[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Prevail Therapeutics\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":82},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,3,0,[8,42,53],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":18,"targetDuration":4,"studyType":21,"phases":22,"briefSummary":25,"conditions":26,"keywords":4,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":30,"lastUpdatePostDateStruct":31,"startDateStruct":34,"completionDateStruct":36,"leadSponsor":38,"locationsCount":41},"100053288","phase-1-a-clinical-trial-of-pr001-ly3884961-in-patients-with-peripheral-manifestations-of-gaucher-disease-proceed-100053288",false,"NCT05487599","A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED)","An Open-label, Dose-Finding, Phase 1\u002F2 Study to Evaluate the Safety and Tolerability of a Single Intravenous Dose of LY3884961 in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED)","Inclusion Criteria:\n\n1. Age greater or equal to 18 years at the time of informed consent.\n2. Bi-allelic pathogenic GBA1 variants must be centrally confirmed.\n3. On ERT or SRT for at least 2 years and on a stable, maximum tolerated dose, for at least 3 months prior to screening.\n4. Capable of giving signed informed consent, including compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.\n5. Females and males will be eligible for this study. Men and women of childbearing potential must use a highly effective method of contraception consistently and correctly for the duration of the study, including the long-term follow-up.\n6. Patients must agree to abstain from blood, tissue and organ donation; and must agree to abstain from tissue and organ donation for the duration of the study, including long-term follow-up.\n\nExclusion Criteria:\n\n1. Clinically significant neurological signs and symptoms and\u002For behavioral disturbances.\n2. Active and progressive bone disease expected to require surgical treatment in the next 6 months.\n3. History of total splenectomy or planned total splenectomy during the first 18 months of the study. (Partial splenectomy not exclusionary).\n4. Splenomegaly \\> 10 MN as evaluated by centrally read abdominal magnetic resonance imaging (MRI)\n5. Evidence of clinically significant liver disease, fragile liver, or history of exposure to hepatotoxins.\n6. Thrombocytopenia with platelet count \\\u003C 40 × 10\\^3 per μL.\n7. Severe hyperlipidemia (triglycerides \\> 1,000 mg\u002FdL).\n8. Current diagnosis of unstable or clinically significant cardiovascular conditions based on Investigator assessment.\n9. History of certain cancers within 5 years of Screening.\n10. Concomitant disease, condition or treatment which, in the opinion of the Investigator, would pose an unacceptable risk to the patient or interfere with the patient's ability to comply with study procedures or interfere with the conduct of the study.\n11. Women of childbearing potential, pregnant (i.e., positive serum pregnancy result at Screening and\u002For Check-in) or breastfeeding or intending to become pregnant during the course of the trial.\n12. Use of any GD-related chaperone therapy within 4 weeks prior to Screening or expected need to initiate chaperone therapy during at least the first 18 months of the study.\n13. Any type of prior gene or cell therapy.\n14. Use of systemic immunosuppressant or steroid therapy other than protocol-specified immunosuppression.\n15. Participation in another therapeutic investigational drug or device study within 3 months or 5 half-lives of the study agent, whichever is longer.\n16. Have an anti-AAV9 antibody titer of \\>1:40 as determined by central laboratory.\n17. Clinically significant abnormalities in laboratory test results at Screening.\n18. Have any contraindications for MRI, including claustrophobia or the presence of contraindicated metal (ferromagnetic)implants\u002Fcardiac pacemaker.","ALL","18 Years",{"count":19,"type":20},15,"ESTIMATED","INTERVENTIONAL",[23,24],"PHASE1","PHASE2","Study J3Z-MC-OJAE is a Phase 1\u002F2, multicenter, open-label, dose-finding study of LY3884961 evaluating the safety and tolerability in adults with peripheral manifestations of GD.\n\nUp to 3 dose levels of LY3884961 will be assessed in 3 dose-finding cohorts of 3 patients. Following this, up to 6 patients may be enrolled in an expansion cohort.\n\nFor each enrolled patient, the study will be approximately 5 years in duration, including up to a 60-day screening period. During the first 18 months after dosing, subjects will be evaluated for the effects of LY3884961 on safety, tolerability, immunogenicity, biomarkers, and efficacy. Patients will be followed for an additional 42 months to monitor safety, immunogenicity, and selected biomarker and efficacy parameters.",[27,28],"Gaucher Disease","Gaucher Disease, Type 1","RECRUITING","2026-07-10",{"date":32,"type":33},"2026-07-13","ACTUAL",{"date":35,"type":33},"2022-12-20",{"date":37,"type":20},"2032-08-30",{"name":39,"class":40},"Prevail Therapeutics","INDUSTRY",9,{"id":43,"slug":4,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":44,"targetDuration":4,"studyType":21,"phases":45,"briefSummary":25,"conditions":46,"keywords":4,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":47,"lastUpdatePostDateStruct":48,"startDateStruct":50,"completionDateStruct":51,"leadSponsor":52,"locationsCount":41},"100476719",{"count":19,"type":20},[23,24],[27,28],"2026-05-19",{"date":49,"type":33},"2026-05-22",{"date":35,"type":33},{"date":37,"type":20},{"name":39,"class":40},{"id":54,"slug":55,"hasResults":11,"nctId":56,"briefTitle":57,"officialTitle":58,"acronym":59,"eligibilityCriteria":60,"healthyVolunteers":61,"sex":16,"minAge":62,"maxAge":63,"enrollmentInfo":64,"targetDuration":4,"studyType":21,"phases":66,"briefSummary":67,"conditions":68,"keywords":70,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":73,"lastUpdatePostDateStruct":74,"startDateStruct":76,"completionDateStruct":78,"leadSponsor":80,"locationsCount":81},"100559520","phase-1-a-clinical-trial-of-ly3962681-in-healthy-volunteers-and-in-patients-with-parkinsons-disease-100559520","NCT06565195","A Clinical Trial of LY3962681 in Healthy Volunteers and in Patients With Parkinson's Disease","A Randomized, Double-blind (Sponsor-unblinded), Placebo-controlled, Single-ascending-dose Study in Healthy Volunteers and a Double-blind (Sponsor-unblinded), Placebo-controlled, Multiple-ascending-dose Study in Patients With Parkinson's Disease to Evaluate the Safety, Tolerability, and PK\u002FPD of LY3962681","PROSPECT-PD","Inclusion Criteria:\n\n* Participant is overtly healthy as determined by medical evaluation. Rescreening is allowed in this study.\n* A Montreal Cognitive Assessment score greater than or equal to 24.\n* Stable use of background medications at least 8 weeks prior to IP administration, including but not limited to those used for treatment of Parkinson's disease (including deep brain stimulation), and the investigator must expect that participant can tolerate a minimum of 6 months without dose adjustment.\n\nMAD study only\n\n* Participant has a diagnosis of Parkinson's disease per UK Parkinson's Disease Society Brain Bank Clinical Diagnostic Criteria.\n* Modified Hoehn and Yahr Stage 1 to 2.5 in the practically defined OFF state.\n* A positive result on CSF alpha-synuclein Seed Amplification Assay. (A prior positive result \\[within 1 year of screening\\] accepted with sponsor approval if patient did not participate in another Parkinson's disease clinical trial during this period.) (US and Japan only)\n* UPSIT score of 20 percentile or less, corrected for age and sex (EU and UK only).\n* An abnormal DaT-SPECT consistent with parkinsonism. (History of an abnormal DaTSPECT with the report confirmed by study investigator will be accepted.)\n* For participants not taking Parkinson's disease medications, not expected to initiate treatment within 6 months.\n* Have a body weight within 40 kg (88 pounds) to 110 kg (242 pounds), inclusive, and body mass index within the range of 17 to 34 kg\u002Fm\\^2, inclusive.\n\nExclusion Criteria:\n\n* MAD study only: Significant neurological disease affecting the central nervous system other than Parkinson's disease that may be a cause for the participant's clinical symptoms or may confound study objectives.\n* Current concomitant disease or serious or unstable illnesses, including central nervous system (SAD study only), cardiovascular, hepatic, renal, gastroenterology, respiratory, endocrinologic, neurologic (MAD study only: other than Parkinson's disease), psychiatric, immunologic, or hematologic disease and other conditions that, in the investigator's opinion, could interfere with the conduct of the study or that would, in the opinion of the investigator, pose an unacceptable safety risk to the participant.\n* Participant is generally frail or has any medical disorders that, in the opinion of the investigator, could interfere with study-related procedures (including safe performance of IT injection or LP), such as prohibitive spinal diseases, bleeding diathesis, clinically significant coagulopathy, thrombocytopenia, or increased intracranial pressure.\n* Have a 12-lead ECG abnormality at screening that, in the opinion of the investigator, increases the risks associated with participating in the study or may confound ECG data analysis.\n* MAD study only: Treatment with continuous intestinal delivery Parkinson's disease medication (for example, Duodopa).\n* MAD study only: Significant renal impairment (estimated glomerular filtration rate \\[eGFR\\] \\\u003C45 mL\u002Fmin\u002F1.73 m\\^2).\n\nOther protocol-defined inclusion\u002Fexclusion criteria may apply.",true,"30 Years","80 Years",{"count":65,"type":20},124,[23],"The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics\u002Fpharmacodynamics (PK\u002FPD) of LY3962681 in healthy volunteers and patients with Parkinson's disease.\n\nThe study consists of two parts, the Single Ascending Dose (SAD) study and the Multiple Ascending Dose (MAD) study.\n\nDuring the SAD portion of the study, healthy volunteers will receive a single dose of LY3962681 or placebo (artificial cerebrospinal fluid \\[aCSF\\]) administered intrathecally (into the spinal fluid). During the MAD portion of the study, patients with Parkinson's disease will receive two doses of either LY3962681 or placebo (aCSF) administered intrathecally (into the spinal fluid), 12 to 24 weeks apart.\n\n* The treatment period in the SAD study will be 1 day. The treatment period in the MAD study will be 2 dosing days, 12 to 24 weeks apart.\n* The follow-up period in the SAD study will be up to 52 weeks. The follow-up period in the MAD study will be up to 52 weeks after Dose 2.",[69],"Parkinson's Disease",[71,72],"Parkinson's disease","Healthy volunteers","2026-05-14",{"date":75,"type":33},"2026-05-18",{"date":77,"type":33},"2024-08-27",{"date":79,"type":20},"2029-05-05",{"name":39,"class":40},4,""]