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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
FOG-001 in Locally Advanced or Metastatic Solid TumorsAdenomatous Polyps, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cronkhite-Canada syndrome, Digestive System Diseases, Digestive System Neoplasms, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Genital Diseases, Liver Neoplasms, Male Urogenital Diseases, Neoplasm Metastasis, Neoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Connective and Soft Tissue, Neoplasms, Fibrous Tissue, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Neoplastic Processes, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Urogenital Diseases, Urogenital Neoplasms, adamantinous craniopharyngioma, adenocarcinoma, adenoma, cancer, carcinoma, classic familial adenomatous polyposis, colonic disorder, colorectal cancer, colorectal neoplasm, connective tissue neoplasm, craniopharyngioma, desmoid tumor, digestive system disorder, digestive system neoplasm, endometrial carcinoma, endometrium neoplasm, epithelial neoplasm, female reproductive system disorder, female reproductive system neoplasm, fibroma, hepatocellular carcinoma, hereditary disease, hereditary neoplastic syndrome, intestinal disorder, liver disorder, male reproductive organ cancer, male reproductive system disorder, metastatic malignant neoplasm, neoplasm, primitive neuroectodermal tumor, prostate cancer, prostate disorder, rectal disorder, uterine disorderRecruitingPhase 1 / Phase 22026-10-07NCT05919264
MEK Inhibitor Mirdametinib (PD-0325901) in Patients With Neurofibromatosis Type 1 Associated Plexiform NeurofibromasCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Neoplasms by Histologic Type, Neoplasms, Nerve Tissue, hereditary disease, hereditary neoplastic syndrome, inherited neurodegenerative disorder, neoplasm, nerve sheath neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neurofibroma, neurofibromatosis, neurofibromatosis type 1, neuromuscular disease, peripheral nervous system disorder, peripheral nervous system neoplasm, plexiform neurofibromaActive, not recruitingPhase 22026-10-06NCT03962543
Screening for Prostate Cancer Using High Resolution Micro-ultrasound Versus Multiparametric Magnetic Resonance Imaging.Prostate Cancer ScreeningRecruitingPhase 32026-09-25NCT06626022
Suzetrigine for Opioid-Sparing Postoperative Analgesia Following Transvaginal Pelvic Reconstructive SurgeryFemale Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Lower Urinary Tract Symptoms, Male Urogenital Diseases, Neurologic Manifestations, Pain, Pathologic Processes, Pathological Conditions, Anatomical, Pathological Conditions, Signs and Symptoms, Postoperative Complications, Postoperative Pain, Prolapse, Signs and Symptoms, Urinary Incontinence, Urinary Incontinence, Stress, Urination Disorders, Urogenital Diseases, Urological Manifestations, pelvic organ prolapse, urinary system disorderNot yet recruitingPhase 42026-09-15NCT07600697
Safety and Efficacy Study of GEB-101 Injection in Subjects With TGFBI-related Corneal DystrophyAmyloid Neuropathies, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Hereditary, Nutritional and Metabolic Diseases, Thiel-Behnke corneal dystrophy, amyloidosis, corneal disorder, corneal dystrophy, eye disorder, familial amyloid neuropathy, hereditary amyloidosis, hereditary disease, inborn errors of metabolism, inherited neurodegenerative disorder, metabolic disease, nervous system disorder, neurodegenerative disease, neuromuscular disease, peripheral nervous system disorder, proteostasis deficienciesNot yet recruitingPhase 1 / Phase 22026-09-10NCT07750678
Long-term Extension of GTX-102 in Angelman SyndromeAbnormalities, Multiple, Angelman syndrome, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Imprinting Disorders, central nervous system disorder, chromosomal disorder, hereditary disease, movement disorder, nervous system disorderActive, not recruitingPhase 32026-09-09NCT06415344
Gene Therapy for Adenosine Deaminase Severe Combined Immune Deficiency Using Peripheral Blood and EFS ADA Vectorsevere combined immunodeficiency, autosomal recessive, T cell-negative, B cell-negative, NK cell-negative, due to adenosine deaminase deficiencyActive, not recruitingPhase 1 / Phase 22026-08-20NCT05432310
Global Open-Label Extension Study of Del-desiran for the Treatment of DM1Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Disorders, Atrophic, Myotonia, Neurologic Manifestations, Neuromuscular Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, hereditary disease, inherited neurodegenerative disorder, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, myotonic dystrophy, myotonic dystrophy type 1, myotonic syndrome, nervous system disorder, neurodegenerative disease, neuromuscular diseaseEnrolling by invitationPhase 32026-08-19NCT07008469
A Study to Evaluate the Effect of Aficamten in Pediatric Patients With Symptomatic Obstructive Hypertrophic Cardiomyopathy (oHCM).Pediatric; Symptomatic Obstructive Hypertrophic CardiomyopathyActive, not recruitingPhase 2 / Phase 32026-08-18NCT06412666
A Study to Investigate Ronde-cel Versus Investigator's Choice CD19 CAR T-Cell TherapyB-cell neoplasm, Hemic and Lymphatic Diseases, Neoplasms by Histologic Type, diffuse large B-cell lymphoma, immune system disorder, immunoproliferative disorder, large B-cell lymphoma, lymphatic system disorder, lymphoma, lymphoproliferative syndrome, neoplasm, non-Hodgkin lymphomaRecruitingPhase 32026-08-04NCT07188558
Phase 1/1b/2 Study of Oral PMD-026 in Patients With Metastatic Breast CancerNeoplasms by Site, Skin and Connective Tissue Diseases, breast disorder, breast neoplasm, neoplasm, skin disorderActive, not recruitingPhase 1 / Phase 22026-07-16NCT04115306
The HistoSonics Edison™ System for Treatment of Primary Solid Renal Tumors Using Histotripsy (#HOPE4KIDNEY)Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Neoplasms by Site, Urogenital Diseases, Urogenital Neoplasms, benign urinary system neoplasm, kidney cancer, kidney disorder, neoplasm, urinary system disorderActive, not recruitingNot applicable2026-06-23NCT05820087
Secretin in RefeedingAnorexia in AdolescenceRecruitingNot stated2026-02-12NCT05235867
Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic DystrophyCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Disorders, Atrophic, hereditary disease, inherited neurodegenerative disorder, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, myotonic dystrophy, myotonic syndrome, nervous system disorder, neurodegenerative disease, neuromuscular diseaseRecruitingPhase 2 / Phase 32025-05-28NCT05004129