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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
A Study of RM-718 in Healthy Subjects and Patients With MC4R Pathway ImpairmentAbnormalities, Multiple, Body Weight, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Froelich syndrome, Imprinting Disorders, Male Urogenital Diseases, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Overweight, Parkes Weber syndrome, Pathological Conditions, Signs and Symptoms, Prader-Willi syndrome, Signs and Symptoms, Urogenital Abnormalities, Urogenital Diseases, chromosomal disorder, disorder of sexual differentiation, endocrine system disorder, gonadal disorder, gonadal dysgenesis, hereditary disease, hypogonadism, intellectual disability, nervous system disorder, nutritional disorder, obesity disorder, overnutritionActive, not recruitingPhase 1 / Phase 22026-10-09NCT06239116
An Extension Trial of Asedebart in Participants With Congenital Adrenal HyperplasiaCongenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Nutritional and Metabolic Diseases, Urogenital Abnormalities, Urogenital Diseases, adrenal gland disorder, adrenogenital syndrome, congenital adrenal hyperplasia, disorder of sexual differentiation, endocrine system disorder, gonadal disorder, hereditary disease, inborn errors of metabolism, metabolic disease, steroid inherited metabolic disorderNot yet recruitingPhase 22026-10-05NCT07859293
Gonadal Tissue Freezing for Fertility Preservation in Individuals at Risk for Ovarian Dysfunction, Premature Ovarian Insufficiency and Clinically Indicated GonadectomyBrain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Cardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Nutritional and Metabolic Diseases, Sex Chromosome Disorders, Turner syndrome, Urogenital Abnormalities, Urogenital Diseases, brain disorder, cardiovascular disorder, central nervous system disorder, chromosomal disorder, congenital heart disease, disorder of sexual differentiation, endocrine system disorder, galactosemia, gonadal disorder, gonadal dysgenesis, heart disorder, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, metabolic disease, nervous system disorder, sex chromosome disorder of sex developmentRecruitingNot stated2026-10-05NCT04948658
Data Collection Study of Pediatric and Adolescent Gynecology ConditionsCongenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Urogenital Abnormalities, Urogenital Diseases, disorder of sexual differentiation, endocrine system disorder, gonadal disorderRecruitingNot stated2026-10-05NCT04717349
Natural History of Noncirrhotic Portal HypertensionCardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Hypertrophy, Idiopathic Noncirrhotic Portal Hypertension, Infant, Newborn, Diseases, Male Urogenital Diseases, Pathological Conditions, Anatomical, Pathological Conditions, Signs and Symptoms, Sex Chromosome Disorders, Splenomegaly, Turner syndrome, Urogenital Abnormalities, Urogenital Diseases, cardiovascular disorder, chromosomal disorder, congenital heart disease, cystic fibrosis, disorder of sexual differentiation, endocrine system disorder, gonadal disorder, gonadal dysgenesis, heart disorder, hereditary disease, immune system disorder, inborn error of immunity, isolated congenital hepatic fibrosis, liver disorder, lung disorder, pancreas disorder, portal hypertension, respiratory system disorder, sex chromosome disorder of sex development, varicose disease, vascular disorderRecruitingNot stated2026-10-02NCT02417740
Worldwide Yearly Screening for HypospadiasCongenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Genital Diseases, Male Urogenital Diseases, Urogenital Abnormalities, Urogenital Diseases, hypospadias, male reproductive system disorder, penile disorderRecruitingNot applicable2026-10-01NCT06966375
Efficacy, Safety, Tolerability, and Pharmacokinetics of NBIP-01435 in Adult Participants With Classic Congenital Adrenal Hyperplasia (CAH)Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Nutritional and Metabolic Diseases, Urogenital Abnormalities, Urogenital Diseases, adrenal gland disorder, adrenogenital syndrome, congenital adrenal hyperplasia, disorder of sexual differentiation, endocrine system disorder, gonadal disorder, hereditary disease, inborn errors of metabolism, metabolic disease, steroid inherited metabolic disorderNot yet recruitingPhase 22026-09-30NCT07850739
EXACT Study: A Blinded Study in Patients With Alport Syndrome to Evaluate Exaluren Efficacy and SafetyCollagen Diseases, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Skin and Connective Tissue Diseases, Urogenital Abnormalities, Urogenital Diseases, X-linked Alport syndrome, autosomal recessive Alport syndrome, connective tissue disorder, hereditary nephritis, kidney disorder, nephritis, urinary system disorderRecruitingPhase 22026-09-30NCT07523581
Trial Investigating the Efficacy and Safety of Weekly Lonapegsomatropin Compared to Daily Somatropin in Children and Adolescents With Short Stature or Growth Failure Due to Growth Hormone Sufficient DisordersBone Diseases, Developmental, Cardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Craniofacial Abnormalities, Dwarfism, Failure to Thrive, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Musculoskeletal Abnormalities, Noonan syndrome, Pathological Conditions, Signs and Symptoms, Sex Chromosome Disorders, Signs and Symptoms, Skin and Connective Tissue Diseases, Turner syndrome, Urogenital Abnormalities, Urogenital Diseases, bone disorder, cardiovascular disorder, chromosomal disorder, congenital heart disease, connective tissue disorder, disorder of sexual differentiation, endocrine system disorder, gonadal disorder, gonadal dysgenesis, heart disorder, hereditary disease, musculoskeletal system disorder, sex chromosome disorder of sex developmentRecruitingPhase 32026-09-30NCT07221851
A Study in Pediatric Participants With Congenital Adrenal Hyperplasia (Balance-CAH)Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Nutritional and Metabolic Diseases, Urogenital Abnormalities, Urogenital Diseases, adrenal gland disorder, adrenogenital syndrome, classic congenital adrenal hyperplasia, congenital adrenal hyperplasia, disorder of sexual differentiation, endocrine system disorder, gonadal disorder, hereditary disease, inborn errors of metabolism, metabolic disease, steroid inherited metabolic disorderRecruitingPhase 2 / Phase 32026-09-30NCT07159841
A Study to Evaluate Atumelnant in Adults With Congenital Adrenal HyperplasiaCongenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Nutritional and Metabolic Diseases, Urogenital Abnormalities, Urogenital Diseases, adrenal gland disorder, adrenogenital syndrome, classic congenital adrenal hyperplasia, congenital adrenal hyperplasia, disorder of sexual differentiation, endocrine system disorder, gonadal disorder, hereditary disease, inborn errors of metabolism, metabolic disease, steroid inherited metabolic disorderRecruitingPhase 32026-09-30NCT07144163
SEMAFORCRANIO : Multicenter, Double-blind, Parallel, Randomized Controlled Trial of the Efficacy of Semaglutide in Hypothalamic Obesity Secondary to Craniopharyngioma in Children Aged 12 to 17 YearsCongenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Craniopharyngioma, Child, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Froelich syndrome, Male Urogenital Diseases, Neoplasms by Histologic Type, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Urogenital Abnormalities, Urogenital Diseases, craniopharyngioma, disorder of sexual differentiation, endocrine system disorder, gonadal disorder, gonadal dysgenesis, hypogonadism, neoplasm, primitive neuroectodermal tumorNot yet recruitingPhase 32026-09-29NCT07845994
A Pilot Study Comparison of Programmed Intermittent Bolus Versus Continuous InfusionCongenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Urogenital Abnormalities, Urogenital Diseases, bladder exstrophy, urinary bladder disorder, urinary system disorderNot yet recruitingNot applicable2026-09-28NCT07839039
Metyrapone Study for Patients Diagnosed With Mild Autonomous Cortisol Secretion - MACSCongenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cushing syndrome, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Nutritional and Metabolic Diseases, Paraneoplastic Endocrine Syndromes, Urogenital Abnormalities, Urogenital Diseases, adrenal gland disorder, adrenal gland hyperfunction, adrenogenital syndrome, congenital adrenal hyperplasia, disorder of sexual differentiation, ectopic ACTH secretion syndrome, endocrine system disorder, gonadal disorder, hereditary disease, inborn errors of metabolism, metabolic disease, neoplasm, paraneoplastic syndrome, steroid inherited metabolic disorderRecruitingPhase 22026-09-25NCT07138274
Metyrapone for Mild Autonomous Cortisol Secretion (MACS)Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cushing syndrome, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Nutritional and Metabolic Diseases, Paraneoplastic Endocrine Syndromes, Urogenital Abnormalities, Urogenital Diseases, adrenal gland disorder, adrenal gland hyperfunction, adrenogenital syndrome, congenital adrenal hyperplasia, disorder of sexual differentiation, ectopic ACTH secretion syndrome, endocrine system disorder, gonadal disorder, hereditary disease, inborn errors of metabolism, metabolic disease, neoplasm, paraneoplastic syndrome, steroid inherited metabolic disorderActive, not recruitingPhase 22026-09-25NCT06106295
Concomitant Renal and Urinary Bladder Allograft TransplantationChronic Disease, Chronic Kidney Diseases, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Neurologic Manifestations, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Urinary Bladder, Neurogenic, Urogenital Abnormalities, Urogenital Diseases, bladder exstrophy, bladder neck obstruction, chronic renal failure syndrome, kidney disorder, kidney failure, nervous system disorder, posterior urethral valve, urethral disorder, urethral obstruction, urinary bladder disorder, urinary system disorderRecruitingEarly phase 12026-09-24NCT04626167
Eurbio-Alport (RaDiCo Cohort) (RaDiCo Eurbio-Alport)Alport syndrome, Collagen Diseases, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Rare Diseases, Skin and Connective Tissue Diseases, Urogenital Abnormalities, Urogenital Diseases, connective tissue disorder, hereditary nephritis, kidney disorder, nephritis, urinary system disorderRecruitingNot stated2026-09-21NCT05927467
A Study to Learn About How Well BAY 3401016 Works in Adults With Alport SyndromeAlport syndrome, Collagen Diseases, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Skin and Connective Tissue Diseases, Urogenital Abnormalities, Urogenital Diseases, connective tissue disorder, hereditary nephritis, kidney disorder, nephritis, urinary system disorderActive, not recruitingPhase 22026-09-18NCT07211685
Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Crinecerfont in Participants With Classic Congenital Adrenal Hyperplasia (CAH) Who Are Less Than 4 Years OldCongenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Nutritional and Metabolic Diseases, Urogenital Abnormalities, Urogenital Diseases, adrenal gland disorder, adrenogenital syndrome, congenital adrenal hyperplasia, disorder of sexual differentiation, endocrine system disorder, gonadal disorder, hereditary disease, inborn errors of metabolism, metabolic disease, steroid inherited metabolic disorderRecruitingPhase 22026-09-16NCT07536269
Genetic and Epigenetic Background of Inner Ear Dysfunction in Turner SyndromeCardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Ear Diseases, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Hearing Loss, Sensorineural, Male Urogenital Diseases, Neurologic Manifestations, Pathological Conditions, Signs and Symptoms, Sensation Disorders, Sex Chromosome Disorders, Signs and Symptoms, Turner syndrome, Urogenital Abnormalities, Urogenital Diseases, cardiovascular disorder, chromosomal disorder, congenital heart disease, disorder of sexual differentiation, endocrine system disorder, gonadal disorder, gonadal dysgenesis, hearing disorder, hearing loss disorder, heart disorder, hereditary disease, inner ear disorder, nervous system disorder, otorhinolaryngologic disease, sex chromosome disorder of sex developmentRecruitingNot stated2026-09-11NCT06507007
Hematological Profile in Children With Numerical Chromosomal DisordersAbnormalities, Multiple, Cardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Down syndrome, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Klinefelter syndrome, Male Urogenital Diseases, Neurobehavioral Manifestations, Neurologic Manifestations, Sex Chromosome Disorders, Trisomy 18 Syndrome, Turner syndrome, Urogenital Abnormalities, Urogenital Diseases, cardiovascular disorder, chromosomal disorder, congenital heart disease, disorder of sexual differentiation, endocrine system disorder, gonadal disorder, gonadal dysgenesis, heart disorder, hereditary disease, hypogonadism, intellectual disability, nervous system disorder, sex chromosome disorder of sex development, trisomy 18Not yet recruitingNot stated2026-09-10NCT07807540
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of OMS1620Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Nutritional and Metabolic Diseases, Urogenital Abnormalities, Urogenital Diseases, adrenal gland disorder, adrenogenital syndrome, congenital adrenal hyperplasia, disorder of sexual differentiation, endocrine system disorder, gonadal disorder, hereditary disease, inborn errors of metabolism, metabolic disease, steroid inherited metabolic disorderNot yet recruitingPhase 12026-09-09NCT07811089
Emerging Indications for Hyperbaric Oxygen TreatmentArterial Occlusive Diseases, Axonotmesis, Bacterial Infections and Mycoses, CNS demyelinating autoimmune disease, Clostridium Enterocolitis, Clostridium difficile colitis, Clostridium infectious disease, Cold Injury, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Conjunctival Diseases, Craniocerebral Trauma, Crohn disease, Crohn ileitis, Digestive System Diseases, Digestive System Fistula, Ear Diseases, Embolism and Thrombosis, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Fistula, Genital Diseases, Ileal Diseases, Infections, Invasive Fungal Infections, Male Urogenital Diseases, Necrosis, Neurologic Manifestations, Nonarteritic Anterior Ischemic Optic Neuropathy, Nutritional and Metabolic Diseases, Optic Neuropathy, Ischemic, Pathologic Processes, Pathological Conditions, Anatomical, Pathological Conditions, Signs and Symptoms, Postoperative Complications, Raynaud disease, Rectovaginal Fistula, Sensation Disorders, Signs and Symptoms, Skin Abnormalities, Skin Ulcer, Skin and Connective Tissue Diseases, Urogenital Abnormalities, Urogenital Diseases, Vaginal Fistula, Venous Thrombosis, anal fistula, autoimmune disease, autoimmune disorder of the nervous system, bacterial infectious disease, bone disorder, branch retinal artery occlusion, calcinosis, calciphylaxis, calcium metabolic disease, cardiovascular disorder, central retinal vein occlusion, colitis, colonic disorder, cranial nerve neuropathy, decubitus ulcer, demyelinating disease, digestive system disorder, enteritis, enterocolitis, epidermolysis bullosa, eye disorder, female reproductive system disorder, frostbite, fungal infectious disease, gastroenteritis, graft versus host disease, gram-positive bacterial infections, hearing disorder, hereditary disease, hereditary skin disorder, hypospadias, immune system disorder, inclusion body myositis, inflammatory bowel disease, injury, intestinal disorder, intestinal fistula, livedoid vasculopathy, male reproductive system disorder, malignant otitis externa, metabolic disease, multiple sclerosis, muscle tissue disorder, musculoskeletal system disorder, myositis disease, nervous system disorder, nervous system injury, neuromuscular disease, optic nerve disorder, osteonecrosis, otorhinolaryngologic disease, penile disorder, peripheral vascular disease, pouchitis, prosthesis-related infectious disease, pterygium, pyoderma, pyoderma gangrenosum, rectal disorder, retinal artery occlusion, retinal disorder, retinal vein occlusion, skin disorder, skin vascular disease, thrombotic disease, tinnitus, ulcerative colitis, vaginal disorder, vascular disorder, vesiculobullous skin diseaseNot yet recruitingPhase 42026-09-09NCT07240649
Natural History Study of Patients With Excess AndrogenCongenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Familial Testotoxicosis, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Nutritional and Metabolic Diseases, Urogenital Abnormalities, Urogenital Diseases, adrenal gland disorder, adrenocortical insufficiency, adrenogenital syndrome, classic congenital adrenal hyperplasia due to 21-hydroxylase deficiency, congenital adrenal hyperplasia, disorder of sexual differentiation, endocrine system disorder, gonadal disorder, hereditary disease, inborn errors of metabolism, metabolic disease, precocious puberty, steroid inherited metabolic disorderRecruitingNot stated2026-09-08NCT00250159
Polycystic Ovary Syndrome in Type 1 Diabetes46,XX disorder of sex development, Adnexal Diseases, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cysts, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Genital Diseases, Hyperandrogenism, Insulin Resistance, Male Urogenital Diseases, Nutritional and Metabolic Diseases, Urogenital Abnormalities, Urogenital Diseases, adrenogenital syndrome, autoimmune disease, diabetes mellitus, disorder of sexual differentiation, endocrine system disorder, female reproductive system disorder, glucose metabolism disease, gonadal disorder, hyperinsulinism, immune system disorder, metabolic disease, neoplasm, ovarian cyst, ovarian disorder, polycystic ovary syndrome, type 1 diabetes mellitusRecruitingNot stated2026-09-03NCT07490964