- Conditions
- Mucopolysaccharidosis Type IIIB, MPS IIIB (Sanfilippo B Syndrome)
- Interventions
- Tralesinidase alfa (TA)
- Drug
- Lead sponsor
- Spruce Biosciences
- Industry
- Eligibility
- 12 Months and older
- U.S. locations
- 1
- States / cities
- Oakland, California
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 121
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Hemophilia B, Mucopolysaccharidosis I, Mucopolysaccharidosis II
- Interventions
- SB-318, SB-913, SB-FIX
- Biological
- Lead sponsor
- Sangamo Therapeutics
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 13 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2026
- U.S. locations
- 5
- States / cities
- Oakland, California • Chicago, Illinois • New York, New York + 2 more
- Conditions
- Mucopolysaccharidosis IIIB
- Interventions
- JR-446
- Drug
- Lead sponsor
- JCR Pharmaceuticals Co., Ltd.
- Industry
- Eligibility
- Up to 5 Years
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2028
- U.S. locations
- 3
- States / cities
- Oakland, California • Pittsburgh, Pennsylvania • Fairfax, Virginia
- 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
- 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
- Hunter Syndrome
- Interventions
- Idursulfase-IT, Elaprase
- Drug
- Lead sponsor
- Takeda
- Industry
- Eligibility
- 3 Years to 18 Years · Male only
- Enrollment
- 15 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2024
- U.S. locations
- 7
- States / cities
- Chicago, Illinois • Chapel Hill, North Carolina • Portland, Oregon + 4 more
- 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
- 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
- MPS IV A, Mucopolysaccharidosis IVA, Morquio A Syndrome
- Interventions
- Not listed
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- Not listed
- Enrollment
- 353 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2014
- U.S. locations
- 2
- States / cities
- Oakland, California • Chicago, Illinois
- Conditions
- MPS-IH (Hurler Syndrome)
- Interventions
- Experimental: OTL-203, Active Comparator: Allo-HSCT
- Genetic
- Lead sponsor
- Orchard Therapeutics
- Industry
- Eligibility
- 28 Days to 30 Months
- Enrollment
- 41 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2031
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Mucopolysaccharidosis IVA, Morquio A Syndrome, MPS IVA
- Interventions
- BMN 110
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 7 Years and older
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2014
- U.S. locations
- 4
- States / cities
- Oakland, California • Chicago, Illinois • New York, New York + 1 more
- Conditions
- Morquio Disease, MPS - Mucopolysaccharidosis
- Interventions
- Interview
- Other
- Lead sponsor
- Emory University
- Other
- Eligibility
- 15 Years to 100 Years · Female only
- Enrollment
- 18 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2017 – 2018
- U.S. locations
- 1
- States / cities
- Decatur, Georgia
- Conditions
- Mucopolysaccharidosis Type 7
- Interventions
- UX003
- Drug
- Lead sponsor
- Joyce Fox
- Other
- Eligibility
- Male only
- U.S. locations
- 1
- States / cities
- New Hyde Park, New York
- Conditions
- Mucopolysaccharidosis IV Type A, Morquio A Syndrome, MPS IVA
- Interventions
- Imaging, gait analysis, growth, joint test, hearing test, questionnaire, etc.
- Diagnostic Test
- Lead sponsor
- Nemours Children's Clinic
- Other
- Eligibility
- Not listed
- Enrollment
- 60 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2027
- U.S. locations
- 1
- States / cities
- Wilmington, Delaware
- Conditions
- Mucopolysaccharidosis Type IIIB
- Interventions
- Not listed
- Lead sponsor
- Allievex Corporation
- Industry
- Eligibility
- Up to 18 Years
- Enrollment
- 44 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2025
- U.S. locations
- 1
- States / cities
- Oakland, California
- Conditions
- Sanfilippo Syndrome Type A (MPS IIIA)
- Interventions
- SOBI003
- Drug
- Lead sponsor
- Swedish Orphan Biovitrum
- Industry
- Eligibility
- 18 Months to 78 Months
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2021
- U.S. locations
- 2
- States / cities
- Oakland, California • Chapel Hill, North Carolina
- Conditions
- Mucopolysaccharidosis IIIA, MPS IIIA, Sanfilippo Syndrome, Sanfilippo A
- Interventions
- No Investigational Product, Adjuvant Immunomodulatory (IM) Therapy
- Other · Drug
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- Not listed
- Enrollment
- 41 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2027
- U.S. locations
- 1
- States / cities
- Columbus, Ohio
- Conditions
- Mucopolysaccharidosis Type IIIA
- Interventions
- DNL126
- Drug
- Lead sponsor
- Denali Therapeutics Inc.
- Industry
- Eligibility
- 0 Years to 18 Years
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2028
- U.S. locations
- 4
- States / cities
- Oakland, California • Iowa City, Iowa • Chapel Hill, North Carolina + 1 more
- Conditions
- MLD, Krabbe Disease, ALD, MPS I, MPS II, MPS III, Vanishing White Matter Disease, GM3 Gangliosidosis, PKAN, Tay-Sachs Disease, NP Deficiency, Osteopetrosis, Alpha-Mannosidosis, Sandhoff Disease, Niemann-Pick Diseases, MPS IV, Gaucher Disease, GAN, GM1 Gangliosidoses, Morquio Disease, S-Adenosylhomocysteine Hydrolase Deficiency, Batten Disease, Pelizaeus-Merzbacher Disease, Leukodystrophy, Lysosomal Storage Diseases, Purine Nucleoside Phosphorylase Deficiency, Multiple Sulfatase Deficiency Disease
- Interventions
- Palliative Care, Hematopoetic Stem Cell Transplantation
- Other · Biological
- Lead sponsor
- University of Pittsburgh
- Other
- Eligibility
- Not listed
- Enrollment
- 1,500 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2035
- U.S. locations
- 1
- States / cities
- Pittsburgh, Pennsylvania
- Conditions
- Mucopolysaccharidosis II
- Interventions
- tividenofusp alfa
- Drug
- Lead sponsor
- Denali Therapeutics Inc.
- Industry
- Eligibility
- Up to 18 Years
- Enrollment
- 99 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2027
- U.S. locations
- 8
- States / cities
- Oakland, California • Chicago, Illinois • Hackensack, New Jersey + 5 more
- 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 I, Hurler's Syndrome, Hurler-Scheie Syndrome, Scheie's Syndrome
- Interventions
- laronidase
- Drug
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- Not listed
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2004 – 2007
- U.S. locations
- 3
- States / cities
- Los Angles, California • Hartford, Connecticut • Milwaukee, Wisconsin
- Conditions
- MPS II, Mucopolysaccharidosis II, Hunter Syndrome
- Interventions
- Not listed
- Lead sponsor
- Duke University
- Other
- Eligibility
- Not listed
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2024
- U.S. locations
- 1
- States / cities
- Durham, North Carolina
- Conditions
- Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy, Tay Sachs Disease, Sandhoffs Disease, Wolman Disease, I-Cell Disease, Sanfilippo Syndrome, GM1 Gangliosidosis
- Interventions
- Clofarabine, Total body Irradiation, Melphalan, Hematopoietic Stem Cell Transplantation, Alemtuzumab, mycophenylate mofetil, Cyclosporine A, Hydroxyurea
- Drug · Procedure · Biological + 1 more
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Other
- Eligibility
- Up to 70 Years
- Enrollment
- 38 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2014
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota