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Conditions: Carpal Tunnel Syndrome
Interventions: Not listed
Lead sponsor: University of Utah; Other
Eligibility: 3 Years to 12 Years
Enrollment: 44 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2013 – 2019
U.S. locations: 1
States / cities: Salt Lake City, Utah

Conditions: Hurler Syndrome, Fanconi Anemia, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease, Severe Congenital Neutropenia, Leukocyte Adhesion Deficiency, Shwachman-Diamond Syndrome, Diamond-Blackfan Anemia, Dyskeratosis-congenita, Chediak-Higashi Syndrome, Severe Aplastic Anemia, Thalassemia Major, Hemophagocytic Lymphohistiocytosis, Sickle Cell Disease
Interventions: Abatacept; Drug
Lead sponsor: Emory University; Other
Eligibility: Up to 21 Years
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2019
U.S. locations: 1
States / cities: Atlanta, Georgia

Conditions: Achondroplasia
Interventions: Not listed
Lead sponsor: QED Therapeutics, a BridgeBio company; Industry
Eligibility: 30 Months to 17 Years
Enrollment: 271 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2025
U.S. locations: 8
States / cities: Oakland, California • Aurora, Colorado • Wilmington, Delaware + 5 more

Conditions: Mucopolysaccharidosis Type II (MPS II)
Interventions: RGX-121; Genetic
Lead sponsor: REGENXBIO Inc.; Industry
Eligibility: 5 Years to 17 Years · Male only
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2024
U.S. locations: 1
States / cities: Oakland, California

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: Severe Combined Immunodeficiency, Malignancy, Hematologic, Neuroblastoma, Neoplasm, Mucopolysaccharidosis I
Interventions: Venipuncture; Procedure
Lead sponsor: St. Jude Children's Research Hospital; Other
Eligibility: Not listed
Enrollment: 41 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2024
U.S. locations: 1
States / cities: Memphis, Tennessee

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: 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: Sanfilippo Syndrome, MPS3
Interventions: Not listed
Lead sponsor: Lysosomal and Rare Disorders Research and Treatment Center, Inc.; Other
Eligibility: 5 Years to 99 Years
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2024
U.S. locations: 1
States / cities: Fairfax, Virginia

Conditions: Hunter Syndrome
Interventions: Neurobehavioral testing, Visual and auditory assessments; Behavioral · Other
Lead sponsor: Shire; Industry
Eligibility: 32 Months to 18 Years · Male only
Enrollment: 33 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2011
U.S. locations: 1
States / cities: Chapel Hill, North Carolina

Conditions: Sanfilippo Syndrome Type A (MPS IIIA)
Interventions: SOBI003; Drug
Lead sponsor: Swedish Orphan Biovitrum; Industry
Eligibility: 12 Months to 72 Months
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2019
U.S. locations: 2
States / cities: Oakland, California • Chapel Hill, North Carolina

Conditions: Inherited Metabolic Diseases, Lysosomal Storage Disorders, Peroxisomal Storage Diseases, Inborn Errors of Metabolism, Mucopolysaccharidosis
Interventions: ALD-101; Biological
Lead sponsor: Aldagen; Industry
Eligibility: Up to 16 Years
Enrollment: 40 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2011
U.S. locations: 3
States / cities: Los Angeles, California • New York, New York • Durham, North Carolina

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: Digestive Health, Abdominal Discomfort, Gas and Bloating
Interventions: Bloat Gummies, Placebo; Dietary Supplement
Lead sponsor: Arrae; Industry
Eligibility: 18 Years to 65 Years
Enrollment: 100 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2026
U.S. locations: 1
States / cities: San Francisco, California

Conditions: Sanfilippo Syndrome Type B
Interventions: Not listed
Lead sponsor: Shire; Industry
Eligibility: 1 Year to 10 Years
Enrollment: 19 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2013
U.S. locations: 2
States / cities: Oakland, 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: MPS II, Hunter Syndrome (MPS II)
Interventions: RGX-121-3102; Genetic
Lead sponsor: REGENXBIO Inc.; Industry
Eligibility: 4 Months to 5 Years · Male only
Enrollment: 2 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2031
U.S. locations: 1
States / cities: New Brunswick, New Jersey

Conditions: Mucopolysaccharidosis III
Interventions: anakinra; Biological
Lead sponsor: Lynda E Polgreen; Other
Eligibility: 4 Years and older
Enrollment: 24 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2023
U.S. locations: 1
States / cities: Torrance, California

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: 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 IVA, Morquio A Syndrome, MPS IVA
Interventions: BMN 110; Drug
Lead sponsor: BioMarin Pharmaceutical; Industry
Eligibility: Not listed
U.S. locations: 24
States / cities: Birmingham, Alabama • Little Rock, Arkansas • Oakland, California + 21 more

Conditions: Sanfilippo Syndrome, MPS3
Interventions: Ambroxol Hydrochloride 30 mg tablet - 9 mg/kg/day, Ambroxol Hydrochloride 30 mg tablet - 18 mg/kg/day, Ambroxol Hydrochloride 30 mg tablet - 27 mg/kg/day; Drug
Lead sponsor: Ozlem Goker-Alpan; Other
Eligibility: 18 Years to 99 Years
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2026
U.S. locations: 1
States / cities: Fairfax, Virginia

Conditions: Hypochondroplasia
Interventions: Not listed
Lead sponsor: QED Therapeutics, a BridgeBio company; Industry
Eligibility: 30 Months to 16 Years
Enrollment: 150 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2026
U.S. locations: 8
States / cities: Oakland, California • Aurora, Colorado • Washington D.C., District of Columbia + 5 more

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

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Ultrasonographic Assessment of Carpal Tunnel Syndrome

Bone Marrow Transplant With Abatacept for Non-Malignant Diseases

Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)

RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome)

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

Long Term Follow Up Of Patients Who Have Received Gene Therapy Or Gene Marked Products

Registry of Patients Diagnosed With Lysosomal Storage Diseases

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

The Natural History Study of Patients With Sanfilippo Disease(s) (MPS3)

Screening Study to Identify Pediatric Patients With Hunter Syndrome Who Demonstrate Evidence of Central Nervous System (CNS) Involvement and Who Are Currently Receiving Treatment With Elaprase®

A Study to Assess the Safety and Tolerability of SOBI003 in Pediatric MPS IIIA Patients

ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases

Intrathecal Enzyme Replacement for Hurler Syndrome

Study to Investigate the Efficacy of a Digestive Health Dietary Supplement on Post-meal Bloating in Healthy Participants

Natural History Study of Patients With Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B)

Unrelated Umbilical Cord Blood Transplantation Augmented With ALDHbr Umbilical Cord Blood Cells

RGX-121-3102 Gene Therapy in Participants With MPS II (Hunter Syndrome)

Open-label Study of Anakinra in MPS III

MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis

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

BMN 110 US Expanded Access Program

An Open Label Dose Escalation Study to Assess the Safety, Tolerability, and Pharmacologic Properties of High Dose Ambroxol Hydrochloride in Adult (≥ 18 Years of Age) Subjects With MPS III

Prospective Clinical Assessment Study in Children With Hypochondroplasia

Stem Cell Transplantation for Hurler