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ClinicalTrials.gov public records Last synced May 21, 2026, 8:09 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 1–24 of 37 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: Sanfilippo Mucopolysaccharidoses Clear all
Not listed No phase listed Observational
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 May 21, 2026, 8:09 PM EDT
Completed Phase 1Phase 2 Interventional Results available
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 May 21, 2026, 8:09 PM EDT
Enrolling by invitation Phase 4 Interventional
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 May 21, 2026, 8:09 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional
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 Dec 4, 2025 · Synced May 21, 2026, 8:09 PM EDT
Not listed Phase 2 Interventional
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 May 21, 2026, 8:09 PM EDT
Recruiting No phase listed Observational
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 May 21, 2026, 8:09 PM EDT
Recruiting Phase 1 Interventional
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 Aug 27, 2025 · Synced May 21, 2026, 8:09 PM EDT
Completed Not applicable Interventional Results available
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 4, 2017 · Synced May 21, 2026, 8:09 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Sanfilippo Syndrome Type B
Interventions
Not listed
Lead sponsor
Shire
Industry
Eligibility
1 Year to 10 Years
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
2
States / cities
Oakland, California • Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Mar 16, 2021 · Synced May 21, 2026, 8:09 PM EDT
Recruiting Phase 2Phase 3 Interventional
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 May 21, 2026, 8:09 PM EDT
Recruiting Phase 2Phase 3 Interventional
View directory record Official record
Conditions
Sanfilippo Syndrome, Mucopolysaccharidosis III
Interventions
Epidiolex, Placebo
Drug
Lead sponsor
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Other
Eligibility
4 Years and older
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Torrance, California
Source: ClinicalTrials.gov public record
Updated Apr 5, 2026 · Synced May 21, 2026, 8:09 PM EDT
Recruiting Phase 2Phase 3 Interventional
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 – 2027
U.S. locations
2
States / cities
Columbus, Ohio • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 23, 2026 · Synced May 21, 2026, 8:09 PM EDT
Not listed Phase 2Phase 3 Interventional
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 30, 2021 · Synced May 21, 2026, 8:09 PM EDT
Terminated Phase 2 Interventional Results available
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 May 21, 2026, 8:09 PM EDT
Not listed No phase listed Observational
View directory record Official record
Conditions
Sanfilippo Syndrome, MPS3
Interventions
Not listed
Lead sponsor
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Other
Eligibility
5 Years to 99 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Mar 11, 2024 · Synced May 21, 2026, 8:09 PM EDT
Terminated Phase 1Phase 2 Interventional
View directory record Official record
Conditions
Mucopolysaccharidosis Type 3 B
Interventions
rAAV9.CMV.hNAGLU
Biological
Lead sponsor
Abeona Therapeutics, Inc
Industry
Eligibility
Not listed
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated May 4, 2022 · Synced May 21, 2026, 8:09 PM EDT
Completed No phase listed Observational
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 May 21, 2026, 8:09 PM EDT
Completed No phase listed Observational
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 May 21, 2026, 8:09 PM EDT
Terminated No phase listed Observational
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 May 21, 2026, 8:09 PM EDT
Completed No phase listed Observational Results available
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 May 21, 2026, 8:09 PM EDT
Completed No phase listed Observational
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 May 21, 2026, 8:09 PM EDT
Completed No phase listed Observational
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 Oct 31, 2019 · Synced May 21, 2026, 8:09 PM EDT
Completed Phase 1 Interventional Results available
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 May 21, 2026, 8:09 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, 8:09 PM EDT