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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Conditions
Angelman Syndrome
Interventions
No intervention, GTX-102
Other · Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
1 Year to 64 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
8
States / cities
Los Angeles, California • Chicago, Illinois • Baltimore, Maryland + 5 more
Completed Phase 2 Interventional Results available

RAPAMYCIN FOR KIDNEY ANGIOMYOLIPOMAS

NCT00126672
Conditions
Nonmalignant Neoplasm, Tuberous Sclerosis, Lymphangioleimyomatosis, Kidney Angiomyolipoma
Interventions
Rapamycin
Drug
Lead sponsor
Dana-Farber Cancer Institute
Other
Eligibility
3 Years to 65 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2010
U.S. locations
7
States / cities
Loma Linda, California • Hartford, Connecticut • Boston, Massachusetts + 4 more
Conditions
Metastatic Colorectal Cancer
Interventions
Pembrolizumab, Binimetinib, Oxaliplatin, Leucovorin, 5-Fluorouracil [5-FU], Irinotecan
Biological · Drug
Lead sponsor
Merck Sharp & Dohme LLC
Industry
Eligibility
18 Years and older
Enrollment
116 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
10
States / cities
Duarte, California • Aurora, Colorado • New Haven, Connecticut + 7 more
Conditions
Primary Hyperoxaluria
Interventions
Oxalobacter formigenes, Placebo
Biological · Drug
Lead sponsor
OxThera
Industry
Eligibility
5 Years and older
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2008
U.S. locations
1
States / cities
Rochester, Minnesota
Conditions
Epidermolysis Bullosa
Interventions
SD-101-6.0 cream, Placebo (SD-101-0.0) cream
Drug
Lead sponsor
Scioderm, Inc.
Industry
Eligibility
1 Month and older
Enrollment
169 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
21
States / cities
Phoenix, Arizona • Redwood City, California • Aurora, Colorado + 17 more
Conditions
Epilepsy, Tuberous Sclerosis Complex, Focal Cortical Dysplasia
Interventions
Everolimus
Drug
Lead sponsor
NYU Langone Health
Other
Eligibility
2 Years to 40 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
1
States / cities
New York, New York
Completed Phase 1Phase 2 Interventional Results available

Rivastigmine Study in Adolescents With Down Syndrome

NCT01084135
Conditions
Down Syndrome
Interventions
Rivastigmine, Liquid Placebo
Drug · Other
Lead sponsor
Duke University
Other
Eligibility
10 Years to 17 Years
Enrollment
42 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2014
U.S. locations
2
States / cities
Baltimore, Maryland • Durham, North Carolina
Conditions
Post-Acute COVID-19 Syndrome, ME/CFS, Rheumatic Arthritis, Juvenile Rheumatoid Arthritis (JRA), Psoriatic Arthritis (PsA), Ankylosing Spondylitis (AS), Autoimmune Encephalitis, Celiac Disease, Celiac Disease in Children, Chronic Lyme Disease, Post-treatment Lyme Disease Syndrome, Crohn's Disease, Dysautonomia, Anorexia Nervosa, Bulimia Nervosa, ARFID, Avoidant / Restrictive Food Intake Disorder, Ehlers Danlos Syndrome, Endometriosis, Fibromyalgia (FM), Long COVID, Lupus, Migraines, Mast Cell Activation Syndrome, Multiple Sclerosis, Myalgic Encephalomyelitis (ME), Myasthenia Gravis, Generalized, Myasthenia Gravis in Children, Narcolepsy, Obsessive Compulsive Disorder (OCD), PANDAS, Pediatric Acute-onset Neuropsychiatric Syndrome (PANS), POTS - Postural Orthostatic Tachycardia Syndrome, General Anxiety Disorder, Social Anxiety Disorder, PTSD - Post Traumatic Stress Disorder, Psoriasis, Traumatic Brain Injury, Tourette's Syndrome, Inflammatory Bowel Disease (IBD), Autoimmune Diseases, Neurological Diseases or Conditions, Psychiatric Disorder, Sjogren's Syndrome, Ulcerative Colitis and Crohn's Disease
Interventions
Not listed
Lead sponsor
Brain Inflammation Collaborative
Other
Eligibility
2 Years and older
Enrollment
10,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2030
U.S. locations
1
States / cities
Delafield, Wisconsin
Conditions
Hematopoietic/Lymphoid Cancer, Accelerated Phase Chronic Myelogenous Leukemia, Acute Undifferentiated Leukemia, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(15;17)(q22;q12), Adult Acute Myeloid Leukemia With t(16;16)(p13;q22), Adult Acute Myeloid Leukemia With t(8;21)(q22;q22), Adult Grade III Lymphomatoid Granulomatosis, Adult Nasal Type Extranodal NK/T-cell Lymphoma, Anaplastic Large Cell Lymphoma, Angioimmunoblastic T-cell Lymphoma, Aplastic Anemia, Atypical Chronic Myeloid Leukemia, BCR-ABL Negative, Blastic Phase Chronic Myelogenous Leukemia, Chronic Eosinophilic Leukemia, Chronic Myelomonocytic Leukemia, Chronic Neutrophilic Leukemia, Chronic Phase Chronic Myelogenous Leukemia, Contiguous Stage II Adult Burkitt Lymphoma, Contiguous Stage II Adult Diffuse Large Cell Lymphoma, Contiguous Stage II Adult Diffuse Mixed Cell Lymphoma, Contiguous Stage II Adult Diffuse Small Cleaved Cell Lymphoma, Contiguous Stage II Adult Immunoblastic Large Cell Lymphoma, Contiguous Stage II Adult Lymphoblastic Lymphoma, Contiguous Stage II Grade 1 Follicular Lymphoma, Contiguous Stage II Grade 2 Follicular Lymphoma, Contiguous Stage II Grade 3 Follicular Lymphoma, Contiguous Stage II Mantle Cell Lymphoma, Contiguous Stage II Marginal Zone Lymphoma, Contiguous Stage II Small Lymphocytic Lymphoma, Cutaneous B-cell Non-Hodgkin Lymphoma, Cytomegalovirus Infection, de Novo Myelodysplastic Syndromes, Essential Thrombocythemia, Extramedullary Plasmacytoma, Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue, Intraocular Lymphoma, Isolated Plasmacytoma of Bone, Mast Cell Leukemia, Meningeal Chronic Myelogenous Leukemia, Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable, Nodal Marginal Zone B-cell Lymphoma, Noncontiguous Stage II Adult Burkitt Lymphoma, Noncontiguous Stage II Adult Diffuse Large Cell Lymphoma, Noncontiguous Stage II Adult Diffuse Mixed Cell Lymphoma, Noncontiguous Stage II Adult Diffuse Small Cleaved Cell Lymphoma, Noncontiguous Stage II Adult Immunoblastic Large Cell Lymphoma, Noncontiguous Stage II Adult Lymphoblastic Lymphoma, Noncontiguous Stage II Grade 1 Follicular Lymphoma, Noncontiguous Stage II Grade 2 Follicular Lymphoma, Noncontiguous Stage II Grade 3 Follicular Lymphoma, Noncontiguous Stage II Mantle Cell Lymphoma, Noncontiguous Stage II Marginal Zone Lymphoma, Noncontiguous Stage II Small Lymphocytic Lymphoma, Polycythemia Vera, Post-transplant Lymphoproliferative Disorder, Previously Treated Myelodysplastic Syndromes, Primary Myelofibrosis, Primary Systemic Amyloidosis, Progressive Hairy Cell Leukemia, Initial Treatment, Prolymphocytic Leukemia, Recurrent Adult Acute Lymphoblastic Leukemia, Recurrent Adult Acute Myeloid Leukemia, Recurrent Adult Burkitt Lymphoma, Recurrent Adult Diffuse Large Cell Lymphoma, Recurrent Adult Diffuse Mixed Cell Lymphoma, Recurrent Adult Diffuse Small Cleaved Cell Lymphoma, Recurrent Adult Grade III Lymphomatoid Granulomatosis, Recurrent Adult Hodgkin Lymphoma, Recurrent Adult Immunoblastic Large Cell Lymphoma, Recurrent Adult Lymphoblastic Lymphoma, Recurrent Adult T-cell Leukemia/Lymphoma, Recurrent Cutaneous T-cell Non-Hodgkin Lymphoma, Recurrent Grade 1 Follicular Lymphoma, Recurrent Grade 2 Follicular Lymphoma, Recurrent Grade 3 Follicular Lymphoma, Recurrent Mantle Cell Lymphoma, Recurrent Marginal Zone Lymphoma, Recurrent Mycosis Fungoides/Sezary Syndrome, Recurrent Small Lymphocytic Lymphoma, Refractory Chronic Lymphocytic Leukemia, Refractory Hairy Cell Leukemia, Refractory Multiple Myeloma, Relapsing Chronic Myelogenous Leukemia, Secondary Acute Myeloid Leukemia, Secondary Myelodysplastic Syndromes, Secondary Myelofibrosis, Splenic Marginal Zone Lymphoma, Stage 0 Chronic Lymphocytic Leukemia, Stage I Adult Burkitt Lymphoma, Stage I Adult Diffuse Large Cell Lymphoma, Stage I Adult Diffuse Mixed Cell Lymphoma, Stage I Adult Diffuse Small Cleaved Cell Lymphoma, Stage I Adult Hodgkin Lymphoma, Stage I Adult Immunoblastic Large Cell Lymphoma, Stage I Adult Lymphoblastic Lymphoma, Stage I Adult T-cell Leukemia/Lymphoma, Stage I Chronic Lymphocytic Leukemia, Stage I Cutaneous T-cell Non-Hodgkin Lymphoma, Stage I Grade 1 Follicular Lymphoma, Stage I Grade 2 Follicular Lymphoma, Stage I Grade 3 Follicular Lymphoma, Stage I Mantle Cell Lymphoma, Stage I Marginal Zone Lymphoma, Stage I Multiple Myeloma, Stage I Mycosis Fungoides/Sezary Syndrome, Stage I Small Lymphocytic Lymphoma, Stage II Adult Hodgkin Lymphoma, Stage II Adult T-cell Leukemia/Lymphoma, Stage II Chronic Lymphocytic Leukemia, Stage II Cutaneous T-cell Non-Hodgkin Lymphoma, Stage II Multiple Myeloma, Stage II Mycosis Fungoides/Sezary Syndrome, Stage III Adult Burkitt Lymphoma, Stage III Adult Diffuse Large Cell Lymphoma, Stage III Adult Diffuse Mixed Cell Lymphoma, Stage III Adult Diffuse Small Cleaved Cell Lymphoma, Stage III Adult Hodgkin Lymphoma, Stage III Adult Immunoblastic Large Cell Lymphoma, Stage III Adult Lymphoblastic Lymphoma, Stage III Adult T-cell Leukemia/Lymphoma, Stage III Chronic Lymphocytic Leukemia, Stage III Cutaneous T-cell Non-Hodgkin Lymphoma, Stage III Grade 1 Follicular Lymphoma, Stage III Grade 2 Follicular Lymphoma, Stage III Grade 3 Follicular Lymphoma, Stage III Mantle Cell Lymphoma, Stage III Marginal Zone Lymphoma, Stage III Multiple Myeloma, Stage III Mycosis Fungoides/Sezary Syndrome, Stage III Small Lymphocytic Lymphoma, Stage IV Adult Burkitt Lymphoma, Stage IV Adult Diffuse Large Cell Lymphoma, Stage IV Adult Diffuse Mixed Cell Lymphoma, Stage IV Adult Diffuse Small Cleaved Cell Lymphoma, Stage IV Adult Hodgkin Lymphoma, Stage IV Adult Immunoblastic Large Cell Lymphoma, Stage IV Adult Lymphoblastic Lymphoma, Stage IV Adult T-cell Leukemia/Lymphoma, Stage IV Chronic Lymphocytic Leukemia, Stage IV Cutaneous T-cell Non-Hodgkin Lymphoma, Stage IV Grade 1 Follicular Lymphoma, Stage IV Grade 2 Follicular Lymphoma, Stage IV Grade 3 Follicular Lymphoma, Stage IV Mantle Cell Lymphoma, Stage IV Marginal Zone Lymphoma, Stage IV Mycosis Fungoides/Sezary Syndrome, Stage IV Small Lymphocytic Lymphoma, T-cell Large Granular Lymphocyte Leukemia, Waldenstrom Macroglobulinemia
Interventions
infection prophylaxis and management, laboratory biomarker analysis, flow cytometry, DNA analysis, RNA analysis, management of therapy complications, ganciclovir, valganciclovir, foscarnet sodium, antiviral therapy, polymerase chain reaction, protein expression analysis
Procedure · Other · Genetic + 1 more
Lead sponsor
City of Hope Medical Center
Other
Eligibility
18 Years and older
Enrollment
153 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2026
U.S. locations
1
States / cities
Duarte, California
Conditions
Cystic Fibrosis
Interventions
Riociguat (Adempas, BAY63-2521), Placebo
Drug
Lead sponsor
Bayer
Industry
Eligibility
18 Years and older
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
3
States / cities
Birmingham, Alabama • Denver, Colorado • St Louis, Missouri
Conditions
Cancer
Interventions
graft versus host disease prophylaxis/therapy, allogeneic bone marrow transplantation
Biological · Procedure
Lead sponsor
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Other
Eligibility
Up to 65 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
1997 – 2004
U.S. locations
1
States / cities
Baltimore, Maryland
Conditions
Fabry Disease
Interventions
Pegunigalsidase-alfa
Drug
Lead sponsor
Chiesi Farmaceutici S.p.A.
Industry
Eligibility
18 Years and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
6
States / cities
Birmingham, Alabama • Atlanta, Georgia • Chicago, Illinois + 3 more
Conditions
Hypothalamic Obesity, Multiple Pituitary Hormone Deficiency Genetic Form, Septo-Optic Dysplasia, Optic Nerve Hypoplasia, Childhood-onset Combined Pituitary Hormone Deficiency, Pituitary Stalk Interruption Syndrome
Interventions
Setmelanotide, Placebo
Drug
Lead sponsor
Rhythm Pharmaceuticals, Inc.
Industry
Eligibility
4 Years and older
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
8
States / cities
Birmingham, Alabama • Aurora, Colorado • Chicago, Illinois + 5 more
Recruiting No phase listed Observational Accepts healthy volunteers

MyVHL: Patient Natural History Study

NCT03749980
Conditions
Von Hippel-Lindau Disease, Hereditary Leiomyomatosis and Renal Cell Cancer, Birt-Hogg-Dube Syndrome, SDHB Gene Mutation
Interventions
Not listed
Lead sponsor
Joshua Mann, MPH
Other
Eligibility
Not listed
Enrollment
10,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2028
U.S. locations
1
States / cities
Boston, Massachusetts
Conditions
Down Syndrome, Fetal Aneuploidy
Interventions
Not listed
Lead sponsor
Sequenom, Inc.
Industry
Eligibility
18 Years to 60 Years · Female only
Enrollment
3,062 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2015
U.S. locations
27
States / cities
Mobile, Alabama • Tucson, Arizona • Campbell, California + 24 more
Conditions
Primary Hyperoxaluria
Interventions
DCR-PHXC, Placebo
Drug
Lead sponsor
Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
Industry
Eligibility
6 Years and older
Enrollment
43 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2019
U.S. locations
1
States / cities
Boston, Massachusetts
Conditions
Alpha 1-Antitrypsin Deficiency
Interventions
Alpha-1 MP, alpha-1 proteinase inhibitor (human)
Drug
Lead sponsor
Grifols Therapeutics LLC
Industry
Eligibility
18 Years and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2007
U.S. locations
8
States / cities
Denver, Colorado • Gainesville, Florida • Miami, Florida + 5 more
Conditions
Ankle Arthrodesis or Arthroplasty, Hindfoot Fusion, Ankle Deformity, Ankle Disease, Hindfoot Arthrodesis, Hindfoot Pathologies
Interventions
MagnetOs, Autograft
Device · Other
Lead sponsor
Kuros Biosurgery AG
Industry
Eligibility
18 Years to 75 Years
Enrollment
126 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
5
States / cities
Vail, Colorado • Columbus, Georgia • Charlotte, North Carolina + 2 more
Conditions
Autosomal Dominant Polycystic Kidney Disease
Interventions
Not listed
Lead sponsor
Mayo Clinic
Other
Eligibility
18 Years to 40 Years
Enrollment
18 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2020
U.S. locations
1
States / cities
Rochester, Minnesota
Conditions
Prader-Willi Syndrome, Excessive Daytime Sleepiness, Hyperphagia, Body Weight, Mood, Behavior
Interventions
Sham Light, Bright Light Therapy
Other
Lead sponsor
Maimonides Medical Center
Other
Eligibility
6 Years to 88 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
1
States / cities
Brooklyn, New York
Conditions
Vasomotor Symptoms as a Sex Hormone-dependent Disorder in Women and Men
Interventions
Elinzanetant (BAY3427080)
Drug
Lead sponsor
Bayer
Industry
Eligibility
18 Years to 79 Years
Enrollment
31 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2022
U.S. locations
2
States / cities
Miami, Florida • Orlando, Florida
Conditions
Von Hippel-Lindau Disease
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
12 Years to 80 Years
Enrollment
340 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2017
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Polycystic Liver Disease
Interventions
PLD-Q
Other
Lead sponsor
Mayo Clinic
Other
Eligibility
18 Years and older
Enrollment
594 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2024
U.S. locations
1
States / cities
Rochester, Minnesota
Conditions
Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes
Interventions
anti-thymocyte globulin, sargramostim, busulfan, etoposide, fludarabine phosphate, prednisone, tacrolimus, allogeneic hematopoietic stem cell transplantation, umbilical cord blood transplantation
Biological · Drug · Procedure
Lead sponsor
University of California, San Francisco
Other
Eligibility
18 Years to 55 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2009
U.S. locations
1
States / cities
San Francisco, California