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ClinicalTrials.gov public records Last synced May 21, 2026, 7:59 PM EDT

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Showing 1–24 of 30 matching trials from the live ClinicalTrials.gov search.
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Terminated Phase 1 Interventional
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 May 21, 2026, 7:59 PM EDT
Completed No phase listed Observational
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 May 21, 2026, 7:59 PM EDT
Available No phase listed Expanded access
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 May 21, 2026, 7:59 PM EDT
Completed Phase 2 Interventional Results available
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 May 21, 2026, 7:59 PM EDT
Recruiting Phase 2 Interventional
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 May 21, 2026, 7:59 PM EDT
Completed Phase 3 Interventional
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 May 21, 2026, 7:59 PM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
MLD, Krabbe Disease, ALD, MPS I, MPS II, MPS III, Vanishing White Matter Disease, GM3 Gangliosidosis, PKAN, Tay-Sachs Disease, NP Deficiency, Osteopetrosis, Alpha-Mannosidosis, Sandhoff Disease, Niemann-Pick Diseases, MPS IV, Gaucher Disease, GAN, GM1 Gangliosidoses, Morquio Disease, S-Adenosylhomocysteine Hydrolase Deficiency, Batten Disease, Pelizaeus-Merzbacher Disease, Leukodystrophy, Lysosomal Storage Diseases, Purine Nucleoside Phosphorylase Deficiency, Multiple Sulfatase Deficiency Disease
Interventions
Palliative Care, Hematopoetic Stem Cell Transplantation
Other · Biological
Lead sponsor
University of Pittsburgh
Other
Eligibility
Not listed
Enrollment
1,500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2035
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Feb 8, 2026 · Synced May 21, 2026, 7:59 PM EDT
Completed Phase 1Phase 2 Interventional
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 May 21, 2026, 7:59 PM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Leukodystrophy, White Matter Disease, Leukoencephalopathies, 4H Syndrome, Adrenoleukodystrophy, AMN, ALD, ALD Gene Mutation, 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, MLD, Metachromatic Leukodystrophy, PMD, Pelizaeus-Merzbacher Disease, PLP1 Null Syndrome, PLP1 Gene Duplication | Blood or Tissue | Mutations, Pelizaeus Merzbacher Like Disease, 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
Not listed
Enrollment
12,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2030
U.S. locations
23
States / cities
Los Angeles, California • Orange, California • Palo Alto, California + 18 more
Source: ClinicalTrials.gov public record
Updated Oct 22, 2025 · Synced May 21, 2026, 7:59 PM EDT
Terminated Not applicable Interventional
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 May 21, 2026, 7:59 PM EDT
Completed Phase 1 Interventional
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 May 21, 2026, 7:59 PM EDT
Recruiting Phase 1 Interventional
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 May 21, 2026, 7:59 PM EDT
Completed Phase 1 Interventional
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 May 21, 2026, 7:59 PM EDT
Withdrawn Phase 1 Interventional
View directory record Official record
Conditions
Lysosomal Storage Diseases, Inborn Errors of Metabolism
Interventions
ALD-601
Biological
Lead sponsor
Joanne Kurtzberg, MD
Other
Eligibility
18 Years and older · Female only
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2017
U.S. locations
2
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jun 28, 2017 · Synced May 21, 2026, 7:59 PM EDT
Recruiting Phase 1 Interventional
View directory record Official record
Conditions
Advanced Malignancies With Homologous Recombination Deficiency (HRD) (Breast, Ovarian, mCRPC, Pancreatic Ductal Adenocarcinoma (PDAC), Brain Metastases)
Interventions
DSB2455
Drug
Lead sponsor
Duke Street Bio Ltd
Industry
Eligibility
18 Years and older
Enrollment
180 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
2
States / cities
New Haven, Connecticut • Houston, Texas
Source: ClinicalTrials.gov public record
Updated May 13, 2026 · Synced May 21, 2026, 7:59 PM EDT
Completed No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Psychogenic Movement Disorders, Non-epileptic Seizures
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
18 Years and older
Enrollment
190 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2005 – 2009
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 1, 2017 · Synced May 21, 2026, 7:59 PM EDT
Completed Phase 3 Interventional
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 May 21, 2026, 7:59 PM EDT
Terminated Phase 3 Interventional
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 May 21, 2026, 7:59 PM EDT
Completed Phase 1Phase 2 Interventional Results available
View directory record Official record
Conditions
Metastatic Pancreatic Ductal Adenocarcinoma
Interventions
MEDI4736 in combination with nab-paclitaxel and gemcitabine, MEDI4736 in combination with AZD5069
Drug
Lead sponsor
AstraZeneca
Industry
Eligibility
18 Years to 130 Years
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated Aug 13, 2019 · Synced May 21, 2026, 7:59 PM EDT
Completed Phase 3 Interventional
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 May 21, 2026, 7:59 PM EDT
Completed Phase 1 Interventional
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 May 21, 2026, 7:59 PM EDT
Recruiting No phase listed Observational
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 May 21, 2026, 7:59 PM EDT
Recruiting Phase 1 Interventional
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
18
States / cities
Duarte, California • Irvine, California • Chicago, Illinois + 14 more
Source: ClinicalTrials.gov public record
Updated May 12, 2026 · Synced May 21, 2026, 7:59 PM EDT
Completed No phase listed Observational Results available
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 May 21, 2026, 7:59 PM EDT