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Conditions: Morquio A Syndrome, Mucopolysaccharidosis IV A
Interventions: Not listed
Lead sponsor: Nadia Ali, PhD; Other
Eligibility: 18 Years and older
Enrollment: 12 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2018
U.S. locations: 1
States / cities: Decatur, Georgia

Conditions: Niemann-Pick Disease, Type C
Interventions: Parts A/B: Adrabetadex, Parts A/B: Sham Control; Drug · Other
Lead sponsor: Mandos LLC; Industry
Eligibility: 4 Years to 21 Years
Enrollment: 56 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2018
U.S. locations: 14
States / cities: Birmingham, Alabama • Orange, California • San Francisco, California + 11 more

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: 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: Fabry Disease
Interventions: Replagal; Biological
Lead sponsor: Shire; Industry
Eligibility: 18 Years and older
Enrollment: 35 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2013
U.S. locations: 2
States / cities: Iowa City, Iowa • Fairfax, Virginia

Conditions: Pompe Disease (Late-onset), Pompe Disease Late-Onset, LOPD
Interventions: AB-1009 (GAA Gene); Genetic
Lead sponsor: AskBio Inc; Industry
Eligibility: 18 Years to 65 Years
Enrollment: 12 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2032
U.S. locations: 9
States / cities: Phoenix, Arizona • Irvine, California • Palo Alto, California + 6 more

Conditions: Parkinson Disease
Interventions: LY3884961, Methylprednisolone, Sirolimus; Biological · Drug
Lead sponsor: Prevail Therapeutics; Industry
Eligibility: 35 Years to 80 Years
Enrollment: 32 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2031
U.S. locations: 11
States / cities: Scottsdale, Arizona • Murrieta, California • Englewood, Colorado + 7 more

Conditions: Glycogen Storage Disease
Interventions: Glycogen Storage Disease markers; Genetic
Lead sponsor: University of Florida; Other
Eligibility: 18 Years to 100 Years
Enrollment: 114 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2016
U.S. locations: 1
States / cities: Gainesville, Florida

Conditions: Pompe Disease (Late-onset)
Interventions: zocaglusagene nuzaparvovec; Genetic
Lead sponsor: Astellas Gene Therapies; Industry
Eligibility: 18 Years to 80 Years
Enrollment: 11 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2035
U.S. locations: 3
States / cities: Orange, California • Palo Alto, California • Salt Lake City, Utah

Conditions: Tay-Sachs Disease, Sandhoff Disease
Interventions: AXO-AAV-GM2 Starting Dose, AXO-AAV-GM2 Low Dose, AXO-AAV-GM2 Middle Dose, AXO-AAV-GM2 High Dose; Biological
Lead sponsor: Terence Flotte; Other
Eligibility: 6 Months to 12 Years
Enrollment: 9 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2024
U.S. locations: 2
States / cities: Boston, Massachusetts • Worcester, Massachusetts

Conditions: Hypochondroplasia
Interventions: infigratinib 0.128 mg/kg/day, infigratinib 0.25 mg/kg/day; Drug
Lead sponsor: QED Therapeutics, a BridgeBio company; Industry
Eligibility: 3 Years to 18 Years
Enrollment: 24 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2027
U.S. locations: 8
States / cities: Oakland, California • Aurora, Colorado • Washington D.C., District of Columbia + 5 more

Conditions: Sphingomyelin Lipidosis
Interventions: placebo (saline), Olipudase alfa; Drug
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 18 Years and older
Enrollment: 36 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2015 – 2023
U.S. locations: 3
States / cities: San Francisco, California • Decatur, Georgia • The Bronx, New York

Conditions: Pompe Disease, Glycogen Storage Disease Type II, Acid Maltase Deficiency Disease, Glycogenosis 2
Interventions: recombinant human acid alpha-glucosidase (rhGAA); Drug
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: Not listed
Enrollment: 8 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2001 – 2002
U.S. locations: 1
States / cities: Durham, North Carolina

Conditions: Melanoma
Interventions: Blood Sample, Tumor Sample; Procedure
Lead sponsor: M.D. Anderson Cancer Center; Other
Eligibility: Not listed
Enrollment: 131 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2006 – 2022
U.S. locations: 1
States / cities: Houston, Texas

Conditions: Anemia
Interventions: Short storage RBC age; Biological
Lead sponsor: Washington University School of Medicine; Other
Eligibility: 3 Days to 15 Years
Enrollment: 1,538 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2018
U.S. locations: 20
States / cities: Birmingham, Alabama • Tucson, Arizona • Los Angeles, California + 17 more

Conditions: Mucopolysaccharidosis IIIB
Interventions: SBC-103; Drug
Lead sponsor: Alexion Pharmaceuticals, Inc.; Industry
Eligibility: 2 Years to 12 Years
Enrollment: 11 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2017
U.S. locations: 2
States / cities: Minneapolis, Minnesota • Pittsburgh, Pennsylvania

Conditions: IBS (Irritable Bowel Syndrome), Bloating, Abdominal Pain, Gut Health
Interventions: FODMAP-targeting digestive enzyme blend, Placebo; Dietary Supplement · Other
Lead sponsor: Kiwi Health Inc; Industry
Eligibility: 18 Years and older
Enrollment: 150 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2026
U.S. locations: 1
States / cities: Los Angeles, California

Conditions: MPS IV A, Mucopolysaccharidosis IVA, Morquio A Syndrome
Interventions: Not listed
Lead sponsor: BioMarin Pharmaceutical; Industry
Eligibility: Not listed
Enrollment: 353 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2014
U.S. locations: 2
States / cities: Oakland, California • Chicago, Illinois

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: Sickle Cell Disease, Sickle Cell Trait
Interventions: Not listed
Lead sponsor: National Heart, Lung, and Blood Institute (NHLBI); NIH
Eligibility: 18 Years to 45 Years
Enrollment: 307 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2001 – 2023
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Gaucher's Disease, Osteopenia
Interventions: alendronate sodium, calcium carbonate, cholecalciferol; Drug
Lead sponsor: Children's Hospital Medical Center, Cincinnati; Other
Eligibility: 18 Years to 50 Years
Enrollment: 82 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 1998 – 2002
U.S. locations: 2
States / cities: Cincinnati, Ohio • Dayton, Ohio

Conditions: Fabry Disease
Interventions: Migalastat HCl, Agalsidase Beta, Agalsidase Alfa; Drug · Biological
Lead sponsor: Amicus Therapeutics; Industry
Eligibility: 18 Years to 65 Years · Male only
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2012
U.S. locations: 5
States / cities: Birmingham, Alabama • Decatur, Georgia • Iowa City, Iowa + 2 more

Conditions: Skin Cancer, Screening, Telemedicine
Interventions: In-person dermatology evaluation, Store-and-forward teledermatology evaluation; Other
Lead sponsor: University of California, Davis; Other
Eligibility: 18 Years and older
Enrollment: 170 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2010 – 2011
U.S. locations: 1
States / cities: Sacramento, California

Conditions: Pompe Disease Late-Onset, Glycogen Storage Disease Type II GSD II
Interventions: Myozyme; Biological
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 16 Years and older · Female only
Enrollment: 1 participant
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2003 – 2006
U.S. locations: 1
States / cities: San Antonio, Texas

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Psychological Concomitants of Morquio A Syndrome - Longitudinal Effects of Enzyme Replacement Therapy (The MAPLE Study)

VTS-270 to Treat Niemann-Pick Type C1 (NPC1) Disease

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

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

Extension Study of TKT028 Evaluating Safety and Clinical Outcomes of Replagal® in Adult Patients With Fabry Disease

A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late-Onset Pompe Disease (PROGRESS-GT LOPD)

Phase 1/2a Clinical Trial of PR001 (LY3884961) in Patients With Parkinson's Disease With at Least One GBA1 Mutation (PROPEL)

Study of Glycogen Storage Disease Expression in Carriers

Gene Transfer Study in Patients With Late Onset Pompe Disease

A Dose-escalation and Safety & Efficacy Study of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease

An Interventional Study of Infigratinib in Children With Hypochondroplasia

Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency

Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe Disease

Sample Collection From Melanoma Patients

Age of Blood in Children in Pediatric Intensive Care Units

Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in Mucopolysaccharidosis III, Type B (MPS IIIB)

A Study Evaluating the Impact of Regular FODMAP-targeting Digestive Enzyme Blend Use on Gastrointestinal Symptoms in Individuals With Self-Reported Bloating

A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)

Registry of Patients Diagnosed With Lysosomal Storage Diseases

Collection and Storage of Umbilical Cord Stem Cells for Treatment of Sickle Cell Disease

Phase II Randomized Study of Alendronate Sodium for Osteopenia in Patients With Gaucher's Disease

Drug-Drug Interaction Study Between AT1001 (Migalastat Hydrochloride) and Agalsidase in Participants With Fabry Disease

Evaluation of Store-and-Forward Teledermatology Versus a Face-to-Face Assessment During a Skin Cancer Screening Event

Extension Study of Long-term Safety and Efficacy of Myozyme for a Single Patient With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored ERT Studies.