- Conditions
- Hematopoietic Cell Transplantation, Mucopolysaccharidosis Type I
- Interventions
- Laronidase therapy and a stem cell transplant
- Drug
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Other
- Eligibility
- 0 Years to 3 Years
- Enrollment
- 13 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2026
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
Search Results
Search by objective public record fields.
Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results come from ClinicalTrials.gov. When the live registry is unavailable, a clearly labeled stored copy may be shown.
These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.
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 51
matching trials from the live ClinicalTrials.gov search.
- 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
- 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
- Mucopolysaccharidosis Disorders, Hurler Syndrome, Hunter Syndrome, Maroteaux Lamy Syndrome, Sly Syndrome, Alpha-Mannosidosis, Fucosidosis, Aspartylglucosaminuria, Glycoprotein Metabolic Disorders, Sphingolipidoses, Recessive Leukodystrophies, Globoid Cell Leukodystrophy, Metachromatic Leukodystrophy, Niemann-Pick B, Niemann-Pick C Subtype 2, Sphingomyelin Deficiency, Peroxisomal Disorders, Adrenoleukodystrophy With Cerebral Involvement, Zellweger Syndrome, Neonatal Adrenoleukodystrophy, Infantile Refsum Disease, Acyl-CoA Oxidase Deficiency, D-Bifunctional Enzyme Deficiency, Multifunctional Enzyme Deficiency, Alpha-methylacyl-CoA Racmase Deficiency, Mitochondrial Neurogastrointestingal Encephalopathy, Severe Osteopetrosis, Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation), Inherited Metabolic Disorders
- Interventions
- Stem Cell Transplantation, IMD Preparative Regimen, Osteopetrosis Only Preparative Regimen, Osteopetrosis Haploidentical Only Preparative Regimen, cALD SR-A (Standard-Risk, Regimen A), cALD SR-B (Standard-Risk, Regimen B), cALD HR-D (High-Risk, Regimen C), cALD HR-D (High-Risk, Regimen D)
- Biological · Drug
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Other
- Eligibility
- Up to 55 Years
- Enrollment
- 149 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2029
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- 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
- 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 Type I (MPS I), Hurler Syndrome, Hurler-Scheie Syndrome
- Interventions
- RGX-111
- Genetic
- Lead sponsor
- REGENXBIO Inc.
- Industry
- Eligibility
- 4 Months and older
- Enrollment
- 21 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2029
- U.S. locations
- 1
- States / cities
- Orange, California
- Conditions
- Spinal Cord Compression, Mucopolysaccharidosis I, Hurler-Scheie Syndrome, Scheie Syndrome, Lysosomal Storage Disease
- Interventions
- laronidase
- Drug
- Lead sponsor
- Patricia I. Dickson, M.D.
- Individual
- Eligibility
- 8 Years and older
- Enrollment
- 3 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2011
- U.S. locations
- 1
- States / cities
- Torrance, California
- 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
- Inherited Metabolic Disorders (IMD)
- Interventions
- MGTA-456
- Drug
- Lead sponsor
- Magenta Therapeutics, Inc.
- Industry
- Eligibility
- 0 Years to 17 Years
- Enrollment
- 8 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2021
- U.S. locations
- 2
- States / cities
- Minneapolis, Minnesota • Cincinnati, Ohio
- Conditions
- Hurler Syndrome (MPS I), Hurler-Scheie Syndrome, Hunter Syndrome (MPS II), Sanfilippo Syndrome (MPS III), Krabbe Disease (Globoid Leukodystrophy), Metachromatic Leukodystrophy (MLD), Adrenoleukodystrophy (ALD and AMN), Sandhoff Disease, Tay Sachs Disease, Pelizaeus Merzbacher (PMD), Niemann-Pick Disease, Alpha-mannosidosis
- Interventions
- hematopoietic stem cell infusion
- Biological
- Lead sponsor
- Talaris Therapeutics Inc.
- Industry
- Eligibility
- Not listed
- Enrollment
- 3 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2016
- U.S. locations
- 1
- States / cities
- Durham, North Carolina
- 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
- 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
- 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 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
- 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
- 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
- Mucopolysaccharidosis Type I, Mucopolysaccharidosis Type II, Mucopolysaccharidosis Type VI, Mucopolysaccharidosis Type IV, Mucopolysaccharidosis Type VII
- Interventions
- Not listed
- Lead sponsor
- University of Minnesota
- Other
- Eligibility
- 6 Years and older
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2019
- U.S. locations
- 3
- States / cities
- Oakland, California • Minneapolis, Minnesota • New York, New York
- Conditions
- Hurler Syndrome
- Interventions
- IRT Laronidase
- Drug
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Other
- Eligibility
- 6 Months to 3 Years
- Enrollment
- 26 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2018
- 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 (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
- 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
- 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
- Hurler Syndrome
- Interventions
- Laronidase
- Drug
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Other
- Eligibility
- Up to 14 Years
- Enrollment
- 11 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2016
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota