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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
A Study of TYRA-300 in Children With Achondroplasia: BEACH301Bone Diseases, Developmental, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dwarfism, achondroplasia, bone disorder, hereditary disease, musculoskeletal system disorder, osteochondrodysplasiaRecruitingPhase 22026-10-01NCT06842355
Modality Choice Before and After Failure for the Treatment of Painful Diabetic NeuropathyDiabetes Complications, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Urogenital Diseases, diabetes mellitus, diabetic kidney disease, diabetic neuropathy, endocrine system disorder, kidney disorder, nervous system disorder, neuromuscular disease, peripheral nervous system disorder, urinary system disorderNot yet recruitingPhase 42026-09-28NCT07795593
Long-Term Extension Study of Vosoritide to Treat Children With HypochondroplasiahypochondroplasiaEnrolling by invitationPhase 32026-09-09NCT07073014
Open-Label, Long-Term, Extension Study of Infigratinib in Children With HypochondroplasiaBone Diseases, Developmental, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dwarfism, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, bone disorder, connective tissue disorder, cutaneous mucinosis, endocrine system disorder, hereditary disease, hypochondroplasia, inborn carbohydrate metabolic disorder, inborn errors of metabolism, lysosomal storage disease, metabolic disease, mucopolysaccharidosis, mucopolysaccharidosis type 4, musculoskeletal system disorder, osteochondrodysplasiaEnrolling by invitationPhase 22026-08-26NCT07393373
An Interventional Study of Infigratinib in Children With HypochondroplasiaBone Diseases, Developmental, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dwarfism, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, bone disorder, connective tissue disorder, cutaneous mucinosis, endocrine system disorder, hereditary disease, hypochondroplasia, inborn carbohydrate metabolic disorder, inborn errors of metabolism, lysosomal storage disease, metabolic disease, mucopolysaccharidosis, mucopolysaccharidosis type 4, musculoskeletal system disorder, osteochondrodysplasiaEnrolling by invitationPhase 2 / Phase 32026-07-16NCT06873035
Prospective Clinical Assessment Study in Children With HypochondroplasiaBone Diseases, Developmental, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dwarfism, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, bone disorder, connective tissue disorder, cutaneous mucinosis, endocrine system disorder, hereditary disease, hypochondroplasia, inborn carbohydrate metabolic disorder, inborn errors of metabolism, lysosomal storage disease, metabolic disease, mucopolysaccharidosis, mucopolysaccharidosis type 4, musculoskeletal system disorder, osteochondrodysplasiaRecruitingNot stated2026-07-16NCT06410976
An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With AchondroplasiaBone Diseases, Developmental, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dwarfism, achondroplasia, bone disorder, endocrine system disorder, hereditary disease, musculoskeletal system disorder, osteochondrodysplasiaActive, not recruitingPhase 32026-03-13NCT03424018
The MIRROR Registry: Minimally Invasive IntRaceRebral HemORrhage EvacuationSupratentorial HemorrhageRecruitingNot stated2026-02-06NCT04494295
Development of Novel Psychological Assessment Tools and Anxiety Intervention for PhenylketonuriaBrain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, hereditary disease, inborn disorder of amino acid metabolism, inborn errors of metabolism, metabolic disease, nervous system disorder, phenylketonuriaRecruitingNot applicable2025-12-23NCT06560736