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Showing 1–24 of 63 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Mucopolysaccharidosis Type 2 Clear all
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 21, 2026, 1:26 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II

NCT03041324
View directory record Official record
Conditions
Mucopolysaccharidosis II, MPS II
Interventions
SB-913
Biological
Lead sponsor
Sangamo Therapeutics
Industry
Eligibility
5 Years to 65 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
5
States / cities
Oakland, California • Chicago, Illinois • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Oct 24, 2022 · Synced Jul 21, 2026, 1:26 PM 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 21, 2026, 1:26 PM EDT
Terminated No phase listed Observational

Mucopolysaccharidosis (MPS) I, II, and VI Screening in a High-Risk Population With Previous Surgical Repair or Presence of Inguinal and/or Umbilical Hernia in Combination With Pediatric ENT Surgery (The HATT Project)

NCT02095015
View directory record Official record
Conditions
Mucopolysaccharidosis (MPS)
Interventions
Not listed
Lead sponsor
Shire
Industry
Eligibility
Up to 7 Years · Male only
Enrollment
159 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
7
States / cities
Los Angeles, California • Jacksonville, Florida • Minneapolis, Minnesota + 4 more
Source: ClinicalTrials.gov public record
Updated Mar 16, 2021 · Synced Jul 21, 2026, 1:26 PM 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 27, 2025 · Synced Jul 21, 2026, 1:26 PM EDT
Terminated Phase 2 Interventional Results available

Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation

NCT01697319
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 Jul 21, 2026, 1:26 PM 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 21, 2026, 1:26 PM 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 10, 2021 · Synced Jul 21, 2026, 1:26 PM EDT
Completed Phase 2 Interventional Results available

Stem Cell Transplantation for Hurler

NCT00176917
View directory record Official record
Conditions
Mucopolysaccharidosis I, Mucopolysaccharidosis VI, Mannosidosis, Mucolipidosis Type II (I-cell Disease)
Interventions
Stem Cell Transplant, Busulfan, Cyclophosphamide, ATG
Procedure · Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Not listed
Enrollment
41 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2010
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 27, 2017 · Synced Jul 21, 2026, 1:26 PM EDT
Completed Phase 1 Interventional

Unrelated Umbilical Cord Blood Transplantation Augmented With ALDHbr Umbilical Cord Blood Cells

NCT00692926
View directory record Official record
Conditions
MDS, Anemia, Aplastic, Inborn Errors of Metabolism, Congenital Marrow Failure, Congenital Immunodeficiency Syndrome
Interventions
ALDHbr Umbilical Cord Blood Cells
Biological
Lead sponsor
Joanne Kurtzberg, MD
Other
Eligibility
Up to 55 Years
Enrollment
37 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2012
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jun 28, 2017 · Synced Jul 21, 2026, 1:26 PM EDT
Completed Phase 2 Interventional Results available

Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of rhHNS Administration Via an IDDD in Pediatric Patients With Early Stage MPS IIIA Disease

NCT02060526
View directory record Official record
Conditions
Sanfilippo Syndrome
Interventions
Recombinant human heparan N-sulfatase [rhHNS]
Drug
Lead sponsor
Shire
Industry
Eligibility
12 Months to 48 Months
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
3
States / cities
Torrance, California • Minneapolis, Minnesota • Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Jun 7, 2021 · Synced Jul 21, 2026, 1:26 PM EDT
Active, not recruiting Phase 3 Interventional

A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT)

NCT04573023
View directory record Official record
Conditions
Mucopolysaccharidosis II
Interventions
JR-141, Idursulfase, JR-141 or Idursulfase
Drug
Lead sponsor
JCR Pharmaceuticals Co., Ltd.
Industry
Eligibility
Not listed
Enrollment
86 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
7
States / cities
Phoenix, Arizona • Oakland, California • Chicago, Illinois + 4 more
Source: ClinicalTrials.gov public record
Updated Jun 3, 2026 · Synced Jul 21, 2026, 1:26 PM 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 6, 2026 · Synced Jul 21, 2026, 1:26 PM 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 21, 2026, 1:26 PM 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 21, 2026, 1:26 PM EDT
Completed Phase 1Phase 2 Interventional Results available

An Extension Study of HGT-HIT-045 Evaluating Long-Term Safety and Clinical Outcomes of Idursulfase-IT in Conjunction With Elaprase in Pediatric Participants With Hunter Syndrome and Cognitive Impairment

NCT01506141
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Aug 5, 2025 · Synced Jul 21, 2026, 1:26 PM EDT
Completed Phase 1Phase 2 Interventional

A Treatment Study of Mucopolysaccharidosis Type IIIB

NCT02754076
View directory record Official record
Conditions
MPS III B, Mucopolysaccharidosis Type IIIB
Interventions
AX 250
Drug
Lead sponsor
Allievex Corporation
Industry
Eligibility
1 Year to 10 Years
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2020
U.S. locations
1
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Aug 5, 2020 · Synced Jul 21, 2026, 1:26 PM EDT
Completed No phase listed Observational Results available

Collection and Study of Cerebrospinal Fluid in Patients With Hunter Syndrome

NCT01449240
View directory record Official record
Conditions
Hunter Syndrome
Interventions
No treatment
Other
Lead sponsor
Shire
Industry
Eligibility
Up to 70 Years · Male only
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
5
States / cities
Decatur, Georgia • Chicago, Illinois • Minneapolis, Minnesota + 2 more
Source: ClinicalTrials.gov public record
Updated Jun 8, 2021 · Synced Jul 21, 2026, 1:26 PM 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 21, 2026, 1:26 PM EDT
Terminated No phase listed Observational

Study to Detect Unrecognized Mucopolysaccharidosis in Children Visiting Rheumatology, Hand or Skeletal Dysplasia Clinics

NCT01675674
View directory record Official record
Conditions
Mucopolysaccharidoses, Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis IV, Mucopolysaccharidosis VI
Interventions
Dried blood spot test for MPS
Other
Lead sponsor
National MPS Society
Other
Eligibility
6 Months to 18 Years
Enrollment
3,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
2
States / cities
New Brunswick, New Jersey • New York, New York
Source: ClinicalTrials.gov public record
Updated May 23, 2013 · Synced Jul 21, 2026, 1:26 PM 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 7, 2026 · Synced Jul 21, 2026, 1:26 PM EDT
Terminated Phase 2 Interventional Results available

Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome

NCT01609062
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jan 31, 2016 · Synced Jul 21, 2026, 1:26 PM EDT
Withdrawn Phase 4 Interventional

A Study of ELAPRASE in Treatment-naïve Participants With Hunter Syndrome (Mucopolysaccharidosis [MPS] II)

NCT05494593
View directory record Official record
Conditions
Mucopolysaccharidosis (MPS), Hunter Syndrome
Interventions
ELAPRASE, Rituximab, Methotrexate, Intravenous Immunoglobulin (IVIG)
Drug
Lead sponsor
Takeda
Industry
Eligibility
Up to 6 Years · Male only
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
8
States / cities
Phoenix, Arizona • Oakland, California • San Diego, California + 5 more
Source: ClinicalTrials.gov public record
Updated Dec 8, 2025 · Synced Jul 21, 2026, 1:26 PM EDT
Terminated No phase listed Observational

MPS II Immunophenotyping

NCT04976231
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Sep 18, 2024 · Synced Jul 21, 2026, 1:26 PM EDT