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Conditions: Biliary Atresia
Interventions: Granulocyte Colony-Stimulating Factor; Drug
Lead sponsor: Holterman, Ai-Xuan, M.D.; Individual
Eligibility: 2 Weeks to 180 Days
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2020
U.S. locations: 1
States / cities: Washington D.C., District of Columbia

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: Abnormal Vitreous Humor Morphology, Abnormal Choroid Morphology, Optic Nerve Diseases, Retinal Disease, Retinal Hemorrhage
Interventions: Direct ophthalmoscope; Diagnostic Test
Lead sponsor: Children's National Research Institute; Other
Eligibility: Up to 18 Years
Enrollment: 84 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2020 – 2023
U.S. locations: 1
States / cities: Washington D.C., District of Columbia

Conditions: Viral Infections, Bone Marrow Transplant Infection
Interventions: Virus Specific T cells (VSTs); Biological
Lead sponsor: Children's National Research Institute; Other
Eligibility: Not listed
Enrollment: 32 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2026
U.S. locations: 1
States / cities: Washington D.C., District of Columbia

Conditions: Venipuncture
Interventions: Lavender Aromatherapy, Jojoba Aromatherapy; Other
Lead sponsor: Children's National Research Institute; Other
Eligibility: 7 Years to 11 Years
Enrollment: 21 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2015 – 2016
U.S. locations: 1
States / cities: Washington D.C., District of Columbia

Conditions: Muscular Dystrophy, Facioscapulohumeral
Interventions: Not listed
Lead sponsor: University of Kansas Medical Center; Other
Eligibility: 5 Years to 17 Years
Enrollment: 80 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2028
U.S. locations: 6
States / cities: Palo Alto, California • Iowa City, Iowa • Kansas City, Kansas + 3 more

Conditions: Congenital Heart Disease (CHD)
Interventions: BM-MSC; Biological
Lead sponsor: Catherine Bollard; Other
Eligibility: Up to 6 Months
Enrollment: 17 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2026
U.S. locations: 1
States / cities: Washington D.C., District of Columbia

Conditions: Amyotrophic Lateral Sclerosis, Frontotemporal Dementia, Primary Lateral Sclerosis, Hereditary Spastic Paraplegia, Progressive Muscular Atrophy, Multisystem Proteinopathy
Interventions: Not listed
Lead sponsor: University of Miami; Other
Eligibility: Not listed
Enrollment: 708 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2015 – 2031
U.S. locations: 13
States / cities: Palo Alto, California • San Diego, California • San Francisco, California + 10 more

Conditions: Craniofacial Microsomia, Hemifacial Microsomia, Oculo-Auriculo-Vertebral-Syndrome, Goldenhar Syndrome, Microtia
Interventions: Not listed
Lead sponsor: Seattle Children's Hospital; Other
Eligibility: 12 Months to 24 Months
Enrollment: 417 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2019
U.S. locations: 5
States / cities: Los Angeles, California • Chicago, Illinois • Chapel Hill, North Carolina + 2 more

Conditions: Analgesics, Antipyretics and Anti-Inflammatory Drugs Causing Adverse Effects in Therapeutic Use
Interventions: Not listed
Lead sponsor: Julia Finkel; Other
Eligibility: 7 Years to 21 Years
Enrollment: 40 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2026
U.S. locations: 1
States / cities: Washington D.C., District of Columbia

Conditions: Sickle Cell Disease
Interventions: Interview; Other
Lead sponsor: St. Jude Children's Research Hospital; Other
Eligibility: 8 Years and older
Enrollment: 145 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2023 – 2027
U.S. locations: 1
States / cities: Memphis, Tennessee

Conditions: All Malignancies, Children Being Treated for Catastrophic Illness
Interventions: Not listed
Lead sponsor: St. Jude Children's Research Hospital; Other
Eligibility: Not listed
Enrollment: 8,800 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 1998 – 2034
U.S. locations: 1
States / cities: Memphis, Tennessee

Conditions: GM2 Gangliosidoses, Tay-Sachs, Sandhoff Disease
Interventions: Zavesca (Miglustat); Drug
Lead sponsor: Children's National Research Institute; Other
Eligibility: 6 Months to 5 Years
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2004 – 2007
U.S. locations: 1
States / cities: Washington D.C., District of Columbia

Conditions: Cardiovascular Disease, Acute Rejection of Cardiac Transplant, Cardiac Transplant Rejection, Heart Transplant Failure and Rejection
Interventions: Blood Draw of up to 10 ml; Diagnostic Test
Lead sponsor: Medical College of Wisconsin; Other
Eligibility: Not listed
Enrollment: 540 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2020
U.S. locations: 8
States / cities: Little Rock, Arkansas • Atlanta, Georgia • Chicago, Illinois + 4 more

Conditions: Insulin Resistance, Obesity
Interventions: Aerobic exercise; Procedure
Lead sponsor: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD); NIH
Eligibility: 14 Years to 18 Years
Enrollment: 40 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2005 – 2009
U.S. locations: 1
States / cities: Washington D.C., District of Columbia

Conditions: Osteogenesis Imperfecta
Interventions: Not listed
Lead sponsor: Baylor College of Medicine; Other
Eligibility: Not listed
Enrollment: 1,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2031
U.S. locations: 11
States / cities: Phoenix, Arizona • Los Angeles, California • Wilmington, Delaware + 8 more

Conditions: Pediatric Crohn's Disease
Interventions: Methotrexate, Sugar pill (placebo); Drug · Other
Lead sponsor: University of North Carolina, Chapel Hill; Other
Eligibility: Up to 20 Years
Enrollment: 306 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2022
U.S. locations: 32
States / cities: Birmingham, Alabama • Alto, California • New Haven, Connecticut + 27 more

Conditions: Cancer
Interventions: Remote Telegenetic Counseling by Phone, Remote Telegenetic Counseling by Videoconferencing, Usual Care Arm; Behavioral
Lead sponsor: University of Chicago; Other
Eligibility: 18 Years and older
Enrollment: 511 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2021 – 2025
U.S. locations: 3
States / cities: Chicago, Illinois • Philadelphia, Pennsylvania • Memphis, Tennessee

Conditions: Dilated Cardiomyopathy, Hypertrophic Cardiomyopathy, Restrictive Cardiomyopathy
Interventions: Not listed
Lead sponsor: Wayne State University; Other
Eligibility: Not listed
Enrollment: 544 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2018
U.S. locations: 11
States / cities: Aurora, Colorado • Miami, Florida • Chicago, Illinois + 8 more

Conditions: Lower Urinary Tract Symptoms
Interventions: Lactobacillus rhamnosus GG; Drug
Lead sponsor: Medstar Health Research Institute; Other
Eligibility: 18 Years and older
Enrollment: 96 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2018
U.S. locations: 2
States / cities: Washington D.C., District of Columbia

Conditions: Acute Renal Failure
Interventions: Not listed
Lead sponsor: Asha Moudgil; Other
Eligibility: 1 Year to 21 Years
Enrollment: 29 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2019
U.S. locations: 1
States / cities: Washington D.C., District of Columbia

Conditions: Propionic Acidemia, Methylmalonic Acidemia
Interventions: N-carbamylglutamate, Standard of Care; Drug · Other
Lead sponsor: Mendel Tuchman; Other
Eligibility: 1 Hour to 4 Weeks
Enrollment: 1 participant
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2015
U.S. locations: 7
States / cities: Los Angeles, California • Palo Alto, California • Aurora, Colorado + 4 more

Conditions: Pediatric Multiple Sclerosis
Interventions: Not listed
Lead sponsor: University of California, San Francisco; Other
Eligibility: 3 Years to 21 Years
Enrollment: 1,276 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2011 – 2018
U.S. locations: 17
States / cities: Birmingham, Alabama • San Bernardino, California • San Francisco, California + 13 more

Conditions: Urea Cycle Disorder, Ornithine Transcarbamylase Deficiency, Citrullinemia 1, ARGI Deficiency, ASL Deficiency, Argininosuccinic Aciduria, ASS Deficiency, Hyperargininemia, Carbamyl Phosphate Synthetase Deficiency, NAGS Deficiency
Interventions: Not listed
Lead sponsor: Baylor College of Medicine; Other
Eligibility: Not listed
Enrollment: 70 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2026
U.S. locations: 2
States / cities: Washington D.C., District of Columbia • Houston, Texas

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GCSF Adjunct Therapy for Biliary Atresia

Palliative Care Needs of Children With Rare Diseases and Their Families

Accuracy of the Red Reflex Test in the Pediatric Population

T-Lymphocytes for Prevention or Treatment of Viral Infections Following Hematopoietic Stem Cell Transplantation

The Effects of Aromatherapy on the Incidence and Severity of Acute Pain

Motor Outcomes to Validate Evaluations in Pediatric FSHD (MOVE Peds)

Mesenchymal Stromal Cells for Infants With Congenital Heart Disease (MedCaP)

Phenotype, Genotype & Biomarkers in ALS and Related Disorders

Craniofacial Microsomia: Longitudinal Outcomes in Children Pre-Kindergarten (CLOCK)

Developing a Method to Objectively Measure Opioid Analgesia

Gene Therapy Communication: Use of a Needs Assessment to Drive Decision-AIDS for Gene Therapy for Rare Diseases (GENETX)

Pharmacogenetic Determinants Of Treatment Response In Children

Pharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset Gangliosidosis: Single and Steady State Oral Doses

Quantitative Detection of Circulating Donor-Specific DNA in Organ Transplant Recipients (DTRT-Multi-Center Study)

An Exercise Intervention Study in Insulin-Resistant Adolescents

BBD Longitudinal Study of Osteogenesis Imperfecta

Low Dose Oral Methotrexate in Pediatric Crohn's Disease Patients Initiating Anti-Tumor Necrosis Factor (Anti-TNF) Therapy

Engaging and Activating Cancer Survivors in Genetic Services Study

Genotype-Phenotype Associations in Pediatric Cardiomyopathy (PCM GENES)

Impact of Probiotics on Urinary Symptoms in Spinal Cord Injury SCI and SB

Carnitine, Aclycarnitine, Myocardial Function, and CRRT

Long-term Outcome of N-Carbamylglutamate Treatment in Propionic Acidemia and Methylmalonic Acidemia

Environmental and Genetic Risk Factors for Pediatric Multiple Sclerosis

Hepatic Histopathology in Urea Cycle Disorders