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ClinicalTrials.gov public records Last synced Oct 10, 2026, 12:53 AM EDT

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

Showing 49–62 of 62 matching trials from the live ClinicalTrials.gov search.

Filters: Condition: Mucopolysaccharidosis Type I (MPS I) Clear all

Conditions: MPS I, MPS IV A, MPS - Mucopolysaccharidosis, MPS IVA
Interventions: Not listed
Lead sponsor: Children's Hospital of Orange County; Other
Eligibility: 0 Years to 99 Years
Enrollment: 240 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2025 – 2030
U.S. locations: 3
States / cities: Orange, California • Minneapolis, Minnesota • St Louis, Missouri

Conditions: MPS I
Interventions: SB-318; Biological
Lead sponsor: Sangamo Therapeutics; Industry
Eligibility: 5 Years and older
Enrollment: 3 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2021
U.S. locations: 1
States / cities: Oakland, California

Conditions: MDS, Anemia, Aplastic, Inborn Errors of Metabolism, Congenital Marrow Failure, Congenital Immunodeficiency Syndrome
Interventions: ALDHbr Umbilical Cord Blood Cells; Biological
Lead sponsor: Joanne Kurtzberg, MD; Other
Eligibility: Up to 55 Years
Enrollment: 37 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2012
U.S. locations: 1
States / cities: Durham, North Carolina

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: Hurler Syndrome, Cerebral Palsy, Juvenile Rheumatoid Arthritis, Crohn Disease, HIV Infections
Interventions: Growth Hormone, Whole body Protein turnover, DEXA scan; Drug · Procedure
Lead sponsor: University of Texas Southwestern Medical Center; Other
Eligibility: 3 Years to 17 Years
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2006
U.S. locations: 1
States / cities: Dallas, Texas

Conditions: Inherited Metabolic Disorders (IMD)
Interventions: Safety and efficacy assessments; Other
Lead sponsor: Magenta Therapeutics, Inc.; Industry
Eligibility: Up to 16 Years
Enrollment: 3 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2020
U.S. locations: 1
States / cities: Minneapolis, Minnesota

Conditions: Mucopolysaccharidosis I, Hurler's Syndrome, Hurler-Scheie Syndrome, Scheie Syndrome
Interventions: Aldurazyme, placebo; Biological
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: Not listed
Enrollment: 45 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2001 – 2005
U.S. locations: 8
States / cities: Mobile, Alabama • Atlanta, Georgia • Rochester, New York + 5 more

Conditions: Mucopolysaccharidosis I
Interventions: AGT-181 (HIRMAb-IDUA); Drug
Lead sponsor: ArmaGen, Inc; Industry
Eligibility: 18 Years and older
Enrollment: 3 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2017
U.S. locations: 6
States / cities: Oakland, California • Orange, California • Decatur, Georgia + 3 more

Conditions: Mucopolysaccharidosis I (MPS I)
Interventions: Not listed
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: Not listed
Enrollment: 1,406 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2003 – 2024
U.S. locations: 53
States / cities: Phoenix, Arizona • Little Rock, Arkansas • Los Angeles, California + 44 more

Conditions: Mucopolysaccharidosis I, Cognitive Decline
Interventions: Intrathecal recombinant human alpha iduronidase; Drug
Lead sponsor: agnes chen; Other
Eligibility: 6 Years and older
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2020
U.S. locations: 1
States / cities: Torrance, California

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: MPS I, MPS II, MPS IVA, MPS VI, Mps VII, Gaucher Disease, Type 2, Gaucher Disease, Type 3, Pompe Disease Infantile-Onset, Wolman Disease
Interventions: Aldurazyme (laronidase); Drug
Lead sponsor: University of California, San Francisco; Other
Eligibility: 18 Years to 50 Years · Female only
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2032
U.S. locations: 1
States / cities: San Francisco, California

Conditions: Mucopolysaccharidosis I
Interventions: JR-171 (lepunafusp alfa); Drug
Lead sponsor: JCR Pharmaceuticals Co., Ltd.; Industry
Eligibility: 0 Years and older
Enrollment: 18 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2022
U.S. locations: 1
States / cities: Oakland, California

Conditions: Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis VI
Interventions: Adalimumab Injection [Humira], Saline Solution for Injection; Drug
Lead sponsor: Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center; Other
Eligibility: 5 Years and older
Enrollment: 14 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2027
U.S. locations: 1
States / cities: Torrance, California

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Cardiac Structure and Function in MPS

Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects With MPS I

Unrelated Umbilical Cord Blood Transplantation Augmented With ALDHbr Umbilical Cord Blood Cells

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

Effects of Growth Hormone in Chronically Ill Children

A Follow-up Study in Patients With Inherited Metabolic Disorders (IMD) Who Underwent Hematopoietic Stem Cell Transplantation (HSCT) With MGTA-456

Phase 3 Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients

Safety and Dose Ranging Study of Insulin Receptor MAb-IDUA Fusion Protein in Patients With MPS I

Mucopolysaccharidosis I (MPS I) Registry

Extension Study of Intrathecal Enzyme Replacement for Cognitive Decline in MPS I

Longitudinal Study of Neurodegenerative Disorders

PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

A Study of JR-171 in Patients With Mucopolysaccharidosis I

Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I, II, and VI