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ClinicalTrials.gov public records Last synced Oct 26, 2026, 5:57 AM EDT

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Showing 49–67 of 67 matching trials from the live ClinicalTrials.gov search.
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Available No phase listed Expanded access

Post-trial Access Program of Idursulfase-IT Along With Elaprase in Children With Hunter Syndrome

NCT05795361
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 13, 2026 · Synced Oct 26, 2026, 5:57 AM EDT
Completed No phase listed Observational

Lysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool Children

NCT01938014
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Oct 4, 2019 · Synced Oct 26, 2026, 5:57 AM EDT
Completed Phase 2Phase 3 Interventional Results available

Study of Intrathecal Idursulfase-IT Administered in Conjunction With Elaprase® in Pediatric Patients With Hunter Syndrome and Early Cognitive Impairment

NCT02055118
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 11, 2021 · Synced Oct 26, 2026, 5:57 AM EDT
Completed No phase listed Observational

A Study of Potential Treatment-Responsive Biomarkers and Clinical Outcomes in Hunter Syndrome

NCT04007536
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jun 10, 2024 · Synced Oct 26, 2026, 5:57 AM EDT
Completed Phase 2Phase 3 Interventional Results available

Study of Long Term Safety and Clinical Outcomes of Idursulfase IT and Elaprase Treatment in Pediatric Participants Who Have Completed Study HGT-HIT-094

NCT02412787
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jun 19, 2025 · Synced Oct 26, 2026, 5:57 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Incorporation of Safe Sleep Education Into m-Health Technology

NCT06074211
View directory record Official record
Conditions
Sudden Infant Death
Interventions
Safety in Seconds mobile app: Safe Sleep Education, Safety in Seconds mobile app: Car Seat Safety Education
Behavioral
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years and older
Enrollment
130 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2026
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 17, 2026 · Synced Oct 26, 2026, 5:57 AM EDT
Completed Phase 4 Interventional Results available

Long-term Evaluation on Height and Weight in Patients With MPS II Who Started Treatment at < 6 Years of Age

NCT02455622
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Apr 2, 2026 · Synced Oct 26, 2026, 5:57 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Improving the Self-Efficacy of African American Parents in Infant Supine Sleep

NCT01707173
View directory record Official record
Conditions
African American Infant Sleep Beliefs and Practices
Interventions
Tailored education, Standard Education
Behavioral
Lead sponsor
University of Michigan
Other
Eligibility
18 Years and older
Enrollment
735 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2016
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Jun 3, 2016 · Synced Oct 26, 2026, 5:57 AM EDT
Active, not recruiting Phase 2Phase 3 Interventional

CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome)

NCT03566043
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jan 28, 2025 · Synced Oct 26, 2026, 5:57 AM EDT
Enrolling by invitation Phase 3 Interventional

An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in MPS II (Hunter Syndrome) Subjects

NCT05594992
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jan 21, 2026 · Synced Oct 26, 2026, 5:57 AM EDT
Recruiting No phase listed Observational

Registry of Patients Diagnosed With Lysosomal Storage Diseases

NCT05619900
View directory record Official record
Conditions
Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis IV A, Mucopolysaccharidosis VI, Mucopolysaccharidosis VII, Pompe Disease Infantile-Onset, Neuronopathic Gaucher Disease, Wolman Disease
Interventions
There is no intervention
Other
Lead sponsor
University of California, San Francisco
Other
Eligibility
Up to 64 Years
Enrollment
250 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2050
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Apr 8, 2026 · Synced Oct 26, 2026, 5:57 AM EDT
Active, not recruiting Phase 2 Interventional

MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis

NCT02171104
View directory record Official record
Conditions
Mucopolysaccharidosis Disorders, Hurler Syndrome, Hunter Syndrome, Maroteaux Lamy Syndrome, Sly Syndrome, Alpha-Mannosidosis, Fucosidosis, Aspartylglucosaminuria, Glycoprotein Metabolic Disorders, Sphingolipidoses, Recessive Leukodystrophies, Globoid Cell Leukodystrophy, Metachromatic Leukodystrophy, Niemann-Pick B, Niemann-Pick C Subtype 2, Sphingomyelin Deficiency, Peroxisomal Disorders, Adrenoleukodystrophy With Cerebral Involvement, Zellweger Syndrome, Neonatal Adrenoleukodystrophy, Infantile Refsum Disease, Acyl-CoA Oxidase Deficiency, D-Bifunctional Enzyme Deficiency, Multifunctional Enzyme Deficiency, Alpha-methylacyl-CoA Racmase Deficiency, Mitochondrial Neurogastrointestingal Encephalopathy, Severe Osteopetrosis, Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation), Inherited Metabolic Disorders
Interventions
Stem Cell Transplantation, IMD Preparative Regimen, Osteopetrosis Only Preparative Regimen, Osteopetrosis Haploidentical Only Preparative Regimen, cALD SR-A (Standard-Risk, Regimen A), cALD SR-B (Standard-Risk, Regimen B), cALD HR-D (High-Risk, Regimen C), cALD HR-D (High-Risk, Regimen D)
Biological · Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 55 Years
Enrollment
149 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2029
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jan 7, 2026 · Synced Oct 26, 2026, 5:57 AM EDT
Completed No phase listed Observational Results available

An Observational Study Evaluating Anti-Idursulfase Serum Antibody Response in Hunter Syndrome Patients

NCT00882921
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jun 8, 2021 · Synced Oct 26, 2026, 5:57 AM EDT
Not listed Not applicable Interventional Accepts healthy volunteers

Generations in Families Talking Safe Sleep

NCT03186469
View directory record Official record
Conditions
Sudden Infant Death
Interventions
Safety Baby shower, Standard of Care
Behavioral
Lead sponsor
Arkansas Children's Hospital Research Institute
Other
Eligibility
13 Years to 19 Years · Female only
Enrollment
146 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2020
U.S. locations
1
States / cities
Little Rock, Arkansas
Source: ClinicalTrials.gov public record
Updated Jul 18, 2019 · Synced Oct 26, 2026, 5:57 AM EDT
Terminated Phase 2Phase 3 Interventional Results available

Clinical Trial of Growth Hormone in MPS I, II, and VI

NCT00748969
View directory record Official record
Conditions
Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis VI
Interventions
Somatropin (DNA origin)
Drug
Lead sponsor
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Other
Eligibility
5 Years to 17 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2013
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Sep 5, 2018 · Synced Oct 26, 2026, 5:57 AM EDT
Active, not recruiting No phase listed Observational

Long-term Follow-Up for RGX-121

NCT04597385
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Aug 19, 2026 · Synced Oct 26, 2026, 5:57 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Re-enforcing Safe Sleep Practices for Caregivers With Tangible Incentives

NCT02010450
View directory record Official record
Conditions
Safe Sleep Education
Interventions
Wearable Blanket (Sleep Sack), Incentive Item
Other
Lead sponsor
University of Kansas
Other
Eligibility
18 Years and older
Enrollment
152 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2014
U.S. locations
1
States / cities
Wichita, Kansas
Source: ClinicalTrials.gov public record
Updated Apr 16, 2015 · Synced Oct 26, 2026, 5:57 AM EDT
Recruiting Not applicable Interventional Accepts healthy volunteers

Improving Pediatrician Counseling About Infant Safe Sleep Using the Electronic Medical Record

NCT05503771
View directory record Official record
Conditions
Sudden Infant Death, Sudden Unexplained Infant Death
Interventions
ISA-MI, Standard of Care (SOC)
Behavioral · Other
Lead sponsor
Johns Hopkins University
Other
Eligibility
3 Days to 75 Years
Enrollment
350 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2027
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 21, 2026 · Synced Oct 26, 2026, 5:57 AM EDT
Enrolling by invitation Phase 2Phase 3 Interventional

An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007

NCT06075537
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jun 11, 2026 · Synced Oct 26, 2026, 5:57 AM EDT