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Showing 25–39 of 15 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: MPS III Clear all
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 30, 2022 · Synced Sep 17, 2026, 2:24 AM EDT
Terminated No phase listed Observational

Natural History Study to Characterise the Course of Disease Progression in Participants With Mucopolysaccharidosis Type IIIB

NCT02293408
View directory record Official record
Conditions
MPS IIIB (Sanfilippo B Syndrome)
Interventions
Not listed
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
1 Year and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
2
States / cities
Minneapolis, Minnesota • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Sep 12, 2018 · Synced Sep 17, 2026, 2:24 AM EDT
Completed Phase 1Phase 2 Interventional Results available

A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA Patients

NCT03811028
View directory record Official record
Conditions
Sanfilippo Syndrome Type A (MPS IIIA)
Interventions
SOBI003
Drug
Lead sponsor
Swedish Orphan Biovitrum
Industry
Eligibility
18 Months to 78 Months
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
2
States / cities
Oakland, California • Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Feb 24, 2022 · Synced Sep 17, 2026, 2:24 AM EDT
Enrolling by invitation Phase 4 Interventional

Safety, Tolerability and Efficacy of ICV AX 250 Treatment in MPS IIIB -OLE

NCT05492799
View directory record Official record
Conditions
MPS III B
Interventions
AX 250
Drug
Lead sponsor
Allievex Corporation
Industry
Eligibility
Not listed
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
2
States / cities
Oakland, California • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 8, 2023 · Synced Sep 17, 2026, 2:24 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 Sep 17, 2026, 2:24 AM 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 18, 2021 · Synced Sep 17, 2026, 2:24 AM 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 Sep 17, 2026, 2:24 AM 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 7, 2026 · Synced Sep 17, 2026, 2:24 AM 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 Sep 17, 2026, 2:24 AM EDT
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 15, 2015 · Synced Sep 17, 2026, 2:24 AM EDT
Completed No phase listed Observational Results available

Carotid Structure and Function in MPS Syndromes: A Multicenter Study of the Lysosomal Disease Network

NCT01586871
View directory record Official record
Conditions
Mucopolysaccharidoses
Interventions
Not listed
Lead sponsor
University of Minnesota
Other
Eligibility
3 Years to 18 Years
Enrollment
1,147 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2017
U.S. locations
2
States / cities
Orange, California • Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Feb 23, 2021 · Synced Sep 17, 2026, 2:24 AM EDT
Terminated Phase 2 Interventional Results available

An Extension Study to Determine Safety and Efficacy for Pediatric Patients With MPS Type IIIA Disease Who Participated in Study HGT-SAN-093.

NCT02350816
View directory record Official record
Conditions
Sanfilippo Syndrome, Mucopolysaccharidosis (MPS)
Interventions
HGT-1410
Drug
Lead sponsor
Shire
Industry
Eligibility
12 Months to 48 Months
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
2
States / cities
Minneapolis, Minnesota • Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Jun 10, 2021 · Synced Sep 17, 2026, 2:24 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 Sep 17, 2026, 2:24 AM EDT
Completed No phase listed Observational

A Natural History Study of Biomarkers and Clinical Outcomes in Mucopolysaccharidosis Type IIIA (MPS IIIA; Sanfilippo Syndrome)

NCT05523206
View directory record Official record
Conditions
Mucopolysaccharidosis III-A
Interventions
No intervention
Other
Lead sponsor
Sanguine Biosciences
Industry
Eligibility
4 Months to 13 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
1
States / cities
Woburn, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jan 29, 2025 · Synced Sep 17, 2026, 2:24 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 Sep 17, 2026, 2:24 AM EDT