[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"trial:NCT07281079":3,"trial-entities:NCT07281079":235,"trial-summary:NCT07281079":239},{"id":4,"nct_id":4,"org_study_id":5,"brief_title":6,"official_title":7,"overall_status":8,"completion_date":9,"status_verified_date":10,"last_update_date":11,"start_date":12,"sponsor_name":13,"lead_sponsor_class":14,"has_dmc":15,"brief_summary":16,"detailed_description":17,"conditions":18,"keywords":20,"study_type":24,"primary_purpose":25,"phases":26,"enrollment_info":28,"interventions":31,"primary_outcomes":41,"secondary_outcomes":49,"sex":68,"minimum_age":69,"maximum_age":70,"healthy_volunteers":71,"eligibility_criteria":72,"std_ages":76,"locations":78,"central_contacts":229,"overall_officials":232,"references":233,"see_also_links":234},"NCT07281079","NEU-2591-PMS-301","A Study of NNZ-2591 in Pediatric Participants With Phelan-McDermid Syndrome","A Phase 3 Randomized, Double-blind, Placebo-controlled Study to Investigate the Efficacy and Safety of Orally Administered NNZ-2591 Compared With Placebo in Pediatric Participants With Phelan-McDermid Syndrome","RECRUITING","2027-11-15","2026-08","2026-08-25","2025-11-12","Neuren Pharmaceuticals Limited","INDUSTRY",true,"This Phase 3, randomized, double-blind, parallel-group (2-arm), placebo-controlled, multicenter study will evaluate the efficacy and safety of NNZ-2591 compared to placebo in pediatric participants with Phelan- McDermid Syndrome.","After providing informed consent\u002Fassent, pediatric participants with Phelan-McDermid syndrome ages 3-12 years of age will enter the 4-week Screening Period and undergo assessments for eligibility, baseline characteristics and symptom severity.\n\nOnce eligibility is confirmed, participants will be randomized in a 1:1 ratio to receive either orally administered NNZ-2591 or matching placebo during the 13-week Treatment Period. Subsequently, a 2-week safety follow-up period will occur immediately after the completion of the Treatment Period.",[19],"Phelan-McDermid Syndrome",[21,5,22,23],"Phelan McDermid Syndrome","NNZ-2591","Neuren","INTERVENTIONAL","TREATMENT",[27],"PHASE3",{"count":29,"type":30},160,"ESTIMATED",[32,37],{"type":33,"name":22,"description":34,"armGroupLabels":35},"DRUG","The study drug will be administered twice daily orally.",[36],"NNZ-2591 Arm",{"type":33,"name":38,"description":34,"armGroupLabels":39},"Placebo",[40],"Placebo Arm",[42,46],{"measure":43,"description":44,"timeFrame":45},"Efficacy of NNZ-2591 compared with placebo as measured by the Phelan-McDermid Syndrome Assessment of Change (PMSA-C) overall score.","Efficacy of NNZ-2591 compared with placebo as measured by the Phelan-McDermid Syndrome Assessment of Change (PMSA-C) overall score. The PMSA-C scores range from 1 to 7 with 1 indicating very much improved and 7 indicating very much worse.","Week 13",{"measure":47,"description":48,"timeFrame":45},"Efficacy of NNZ-2591 compared with placebo as measured by the change from baseline in the Vineland Adaptive Behavior Scales-3, Interview version (Vineland-3) receptive communication subdomain raw score.","Efficacy of NNZ-2591 compared with placebo as measured by the change from baseline in the Vineland Adaptive Behavior Scales-3, Interview version (Vineland-3) receptive communication subdomain raw score. A higher raw score for the receptive communication subdomain indicates better adaptive behavior.",[50,53,56,59,62,65],{"measure":51,"description":52,"timeFrame":45},"Efficacy of NNZ-2591 compared with placebo as measured by the Caregiver Impression of Change (CIC) overall score.","Efficacy of NNZ-2591 compared with placebo as measured by the Caregiver Impression of Change (CIC) overall score. The CIC scores range from 1 to 7 with 1 indicating very much improved and 7 indicating very much worse.",{"measure":54,"description":55,"timeFrame":45},"Efficacy of NNZ-2591 compared with placebo as measured by the Phelan-McDermid Syndrome Assessment of Change (PMSA-C) domain scores.","Efficacy of NNZ-2591 compared with placebo as measured by the Phelan-McDermid Syndrome Assessment of Change (PMSA-C) domain scores. The PMSA-C domain scores range from 1 to 7 with 1 indicating very much improved and 7 indicating very much worse.",{"measure":57,"description":58,"timeFrame":45},"Efficacy of NNZ-2591 compared with placebo as measured by the Caregiver Impression of Change (CIC) domain scores.","Efficacy of NNZ-2591 compared with placebo as measured by the Caregiver Impression of Change (CIC) domain scores. The CIC scores range from 1 to 7 with 1 indicating very much improved and 7 indicating very much worse.",{"measure":60,"description":61,"timeFrame":45},"Efficacy of NNZ-2591 compared with placebo as measured by the change from baseline in Phelan-McDermid Syndrome Assessment of Severity (PMSA-S) domain scores.","Efficacy of NNZ-2591 compared with placebo as measured by the change from baseline in Phelan-McDermid Syndrome Assessment of Severity (PMSA-S) domain scores. The PMSA-S scores range from 1 to 7 with 1 indicating typical for age, not at all impaired and 7 among the most severely impaired.",{"measure":63,"description":64,"timeFrame":45},"Efficacy of NNZ-2591 compared with placebo as measured by the change from baseline in PMSA-S overall score.","Efficacy of NNZ-2591 compared with placebo as measured by the change from baseline in PMSA-S overall score. The PMSA-S scores range from 1 to 7 with 1 indicating typical for age, not at all impaired and 7 among the most severely impaired.",{"measure":66,"description":67,"timeFrame":45},"Efficacy of NNZ-2591 compared with placebo as measured by the change from baseline in PMS Clinician Domain Specific Rating Scale (PMS-DSRS) scores.","Efficacy of NNZ-2591 compared with placebo as measured by the change from baseline in PMS Clinician Domain Specific Rating Scale (PMS-DSRS) scores. The PMS-DSRS scores range from 0 to 4 with 0 indicating Symptom Not Present and 4 indicating Very Severe.","ALL","3 Years","12 Years",false,{"inclusion":73,"exclusion":74,"raw_text":75},[],[],"Inclusion Criteria:\n\n1. Male or female pediatric participants with Phelan-McDermid syndrome ages 3 to 12 years (inclusive) at the time of signing the informed consent.\n2. Clinical diagnosis of Phelan-McDermid syndrome with a documented disease-causing genetic abnormality of SHANK3.\n3. Body weight ≥ 10 kg at Screening.\n4. Participants with a PMSA-S overall score ≥ 3 at the Screening and Baseline visits.\n5. Not actively undergoing regression or loss of skills.\n\nExclusion Criteria:\n\n1. Use of exclusionary medication or unstable treatment regimens of acceptable concomitant medications as required by the protocol.\n2. Current treatment with more than 3 allowable psychotropic medications.\n3. Participants with seizures must be controlled on no more than 2 anticonvulsant medications (not counting rescue medications).\n4. Psychotropic medications or any other medication used for a chronic illness (not including antibiotics, pain relievers, anti-diarrheals, and laxatives) with doses and dosing regimen that have not been stable for at least 4 weeks before Screening. If the treatment was discontinued, the discontinuation must have occurred no fewer than 2 weeks before the start of Screening.\n5. Any intercurrent seizures in the past 6 months and \u002For more than 1 seizure in the past 12 months. •A single febrile seizure in the 6 months prior to screening is allowable if no rescue medication was required.\n6. Abnormal liver function laboratory results during the Screening period, as defined by the protocol\n7. Abnormal QT interval on Screening ECG as defined by the protocol.",[77],"CHILD",[79,93,103,112,121,130,140,150,160,169,179,188,198,208,218],{"facility":80,"status":8,"city":81,"state":82,"zip":83,"country":84,"contacts":85,"geoPoint":90},"Neuren PMS-301 Site #116","Birmingham","Alabama","35233","United States",[86],{"name":87,"role":88,"phone":89,"email":87},"medicalinformation@neurenpharma.com","CONTACT","231-203-8050",{"lat":91,"lon":92},33.52066,-86.80249,{"facility":94,"status":8,"city":95,"state":96,"zip":97,"country":84,"contacts":98,"geoPoint":100},"Neuren PMS-301 Site#122","Glendale","California","91203",[99],{"name":87,"role":88,"phone":89,"email":87},{"lat":101,"lon":102},34.14251,-118.25508,{"facility":104,"status":8,"city":105,"state":96,"zip":106,"country":84,"contacts":107,"geoPoint":109},"Neuren PMS-301 Site #112","Palo Alto","94305",[108],{"name":87,"role":88,"phone":89,"email":87},{"lat":110,"lon":111},37.44188,-122.14302,{"facility":113,"status":8,"city":114,"state":96,"zip":115,"country":84,"contacts":116,"geoPoint":118},"Neuren PMS-301 Site#103","San Diego","92123",[117],{"name":87,"role":88,"phone":89,"email":87},{"lat":119,"lon":120},32.71571,-117.16472,{"facility":122,"status":8,"city":123,"state":96,"zip":124,"country":84,"contacts":125,"geoPoint":127},"Neuren PMS-301 Site#111","San Rafael","94903",[126],{"name":87,"role":88,"phone":89,"email":87},{"lat":128,"lon":129},37.97353,-122.53109,{"facility":131,"status":8,"city":132,"state":133,"zip":134,"country":84,"contacts":135,"geoPoint":137},"Neuren PMS-301 Site#102","Chicago","Illinois","60612",[136],{"name":87,"role":88,"phone":89,"email":87},{"lat":138,"lon":139},41.85003,-87.65005,{"facility":141,"status":8,"city":142,"state":143,"zip":144,"country":84,"contacts":145,"geoPoint":147},"Neuren PMS-301 Site#109","Chevy Chase","Maryland","20815",[146],{"name":87,"role":88,"phone":89,"email":87},{"lat":148,"lon":149},39.00287,-77.07115,{"facility":151,"status":8,"city":152,"state":153,"zip":154,"country":84,"contacts":155,"geoPoint":157},"Neuren PMS-301 Site#106","Brookline","Massachusetts","02445",[156],{"name":87,"role":88,"phone":89,"email":87},{"lat":158,"lon":159},42.33176,-71.12116,{"facility":161,"status":8,"city":162,"state":153,"zip":163,"country":84,"contacts":164,"geoPoint":166},"Neuren PMS-301 Site#104","Lexington","02421",[165],{"name":87,"role":88,"phone":89,"email":87},{"lat":167,"lon":168},42.44732,-71.2245,{"facility":170,"status":8,"city":171,"state":172,"zip":173,"country":84,"contacts":174,"geoPoint":176},"Neuren PMS-301 Site #117","Ann Arbor","Michigan","48109",[175],{"name":87,"role":88,"phone":89,"email":87},{"lat":177,"lon":178},42.27756,-83.74088,{"facility":180,"status":8,"city":181,"state":181,"zip":182,"country":84,"contacts":183,"geoPoint":185},"Neuren PMS-301 Site#101","New York","10029",[184],{"name":87,"role":88,"phone":89,"email":87},{"lat":186,"lon":187},40.71427,-74.00597,{"facility":189,"status":8,"city":190,"state":191,"zip":192,"country":84,"contacts":193,"geoPoint":195},"Neuren PMS-301 Site#108","Cincinnati","Ohio","45229",[194],{"name":87,"role":88,"phone":89,"email":87},{"lat":196,"lon":197},39.12711,-84.51439,{"facility":199,"status":8,"city":200,"state":201,"zip":202,"country":84,"contacts":203,"geoPoint":205},"Neuren PMS-301 Site#115","Houston","Texas","77030",[204],{"name":87,"role":88,"phone":89,"email":87},{"lat":206,"lon":207},29.76328,-95.36327,{"facility":209,"status":8,"city":210,"state":211,"zip":212,"country":84,"contacts":213,"geoPoint":215},"Neuren PMS-301 Site #113","Charlottesville","Virginia","22903",[214],{"name":87,"role":88,"phone":89,"email":87},{"lat":216,"lon":217},38.02931,-78.47668,{"facility":219,"status":8,"city":220,"state":221,"zip":222,"country":223,"contacts":224,"geoPoint":226},"Neuren PMS-301 Site#201","Toronto","Ontario","M4G 1R8","Canada",[225],{"name":87,"role":88,"phone":89,"email":87},{"lat":227,"lon":228},43.70643,-79.39864,[230],{"name":231,"role":88,"phone":89,"email":87},"Medical Information Lead",[],[],[],{"nct_id":4,"conditions":236,"biomarkers":237},[19],[238],"SHANK3 Gene",{"nct_id":4,"found":15,"summary":240,"prompt_version":250},{"design":241,"status":242,"heading":243,"summary":244,"follow_up":245,"word_count":246,"commitments":247,"compensation":248,"drugs_mentioned":249},"This is a Phase 3, randomized, double-blind study, meaning participants will be randomly assigned to receive either NNZ-2591 or a placebo, and neither you nor the study team will know which you are receiving. It plans to enroll 160 participants.","completed","Study of NNZ-2591 for Phelan-McDermid Syndrome in Children","This study is testing a medication called NNZ-2591 for children with Phelan-McDermid Syndrome. We want to see if NNZ-2591 can help improve symptoms compared to a placebo (an inactive substance). Children aged 3 to 12 years with a confirmed diagnosis of Phelan-McDermid Syndrome and a specific genetic change (SHANK3 abnormality) can join. We will measure success by looking at changes in a special assessment for Phelan-McDermid Syndrome (PMSA-C) and improvements in communication skills (Vineland-3). The current status of this study is unclear, but it plans to enroll 160 participants.","There will be a 2-week safety follow-up period immediately after the 13-week treatment period.",89,"After an initial 4-week screening period, participants will take either NNZ-2591 or placebo twice daily orally for 13 weeks.","Not stated in the trial record.",[22,38],null]