[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"AskBio Inc\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":147},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,4,0,[8,44,71,110],{"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":26,"conditions":27,"keywords":29,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":32,"lastUpdatePostDateStruct":33,"startDateStruct":36,"completionDateStruct":38,"leadSponsor":40,"locationsCount":43},"100538039","phase-2-a-study-of-aav2-gdnf-in-adults-with-moderate-parkinsons-disease-regenerate-pd-100538039",false,"NCT06285643","A Study of AAV2-GDNF in Adults With Moderate Parkinson's Disease (REGENERATE-PD)","A Phase 2, Randomized, Double-blind, Sham Surgery-controlled Study of the Efficacy and Safety of Intraputaminal AAV2-GDNF in the Treatment of Adults With Moderate Stage Parkinson's Disease","REGENERATE-PD","Inclusion Criteria:\n\nAge\n\n1. Male and female adults 45-75 years of age inclusive, at the time of signing of informed consent Type of Subject and Disease Characteristics\n2. Diagnosed with Parkinson's disease in the past 4-10 years (inclusive) as defined by the following:\n\n   1. Presence of bradykinesia PLUS any of the following:\n\n      * Rigidity\n      * Rest tremor\n      * Postural instability\n   2. Presence of motor fluctuations as measured by the PD Motor Diary\n   3. Stable anti-parkinsonian medication regimen for \\>\u002F= 4 weeks prior to screening\n   4. Must demonstrate responsiveness to levodopa therapy\n\nExclusion Criteria:\n\n* Known history or current evidence of medical, genetic, or neurological conditions that may provide an alternative to idiopathic PD diagnosis\n* Presence or history of significant vascular and\u002For cardiovascular disease\n* Presence of significant cognitive impairment, poorly controlled depression\u002Fanxiety\n* Presence or history of psychosis or impulse control disorder\n* History of malignancy other than treated cutaneous squamous or basal cell carcinomas\n* Presence of clinically relevant conditions that could compromise surgical suitability and\u002For subject safety\n* Contraindication to magnetic resonance imaging and\u002For use gadolinium-based contrast agents\n* Prior history of brain surgery including, but no limited: DBS, pallidotomy focused ultrasound thalamotomy, or other experimental neurosurgical procedure\n* Chronic immunosuppressive therapy","ALL","45 Years","75 Years",{"count":21,"type":22},127,"ESTIMATED","INTERVENTIONAL",[25],"PHASE2","The objective of this randomized, surgically controlled, double-blinded, Phase 2 study is to evaluate the safety and efficacy of AAV2-GDNF delivered to the putamen in subjects with moderate Parkinson's Disease.",[28],"Parkinson Disease",[30],"Gene therapy","RECRUITING","2026-06-25",{"date":34,"type":35},"2026-06-29","ACTUAL",{"date":37,"type":35},"2024-06-11",{"date":39,"type":22},"2028-08-31",{"name":41,"class":42},"AskBio Inc","INDUSTRY",46,{"id":45,"slug":46,"hasResults":11,"nctId":47,"briefTitle":48,"officialTitle":49,"acronym":50,"eligibilityCriteria":51,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":52,"targetDuration":4,"studyType":23,"phases":54,"briefSummary":55,"conditions":56,"keywords":58,"overallStatus":61,"whyStopped":4,"lastUpdateSubmitDate":62,"lastUpdatePostDateStruct":63,"startDateStruct":65,"completionDateStruct":67,"leadSponsor":69,"locationsCount":70},"100609501","phase-2-prescriptive-infusion-algorithm-pia-100609501","NCT07215403","Prescriptive Infusion Algorithm (PIA)","Open-Label, Multi-Stage Study to Optimize the Intraputaminal Administration of AB-1005 Using a Prescriptive Infusion Algorithm (PIA)","PIA","Inclusion Criteria:\n\n* Participant must be 45 to 75 years of age inclusive, at the time of signing the informed consent.\n* \\>10 years since diagnosis of PD (at time of consenting \u002F Screening Visit 1)\n* Presence of bradykinesia plus any of the following:\n* Rigidity\n* Resting tremor\n* Postural instability\n* Modified Hoehn and Yahr stage III-IV in the practically defined OFF state\n* Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part III score \\>40 in the practically defined OFF state\n* Stable anti-PD medication regimen for at least 4 weeks prior to Screening Visit 1 and through Baseline Visit\n* ≥30% reduction in MDS-UPDRS Part III following a levodopa challenge\n* Must agree to use barrier method protection when engaging in intercourse\u002Fsexual activity with another person for at least 3 months post-dosing.\n* Male participants must refrain from donating sperm for at least 3 months post-dosing.\n* Female participants cannot be pregnant or breastfeeding at the time of screening. A woman of childbearing potential (WOCBP) must have a negative highly sensitive pregnancy test (urine or serum as required by local regulations) at the required assessments\n* Provision of signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol\n* Provision of signed consent to also participate in LTFU study ASK-PD0-CS002\n\nExclusion Criteria:\n\n* Evidence of secondary or atypical parkinsonism (as determined by the neurologist)\n* Presence or history of psychosis or impulse control disorder (as determined by the neurologist)\n* Presence or history (within 2 years prior to screening) of substance use disorder (including alcohol) as defined by the Diagnostic and Statistical Manual of Mental Disorders (DSM-5) criteria (or in the judgment of the neurologist)\n* Presence of untreated or sub optimally treated depression (Beck Depression Inventory \\[BDI\\]-II score ≥20)\n* Current suicidal ideation as indicated by positive response to items 4 or 5 on the Columbia-Suicide Severity Rating Scale (C SSRS), or any history of a suicide attempt\n* Clinically significant cognitive impairment (Montreal Cognitive Assessment \\[MoCA\\] score \\\u003C25)\n* Presence or history of malignancy other than treated cutaneous squamous or basal cell carcinomas\n* Presence of clinically active infection, including acute or chronic scalp infection\n* Known contraindications to MRI\n* Presence or history of significant cerebrovascular or cardiovascular disease, including:\n* Stroke, transient ischemic attack, or other suspected cerebrovascular accident within 1 year prior to screening\n* Unstable angina pectoris or myocardial infarction within 1 year prior to screening\n* Revascularization procedure(s) within 1 year prior to screening\n* Poorly controlled hypertension, poorly controlled diabetes mellitus or prediabetes mellitus with known significant microvascular injury, or other significant cardiovascular history or risk factor\n* Known history of complications of anesthesia including difficult airway management and\u002For difficult endotracheal intubation, malignant hyperthermia, or other related issues that would compromise participant safety during general anesthesia (as determined by the anesthesiologist)\n* Inability to identify a safe trajectory to each putamen via an occipitoparietal entry point, or known contraindications to brain surgery in prone position (as determined by the neurosurgeon)\n* Known allergy or sensitivity to ingredients in the intervention formulation and\u002For to gadolinium-based contrast agents\n* Concurrent use of percutaneous levodopa\u002Fcarbidopa intestinal gel, subcutaneous levodopa, or apomorphine pump\n* History of brain surgery (including deep brain stimulation \\[DBS\\] or focused ultrasound)\n* Chronic immunosuppressive therapy\n* History of prior cell or gene therapy\n* Participation in other interventional clinical trials within 12 weeks prior to screening or unwilling to refrain from starting new investigational agents throughout the course of the study\n* Laboratory values at screening:\n* Platelets ≤100,000\u002Fmm3\n* PT \\>15 s, aPTT \\>40 s, and\u002For INR \\>1.3\n* Absolute neutrophil count (ANC) ≤1500\u002Fmm3\n* Hemoglobin ≤10.0 g\u002FdL\n* Aspartate aminotransferase or alanine aminotransferase ≥2.5 times the upper limit of normal\n* Total bilirubin ≥2.5 mg\u002FdL\n* eGFR \\\u003C45 mL\u002Fmin\u002F1.73 m2\n* HbA1C ≥8%\n* Unable to comply with the protocol procedures, including frequent and prolonged follow-up assessments (during LTFU study ASK-PD0-CS002)\n* Unwilling to defer any vaccination from screening through 1 month after surgery\n* Any significant issue raised by the neurologist, neurosurgeon, or anesthesiologist that may make a participant unsuitable for the study",{"count":53,"type":22},18,[25],"This study is being done to test a new way of delivering AB-1005 into the brain. The goal is to make the procedure easier and quicker to perform, while providing similar amounts of drug to the part of the brain that needs treatment.\n\nTechnical (Stage 0) To test if the new delivery method (prefrontal surgical approach) can consistently deliver AB-1005 to the putamen using MRI monitoring.\n\nTechnical (Stage 1) To test if the new delivery method (PIA-based infusion) can consistently deliver AB-1005 to the putamen using brain imaging by MRI.\n\nTechnical (Stage 2) To confirm the new delivery method works without brain imaging by MRI, using standard operating room tools including brain imaging by CT.\n\nSafety (Stage 1 and 2) To assess the safety and tolerability of the new delivery method for AB-1005 up to 6 months after surgery",[57],"PARKINSON DISEASE (Disorder)",[59,60],"PD","Parkinsons Disease","NOT_YET_RECRUITING","2026-06-24",{"date":64,"type":35},"2026-06-26",{"date":66,"type":22},"2026-07-15",{"date":68,"type":22},"2028-03-01",{"name":41,"class":42},1,{"id":72,"slug":73,"hasResults":11,"nctId":74,"briefTitle":75,"officialTitle":76,"acronym":4,"eligibilityCriteria":77,"healthyVolunteers":11,"sex":17,"minAge":78,"maxAge":4,"enrollmentInfo":79,"targetDuration":4,"studyType":23,"phases":81,"briefSummary":83,"conditions":84,"keywords":88,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":101,"lastUpdatePostDateStruct":102,"startDateStruct":104,"completionDateStruct":106,"leadSponsor":108,"locationsCount":109},"100614688","phase-1-a-study-to-evaluate-safety-tolerability-and-efficacy-of-ab-1009-gene-therapy-gaa-gene-in-adult-participants-with-late-onset-pompe-disease-progress-gt-lopd-100614688","NCT07282847","A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late Onset Pompe Disease (PROGRESS-GT LOPD)","A Single-Arm, Open-Label, Dose-Escalation Study to Evaluate the Safety, Tolerability and Efficacy of a Single Intravenous Infusion of AB-1009 in Adult Participants With Late Onset Pompe Disease (LOPD)","Inclusion Criteria:\n\n1. Participant must be ≥18 years of age at the time of signing the informed consent form.\n2. Confirmed GAA enzyme deficiency from any tissue source and\u002For confirmed biallelic GAA gene mutations.\n3. Undergone enzyme replacement treatment (ERT) (either alglucosidase alfa (Lumizyme®) or avalglucosidase alfa-ngpt (Nexviazyme®)), for at least 6 months (at least 10 infusions) before signing the initial informed consent form. During the screening process, participants need to remain on their current ERT until close to dosing;\n4. FVC in the upright position ≥30% and ≤80% of predicted;\n5. Capable of walking at least 100 meters in the 6MWT (use of a cane, quad cane, or standard walker is permitted);\n6. Contraceptive\u002Fbarrier use by men and women requirements as per protocol.\n7. Capable of giving informed consent and able to understand and comply with all study procedures.\n\nExclusion Criteria:\n\n1. Severe cardiomyopathy, defined as left ventricular ejection fraction (LVEF) \\\u003C40% or New York Heart Association (NYHA) functional class 3 or above;\n2. Require invasive mechanical ventilation, or rely on noninvasive ventilation during the day;\n3. Intolerance to ERT or investigator-assessed intolerance to ERT, prior experience of serious ERT-related infusion-associated reactions (IARs);\n4. Have known intrinsic liver diseases, including hepatitis, HIV-related liver disease, prior diagnosis of portal hypertension, splenomegaly, hepatic encephalopathy, severe fatty liver, cirrhosis or liver fibrosis ≥stage 2, ultrasound-identified liver neoplasms, or laboratory tests suggesting elevated alpha-fetoprotein. Patients with liver function tests including ALT or AST \\>3× upper limit of normal (ULN) or any total bilirubin above ULN during screening will also be excluded;\n5. Prior or ongoing medical condition(s), physical finding(s), assessment findings, or laboratory abnormality that, in the investigator's opinion, would impact participant's safety and compliance with the study procedures.\n6. Have received gene therapy prior to screening;\n7. Have received any systemic immunosuppressants (except inhalation or topical use) other than glucocorticoids or investigator-recommended immunosuppressants 30 days prior to screening through completion of screening, and\u002For known intolerance to immunosuppressants such as glucocorticoids;\n8. Use of investigational drugs or drugs that could affect this study as evaluated by the investigator within 30 days prior to screening through completion of Week 52 or within 5 half-lives of the investigational drug (whichever is longer);\n9. Have received any vaccine within 30 days prior to dosing;\n10. Other conditions that make the participant not eligible for the study according to the investigator.","18 Years",{"count":80,"type":22},12,[82,25],"PHASE1","This is a single-arm, open-label, dose-escalation study to evaluate the safety, tolerability and efficacy of a single intravenous infusion of AB-1009 in adult participants with late-onset Pompe disease (LOPD).",[85,86,87],"Pompe Disease (Late-onset)","Pompe Disease Late-Onset","LOPD",[89,90,91,92,93,94,95,96,87,97,98,99,100],"Pompe Disease","Glycogen Storage Disease","Lysosomal Storage Diseases","Acid Maltase Deficiency","Acid Maltase Deficiency Disease","Gene Therapy","AB-1009","Neuromuscular Disease","Acid-Alpha Glucoside (GAA)","GAA gene","Adeno-Associated Virus (AAV)","Late-Onset Pompe Disease","2026-05-27",{"date":103,"type":35},"2026-05-28",{"date":105,"type":35},"2026-04-15",{"date":107,"type":22},"2032-09",{"name":41,"class":42},9,{"id":111,"slug":112,"hasResults":11,"nctId":113,"briefTitle":114,"officialTitle":115,"acronym":4,"eligibilityCriteria":116,"healthyVolunteers":11,"sex":17,"minAge":78,"maxAge":117,"enrollmentInfo":118,"targetDuration":4,"studyType":23,"phases":120,"briefSummary":121,"conditions":122,"keywords":132,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":138,"lastUpdatePostDateStruct":139,"startDateStruct":141,"completionDateStruct":143,"leadSponsor":145,"locationsCount":146},"100456966","phase-1-a-study-to-evaluate-the-safety-of-ab-1003-previously-lion-101-in-subjects-with-genetic-confirmation-of-lgmd2ir9-part1-100456966","NCT05230459","A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I\u002FR9 (Part1)","A Two-part Multicenter Study: a Randomized, Double-blind, Placebo-controlled Dose-escalation Safety Phase (Part 1) Followed by Double-blind, Placebo-controlled, Adaptive Phase (Part 2) Study to Evaluate the Safety and Efficacy of AB-1003 in Adult Subjects With LGMD2I\u002FR9 Mutations in the Gene Encoding Fukutin Related Protein (FKRP)","Inclusion Criteria:\n\n1. Male and female subjects aged 18 and 65 years with clinical diagnosis of LGMD2I\u002FR9 and confirmation of FKRP gene mutation.\n2. Ability to walk\u002Frun 10 meters in \\\u003C30 seconds.\n3. Able to understand and comply with all study procedures.\n4. Sexually active females of childbearing potential and female and male partners of male subjects receiving study intervention must use a barrier method of contraception for the first 6 months after dosing.\n\nExclusion Criteria:\n\n1. Significant cardiomyopathy as defined by echocardiogram (left ventricular ejection fraction \\\u003C40%), evidence of conduction defect (increased PR and RR intervals, left bundle branch block and QTcF \\>480m\u002Fsec), NYHA Class 3 or 4 heart failure, or MRI gadolinium enhancement evidence of clinically important myocardial fibrosis.\n2. Contraindication to MRI or hypersensitivity to contrast dyes, shellfish or iodine.\n3. Implanted spinal rods, cardiac pacemaker or other implantation that would distort cardiac MRI images.\n4. History of active, ongoing chronic liver disease (e.g. hepatitis, HIV-related liver disease, hemochromatosis, steatosis, etc.) or abnormal liver function tests (abnormal GGT and\u002For abnormal total\u002Fdirect bilirubin \\>upper limit of normal \\[ULN\\] and\u002For elevated AST and ALT \\>2 ULN).\n5. Abnormal renal function (GFR \\\u003C60 ml\u002Fmin, using the Modification of Diet in Renal Disease equation).\n6. Any life-threatening disease, including malignant neoplasms and medical history or malignant neoplasms within the past 5 years prior to screening (except basal and squamous cell skin cancer).\n7. In the opinion of the investigator, a pre-existing medical condition that predisposes the subject to risks that outweighs the potential benefits.\n8. Requirement for daytime ventilatory support.\n9. Change in glucocorticosteroid treatment within 3 months prior to screening visit.\n10. Exposure to another investigational drug within 3 months prior to study treatment or any previous treatment with gene therapy.\n11. Ongoing participation in any other therapeutic clinical trial.\n12. Neutralizing antibody titer to AAV9 \\>1:5.\n13. Female subjects who are pregnant, plan to become pregnant in the next 12 months, or breastfeeding.","65 Years",{"count":119,"type":22},10,[82,25],"The purpose of this study is to evaluate the safety and tolerability of a single intravenous infusion of AB-1003 in adults diagnosed with limb girdle muscular dystrophy type 2I\u002FR9 (LGMD2I\u002FR9). Participants will be treated in sequential, dose-level cohorts. (Part 1)",[123,124,125,126,127,128,129,130,131],"Limb Girdle Muscular Dystrophy","Limb-Girdle Muscular Dystrophy Type 2","LGMD2I","Muscular Dystrophy","LGMD2","LGMD","FKRP","FKRP Mutation","Fukutin Related Protein",[133,125,134,135,129,136,137],"gene therapy","LGMD2I\u002FR9","gene augmentation therapy","fukutin related protein","FKRP mutation","2026-02-18",{"date":140,"type":35},"2026-02-20",{"date":142,"type":35},"2023-05-15",{"date":144,"type":22},"2032-12",{"name":41,"class":42},6,""]