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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

Conditions: Advanced Cancers (Breast, Ovarian, mCRPC, Pancreatic Ductal Adenocarcinoma (PDAC), Brain Mets) With Specific Mutations (Homologous Recombination Deficiency)
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: 4
States / cities: Sacramento, California • New Haven, Connecticut • New York, New York + 1 more

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

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

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

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

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

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

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

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

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 &#X7C; Blood or Tissue &#X7C; 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

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

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, 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: 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

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

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

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

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 &#X7C; Blood or Tissue &#X7C; 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

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

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

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

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

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

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

Study to Assess Safety, Tolerability and Activity of DSB2455 in Participants With Advanced Malignancies

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

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

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

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

Psychiatric Correlates of Psychogenic Movement Disorder and Non-Epileptic Seizure

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

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"

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

The Myelin Disorders Biorepository Project

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

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

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

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

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

Fetal Umbilical Cord Blood (UCB) Transplant for Lysosomal Storage Diseases

Embodied Virtual Reality Therapy for Functional Neurological Symptom/ Conversion Disorder

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

Longitudinal Study of Neurodegenerative Disorders

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

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

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

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