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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: MPS II Clear all
Active, not recruiting Phase 3 Interventional

RGX-121-3102 Gene Therapy in Participants With MPS II (Hunter Syndrome)

NCT07236606
View directory record Official record
Conditions
MPS II, Hunter Syndrome (MPS II)
Interventions
RGX-121-3102
Genetic
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
4 Months to 5 Years · Male only
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2031
U.S. locations
1
States / cities
New Brunswick, New Jersey
Source: ClinicalTrials.gov public record
Updated Jun 3, 2026 · Synced Jul 22, 2026, 8:49 AM EDT
Terminated Phase 3 Interventional

ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases

NCT00654433
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jul 7, 2014 · Synced Jul 22, 2026, 8:49 AM EDT
Active, not recruiting No phase listed Observational

Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX

NCT04628871
View directory record Official record
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 – 2030
U.S. locations
5
States / cities
Oakland, California • Chicago, Illinois • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated May 2, 2024 · Synced Jul 22, 2026, 8:49 AM EDT
Recruiting Phase 1 Interventional

UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells

NCT02254863
View directory record Official record
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 – 2026
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Sep 7, 2025 · Synced Jul 22, 2026, 8:49 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 3, 2019 · Synced Jul 22, 2026, 8:49 AM EDT
Withdrawn Phase 1 Interventional

Safety and Efficacy of HMI-203 in ERT-Treated Adults With MPS II

NCT05238324
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Aug 27, 2023 · Synced Jul 22, 2026, 8:49 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in Mucopolysaccharidosis III, Type B (MPS IIIB)

NCT02324049
View directory record Official record
Conditions
Mucopolysaccharidosis IIIB
Interventions
SBC-103
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
2 Years to 12 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
2
States / cities
Minneapolis, Minnesota • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 20, 2018 · Synced Jul 22, 2026, 8:49 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 20, 2026 · Synced Jul 22, 2026, 8:49 AM EDT
Completed Phase 1Phase 2 Interventional Results available

A Safety and Dose Ranging Study of Idursulfase (Intrathecal) Administration Via an Intrathecal Drug Delivery Device in Pediatric Patients With Hunter Syndrome Who Have Central Nervous System Involvement and Are Receiving Treatment With Elaprase®

NCT00920647
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jun 27, 2021 · Synced Jul 22, 2026, 8:49 AM EDT
Completed Phase 1 Interventional

Safety and Dose Ranging Study of Insulin Receptor MoAb-IDS Fusion Protein in Patients With Hunter Syndrome

NCT02262338
View directory record Official record
Conditions
Mucopolysaccharidosis II
Interventions
AGT-182
Drug
Lead sponsor
ArmaGen, Inc
Industry
Eligibility
18 Years and older · Male only
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
3
States / cities
Oakland, California • Orange, California • Decatur, Georgia
Source: ClinicalTrials.gov public record
Updated Sep 17, 2018 · Synced Jul 22, 2026, 8:49 AM EDT
Completed Phase 1 Interventional Results available

Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders

NCT00744692
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Aug 12, 2014 · Synced Jul 22, 2026, 8:49 AM EDT
Completed Phase 2Phase 3 Interventional Results available

Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Idursulfase

NCT00630747
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jun 9, 2021 · Synced Jul 22, 2026, 8:49 AM EDT
Completed No phase listed Observational

Observational Study to Evaluate Neurodevelopmental Status in Pediatric Patients With Hunter Syndrome (MPS II)

NCT01822184
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 16, 2021 · Synced Jul 22, 2026, 8:49 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 9, 2024 · Synced Jul 22, 2026, 8:49 AM EDT
Terminated Phase 1Phase 2 Interventional

Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders

NCT01372228
View directory record Official record
Conditions
Hurler Syndrome (MPS I), Hurler-Scheie Syndrome, Hunter Syndrome (MPS II), Sanfilippo Syndrome (MPS III), Krabbe Disease (Globoid Leukodystrophy), Metachromatic Leukodystrophy (MLD), Adrenoleukodystrophy (ALD and AMN), Sandhoff Disease, Tay Sachs Disease, Pelizaeus Merzbacher (PMD), Niemann-Pick Disease, Alpha-mannosidosis
Interventions
hematopoietic stem cell infusion
Biological
Lead sponsor
Talaris Therapeutics Inc.
Industry
Eligibility
Not listed
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Apr 11, 2023 · Synced Jul 22, 2026, 8:49 AM EDT
Not yet recruiting Phase 1Phase 2 Interventional

A Phase I/II Trial of JR-446 in Mucopolysaccharidosis Type IIIB (MPS IIIB)

NCT07640984
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jul 6, 2026 · Synced Jul 22, 2026, 8:49 AM EDT
Suspended Phase 1Phase 2 Interventional

RGX-111 Gene Therapy in Patients With MPS I

NCT03580083
View directory record Official record
Conditions
Mucopolysaccharidosis Type I (MPS I), Hurler Syndrome, Hurler-Scheie Syndrome
Interventions
RGX-111
Genetic
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
4 Months and older
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2029
U.S. locations
1
States / cities
Orange, California
Source: ClinicalTrials.gov public record
Updated Mar 11, 2026 · Synced Jul 22, 2026, 8:49 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease

NCT04088734
View directory record Official record
Conditions
MPS IIIA, Sanfilippo Syndrome, Sanfilippo A, Mucopolysaccharidosis III
Interventions
ABO-102
Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
2 Years to 18 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 24, 2023 · Synced Jul 22, 2026, 8:49 AM EDT
Completed No phase listed Observational

Longitudinal Studies of Brain Structure and Function in MPS Disorders

NCT01870375
View directory record Official record
Conditions
Mucopolysaccharidosis Type I, Mucopolysaccharidosis Type II, Mucopolysaccharidosis Type VI, Mucopolysaccharidosis Type IV, Mucopolysaccharidosis Type VII
Interventions
Not listed
Lead sponsor
University of Minnesota
Other
Eligibility
6 Years and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2019
U.S. locations
3
States / cities
Oakland, California • Minneapolis, Minnesota • New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 5, 2019 · Synced Jul 22, 2026, 8:49 AM EDT
Not listed 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
28 Months and older · Male only
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 9, 2021 · Synced Jul 22, 2026, 8:49 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 4, 2018 · Synced Jul 22, 2026, 8:49 AM EDT
Recruiting No phase listed Observational

Longitudinal Study of Neurodegenerative Disorders

NCT03333200
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 Jul 22, 2026, 8:49 AM EDT
Completed No phase listed Observational

Hunter Outcome Survey (HOS)

NCT03292887
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 Jul 22, 2026, 8:49 AM EDT
Completed Phase 2 Interventional Results available

Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders

NCT01043640
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Feb 4, 2018 · Synced Jul 22, 2026, 8:49 AM EDT