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Conditions: Disorder; Trance, Anxiety
Interventions: Standardized Hypnotic Susceptibility Testing; Behavioral
Lead sponsor: Icahn School of Medicine at Mount Sinai; Other
Eligibility: 18 Years to 65 Years
Enrollment: 50 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2025
U.S. locations: 1
States / cities: New York, New York

Conditions: Congenital Amegakaryocytic Thrombocytopenia, Leukemia, Myelodysplastic Syndromes, Severe Congenital Neutropenia
Interventions: anti-thymocyte globulin, therapeutic allogeneic lymphocytes, fludarabine phosphate, thiotepa, allogeneic bone marrow transplantation, allogeneic hematopoietic stem cell transplantation, in vitro-treated peripheral blood stem cell transplantation, total-body irradiation; Biological · Drug · Procedure + 1 more
Lead sponsor: University of California, San Francisco; Other
Eligibility: 1 Year to 17 Years
Enrollment: 21 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2011
U.S. locations: 2
States / cities: San Francisco, California • Chapel Hill, North Carolina

Conditions: Antibody-mediated Rejection, Hyperacute Rejection of Cardiac Transplant, Left Ventricular Dysfunction, Cardiac Allograft Vasculopathy, Heart Graft Dysfunction
Interventions: Eculizumab; Drug
Lead sponsor: Cedars-Sinai Medical Center; Other
Eligibility: 18 Years to 75 Years
Enrollment: 36 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2020
U.S. locations: 1
States / cities: Los Angeles, California

Conditions: Retinal Degeneration, Retinitis Pigmentosa, Eye Diseases, Hereditary
Interventions: Not listed
Lead sponsor: Jaeb Center for Health Research; Other
Eligibility: 8 Years and older
Enrollment: 44 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2027
U.S. locations: 3
States / cities: San Francisco, California • Baltimore, Maryland • Durham, North Carolina

Conditions: Chronic Obstructive Airway Disease
Interventions: Bi-level positive airway pressure (BPAP), Oxygen, CPAP; Device · Drug
Lead sponsor: Brigham and Women's Hospital; Other
Eligibility: 18 Years to 75 Years
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2011
U.S. locations: 2
States / cities: Boston, Massachusetts

Conditions: Autosomal Recessive Ichthyosis
Interventions: KB105, Placebo; Biological · Other
Lead sponsor: Krystal Biotech, Inc.; Industry
Eligibility: 6 Months and older
Enrollment: 15 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2024
U.S. locations: 1
States / cities: Rancho Santa Margarita, California

Conditions: Parkinson Disease 6, Early-Onset, Parkinson Disease (Autosomal Recessive, Early Onset) 7, Human, Parkinson Disease Autosomal Recessive, Early Onset, Parkinson Disease, Autosomal Recessive Early-Onset, Digenic, Pink1/Dj1
Interventions: Not listed
Lead sponsor: National Heart, Lung, and Blood Institute (NHLBI); NIH
Eligibility: 18 Years to 80 Years
Enrollment: 31 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2017
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Polycystic Kidney, Autosomal Recessive
Interventions: Tesevatinib; Drug
Lead sponsor: Kadmon Corporation, LLC; Industry
Eligibility: 5 Years to 12 Years
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2019
U.S. locations: 2
States / cities: Philadelphia, Pennsylvania • Milwaukee, Wisconsin

Conditions: Stargardt Disease, Stargardt Macular Degeneration, Stargardt Macular Dystrophy, Autosomal Recessive Stargardt Disease 1 (ABCA4-related)
Interventions: ALK-001; Drug
Lead sponsor: Alkeus Pharmaceuticals, Inc.; Industry
Eligibility: 8 Years to 70 Years
Enrollment: 200 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2026
U.S. locations: 8
States / cities: Phoenix, Arizona • Los Angeles, California • Aurora, Colorado + 5 more

Conditions: Obstructive Sleep Apnea, Sleep Apnea, Sleep Apnea, Obstructive, Sleep, Sleep Disorder
Interventions: Eszopiclone, Supplemental Oxygen, Continuous Positive Airway Pressure (CPAP); Drug · Other · Device
Lead sponsor: University of California, San Diego; Other
Eligibility: 21 Years to 65 Years
Enrollment: 233 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2026
U.S. locations: 1
States / cities: La Jolla, California

Conditions: GJB2-mediated Hearing Loss, GJB2 Gene Mutation, DFNB1A
Interventions: SKY-GJB2; Combination Product
Lead sponsor: Skylark Bio Inc.; Industry
Eligibility: 9 Months to 7 Years
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2028
U.S. locations: 4
States / cities: Bethesda, Maryland • Boston, Massachusetts • Oklahoma City, Oklahoma + 1 more

Conditions: Aortopathies, Thoracic Aortic Aneurysm, Aortic Valve Disease, Thoracic Aortic Disease, Thoracic Aortic Dissection, Thoracic Aortic Rupture, Ascending Aortic Disease, Descending Aortic Disease, Ascending Aortic Aneurysm, Descending Aortic Aneurysm, Marfan Syndrome, Loeys-Dietz Syndrome, Vascular Ehlers-Danlos Syndrome, Shprintzen-Goldberg Syndrome, Turner Syndrome, PHACE Syndrome, Autosomal Recessive Cutis Laxa, Congenital Contractural Arachnodactyly, Arterial Tortuosity Syndrome, Bicuspid Aortic Valve-Associated Aortopathy, Bicuspid Aortic Valve, Familial Thoracic Aortic Aneurysm and Aortic Dissection
Interventions: Not listed
Lead sponsor: Yale University; Other
Eligibility: Not listed
Enrollment: 3,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2030
U.S. locations: 2
States / cities: Atlanta, Georgia • Indianapolis, Indiana

Conditions: Erectile Dysfunction Due to Arterial Insufficiency, Erectile Dysfunction, Erectile Dysfunction Due to Arterial Disease, Chronic Pelvic Pain Syndrome, Chronic Prostatitis, Erectile Dysfunction Following Radical Prostatectomy, Erectile Dysfunction Following Radiation Therapy
Interventions: Focused shockwave, Radial wave, Sham treatment; Device
Lead sponsor: The Cleveland Clinic; Other
Eligibility: 40 Years and older · Male only
Enrollment: 186 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2027
U.S. locations: 1
States / cities: Cleveland, Ohio

Conditions: Obstructive Sleep Apnea Syndrome
Interventions: OxiplexTS; Device
Lead sponsor: ISS, Inc.; Industry
Eligibility: 21 Years to 55 Years
Enrollment: 309 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2007 – 2010
U.S. locations: 4
States / cities: Orange, California • Stanford, California • Chicago, Illinois + 1 more

Conditions: Chronic Myeloid Leukemia (CML), Acute Myelogenous Leukemia (AML), Myelodysplastic Syndrome (MDS), Juvenile Myelomonocytic Leukemia (JMML), Acute Lymphoblastic Leukemia (ALL), Lymphoma (Hodgkin's and Non-Hodgkin's)
Interventions: CliniMACS CD34+ Reagent System, Thiotepa, Cyclophosphamide, Alemtuzumab, Tacrolimus, Melphalan, Busulfan, Fludarabine, Methylprednisolone; Device · Drug
Lead sponsor: Diane George; Other
Eligibility: Up to 22 Years
Enrollment: 14 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2026
U.S. locations: 1
States / cities: New York, New York

Conditions: Osteopetrosis
Interventions: Stem Cell Transplantation, Miltenyi Biotec CliniMACS, Systemic chemotherapy and antibodies; Procedure · Device · Drug
Lead sponsor: St. Jude Children's Research Hospital; Other
Eligibility: Not listed
Enrollment: 15 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2004 – 2009
U.S. locations: 1
States / cities: Memphis, Tennessee

Conditions: Bone Marrow Failure Syndromes, Erythrocyte Disorder, Leukocyte Disorder, Hemostasis, Blood Coagulation Disorder, Sickle Cell Disease, Dyskeratosis Congenita, Diamond-Blackfan Anemia, Congenital Thrombocytopenia, Severe Congenital Neutropenia, Fanconi Anemia, Myelodysplastic Syndromes, Myeloproliferative Diseases
Interventions: Not listed
Lead sponsor: St. Jude Children's Research Hospital; Other
Eligibility: Not listed
Enrollment: 1,716 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2050
U.S. locations: 1
States / cities: Memphis, Tennessee

Conditions: Bone Marrow Failure Syndrome, Severe Aplastic Anemia, Severe Congenital Neutropenia, Amegakaryocytic Thrombocytopenia, Diamond-Blackfan Anemia, Schwachman Diamond Syndrome, Primary Immunodeficiency Syndromes, Acquired Immunodeficiency Syndromes, Histiocytic Syndrome, Familial Hemophagocytic Lymphocytosis, Lymphohistiocytosis, Macrophage Activation Syndrome, Langerhans Cell Histiocytosis (LCH), Hemoglobinopathies, Sickle Cell Disease, Sickle Cell-beta-thalassemia
Interventions: CD34 Stem Cell Selection Therapy; Biological
Lead sponsor: Diane George; Other
Eligibility: Up to 40 Years
Enrollment: 37 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2030
U.S. locations: 1
States / cities: New York, New York

Conditions: Congenital Amegakaryocytic Thrombocytopenia, Diamond-blackfan Anemia, Leukemia, Myelodysplastic Syndromes, Severe Congenital Neutropenia
Interventions: alemtuzumab, busulfan, cyclosporine, fludarabine phosphate, methotrexate, methylprednisolone, allogeneic bone marrow transplantation, allogeneic hematopoietic stem cell transplantation, peripheral blood stem cell transplantation, umbilical cord blood transplantation; Biological · Drug · Procedure
Lead sponsor: University of California, San Francisco; Other
Eligibility: Up to 21 Years
Enrollment: 35 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2011
U.S. locations: 2
States / cities: San Francisco, California • Madison, Wisconsin

Conditions: Autosomal Recessive Polycystic Kidney (ARPKD)
Interventions: Tolvaptan Suspension, Tolvaptan Tablets; Drug
Lead sponsor: Otsuka Pharmaceutical Development & Commercialization, Inc.; Industry
Eligibility: 28 Days to 18 Years
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2028
U.S. locations: 12
States / cities: Washington D.C., District of Columbia • Atlanta, Georgia • Chicago, Illinois + 9 more

Conditions: Obstructive Sleep Apnea
Interventions: Artificial Neural Network; Procedure
Lead sponsor: State University of New York at Buffalo; Other
Eligibility: 18 Years to 80 Years
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2009
U.S. locations: 1
States / cities: Buffalo, New York

Conditions: Autosomal Recessive Disorder, Aneuploidy
Interventions: Computer-Aided Genetic Education Module; Other
Lead sponsor: Wayne State University; Other
Eligibility: 18 Years and older · Female only
Enrollment: 100 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2018 – 2019
U.S. locations: 1
States / cities: Detroit, Michigan

Conditions: Respiratory Distress Syndrome
Interventions: HHHFNC, NCPAP; Device
Lead sponsor: Sidra Medicine; Other
Eligibility: Not listed
Enrollment: 60 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2012
U.S. locations: 1
States / cities: Akron, Ohio

Conditions: Molybdenum Cofactor Deficiency (MoCD), Rare Autosomal Recessive Disorder, Deficiency of Activity of Molybdenum-dependent Enzymes (Sulfite Oxidase [SOX], Xanthine Dehydrogenase, and Aldehyde Oxidase)
Interventions: ALXN1101, Placebo; Drug
Lead sponsor: Origin Biosciences; Industry
Eligibility: 18 Years to 60 Years
Enrollment: 24 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2013
U.S. locations: 1
States / cities: Baltimore, Maryland

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Hypnosis-Based Machine Learning Biomarker Study

Donor Stem Cell Transplant in Treating Young Patients With Myelodysplastic Syndrome, Leukemia, Bone Marrow Failure Syndrome, or Severe Immunodeficiency Disease

The De-novo Use of Eculizumab in Presensitized Patients Receiving Cardiac Transplantation

Rate of Progression of PCDH15-Related Retinal Degeneration in Usher Syndrome 1F

Cardiac Hemodynamics in Overlap Syndrome (COPD With Obstructive Sleep Apnea)

Topical KB105 for the Treatment of TGM1-deficient Autosomal Recessive Congenital Ichthyosis (ARCI)

Hereditary Parkinson s Disease Natural History Protocol

A Safety, Pharmacokinetic, Single Ascending Dose Study of Tesevatinib in Pediatric Subjects With Autosomal Recessive Polycystic Kidney Disease (ARPKD)

Open-Label Extension: Tolerability and Effects of ALK-001 on Stargardt Disease (TEASE)

Do Endotypes Predict Response and Sequelae in OSA Patients

Open-label Study of SKY-GJB2 in Pediatric Subjects With GJB2-mediated Hearing Loss

Pathogenetic Basis of Aortopathy and Aortic Valve Disease

Efficacy of Low-intensity Shockwave vs Radial Wave for Treatment of Erectile Dysfunction and Pelvic Pain

Brain Oxygenation and Hemodynamics During Sleep in Obstructive Sleep Apnea Sufferers

CD34+ (Malignant) Stem Cell Selection for Patients Receiving Allogenic Stem Cell Transplant

Stem Cell Transplantation for Children Affected With Osteopetrosis

Investigation of the Genetics of Hematologic Diseases

CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation

Alemtuzumab, Fludarabine, and Busulfan Followed By Donor Stem Cell Transplant in Treating Young Patients With Hematologic Disorders

A Study to See if Tolvaptan is Safe in Infants and Children Who at Enrollment Are 28 Days to Less Than 18 Years Old With Autosomal Recessive Polycystic Kidney Disease (ARPKD)

CPAP Titration Using an Artificial Neural Network: A Randomized Controlled Study

Prenatal Computer-Aided Genetics Education Module

High Flow Nasal Cannula in Comparison With Nasal Continuous Positive Airway Pressure in the Management of Respiratory Distress Syndrome

Phase 1 Single Dose Study of ALXN1101 in Healthy Volunteers