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ClinicalTrials.gov public records Last synced Sep 30, 2026, 7:03 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 189 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Osteochondrodysplasia Clear all
Recruiting No phase listed Observational

Screening and Natural History of Patients With Polyostotic Fibrous Dysplasia and the McCune-Albright Syndrome

NCT00001727
View directory record Official record
Conditions
McCune-Albright Syndrome
Interventions
Not listed
Lead sponsor
National Institute of Dental and Craniofacial Research (NIDCR)
NIH
Eligibility
1 Day to 100 Years
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1998
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 28, 2026 · Synced Sep 30, 2026, 7:03 AM EDT
Not listed Not applicable Interventional

Evaluation of Skeletal Maturity for Slipped Capital Femoral Epiphysis

NCT01220882
View directory record Official record
Conditions
Slipped Capital Femoral Epiphysis
Interventions
Radiographic skeletal age assessment
Radiation
Lead sponsor
Rutgers, The State University of New Jersey
Other
Eligibility
8 Years to 16 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2015
U.S. locations
1
States / cities
Newark, New Jersey
Source: ClinicalTrials.gov public record
Updated Dec 25, 2013 · Synced Sep 30, 2026, 7:03 AM EDT
Terminated Phase 1 Interventional Accepts healthy volunteers

Bioequivalence Study to Compare Two Injection Devices for BMN 111 in Healthy Participants

NCT05813314
View directory record Official record
Conditions
Achondroplasia
Interventions
BMN 111 administration via Injector Pen, BMN 111 administration via vial and syringe
Combination Product · Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
18 Years to 55 Years
Enrollment
36 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Oct 12, 2023 · Synced Sep 30, 2026, 7:03 AM EDT
Recruiting Phase 1 Interventional

PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

NCT04532047
View directory record Official record
Conditions
MPS I, MPS II, MPS IVA, MPS VI, Mps VII, Gaucher Disease, Type 2, Gaucher Disease, Type 3, Pompe Disease Infantile-Onset, Wolman Disease
Interventions
Aldurazyme (laronidase)
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
18 Years to 50 Years · Female only
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2032
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Mar 17, 2026 · Synced Sep 30, 2026, 7:03 AM EDT
Completed Not applicable Interventional Results available

North Carolina Genomic Evaluation by Next-generation Exome Sequencing, 2

NCT03548779
View directory record Official record
Conditions
Epilepsy; Seizure, Neuromuscular Diseases, Brain Malformation, Intellectual Disability, Autism Spectrum Disorder, Hypotonia, Inborn Errors of Metabolism, Movement Disorders, Genetic Disease, Development Delay, Chromosome Abnormality, Hearing Loss, Dysmorphic Features, Skeletal Dysplasia, Congenital Abnormality, Microcephaly, Macrocephaly
Interventions
Pre-visit prep, usual care + exome seq
Behavioral · Diagnostic Test
Lead sponsor
University of North Carolina, Chapel Hill
Other
Eligibility
0 Years and older
Enrollment
548 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2024
U.S. locations
3
States / cities
Asheville, North Carolina • Chapel Hill, North Carolina • Greenville, North Carolina
Source: ClinicalTrials.gov public record
Updated May 13, 2025 · Synced Sep 30, 2026, 7:03 AM EDT
Recruiting Phase 4 Interventional Accepts healthy volunteers

Effects of Bisphosphonates on OI-Related Hearing Loss

NCT04152551
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 28, 2026 · Synced Sep 30, 2026, 7:03 AM EDT
Recruiting Phase 2 Interventional

Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia (ACH)

NCT07169279
View directory record Official record
Conditions
Achondroplasia
Interventions
Infigratinib is provided as a single dose of minitablets for oral administration, Infigratinib is provided as sprinkle capsules for daily oral administration, Infigratinib or placebo comparator is provided as sprinkle capsules for daily oral administration
Drug
Lead sponsor
QED Therapeutics, a BridgeBio company
Industry
Eligibility
0 Years to 32 Months
Enrollment
77 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
3
States / cities
Oakland, California • Baltimore, Maryland • Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated Jul 13, 2026 · Synced Sep 30, 2026, 7:03 AM EDT
Completed Phase 2Phase 3 Interventional Results available

Allogeneic Transplantation For Severe Osteopetrosis

NCT00775931
View directory record Official record
Conditions
Severe Osteopetrosis
Interventions
umbilical cord blood transplantation, Campath-1H, Total Lymphoid Irradiation, Cyclophosphamide, Busulfan, Fludarabine monophosphate, marrow graft transplantation
Procedure · Drug · Radiation
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
1 Day to 45 Years
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2013
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jul 31, 2019 · Synced Sep 30, 2026, 7:03 AM EDT
Recruiting Phase 2 Interventional

A Study Evaluating Ficerafusp Alfa (BCA101) QW in Combination With Pembrolizumab vs Alternative Ficerafusp Alfa Dosing in 1L R or M HNSCC

NCT07807163
View directory record Official record
Conditions
Metastatic Head and Neck Squamous Cell Carcinoma, Recurrent Head and Neck Squamous Cell Carcinoma
Interventions
Ficerafusp alfa, Pembrolizumab (KEYTRUDA®)
Drug
Lead sponsor
Bicara Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
160 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
1
States / cities
Tyler, Texas
Source: ClinicalTrials.gov public record
Updated Sep 8, 2026 · Synced Sep 30, 2026, 7:03 AM EDT
Completed Not applicable Interventional Results available

Prevention of Post Operative Bone Loss in Children

NCT00655681
View directory record Official record
Conditions
Osteoporosis, Cerebral Palsy, Spina Bifida, Osteopenia, Osteogenesis Imperfecta
Interventions
pamidronate, saline
Drug · Other
Lead sponsor
University of New Mexico
Other
Eligibility
4 Years to 18 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2011
U.S. locations
1
States / cities
Albuquerque, New Mexico
Source: ClinicalTrials.gov public record
Updated Aug 22, 2023 · Synced Sep 30, 2026, 7:03 AM EDT
Terminated Phase 3 Interventional Results available

Multicenter,Single-arm Study to Evaluate Efficacy, Safety, & Pharmacokinetics of Denosumab in Children w/ OI

NCT02352753
View directory record Official record
Conditions
Osteogenesis Imperfecta
Interventions
Denosumab
Drug
Lead sponsor
Amgen
Industry
Eligibility
2 Years to 17 Years
Enrollment
153 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2022
U.S. locations
13
States / cities
Los Angeles, California • Torrance, California • Aurora, Colorado + 10 more
Source: ClinicalTrials.gov public record
Updated Dec 28, 2022 · Synced Sep 30, 2026, 7:03 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Effectiveness of a Butyrate Formulation and Butyrate + Polyphenol Formulation on Gut Health, Permeability and Associated Symptoms

NCT06419231
View directory record Official record
Conditions
Gut Health
Interventions
Butyrate Formulation, Butyrate + Polyphenol Formulation, Placebo
Dietary Supplement
Lead sponsor
Supplement Formulators, Inc.
Industry
Eligibility
21 Years to 70 Years
Enrollment
143 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2025
U.S. locations
1
States / cities
Fort Lauderdale, Florida
Source: ClinicalTrials.gov public record
Updated Jan 13, 2026 · Synced Sep 30, 2026, 7:03 AM EDT
Completed Phase 3 Interventional Results available

Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

NCT01415427
View directory record Official record
Conditions
Mucopolysaccharidosis IV A, Morquio A Syndrome, MPS IVA
Interventions
BMN 110 - Weekly, BMN 110 - Every Other Week
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
5 Years and older
Enrollment
173 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
10
States / cities
Phoenix, Arizona • Oakland, California • Orange, California + 7 more
Source: ClinicalTrials.gov public record
Updated Jul 21, 2021 · Synced Sep 30, 2026, 7:03 AM EDT
Active, not recruiting Phase 2 Interventional

An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia

NCT03989947
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 13, 2026 · Synced Sep 30, 2026, 7:03 AM EDT
Completed Phase 4 Interventional Accepts healthy volunteers Results available

Pregabalin and Colonic Function

NCT01094808
View directory record Official record
Conditions
Healthy
Interventions
Pregabalin, Placebo, Bowel preparation
Drug · Other
Lead sponsor
Mayo Clinic
Other
Eligibility
18 Years to 75 Years
Enrollment
62 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2010 – 2011
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Mar 7, 2012 · Synced Sep 30, 2026, 7:03 AM EDT
Terminated No phase listed Observational

Study to Detect Unrecognized Mucopolysaccharidosis in Children Visiting Rheumatology, Hand or Skeletal Dysplasia Clinics

NCT01675674
View directory record Official record
Conditions
Mucopolysaccharidoses, Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis IV, Mucopolysaccharidosis VI
Interventions
Dried blood spot test for MPS
Other
Lead sponsor
National MPS Society
Other
Eligibility
6 Months to 18 Years
Enrollment
3,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
2
States / cities
New Brunswick, New Jersey • New York, New York
Source: ClinicalTrials.gov public record
Updated May 24, 2013 · Synced Sep 30, 2026, 7:03 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Natural History of Cherubism Observational Study

NCT01916772
View directory record Official record
Conditions
Cherubism
Interventions
no interventions
Other
Lead sponsor
The Cleveland Clinic
Other
Eligibility
Not listed
Enrollment
33 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2016
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Jan 10, 2017 · Synced Sep 30, 2026, 7:03 AM EDT
Completed Not applicable Interventional Results available

A Study to Evaluate the Safety, Tolerability and Effectiveness of the Renew Insert in Treating Accidental Bowel Leakage

NCT01475474
View directory record Official record
Conditions
Bowel Incontinence
Interventions
Renew Insert
Device
Lead sponsor
Renew Medical
Industry
Eligibility
18 Years and older
Enrollment
97 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2011
U.S. locations
3
States / cities
Los Gatos, California • San Diego, California • Weston, Florida
Source: ClinicalTrials.gov public record
Updated Nov 28, 2013 · Synced Sep 30, 2026, 7:03 AM EDT
Active, not recruiting Phase 3 Interventional

Setrusumab vs Bisphosphonates in Pediatric Subjects With Osteogenesis Imperfecta

NCT05768854
View directory record Official record
Conditions
Osteogenesis Imperfecta
Interventions
Bisphosphonate, Setrusumab
Drug · Biological
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
2 Years to 6 Years
Enrollment
69 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
13
States / cities
Phoenix, Arizona • Los Angeles, California • Aurora, Colorado + 10 more
Source: ClinicalTrials.gov public record
Updated Feb 25, 2026 · Synced Sep 30, 2026, 7:03 AM EDT
Enrolling by invitation Phase 2Phase 3 Interventional

An Interventional Study of Infigratinib in Children With Hypochondroplasia

NCT06873035
View directory record Official record
Conditions
Hypochondroplasia
Interventions
infigratinib 0.128 mg/kg/day, infigratinib 0.25 mg/kg/day
Drug
Lead sponsor
QED Therapeutics, a BridgeBio company
Industry
Eligibility
3 Years to 18 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
8
States / cities
Oakland, California • Aurora, Colorado • Washington D.C., District of Columbia + 5 more
Source: ClinicalTrials.gov public record
Updated Jul 16, 2026 · Synced Sep 30, 2026, 7:03 AM EDT
Recruiting No phase listed Observational

Registry of Patients Diagnosed With Lysosomal Storage Diseases

NCT05619900
View directory record Official record
Conditions
Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis IV A, Mucopolysaccharidosis VI, Mucopolysaccharidosis VII, Pompe Disease Infantile-Onset, Neuronopathic Gaucher Disease, Wolman Disease
Interventions
There is no intervention
Other
Lead sponsor
University of California, San Francisco
Other
Eligibility
Up to 64 Years
Enrollment
250 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2050
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Apr 8, 2026 · Synced Sep 30, 2026, 7:03 AM EDT
Recruiting No phase listed Observational

Prospective Clinical Assessment Study in Children With Hypochondroplasia

NCT06410976
View directory record Official record
Conditions
Hypochondroplasia
Interventions
Not listed
Lead sponsor
QED Therapeutics, a BridgeBio company
Industry
Eligibility
30 Months to 16 Years
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
8
States / cities
Oakland, California • Aurora, Colorado • Washington D.C., District of Columbia + 5 more
Source: ClinicalTrials.gov public record
Updated Jul 16, 2026 · Synced Sep 30, 2026, 7:03 AM EDT
Terminated Phase 1 Interventional Results available

PF-06952229 Treatment in Adult Patients With Advanced Solid Tumors

NCT03685591
View directory record Official record
Conditions
Breast Neoplasms, Prostate Neoplasms, Neoplasms, Squamous Cell, Melanoma, Mesothelioma, Pancreatic Neoplasms, Colorectal Neoplasms, Carcinoma, Renal Cell, Liver Neoplasms
Interventions
PF-06952229, Enzalutamide
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older
Enrollment
49 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
15
States / cities
Scottsdale, Arizona • Santa Monica, California • Boston, Massachusetts + 6 more
Source: ClinicalTrials.gov public record
Updated Jun 26, 2024 · Synced Sep 30, 2026, 7:03 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Study of Gene Modified Donor T-cells Following TCR Alpha Beta Positive Depleted Stem Cell Transplant

NCT03301168
View directory record Official record
Conditions
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndromes, Primary Immune Deficiency Disorder, Osteopetrosis, Cytopenia, Hemoglobinopathy in Children, Anemia, Aplastic
Interventions
BPX-501 T cells, Rimiducid
Biological · Drug
Lead sponsor
Bellicum Pharmaceuticals
Industry
Eligibility
1 Month to 26 Years
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2034
U.S. locations
10
States / cities
Los Angeles, California • Palo Alto, California • Washington D.C., District of Columbia + 7 more
Source: ClinicalTrials.gov public record
Updated Jul 12, 2022 · Synced Sep 30, 2026, 7:03 AM EDT