- 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
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 68
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis IV A, Mucopolysaccharidosis VI, Mucopolysaccharidosis VII, Pompe Disease Infantile-Onset, Neuronopathic Gaucher Disease, Wolman Disease
- Interventions
- There is no intervention
- Other
- Lead sponsor
- University of California, San Francisco
- Other
- Eligibility
- Up to 64 Years
- Enrollment
- 250 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2050
- U.S. locations
- 1
- States / cities
- San Francisco, California
- Conditions
- Hurler Syndrome, Inherited Metabolic Disorder, Lysosomal Storage Disorder, Metachromatic Leukodystrophy, Inborn Errors of Metabolism
- Interventions
- rivogenlecleucel, rimiducid
- Biological · Drug
- Lead sponsor
- Bellicum Pharmaceuticals
- Industry
- Eligibility
- 3 Months to 21 Years
- U.S. locations
- 2
- States / cities
- Los Angeles, California • Palo Alto, California
- 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
- 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
- Mucopolysaccharidoses, Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis IV, Mucopolysaccharidosis VI
- Interventions
- Dried blood spot test for MPS
- Other
- Lead sponsor
- National MPS Society
- Other
- Eligibility
- 6 Months to 18 Years
- Enrollment
- 3,000 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2014
- U.S. locations
- 2
- States / cities
- New Brunswick, New Jersey • New York, New York
- Conditions
- MPS-IH (Hurler Syndrome)
- Interventions
- Experimental: OTL-203, Active Comparator: Allo-HSCT
- Genetic
- Lead sponsor
- Orchard Therapeutics
- Industry
- Eligibility
- 28 Days to 30 Months
- Enrollment
- 41 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2031
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- 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
- 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
- Mucopolysaccharidosis I, Hurler's Syndrome, Hurler-Scheie Syndrome, Scheie's Syndrome
- Interventions
- laronidase
- Drug
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- Not listed
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2004 – 2007
- U.S. locations
- 3
- States / cities
- Los Angles, California • Hartford, Connecticut • Milwaukee, Wisconsin
- 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
- Conditions
- Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy, Tay Sachs Disease, Sandhoffs Disease, Wolman Disease, I-Cell Disease, Sanfilippo Syndrome, GM1 Gangliosidosis
- Interventions
- Clofarabine, Total body Irradiation, Melphalan, Hematopoietic Stem Cell Transplantation, Alemtuzumab, mycophenylate mofetil, Cyclosporine A, Hydroxyurea
- Drug · Procedure · Biological + 1 more
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Other
- Eligibility
- Up to 70 Years
- Enrollment
- 38 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2014
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- 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
- Conditions
- Hunter Syndrome
- Interventions
- Control, Idursulfase IT (1 mg), Idursulfase IT (10 mg), Idursulfase IT (30 mg)
- Other · Drug
- Lead sponsor
- Shire
- Industry
- Eligibility
- 3 Years to 18 Years · Male only
- Enrollment
- 16 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2012
- U.S. locations
- 1
- States / cities
- Chapel Hill, North Carolina
- 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
- 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
- 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
- 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
- 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 II, MPS II
- Interventions
- SB-913
- Biological
- Lead sponsor
- Sangamo Therapeutics
- Industry
- Eligibility
- 5 Years to 65 Years
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2021
- U.S. locations
- 5
- States / cities
- Oakland, California • Chicago, Illinois • New York, New York + 2 more
- 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
- Mucopolysaccharidosis I, Mucopolysaccharidosis VI, Adrenoleukodystrophy, Niemann-Pick Disease, Metachromatic Leukodystrophy, Wolman Disease, Krabbe's Disease, Gaucher's Disease, Fucosidosis, Batten Disease, Severe Aplastic Anemia, Diamond-Blackfan Anemia, Amegakaryocytic Thrombocytopenia, Myelodysplastic Syndrome, Acute Myelogenous Leukemia, Acute Lymphocytic Leukemia
- Interventions
- Human Placental Derived Stem Cell
- Drug
- Lead sponsor
- New York Medical College
- Other
- Eligibility
- 0 Years to 55 Years
- Enrollment
- 43 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2022
- U.S. locations
- 3
- States / cities
- Denver, Colorado • Valhalla, New York • Salt Lake City, Utah
- Conditions
- Bone Marrow Failure Syndrome, Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia, Acquired Neutropenia in Newborn, Acquired Anemia Hemolytic, Acquired Thrombocytopenia, Hemophagocytic Lymphohistiocytoses, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease, Common Variable Immunodeficiency, X-linked Lymphoproliferative Disease, Severe Combined Immunodeficiency, Hurler Syndrome, Mannosidosis, Adrenoleukodystrophy
- Interventions
- Thiotepa--single daily dose, Thiotepa--escalated dose
- Drug
- Lead sponsor
- University of Florida
- Other
- Eligibility
- 3 Months to 39 Years
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2023
- U.S. locations
- 1
- States / cities
- Gainesville, Florida
- Conditions
- Mucopolysaccharidosis I, Hurler Syndrome
- Interventions
- Stem Cell Transplant, Laronidase ERT
- Procedure · Drug
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Other
- Eligibility
- Up to 7 Years
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2004 – 2012
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota