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Conditions: Mucopolysaccharidosis, Hurler Syndrome, Hunter Syndrome, Maroteaux-Lamy Syndrome, Sly Syndrome, Alpha Mannosidosis, Fucosidosis, Aspartylglucosaminuria, Adrenoleukodystrophy (ALD), Krabbe Disease, Metachromatic Leukodystrophy (MLD), Sphingolipidoses, Peroxisomal Disorders
Interventions: Campath-1H, Cyclophosphamide, Busulfan, Allogeneic stem cell transplantation, Cyclosporine A, Mycophenolate Mofetil; Drug · Procedure
Lead sponsor: Masonic Cancer Center, University of Minnesota; Other
Eligibility: Up to 21 Years
Enrollment: 46 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2017
U.S. locations: 1
States / cities: Minneapolis, Minnesota

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: 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: 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: 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: 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: Mucopolysaccharidosis I
Interventions: JR-171; Drug
Lead sponsor: JCR Pharmaceuticals Co., Ltd.; Industry
Eligibility: Not listed
Enrollment: 14 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2025
U.S. locations: 1
States / cities: Oakland, California

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: 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: 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: Cognitive Decline, Mucopolysaccharidosis I, Hurler-Scheie Syndrome, Scheie Syndrome, Lysosomal Storage Disease
Interventions: laronidase; Drug
Lead sponsor: Patricia I. Dickson, M.D.; Individual
Eligibility: 6 Years and older
Enrollment: 9 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2015
U.S. locations: 3
States / cities: Oakland, California • Torrance, 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: 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: 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: 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: 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
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 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: 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 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, 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

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: 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

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Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders

Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders

RGX-111 Gene Therapy in Patients With MPS I

Administration of IV Laronidase Post Bone Marrow Transplant in Hurler

A Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG (Glycosaminoglycan) Levels in Aldurazyme® Treated Patients

Cardiac Structure and Function in MPS

An Extension Study of JR-171-101 Study in Patients With Mucopolysaccharidosis Type I (MPS I)

Evaluation of Intravenous Laronidase Pharmacokinetics Before and After Hematopoietic Cell Transplantation in Patients With Mucopolysaccharidosis Type IH.

MRS to Determine Neuroinflammation and Oxidative Stress in MPS I

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

A Study of Intrathecal Enzyme Therapy for Cognitive Decline in MPS I

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

Effects of Growth Hormone in Chronically Ill Children

Extension Study of Intrathecal Enzyme Replacement Therapy for MPS I

Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders

Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation

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

Longitudinal Studies of Brain Structure and Function in MPS Disorders

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

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

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

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

Neurobehavioral Phenotypes in MPS III