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Conditions: Urinary Incontinence, Cystic Fibrosis
Interventions: Reusable Underwear, Disposable urethral support, Pelvic floor physical therapy; Device · Behavioral
Lead sponsor: Megan Bradley; Other
Eligibility: 18 Years to 99 Years · Female only
Enrollment: 15 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2023
U.S. locations: 1
States / cities: Pittsburgh, Pennsylvania

Conditions: Asthma, COPD, Cystic Fibrosis, Ciliary Motility Disorders, Bronchiectasis, Airway Malacia, Healthy Control
Interventions: AWARE; Diagnostic Test
Lead sponsor: Indiana University; Other
Eligibility: 8 Years to 70 Years
Enrollment: 800 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2024 – 2029
U.S. locations: 2
States / cities: Indianapolis, Indiana • Pittsburgh, Pennsylvania

Conditions: Bronchopulmonary Dysplasia
Interventions: 16S rRNA stool microbiome sequencing.; Diagnostic Test
Lead sponsor: University of Tennessee; Other
Eligibility: 0 Days to 7 Days
Enrollment: 197 participants
Timeline: 2017 – 2020
U.S. locations: 2
States / cities: Memphis, Tennessee

Conditions: Type 1 Diabetes Mellitus
Interventions: 180 mg/kg Alpha1-PI, 90 mg/kg Alpha1-PI, Placebo; Biological
Lead sponsor: Grifols Therapeutics LLC; Industry
Eligibility: 6 Years to 35 Years
Enrollment: 76 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2017
U.S. locations: 36
States / cities: Tucson, Arizona • Los Angeles, California • Orange, California + 28 more

Conditions: Cystic Fibrosis, Exocrine Pancreatic Insufficiency
Interventions: Liprotamase; Drug
Lead sponsor: Anthera Pharmaceuticals; Industry
Eligibility: 7 Years and older
Enrollment: 214 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2009
U.S. locations: 39
States / cities: Oakland, California • Denver, Colorado • Jacksonville, Florida + 35 more

Conditions: Malignant Head and Neck Neoplasm, Mucositis, Radiation-Induced Disorder
Interventions: Educational Intervention, Gabapentin, Pain Therapy, Quality-of-Life Assessment, Questionnaire Administration, Oxycodone/Acetaminophen, Hydrocodone/Acetaminophen, Fentanyl, Ibuprofen, Magic Mouthwash; Other · Drug
Lead sponsor: Vanderbilt-Ingram Cancer Center; Other
Eligibility: 21 Years and older
Enrollment: 79 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2020
U.S. locations: 1
States / cities: Nashville, Tennessee

Conditions: Cystic Fibrosis, Chronic Disease
Interventions: coopeRATE Prompt; Behavioral
Lead sponsor: Dartmouth-Hitchcock Medical Center; Other
Eligibility: 18 Years and older
Enrollment: 219 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021
U.S. locations: 4
States / cities: Denver, Colorado • Kansas City, Kansas • Lebanon, New Hampshire + 1 more

Conditions: Cystic Fibrosis
Interventions: Not listed
Lead sponsor: University of Minnesota; Other
Eligibility: 7 Days to 3 Years
Enrollment: 7 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2019
U.S. locations: 1
States / cities: Minneapolis, Minnesota

Conditions: Cystic Fibrosis
Interventions: ELX/TEZ/IVA, IVA; Drug
Lead sponsor: Vertex Pharmaceuticals Incorporated; Industry
Eligibility: 12 Years and older
Enrollment: 458 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2022
U.S. locations: 46
States / cities: Birmingham, Alabama • Palo Alto, California • Aurora, Colorado + 42 more

Conditions: Familial Adenomatous Polyposis
Interventions: Curcumin, Laboratory Biomarker Analysis, Placebo; Drug · Other
Lead sponsor: National Cancer Institute (NCI); NIH
Eligibility: 18 Years to 85 Years
Enrollment: 44 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2016
U.S. locations: 1
States / cities: Baltimore, Maryland

Conditions: Cystic Fibrosis
Interventions: Ivacaftor, β-Adrenergic cocktail, Pilocarpine Nitrate 5%, Macroduct sweat stimulator; Drug · Device
Lead sponsor: Richard Barry Moss; Other
Eligibility: 18 Years and older
Enrollment: 8 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2015 – 2017
U.S. locations: 1
States / cities: Stanford, California

Conditions: Lynch Syndrome, FAP, Hereditary Diffuse Gastric Cancer, Juvenile Polyposis Syndrome, Peutz-Jeghers Syndrome
Interventions: Research Registry; Other
Lead sponsor: University of Pittsburgh; Other
Eligibility: 8 Years to 100 Years
Enrollment: 114 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2018
U.S. locations: 1
States / cities: Pittsburgh, Pennsylvania

Conditions: Cystic Fibrosis, Patient Engagement
Interventions: 4-part educational program to build patient-engagement methodology capacity; Other
Lead sponsor: University of Washington; Other
Eligibility: Not listed
Enrollment: 100 participants
Timeline: 2019 – 2021
U.S. locations: 1
States / cities: Seattle, Washington

Conditions: Chronic Constipation, Hirschsprung's Disease
Interventions: Endoscopic Mucosal Resection (EMR); Procedure
Lead sponsor: Massachusetts General Hospital; Other
Eligibility: 10 Years and older
Enrollment: 50 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2009
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Vitamin D Deficiency, Cystic Fibrosis
Interventions: High-Dose Vitamin D3, Stool Sample, Sputum Sample, Sham Comparator, Blood draw; Dietary Supplement · Other · Procedure
Lead sponsor: Emory University; Other
Eligibility: 18 Years and older
Enrollment: 41 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2017
U.S. locations: 1
States / cities: Atlanta, Georgia

Conditions: Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia, Asparagine Synthetase Deficiency, CHARGE Syndrome, Early Infantile Epileptic Encephalopathy, FOXG1 Syndrome, KBG Syndrome, Noonan Syndrome, Severe Hemophilia A, Short Bowel Syndrome, Beta-Propeller Protein-Associated Neurodegeneration, Brain Injury of Prematurity With Periventricular Leukomalacia, Chromosome 17p13.3 Microdeletion Syndrome, Chromosome 1q43-1q44 Deletion, Cockayne Syndrome, Congenital Diaphragmatic Hernia, End-Stage Renal Disease With Cloacal Anomaly, Mitochondrial Depletion Disorder, Severe Factor VII Deficiency
Interventions: Family Centered pediatric palliative care for family caregivers of children with rare diseases.; Behavioral
Lead sponsor: Children's National Research Institute; Other
Eligibility: 12 Months to 99 Years
Enrollment: 480 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2025 – 2029
U.S. locations: 1
States / cities: Washington D.C., District of Columbia

Conditions: Cystic Fibrosis
Interventions: ELX/TEZ/IVA, IVA; Drug
Lead sponsor: Vertex Pharmaceuticals Incorporated; Industry
Eligibility: 12 Years and older
Enrollment: 251 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2022
U.S. locations: 33
States / cities: Tucson, Arizona • Long Beach, California • Palo Alto, California + 28 more

Conditions: Colic, Gastrointestinal Tract Infections
Interventions: L. reuteri, Sunflower Oil; Biological
Lead sponsor: The University of Texas Health Science Center, Houston; Other
Eligibility: 18 Years to 60 Years
Enrollment: 40 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2009 – 2011
U.S. locations: 1
States / cities: Houston, Texas

Conditions: Hepatoblastoma, Hepatocellular Carcinoma, Wilms Tumor, Malignant Rhabdoid Tumor, Yolk Sac Tumor, Rhabdomyosarcoma, Liposarcoma, Embryonal Sarcoma of Liver
Interventions: 21.15.GPC3-CAR T cells; Genetic
Lead sponsor: Baylor College of Medicine; Other
Eligibility: Up to 21 Years
Enrollment: 21 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2043
U.S. locations: 1
States / cities: Houston, Texas

Conditions: Lymphatic Diseases, Lymphatic Abnormalities
Interventions: Not listed
Lead sponsor: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD); NIH
Eligibility: 1 Day to 100 Years
Enrollment: 1,200 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2023 – 2028
U.S. locations: 2
States / cities: Bethesda, Maryland • Philadelphia, Pennsylvania

Conditions: Esophageal Atresia With Tracheo-esophageal Fistula, Esophageal Atresia, Esophageal Strictures
Interventions: Endoscopic Balloon Dilation; Procedure
Lead sponsor: Boston Children's Hospital; Other
Eligibility: Up to 18 Years
Enrollment: 128 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2028
U.S. locations: 2
States / cities: Boston, Massachusetts • Philadelphia, Pennsylvania

Conditions: Cystic Fibrosis
Interventions: VX-809, Placebo, VX-770; Drug
Lead sponsor: Vertex Pharmaceuticals Incorporated; Industry
Eligibility: 6 Years to 11 Years
Enrollment: 206 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2016
U.S. locations: 29
States / cities: Birmingham, Alabama • Los Angeles, California • Palo Alto, California + 26 more

Conditions: Colitis, HIV Infections
Interventions: Ganciclovir; Drug
Lead sponsor: Hoffmann-La Roche; Industry
Eligibility: 21 Years and older · Male only
Healthy volunteers: Healthy volunteers not accepted
U.S. locations: 1
States / cities: Palo Alto, California

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

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Cystic Fibrosis and Urinary Incontinence

Acoustic Waveform Respiratory Evaluation

Gastrointestinal Microbiome Influence on the Development of Bronchopulmonary Dysplasia

Study of Human Plasma-Derived Alpha1-Proteinase Inhibitor in Subjects With New-Onset Type 1 Diabetes Mellitus

Open-Label Phase III Long-Term Safety Trial of Liprotamase

Gabapentin Compared to Standard of Care in Preventing Mucositis in Patients With Stage III-IV Head and Neck Cancer Undergoing Primary or Adjuvant Chemoradiation Therapy

Feasibility of a Goal-based Agenda Setting Intervention

The Sinonasal Cavity as a Reservoir for Upper Airway Bacterial Development

A Study Evaluating the Long-term Safety of VX-445 Combination Therapy

Curcumin in Treating Patients With Familial Adenomatous Polyposis

(Study: Vertex IIS) Does Ivacaftor Alter Wild Type CFTR-Open Probability In The Sweat Gland Secretory Coil?

Hereditary Colorectal and Associated Tumor Registry Study

Cystic Fibrosis Reproductive and Sexual Health Collaborative: Building Online Research Partnerships

Endoscopic Mucosal Resection (EMR) for Diagnosis of Hirschsprung's Disease

Vitamin D and Microbiota in Cystic Fibrosis

Palliative Care Needs of Children With Rare Diseases and Their Families

Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy

Safety of Lactobacillus Reuteri (L. Reuteri) in Healthy Adults

Immunotherapy For Adults With GPC3-Positive Solid Tumors Using IL-15 and IL-21 Armored GPC3-CAR T Cells

A Prospective Natural History Study of Lymphatic Anomalies

Balloon Inflation Time for Esophageal Strictures (BITES): A Randomized Multi-Center Study

A Study to Evaluate the Efficacy and Safety of Lumacaftor in Combination With Ivacaftor in Subjects With CF, Homozygous for the F508del-CFTR Mutation

A Study of Ganciclovir in the Treatment of Cytomegalovirus of the Large Intestine in Patients With AIDS

Safety and Efficacy of Pembrolizumab (MK-3475) Plus Binimetinib Alone or Pembrolizumab Plus Chemotherapy With or Without Binimetinib in Metastatic Colorectal Cancer (mCRC) Participants (MK-3475-651/KEYNOTE-651)