- 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
Search Results
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Showing 25–48
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- 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
- 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
- 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