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ClinicalTrials.gov public records Last synced Sep 8, 2026, 8:28 AM EDT

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Showing 1–24 of 125 matching trials from the live ClinicalTrials.gov search.
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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
Nervous System Malformations, Arthropathy, Neurogenic, Urticaria, Papilledema
Interventions
anakinra
Drug
Lead sponsor
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
NIH
Eligibility
Not listed
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2010
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Congenital Diaphragmatic Hernia, Congenital Abnormalities, Neonatal Diseases and Abnormalities, Hernia, DIaphragmatic, Congenital, Internal Hernia, Hernia, Pathological Conditions, Anatomical, Pathological Conditions, Signs and Symptoms
Interventions
Fetal Treatment Arm (FETO Group)
Device
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years and older · Female only
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2031
U.S. locations
1
States / cities
Baltimore, Maryland
Conditions
Atopic Dermatitis
Interventions
Crisaborole 2% Topical Application Ointment [EUCRISA], Triamcinolone Acetonide 0.1% Ointment
Drug
Lead sponsor
Clinical Research Center of the Carolinas
Other
Eligibility
2 Years to 79 Years
Enrollment
16 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2020
U.S. locations
1
States / cities
Charleston, South Carolina
Conditions
Preterm Less Than 32wks With IVH, WMI/PVL, Late Preterm or Term (37-42wks) With Neonatal Encephalopathy Treated With Hypothermia for HIE
Interventions
Prospective Clinical and Neurodevelopmental Data Collection
Other
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
31 Weeks to 42 Weeks
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
1
States / cities
Boston, Massachusetts
Conditions
Congestive Heart Failure
Interventions
carvedilol, placebo
Drug
Lead sponsor
Shaddy, Robert, M.D.
Individual
Eligibility
1 Day to 17 Years
Enrollment
161 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2006
U.S. locations
21
States / cities
Birmingham, Alabama • Los Angeles, California • Palo Alto, California + 16 more
Conditions
Neonatal Abstinence Syndrome
Interventions
Structured psychosocial intervention
Behavioral
Lead sponsor
Dr. Henry Akinbi
Other
Eligibility
Female only
Enrollment
113 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013
U.S. locations
1
States / cities
Cincinnati, Ohio
Completed No phase listed Observational Accepts healthy volunteers

Osteogenesis Imperfecta Blood Pressure Study

NCT04169568
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
Exocrine Pancreatic Insufficiency, Cystic Fibrosis
Interventions
Liprotamase, porcine PERT
Drug
Lead sponsor
Anthera Pharmaceuticals
Industry
Eligibility
7 Years and older
Enrollment
140 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2018
U.S. locations
28
States / cities
Little Rock, Arkansas • Long Beach, California • Los Angeles, California + 24 more
Completed No phase listed Observational

FuRST 2.0 Cognitive Pre-Testing

NCT02881931
Conditions
Huntington's Disease
Interventions
FuRST 2.0
Behavioral
Lead sponsor
CHDI Foundation, Inc.
Other
Eligibility
18 Years and older
Enrollment
75 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017
U.S. locations
3
States / cities
Englewood, Colorado • Wichita, Kansas • New York, New York
Conditions
Propionic Acidemia, Type I and/or Type II, Methylmalonic Acidemia, Carbamoyl-Phosphate Synthase I Deficiency Disease, Ornithine Carbamoyltransferase Deficiency
Interventions
Carbaglu, Placebo
Drug
Lead sponsor
Mendel Tuchman
Other
Eligibility
1 Week to 99 Years
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2020
U.S. locations
9
States / cities
Los Angeles, California • Palo Alto, California • Aurora, Colorado + 6 more
Completed No phase listed Observational

GAS-Hem Feasibility Study

NCT02674997
Conditions
Hemophilia A
Interventions
Factor VIII
Biological
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
5 Years to 65 Years
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016
U.S. locations
2
States / cities
Peoria, Illinois • Portland, Oregon
Conditions
Labor, Premature
Interventions
Magnesium, Nifedlipine
Drug
Lead sponsor
MemorialCare
Other
Eligibility
18 Years to 55 Years · Female only
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2012
U.S. locations
1
States / cities
Long Beach, California
Conditions
Hyperbilirubinemia, Neonatal, Jaundice, Neonatal, Infant, Newborn, Infant, Low Birth Weight, Infant, Small for Gestational Age, Infant, Premature
Interventions
Aggressive Phototherapy 501-750g, Aggressive Phototherapy 751-1000g, Conservative Phototherapy 501-750g, Conservative Phototherapy 751-1000g
Procedure
Lead sponsor
NICHD Neonatal Research Network
Network
Eligibility
12 Hours to 36 Hours
Enrollment
1,974 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2007
U.S. locations
17
States / cities
Birmingham, Alabama • Palo Alto, California • San Diego, California + 13 more
Conditions
Neonatal Congenital Heart Disease
Interventions
Not listed
Lead sponsor
Emory University
Other
Eligibility
Up to 30 Days
Enrollment
92 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2016
U.S. locations
1
States / cities
Atlanta, Georgia
Conditions
Retinopathy of Prematurity (ROP)
Interventions
rhIGF-1/rhIGFBP-3
Drug
Lead sponsor
Shire
Industry
Eligibility
40 Weeks to 108 Weeks
Enrollment
76 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2021
U.S. locations
4
States / cities
Mobile, Alabama • Greenville, North Carolina • Oklahoma City, Oklahoma + 1 more
Conditions
Hunter Syndrome
Interventions
Idursulfase
Biological
Lead sponsor
Shire
Industry
Eligibility
5 Years and older · Male only
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2013
U.S. locations
2
States / cities
Oakland, California • Minneapolis, Minnesota
Completed Not applicable Interventional Accepts healthy volunteers Results available

Absorptive Clearance in the Cystic Fibrosis Airway

NCT00541190
Conditions
Cystic Fibrosis
Interventions
Technetium [Tc-99m] sulfur colloid and Indium [In-111] DTPA
Other
Lead sponsor
University of Pittsburgh
Other
Eligibility
18 Years and older
Enrollment
21 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2007 – 2008
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
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
Cleft Palate
Interventions
Not listed
Lead sponsor
University of California, Davis
Other
Eligibility
1 Year to 10 Years
Enrollment
88 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2015
U.S. locations
1
States / cities
Sacramento, California
Completed No phase listed Observational

Hunter Outcome Survey (HOS)

NCT03292887
Conditions
Hunter Syndrome
Interventions
Not listed
Lead sponsor
Shire
Industry
Eligibility
Not listed
Enrollment
1,443 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2023
U.S. locations
1
States / cities
Lexington, Massachusetts
Completed Phase 2 Interventional Results available

Androgen Effect on Klinefelter Syndrome Motor Outcome

NCT00348946
Conditions
Klinefelter Syndrome
Interventions
androgen oxandrolone, placebo
Drug · Other
Lead sponsor
Thomas Jefferson University
Other
Eligibility
4 Years to 12 Years · Male only
Enrollment
93 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2017
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Active, not recruiting No phase listed Observational

Database of Monochorionic Pregnancies

NCT05543499
Conditions
Monochorionic Diamniotic Placenta, Monochorial Monoamniotic Placenta, Twin; Complicating Pregnancy, Twin to Twin Transfusion Syndrome, Twin Reversal Arterial Perfusion Syndrome
Interventions
Not listed
Lead sponsor
University of Miami
Other
Eligibility
Not listed
Enrollment
1,200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
1
States / cities
Miami, Florida
Conditions
Duchenne Muscular Dystrophy (DMD), Cardiomyopathy, Becker Muscular Dystrophy, Carrier of Duchenne Muscular Dystrophy
Interventions
Not listed
Lead sponsor
Vanderbilt University Medical Center
Other
Eligibility
Not listed
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2029
U.S. locations
9
States / cities
Sacramento, California • Washington D.C., District of Columbia • Chicago, Illinois + 6 more