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Showing 25–39 of 15 matching trials from the live ClinicalTrials.gov search.

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Filters: Condition: Mucopolysaccharidosis Type 3 A Clear all

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

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

Conditions: Sickle Cell Disease, Thalassemia, Anemia, Granuloma, Wiskott-Aldrich Syndrome, Chediak Higashi Syndrome, Osteopetrosis, Neutropenia, Thrombocytopenia, Hurler Disease, Niemann-Pick Disease, Fucosidosis
Interventions: Hematopoietic stem cell transplantation; Procedure
Lead sponsor: Children's Hospital Los Angeles; Other
Eligibility: Up to 21 Years
Enrollment: 25 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2015
U.S. locations: 1
States / cities: Los Angeles, California

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

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

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

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

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

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

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

Conditions: MPS IV A
Interventions: BMN 110 Weekly, Placebo, BMN 110 Every Other Week; Drug
Lead sponsor: BioMarin Pharmaceutical; Industry
Eligibility: 5 Years and older
Enrollment: 177 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2012
U.S. locations: 6
States / cities: Oakland, California • Wilmington, Delaware • Washington D.C., District of Columbia + 3 more

Conditions: Mucopolysaccharidosis II
Interventions: tividenofusp alfa, idursulfase; Drug
Lead sponsor: Denali Therapeutics Inc.; Industry
Eligibility: 2 Years to 25 Years
Enrollment: 63 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2027
U.S. locations: 8
States / cities: Oakland, California • Chicago, Illinois • Hackensack, New Jersey + 5 more

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

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

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

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Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease

Longitudinal Study of Neurodegenerative Disorders

Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells

A Treatment Study of Mucopolysaccharidosis Type IIIB

Natural History Study of Participants With Sanfilippo Syndrome Type IIIC

A Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)

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

A Natural History Study of Sanfilippo Syndrome Type D

Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of rhHNS Administration Via an IDDD in Pediatric Patients With Early Stage MPS IIIA Disease

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

A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)

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

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

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