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Showing 1–24 of 59 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Mucopolysaccharidosis IV Clear all
Completed Not applicable Interventional Results available

North Carolina Genomic Evaluation by Next-generation Exome Sequencing, 2

NCT03548779
View directory record Official record
Conditions
Epilepsy; Seizure, Neuromuscular Diseases, Brain Malformation, Intellectual Disability, Autism Spectrum Disorder, Hypotonia, Inborn Errors of Metabolism, Movement Disorders, Genetic Disease, Development Delay, Chromosome Abnormality, Hearing Loss, Dysmorphic Features, Skeletal Dysplasia, Congenital Abnormality, Microcephaly, Macrocephaly
Interventions
Pre-visit prep, usual care + exome seq
Behavioral · Diagnostic Test
Lead sponsor
University of North Carolina, Chapel Hill
Other
Eligibility
0 Years and older
Enrollment
548 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2024
U.S. locations
3
States / cities
Asheville, North Carolina • Chapel Hill, North Carolina • Greenville, North Carolina
Source: ClinicalTrials.gov public record
Updated May 12, 2025 · Synced Sep 17, 2026, 7:19 AM EDT
Completed No phase listed Observational

Study of Skeletal Disorders and Short Stature

NCT00001754
View directory record Official record
Conditions
Developmental Bone Disease, Dwarfism, Skeletal Dysplasias
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
Not listed
Enrollment
600 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2001
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced Sep 17, 2026, 7:19 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Pregnancy With Morquio Syndrome - What Are Patients' Perspectives and Has ERT Changed Them?

NCT03150069
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 Sep 17, 2026, 7:19 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Cardiac Structure and Function in MPS

NCT07361536
View directory record Official record
Conditions
MPS I, MPS IV A, MPS - Mucopolysaccharidosis, MPS IVA
Interventions
Not listed
Lead sponsor
Children's Hospital of Orange County
Other
Eligibility
0 Years to 99 Years
Enrollment
240 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2030
U.S. locations
3
States / cities
Orange, California • Minneapolis, Minnesota • St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Jan 25, 2026 · Synced Sep 17, 2026, 7:19 AM EDT
Completed No phase listed Observational

Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)

NCT04035811
View directory record Official record
Conditions
Achondroplasia
Interventions
Not listed
Lead sponsor
QED Therapeutics, a BridgeBio company
Industry
Eligibility
30 Months to 17 Years
Enrollment
271 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
8
States / cities
Oakland, California • Aurora, Colorado • Wilmington, Delaware + 5 more
Source: ClinicalTrials.gov public record
Updated Apr 2, 2026 · Synced Sep 17, 2026, 7:19 AM EDT
Enrolling by invitation No phase listed Observational Accepts healthy volunteers

ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program

NCT05368038
View directory record Official record
Conditions
Acid Sphingomyelinase Deficiency, Ceroid Lipofuscinosis, Neuronal, 2, Cerebrotendinous Xanthomatosis, Fabry Disease, GM1 Gangliosidosis, Gaucher Disease, Lysosomal Acid Lipase Deficiency, Metachromatic Leukodystrophy, Mucopolysaccharidosis II, Mucopolysaccharidosis III-B, Mucopolysaccharidosis IV A, Mucopolysaccharidosis VI, Mucopolysaccharidosis VII, Niemann-Pick Disease, Type C
Interventions
Confirmatory Testing
Diagnostic Test
Lead sponsor
Albert Einstein College of Medicine
Other
Eligibility
Up to 4 Weeks
Enrollment
100,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2029
U.S. locations
9
States / cities
Brooklyn, New York • Manhasset, New York • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Jul 27, 2026 · Synced Sep 17, 2026, 7:19 AM EDT
Completed No phase listed Observational

Psychological Concomitants of Morquio A Syndrome - Longitudinal Effects of Enzyme Replacement Therapy (The MAPLE Study)

NCT02208661
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Apr 10, 2018 · Synced Sep 17, 2026, 7:19 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Safety and Effect of L. Reuteri on Biomarkers of Inflammation in Healthy Infants With Colic

NCT01849991
View directory record Official record
Conditions
Gastrointestinal, Colic
Interventions
Lactobacillus reuteri, Placebo
Biological
Lead sponsor
The University of Texas Health Science Center, Houston
Other
Eligibility
3 Weeks to 3 Months
Enrollment
21 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2016
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jan 29, 2018 · Synced Sep 17, 2026, 7:19 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Safety and Efficacy of Voxzogo for Growth Deficits in MPS IVA and VI

NCT05845749
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 Sep 17, 2026, 7:19 AM EDT
Active, not recruiting Not applicable Interventional Accepts healthy volunteers

Impact of B. Adolescentis iVS-1 Probiotic on Gastrointestinal Outcomes

NCT07408518
View directory record Official record
Conditions
Digestive Disease, Gut Health
Interventions
Probiotic, Placebo
Dietary Supplement · Other
Lead sponsor
Synbiotic Health
Industry
Eligibility
18 Years to 75 Years
Enrollment
92 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2026
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Mar 31, 2026 · Synced Sep 17, 2026, 7:19 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Study of Skeletal Disorders

NCT05031507
View directory record Official record
Conditions
Skeletal Disorders
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
2 Months to 100 Years
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2027
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 19, 2026 · Synced Sep 17, 2026, 7:19 AM EDT
Completed No phase listed Observational

A Non-Interventional Study of Clinical Characteristics and Mortality of US Patients With Fibrodysplasia Ossificans Progressiva (FOP)

NCT06064656
View directory record Official record
Conditions
Fibrodysplasia Ossificans Progressiva (FOP)
Interventions
Non-Interventional
Other
Lead sponsor
Regeneron Pharmaceuticals
Industry
Eligibility
Not listed
Enrollment
131 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023
U.S. locations
1
States / cities
Tarrytown, New York
Source: ClinicalTrials.gov public record
Updated Jul 2, 2025 · Synced Sep 17, 2026, 7:19 AM EDT
Recruiting Phase 1 Interventional

PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

NCT04532047
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 16, 2026 · Synced Sep 17, 2026, 7:19 AM EDT
Completed No phase listed Observational

Screening an Orthopedic Population for Mildly-affected Individuals With Morquio Syndrome A and Maroteaux-Lamy Syndrome

NCT01961518
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Aug 12, 2015 · Synced Sep 17, 2026, 7:19 AM EDT
Terminated No phase listed Observational

Discovering New Biomarkers For Monitoring Disease Progression in Patients With Mucopolysaccharidosis IVA

NCT01733615
View directory record Official record
Conditions
Mucopolysaccharidosis Type IVA
Interventions
Not listed
Lead sponsor
Emory University
Other
Eligibility
Not listed
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
1
States / cities
Decatur, Georgia
Source: ClinicalTrials.gov public record
Updated Sep 3, 2014 · Synced Sep 17, 2026, 7:19 AM EDT
Completed No phase listed Observational

Psychological Concomitants of Morquio Syndrome (The MAP Study)

NCT01752296
View directory record Official record
Conditions
Morquio Disease, Mucopolysaccharidosis IV
Interventions
Not listed
Lead sponsor
Nadia Ali, PhD
Other
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
1
States / cities
Decatur, Georgia
Source: ClinicalTrials.gov public record
Updated Dec 3, 2013 · Synced Sep 17, 2026, 7:19 AM EDT
Recruiting Phase 4 Interventional Accepts healthy volunteers

Effects of Bisphosphonates on OI-Related Hearing Loss

NCT04152551
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 Sep 17, 2026, 7:19 AM EDT
Completed Not applicable Interventional Results available

A Study to Evaluate the Safety, Tolerability and Effectiveness of the Renew Insert in Treating Accidental Bowel Leakage

NCT01475474
View directory record Official record
Conditions
Bowel Incontinence
Interventions
Renew Insert
Device
Lead sponsor
Renew Medical
Industry
Eligibility
18 Years and older
Enrollment
97 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2011
U.S. locations
3
States / cities
Los Gatos, California • San Diego, California • Weston, Florida
Source: ClinicalTrials.gov public record
Updated Nov 27, 2013 · Synced Sep 17, 2026, 7:19 AM EDT
Completed No phase listed Observational

Diagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilateral Hip Disease

NCT01707433
View directory record Official record
Conditions
Mucopolysaccharidosis IV A, Mucopolysaccharidosis VI
Interventions
Enzyme testing
Other
Lead sponsor
Children's Hospitals and Clinics of Minnesota
Other
Eligibility
Up to 21 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2015
U.S. locations
2
States / cities
Minneapolis, Minnesota • Saint Paul, Minnesota
Source: ClinicalTrials.gov public record
Updated Aug 3, 2015 · Synced Sep 17, 2026, 7:19 AM EDT
Completed Phase 3 Interventional Results available

Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

NCT01415427
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jul 20, 2021 · Synced Sep 17, 2026, 7:19 AM EDT
Completed Phase 4 Interventional Accepts healthy volunteers Results available

Pregabalin and Colonic Function

NCT01094808
View directory record Official record
Conditions
Healthy
Interventions
Pregabalin, Placebo, Bowel preparation
Drug · Other
Lead sponsor
Mayo Clinic
Other
Eligibility
18 Years to 75 Years
Enrollment
62 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2010 – 2011
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Mar 6, 2012 · Synced Sep 17, 2026, 7:19 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Clinical Evaluation of Acuity 200 (Fluoroxyfocon A) RGP Contact Lens

NCT03971227
View directory record Official record
Conditions
Refractive Errors
Interventions
Acuity 200 (Fluoroxyfocon A) contact lens, Acuity 100 (Hexafocon A) contact lens
Device
Lead sponsor
Acuity Polymers, Inc.
Industry
Eligibility
18 Years and older
Enrollment
74 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2020
U.S. locations
3
States / cities
Honolulu, Hawaii • Edina, Minnesota • Pittsford, New York
Source: ClinicalTrials.gov public record
Updated May 11, 2020 · Synced Sep 17, 2026, 7:19 AM EDT
Recruiting No phase listed Observational

Uncovering Genes Behind Cartilage Tumors and Vascular Anomalies Using Genomic Sequencing

NCT06749366
View directory record Official record
Conditions
Enchondromatosis
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
2 Years to 100 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 14, 2026 · Synced Sep 17, 2026, 7:19 AM EDT
Completed Phase 2 Interventional Results available

Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)

NCT01515956
View directory record Official record
Conditions
Mucopolysaccharidosis IVA, Morquio A Syndrome, MPS IVA
Interventions
BMN 110
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
Up to 5 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
2
States / cities
Oakland, California • Manhasset, New York
Source: ClinicalTrials.gov public record
Updated Aug 9, 2017 · Synced Sep 17, 2026, 7:19 AM EDT