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Showing 1–15 of 15 matching trials from the live ClinicalTrials.gov search.
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Terminated Phase 1 Interventional

Extension Study of Intrathecal Enzyme Replacement Therapy for MPS I

NCT00786968
View directory record Official record
Conditions
Spinal Cord Compression, Mucopolysaccharidosis I, Hurler-Scheie Syndrome, Scheie Syndrome, Lysosomal Storage Disease
Interventions
laronidase
Drug
Lead sponsor
Patricia I. Dickson, M.D.
Individual
Eligibility
8 Years and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2011
U.S. locations
1
States / cities
Torrance, California
Source: ClinicalTrials.gov public record
Updated Feb 20, 2013 · Synced Jul 22, 2026, 8:48 AM EDT
Completed Phase 3 Interventional Results available

Clinical Study of Aldurazyme in Patients With Mucopolysaccharidosis (MPS) I

NCT00912925
View directory record Official record
Conditions
Mucopolysaccharidosis I, Hurlers Syndrome, Hurler-Scheie Syndrome
Interventions
rhIDU (recombinant human-Alpha-L-Iduronidase), Placebo
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
5 Years and older
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2001
U.S. locations
2
States / cities
New York, New York • Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Apr 6, 2015 · Synced Jul 22, 2026, 8:48 AM 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 22, 2026, 8:48 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, 8:48 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, 8:48 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, 8:48 AM EDT
Completed No phase listed Observational

Mucopolysaccharidosis I (MPS I) Registry

NCT00144794
View directory record Official record
Conditions
Mucopolysaccharidosis I (MPS I)
Interventions
Not listed
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Not listed
Enrollment
1,406 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2024
U.S. locations
53
States / cities
Phoenix, Arizona • Little Rock, Arkansas • Los Angeles, California + 44 more
Source: ClinicalTrials.gov public record
Updated Feb 26, 2026 · Synced Jul 22, 2026, 8:48 AM EDT
Completed Not applicable Interventional

A Study of Intrathecal Enzyme Therapy for Cognitive Decline in MPS I

NCT00852358
View directory record Official record
Conditions
Cognitive Decline, Mucopolysaccharidosis I, Hurler-Scheie Syndrome, Scheie Syndrome, Lysosomal Storage Disease
Interventions
laronidase
Drug
Lead sponsor
Patricia I. Dickson, M.D.
Individual
Eligibility
6 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2015
U.S. locations
3
States / cities
Oakland, California • Torrance, California • Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jan 25, 2016 · Synced Jul 22, 2026, 8:48 AM EDT
Recruiting Phase 1 Interventional

ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I

NCT05682144
View directory record Official record
Conditions
Mucopolysaccharidosis IH/S, Mucopolysaccharidosis IS
Interventions
Autologous Plasmablasts (B cells)
Biological
Lead sponsor
Immusoft of CA, Inc.
Industry
Eligibility
10 Years and older
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2044
U.S. locations
2
States / cities
Oakland, California • Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jul 16, 2026 · Synced Jul 22, 2026, 8:48 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, 8:48 AM EDT
Completed Phase 4 Interventional

A Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG (Glycosaminoglycan) Levels in Aldurazyme® Treated Patients

NCT00144768
View directory record Official record
Conditions
Mucopolysaccharidosis I, Hurler's Syndrome, Hurler-Scheie Syndrome, Scheie's Syndrome
Interventions
laronidase
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Not listed
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2007
U.S. locations
3
States / cities
Los Angles, California • Hartford, Connecticut • Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated May 4, 2014 · Synced Jul 22, 2026, 8:48 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, 8:48 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, 8:48 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects With MPS I

NCT02702115
View directory record Official record
Conditions
MPS I
Interventions
SB-318
Biological
Lead sponsor
Sangamo Therapeutics
Industry
Eligibility
5 Years and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
1
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Jan 25, 2023 · Synced Jul 22, 2026, 8:48 AM EDT
Completed Phase 3 Interventional Results available

Phase 3 Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients

NCT00146770
View directory record Official record
Conditions
Mucopolysaccharidosis I, Hurler's Syndrome, Hurler-Scheie Syndrome, Scheie Syndrome
Interventions
Aldurazyme, placebo
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Not listed
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2005
U.S. locations
8
States / cities
Mobile, Alabama • Atlanta, Georgia • Rochester, New York + 5 more
Source: ClinicalTrials.gov public record
Updated Apr 2, 2015 · Synced Jul 22, 2026, 8:48 AM EDT