- Conditions
- Achondroplasia
- Interventions
- BMN 111, Placebo
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 5 Years to 18 Years
- Enrollment
- 121 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2019
- U.S. locations
- 11
- States / cities
- Oakland, California • Torrance, California • Wilmington, Delaware + 8 more
Search Results
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Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results are retrieved from ClinicalTrials.gov and synchronized into the directory. Search pages remain noindex by default.
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Showing 1–24
of 28
matching trials from the live ClinicalTrials.gov search.
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- Conditions
- Achondroplasia
- Interventions
- BMN 333, Vosoritide Injection [Voxzogo]
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 2 Years to 17 Years
- Enrollment
- 160 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2029
- U.S. locations
- 9
- States / cities
- Oakland, California • Wilmington, Delaware • Chicago, Illinois + 6 more
- 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
- Conditions
- Achondroplasia
- Interventions
- Recifercept
- Biological
- Lead sponsor
- Pfizer
- Industry
- Eligibility
- 3 Months to 10 Years
- Enrollment
- 60 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2023
- U.S. locations
- 6
- States / cities
- Aliso Viejo, California • Irvine, California • Long Beach, California + 3 more
- Conditions
- Achondroplasia
- Interventions
- Not listed
- Lead sponsor
- QED Therapeutics, a BridgeBio company
- Industry
- Eligibility
- 30 Months to 17 Years
- Enrollment
- 271 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2025
- U.S. locations
- 8
- States / cities
- Oakland, California • Aurora, Colorado • Wilmington, Delaware + 5 more
- Conditions
- Syringomyelia, Type I Arnold Chiari Malformation
- Interventions
- Not listed
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
- NIH
- Eligibility
- 1 Year and older
- Enrollment
- 152 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 2001
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Achondroplasia
- Interventions
- BMN 111
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 6 Years and older
- Enrollment
- 119 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2031
- U.S. locations
- 11
- States / cities
- Oakland, California • Torrance, California • Wilmington, Delaware + 8 more
- 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
- 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
- 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
- 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
- Conditions
- Developmental Bone Disease, Dwarfism, Skeletal Dysplasias
- Interventions
- Not listed
- Lead sponsor
- National Human Genome Research Institute (NHGRI)
- NIH
- Eligibility
- Not listed
- Enrollment
- 600 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1998 – 2001
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- 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
- 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
- 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
- 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
- Conditions
- Fibrous Dysplasia
- Interventions
- denosumab
- Drug
- Lead sponsor
- National Institute of Dental and Craniofacial Research (NIDCR)
- NIH
- Eligibility
- 4 Years to 14 Years
- Enrollment
- 15 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2026
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- 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
- 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
- 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
- Conditions
- Tumor Induced Osteomalcia, Osteomalacia, Familial Tumoral Calcinosis
- Interventions
- Not listed
- Lead sponsor
- National Institute of Dental and Craniofacial Research (NIDCR)
- NIH
- Eligibility
- 1 Day to 100 Years
- Enrollment
- 1,000 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 2001
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Hypochondroplasia
- Interventions
- Infigratinib
- Drug
- Lead sponsor
- QED Therapeutics, a BridgeBio company
- Industry
- Eligibility
- 3 Years to 18 Years
- Enrollment
- 135 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2036
- U.S. locations
- 8
- States / cities
- Oakland, California • Aurora, Colorado • Washington D.C., District of Columbia + 5 more
- 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
- Conditions
- Rheumatoid Arthritis, Ankylosing Spondylitis, Fibromyalgia, Gout, Crohn Disease, Juvenile Idiopathic Arthritis, Lupus Erythematosus, Myositis, Osteoarthritis, Osteoporosis, Psoriasis, Psoriatic Arthritis, Scleroderma, Dermatomyositis, Inflammatory Bowel Diseases, Polymyositis, Axial Spondyloarthritis, Diffuse Idiopathic Skeletal Hyperostosis, Polymyalgia Rheumatica, Giant Cell Arteritis, Temporal Arteritis, Wegener, Relapsing Polychondritis, Undifferentiated Connective Tissue Disease, Spinal Cord Injuries, Alzheimer Disease, Amyotrophic Lateral Sclerosis, Ataxia, Bell Palsy, Brain Tumor, Cerebral Aneurysm, Epilepsy, Guillain-Barre Syndrome, Headache, Head Injury, Hydrocephalus, Lumbar Disc Disease, Meningitis, Multiple Sclerosis, Muscular Dystrophy, Neurocutaneous Syndromes, Parkinson Disease, Stroke, Cluster Headache, Tension-Type Headache, Chronic Obstructive Pulmonary Disease, Asthma, Lung Cancer, Cystic Fibrosis, Sleep Apnea, Eczema, Alopecia, Chronic Inflammation, Unstable Angina, Heart Attack, Heart Failure, Arrythmia, Valve Heart Disease, High Blood Pressure, Congenital Heart Disease, Peripheral Arterial Disease, Diabetes, Chronic Liver Disease, Obesity
- Interventions
- Not listed
- Lead sponsor
- Global Healthy Living Foundation
- Other
- Eligibility
- 19 Years and older
- Enrollment
- 40,000 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2015 – 2025
- U.S. locations
- 1
- States / cities
- Upper Nyack, New York