals Co., Ltd.
- Industry
- Eligibility
- Not listed
- Enrollment
- 80 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2028
- U.S. locations
- 2
- States / cities
- Oakland, California • Philadelphia, Pennsylvania
- Conditions
- Hunter Syndrome
- Interventions
- No treatment
- Other
- Lead sponsor
- Shire
- Industry
- Eligibility
- Up to 70 Years · Male only
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2013
- U.S. locations
- 5
- States / cities
- Decatur, Georgia • Chicago, Illinois • Minneapolis, Minnesota + 2 more
- Conditions
- Mucopolysaccharidosis II
- Interventions
- Long-term Follow-Up
- Other
- Lead sponsor
- REGENXBIO Inc.
- Industry
- Eligibility
- Male only
- Enrollment
- 27 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2028
- U.S. locations
- 3
- States / cities
- San Francisco, California • New Brunswich, New Jersey • Philadelphia, Pennsylvania
- 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