[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"UniQure Biopharma B.V.\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":76},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,2,0,[8,48],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":20,"targetDuration":4,"studyType":23,"phases":24,"briefSummary":27,"conditions":28,"keywords":30,"overallStatus":35,"whyStopped":4,"lastUpdateSubmitDate":36,"lastUpdatePostDateStruct":37,"startDateStruct":40,"completionDateStruct":42,"leadSponsor":44,"locationsCount":47},"100520998","phase-1-amt-260-gene-therapy-study-in-adults-with-unilateral-refractory-mesial-temporal-lobe-epilepsy-100520998",false,"NCT06063850","AMT-260 Gene Therapy Study in Adults With Unilateral Refractory Mesial Temporal Lobe Epilepsy","A Multi-center, Phase 1\u002F2a, First-in-human (FIH) Study Investigating the Safety, Tolerability, and Efficacy of AMT-260 in Adults With Unilateral Refractory Mesial Temporal Lobe Epilepsy (MTLE) Administered Via Magnetic Resonance Imaging (MRI)-Guided Convection-enhanced Delivery (CED)","GenTLE","Inclusion Criteria:\n\n* Diagnosis of unilateral refractory MTLE\n* History of seizures with an average of ≥ 2 documented focal impaired awareness seizures or focal to bilateral tonic-clonic seizures per 30-day period over the 3 month Retrospective Period.\n* On a stable type and dose regimen of up to a maximum of 4 approved Anti Seizure Drugs for at least 1 month prior to the Retrospective Period.\n* Confirmed unilateral hippocampal pathology and concordant unilateral seizure focus\n* No evidence of focal neurocognitive dysfunction, inconsistent with disease pathology- related MRI and\u002For (18F)FDG-PET findings.\n* Women of childbearing potential (WOCBP) and fertile male subjects must be willing and able to use highly effective methods of birth control consistently and correctly throughout the study.\n* For WOCBP only: Negative pregnancy test.\n\nExclusion Criteria:\n\n* Implanted devices that would contraindicate MRI; MRI-compatible devices must be implanted ≥3 months prior to Screening (vagus nerve stimulation devices will be up to discretion of the Investigator).\n* Any other contraindications to general anesthesia, surgery, or intra-operative magnetic resonance imaging (MRI).\n* Medications that could confound clinical (e.g., antipsychotic medication and anti-viral therapy) and laboratory evaluations or could affect a participant's safety or their ability to undergo the neurosurgical procedure or comply with the procedures and study visit schedule.\n* Bilateral or multifocal epilepsy; or inability to confidently lateralize seizure onset.\n* Dementia or other progressive neurological disorders and progressive brain lesions.\n* Previous major disease-unrelated neurosurgical intervention due to intracranial tumor, trauma, or bleeding and\u002For history of previous intracranial surgery for treatment of epileptic seizures, not including diagnostic stereo-EEG.\n* MRI evidence of epileptogenic, extra-temporal lesions, or dual temporal lobe pathology.\n* Status epilepticus within the year prior to Screening.","ALL","18 Years","75 Years",{"count":21,"type":22},12,"ESTIMATED","INTERVENTIONAL",[25,26],"PHASE1","PHASE2","The main goals of this clinical study are to learn if AMT-260 is safe and tolerable and works to reduce the frequency of seizures in adults with unilateral mesial temporal lobe epilepsy (MTLE).",[29],"Mesial Temporal Lobe Epilepsy",[31,32,33,34],"Epilepsy","Temporal Lobe","Hippocampal Sclerosis","Epileptic Syndromes","RECRUITING","2026-05-28",{"date":38,"type":39},"2026-06-02","ACTUAL",{"date":41,"type":39},"2024-06-12",{"date":43,"type":22},"2031-12",{"name":45,"class":46},"UniQure Biopharma B.V.","INDUSTRY",18,{"id":49,"slug":50,"hasResults":11,"nctId":51,"briefTitle":52,"officialTitle":53,"acronym":4,"eligibilityCriteria":54,"healthyVolunteers":11,"sex":55,"minAge":18,"maxAge":56,"enrollmentInfo":57,"targetDuration":4,"studyType":23,"phases":58,"briefSummary":59,"conditions":60,"keywords":62,"overallStatus":35,"whyStopped":4,"lastUpdateSubmitDate":67,"lastUpdatePostDateStruct":68,"startDateStruct":70,"completionDateStruct":72,"leadSponsor":74,"locationsCount":75},"100536860","phase-1-safety-pkpd-and-exploratory-efficacy-study-of-amt-191-in-classic-fabry-disease-100536860","NCT06270316","Safety, PK\u002FPD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease","A Phase 1\u002F2, Single Dose, Dose Ranging Study of Intravenous AAV5-GLA (AMT-191) in Adult Males With Classic Fabry Disease","Key Inclusion Criteria:\n\n* Male of age ≥ 18 years and ≤50 years\n* Confirmed clinical diagnosis of classic Fabry disease (FD) defined as:\n\n  1. Absent or minimal αGAL A enzyme activity \\\u003C 1% of mean normal measured in plasma regardless of variant status; OR\n  2. α-galactosidase A (GLA) pathogenic or likely pathogenic variant associated with classic FD phenotype identified on molecular genetic testing with plasma αGLA A enzyme activity below lower bound of the reference range (as measured at trough enzyme replacement therapy \\[ERT\\] levels).\n* eGFR ≥ 40 mL\u002Fmin\u002F1.73 m2\n* Suboptimal response after at least 12 months of enzyme replacement therapy (ERT) treatment. Suboptimal response is defined as plasma lyso-Gb3 ≥ 2.3 nanograms per milliliter (ng\u002FmL) at Screening and one or both of the following:\n* Persistent moderate or severe neuropathic pain (intermittent or continuous) over a period of at least 3 months prior to consent\n* Presence of gastrointestinal symptoms (abdominal cramping, constipation, or diarrhea), reported by the Participant as moderate or severe and that are either persistent or occurring two or more times over the 12 weeks prior to consent\n* Weight ≤ 120 kilograms (kg)\n\nKey Exclusion Criteria:\n\n* Any allergic hypersensitivity reaction to ERT or infusion reaction in the 12 months prior to consent that was of severity grade 3 or above based on Common Terminology Criteria for Adverse Events (CTCAE v5.0) and required emergency intervention for hypertension\u002Fhypotension to stabilize blood pressure or hypoxia OR any other life-threatening complication.\n* Proteinuria, with random urine protein\u002Fcreatinine ratio (rUPCR) ≥1 mg\u002Fmg at Screening\n* Current use of chaperone therapy such as migalastat (Galafold®)\n* Malignancy within 5 years of Screening, except for basal or squamous cell carcinoma of the skin\n* Presence of chronic, active, or latent infection with hepatitis B or C, human immunodeficiency virus (HIV), or tuberculosis (TB) as assessed at the screening visit\n* Active or ongoing infection or any other significant concurrent, uncontrolled medical condition including, but not limited to, renal, hepatic, cardiovascular, hematological, GI, endocrine (such as diabetes mellitus with poor glycemic control), pulmonary, neurological, cerebral, or psychiatric disease, alcoholism, drug dependency, or any other psychological disorder that could, in the opinion of the Investigator, risk the safety of the Participant, or interfere with adherence to the protocol procedures or interpretation of results\n* Evidence of any liver disease, including hepatitis, fibrosis, cirrhosis of the liver, neoplastic lesion, or any known medical condition that could impact the intended transduction of the vector and\u002For expression and activity of the protein\n* History of kidney transplantation or currently on hemodialysis or peritoneal dialysis\n* Uncontrolled hypertension, defined as systolic blood pressure \\>140 millimeters of mercury (mmHg) (inclusive) and\u002For diastolic blood pressure outside the range of 60 to 85 mmHg (inclusive) at Screening, confirmed on at least 2 repeated measurements\n* Patients taking blood pressure medication to control blood pressure or proteinuria (eg, angiotensin-converting enzyme \\[ACE\\] inhibitors and angiotensin II receptor blockers \\[ARBs\\]) and have been titrated to a stable dose for at least 3 months prior to Screening are allowed in the study.\n* Glycated hemoglobin (HbA1c) at Screening ≥7%\n* Contraindication to systemic corticosteroid therapy or immunosuppressive therapy\n* Chronic steroid use, defined as ≥ 3 months of oral corticosteroid use within the 12 months prior to Screening\n* Screening laboratory values for renal and liver function that meet or exceed any of the following:\n\n  1. Alanine transaminase (ALT) \\> 2 x upper limit of normal for the testing laboratory (ULN)\n  2. Aspartate aminotransferase (AST) \\> 2 x ULN\n  3. Total Bilirubin \\> 2 x ULN (except if this is caused by Gilbert disease)\n  4. Alkaline phosphatase (ALP) \\> 2 x ULN\n  5. Creatinine \\> 2 x ULN\n* Screening laboratory values for hematologic and coagulation function that meet any of the following:\n\n  1. Hemoglobin \\\u003C lower limit of normal (LLN) (as per reference laboratory ranges)\n  2. Platelet count \\\u003C 150 x1000\u002Fμl\n  3. International normalized ratio (INR) \\>1.1\n  4. Soluble terminal complement complex (sC5b-9)\\>ULN\n* Significant anatomical abnormalities on renal ultrasound such as the presence of only 1 kidney, significant differences in kidney sizes between the right and left kidneys \\>1.5 centimeters (about 0.59 inch), or presence of kidney cysts","MALE","50 Years",{"count":21,"type":22},[25,26],"The main goals of this clinical study are to characterize safety and PK\u002FPD of AMT-191 i.e. if drug doses used in the study are safe and tolerable and to understand how it acts in the body of people with Fabry disease.",[61],"Fabry Disease",[63,64,65,66],"GLA","gene therapy","ERT","FD","2025-10-21",{"date":69,"type":39},"2025-10-23",{"date":71,"type":39},"2024-06-05",{"date":73,"type":22},"2031-04-30",{"name":45,"class":46},8,""]