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Showing 1–24 of 37 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Sanfilippo A Clear all
Completed No phase listed Observational

A Natural History Study of Sanfilippo Syndrome Type D

NCT05648851
View directory record Official record
Conditions
Sanfilippo Syndrome Type D
Interventions
Not listed
Lead sponsor
Phoenix Nest
Industry
Eligibility
12 Months and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 31, 2025 · Synced Jul 23, 2026, 2:18 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in Mucopolysaccharidosis III, Type B (MPS IIIB)

NCT02324049
View directory record Official record
Conditions
Mucopolysaccharidosis IIIB
Interventions
SBC-103
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
2 Years to 12 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
2
States / cities
Minneapolis, Minnesota • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 20, 2018 · Synced Jul 23, 2026, 2:18 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 10, 2021 · Synced Jul 23, 2026, 2:18 PM EDT
Recruiting 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 19, 2025 · Synced Jul 23, 2026, 2:18 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 17, 2023 · Synced Jul 23, 2026, 2:18 PM 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 Jul 23, 2026, 2:18 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 Jul 16, 2026 · Synced Jul 23, 2026, 2:18 PM EDT
Not listed No phase listed Observational

A Prospective Natural History Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)

NCT03227042
View directory record Official record
Conditions
Mucopolysaccharidosis Type IIIB
Interventions
Not listed
Lead sponsor
Allievex Corporation
Industry
Eligibility
Up to 18 Years
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2025
U.S. locations
1
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Oct 25, 2022 · Synced Jul 23, 2026, 2:18 PM EDT
Recruiting No phase listed Observational

Natural History Study of Participants With Sanfilippo Syndrome Type IIIC

NCT05825131
View directory record Official record
Conditions
Sanfilippo Syndrome Type C
Interventions
Not listed
Lead sponsor
Phoenix Nest
Industry
Eligibility
12 Months and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated May 11, 2026 · Synced Jul 23, 2026, 2:18 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 5, 2020 · Synced Jul 23, 2026, 2:18 PM 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 Jul 23, 2026, 2:18 PM EDT
Not yet recruiting No phase listed Observational

Natural History to Assess Disease in Patients With MPS IIIC

NCT07712003
View directory record Official record
Conditions
Sanfilippo Syndrome Type C, Mucopolysaccharidosis (MPS) IIIC
Interventions
Not listed
Lead sponsor
Phoenix Nest
Industry
Eligibility
1 Year to 25 Years
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Jul 16, 2026 · Synced Jul 23, 2026, 2:18 PM EDT
Completed Phase 1Phase 2 Interventional

Open-label Study of Anakinra in MPS III

NCT04018755
View directory record Official record
Conditions
Mucopolysaccharidosis III
Interventions
anakinra
Biological
Lead sponsor
Lynda E Polgreen
Other
Eligibility
4 Years and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
1
States / cities
Torrance, California
Source: ClinicalTrials.gov public record
Updated Oct 21, 2024 · Synced Jul 23, 2026, 2:18 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 18, 2021 · Synced Jul 23, 2026, 2:18 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 2, 2017 · Synced Jul 23, 2026, 2:18 PM 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 Jul 23, 2026, 2:18 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 23, 2026, 2:18 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 30, 2022 · Synced Jul 23, 2026, 2:18 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 – 2026
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Sep 7, 2025 · Synced Jul 23, 2026, 2:18 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)

NCT06181136
View directory record Official record
Conditions
Mucopolysaccharidosis Type IIIA
Interventions
DNL126
Drug
Lead sponsor
Denali Therapeutics Inc.
Industry
Eligibility
0 Years to 18 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
4
States / cities
Oakland, California • Iowa City, Iowa • Chapel Hill, North Carolina + 1 more
Source: ClinicalTrials.gov public record
Updated Jul 12, 2026 · Synced Jul 23, 2026, 2:18 PM 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 Jul 23, 2026, 2:18 PM EDT
Completed Phase 1 Interventional Results available

Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders

NCT00744692
View directory record Official record
Conditions
Non Malignant Disorders, Immunodeficiencies, Congenital Marrow Failures, Hemoglobinopathies, Inborn Errors of Metabolism, Sickle Cell, Thalassemia, Lysosomal Storage Disease
Interventions
Unrelated Umbilical Cord Blood Transplant, Reduced Intensity Conditioning
Biological · Drug
Lead sponsor
Duke University
Other
Eligibility
Up to 21 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2014
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Aug 12, 2014 · Synced Jul 23, 2026, 2:18 PM 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 Jul 23, 2026, 2:18 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 23, 2026, 2:18 PM EDT