Testing effectiveness (Phase 2)Ended earlyNCT00638820What this trial is testingReduced Intensity AlloTransplant For OsteopetrosisWho this might be right forOsteopetrosis Masonic Cancer Center, University of Minnesota 3
Large-scale testing (Phase 3)Study completedNCT01275066What this trial is testingStudy to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)Who this might be right forMPS IV A BioMarin Pharmaceutical 177
Not applicableStudy completedNCT00318097What this trial is testingHistamine Responsiveness in McCune-Albright SyndromeWho this might be right forMcCune-Albright Syndrome Children's Mercy Hospital Kansas City 22
Post-approval studies (Phase 4)Study completedNCT02303873What this trial is testingEfficacy and Safety of Alendronate in Chinese Children or Adolescents With Osteogenesis ImperfectaWho this might be right forOsteogenesis Imperfecta Peking Union Medical College Hospital 99
Not applicableLooking for participantsNCT07287241What this trial is testingProspective Observational Cohort Study of Cardiac Structure and Function in Children and Adults With Osteogenesis ImperfectaWho this might be right forOsteogenesis Imperfecta (OI)Osteogenesis Imperfecta Istituto Ortopedico Rizzoli 40
Not applicableLooking for participantsNCT05031507What this trial is testingStudy of Skeletal DisordersWho this might be right forSkeletal Disorders Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 100
Not applicableStudy completedNCT05406544What this trial is testingDf-Life : Quality of Life in Patients With Fibrous DysplasiaWho this might be right forFibrous Dysplasia Hospices Civils de Lyon 149
Not applicableStudy completedNCT04035811What this trial is testingProspective Clinical Assessment Study in Children With Achondroplasia (ACH)Who this might be right forAchondroplasia QED Therapeutics, a BridgeBio company 271
Testing effectiveness (Phase 2)Active Not RecruitingNCT02724228What this trial is testingEvaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)Who this might be right forAchondroplasia BioMarin Pharmaceutical 30
Not applicableNot Yet RecruitingNCT05258019What this trial is testingSite Preservation After Tooth ExtractionWho this might be right forOsteogenesis ImperfectaTotal Absence of Permanent TeethTooth Socket Nanfang Hospital, Southern Medical University 88
Not applicableLooking for participantsNCT03333200What this trial is testingLongitudinal Study of Neurodegenerative DisordersWho this might be right forMLDKrabbe DiseaseALD+24 more University of Pittsburgh 1,500
Not applicableUnknownNCT04624789What this trial is testingRegistry GangliosidosesWho this might be right forGangliosidosesGM1 GangliosidosisMorquio B Disease+6 more SphinCS Lyso Gemeinnutzige UG (Haftungsbeschrankt) 40
Not applicableStudy completedNCT00056784What this trial is testingThe Role of Connective Tissue Growth Factor in the Development of Kidney Disease After Organ TransplantationWho this might be right forKidney Transplantation National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) 134
Not applicableLooking for participantsNCT05858606What this trial is testingMultidisciplinary Evaluation and a Genome-wide Analysis in a Cohort of Idiopathic Short Stature PatientsWho this might be right forIdiopathic Short Stature University Hospital, Montpellier 200
Post-approval studies (Phase 4)Looking for participantsNCT05966064What this trial is testingDEnosumab for the Treatment of FIbrous Dysplasia/McCune-Albright Syndrome in Adults (DeFiD)Who this might be right forFibrous DysplasiaMcCune Albright Syndrome Natasha Appelman-Dijkstra 82
Not applicableStudy completedNCT07230821What this trial is testingRadicle GI Health RDH™: A Study Assessing the Impact of Health and Wellness Products on Gastrointestinal (GI) Health and Related Health OutcomesWho this might be right forGas and BloatingDiarrheaConstipation Radicle Science 500
Not applicableLooking for participantsNCT02504853What this trial is testingNatural History and Genetics of Food Allergy and Related ConditionsWho this might be right forFood AllergyLoeys-Dietz SyndromeAtopic Dermatitis+1 more National Institute of Allergy and Infectious Diseases (NIAID) 1,800
Not applicableLooking for participantsNCT06595407What this trial is testingLoss of Y Chromosome in Aortic StenosisWho this might be right forAortic Stenosis University of Virginia 200
Not applicableStudy completedNCT03872713What this trial is testingEstablishment of Human Cellular Disease Models for Morquio DiseaseWho this might be right forMorquio Disease CENTOGENE GmbH Rostock 40
Not applicableEnrolling By InvitationNCT03655223What this trial is testingEarly Check: Expanded Screening in NewbornsWho this might be right forSpinal Muscular AtrophyFragile X SyndromeFragile X - Premutation+182 more RTI International 30,000