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ClinicalTrials.gov public records Last synced Oct 29, 2026, 5:24 AM EDT

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Showing 49–72 of 607 matching trials from the live ClinicalTrials.gov search.
Conditions
Acute Leukemia, Adenomatous Polyposis, Adrenocortical Carcinoma, AML, BAP1 Tumor Predisposition Syndrome, Carney Complex, Choroid Plexus Carcinoma, Constitutional Mismatch Repair Deficiency Syndrome, Diamond-Blackfan Anemia, DICER1 Syndrome, Dyskeratosis Congenita, Emberger Syndrome, Familial Acute Myeloid Leukemia, Familial Adenomatous Polyposis, Fanconi Anemia, Familial Cancer, Familial Wilms Tumor, Familial Neuroblastoma, GIST, Hereditary Breast and Ovarian Cancer, Hereditary Paraganglioma-Pheochromocytoma Syndrome, Hodgkin Lymphoma, Juvenile Polyposis, Li-Fraumeni Syndrome, Lynch Syndrome, MDS, Melanoma Syndrome, Multiple Endocrine Neoplasia Type 1, Multiple Endocrine Neoplasia Type 2, Neuroblastoma, Neurofibromatosis Type 1, Neurofibromatosis Type II, Nevoid Basal Cell Carcinoma Syndrome, Non Hodgkin Lymphoma, Noonan Syndrome and Other Rasopathy, Overgrowth Syndromes, Pancreatic Cancer, Peutz-Jeghers Syndrome, Pheochromocytoma/Paraganglioma, PTEN Hamartoma Tumor Syndrome, Retinoblastoma, Rhabdoid Tumor Predisposition Syndrome, Rhabdomyosarcoma, Rothmund-Thomson Syndrome, Tuberous Sclerosis, Von Hippel-Lindau Disease
Interventions
Not listed
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Not listed
Enrollment
1,500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2037
U.S. locations
1
States / cities
Memphis, Tennessee
Conditions
Urothelial Carcinoma
Interventions
Mocetinostat
Drug
Lead sponsor
Mirati Therapeutics Inc.
Industry
Eligibility
18 Years and older
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
14
States / cities
Birmingham, Alabama • Duarte, California • Fort Myers, Florida + 11 more
Conditions
Fibrous Dysplasia, McCune Albright Syndrome, Mazabraud Syndrome
Interventions
Not listed
Lead sponsor
Tovah Burstein
Other
Eligibility
Not listed
Enrollment
600 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2028
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
Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome, Adult Acute Basophilic Leukemia, Adult Acute Eosinophilic Leukemia, Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0), Adult Acute Monoblastic Leukemia (M5a), Adult Acute Monocytic Leukemia (M5b), Adult Acute Myeloblastic Leukemia With Maturation (M2), Adult Acute Myeloblastic Leukemia Without Maturation (M1), Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(16;16)(p13;q22), Adult Acute Myeloid Leukemia With t(8;21)(q22;q22), Adult Acute Myelomonocytic Leukemia (M4), Adult Erythroleukemia (M6a), Adult Pure Erythroid Leukemia (M6b), Secondary Acute Myeloid Leukemia
Interventions
eltrombopag olamine
Drug
Lead sponsor
Case Comprehensive Cancer Center
Other
Eligibility
60 Years and older
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
1
States / cities
Cleveland, Ohio
Conditions
Osteogenesis Imperfecta, Brittle Bone Disorders
Interventions
Web-based Survey
Other
Lead sponsor
University of South Florida
Other
Eligibility
2 Years and older
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016
U.S. locations
1
States / cities
Tampa, Florida
Conditions
Achondroplasia
Interventions
Navepegritide, Placebo for Navepegritide
Drug
Lead sponsor
Ascendis Pharma A/S
Industry
Eligibility
0 Years to 2 Years
Enrollment
72 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
3
States / cities
Saint Paul, Minnesota • Houston, Texas • Madison, Wisconsin
Conditions
Growth Hormone Deficiency
Interventions
Somatropin
Drug
Lead sponsor
Altus Pharmaceuticals
Industry
Eligibility
3 Years to 13 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2009
U.S. locations
11
States / cities
Little Rock, Arkansas • Orlando, Florida • Springfield, Massachusetts + 7 more
Conditions
Short Stature
Interventions
Norditropin
Drug
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
2 Years and older
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
1
States / cities
Cincinnati, Ohio
Conditions
Leukemia
Interventions
sargramostim, cyclophosphamide, cytarabine, gemtuzumab ozogamicin, liposomal daunorubicin citrate, topotecan hydrochloride
Biological · Drug
Lead sponsor
Eastern Cooperative Oncology Group
Network
Eligibility
18 Years to 120 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2007
U.S. locations
62
States / cities
Scottsdale, Arizona • Duarte, California • Palo Alto, California + 44 more
Conditions
Vertebral Cervical Fusion Syndrome, Degenerative Disc Disease
Interventions
CervicalStim bone growth stimulator
Device
Lead sponsor
Orthofix Inc.
Industry
Eligibility
18 Years and older
Enrollment
211 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
1
States / cities
Johnstown, Colorado
Completed Not applicable Interventional Results available

Prevention of Post Operative Bone Loss in Children

NCT00655681
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
Active, not recruiting Phase 4 Interventional Accepts healthy volunteers

Assessing the Impact of Contraceptives on Bone Health Using 41Ca

NCT02367846
Conditions
Other Disorders of Bone Development and Growth
Interventions
Combined Oral Contraceptive (COC), Contraceptive Vaginal Ring (CVR)
Drug
Lead sponsor
Penn State University
Other
Eligibility
18 Years to 25 Years · Female only
Enrollment
6 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2028
U.S. locations
1
States / cities
University Park, Pennsylvania
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
Basal Cell Nevus Syndrome, Gorlin Syndrome
Interventions
GDC-0449
Drug
Lead sponsor
UCSF Benioff Children's Hospital Oakland
Other
Eligibility
35 Years to 75 Years
Enrollment
41 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2014
U.S. locations
2
States / cities
Oakland, California • New York, New York
Completed No phase listed Observational Accepts healthy volunteers

Natural History of Cherubism Observational Study

NCT01916772
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
Conditions
RCDP - Rhizomelic Chondrodysplasia Punctata, RCDP1, RCDP2, RCDP3, RCDP4, RCDP5
Interventions
Not listed
Lead sponsor
Nemours Children's Clinic
Other
Eligibility
Not listed
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2030
U.S. locations
1
States / cities
Wilmington, Delaware
Completed No phase listed Observational

Child and Infant Learning Project

NCT00077831
Conditions
Craniosynostosis
Interventions
neurobehavioral development
Behavioral
Lead sponsor
Seattle Children's Hospital
Other
Eligibility
2 Months to 7 Years
Enrollment
535 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2015
U.S. locations
4
States / cities
Atlanta, Georgia • Westchester, Illinois • St Louis, Missouri + 1 more
Conditions
Craniosynostosis
Interventions
Epsilon-Aminocaproic Acid
Drug
Lead sponsor
Paul Stricker
Other
Eligibility
2 Months to 24 Months
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2011
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
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
Pituitary Disorders, Adult Growth Hormone Deficiency
Interventions
growth hormone
Drug
Lead sponsor
LG Life Sciences
Industry
Eligibility
23 Years to 70 Years
Enrollment
147 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2008
U.S. locations
1
States / cities
Portland, Oregon
Recruiting Not applicable Interventional Accepts healthy volunteers

Physical Activity and Community EmPOWERment Project

NCT06740162
Conditions
Intellectual Disability, Neurodevelopmental Disorders, Autism Spectrum Disorder, Down Syndrome, Fragile X Syndrome, Cri-du-Chat Syndrome, De Lange Syndrome, Mental Retardation, X-Linked, Prader-Willi Syndrome, Rubinstein-Taybi Syndrome, Trisomy 13 Syndrome, WAGR Syndrome, Williams Syndrome
Interventions
PACE Program
Behavioral
Lead sponsor
University of North Carolina, Chapel Hill
Other
Eligibility
18 Years and older
Enrollment
376 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2028
U.S. locations
2
States / cities
Fayetteville, Arkansas • Chapel Hill, North Carolina
Terminated Phase 2 Interventional Results available

Reduced Intensity AlloTransplant For Osteopetrosis

NCT00638820
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
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