- 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 156
matching trials from the live ClinicalTrials.gov search.
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- Conditions
- Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Chronic Myeloid Leukemia, Myelodysplastic Syndrome, Lymphomas, Bone Marrow Failure, Hemoglobinopathy, Immune Deficiency, Osteopetrosis, Cytopenias, Leukocyte Disorders, Anemia Due to Intrinsic Red Cell Abnormality
- Interventions
- CD34+ enriched, T Cell Depleted donor stem cell product
- Biological
- Lead sponsor
- Christopher Dvorak
- Other
- Eligibility
- 2 Months to 30 Years
- Healthy volunteers
- Healthy volunteers not accepted
- U.S. locations
- 1
- States / cities
- San Francisco, California
- Conditions
- Exostoses, Multiple Hereditary
- Interventions
- Palovarotene 2.5 mg, Palovarotene 5.0 mg, Placebo
- Drug · Other
- Lead sponsor
- Clementia Pharmaceuticals Inc.
- Industry
- Eligibility
- 2 Years to 14 Years
- Enrollment
- 193 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2020
- U.S. locations
- 13
- States / cities
- Los Angeles, California • Sacramento, California • San Francisco, California + 9 more
- Conditions
- Osteogenesis Imperfecta
- Interventions
- SAR439459, Placebo
- Drug
- Lead sponsor
- Sanofi
- Industry
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 16 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2024
- U.S. locations
- 7
- States / cities
- Los Angeles, California • New Haven, Connecticut • Indianapolis, Indiana + 4 more
- Conditions
- McCune Albright Syndrome, Polyostotic Fibrous Dysplasia, Precocious Puberty
- Interventions
- Letrozole
- Drug
- Lead sponsor
- National Institute of Dental and Craniofacial Research (NIDCR)
- NIH
- Eligibility
- 1 Year to 8 Years · Female only
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2000 – 2009
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- 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
- Conditions
- Connective Tissue Disorders, Osteogenesis Imperfecta, Bone Diseases, Metabolic, Melorheostosis
- Interventions
- Not listed
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- NIH
- Eligibility
- Up to 70 Years
- Enrollment
- 180 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- Started 2004
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- 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
- Conditions
- Polyostotic Fibrous Dysplasia
- Interventions
- Not listed
- Lead sponsor
- National Institute of Dental and Craniofacial Research (NIDCR)
- NIH
- Eligibility
- Not listed
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1996 – 2008
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Autosomal Dominant Osteopetrosis Type 2
- Interventions
- ACTIMMUNE
- Drug
- Lead sponsor
- Indiana University
- Other
- Eligibility
- 3 Years to 65 Years
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2019
- U.S. locations
- 1
- States / cities
- Indianapolis, Indiana
- 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
- Osteogenesis Imperfecta
- Interventions
- Zoledronic Acid
- Drug
- Lead sponsor
- Novartis Pharmaceuticals
- Industry
- Eligibility
- 1 Year to 17 Years
- Enrollment
- 127 participants
- Timeline
- 2004 – 2007
- U.S. locations
- 10
- States / cities
- Los Angeles, California • Wilmington, Delaware • Boise, Idaho + 7 more
- Conditions
- Advanced Solid Tumor, Refractory Hodgkin Lymphoma
- Interventions
- HCB301
- Drug
- Lead sponsor
- FBD Biologics Limited
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2027
- U.S. locations
- 1
- States / cities
- Greenville, South Carolina
- 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
- Osteopetrosis
- Interventions
- Stem Cell or Umbilical Cord Blood Transplantation, Campath, Busulfan, Clofarabine, Total Lymphoid Irradiation
- Procedure · Drug
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Other
- Eligibility
- Up to 45 Years
- Enrollment
- 3 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2008
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Locally Advanced or Metastatic Cancers, Metastatic Human Papillomavirus-Related Malignant Neoplasm
- Interventions
- TST005
- Drug
- Lead sponsor
- Suzhou Transcenta Therapeutics Co., Ltd.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 19 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2023
- U.S. locations
- 3
- States / cities
- Canton, Ohio • Dallas, Texas • San Antonio, Texas
- Conditions
- Osteogenesis Imperfecta
- Interventions
- Not listed
- Lead sponsor
- Baylor College of Medicine
- Other
- Eligibility
- 10 Years to 100 Years
- Enrollment
- 75 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2027
- U.S. locations
- 2
- States / cities
- Los Angeles, California • Houston, Texas
- 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
- Osteogenesis Imperfecta (OI)
- Interventions
- AGA2115, Placebo
- Drug · Other
- Lead sponsor
- Angitia Incorporated Limited
- Industry
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 80 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2029
- U.S. locations
- 9
- States / cities
- Phoenix, Arizona • New Haven, Connecticut • Wilmington, Delaware + 6 more
- 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
- Conditions
- Solid Tumor
- Interventions
- LY3200882, LY3300054, Gemcitabine, nab-Paclitaxel, Cisplatin, Intensity Modulated Radiotherapy
- Drug · Radiation
- Lead sponsor
- Eli Lilly and Company
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 223 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2027
- U.S. locations
- 3
- States / cities
- Louisville, Kentucky • New York, New York • Houston, Texas
- 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