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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
BXQ-350 in Newly Diagnosed Metastatic Colorectal CarcinomaDigestive System Diseases, Digestive System Neoplasms, Neoplasms by Site, colonic disorder, colorectal neoplasm, digestive system disorder, digestive system neoplasm, intestinal disorder, neoplasm, peripheral neuropathy, rectal disorderActive, not recruitingPhase 1 / Phase 22026-09-29NCT05322590
A Study to Evaluate the Treatment Outcomes of Subcutaneous Anifrolumab in Immunosuppressant-naïve and Biologic-naïve Systemic Lupus ErythematosusSkin and Connective Tissue Diseases, autoimmune disease, connective tissue disorder, immune system disorder, systemic lupus erythematosusRecruitingPhase 32026-09-25NCT07430306
Observational Study Protocol: LIVER-RHepatobiliary CancersRecruitingNot stated2026-09-25NCT06252753
Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy-SUMMITCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne muscular dystrophy, Muscular Disorders, Atrophic, X-linked disease, hereditary disease, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, nervous system disorder, neuromuscular diseaseRecruitingNot stated2026-09-24NCT06564974
Non-interventional Study of Patients With Transthyretin (ATTR) AmyloidosisAmyloid Neuropathies, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, amyloidosis, 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, polyneuropathy, proteostasis deficienciesRecruitingNot stated2026-09-23NCT06465810
Open Label Extension to Assess Long Term Safety and Efficacy of KL1333 in Patients With Primary Mitochondrial DiseaseNutritional and Metabolic Diseases, inborn mitochondrial metabolism disorder, metabolic diseaseRecruitingPhase 22026-09-16NCT07514338
A Study to Investigate the Long-term Safety and Efficacy of Belimumab in Adults With Interstitial Lung Disease (ILD) Associated With Systemic Sclerosis (SSc) and Other Connective Tissue Diseases (CTD) (BLISSconneCTD-OLE)Skin and Connective Tissue Diseases, connective tissue disorder, diffuse scleroderma, interstitial lung disease, lung disorder, respiratory system disorder, skin disorder, systemic sclerosisRecruitingPhase 32026-09-09NCT06716606
A Phase IIIb Study to Evaluate Camizestrant Plus Ribociclib in ER-positive, HER2-negative Advanced Breast CancerNeoplasm Metastasis, Neoplasms by Site, Neoplastic Processes, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Skin and Connective Tissue Diseases, breast disorder, breast neoplasm, neoplasm, skin disorderRecruitingPhase 32026-09-08NCT07647328
The Eplontersen Pregnancy and Lactation Outcomes Studyfamilial amyloid neuropathyRecruitingNot stated2026-08-27NCT07205666
A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry DiseaseBrain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Cerebral Small Vessel Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Fabry disease, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, X-linked disease, brain disorder, cardiovascular disorder, central nervous system disorder, cerebrovascular disorder, hereditary disease, inborn errors of metabolism, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, sphingolipidosis, vascular disorderRecruitingPhase 2 / Phase 32026-08-10NCT06328608
ENdoluminal LIGHT ActivatED Treatment of Upper Tract Urothelial Cancer (ENLIGHTED) Studyrenal pelvis/ureter urothelial carcinomaActive, not recruitingPhase 32026-07-30NCT04620239
Efficacy and Safety of MB-CART2019.1 vs. SoC in Lymphoma PatientsB-cell neoplasm, Hemic and Lymphatic Diseases, Neoplasms by Histologic Type, Neoplasms by Site, diffuse large B-cell lymphoma, hematologic disorder, hematopoietic and lymphoid system neoplasm, immune system disorder, immunoproliferative disorder, lymphatic system disorder, lymphoma, lymphoproliferative syndrome, neoplasmRecruitingPhase 22026-06-10NCT04844866
Comparability Trial of the MVA-BN Vaccine Manufactured in Different Production CellsDNA Virus Infections, Infections, milker's nodule, monkeypox, non-human animal disease, non-human primate disease, rodent disease, viral infectious diseaseActive, not recruitingPhase 22026-04-08NCT07199569
Maternal and Postnatal Outcomes Study (MOS): A Global Observational Registry Assessing the Safety of Elfabrio® in Women With Fabry Disease and Their Infants During Pregnancy and BreastfeedingBrain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Cerebral Small Vessel Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Fabry disease, Female Urogenital Diseases and Pregnancy Complications, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, Urogenital Diseases, X-linked disease, brain disorder, cardiovascular disorder, central nervous system disorder, cerebrovascular disorder, hereditary disease, inborn errors of metabolism, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, pregnancy disorder, sphingolipidosis, vascular disorderRecruitingNot stated2026-03-19NCT06941025
Study to Evaluate the Safety, PK, PD, and Efficacy of PRX-102 in Japanese Patients With Fabry DiseaseBrain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Cerebral Small Vessel Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Fabry disease, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, X-linked disease, brain disorder, cardiovascular disorder, central nervous system disorder, cerebrovascular disorder, hereditary disease, inborn errors of metabolism, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, sphingolipidosis, vascular disorderRecruitingPhase 2 / Phase 32026-03-18NCT05710692
A Safety, Tolerability and Efficacy Study of Sernova's Cell Pouch™ for Clinical Islet TransplantationNutritional and Metabolic Diseases, autoimmune disease, diabetes mellitus, endocrine system disorder, glucose metabolism disease, hypoglycemia, immune system disorder, metabolic disease, type 1 diabetes mellitusActive, not recruitingPhase 1 / Phase 22026-02-25NCT03513939
A Trial Testing SP-420 in Subjects With Transfusion-dependent β-thalassemia or Low-risk Myelodysplastic SyndromesHemic and Lymphatic Diseases, bone marrow disorder, hematologic disorder, myelodysplastic syndromeRecruitingPhase 22025-09-24NCT05693909
Expanded Study of the HighLife 28mm Trans-septal Trans-catheter Mitral Valve in Patients With Moderate-severe or Severe Mitral Regurgitation and at High Surgical Riskcardiovascular disorder, heart disorder, heart valve disorder, mitral valve insufficiencyRecruitingNot applicable2025-09-15NCT04029363
French Registry Conducted on E-vita OPEN NEOAneurysm, cardiovascular disorder, vascular disorderActive, not recruitingNot stated2025-03-28NCT05721001
A Study of XmAb20717 (Vudalimab)in Patients With Selected Advanced Gynecologic and Genitourinary MalignanciesAdnexal Diseases, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Genital Diseases, Male Urogenital Diseases, Neoplasms by Histologic Type, Neoplasms by Site, Urogenital Diseases, Urogenital Neoplasms, Uterine Cervical Neoplasms, adenocarcinoma, carcinoma, cervical carcinoma, cervix disorder, clear cell adenocarcinoma, endocrine system disorder, endometrium neoplasm, epithelial neoplasm, fallopian tube disorder, fallopian tube neoplasm, female reproductive system disorder, female reproductive system neoplasm, gonadal disorder, male reproductive organ cancer, male reproductive system disorder, malignant endocrine neoplasm, neoplasm, ovarian cancer, ovarian disorder, prostate cancer, prostate disorder, uterine disorderActive, not recruitingPhase 22025-02-07NCT05032040
US Pivotal Clinical Study of the Adient Absorbable Filter for the Prevention of Pulmonary EmbolismEmbolism, Embolism and Thrombosis, cardiovascular disorder, lung disorder, pulmonary embolism, respiratory system disorder, vascular disorderActive, not recruitingNot applicable2023-08-31NCT05127915