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ClinicalTrials.gov public records Last synced Oct 6, 2026, 4:58 PM EDT

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Showing 49–65 of 65 matching trials from the live ClinicalTrials.gov search.

Filters: Condition: Mucopolysaccharidosis Type 1 Clear all

Conditions: Mucopolysaccharidosis I, Lysosomal Storage Diseases, Spinal Cord Compression
Interventions: laronidase; Drug
Lead sponsor: Patricia I. Dickson, M.D.; Individual
Eligibility: 8 Years and older
Enrollment: 4 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2011
U.S. locations: 1
States / cities: Torrance, California

Conditions: Mucopolysaccharidosis IH/S, Mucopolysaccharidosis IS
Interventions: Autologous Plasmablasts (B cells); Biological
Lead sponsor: Immusoft of CA, Inc.; Industry
Eligibility: 10 Years and older
Enrollment: 11 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2044
U.S. locations: 2
States / cities: Oakland, California • Minneapolis, Minnesota

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: Mucopolysaccharidosis Type I (MPS I), Mucopolysaccharidosis Type II (MPS II), Mucopolysaccharidosis Type III (MPS III), Mucopolysaccharidosis Type VI (MPS VI), Krabbe Disease
Interventions: Not listed
Lead sponsor: University of Chicago; Other
Eligibility: 1 Day to 18 Years
Enrollment: 19 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2016
U.S. locations: 3
States / cities: Chicago, Illinois • Minneapolis, Minnesota • Buffalo, New York

Conditions: Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions, Systemic Juvenile Idiopathic Arthritis (sJIA), Juvenile Rheumatoid Arthritis (JRA)
Interventions: Hydroxyurea, Alemtuzumab, Fludarabine, Melphalan, Thiotepa; Drug
Lead sponsor: Paul Szabolcs; Other
Eligibility: 2 Months to 55 Years
Enrollment: 100 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2027
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: Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions
Interventions: data collection; Drug
Lead sponsor: Paul Szabolcs; Other
Eligibility: 2 Months to 60 Years
Enrollment: 50 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2028
U.S. locations: 1
States / cities: Pittsburgh, Pennsylvania

Conditions: Mucopolysaccharidosis I
Interventions: No Intervention; Genetic
Lead sponsor: REGENXBIO Inc.; Industry
Eligibility: Not listed
Enrollment: 21 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2027
U.S. locations: 1
States / cities: Orange, California

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: Mucopolysaccharidosis I, Mucopolysaccharidosis VI, Mannosidosis, Mucolipidosis Type II (I-cell Disease)
Interventions: Stem Cell Transplant, Busulfan, Cyclophosphamide, ATG; Procedure · Drug
Lead sponsor: Masonic Cancer Center, University of Minnesota; Other
Eligibility: Not listed
Enrollment: 41 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 1999 – 2010
U.S. locations: 1
States / cities: Minneapolis, Minnesota

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

Conditions: Mucopolysaccharidosis Type I
Interventions: Not listed
Lead sponsor: University of Minnesota; Other
Eligibility: 6 Years and older
Enrollment: 30 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2018 – 2019
U.S. locations: 1
States / cities: Minneapolis, Minnesota

Conditions: Mucopolysaccharidoses
Interventions: Not listed
Lead sponsor: Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center; Other
Eligibility: 5 Years to 35 Years
Enrollment: 55 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2019
U.S. locations: 3
States / cities: Oakland, California • Torrance, California • Minneapolis, Minnesota

Conditions: Hurler's Syndrome, Maroteaux-Lamy Syndrome, Sly Syndrome, Alpha Mannosidosis, Fucosidosis, Aspartylglucosaminuria, Sphingolipidoses, Krabbe Disease, Wolman's Disease, Niemann-Pick Disease Type B, Niemann-Pick Disease, Type C
Interventions: Stem Cell Transplantation, Cyclophosphamide, Campath-1H, Busulfan; Procedure · Drug
Lead sponsor: Masonic Cancer Center, University of Minnesota; Other
Eligibility: Up to 21 Years
Enrollment: 18 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2010
U.S. locations: 1
States / cities: Minneapolis, Minnesota

Conditions: Mucopolysaccharidosis I, Hurlers Syndrome, Hurler-Scheie Syndrome
Interventions: rhIDU (recombinant human-Alpha-L-Iduronidase), Placebo; Biological
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 5 Years and older
Enrollment: 45 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2000 – 2001
U.S. locations: 2
States / cities: New York, New York • Chapel Hill, North Carolina

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 (MPS)
Interventions: Not listed
Lead sponsor: Shire; Industry
Eligibility: Up to 7 Years · Male only
Enrollment: 159 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2015
U.S. locations: 7
States / cities: Los Angeles, California • Jacksonville, Florida • Minneapolis, Minnesota + 4 more

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Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) I

ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I

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

Lysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool Children

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT

PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC

Long Term Follow-Up for RGX-111

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

Stem Cell Transplantation for Hurler

Neurobehavioral Phenotypes in MPS III

MRS to Determine Neuroinflammation and Oxidative Stress in MPS I

Longitudinal Study of Bone Disease in Children with Mucopolysaccharidoses (MPS) I, II, and VI

Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism

Clinical Study of Aldurazyme in Patients With Mucopolysaccharidosis (MPS) I

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

Mucopolysaccharidosis (MPS) I, II, and VI Screening in a High-Risk Population With Previous Surgical Repair or Presence of Inguinal and/or Umbilical Hernia in Combination With Pediatric ENT Surgery (The HATT Project)