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Conditions: Parkinson's Disease, Sleep Disordered Breathing, Sleep Obstructive Apnea
Interventions: Neurocognitive function, Sleep evaluations; Other
Lead sponsor: University of Miami; Other
Eligibility: 18 Years and older
Enrollment: 89 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2016
U.S. locations: 1
States / cities: Miami, Florida

Conditions: Lamellar Ichthyosis
Interventions: CD5789 Cream 200 µg/g, CD5789 Cream 100 µg/g, CD5789 Cream Vehicle; Drug
Lead sponsor: Mayne Pharma International Pty Ltd; Industry
Eligibility: 12 Years and older
Enrollment: 65 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2021
U.S. locations: 10
States / cities: San Diego, California • Aurora, Colorado • New Haven, Connecticut + 7 more

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: Canavan Disease
Interventions: rAAV-Olig001-ASPA, Levetiracetam, Prednisone; Drug
Lead sponsor: Myrtelle Inc.; Industry
Eligibility: 3 Months to 60 Months
Enrollment: 24 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2027
U.S. locations: 1
States / cities: Dayton, Ohio

Conditions: Epilepsy, GLUT1DS1, Glut1 Deficiency Syndrome 1, Autosomal Recessive, Glucose Metabolism Disorders, Glucose Transport Defect, Glucose Transporter Type 1 Deficiency Syndrome, Glucose Transporter Protein Type 1 Deficiency Syndrome
Interventions: Triheptanoin; Drug
Lead sponsor: Juan Pascual; Other
Eligibility: 2 Years to 35 Years
Enrollment: 12 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017
U.S. locations: 1
States / cities: Dallas, Texas

Conditions: Gene Mutations, Pseudoxanthoma Elasticum, Arterial Calcification, Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets Type 2
Interventions: INZ-701; Drug
Lead sponsor: Inozyme Pharma; Industry
Eligibility: 1 Year and older
Enrollment: 22 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2026
U.S. locations: 2
States / cities: Rochester, Minnesota • Eatontown, New Jersey

Conditions: Glut1 Deficiency Syndrome 1, Glucose Metabolism Disorders, Epilepsy, Glut1 Deficiency Syndrome 1, Autosomal Recessive, Glucose Transporter Type 1 Deficiency Syndrome, Glucose Transporter Protein Type 1 Deficiency Syndrome, Glucose Transport Defect
Interventions: Magnetic resonance imaging; Device
Lead sponsor: Weill Medical College of Cornell University; Other
Eligibility: 16 Years to 80 Years
Enrollment: 20 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2022 – 2029
U.S. locations: 2
States / cities: New York, New York • Dallas, Texas

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: 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: 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: 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: Myocardial Infarction
Interventions: BB3, Normal saline; Drug
Lead sponsor: Angion Biomedica Corp; Industry
Eligibility: 21 Years to 80 Years
Enrollment: 5 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2013
U.S. locations: 2
States / cities: Minneapolis, Minnesota • New Haven, New York

Conditions: Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, ATP-Binding Cassette Subfamily C Member 6 Deficiency, Generalized Arterial Calcification of Infancy, Autosomal Recessive Hypophosphatemic Rickets, PXE (Pseudoxanthoma Elasticum)
Interventions: Not listed
Lead sponsor: Inozyme Pharma; Industry
Eligibility: 1 Day and older
Enrollment: 23 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2025
U.S. locations: 1
States / cities: Philadelphia, Pennsylvania

Conditions: Neutropenia
Interventions: Mavorixafor, Placebo; Drug
Lead sponsor: X4 Pharmaceuticals; Industry
Eligibility: 12 Years and older
Enrollment: 176 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2027
U.S. locations: 22
States / cities: Phoenix, Arizona • Irvine, California • La Jolla, California + 19 more

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: Cyclic Neutropenia, Congenital Neutropenia, Neutropenia
Interventions: Serial Blood Count Study, Athelas Home device; Device
Lead sponsor: University of Washington; Other
Eligibility: 5 Years and older
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2025
U.S. locations: 1
States / cities: Seattle, Washington

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: TNNT1-associated Myopathy, Infantile-onset Nemaline Rod Myopathy, Myopathies, Nemaline, Myopathy, Myopathy, Rod, Myopathy; Hereditary, Amish Nemaline Myopathy, Nemaline Myopathy 5, NEM5, Genetic Muscle Disease, Recessive Hereditary Disorder (Autosomal), ANM
Interventions: Not listed
Lead sponsor: Clinic for Special Children; Other
Eligibility: Not listed
Enrollment: 40 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2027
U.S. locations: 1
States / cities: Gordonville, Pennsylvania

Conditions: Sickle Cell Disease, Thalassemia, Anemia, Granuloma, Wiskott-Aldrich Syndrome, Chediak Higashi Syndrome, Osteopetrosis, Neutropenia, Thrombocytopenia, Hurler Disease, Niemann-Pick Disease, Fucosidosis
Interventions: Hematopoietic stem cell transplantation; Procedure
Lead sponsor: Children's Hospital Los Angeles; Other
Eligibility: Up to 21 Years
Enrollment: 25 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2015
U.S. locations: 1
States / cities: Los Angeles, California

Conditions: Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets, Generalized Arterial Calcification of Infancy, ATP-Binding Cassette Subfamily C Member 6 Deficiency, Pseudoxanthoma Elasticum
Interventions: INZ-701; Drug
Lead sponsor: Inozyme Pharma; Industry
Eligibility: Up to 1 Year
Enrollment: 16 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2026
U.S. locations: 3
States / cities: San Diego, California • Columbus, Ohio • Philadelphia, Pennsylvania

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: 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: Leber Congenital Amaurosis, Leber Congenital Amaurosis (LCA)
Interventions: OPGx-RDH12; Drug
Lead sponsor: Opus Genetics, Inc; Industry
Eligibility: 18 Years and older
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2034
U.S. locations: 3
States / cities: Phoenix, Arizona • Philadelphia, Pennsylvania • Houston, Texas

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

Search by objective public record fields.

Sleep Disordered Breathing and Its Impact on Neuro-cognitive Performance and Quality of Life in Parkinson Disease

A Safety, Efficacy and Systemic Exposure Study of CD5789 Cream in Adults and Adolescents With Lamellar Ichthyosis

Hypnosis-Based Machine Learning Biomarker Study

rAAV-Olig001-ASPA Gene Therapy for Treatment of Children With Typical Canavan Disease

Treatment Development of Triheptanoin (G1D)

ADAPT Study: Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 Deficiency

Brain Metabolism Observed at 3 Tesla or 7 Tesla in Health and Metabolic Disease

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

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

Hereditary Parkinson s Disease Natural History Protocol

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

Study to Evaluate the Safety and Activity of BB3 to Treat Heart Attack

Natural History Study: ENPP1 Deficiency or the Early-Onset Form of ABCC6 Deficiency

A Study of Mavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenic Disorders Who Are Experiencing Recurrent and/or Serious Infections

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)

Serial Blood Count Study

Do Endotypes Predict Response and Sequelae in OSA Patients

WiTNNess - TNNT1 Myopathy Natural History Study

Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells

The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency

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

Investigation of the Genetics of Hematologic Diseases

Safety and Tolerability of Subretinal OPGx-RDH12-1001 for LCA-Associated Inherited Retinal Degeneration (LCA-IRD)

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