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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: 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: 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, Mucopolysaccharidosis II, Mucopolysaccharidosis VI
Interventions: Somatropin (DNA origin); Drug
Lead sponsor: Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center; Other
Eligibility: 5 Years to 17 Years
Enrollment: 2 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2013
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: 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: 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: 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 (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: 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: 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: Hemophilia B, Mucopolysaccharidosis I, Mucopolysaccharidosis II
Interventions: SB-318, SB-913, SB-FIX; Biological
Lead sponsor: Sangamo Therapeutics; Industry
Eligibility: 18 Years and older
Enrollment: 13 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2026
U.S. locations: 5
States / cities: Oakland, California • Chicago, Illinois • New York, New York + 2 more

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: 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: 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 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 (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: 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-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: 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: 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 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: 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 IH, MPS I, Hurler Syndrome
Interventions: Laronidase; Drug
Lead sponsor: Masonic Cancer Center, University of Minnesota; Other
Eligibility: Not listed
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2018
U.S. locations: 1
States / cities: Minneapolis, Minnesota

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

Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders

Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation

Clinical Trial of Growth Hormone in MPS I, II, and VI

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

Extension Study of Intrathecal Enzyme Replacement Therapy for MPS I

Longitudinal Study of Neurodegenerative Disorders

Administration of IV Laronidase Post Bone Marrow Transplant in Hurler

Mucopolysaccharidosis I (MPS I) Registry

PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

Neurobehavioral Phenotypes in MPS III

Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX

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

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

MRS to Determine Neuroinflammation and Oxidative Stress in MPS I

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

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)

Cardiac Structure and Function in MPS

A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT

BPX-501 T Cells Infused Post Stem Cell Transplant in Pediatrics With Non-Malignant Disorders Ineligible for BPU004 Study

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

Effects of Growth Hormone in Chronically Ill Children

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

Laronidase (Aldurazyme TM) Enzyme Replacement Therapy With Hematopoietic Stem Cell Transplant for Hurler Syndrome