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Showing 1–24 of 30 matching trials from the live ClinicalTrials.gov search.
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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 7, 2014 · Synced Jul 21, 2026, 4:41 PM EDT
Completed Phase 3 Interventional

Study to Evaluate Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Disease From Nuclear DNA Mutations (nPMD)

NCT05162768
View directory record Official record
Conditions
Mitochondrial Myopathies, Mitochondrial Pathology, Mitochondrial DNA Mutation, Mitochondrial Diseases, Mitochondrial DNA Deletion, Mitochondrial DNA Depletion, Mitochondrial Metabolism Defect, Mitochondrial Complex I Deficiency
Interventions
Elamipretide, Placebo
Drug
Lead sponsor
Stealth BioTherapeutics Inc.
Industry
Eligibility
18 Years to 70 Years
Enrollment
102 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
9
States / cities
San Diego, California • Atlanta, Georgia • Boston, Massachusetts + 6 more
Source: ClinicalTrials.gov public record
Updated Oct 23, 2025 · Synced Jul 21, 2026, 4:41 PM EDT
Completed Phase 1 Interventional

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

NCT00692926
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jun 28, 2017 · Synced Jul 21, 2026, 4:41 PM EDT
Completed Phase 3 Interventional

A United States Extension Study of Corlux for Recurrent Psychotic Symptoms in Psychotic Major Depression

NCT00208156
View directory record Official record
Conditions
Depressive Disorder, Major
Interventions
Mifepristone
Drug
Lead sponsor
Corcept Therapeutics
Industry
Eligibility
18 Years to 75 Years
Enrollment
87 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2006
U.S. locations
13
States / cities
San Diego, California • Atlanta, Georgia • Las Vegas, Nevada + 10 more
Source: ClinicalTrials.gov public record
Updated Feb 14, 2012 · Synced Jul 21, 2026, 4:41 PM EDT
Terminated Phase 1 Interventional

(PM-01) IMPRIME PGG® With BTH1704 and Gemcitabine for Advanced Pancreatic Cancer

NCT02132403
View directory record Official record
Conditions
Pancreatic Cancer
Interventions
BTH1704, IMPRIME PGG, Gemcitabine
Drug
Lead sponsor
University of Illinois at Chicago
Other
Eligibility
18 Years and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Apr 30, 2015 · Synced Jul 21, 2026, 4:41 PM EDT
Completed Phase 1 Interventional

Evaluate the Safety and Feasibility of Injecting PMD-MSC Into the Penis to Treat the Symptoms of Mild to Moderate ED

NCT02398370
View directory record Official record
Conditions
Erectile Dysfunction
Interventions
Placental Matrix-Derived Mesenchymal Stem Cells (PMD-MSCs)
Biological
Lead sponsor
Melissa Marchand
Other
Eligibility
40 Years to 70 Years · Male only
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
1
States / cities
Coral Springs, Florida
Source: ClinicalTrials.gov public record
Updated Apr 26, 2015 · Synced Jul 21, 2026, 4:41 PM EDT
Completed No phase listed Observational Results available

LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies

NCT02699190
View directory record Official record
Conditions
Leukodystrophy, White Matter Disease, 4H Syndrome, Adrenoleukodystrophy, AMN, ALD, ALD (Adrenoleukodystrophy), X-linked Adrenoleukodystrophy, X-ALD, Adrenomyeloneuropathy, Aicardi Goutieres Syndrome, AGS, Alexander Disease, Alexanders Leukodystrophy, AxD, ADLD, Canavan Disease, CTX, Cerebrotendinous Xanthomatoses, Krabbe Disease, GALC Deficiency, Globoid Leukodystrophy, TUBB4A-Related Leukodystrophy, H-ABC - Hypomyelination, Atrophy of Basal Ganglia and Cerebellum, HBSL, HBSL - Hypomyelination, Brain Stem, Spinal Cord, Leg Spasticity, LBSL, Leukoencephalopathy With Brain Stem and Spinal Cord Involvement and High Lactate Syndrome (Disorder), Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation, ALSP, CSF1R Gene Mutation, HCC - Hypomyelination and Congenital Cataract, MLC1, Megalencephalic Leukoencephalopathy With Subcortical Cysts 1, MLD, Metachromatic Leukodystrophy, PMD, Pelizaeus-Merzbacher Disease, PLP1 Null Syndrome, PLP1 Gene Duplication | Blood or Tissue | Mutations, Pelizaeus-Merzbacher-Like Disease, 1, Peroxisomal Biogenesis Disorder, Zellweger Syndrome, Refsum Disease, Salla Disease, Sialic Storage Disease, Sjögren, Sjogren-Larsson Syndrome, Van Der Knapp Disease, Vanishing White Matter Disease, Charcot-Marie-Tooth, CMT, Mct8 (Slc16A2)-Specific Thyroid Hormone Cell Transporter Deficiency, Allan-Herndon-Dudley Syndrome, Cadasil, Cockayne Syndrome, Multiple Sulfatase Deficiency, Gangliosidoses, GM2 Gangliosidosis, BPAN, Labrune Syndrome, LCC, Mucopolysaccharidoses, TBCK-Related Intellectual Disability Syndrome
Interventions
Not listed
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
Up to 18 Years
Enrollment
236 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2024
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Nov 9, 2025 · Synced Jul 21, 2026, 4:41 PM EDT
Available No phase listed Expanded access

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

NCT01881334
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Nov 19, 2025 · Synced Jul 21, 2026, 4:41 PM EDT
Terminated Not applicable Interventional

Embodied Virtual Reality Therapy for Functional Neurological Symptom/ Conversion Disorder

NCT02764476
View directory record Official record
Conditions
Conversion Disorder, Psychogenic Movement Disorder, Functional Movement Disorder, Functional Neurological Disorder, Non-epileptic Seizures
Interventions
Embodied Virtual Reality Therapy, Virtual reality
Other
Lead sponsor
Stanford University
Other
Eligibility
18 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2021
U.S. locations
1
States / cities
Stanford, California
Source: ClinicalTrials.gov public record
Updated Aug 29, 2023 · Synced Jul 21, 2026, 4:41 PM EDT
Completed Phase 1 Interventional

Evaluate the Safety and Feasibility of Injecting PMD-MSC Into the Penis to Treat the Symptoms of PD

NCT02395029
View directory record Official record
Conditions
Peyronie's Disease
Interventions
Placental Matrix-Derived Mesenchymal Stem Cells (PMD-MSCs)
Biological
Lead sponsor
Melissa Marchand
Other
Eligibility
18 Years and older · Male only
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
1
States / cities
Coral Springs, Florida
Source: ClinicalTrials.gov public record
Updated Mar 19, 2015 · Synced Jul 21, 2026, 4:41 PM EDT
Completed Phase 1Phase 2 Interventional

Promoting Resilience in Stress Management (PRISM) and Clinical-focused Narrative (CFN) Pilot in Adults With Primary Mitochondrial Disease (PMD).

NCT06051448
View directory record Official record
Conditions
Mitochondrial Diseases, Mitochondrial Myopathies, Mitochondrial Encephalomyopathies, MELAS, MERRF
Interventions
Promoting Resilience in Stress Management (PRISM), Clinical-focused narrative (CFN)
Behavioral
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
18 Years and older
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Nov 28, 2024 · Synced Jul 21, 2026, 4:41 PM EDT
Completed Phase 1 Interventional

Study of Human Central Nervous System (CNS) Stem Cells Transplantation in Pelizaeus-Merzbacher Disease (PMD) Subjects

NCT01005004
View directory record Official record
Conditions
Pelizaeus-Merzbacher Disease
Interventions
HuCNS-SC cells
Biological
Lead sponsor
StemCells, Inc.
Industry
Eligibility
6 Months to 5 Years · Male only
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2012
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Jan 14, 2015 · Synced Jul 21, 2026, 4:41 PM EDT
Recruiting No phase listed Observational

Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher Disease

NCT05659901
View directory record Official record
Conditions
Pelizaeus-Merzbacher Disease
Interventions
Not listed
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
6 Months to 17 Years · Male only
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2029
U.S. locations
2
States / cities
Atlanta, Georgia • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 16, 2026 · Synced Jul 21, 2026, 4:41 PM EDT
Completed Phase 2 Interventional Results available

Phase II Study of MEDI4736 Monotherapy or in Combinations With Tremelimumab in Metastatic Pancreatic Ductal Carcinoma

NCT02558894
View directory record Official record
Conditions
Metastatic Pancreatic Ductal Adenocarcinoma
Interventions
MEDI4736 monotherapy, tremelimumab+MEDI4736
Drug
Lead sponsor
AstraZeneca
Industry
Eligibility
18 Years and older
Enrollment
65 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
2
States / cities
Tampa, Florida • New York, New York
Source: ClinicalTrials.gov public record
Updated Aug 1, 2018 · Synced Jul 21, 2026, 4:41 PM EDT
Completed Not applicable Interventional

The Impact of Patient Motivational Dialogue (PMD) on Patient Self-advocacy for Hand Hygiene

NCT04360057
View directory record Official record
Conditions
Hand Disinfection
Interventions
PMD, PMDPlus
Other
Lead sponsor
The University of Texas Health Science Center at San Antonio
Other
Eligibility
3 Years to 85 Years
Enrollment
63 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
1
States / cities
San Antonio, Texas
Source: ClinicalTrials.gov public record
Updated Apr 23, 2020 · Synced Jul 21, 2026, 4:41 PM EDT
Recruiting Phase 1 Interventional

Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)

NCT06150716
View directory record Official record
Conditions
Pelizaeus-Merzbacher Disease
Interventions
ION356
Drug
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
2 Years to 17 Years · Male only
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
3
States / cities
Atlanta, Georgia • Philadelphia, Pennsylvania • Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Dec 11, 2025 · Synced Jul 21, 2026, 4:41 PM EDT
Completed Phase 3 Interventional

A United States Study of the Safety and Tolerability of Corlux for Psychotic Symptoms in Psychotic Major Depression

NCT00128479
View directory record Official record
Conditions
Major Depressive Disorder, Psychotic Disorders
Interventions
mifepristone, mifepristone matched placebo, mifepristone 600 mg, mifepristone 1200 mg
Drug
Lead sponsor
Corcept Therapeutics
Industry
Eligibility
18 Years to 75 Years
Enrollment
443 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2007
U.S. locations
44
States / cities
Little Rock, Arkansas • Cerritos, California • Fountain Valley, California + 40 more
Source: ClinicalTrials.gov public record
Updated Feb 14, 2012 · Synced Jul 21, 2026, 4:41 PM 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 21, 2026, 4:41 PM EDT
Recruiting Phase 2 Interventional

A Phase 2 Study of Radiotherapy-induced Immune Priming to Enhance Elranatamab (Elra) in Relapsed Refractory Multiple Myeloma (RRMM) With Extramedullary Disease (EMD) and Paramedullary Disease (PMD) "PRIME-EMD-PMD"

NCT07382739
View directory record Official record
Conditions
Phase 2, Radiotherapy-Induced Immune Priming, Elranatamab, Relapsed Refractory Multiple Myeloma (RRMM), Extramedullary Disease in Multiple Myeloma, Paramedullary Disease, PMD
Interventions
Elranatamab
Drug
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
Not listed
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Apr 13, 2026 · Synced Jul 21, 2026, 4:41 PM EDT
Completed Phase 3 Interventional

A United States Study of Corlux for Psychotic Symptoms in Psychotic Major Depression

NCT00130676
View directory record Official record
Conditions
Major Depressive Disorder, Psychotic Disorders
Interventions
Mifepristone, matching placebo
Drug
Lead sponsor
Corcept Therapeutics
Industry
Eligibility
18 Years to 75 Years
Enrollment
257 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2006
U.S. locations
23
States / cities
Pico Rivera, California • Riverside, California • San Diego, California + 20 more
Source: ClinicalTrials.gov public record
Updated Feb 14, 2012 · Synced Jul 21, 2026, 4:41 PM EDT
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 21, 2026, 4:41 PM EDT
Completed No phase listed Observational

Long-Term Follow-Up Study of Human Stem Cells Transplanted in Subjects With Connatal Pelizaeus-Merzbacher Disease (PMD)

NCT01391637
View directory record Official record
Conditions
Pelizaeus-Merzbacher Disease, PMD
Interventions
HuCNS-SC transplant in the lead-in phase
Biological
Lead sponsor
StemCells, Inc.
Industry
Eligibility
Male only
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated May 12, 2016 · Synced Jul 21, 2026, 4:41 PM EDT
Recruiting Phase 1 Interventional

Study to Assess Adverse Events and Pharmacokinetics in Adult Participants With Non-Small Cell Lung Cancer, Head and Neck Squamous Cell Carcinoma and Other Solid Tumors, Receiving Intravenous Infusion of Azirkitug Alone or in Combination(s) With Budigalimab, Bevacizumab, or Telisotuzumab Adizutecan

NCT05005403
View directory record Official record
Conditions
Non-Small Cell Lung Cancer, Head and Neck Squamous Cell Carcinoma, Micro Satellite Stable Colorectal Cancer, Gastric/Esophageal Cancer, High-Grade Serous Ovarian Cancer, Pancreatic Cancer, Triple Negative Breast Cancer
Interventions
Azirkitug, Budigalimab, Bevacizumab, Telisotuzumab Adizutecan
Drug
Lead sponsor
AbbVie
Industry
Eligibility
18 Years and older
Enrollment
694 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
20
States / cities
Duarte, California • Irvine, California • Los Angeles, California + 15 more
Source: ClinicalTrials.gov public record
Updated Jun 22, 2026 · Synced Jul 21, 2026, 4:41 PM EDT
Terminated Phase 3 Interventional Results available

A Trial to Evaluate Safety and Efficacy of Elamipretide Primary Mitochondrial Myopathy Followed by Open-Label Extension

NCT03323749
View directory record Official record
Conditions
Primary Mitochondrial Myopathy
Interventions
elamipretide, placebo comparator, elamipretide open label treatment
Combination Product
Lead sponsor
Stealth BioTherapeutics Inc.
Industry
Eligibility
16 Years to 80 Years
Enrollment
218 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2020
U.S. locations
13
States / cities
La Jolla, California • Palo Alto, California • Aurora, Colorado + 9 more
Source: ClinicalTrials.gov public record
Updated Jan 23, 2022 · Synced Jul 21, 2026, 4:41 PM EDT