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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Recruiting Phase 1 Interventional

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

NCT02254863
View directory record Official record
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 – 2026
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Sep 7, 2025 · Synced Jul 22, 2026, 11:55 AM EDT
Recruiting Phase 2 Interventional

LGG Supplementation in Patients With AUD and ALD

NCT05178069
View directory record Official record
Conditions
Alcohol Use Disorder, Alcohol-associated Liver Disease
Interventions
: Placebo for Probiotic, Lactobacillus Rhamnosus GG
Drug · Dietary Supplement
Lead sponsor
University of Louisville
Other
Eligibility
21 Years to 65 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
1
States / cities
Louisville, Kentucky
Source: ClinicalTrials.gov public record
Updated Oct 30, 2025 · Synced Jul 22, 2026, 11:55 AM EDT
Not yet recruiting Not applicable Interventional

Peer Support for Liver Transplant Recipients With History of ALD

NCT07397598
View directory record Official record
Conditions
Alcohol Use Disorder, Alcohol Related Liver Disease, Liver Transplant Recipient
Interventions
Peer Support
Behavioral
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years and older
Enrollment
95 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 29, 2026 · Synced Jul 22, 2026, 11:55 AM 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 2, 2021 · Synced Jul 22, 2026, 11:55 AM EDT
Completed Phase 3 Interventional Results available

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

NCT03852498
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated May 23, 2024 · Synced Jul 22, 2026, 11:55 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation

NCT03513328
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Sep 14, 2023 · Synced Jul 22, 2026, 11:55 AM EDT
Completed Phase 1 Interventional Results available

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

NCT00744692
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Aug 12, 2014 · Synced Jul 22, 2026, 11:55 AM EDT
Active, not recruiting No phase listed Observational

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

NCT02698579
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Feb 19, 2025 · Synced Jul 22, 2026, 11:55 AM EDT
Recruiting Phase 2 Interventional

RESTORE TRIAL: A Phase 2 Study Evaluating the Efficacy and Safety of Pemvidutide in the Treatment of Alcohol-Associated Liver Disease (ALD)

NCT07009860
View directory record Official record
Conditions
Alcohol Liver Disease
Interventions
Pemvidutide, Placebo
Drug · Other
Lead sponsor
Altimmune, Inc.
Industry
Eligibility
18 Years to 75 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
41
States / cities
Peoria, Arizona • Tucson, Arizona • Tuscon, Arizona + 35 more
Source: ClinicalTrials.gov public record
Updated Mar 19, 2026 · Synced Jul 22, 2026, 11:55 AM 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 23, 2005 · Synced Jul 22, 2026, 11:55 AM EDT
Completed Phase 2Phase 3 Interventional Results available

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

NCT01896102
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Apr 24, 2022 · Synced Jul 22, 2026, 11:55 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

BREATHE ALD: A Shared Decision-Making Intervention for Adults With Advanced Lung Disease

NCT04930666
View directory record Official record
Conditions
Pulmonary Disease, Chronic Obstructive, Lung Diseases, Interstitial
Interventions
BRief intervention to Enhance Adherence to Treatment and HEalth advice in Advanced Lung Disease
Other
Lead sponsor
Columbia University
Other
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2025
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Apr 14, 2025 · Synced Jul 22, 2026, 11:55 AM 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 1, 2012 · Synced Jul 22, 2026, 11:55 AM 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 16, 2018 · Synced Jul 22, 2026, 11:55 AM EDT
Terminated Phase 1Phase 2 Interventional

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

NCT01372228
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Apr 11, 2023 · Synced Jul 22, 2026, 11:55 AM EDT
Recruiting Phase 3 Interventional

Study to Evaluate the Safety and Efficacy of Larsucosterol in Participants With Alcohol-associated Hepatitis (AH)

NCT07428538
View directory record Official record
Conditions
Hepatitis
Interventions
Larsucosterol, Placebo
Drug
Lead sponsor
Bausch Health Americas, Inc.
Industry
Eligibility
18 Years and older
Enrollment
350 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
84
States / cities
Birmingham, Alabama • Mobile, Alabama • Phoenix, Arizona + 61 more
Source: ClinicalTrials.gov public record
Updated Jul 21, 2026 · Synced Jul 22, 2026, 11:55 AM EDT
Completed Phase 1 Interventional

A Study to Assess the Pharmacodynamics of VK0214 in Male Subjects With AMN

NCT04973657
View directory record Official record
Conditions
Adrenomyeloneuropathy Form (AMN) of X-linked Adrenoleukodystrophy (X-ALD)
Interventions
VK0214, Placebo
Drug · Other
Lead sponsor
Viking Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
4
States / cities
Palo Alto, California • Gainesville, Florida • Baltimore, Maryland + 1 more
Source: ClinicalTrials.gov public record
Updated Feb 16, 2025 · Synced Jul 22, 2026, 11:55 AM EDT
Terminated Phase 2 Interventional

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

NCT03367546
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jul 11, 2024 · Synced Jul 22, 2026, 11:55 AM 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 2, 2023 · Synced Jul 22, 2026, 11:55 AM EDT
Terminated Phase 2 Interventional Results available

Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy

NCT00004418
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Oct 22, 2020 · Synced Jul 22, 2026, 11:55 AM 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 5, 2018 · Synced Jul 22, 2026, 11:55 AM EDT
Active, not recruiting Not applicable Interventional Accepts healthy volunteers

CM for Patients With ALD After Liver Transplant

NCT06304467
View directory record Official record
Conditions
Alcohol Use Disorder, Alcohol; Liver, Post Liver Transplant, Contingency Management, Alcohol Liver Disease
Interventions
Contingency Management
Behavioral
Lead sponsor
Arpan A. Patel, MD
Other
Eligibility
18 Years to 99 Years
Enrollment
30 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024 – 2026
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Apr 13, 2026 · Synced Jul 22, 2026, 11:55 AM 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 7, 2018 · Synced Jul 22, 2026, 11:55 AM 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 27, 2017 · Synced Jul 22, 2026, 11:55 AM EDT