hereditary disease
hereditary disease
Explore linked clinical trials. Standard reference: MONDO:00038473,937 results · Page 1 of 158
Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| A Study to Investigate the Safety, Tolerability, and Pharmacokinetics of ATI-105 in Healthy Adults | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Friedreich ataxia, Mitochondrial Dysfunction, Nutritional and Metabolic Diseases, Spinocerebellar Degenerations, brain disorder, cardiomyopathy, cardiovascular disorder, central nervous system disorder, cerebellar disorder, heart disorder, hereditary disease, inborn mitochondrial metabolism disorder, inherited neurodegenerative disorder, metabolic disease, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder | Not yet recruiting | Phase 1 | 2026-10-09 | NCT07869654 | |
| Absorption, Distribution, Metabolism, and Excretion of 14C-VX-407 After a Single Oral Dose | Abnormalities, Multiple, Ciliopathies, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Urogenital Diseases, autosomal dominant polycystic kidney disease, cystic kidney disease, hereditary disease, kidney disorder, polycystic kidney disease, urinary system disorder | Not yet recruiting | Phase 1 | 2026-10-09 | NCT07868926 | |
| Brain Iron in Beta-Propeller Protein Associated Neurodegeneration (BPAN) | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Nerve Degeneration, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, basal ganglia disorder, brain disorder, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, iron metabolism disease, metabolic disease, movement disorder, nervous system disorder, neuroaxonal dystrophy, neurodegeneration with brain iron accumulation, neurodegenerative disease, pantothenate kinase-associated neurodegeneration | Not yet recruiting | Not applicable | 2026-10-09 | NCT07868120 | |
| Tight Control of Treatment Adherence and Efficacy by Telemedicine for an Improved Personalized Management of Patients With Hemophilia | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, blood coagulation disease, coagulation protein disease, hematologic disorder, hemophilia A, hemorrhagic disease, hereditary disease, inherited blood coagulation disorder | Recruiting | Not applicable | 2026-10-09 | NCT07867834 | |
| Identification of New Biomarkers in Haemophilic Arthropathy Through Transcriptome Analysis | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, blood coagulation disease, coagulation protein disease, hematologic disorder, hemophilia A, hemorrhagic disease, hereditary disease, inherited blood coagulation disorder | Recruiting | Not applicable | 2026-10-09 | NCT07867821 | |
| Market Research of a Slow Release Protein Substitute for the Dietary Management of PKU | Brain 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, phenylketonuria | Not yet recruiting | Not applicable | 2026-10-09 | NCT07802366 | |
| A Trial to Assess Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Preliminary Efficacy of SFL-0821 in Adults With FSHD | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Disorders, Atrophic, facioscapulohumeral muscular dystrophy, hereditary disease, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, nervous system disorder, neuromuscular disease | Recruiting | Phase 1 / Phase 2 | 2026-10-09 | NCT07798609 | |
| A Study to Evaluate the Efficacy and Safety of Subcutaneous Nomlabofusp in Subjects With Friedreich's Ataxia | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Friedreich ataxia, Nutritional and Metabolic Diseases, Spinocerebellar Degenerations, brain disorder, central nervous system disorder, cerebellar disorder, hereditary disease, inborn mitochondrial metabolism disorder, inherited neurodegenerative disorder, metabolic disease, nervous system disorder, neurodegenerative disease, spinal cord disorder | Recruiting | Phase 3 | 2026-10-09 | NCT07778836 | |
| Evaluation of 611 in Chinese Children With Moderate to Severe Atopic Dermatitis(AD) | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hypersensitivity, Immediate, Skin Diseases, Eczematous, Skin and Connective Tissue Diseases, atopic eczema, dermatitis, hereditary disease, hereditary skin disorder, immune system disorder, skin disorder | Recruiting | Phase 3 | 2026-10-09 | NCT07767253 | |
| Study of EO2002 in Subjects With Corneal Edema Secondary to Corneal Endothelial Dysfunction | Chandler syndrome, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Corneal Endothelial Cell Loss, Corneal Endothelial Dystrophy 1, Eye Diseases, Hereditary, Eye Manifestations, Fuchs' endothelial dystrophy, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Postoperative Complications, Signs and Symptoms, congenital hereditary endothelial dystrophy of cornea, corneal disorder, corneal dystrophy, corneal edema, eye disorder, hereditary disease | Recruiting | Phase 2 | 2026-10-09 | NCT07729137 | |
| Study of YOLT-203 in Children and Adults With Primary Hyperoxaluria Type 1 (PH1) | Calculi, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Hyperoxaluria, Kidney Calculi, Male Urogenital Diseases, Nutritional and Metabolic Diseases, Pathological Conditions, Anatomical, Pathological Conditions, Signs and Symptoms, Urinary Calculi, Urogenital Diseases, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, kidney disorder, metabolic disease, nephrolithiasis, primary hyperoxaluria, primary hyperoxaluria type 1, urinary system disorder, urolithiasis | Recruiting | Phase 2 | 2026-10-09 | NCT07587021 | |
| Interfacing With NeuroTechnology to Expand Neural Throughput (INTENT) | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Infarction, Muscular Disorders, Atrophic, Necrosis, Neurologic Manifestations, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, TDP-43 proteinopathy, amyotrophic lateral sclerosis, brain disorder, brain infarction, brain ischemia, brain stem infarction, cardiovascular disorder, central nervous system disorder, cerebrovascular disorder, hereditary disease, injury, ischemic disease, metabolic disease, motor neuron disorder, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, nervous system disorder, nervous system injury, neurodegenerative disease, neuromuscular disease, palsy, progressive muscular atrophy, proteostasis deficiencies, quadriplegia, spinal cord disorder, spinal cord injury, spinal muscular atrophy, stroke disorder, vascular disorder | Recruiting | Not applicable | 2026-10-09 | NCT07521930 | |
| A Study to Investigate the Effects and Safety of Codabakalner for the Treatment of FXS in Male Participants and Adult Participants With ADS | Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neurobehavioral Manifestations, Neurologic Manifestations, Sex Chromosome Disorders, X-linked disease, autism, autism spectrum disorder, chromosomal disorder, fragile X syndrome, hereditary disease, inherited neurodegenerative disorder, intellectual disability, nervous system disorder, neurodevelopmental disorder, pervasive developmental disorder, psychiatric disorder | Not yet recruiting | Phase 2 / Phase 3 | 2026-10-09 | NCT07439510 | |
| Self-Efficacy Enhancement Among Online Support Group of Primary Caregivers of Children With Neuro Muscular Dystrophy | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne muscular dystrophy, Muscular Disorders, Atrophic, X-linked disease, central nervous system disorder, hereditary disease, motor neuron disorder, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophy | Active, not recruiting | Not applicable | 2026-10-09 | NCT07356063 | |
| Evaluating the Safety and Tolerability of Baricitinib in Patients With Job Syndrome With Lupus-Like Disease and/or Atopic Dermatitis | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hypersensitivity, Immediate, Skin Diseases, Eczematous, Skin and Connective Tissue Diseases, atopic eczema, dermatitis, hereditary disease, hereditary skin disorder, hyper-IgE syndrome, immune system disorder, skin disorder | Recruiting | Phase 1 | 2026-10-09 | NCT07262983 | |
| Clinical Genetics Branch Eligibility Screening Survey | Abnormalities, Multiple, Bone Marrow Failure Disorders, Congenital Abnormalities, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Costello syndrome, Craniofacial Abnormalities, DNA repair disease, Fanconi anemia, Hemic and Lymphatic Diseases, Infant, Newborn, Diseases, Legius syndrome, Li-Fraumeni syndrome, Musculoskeletal Abnormalities, Neoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, Thoracic Neoplasms, anemia, aplastic anemia, bone marrow disorder, cardiofaciocutaneous syndrome, chordoma, hematologic disorder, hereditary disease, hereditary neoplastic syndrome, inherited aplastic anemia, lung neoplasm, melanocytic neoplasm, melanoma, metabolic disease, mixed neoplasm, musculoskeletal system disorder, neoplasm, neuroendocrine neoplasm, primitive neuroectodermal tumor, pulmonary blastoma, respiratory tract neoplasm, skin disorder, skin neoplasm, susceptibility to familial cutaneous melanoma | Not yet recruiting | Not stated | 2026-10-09 | NCT07005297 | |
| A Study to Assess the Efficacy and Safety of Emicizumab in Participants With Type 3 Von Willebrand Disease | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, blood coagulation disease, coagulation protein disease, hematologic disorder, hemorrhagic disease, hereditary disease, inherited blood coagulation disorder, von Willebrand disease (hereditary or acquired), von Willebrand disease 3 | Recruiting | Phase 3 | 2026-10-09 | NCT06998524 | |
| MHB018A Treatment in Patients With Active Thyroid Eye Disease | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Hereditary, Graves Ophthalmopathy, Graves disease, autoimmune disease, disease of orbital part of eye adnexa, endocrine system disorder, exophthalmos, eye disorder, goiter, hereditary disease, hyperthyroidism, immune system disorder, thyroid gland disorder | Active, not recruiting | Phase 3 | 2026-10-09 | NCT06989918 | |
| A Biorepository of Multiple Allergic Diseases (MADREP) With Longitudinal Follow-Up | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hypersensitivity, Immediate, Respiratory Hypersensitivity, Rhinitis, Allergic, Skin Diseases, Eczematous, Skin and Connective Tissue Diseases, asthma, atopic eczema, bronchial disorder, dermatitis, hereditary disease, hereditary skin disorder, immune system disorder, lung disorder, nasal disorder, obstructive lung disease, otorhinolaryngologic disease, respiratory system disorder, rhinitis, skin disorder | Recruiting | Not stated | 2026-10-09 | NCT06732414 | |
| A Phase I/II Study of ITU512 in Healthy Participants and Patients With Sickle Cell Disease | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, anemia, familial hemolytic anemia, hematologic disorder, hemolytic anemia, hereditary disease, inherited hemoglobinopathy, sickle cell disease | Recruiting | Phase 1 / Phase 2 | 2026-10-09 | NCT06546670 | |
| An Open-Label Study of CTI-1601 in Subjects With Friedreich's Ataxia | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Friedreich ataxia, Nutritional and Metabolic Diseases, Spinocerebellar Degenerations, brain disorder, central nervous system disorder, cerebellar disorder, hereditary disease, inborn mitochondrial metabolism disorder, inherited neurodegenerative disorder, metabolic disease, nervous system disorder, neurodegenerative disease, spinal cord disorder | Recruiting | Phase 2 | 2026-10-09 | NCT06447025 | |
| Prescreening Study to Identify Potential Stargardt Participants for ACDN-01 Clinical Trials (STARPATH) | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Hereditary, Juvenile Macular Degeneration, Stargardt Disease 1, Stargardt disease, cone-rod dystrophy, eye disorder, hereditary disease, inherited retinal dystrophy, macular degeneration, retinal degeneration, retinal disorder | Recruiting | Not stated | 2026-10-09 | NCT06445322 | |
| A Study of RM-718 in Healthy Subjects and Patients With MC4R Pathway Impairment | Abnormalities, 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, overnutrition | Active, not recruiting | Phase 1 / Phase 2 | 2026-10-09 | NCT06239116 | |
| HELIOS: Open-Label, Long-Term Extension Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants With EPP or XLP | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, erythropoietic protoporphyria, hepatic porphyria, hereditary disease, hereditary skin disorder, liver disorder, metabolic disease, porphyria, skin disorder | Enrolling by invitation | Phase 2 / Phase 3 | 2026-10-09 | NCT05883748 | |
| Allogeneic Hematopoietic Stem Cell Transplantation for Chronic Granulomatous Disease (CGD) With an Alemtuzumab, Busulfan and TBI-based Conditioning Regimen Combined With Cytokine (IL-6, +/- IFN-gamma) Antagonists | Chronic Disease, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, Hemic and Lymphatic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, X-linked disease, chronic granulomatous disease, hematologic disorder, hereditary disease, immune system disorder, inborn error of immunity, leukocyte disorder, phagocyte bactericidal dysfunction | Recruiting | Phase 1 / Phase 2 | 2026-10-09 | NCT05463133 |