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ClinicalTrials.gov public records Last synced May 22, 2026, 12:11 AM 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 155 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: Skeletal Dysplasia Clear all
Terminated Phase 2 Interventional Results available
View directory record Official record
Conditions
Urothelial Cancer
Interventions
Bintrafusp alfa (M7824)
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 21, 2022 · Synced May 22, 2026, 12:11 AM 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 22, 2026, 12:11 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Food Allergy, Loeys-Dietz Syndrome, Atopic Dermatitis, Eosinophilic Esophagitis
Interventions
Not listed
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
1 Day to 99 Years
Enrollment
1,800 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2026
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated May 19, 2026 · Synced May 22, 2026, 12:11 AM EDT
Completed Phase 1Phase 2 Interventional
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia, Anemia, Granuloma, Wiskott-Aldrich Syndrome, Chediak Higashi Syndrome, Osteopetrosis, Neutropenia, Thrombocytopenia, Hurler Disease, Niemann-Pick Disease, Fucosidosis
Interventions
Hematopoietic stem cell transplantation
Procedure
Lead sponsor
Children's Hospital Los Angeles
Other
Eligibility
Up to 21 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2015
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jun 22, 2016 · Synced May 22, 2026, 12:11 AM EDT
Completed Phase 2 Interventional Results available
View directory record Official record
Conditions
Fibrosis, Focal Glomerulosclerosis, Kidney Failure, Nephrotic Syndrome, Proteinuria
Interventions
Pirfenidone
Drug
Lead sponsor
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
NIH
Eligibility
18 Years and older
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2008
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated May 25, 2014 · Synced May 22, 2026, 12:11 AM 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 22, 2026, 12:11 AM 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 22, 2026, 12:11 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Aortic Stenosis
Interventions
Blood analysis for loss-of-Y chromosome
Diagnostic Test
Lead sponsor
University of Virginia
Other
Eligibility
40 Years and older · Male only
Enrollment
200 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024 – 2026
U.S. locations
1
States / cities
Charlottesville, Virginia
Source: ClinicalTrials.gov public record
Updated Sep 18, 2024 · Synced May 22, 2026, 12:11 AM EDT
Terminated Phase 3 Interventional Results available
View directory record Official record
Conditions
Osteogenesis Imperfecta (OI)
Interventions
Denosumab, Alternative osteoporosis medications
Drug
Lead sponsor
Amgen
Industry
Eligibility
Not listed
Enrollment
75 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
2
States / cities
Los Angeles, California • Indianapolis, Indiana
Source: ClinicalTrials.gov public record
Updated Dec 19, 2022 · Synced May 22, 2026, 12:11 AM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions
Interventions
data collection
Drug
Lead sponsor
Paul Szabolcs
Other
Eligibility
2 Months to 60 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2028
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 12, 2026 · Synced May 22, 2026, 12:11 AM 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 22, 2026, 12:11 AM EDT
Completed Phase 1 Interventional Results available
View directory record Official record
Conditions
Osteogenesis Imperfecta
Interventions
Romosozumab, Calcium, Vitamin D
Drug · Dietary Supplement
Lead sponsor
Amgen
Industry
Eligibility
5 Years to 17 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
3
States / cities
Indianapolis, Indiana • Nashville, Tennessee • Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Apr 14, 2024 · Synced May 22, 2026, 12:11 AM 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 22, 2026, 12:11 AM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Achondroplasia, Dwarfism
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
Not listed
Enrollment
2,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1996 – 2000
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced May 22, 2026, 12:11 AM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Achondroplasia
Interventions
Not listed
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
0 Months to 18 Years
Enrollment
170 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
7
States / cities
Oakland, California • San Francisco, California • Statesville, North Carolina + 4 more
Source: ClinicalTrials.gov public record
Updated Dec 17, 2025 · Synced May 22, 2026, 12:11 AM EDT
Active, not recruiting Phase 2 Interventional
View directory record Official record
Conditions
Mucopolysaccharidosis Disorders, Hurler Syndrome, Hunter Syndrome, Maroteaux Lamy Syndrome, Sly Syndrome, Alpha-Mannosidosis, Fucosidosis, Aspartylglucosaminuria, Glycoprotein Metabolic Disorders, Sphingolipidoses, Recessive Leukodystrophies, Globoid Cell Leukodystrophy, Metachromatic Leukodystrophy, Niemann-Pick B, Niemann-Pick C Subtype 2, Sphingomyelin Deficiency, Peroxisomal Disorders, Adrenoleukodystrophy With Cerebral Involvement, Zellweger Syndrome, Neonatal Adrenoleukodystrophy, Infantile Refsum Disease, Acyl-CoA Oxidase Deficiency, D-Bifunctional Enzyme Deficiency, Multifunctional Enzyme Deficiency, Alpha-methylacyl-CoA Racmase Deficiency, Mitochondrial Neurogastrointestingal Encephalopathy, Severe Osteopetrosis, Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation), Inherited Metabolic Disorders
Interventions
Stem Cell Transplantation, IMD Preparative Regimen, Osteopetrosis Only Preparative Regimen, Osteopetrosis Haploidentical Only Preparative Regimen, cALD SR-A (Standard-Risk, Regimen A), cALD SR-B (Standard-Risk, Regimen B), cALD HR-D (High-Risk, Regimen C), cALD HR-D (High-Risk, Regimen D)
Biological · Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 55 Years
Enrollment
149 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2029
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jan 6, 2026 · Synced May 22, 2026, 12:11 AM EDT
Active, not recruiting Phase 2Phase 3 Interventional
View directory record Official record
Conditions
Osteogenesis Imperfecta
Interventions
Setrusumab, Placebo
Biological · Other
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
5 Years to 25 Years
Enrollment
183 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
23
States / cities
Little Rock, Arkansas • Los Angeles, California • Sacramento, California + 20 more
Source: ClinicalTrials.gov public record
Updated Feb 24, 2026 · Synced May 22, 2026, 12:11 AM EDT
Active, not recruiting Phase 1 Interventional
View directory record Official record
Conditions
EBV-positive Nasopharyngeal Carcinoma
Interventions
DNR.NPC-specific T cells, DNR.NPC-specific T cells + cyclophosphamide + fludarabine
Biological
Lead sponsor
Baylor College of Medicine
Other
Eligibility
Not listed
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2033
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jan 28, 2026 · Synced May 22, 2026, 12:11 AM EDT
Completed Phase 1 Interventional
View directory record Official record
Conditions
Breast Cancer, Breast Neoplasms
Interventions
fenretinide
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
Not listed
Enrollment
75 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1994 – 2000
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced May 22, 2026, 12:11 AM EDT
Terminated Phase 1Phase 2 Interventional Results available
View directory record Official record
Conditions
Hip Osteoarthritis, Hip Impingement Syndrome, Cartilage Damage, Fibrosis
Interventions
Losartan, Placebo
Drug · Other
Lead sponsor
Steadman Philippon Research Institute
Other
Eligibility
18 Years to 60 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020
U.S. locations
1
States / cities
Vail, Colorado
Source: ClinicalTrials.gov public record
Updated Oct 29, 2023 · Synced May 22, 2026, 12:11 AM EDT
Completed Phase 2 Interventional Results available
View directory record Official record
Conditions
Achondroplasia
Interventions
BMN 111
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
5 Years to 14 Years
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
6
States / cities
Oakland, California • Torrance, California • Chicago, Illinois + 3 more
Source: ClinicalTrials.gov public record
Updated Jan 14, 2021 · Synced May 22, 2026, 12:11 AM EDT
Not listed No phase listed Observational
View directory record Official record
Conditions
Rhizomelic Chondrodysplasia Punctata
Interventions
Not listed
Lead sponsor
MED-LIFE DISCOVERIES LP
Industry
Eligibility
6 Months to 21 Years
Enrollment
75 participants
Timeline
2019 – 2021
U.S. locations
1
States / cities
Wilmington, Delaware
Source: ClinicalTrials.gov public record
Updated Aug 1, 2019 · Synced May 22, 2026, 12:11 AM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Rheumatic Disease
Interventions
Not listed
Lead sponsor
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
NIH
Eligibility
18 Years to 90 Years
Enrollment
350 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2030
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated May 6, 2026 · Synced May 22, 2026, 12:11 AM EDT
Recruiting Phase 3 Interventional
View directory record Official record
Conditions
Osteogenesis Imperfecta
Interventions
Romosozumab, Bisphosphonate
Drug
Lead sponsor
Amgen
Industry
Eligibility
5 Years to 17 Years
Enrollment
106 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
14
States / cities
Birmingham, Alabama • Phoenix, Arizona • Los Angeles, California + 11 more
Source: ClinicalTrials.gov public record
Updated Apr 30, 2026 · Synced May 22, 2026, 12:11 AM EDT