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Conditions: Cerebral Adrenoleukodystrophy (CALD), Adrenoleukodystrophy (ALD), X-Linked Adrenoleukodystrophy (X-ALD)
Interventions: No interventional drug product utilized in this follow-up study; Genetic
Lead sponsor: Genetix Biotherapeutics Inc.; Industry
Eligibility: Up to 19 Years · Male only
Enrollment: 64 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2038
U.S. locations: 4
States / cities: Los Angeles, California • Palo Alto, California • Boston, Massachusetts + 1 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: Adrenoleukodystrophy, Batten Disease, Mucopolysaccharidosis II, Leukodystrophy, Globoid Cell, Leukodystrophy, Metachromatic, Neimann Pick Disease, Pelizaeus-Merzbacher Disease, Sandhoff Disease, Tay-Sachs Disease, Brain Diseases, Metabolic, Inborn, Alpha-Mannosidosis, Sanfilippo Mucopolysaccharidoses
Interventions: DUOC-01; Biological
Lead sponsor: Joanne Kurtzberg, MD; Other
Eligibility: 1 Week to 22 Years
Enrollment: 40 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2027
U.S. locations: 1
States / cities: Durham, North Carolina

Conditions: Neurodegenerative Diseases, Leukodystrophy, Ataxia, LBSL, Leukoencephalopathy with Brain Stem and Spinal Cord Involvement and High Lactate Syndrome (Disorder), Leukoencephalopathy with Brainstem and Spinal Cord Involvement and Lactate Elevation, Cerebellar Ataxia, Adrenomyeloneuropathy
Interventions: Home exercise; Behavioral
Lead sponsor: Hugo W. Moser Research Institute at Kennedy Krieger, Inc.; Other
Eligibility: 5 Years to 75 Years
Enrollment: 30 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2023 – 2027
U.S. locations: 1
States / cities: Baltimore, Maryland

Conditions: Sickle Cell Disease, Thalassemia, High Risk Hematologic Disorders, Cerebral Adrenoleukodystrophy, Inherited Metabolic Disorders
Interventions: Blood and Marrow Transplant; Procedure
Lead sponsor: Masonic Cancer Center, University of Minnesota; Other
Eligibility: 0 Years to 25 Years
Enrollment: 5 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2022
U.S. locations: 1
States / cities: Minneapolis, Minnesota

Conditions: X-Linked Adrenoleukodystrophy
Interventions: Not listed
Lead sponsor: NeuroVia, Inc.; Industry
Eligibility: 2 Years to 13 Years · Male only
Enrollment: 100 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2021
U.S. locations: 3
States / cities: Palo Alto, California • Boston, Massachusetts • Pittsburgh, Pennsylvania

Conditions: X-linked Adrenoleukodystrophy
Interventions: vitamin D3; Dietary Supplement
Lead sponsor: Stanford University; Other
Eligibility: 18 Months to 25 Years · Male only
Enrollment: 21 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2020
U.S. locations: 1
States / cities: Palo Alto, California

Conditions: ALD (Adrenoleukodystrophy), Adrenoleukodystrophy, Cerebral Adrenoleukodystrophy
Interventions: Medical Record Abstraction, Biospecimen Sample Collection; Other
Lead sponsor: Masonic Cancer Center, University of Minnesota; Other
Eligibility: Not listed
Enrollment: 1,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2030
U.S. locations: 1
States / cities: Minneapolis, Minnesota

Conditions: Cerebral Adrenoleukodystrophy (CALD)
Interventions: Lenti-D; Genetic
Lead sponsor: Genetix Biotherapeutics Inc.; Industry
Eligibility: Up to 17 Years · Male only
Enrollment: 35 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2023
U.S. locations: 3
States / cities: Palo Alto, California • Boston, Massachusetts • Minneapolis, Minnesota

Conditions: Cerebral Adrenoleukodystrophy (CALD)
Interventions: Lenti-D Drug Product (eli-cel); Genetic
Lead sponsor: Genetix Biotherapeutics Inc.; Industry
Eligibility: Up to 17 Years · Male only
Enrollment: 32 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2021
U.S. locations: 3
States / cities: Los Angeles, California • Boston, Massachusetts • 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: Hematologic Malignancies, Inborn Errors of Metabolism Disorders, Immune Deficiencies
Interventions: CliniMACS CD34 Reagent System; Biological
Lead sponsor: Joanne Kurtzberg, MD; Other
Eligibility: Up to 65 Years
U.S. locations: 1
States / cities: Durham, North Carolina

Conditions: Adrenoleukodystrophy, Restless Legs Syndrome
Interventions: Pramipexole, Placebo; Drug
Lead sponsor: Massachusetts General Hospital; Other
Eligibility: 18 Years to 75 Years · Female only
Enrollment: 24 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2026
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: X-Linked Adrenoleukodystrophy (X-ALD), Cerebral Adrenoleukodystrophy (CALD), Adrenoleukodystrophy (ALD)
Interventions: Allo-HSCT; Genetic
Lead sponsor: Genetix Biotherapeutics Inc.; Industry
Eligibility: Up to 17 Years · Male only
Enrollment: 59 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2019
U.S. locations: 6
States / cities: Los Angeles, California • Palo Alto, California • Boston, Massachusetts + 3 more

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, 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: 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: AMN, AMN Gene Mutation, X-ALD
Interventions: SBT101 Treatment; Genetic
Lead sponsor: SwanBio Therapeutics, Inc.; Industry
Eligibility: 18 Years to 65 Years · Male only
Enrollment: 9 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2025
U.S. locations: 1
States / cities: Worcester, Massachusetts

Conditions: Inherited Metabolic Disorders (IMD)
Interventions: MGTA-456; Drug
Lead sponsor: Magenta Therapeutics, Inc.; Industry
Eligibility: 0 Years to 17 Years
Enrollment: 8 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2021
U.S. locations: 2
States / cities: Minneapolis, Minnesota • Cincinnati, Ohio

Conditions: X-Linked Adrenoleukodystrophy, Adrenomyeloneuropathy
Interventions: Sobetirome; Drug
Lead sponsor: Thomas S. Scanlan; Other
Eligibility: 18 Years to 64 Years · Male only
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2014
U.S. locations: 1
States / cities: Portland, Oregon

Conditions: Adrenoleukodystrophy
Interventions: glyceryl trierucate/glyceryl trioleate; Drug
Lead sponsor: Hugo W. Moser Research Institute at Kennedy Krieger, Inc.; Other
Eligibility: 18 Months to 8 Years · Male only
Enrollment: 126 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 1998 – 2014
U.S. locations: 2
States / cities: Baltimore, Maryland

Conditions: AMN, AMN Gene Mutation, X-ALD
Interventions: Natural History Observation; Other
Lead sponsor: SwanBio Therapeutics, Inc.; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 65 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2025
U.S. locations: 4
States / cities: Stanford, California • Boston, Massachusetts • New York, New York + 1 more

Conditions: Adrenoleukodystrophy
Interventions: Not listed
Lead sponsor: Masonic Cancer Center, University of Minnesota; Other
Eligibility: 1 Year to 5 Years · Male only
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2023
U.S. locations: 1
States / cities: Minneapolis, Minnesota

Conditions: X-linked Adrenoleukodystrophy
Interventions: exercise training; Behavioral
Lead sponsor: Hugo W. Moser Research Institute at Kennedy Krieger, Inc.; Other
Eligibility: 21 Years to 70 Years · Female only
Enrollment: 31 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2012 – 2014
U.S. locations: 1
States / cities: Baltimore, Maryland

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Long-term Follow-up of Participants With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product

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

UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells

Home Exercise for Individuals with Neurodegenerative Disease

Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation (HaploHCT) Following Reduced Intensity Conditioning (RIC) for Selected High Risk Non-Malignant Diseases

A Study to Prospectively Assess Disease Progression in Male Children With X-ALD

A Pilot Study of Vitamin D in Boys With X-linked Adrenoleukodystrophy

Adrenoleukodystrophy National Registry Study

A Clinical Study to Assess the Efficacy and Safety of Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy (CALD)

A Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD)

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

Expanded Access to T-cell Depleted Haplo-Identical Stem Cells for Patients Receiving Haplo-Identical and Unrelated Cord Blood Transplants

Treating Leg Symptoms in Women With X-linked Adrenoleukodystrophy

Observational Study to Evaluate Allogeneic HSCT Outcomes for Cerebral Adrenoleukodystrophy (CALD)

A Follow-up Study in Patients With Inherited Metabolic Disorders (IMD) Who Underwent Hematopoietic Stem Cell Transplantation (HSCT) With MGTA-456

Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders

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

A Study to Evaluate Administration of SBT101 Gene Therapy in Adult Patients With Adrenomyeloneuropathy (AMN)

MGTA-456 in Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplantation (HSCT)

Safety and Pharmacodynamic Study of Sobetirome in X-Linked Adrenoleukodystrophy (X-ALD)

Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy

Study of Disease Progression in Adults With Inherited Forms of Spastic Paraplegia

Early Diagnosis Of Childhood Cerebral ALD

Exercise Study of Function and Pathology for Women With X-linked Adrenoleukodystrophy