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ClinicalTrials.gov public records Last synced Sep 30, 2026, 4:54 PM EDT

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Showing 25–43 of 43 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Cerebral Adrenoleukodystrophy Clear all
Completed Phase 2 Interventional Results available

HSCT for High Risk Inherited Inborn Errors

NCT00383448
View directory record Official record
Conditions
Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy, Tay Sachs Disease, Sandhoffs Disease, Wolman Disease, I-Cell Disease, Sanfilippo Syndrome, GM1 Gangliosidosis
Interventions
Clofarabine, Total body Irradiation, Melphalan, Hematopoietic Stem Cell Transplantation, Alemtuzumab, mycophenylate mofetil, Cyclosporine A, Hydroxyurea
Drug · Procedure · Biological + 1 more
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 70 Years
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2014
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jul 11, 2019 · Synced Sep 30, 2026, 4:54 PM EDT
Completed Phase 2Phase 3 Interventional Results available

Stem Cell Transplant for Inborn Errors of Metabolism

NCT00176904
View directory record Official record
Conditions
Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy, Gaucher's Disease, Fucosidosis, Wolman Disease, Niemann-Pick Disease, Batten Disease, GM1 Gangliosidosis, Tay Sachs Disease, Sandhoff Disease
Interventions
Stem Cell Transplant, Busulfan, Cyclophosphamide, Antithymocyte Globulin
Procedure · Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Not listed
Enrollment
135 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1995 – 2010
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 28, 2017 · Synced Sep 30, 2026, 4:54 PM EDT
Terminated Phase 3 Interventional

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

NCT00654433
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jul 8, 2014 · Synced Sep 30, 2026, 4:54 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of OP-101 After Intravenous Administration in Healthy Volunteers

NCT03500627
View directory record Official record
Conditions
Healthy
Interventions
OP-101
Drug
Lead sponsor
Orpheris, Inc.
Industry
Eligibility
18 Years to 65 Years
Enrollment
8 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Sep 17, 2018 · Synced Sep 30, 2026, 4:54 PM EDT
Terminated Phase 3 Interventional

A Phase III Trial of Lorenzo's Oil in Adrenomyeloneuropathy

NCT00545597
View directory record Official record
Conditions
Adrenomyeloneuropathy, Adrenoleukodystrophy
Interventions
Lorenzo's oil
Drug
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
18 Years and older
Enrollment
240 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2007
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 2, 2012 · Synced Sep 30, 2026, 4:54 PM EDT
Completed Phase 3 Interventional Results available

Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid

NCT00007020
View directory record Official record
Conditions
Infantile Refsum's Disease, Zellweger Syndrome, Adrenoleukodystrophy, Peroxisomal Disorders, Cholestasis
Interventions
Cholic Acids
Drug
Lead sponsor
Mirum Pharmaceuticals, Inc.
Industry
Eligibility
Not listed
Enrollment
85 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1992 – 2009
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Oct 3, 2023 · Synced Sep 30, 2026, 4:54 PM EDT
Terminated No phase listed Observational

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

NCT05008874
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Oct 30, 2025 · Synced Sep 30, 2026, 4:54 PM EDT
Completed Phase 1 Interventional

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

NCT02595489
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jun 9, 2022 · Synced Sep 30, 2026, 4:54 PM EDT
Not listed No phase listed Observational

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

NCT03278899
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Aug 8, 2018 · Synced Sep 30, 2026, 4:54 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

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

NCT05394064
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Aug 26, 2026 · Synced Sep 30, 2026, 4:54 PM EDT
Recruiting No phase listed Observational

Adrenoleukodystrophy National Registry Study

NCT03789721
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated May 12, 2026 · Synced Sep 30, 2026, 4:54 PM EDT
Completed Phase 1 Interventional

Human Placental-Derived Stem Cell Transplantation

NCT01586455
View directory record Official record
Conditions
Mucopolysaccharidosis I, Mucopolysaccharidosis VI, Adrenoleukodystrophy, Niemann-Pick Disease, Metachromatic Leukodystrophy, Wolman Disease, Krabbe's Disease, Gaucher's Disease, Fucosidosis, Batten Disease, Severe Aplastic Anemia, Diamond-Blackfan Anemia, Amegakaryocytic Thrombocytopenia, Myelodysplastic Syndrome, Acute Myelogenous Leukemia, Acute Lymphocytic Leukemia
Interventions
Human Placental Derived Stem Cell
Drug
Lead sponsor
New York Medical College
Other
Eligibility
0 Years to 55 Years
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2022
U.S. locations
3
States / cities
Denver, Colorado • Valhalla, New York • Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Oct 25, 2022 · Synced Sep 30, 2026, 4:54 PM EDT
Recruiting No phase listed Observational

Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC

NCT04528355
View directory record Official record
Conditions
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions
Interventions
data collection
Drug
Lead sponsor
Paul Szabolcs
Other
Eligibility
2 Months to 60 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2028
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 13, 2026 · Synced Sep 30, 2026, 4:54 PM EDT
Not listed No phase listed Observational

Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation

NCT00005900
View directory record Official record
Conditions
I Cell Disease, Fucosidosis, Globoid Cell Leukodystrophy, Adrenoleukodystrophy, Mannosidosis, Niemann-Pick Disease, Pulmonary Complications, Mucopolysaccharidosis I, Mucopolysaccharidosis VI, Metachromatic Leukodystrophy, Gaucher's Disease, Wolman Disease
Interventions
Not listed
Lead sponsor
Fairview University Medical Center
Other
Eligibility
0 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1999
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Sep 30, 2026, 4:54 PM EDT
Active, not recruiting Phase 3 Interventional

A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adult Male Subjects With Cerebral Adrenoleukodystrophy

NCT05819866
View directory record Official record
Conditions
Cerebral Adrenoleukodystrophy (cALD)
Interventions
Leriglitazone, Placebo
Drug
Lead sponsor
Minoryx Therapeutics, S.L.
Industry
Eligibility
18 Years and older · Male only
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
4
States / cities
Palo Alto, California • Baltimore, Maryland • Boston, Massachusetts + 1 more
Source: ClinicalTrials.gov public record
Updated Jul 29, 2026 · Synced Sep 30, 2026, 4:54 PM EDT
Withdrawn No phase listed Observational

Early Diagnosis Of Childhood Cerebral ALD

NCT02948062
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jul 6, 2018 · Synced Sep 30, 2026, 4:54 PM EDT
Suspended Phase 1 Interventional

MT2022-01: MSCs for ALD

NCT06030648
View directory record Official record
Conditions
Cerebral Adrenoleukodystrophy
Interventions
Mesenchymal stem cells (IV-MSC)
Biological
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
3 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2029
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jun 18, 2026 · Synced Sep 30, 2026, 4:54 PM EDT
Completed Phase 2 Interventional Results available

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

NCT03406962
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Nov 3, 2021 · Synced Sep 30, 2026, 4:54 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

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

NCT01594853
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Aug 29, 2018 · Synced Sep 30, 2026, 4:54 PM EDT