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ClinicalTrials.gov public records Last synced May 21, 2026, 7:22 PM EDT

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Showing 1–15 of 15 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Hurler-Scheie Syndrome Clear all
Completed Phase 3 Interventional Results available
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 May 21, 2026, 7:22 PM EDT
Completed Phase 3 Interventional Results available
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 May 21, 2026, 7:22 PM EDT
Terminated Phase 1Phase 2 Interventional Results available
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 May 21, 2026, 7:22 PM EDT
Active, not recruiting No phase listed Observational
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 May 21, 2026, 7:22 PM EDT
Completed Phase 4 Interventional
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 May 21, 2026, 7:22 PM EDT
Completed No phase listed Observational
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 May 21, 2026, 7:22 PM EDT
Terminated Phase 3 Interventional
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 May 21, 2026, 7:22 PM EDT
Completed Not applicable Interventional
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 May 21, 2026, 7:22 PM EDT
Terminated Phase 1 Interventional
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 May 21, 2026, 7:22 PM EDT
Terminated No phase listed Observational
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 May 21, 2026, 7:22 PM EDT
Completed No phase listed Observational
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 May 21, 2026, 7:22 PM EDT
Suspended Phase 1Phase 2 Interventional
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 May 21, 2026, 7:22 PM EDT
Completed No phase listed Observational
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 May 21, 2026, 7:22 PM EDT
Terminated Phase 1Phase 2 Interventional
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 May 21, 2026, 7:22 PM EDT
Recruiting Phase 1 Interventional
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 Mar 29, 2026 · Synced May 21, 2026, 7:22 PM EDT