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ClinicalTrials.gov public records Last synced May 21, 2026, 9:56 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 1–24 of 55 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: Skeletal Anomalies Clear all
Completed Phase 3 Interventional Results available
View directory record Official record
Conditions
Hypophosphatemia, Familial, Hyperparathyroidism
Interventions
Paricalcitol, Placebo
Drug · Other
Lead sponsor
Yale University
Other
Eligibility
9 Years and older
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2012
U.S. locations
1
States / cities
New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Mar 16, 2020 · Synced May 21, 2026, 9:56 PM EDT
Completed Phase 3 Interventional
View directory record Official record
Conditions
Achondroplasia
Interventions
Infigratinib 0.25 mg/kg/day, Placebo Comparator 0.25 mg/kg/day
Drug
Lead sponsor
QED Therapeutics, a BridgeBio company
Industry
Eligibility
3 Years to 17 Years
Enrollment
114 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
7
States / cities
San Francisco, California • Aurora, Colorado • Baltimore, Maryland + 4 more
Source: ClinicalTrials.gov public record
Updated Mar 16, 2026 · Synced May 21, 2026, 9:56 PM EDT
Active, not recruiting No phase listed Observational
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 30, 2026 · Synced May 21, 2026, 9:56 PM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Mucopolysaccharidosis IV Type A, Morquio A Syndrome, MPS IVA
Interventions
Imaging, gait analysis, growth, joint test, hearing test, questionnaire, etc.
Diagnostic Test
Lead sponsor
Nemours Children's Clinic
Other
Eligibility
Not listed
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
1
States / cities
Wilmington, Delaware
Source: ClinicalTrials.gov public record
Updated May 12, 2026 · Synced May 21, 2026, 9:56 PM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Enchondromatosis
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
2 Years to 100 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated May 11, 2026 · Synced May 21, 2026, 9:56 PM EDT
Active, not recruiting Phase 2 Interventional
View directory record Official record
Conditions
Achondroplasia
Interventions
Active BMN 111: Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily.
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
15 Months and older
Enrollment
73 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2038
U.S. locations
9
States / cities
Oakland, California • Torrance, California • Wilmington, Delaware + 6 more
Source: ClinicalTrials.gov public record
Updated Mar 12, 2026 · Synced May 21, 2026, 9:56 PM EDT
Completed Phase 2 Interventional
View directory record Official record
Conditions
Achondroplasia
Interventions
Infigratinib 0.016 mg/kg, Infigratinib 0.032 mg/kg, Infigratinib 0.064 mg/kg, Infigratinib 0.128 mg/kg, Infigratinib 0.25 mg/kg
Drug
Lead sponsor
QED Therapeutics, a BridgeBio company
Industry
Eligibility
3 Years to 11 Years
Enrollment
84 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2024
U.S. locations
5
States / cities
Oakland, California • Wilmington, Delaware • Baltimore, Maryland + 2 more
Source: ClinicalTrials.gov public record
Updated Oct 21, 2025 · Synced May 21, 2026, 9:56 PM EDT
Recruiting Phase 4 Interventional
View directory record Official record
Conditions
Musculoskeletal Abnormalities, Obesity, Sarcopenic Obesity, Osteoporosis, Drug Effect
Interventions
Tirzepatide, Lifestyle toolkit
Drug · Behavioral
Lead sponsor
Pennington Biomedical Research Center
Other
Eligibility
18 Years to 50 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
1
States / cities
Baton Rouge, Louisiana
Source: ClinicalTrials.gov public record
Updated Apr 15, 2026 · Synced May 21, 2026, 9:56 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers
View directory record Official record
Conditions
Skeletal Anomalies, Kidney Transplant; Complications
Interventions
measurements to evaluate metabolic bone disease, standard care
Diagnostic Test
Lead sponsor
Yale University
Other
Eligibility
30 Years to 65 Years
Enrollment
8 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2021
U.S. locations
1
States / cities
New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Feb 27, 2023 · Synced May 21, 2026, 9:56 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Adenovirus, Anesthesia, Anxiety, Anxiolysis, Autism, Autistic Disorder, Bacterial Meningitis, Bacterial Septicemia, Benzodiazepine, Bipolar Disorder, Bone and Joint Infections, Central Nervous System Infections, Convulsions, Cytomegalovirus Retinitis, Early-onset Schizophrenia Spectrum Disorders, Epilepsy, General Anesthesia, Gynecologic Infections, Herpes Simplex Virus, Infantile Hemangioma, Infection, Inflammation, Inflammatory Conditions, Intra-abdominal Infections, Lower Respiratory Tract Infections, Migraines, Pain, Pneumonia, Schizophrenia, Sedation, Seizures, Skeletal Muscle Spasms, Skin and Skin-structure Infections, Treatment-resistant Schizophrenia, Urinary Tract Infections, Withdrawal, Sepsis, Gram-negative Infection, Bradycardia, Cardiac Arrest, Cardiac Arrhythmia, Staphylococcal Infections, Nosocomial Pneumonia, Neuromuscular Blockade, Methicillin Resistant Staphylococcus Aureus, Endocarditis, Neutropenia, Headache, Fibrinolytic Bleeding, Pulmonary Arterial Hypertension, CMV Retinitis, Hypertension, Chronic Kidney Diseases, Hyperaldosteronism, Hypokalemia, Heart Failure, Hemophilia, Heavy Menstrual Bleeding, Insomnia
Interventions
The POPS study is collecting PK data on children prescribed the following drugs of interest per standard of care:
Drug
Lead sponsor
Daniel Benjamin
Other
Eligibility
Up to 21 Years
Enrollment
3,520 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2019
U.S. locations
43
States / cities
Anchorage, Alaska • Little Rock, Arkansas • La Jolla, California + 37 more
Source: ClinicalTrials.gov public record
Updated Sep 5, 2023 · Synced May 21, 2026, 9:56 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Cardiomyopathy, Hypertrophic, Muscular Dystrophy, Oculopharyngeal
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
Not listed
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2001
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced May 21, 2026, 9:56 PM EDT
Completed No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Barth Syndrome
Interventions
Not listed
Lead sponsor
Duke University
Other
Eligibility
8 Years to 35 Years · Male only
Enrollment
64 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2020
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Jun 1, 2020 · Synced May 21, 2026, 9:56 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Choreatic Disorder
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
Not listed
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2003
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced May 21, 2026, 9:56 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Myotonic Dystrophy
Interventions
Not listed
Lead sponsor
Wake Forest University Health Sciences
Other
Eligibility
18 Years to 65 Years
Enrollment
75 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2029
U.S. locations
1
States / cities
Winston-Salem, North Carolina
Source: ClinicalTrials.gov public record
Updated Jan 22, 2026 · Synced May 21, 2026, 9:56 PM EDT
Enrolling by invitation Phase 2 Interventional
View directory record Official record
Conditions
Achondroplasia
Interventions
Infigratinib
Drug
Lead sponsor
QED Therapeutics, a BridgeBio company
Industry
Eligibility
3 Years to 18 Years
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2032
U.S. locations
8
States / cities
Oakland, California • Aurora, Colorado • Wilmington, Delaware + 5 more
Source: ClinicalTrials.gov public record
Updated Oct 30, 2025 · Synced May 21, 2026, 9:56 PM EDT
Completed Phase 1Phase 2 Interventional Results available
View directory record Official record
Conditions
Becker Muscular Dystrophy
Interventions
(-)-epicatechin
Drug
Lead sponsor
Craig McDonald, MD
Other
Eligibility
18 Years to 60 Years · Male only
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2018
U.S. locations
1
States / cities
Sacramento, California
Source: ClinicalTrials.gov public record
Updated Dec 21, 2021 · Synced May 21, 2026, 9:56 PM EDT
Terminated Early Phase 1 Interventional Accepts healthy volunteers
View directory record Official record
Conditions
Muscular Dystrophy
Interventions
Tadalafil 20 MG, beetroot juice extract
Drug · Other
Lead sponsor
Cedars-Sinai Medical Center
Other
Eligibility
18 Years to 45 Years · Male only
Enrollment
6 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2018
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jan 13, 2020 · Synced May 21, 2026, 9:56 PM EDT
Recruiting Phase 4 Interventional Accepts healthy volunteers
View directory record Official record
Conditions
Osteogenesis Imperfecta
Interventions
Risedronate Oral Tablet
Drug
Lead sponsor
Hospital for Special Surgery, New York
Other
Eligibility
6 Years to 100 Years
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2027
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Apr 27, 2026 · Synced May 21, 2026, 9:56 PM EDT
Terminated Early Phase 1 Interventional
View directory record Official record
Conditions
Pompe Disease
Interventions
Pyridostigmine Bromide
Drug
Lead sponsor
University of Florida
Other
Eligibility
8 Years to 60 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated May 14, 2018 · Synced May 21, 2026, 9:56 PM EDT
Completed Phase 2 Interventional Results available
View directory record Official record
Conditions
Achondroplasia
Interventions
BMN 111, Placebo
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
Up to 59 Months
Enrollment
75 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
9
States / cities
Oakland, California • Torrance, California • Wilmington, Delaware + 6 more
Source: ClinicalTrials.gov public record
Updated Jun 12, 2024 · Synced May 21, 2026, 9:56 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Skeletal Disorders
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
2 Months to 100 Years
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2027
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Feb 4, 2026 · Synced May 21, 2026, 9:56 PM EDT
Active, not recruiting Phase 3 Interventional
View directory record Official record
Conditions
Hypochondroplasia
Interventions
Vosoritide, Placebo
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
3 Years to 17 Years
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
4
States / cities
Wilmington, Delaware • Washington D.C., District of Columbia • Chicago, Illinois + 1 more
Source: ClinicalTrials.gov public record
Updated Jan 19, 2026 · Synced May 21, 2026, 9:56 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Fibrodysplasia Ossificans Progressiva (FOP)
Interventions
Non-Interventional
Other
Lead sponsor
Regeneron Pharmaceuticals
Industry
Eligibility
Not listed
Enrollment
131 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023
U.S. locations
1
States / cities
Tarrytown, New York
Source: ClinicalTrials.gov public record
Updated Jul 2, 2025 · Synced May 21, 2026, 9:56 PM EDT
Completed Early Phase 1 Interventional Results available
View directory record Official record
Conditions
X-linked Hypophosphatemia, Hypophosphatemic Rickets, Hypophosphatemic Rickets, X-Linked Dominant
Interventions
Calcitriol
Drug
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
3 Years to 70 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2026
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated May 6, 2026 · Synced May 21, 2026, 9:56 PM EDT