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ClinicalTrials.gov public records Last synced Oct 6, 2026, 10:51 PM EDT

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Showing 25–40 of 40 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Sanfilippo Mucopolysaccharidoses Clear all
Completed No phase listed Observational

Natural History Studies of Mucopolysaccharidosis III

NCT02037880
View directory record Official record
Conditions
Mucopolysaccharidosis Type IIIA, Mucopolysaccharidosis Type IIIB
Interventions
Lumbar puncture, Magnetic Resonance Imaging (MRI) of the brain
Procedure · Device
Lead sponsor
Nationwide Children's Hospital
Other
Eligibility
2 Years and older
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Nov 16, 2015 · Synced Oct 6, 2026, 10:51 PM EDT
Not listed Phase 2Phase 3 Interventional

An Open Label Dose Escalation Study to Assess the Safety, Tolerability, and Pharmacologic Properties of High Dose Ambroxol Hydrochloride in Adult (≥ 18 Years of Age) Subjects With MPS III

NCT06614894
View directory record Official record
Conditions
Sanfilippo Syndrome, MPS3
Interventions
Ambroxol Hydrochloride 30 mg tablet - 9 mg/kg/day, Ambroxol Hydrochloride 30 mg tablet - 18 mg/kg/day, Ambroxol Hydrochloride 30 mg tablet - 27 mg/kg/day
Drug
Lead sponsor
Ozlem Goker-Alpan
Other
Eligibility
18 Years to 99 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Aug 20, 2025 · Synced Oct 6, 2026, 10:51 PM EDT
Recruiting Phase 2Phase 3 Interventional

Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH

NCT02716246
View directory record Official record
Conditions
MPS IIIA, Sanfilippo Syndrome, Sanfilippo A, Mucopolysaccharidosis III
Interventions
UX111, Prophylactic Immunomodulatory (IM) Therapy, Optimized Prophylactic IM Therapy, Adjuvant IM Therapy
Biological · Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
Not listed
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2029
U.S. locations
2
States / cities
Columbus, Ohio • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Sep 22, 2026 · Synced Oct 6, 2026, 10:51 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 4, 2019 · Synced Oct 6, 2026, 10:51 PM 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 25, 2023 · Synced Oct 6, 2026, 10:51 PM EDT
Terminated No phase listed Observational

A Long-term Follow-up Study of Patients With MPS IIIB Treated With ABO-101

NCT04655911
View directory record Official record
Conditions
Mucopolysaccharidosis III-B
Interventions
ABO-101
Biological
Lead sponsor
Abeona Therapeutics, Inc
Industry
Eligibility
Not listed
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated May 31, 2022 · Synced Oct 6, 2026, 10:51 PM EDT
Completed No phase listed Observational Results available

A Study of Patients With Sanfilippo Syndrome Type A (MPS IIIA)

NCT01047306
View directory record Official record
Conditions
Sanfilippo Syndrome Type A
Interventions
assessment
Other
Lead sponsor
Shire
Industry
Eligibility
1 Year and older
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jun 11, 2021 · Synced Oct 6, 2026, 10:51 PM EDT
Active, not recruiting Phase 1 Interventional

Phase 1 Study of GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA)

NCT06567769
View directory record Official record
Conditions
Sanfilippo Syndrome Type A
Interventions
GC1130A
Drug
Lead sponsor
GC Biopharma Corp
Industry
Eligibility
12 Months to 18 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
2
States / cities
Oakland, California • Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Sep 8, 2026 · Synced Oct 6, 2026, 10:51 PM EDT
Completed No phase listed Observational

Neurobehavioral Phenotypes in MPS III

NCT01873911
View directory record Official record
Conditions
Sanfilippo Syndrome Type A, Sanfilippo Syndrome Type B, Hurler Syndrome
Interventions
Not listed
Lead sponsor
University of Minnesota
Other
Eligibility
2 Years to 12 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2014
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Nov 1, 2019 · Synced Oct 6, 2026, 10:51 PM EDT
Completed No phase listed Observational

A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIB

NCT02293382
View directory record Official record
Conditions
MPS IIIB (Sanfilippo Syndrome)
Interventions
Not listed
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
Not listed
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Feb 3, 2017 · Synced Oct 6, 2026, 10:51 PM EDT
Not listed Phase 2Phase 3 Interventional

Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA)

NCT03612869
View directory record Official record
Conditions
Mucopolysaccharidosis Type IIIA
Interventions
LYS-SAF302
Drug
Lead sponsor
LYSOGENE
Industry
Eligibility
6 Months and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
4
States / cities
Orange, California • Minneapolis, Minnesota • New York, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 31, 2021 · Synced Oct 6, 2026, 10:51 PM EDT
Completed Phase 1Phase 2 Interventional Results available

A Study to Assess the Safety and Tolerability of SOBI003 in Pediatric MPS IIIA Patients

NCT03423186
View directory record Official record
Conditions
Sanfilippo Syndrome Type A (MPS IIIA)
Interventions
SOBI003
Drug
Lead sponsor
Swedish Orphan Biovitrum
Industry
Eligibility
12 Months to 72 Months
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
2
States / cities
Oakland, California • Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Nov 19, 2021 · Synced Oct 6, 2026, 10:51 PM EDT
Not listed Phase 2 Interventional

A Treatment Extension Study of Mucopolysaccharidosis Type IIIB

NCT03784287
View directory record Official record
Conditions
Mucopolysaccharidosis Type IIIB, MPS III B
Interventions
AX 250
Drug
Lead sponsor
Allievex Corporation
Industry
Eligibility
0 Years to 18 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
2
States / cities
Oakland, California • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 18, 2023 · Synced Oct 6, 2026, 10:51 PM EDT
Completed Not applicable Interventional Results available

Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders

NCT01626092
View directory record Official record
Conditions
Lysosomal Storage Disease, Peroxisomal Disorder
Interventions
Campath-1H, Clofarabine, Melphalan, Total Body Irradiation with Marrow Boosting, Hematopoietic stem cell transplantation, Cyclosporine A, Mycophenolate mofetil
Drug · Radiation · Biological
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 55 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 5, 2017 · Synced Oct 6, 2026, 10:51 PM EDT
Completed No phase listed Observational

A Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)

NCT02493998
View directory record Official record
Conditions
Mucopolysaccharidosis Type IIIB, Mucopolysaccharidosis Type 3 B, MPS III B, MPS 3 B
Interventions
Not listed
Lead sponsor
Allievex Corporation
Industry
Eligibility
1 Year to 10 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
1
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Mar 6, 2020 · Synced Oct 6, 2026, 10:51 PM 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 – 2027
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Sep 3, 2026 · Synced Oct 6, 2026, 10:51 PM EDT