[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Denali Therapeutics Inc.\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":101},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,3,0,[8,43,70],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":25,"conditions":26,"keywords":28,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":31,"lastUpdatePostDateStruct":32,"startDateStruct":35,"completionDateStruct":37,"leadSponsor":39,"locationsCount":42},"100618194","phase-1-a-study-to-evaluate-the-safety-tolerability-pharmacokinetics-and-pharmacodynamics-of-dnl628-in-participants-with-early-alzheimers-disease-100618194",false,"NCT07328451","A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of DNL628 in Participants With Early Alzheimer's Disease","A Phase 1b, Multicenter, Randomized, Placebo-Controlled, Double-Blind Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of DNL628 in Participants With Early Alzheimer's Disease","Key Inclusion Criteria:\n\n* BMI of ≥18 to \\\u003C 32 kg\u002Fm2 and body weight of ≥45 kg\n* Have a diagnosis of probable AD dementia based on NIA AA 2011 criteria, including amnestic or nonamnestic presentation at screening\n* Have supportive evidence of AD pathology via historical records or laboratory testing at screening for amyloid positivity\n* Have AD severity defined as the following at screening:\n\n  * A Clinical Dementia Rating global score of 0.5 or 1\n  * A Mini-Mental State Examination score of 20 to 30 (inclusive)\n\nKey Exclusion Criteria:\n\n* Have clinically significant neurological or cognitive disorders affecting the CNS other than AD, as determined by the investigator\n* Have clinically significant psychiatric conditions\n* Have any history of unstable or poorly controlled endocrine, pulmonary, cardiovascular, gastrointestinal, hepatic, hematological, or other significant medical condition that, in the opinion of the investigator, may interfere with the completion or interpretation of study assessment\n* Have had a malignancy within 5 years before screening, except fully resected basal cell carcinoma or other malignancies (such as prostate cancer) at low risk of recurrence, depending on investigator and medical monitor agreement\n* Have had previous anti amyloid or anti tau immunotherapy (including active immunization)\n\n  * Note: ADAD participants who have participated in previous passive anti-amyloid immunotherapy \\> 6 months previously will be allowed, contingent on investigator and Sponsor agreement\n* Have had previous exposure to gene therapy","ALL","18 Years","75 Years",{"count":20,"type":21},68,"ESTIMATED","INTERVENTIONAL",[24],"PHASE1","This is a Phase 1b, multicenter, randomized, placebo-controlled, double-blind, multiple ascending dose (MAD) study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of DNL628 in participants with early Alzheimer's disease (AD), defined as mild cognitive impairment, or mild AD with biomarker evidence of amyloid positivity.\n\nNote: In the Netherlands, this study includes an open-label extension.",[27],"Alzheimer Disease, Early Onset",[29],"Alzheimer's Disease","RECRUITING","2026-06-29",{"date":33,"type":34},"2026-07-01","ACTUAL",{"date":36,"type":34},"2026-01-30",{"date":38,"type":21},"2027-02",{"name":40,"class":41},"Denali Therapeutics Inc.","INDUSTRY",4,{"id":44,"slug":45,"hasResults":11,"nctId":46,"briefTitle":47,"officialTitle":48,"acronym":4,"eligibilityCriteria":49,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":50,"targetDuration":4,"studyType":22,"phases":52,"briefSummary":53,"conditions":54,"keywords":56,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":63,"lastUpdatePostDateStruct":64,"startDateStruct":65,"completionDateStruct":67,"leadSponsor":69,"locationsCount":5},"100620215","phase-1-a-study-to-evaluate-the-safety-pharmacokinetics-and-pharmacodynamics-of-dnl952-in-adult-participants-with-late-onset-pompe-disease-100620215","NCT07354724","A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease","A Phase 1, Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease","Key Inclusion Criteria:\n\n* Body weight ≥40 kg\n* Diagnosis of LOPD\n* Upright FVC ≥ 30% of predicted normal value\n* Able to ambulate ≥ 40 meters (use of assistive devices is acceptable)\n* \\[Cohorts A1-A4 only\\] Have received avalglucosidase alfa or cipaglucosidase alfa at a dose of 20 mg\u002Fkg every 2 weeks for at least 12 months prior to screening\n* \\[Cohorts B1-B2 only\\] Must not have received any enzyme-replacement therapy for Pompe disease in the 12 months prior to screening\n\nKey Exclusion Criteria:\n\n* Any ongoing, clinically significant, unstable, or poorly controlled neurological, psychiatric, endocrine, pulmonary, cardiovascular, gastrointestinal, hepatic, pancreatic, renal, metabolic, hematological, immunological, allergic, or ophthalmic disease not related to Pompe disease, or other major disorders. Well-controlled conditions are permitted if investigator and Sponsor agree.\n* Wheelchair-dependent\n* Require noninvasive ventilation for an average of more than 6 hours per day while awake or any invasive ventilation. Use of noninvasive ventilation during sleep is acceptable.\n* Received an experimental gene therapy at any time or participation in any other investigational drug trial or use of investigational drug within 60 days or 5 half-lives, whichever is longer, before screening",{"count":51,"type":21},32,[24],"This is a Phase 1, multicenter, open-label study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of DNL952 in adult participants with late-onset Pompe disease. The principal aim of this study is to obtain safety and tolerability data across varous dose levels of DNL952 in participants with late-onset Pompe disease (LOPD).",[55],"Late-onset Pompe Disease",[57,58,59,60,61,62],"LOPD","Pompe","acid maltase deficiency","glycogen storage disease type II","ERT","enzyme replacement therapy","2026-06-26",{"date":31,"type":34},{"date":66,"type":34},"2026-05-12",{"date":68,"type":21},"2028-08",{"name":40,"class":41},{"id":71,"slug":72,"hasResults":11,"nctId":73,"briefTitle":74,"officialTitle":75,"acronym":76,"eligibilityCriteria":77,"healthyVolunteers":11,"sex":16,"minAge":78,"maxAge":79,"enrollmentInfo":80,"targetDuration":4,"studyType":22,"phases":82,"briefSummary":85,"conditions":86,"keywords":88,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":93,"lastUpdatePostDateStruct":94,"startDateStruct":96,"completionDateStruct":98,"leadSponsor":100,"locationsCount":51},"100467810","phase-2-a-study-to-determine-the-efficacy-and-safety-of-tividenofusp-alfa-dnl310-vs-idursulfase-in-pediatric-and-young-adult-participants-with-neuronopathic-nmps-ii-or-non-neuronopathic-mucopolysaccharidosis-type-ii-nnmps-ii-100467810","NCT05371613","A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)","A Phase 2\u002F3, Multicenter, Double-Blind, Randomized Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic or Non-Neuronopathic Mucopolysaccharidosis Type II","COMPASS","Key Inclusion Criteria:\n\n* Participants aged ≥2 to \\\u003C6 years (Cohort A) or ≥6 to \\\u003C26 years (Cohort B)\n* Confirmed diagnosis of MPS II (for Cohort A, nMPS II; for Cohort B, nnMPS II)\n* Have no history of treatment with enzyme replacement therapy (ERT) OR not have received continuous ERT for 4 months prior to screening OR be on maintenance ERT and have tolerated idursulfase for a minimum of 4 months prior to screening\n\nKey Exclusion Criteria:\n\n* Have a documented mutation of other genes or genetic diagnosis accounting for developmental delay\n* Previously received an iduronate 2-sulfatase (IDS) gene therapy or stem cell therapy\n* Received any CNS-targeted MPS ERT within 6 months prior to screening\n* Have a contraindication for lumbar punctures and\u002For magnetic resonance imaging (MRI)\n* Participated in any other investigational drug study or used an investigational drug within 60 days prior to screening or intend to receive another investigational drug during the study","2 Years","25 Years",{"count":81,"type":21},63,[83,84],"PHASE2","PHASE3","This is a Phase 2\u002F3, multiregional, two-arm, double-blind, randomized, active (standard-of-care)-controlled study of the efficacy and safety of tividenofusp alfa (DNL310), an investigational central nervous system (CNS)-penetrant enzyme-replacement therapy (ERT) for mucopolysaccharidosis type II (MPS II).\n\nParticipants may also qualify to enter an open-label treatment phase with DNL310 or idursulfase based on pre-specified criteria.",[87],"Mucopolysaccharidosis II",[89,90,91,92],"Hunter Syndrome","MPS II","nMPS II","nnMPS II","2025-08-04",{"date":95,"type":34},"2025-08-05",{"date":97,"type":34},"2022-07-21",{"date":99,"type":21},"2027-12",{"name":40,"class":41},""]