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Showing 25–46 of 46 matching trials from the live ClinicalTrials.gov search.
Conditions
Erythropoietic Protoporphyria
Interventions
DISC-1459
Drug
Lead sponsor
Disc Medicine, Inc
Industry
Eligibility
12 Years and older
Enrollment
230 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
11
States / cities
Birmingham, Alabama • San Francisco, California • Miami, Florida + 7 more
Conditions
Age-Related Macular Degeneration, Allergies, Alpha-Gal Syndrome, Alzheimer Disease, Amyloidosis, Ankylosing Spondylitis, Arthritis, Alopecia Areata, Asthma, Atopic Dermatitis, Autism, Autoimmune Hepatitis, Behcet's Disease, Beta-Thalassemia, Cancer, Celiac Disease, Kidney Diseases, COPD, Crohn Disease, Cystic Fibrosis, Diabetes, Dravet Syndrome, DMD, Fibromyalgia, Graves Disease, Thyroid Diseases, Hepatitis, Hidradenitis Suppurativa, ITP, Leukemia, ALS, Lupus or SLE, Lymphoma, Multiple Sclerosis, Myasthenia Gravis, Heart Diseases, Parkinson Disease, Pemphigus Vulgaris, Cirrhosis, Psoriasis, Schizophrenia, Scleroderma, Sickle Cell Disease, Stroke, Ulcerative Colitis, Vasculitis, Vitiligo
Interventions
Specimen sample
Diagnostic Test
Lead sponsor
Sanguine Biosciences
Industry
Eligibility
18 Years to 85 Years
Enrollment
20,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2025
U.S. locations
1
States / cities
Waltham, Massachusetts
Conditions
Cancer, Colorectal Cancer, Solid Tumor, Locally Advanced Solid Tumor, Metastatic Cancer, WNT Pathway, HCC, Desmoid, Microsatellite Stable Colorectal Cancer, Metastatic Castration-resistant Prostate Cancer, Familial Adenomatous Polyposis (FAP), Endometrial Carcinoma, Prostate Cancer, Microsatellite Instability-High Colorectal Cancer, Adamantinomatous Craniopharyngioma
Interventions
FOG-001, mFOLFOX-6, Nivolumab, Trifluridine/tipiracil, Bevacizumab
Drug
Lead sponsor
Parabilis Medicines, Inc.
Industry
Eligibility
18 Years and older
Enrollment
619 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
31
States / cities
Phoenix, Arizona • Scottsdale, Arizona • Tucson, Arizona + 25 more
Conditions
Cancer
Interventions
entinostat
Drug
Lead sponsor
National Institutes of Health Clinical Center (CC)
NIH
Eligibility
18 Years and older
Enrollment
75 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2008
U.S. locations
3
States / cities
Bethesda, Maryland
Conditions
Adrenal Cortical Carcinoma, Alveolar Soft Part Sarcoma, Central Nervous System Neoplasm, Childhood Clear Cell Sarcoma of Soft Tissue, Clear Cell Sarcoma of Soft Tissue, Ewing Sarcoma, Hepatoblastoma, Hepatocellular Carcinoma, Osteosarcoma, Recurrent Adrenal Cortical Carcinoma, Recurrent Alveolar Soft Part Sarcoma, Recurrent Clear Cell Sarcoma of Soft Tissue, Recurrent Ewing Sarcoma, Recurrent Hepatoblastoma, Recurrent Hepatocellular Carcinoma, Recurrent Kidney Wilms Tumor, Recurrent Malignant Solid Neoplasm, Recurrent Osteosarcoma, Recurrent Primary Malignant Central Nervous System Neoplasm, Recurrent Renal Cell Carcinoma, Recurrent Rhabdomyosarcoma, Recurrent Soft Tissue Sarcoma, Recurrent Thyroid Gland Medullary Carcinoma, Refractory Adrenal Cortical Carcinoma, Refractory Alveolar Soft Part Sarcoma, Refractory Clear Cell Sarcoma of Soft Tissue, Refractory Ewing Sarcoma, Refractory Hepatoblastoma, Refractory Hepatocellular Carcinoma, Refractory Malignant Solid Neoplasm, Refractory Osteosarcoma, Refractory Primary Central Nervous System Neoplasm, Refractory Primary Malignant Central Nervous System Neoplasm, Refractory Renal Cell Carcinoma, Refractory Rhabdomyosarcoma, Refractory Soft Tissue Sarcoma, Refractory Thyroid Gland Medullary Carcinoma, Refractory Wilms Tumor, Renal Cell Carcinoma, Rhabdomyosarcoma, Soft Tissue Sarcoma, Solid Neoplasm, Thyroid Gland Medullary Carcinoma, Wilms Tumor
Interventions
Cabozantinib, Cabozantinib S-malate, Pharmacological Study
Drug · Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
2 Years to 30 Years
Enrollment
109 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2027
U.S. locations
142
States / cities
Birmingham, Alabama • Anchorage, Alaska • Little Rock, Arkansas + 104 more
Conditions
Childhood Hepatocellular Carcinoma, Papillary Thyroid Cancer, Previously Treated Childhood Rhabdomyosarcoma, Recurrent Childhood Liver Cancer, Recurrent Childhood Rhabdomyosarcoma, Recurrent Thyroid Cancer, Recurrent Wilms Tumor and Other Childhood Kidney Tumors
Interventions
sorafenib tosylate, pharmacological study, laboratory biomarker analysis
Drug · Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
2 Years to 30 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2014
U.S. locations
83
States / cities
Birmingham, Alabama • Little Rock, Arkansas • Downey, California + 70 more
Conditions
Erythropoietic Protoporphyria (EPP), X-Linked Protoporphyria (XLP)
Interventions
DISC-1459
Drug
Lead sponsor
Disc Medicine, Inc
Industry
Eligibility
12 Years and older
U.S. locations
7
States / cities
Miami, Florida • Boston, Massachusetts • Detroit, Michigan + 3 more
Conditions
Erythropoietic Protoporphyria (EPP), X-Linked Protoporphyria (XLP), EPP2
Interventions
PORT-77, Placebo
Drug
Lead sponsor
Portal Therapeutics, Inc.
Industry
Eligibility
12 Years and older
Enrollment
225 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
2
States / cities
Huntington Beach, California • Cincinnati, Ohio
Conditions
Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor, Extragonadal Germ Cell Tumor, Kidney Cancer, Liver Cancer, Neuroblastoma, Ovarian Cancer, Retinoblastoma, Sarcoma
Interventions
arsenic trioxide
Drug
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
Up to 40 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2009
U.S. locations
1
States / cities
New York, New York
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
Conditions
Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor, Chordoma, Kidney Cancer, Liver Cancer, Neuroblastoma, Ovarian Cancer, Retinoblastoma, Sarcoma
Interventions
amifostine trihydrate, busulfan, filgrastim, melphalan, thiotepa, peripheral blood stem cell transplantation (PBSC)
Drug · Procedure
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
1 Year to 45 Years
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2003
U.S. locations
1
States / cities
Minneapolis, Minnesota
Conditions
Alpha 1-Antitrypsin Deficiency
Interventions
ADVM-043
Genetic
Lead sponsor
Adverum Biotechnologies, Inc.
Industry
Eligibility
18 Years and older
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
2
States / cities
Gainesville, Florida • Charleston, South Carolina
Conditions
Colorectal Carcinoma, Endometrial Carcinoma, Melanoma, Neuroblastoma, Ovarian Carcinoma, Pancreatic Ductal Adenocarcinoma, Recurrent Desmoid Fibromatosis, Recurrent Ewing Sarcoma, Recurrent Hepatoblastoma, Recurrent Hepatocellular Carcinoma, Recurrent Malignant Solid Neoplasm, Recurrent Non-Hodgkin Lymphoma, Recurrent Osteosarcoma, Refractory Desmoid Fibromatosis, Refractory Ewing Sarcoma, Refractory Hepatoblastoma, Refractory Hepatocellular Carcinoma, Refractory Malignant Solid Neoplasm, Refractory Non-Hodgkin Lymphoma, Refractory Osteosarcoma, Solid Pseudopapillary Neoplasm of the Pancreas, Wilms Tumor
Interventions
Biospecimen Collection, Dual X-ray Absorptiometry, Tegavivint, X-Ray Imaging
Procedure · Drug
Lead sponsor
Children's Oncology Group
Network
Eligibility
12 Months to 30 Years
Enrollment
147 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2028
U.S. locations
21
States / cities
Birmingham, Alabama • Los Angeles, California • Orange, California + 18 more
Conditions
Relapsed Pediatric Solid Tumors, Refractory Pediatric Solid Tumors, Rhabdomyosarcoma, Ewing Sarcoma, Osteosarcoma, Neuroblastoma, Wilms Tumor, Hepatic Tumor, Germ Cell Tumor, Desmoid Tumor
Interventions
Magnetic Resonance High Intensity Focused Ultrasound
Device
Lead sponsor
AeRang Kim
Other
Eligibility
Up to 30 Years
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2024
U.S. locations
2
States / cities
Washington D.C., District of Columbia • Cincinnati, Ohio
Conditions
Cancer
Interventions
therapeutic allogeneic lymphocytes
Biological
Lead sponsor
University of Medicine and Dentistry of New Jersey
Other
Eligibility
Not listed
Enrollment
37 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2011
U.S. locations
1
States / cities
New Brunswick, New Jersey
Conditions
Ewing Sarcoma, Gastrointestinal Tumor, Germ Cell Tumor, Hepatic Tumor, Lymphoma, Wilms Tumor, Rhabdoid Tumor, Clear Cell Carcinoma, Renal Cell Carcinoma, Melanoma, Neuroblastoma, Rhabdomyosarcoma, Non-rhabdomyosarcoma
Interventions
alemtuzumab, fludarabine, sirolimus, Busulfan, melphalan, stem cells, CliniMACS
Drug · Biological · Device
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
2 Years to 21 Years
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2020
U.S. locations
1
States / cities
Memphis, Tennessee
Conditions
Erythropoietic Protoporphyria (EPP), X-Linked Protoporphyria (XLP)
Interventions
Placebo, DISC-1459
Drug
Lead sponsor
Disc Medicine, Inc
Industry
Eligibility
12 Years and older
Enrollment
183 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
11
States / cities
Huntington Beach, California • San Francisco, California • Miami, Florida + 7 more
Conditions
Alpha 1-Antitrypsin Deficiency, Emphysema, COPD
Interventions
Placebo Oral Tablet, Alvelestat oral tablet - dose 1, Alvelestat oral tablet - dose 2
Drug
Lead sponsor
Mereo BioPharma
Industry
Eligibility
18 Years to 75 Years
Enrollment
99 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
4
States / cities
Birmingham, Alabama • Los Angeles, California • Sacramento, California + 1 more
Conditions
Phenylketonuria
Interventions
Repinatrabit
Drug
Lead sponsor
Otsuka Pharmaceutical Development & Commercialization, Inc.
Industry
Eligibility
18 Years to 65 Years
Enrollment
32 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2026 – 2027
U.S. locations
1
States / cities
Miami, Florida
Conditions
Wilson Disease
Interventions
Not listed
Lead sponsor
Prime Medicine, Inc.
Industry
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
7
States / cities
Los Angeles, California • Sacramento, California • New Haven, Connecticut + 4 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 | 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
Conditions
Recurrent Childhood Brain Stem Glioma, Recurrent Childhood Cerebellar Astrocytoma, Recurrent Childhood Cerebral Astrocytoma, Recurrent Childhood Ependymoma, Recurrent Childhood Large Cell Lymphoma, Recurrent Childhood Liver Cancer, Recurrent Childhood Lymphoblastic Lymphoma, Recurrent Childhood Malignant Germ Cell Tumor, Recurrent Childhood Medulloblastoma, Recurrent Childhood Rhabdomyosarcoma, Recurrent Childhood Small Noncleaved Cell Lymphoma, Recurrent Childhood Soft Tissue Sarcoma, Recurrent Childhood Supratentorial Primitive Neuroectodermal Tumor, Recurrent Childhood Visual Pathway and Hypothalamic Glioma, Recurrent Childhood Visual Pathway Glioma, Recurrent Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor, Recurrent Neuroblastoma, Recurrent Osteosarcoma, Recurrent Retinoblastoma, Recurrent Wilms Tumor and Other Childhood Kidney Tumors, Recurrent/Refractory Childhood Hodgkin Lymphoma, Unspecified Childhood Solid Tumor, Protocol Specific
Interventions
alvocidib, pharmacological study
Drug · Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
Up to 21 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2001
U.S. locations
1
States / cities
Arcadia, California