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ClinicalTrials.gov public records Last synced May 22, 2026, 3:54 AM EDT

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 24 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: MPS - Mucopolysaccharidosis Clear all
Recruiting Not applicable Interventional Accepts healthy volunteers
View directory record Official record
Conditions
IBS (Irritable Bowel Syndrome), Bloating, Abdominal Pain, Gut Health
Interventions
FODMAP-targeting digestive enzyme blend, Placebo
Dietary Supplement · Other
Lead sponsor
Kiwi Health Inc
Industry
Eligibility
18 Years and older
Enrollment
150 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2026
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated May 14, 2026 · Synced May 22, 2026, 3:54 AM EDT
Available No phase listed Expanded access
View directory record Official record
Conditions
Hunter Syndrome
Interventions
Idursulfase-IT
Drug
Lead sponsor
Takeda
Industry
Eligibility
Male only
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
17
States / cities
Phoenix, Arizona • Oakland, California • Wilmington, Delaware + 14 more
Source: ClinicalTrials.gov public record
Updated Apr 12, 2026 · Synced May 22, 2026, 3:54 AM EDT
Recruiting Phase 1 Interventional
View directory record Official record
Conditions
Sanfilippo Syndrome Type A
Interventions
GC1130A
Drug
Lead sponsor
GC Biopharma Corp
Industry
Eligibility
12 Months to 18 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
2
States / cities
Oakland, California • Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Aug 27, 2025 · Synced May 22, 2026, 3:54 AM EDT
Withdrawn Not applicable Interventional
View directory record Official record
Conditions
Hurler Syndrome, Cerebral Palsy, Juvenile Rheumatoid Arthritis, Crohn Disease, HIV Infections
Interventions
Growth Hormone, Whole body Protein turnover, DEXA scan
Drug · Procedure
Lead sponsor
University of Texas Southwestern Medical Center
Other
Eligibility
3 Years to 17 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Dec 13, 2018 · Synced May 22, 2026, 3:54 AM EDT
Completed No phase listed Observational Accepts healthy volunteers
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jan 19, 2021 · Synced May 22, 2026, 3:54 AM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Hunter Syndrome
Interventions
Not listed
Lead sponsor
Shire
Industry
Eligibility
Not listed
Enrollment
1,443 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2023
U.S. locations
1
States / cities
Lexington, Massachusetts
Source: ClinicalTrials.gov public record
Updated Oct 27, 2024 · Synced May 22, 2026, 3:54 AM EDT
Recruiting No phase listed Observational
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Feb 8, 2026 · Synced May 22, 2026, 3:54 AM EDT
Completed Not applicable Interventional Results available
View directory record Official record
Conditions
Lysosomal Storage Disease, Peroxisomal Disorder
Interventions
Campath-1H, Clofarabine, Melphalan, Total Body Irradiation with Marrow Boosting, Hematopoietic stem cell transplantation, Cyclosporine A, Mycophenolate mofetil
Drug · Radiation · Biological
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 55 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 4, 2017 · Synced May 22, 2026, 3:54 AM EDT
Recruiting Not applicable Interventional Accepts healthy volunteers
View directory record Official record
Conditions
Gut Health
Interventions
Butyrate + Polyphenol Formulation, Placebo
Dietary Supplement
Lead sponsor
Supplement Formulators, Inc.
Industry
Eligibility
25 Years to 70 Years
Enrollment
124 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2026
U.S. locations
1
States / cities
Fort Lauderdale, Florida
Source: ClinicalTrials.gov public record
Updated Jan 28, 2026 · Synced May 22, 2026, 3:54 AM EDT
Enrolling by invitation Phase 2 Interventional
View directory record Official record
Conditions
Achondroplasia
Interventions
Infigratinib
Drug
Lead sponsor
QED Therapeutics, a BridgeBio company
Industry
Eligibility
3 Years to 18 Years
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2032
U.S. locations
8
States / cities
Oakland, California • Aurora, Colorado • Wilmington, Delaware + 5 more
Source: ClinicalTrials.gov public record
Updated Oct 30, 2025 · Synced May 22, 2026, 3:54 AM EDT
Completed Phase 1 Interventional
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Nov 2, 2006 · Synced May 22, 2026, 3:54 AM EDT
Terminated Phase 1 Interventional Results available
View directory record Official record
Conditions
Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis, Inherited Bone Marrow Failure Syndrome, Hemoglobinopathies, Metabolic Disorders
Interventions
BPX-501 and Rimiducid
Biological
Lead sponsor
Bellicum Pharmaceuticals
Industry
Eligibility
4 Months to 55 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Mar 24, 2024 · Synced May 22, 2026, 3:54 AM EDT
Terminated Phase 2 Interventional Results available
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jan 11, 2016 · Synced May 22, 2026, 3:54 AM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Mucopolysaccharidosis II
Interventions
Not listed
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
Male only
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 25, 2022 · Synced May 22, 2026, 3:54 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional
View directory record Official record
Conditions
MPS IVA, MPS VI
Interventions
Vosoritide Injection [Voxzogo]
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
5 Years to 10 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2029
U.S. locations
1
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Mar 9, 2026 · Synced May 22, 2026, 3:54 AM EDT
Completed Phase 2Phase 3 Interventional Results available
View directory record Official record
Conditions
Hunter Syndrome
Interventions
idursulfase-IT, No IT treatment
Biological · Other
Lead sponsor
Shire
Industry
Eligibility
Up to 18 Years · Male only
Enrollment
58 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
3
States / cities
Oakland, California • Chicago, Illinois • Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Jun 10, 2021 · Synced May 22, 2026, 3:54 AM EDT
Completed Phase 3 Interventional Results available
View directory record Official record
Conditions
MPS IV A
Interventions
BMN 110 Weekly, Placebo, BMN 110 Every Other Week
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
5 Years and older
Enrollment
177 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2012
U.S. locations
6
States / cities
Oakland, California • Wilmington, Delaware • Washington D.C., District of Columbia + 3 more
Source: ClinicalTrials.gov public record
Updated Jul 6, 2014 · Synced May 22, 2026, 3:54 AM EDT
Recruiting Phase 4 Interventional Accepts healthy volunteers
View directory record Official record
Conditions
Osteogenesis Imperfecta
Interventions
Risedronate Oral Tablet
Drug
Lead sponsor
Hospital for Special Surgery, New York
Other
Eligibility
6 Years to 100 Years
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2027
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Apr 27, 2026 · Synced May 22, 2026, 3:54 AM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Sanfilippo Syndrome Type B
Interventions
Not listed
Lead sponsor
Shire
Industry
Eligibility
1 Year to 10 Years
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
2
States / cities
Oakland, California • Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Mar 16, 2021 · Synced May 22, 2026, 3:54 AM EDT
Recruiting Phase 2Phase 3 Interventional
View directory record Official record
Conditions
Sanfilippo Syndrome, MPS3
Interventions
Ambroxol Hydrochloride 30 mg tablet - 9 mg/kg/day, Ambroxol Hydrochloride 30 mg tablet - 18 mg/kg/day, Ambroxol Hydrochloride 30 mg tablet - 27 mg/kg/day
Drug
Lead sponsor
Ozlem Goker-Alpan
Other
Eligibility
18 Years to 99 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Aug 19, 2025 · Synced May 22, 2026, 3:54 AM EDT
Active, not recruiting Phase 3 Interventional
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Dec 21, 2025 · Synced May 22, 2026, 3:54 AM EDT
Recruiting Phase 2Phase 3 Interventional
View directory record Official record
Conditions
Sanfilippo Syndrome, Mucopolysaccharidosis III
Interventions
Epidiolex, Placebo
Drug
Lead sponsor
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Other
Eligibility
4 Years and older
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Torrance, California
Source: ClinicalTrials.gov public record
Updated Apr 5, 2026 · Synced May 22, 2026, 3:54 AM EDT
Recruiting Phase 2Phase 3 Interventional
View directory record Official record
Conditions
MPS IIIA, Sanfilippo Syndrome, Sanfilippo A, Mucopolysaccharidosis III
Interventions
UX111, Prophylactic Immunomodulatory (IM) Therapy, Optimized Prophylactic IM Therapy, Adjuvant IM Therapy
Biological · Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
Not listed
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2027
U.S. locations
2
States / cities
Columbus, Ohio • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 23, 2026 · Synced May 22, 2026, 3:54 AM EDT
Completed No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Mucopolysaccharidosis Type I
Interventions
Not listed
Lead sponsor
University of Minnesota
Other
Eligibility
6 Years and older
Enrollment
30 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2019
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Oct 31, 2019 · Synced May 22, 2026, 3:54 AM EDT