- Conditions
- Genetic Disorder
- Interventions
- Not listed
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- NIH
- Eligibility
- 18 Years to 99 Years
- Enrollment
- 11 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 2014
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
Search Results
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Showing 25–48
of 24
matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
- Conditions
- Huntington Disease
- Interventions
- Pridopidine, Placebo
- Drug
- Lead sponsor
- Prilenia
- Industry
- Eligibility
- 25 Years and older
- Enrollment
- 499 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2024
- U.S. locations
- 26
- States / cities
- Davis, California • San Diego, California • Englewood, Colorado + 22 more
- Conditions
- Sickle Cell Disease, Sickle Cell Hemoglobin C, Sickle Beta Zero Thalassemia, Sickle B+ Thalassemia
- Interventions
- PROs assessment using sickle cell disease mobile app (SCD-app)
- Behavioral
- Lead sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
- Other
- Eligibility
- 12 Years to 25 Years
- Enrollment
- 40 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2019
- U.S. locations
- 1
- States / cities
- Chicago, Illinois
- Conditions
- Adrenal Hyperplasia, Congenital
- Interventions
- Not listed
- Lead sponsor
- Office of Rare Diseases (ORD)
- NIH
- Eligibility
- 12 Years and older
- Enrollment
- 233 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2008 – 2009
- U.S. locations
- 2
- States / cities
- New York, New York • Dallas, Texas
- Conditions
- Protein C Deficiency
- Interventions
- Protein C Concentrate (Human) Vapor Heated
- Drug
- Lead sponsor
- Baxalta now part of Shire
- Industry
- Eligibility
- Not listed
- Enrollment
- 11 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005
- U.S. locations
- 10
- States / cities
- Los Angeles, California • Denver, Colorado • Fort Carson, Colorado + 7 more
- Conditions
- Gout
- Interventions
- Not listed
- Lead sponsor
- Arthritis & Rheumatism Associates, P.C.
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2017
- U.S. locations
- 1
- States / cities
- Wheaton, Maryland
- Conditions
- NOMID, DIRA, NLRC4-MAS, SAVI, CANDLE
- Interventions
- Not listed
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
- NIH
- Eligibility
- Up to 99 Years
- Enrollment
- 3,200 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2016 – 2032
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Congenital Muscular Dystrophy With ITGA7 (Integrin Alpha-7) Deficiency, Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy and Abnormal Glycosylation of Dystroglycan With Severe Epilepsy), Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy With Fatty Liver and Infantile-onset Cataract Caused by TRAPPC11 Mutations), Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy With Hypoglycosylation of Dystroglycan), Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy With Hypoglycosylation of Dystroglycan and Epilepsy), Alpha-Dystroglycanopathy (Dystroglycanopathy, Congenital With or Without Mental Retardation (Formerly MDC1C)), Alpha-Dystroglycanopathy (Fukuyama CMD), Alpha-Dystroglycanopathy (LGMDR09 FKRP Related (Formerly LGMD2I)), Alpha-Dystroglycanopathy (LGMDR11 POMT1 Related (Formerly LGMD2K)), Alpha-Dystroglycanopathy (LGMDR13 FKTN Related (Formerly LGMD2M)), Alpha-Dystroglycanopathy (LGMDR14 POMT2 Related (Formerly LGMD2N)), Alpha-Dystroglycanopathy (LGMDR15 POMGnT1 Related (Formerly LGMD2O)), Alpha-Dystroglycanopathy (LGMDR19 GMPPB Related (Formerly LGMD2T)), Alpha-Dystroglycanopathy (LGMDR20 ISPD Related (Formerly LGMD2U)), Alpha-Dystroglycanopathy (LGMDR24 POMGnT2 Related), Alpha-Dystroglycanopathy (Muscle Eye Brain Disease (MEB)), Alpha-Dystroglycanopathy (Walker Warburg Syndrome (WWS)), Choline Kinase B Receptor - CHKB, Collagen VI Related Disorders, Collagen XII Related Disorders, Congenital Muscular Dystrophy Not Otherwise Specified (Including Merosin Positive), Congenital Muscular Dystrophy With Cataracts and Intellectual Disability (MDCCAID), Congenital Muscular Dystrophy With Joint Hyperlaxity, Congenital Muscular Dystrophy With Rigid Spine Related to ACTA1, Emery-Dreifuss Muscular Dystrophy, GOLGA2-related Congenital Muscle Dystrophy With Brain Involvement, LMNA Related Disorders, Merosin Deficient CMD (Full or Partial), Nesprin Related MD (SYNE1), SELENON Related Disorders (Previously Known as SEPN1), SELENON Related Myopathy (Aka SEPN1), Telethonin CMD, Congenital Myasthenic Syndrome, Limb-Girdle Muscular Dystrophy, LGMDD01 - DNAJB6 (Formerly LGMD1D), LGMDD05 - Collagen VI Related Bethlem Myopathy (Dominant), LGMDR07 - Telethonin (TCAP) Related (Formerly LGMD2G), LGMDR08 - TRIM Related (Formerly LGMD2H), LGMDR09 - FKRP Related (Formerly LGMD2I), LGMDR10 - Titin (TTN) Related (Formerly LGMD2J), LGMDR11 - POMT1 Related (Formerly LGMD2K), LGMDR13 - Fukutin (FKTN) Related (Formerly LGMD2M), LGMDR14 - POMT2 Related (Formerly LGMD2N), LGMDR15 - POMGnT1 Related (Formerly LGMD2O), LGMDR16 - DAG1 Related Dystroglycanopathy (Formerly LGMD2P), LGMDR17 - Plectin (PLEC) Related (Formerly LGMD2Q), LGMDR18 - TRAPPC11 Related (Formerly LGMD2S), LGMDR19 - GMPPB Related (Formerly LGMD2T), LGMDR20 - ISPD Related (Formerly LGMD2U), LGMDR22 - Collagen VI Related Bethlem Myopathy (Recessive), LGMDR23 - LAMA2 Related, LGMDR24 - POMGnT2 Related
- Interventions
- Not listed
- Lead sponsor
- Cure CMD
- Other
- Eligibility
- Not listed
- Enrollment
- 4,000 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2029
- U.S. locations
- 1
- States / cities
- Lakewood, California
- Conditions
- Osteogenesis Imperfecta
- Interventions
- Manual cuff blood pressure
- Procedure
- Lead sponsor
- Nemours Children's Clinic
- Other
- Eligibility
- 1 Year to 35 Years
- Enrollment
- 50 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2019 – 2025
- U.S. locations
- 1
- States / cities
- Wilmington, Delaware
- Conditions
- Hypoxic-Ischemic Encephalopathy, Neonatal Encephalopathy, Encephalopathy
- Interventions
- Visual Evoked Potential (VEP), Electroretinogram (ERG)
- Device
- Lead sponsor
- University of Wisconsin, Madison
- Other
- Eligibility
- Up to 36 Months
- Enrollment
- 125 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2020 – 2029
- U.S. locations
- 1
- States / cities
- Madison, Wisconsin
- Conditions
- Friedreich Ataxia, Rare Diseases
- Interventions
- Geneticlly confirmed disease causing FXN mutatuion, Healthy Control
- Other
- Lead sponsor
- Friedreich's Ataxia Research Alliance
- Other
- Eligibility
- 4 Years to 21 Years
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2025 – 2028
- U.S. locations
- 3
- States / cities
- Iowa City, Iowa • Philadelphia, Pennsylvania • Memphis, Tennessee
- Conditions
- Arthrogryposis, Craniofacial Abnormalities, Posttraumatic Stress Disorder, Depressive Disorder
- Interventions
- PTSD Checklist-Specific, Modified Flanagan Quality of Life Scale, Center for Epidemiologic Studies Depression Scale, Functional Enquiry Form, Strength, Joint ROM, Girth and Length Measurements, Study Physical Examination, PTSD, Depression, and FSS-Focused Examination, Freeman-Sheldon Specific Quality of Life Survey, Lactate, Glucose, and Adenosine Triphosphate Blood Levels
- Other
- Lead sponsor
- Freeman-Sheldon Research Group, Inc.
- Other
- Eligibility
- Not listed
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2017
- U.S. locations
- 1
- States / cities
- Buckhannon, West Virginia
- Conditions
- Lymphatic Malformation, Generalized Lymphatic Anomaly (GLA), Central Conducting Lymphatic Anomaly, CLOVES Syndrome, Gorham-Stout Disease ("Disappearing Bone Disease"), Blue Rubber Bleb Nevus Syndrome, Kaposiform Lymphangiomatosis, Kaposiform Hemangioendothelioma/Tufted Angioma, Klippel-Trenaunay Syndrome, Lymphangiomatosis
- Interventions
- Not listed
- Lead sponsor
- Boston Children's Hospital
- Other
- Eligibility
- Not listed
- Enrollment
- 1,000 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2035
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Cortical Dysplasia
- Interventions
- Classical ketogenic diet
- Dietary Supplement
- Lead sponsor
- University College, London
- Other
- Eligibility
- 3 Years to 15 Years
- Enrollment
- 3 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2017
- U.S. locations
- 1
- States / cities
- Baltimore, Maryland
- Conditions
- Low Birth Weight Neonates
- Interventions
- Not listed
- Lead sponsor
- Icahn School of Medicine at Mount Sinai
- Other
- Eligibility
- Not listed
- Enrollment
- 81 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2013
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Neurofibromatosis 1, Plexiform Neurofibromas
- Interventions
- Not listed
- Lead sponsor
- National Cancer Institute (NCI)
- NIH
- Eligibility
- 5 Years and older
- Enrollment
- 476 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2015 – 2027
- U.S. locations
- 6
- States / cities
- Washington D.C., District of Columbia • Chicago, Illinois • Baltimore, Maryland + 3 more
- Conditions
- Pregnancy Complications, Pregnancy Outcome, Congenital Abnormalities
- Interventions
- Observational Intervention
- Biological
- Lead sponsor
- Emergent BioSolutions
- Industry
- Eligibility
- Female only
- Enrollment
- 98 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2012 – 2020
- U.S. locations
- 1
- States / cities
- San Diego, California
- Conditions
- Leukemia
- Interventions
- flow cytometry, immunologic technique, laboratory biomarker analysis
- Other
- Lead sponsor
- Alliance for Clinical Trials in Oncology
- Other
- Eligibility
- 15 Years and older
- Enrollment
- 451 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2004 – 2014
- U.S. locations
- 2
- States / cities
- Kinston, North Carolina • Columbus, Ohio
- 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
- Neurodegeneration With Brain Iron Accumulation (NBIA)
- Interventions
- Not listed
- Lead sponsor
- Susan J. Hayflick
- Other
- Eligibility
- Not listed
- Enrollment
- 300 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2025
- U.S. locations
- 1
- States / cities
- Portland, Oregon
- Conditions
- Pompe Disease
- Interventions
- Not listed
- Lead sponsor
- O & O Alpan LLC
- Other
- Eligibility
- 18 Years and older · Female only
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2016
- U.S. locations
- 1
- States / cities
- Fairfax, Virginia
- Conditions
- Down Syndrome
- Interventions
- Body weight supported harness
- Behavioral
- Lead sponsor
- Boston University Charles River Campus
- Other
- Eligibility
- Up to 2 Years
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2025
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Congenital Heart Disease
- Interventions
- Not listed
- Lead sponsor
- Icahn School of Medicine at Mount Sinai
- Other
- Eligibility
- 1 Day to 2 Months
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2011
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Neonatal Seizures
- Interventions
- phenobarbital, placebo
- Drug
- Lead sponsor
- University of Rochester
- Other
- Eligibility
- 2 Days to 2 Weeks
- Enrollment
- 13 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2014
- U.S. locations
- 18
- States / cities
- Little Rock, Arkansas • San Francisco, California • Iowa City, Iowa + 12 more