[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"trial:NCT05996003":3,"trial-entities:NCT05996003":414,"trial-summary:NCT05996003":418},{"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":22,"study_type":25,"primary_purpose":26,"phases":27,"enrollment_info":29,"interventions":32,"primary_outcomes":39,"secondary_outcomes":64,"sex":90,"minimum_age":91,"maximum_age":92,"healthy_volunteers":93,"eligibility_criteria":94,"std_ages":110,"locations":112,"central_contacts":403,"overall_officials":408,"references":409,"see_also_links":410},"NCT05996003","NS-089\u002FNCNP-02-201","NS-089\u002FNCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)","A Phase 2 Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of NS-089\u002FNCNP-02 in Boys With Duchenne Muscular Dystrophy (DMD)","RECRUITING","2026-09-11","2026-03","2026-03-06","2024-02-22","NS Pharma, Inc.","INDUSTRY",true,"This is a Phase 2, open-label, multi-center, 2-part study of NS-089\u002FNCNP-02 administered by weekly IV infusion to ambulant boys aged ≥4 to \\\u003C15 years with DMD due to mutations amenable to exon 44 skipping. Participants will receive a selected dose of NS-089\u002FNCNP-02 administered once weekly.\n\nThe study consists of 2 parts: Part 1 and Part 2. Six participants (Cohort 1) will participate in both Part 1 and Part 2, and 14 participants (Cohort 2) will be added for Part 2.",null,[19,20,21],"Duchenne Muscular Dystrophy","Exon 44","DMD",[23,21,24],"Exon 44 Skipping","Brogidirsen","INTERVENTIONAL","TREATMENT",[28],"PHASE2",{"count":30,"type":31},20,"ESTIMATED",[33],{"type":34,"name":35,"description":36,"armGroupLabels":37,"otherNames":38},"DRUG","NS-089\u002FNCNP-02","Cohort 1:\n\nPart 1 Dose Level 1-3: a 4-week Treatment Phase at each treatment dose level\n\nPart 2 Single Dose Level: a 24-week Treatment Phase at the MTD of Part 1\n\nCohort 2:\n\nPart 2 Single Dose Level: a 24-week Treatment Phase at the MTD of Part 1",[35],[24],[40,43,46,48,50,52,54,56,58,61],{"measure":41,"timeFrame":42},"Adverse Event and Adverse Drug Reaction","through study completion, up to follow-up phone call for Part 2",{"measure":44,"timeFrame":45},"Plasma pharmacokinetic (PK) parameters","Day1, Week4 for each dose for Part 1, Day1 and Week24 for Part 2] Maximum plasma concentration (Cmax) of NS-089\u002FNCNP-02",{"measure":44,"timeFrame":47},"Day1, Week4 for each dose for Part 1, Day1 and Week24 for Part 2] Time of the maximum plasma concentration (Tmax) of NS-089\u002FNCNP-02",{"measure":44,"timeFrame":49},"Day1, Week4 for each dose for Part 1, Day1 and Week24 for Part 2] Terminal half-life (T1\u002F2) of NS-089\u002FNCNP-02",{"measure":44,"timeFrame":51},"Day1, Week4 for each dose for Part 1, Day1 and Week24 for Part 2] Area under the concentration-time curve from time 0 to the last time point (AUC0-t) of NS-089\u002FNCNP-02",{"measure":44,"timeFrame":53},"Day1, Week4 for each dose for Part 1, Day1 and Week24 for Part 2] Area under the concentration-time curve from time 0 to infinity (AUC0-∞) of NS-089\u002FNCNP-02",{"measure":44,"timeFrame":55},"[Time Frame: Day1, Week4 for each dose for Part 1, Day1 and Week24 for Part 2] Total body clearance (CLtot) of NS-089\u002FNCNP-02",{"measure":44,"timeFrame":57},"Day1, Week4 for each dose for Part 1, Day1 and Week24 for Part 2] The volume in the terminal state (Vz) of NS-089\u002FNCNP-02",{"measure":59,"timeFrame":60},"Urine pharmacokinetic parameters","Day1, Week4 for each dose for Part 1, Day1 and Week24 for Part 2] Urinary excretion of NS-089\u002FNCNP-02",{"measure":62,"timeFrame":63},"Change from baseline in skeletal muscle dystrophin protein by immunoblot (Western blot).","Baseline, Week25",[65,67,69,71,75,77,79,81,83,85,87],{"measure":66,"timeFrame":63},"Change from baseline in skeletal muscle dystrophin protein by mass spectrometry.",{"measure":68,"timeFrame":63},"Change from baseline in skeletal muscle dystrophin protein levels by immunofluorescence staining.",{"measure":70,"timeFrame":63},"Change from baseline in percentage of exon 44-skipped mRNA of skeletal muscle dystrophin",{"measure":72,"description":73,"timeFrame":74},"North Star Ambulatory Assessment (NSAA) score","The NSAA is a functional scale devised for use in ambulant children with Duchenne muscular dystrophy (DMD). It consists of 17 activities graded 0 (unable to perform), 1 (performs with modifications), 2 (normal movement). It assesses abilities necessary to remain ambulant that have been found to progressively deteriorate in untreated DMD patients, as well as in other muscular dystrophies such as Becker Muscular Dystrophy. NSAA Total Score ranges from 0 to 34, with a score of 34 implying normal function.","Baseline, Week13, Week25",{"measure":76,"timeFrame":74},"Time to Run\u002FWalk 10 Meters (TTRW)",{"measure":78,"timeFrame":74},"Time to Stand (TTSTAND)",{"measure":80,"timeFrame":74},"Total distance of 6 Minute Walk Test (6MWT)",{"measure":82,"timeFrame":74},"Time to Climb 4 Stairs (TTCLIMB)",{"measure":84,"timeFrame":74},"Muscle strength measured by Quantitative Muscle Testing (QMT)",{"measure":86,"timeFrame":74},"Grip and pinch strength",{"measure":88,"description":89,"timeFrame":74},"Performance of Upper Limb (PUL) 2.0. score","The PUL 2.0 provides both a total score and sub-scores for the 3 domains (shoulder, middle, and distal) that in DMD are progressively involved with a proximal to distal gradient. The PUL includes 22 items with an entry item to define the starting functional level. The 22 items are subdivided into the high-level shoulder dimension (6 items), middle level elbow dimension (9 items), and distal wrist and hand dimension (7 items). For weaker patients, a low score on the entry item (0-2) means high level items do not need to be performed. Scoring options vary across the scale between 0-1 and 0-2 according to performance. Each dimension can be scored separately with a maximum score of 12 for the high-level shoulder dimension, 17 for the middle level elbow dimension, and 13 for the distal wrist and hand dimension. A total score can be achieved by adding the 3 level scores (maximum total score of 42).","MALE","4 Years","14 Years",false,{"inclusion":95,"exclusion":101,"raw_text":109},[96,97,98,99,100],"Male ≥ 4 years and \\\u003C15 years of age","Confirmed DMD mutation(s) in the dystrophin gene that is amenable to skipping of exon 44 to restore the dystrophin mRNA reading frame","Able to walk independently without assistive devices","Ability to complete the TTSTAND without assistance in \\\u003C20 seconds","Stable dose of glucocorticoid for at least 3 months and the dose is expected to remain on a stable dose for the duration of the study.",[102,103,104,105,106,107,108],"Has a body weight of \\\u003C20 kg at the time of informed consent (applies to participants screening for Part 1 only)","Evidence of symptomatic cardiomyopathy","Current or previous treatment with anabolic steroids (e.g., oxandrolone) or products containing resveratrol or adenosine triphosphate within 3 months prior to first dose of study drug","Current or previous treatment with any other investigational drug within 3 months prior to the first dose of study drug or within 5 times the half-life of a medication, whichever is longer","Surgery within the 3 months prior to the first dose of study drug or planned during the study duration","Previously treated in an interventional study of NS-089\u002FNCNP-02","Having received exon skipping oligonucleotide within 1 year prior to the first dose of IP","Inclusion Criteria:\n\n* Male ≥ 4 years and \\\u003C15 years of age\n* Confirmed DMD mutation(s) in the dystrophin gene that is amenable to skipping of exon 44 to restore the dystrophin mRNA reading frame\n* Able to walk independently without assistive devices\n* Ability to complete the TTSTAND without assistance in \\\u003C20 seconds\n* Stable dose of glucocorticoid for at least 3 months and the dose is expected to remain on a stable dose for the duration of the study.\n* Other inclusion criteria may apply.\n\nExclusion Criteria:\n\n* Has a body weight of \\\u003C20 kg at the time of informed consent (applies to participants screening for Part 1 only)\n* Evidence of symptomatic cardiomyopathy\n* Current or previous treatment with anabolic steroids (e.g., oxandrolone) or products containing resveratrol or adenosine triphosphate within 3 months prior to first dose of study drug\n* Current or previous treatment with any other investigational drug within 3 months prior to the first dose of study drug or within 5 times the half-life of a medication, whichever is longer\n* Surgery within the 3 months prior to the first dose of study drug or planned during the study duration\n* Previously treated in an interventional study of NS-089\u002FNCNP-02\n* Having received exon skipping oligonucleotide within 1 year prior to the first dose of IP\n* Other exclusion criteria may apply.",[111],"CHILD",[113,127,139,151,166,178,190,202,213,225,237,251,263,274,285,294,306,318,327,335,347,359,369,381,392],{"facility":114,"status":8,"city":115,"state":116,"zip":117,"country":118,"contacts":119,"geoPoint":124},"Children's Hospital Colorado","Aurora","Colorado","80045","United States",[120],{"name":121,"role":122,"email":123},"Hannah Kleiner","CONTACT","hannah.kleiner@childrenscolorado.org",{"lat":125,"lon":126},39.72943,-104.83192,{"facility":128,"status":8,"city":129,"state":130,"zip":131,"country":118,"contacts":132,"geoPoint":136},"Rare Disease Research, LLC - FL","Kissimmee","Florida","34746",[133],{"name":134,"role":122,"email":135},"Marcial Almaraz","marcial.almaraz@rarediseaseresearch.com",{"lat":137,"lon":138},28.30468,-81.41667,{"facility":140,"status":8,"city":141,"state":142,"zip":143,"country":118,"contacts":144,"geoPoint":148},"Rare Disease Research","Atlanta","Georgia","30329",[145],{"name":146,"role":122,"email":147},"Julia Zhu","julia.zhu@rarediseaseresearch.com",{"lat":149,"lon":150},33.749,-84.38798,{"facility":152,"status":8,"city":153,"state":154,"zip":155,"country":118,"contacts":156,"geoPoint":163},"Ann and Robert H. Lurie Children's Hospital of Chicago","Chicago","Illinois","60611",[157,160],{"name":158,"role":122,"email":159},"Raven Hill","rhill@luriechildrens.org",{"name":161,"role":122,"email":162},"Alka Maheshwari","amaheshwari@luriechildrens.org",{"lat":164,"lon":165},41.85003,-87.65005,{"facility":167,"status":8,"city":168,"state":169,"zip":170,"country":118,"contacts":171,"geoPoint":175},"University of Kansas Medical Center (KUMC)","Kansas City","Kansas","66160",[172],{"name":173,"role":122,"email":174},"Rebecca Clay","rclay@kumc.edu",{"lat":176,"lon":177},39.11417,-94.62746,{"facility":179,"status":8,"city":180,"state":181,"zip":182,"country":118,"contacts":183,"geoPoint":187},"Cincinnati Children's Hospital Medical Center","Cincinnati","Ohio","45229",[184],{"name":185,"role":122,"email":186},"Angela Edmondson","Angela.Edmondson@cchmc.org",{"lat":188,"lon":189},39.12711,-84.51439,{"facility":191,"status":8,"city":192,"state":193,"zip":194,"country":118,"contacts":195,"geoPoint":199},"The Children's Hospital of Philadelphia (CHOP)","Philadelphia","Pennsylvania","19104",[196],{"name":197,"role":122,"email":198},"Karen Monono","mononok@email.chop.edu",{"lat":200,"lon":201},39.95238,-75.16362,{"facility":203,"status":8,"city":204,"state":193,"zip":205,"country":118,"contacts":206,"geoPoint":210},"University of Pittsburgh School of Medicine","Pittsburgh","15224",[207],{"name":208,"role":122,"email":209},"Vibha Chauhan","vibha.chauhan@chp.edu",{"lat":211,"lon":212},40.44062,-79.99589,{"facility":214,"status":8,"city":215,"state":216,"zip":217,"country":118,"contacts":218,"geoPoint":222},"UT Southwestern\u002FChildren's Health","Dallas","Texas","75207",[219],{"name":220,"role":122,"email":221},"Deniece Winston","Deniece.Winston@UTSouthwestern.edu",{"lat":223,"lon":224},32.78306,-96.80667,{"facility":226,"status":8,"city":227,"state":228,"zip":229,"country":118,"contacts":230,"geoPoint":234},"Virginia Commonwealth University Health System","Richmond","Virginia","23298",[231],{"name":232,"role":122,"email":233},"Andrea Jewell","andrea.jewell@vcuhealth.org",{"lat":235,"lon":236},37.55376,-77.46026,{"facility":238,"status":239,"city":240,"state":241,"zip":242,"country":243,"contacts":244,"geoPoint":248},"Perth Children's Hospital","NOT_YET_RECRUITING","Nedlands","Western Australia","6009","Australia",[245],{"name":246,"role":122,"email":247},"Jane Jones","Jane.Jones2@health.wa.gov.au",{"lat":249,"lon":250},-31.98184,115.8073,{"facility":252,"status":239,"city":253,"state":254,"country":255,"contacts":256,"geoPoint":260},"Alberta Children's Hospital","Calgary","Alberta","Canada",[257],{"name":258,"role":122,"email":259},"Julie Dao","julie.dao@ucalgary.ca",{"lat":261,"lon":262},51.05011,-114.08529,{"facility":264,"status":239,"city":265,"state":266,"country":255,"contacts":267,"geoPoint":271},"British Columbia Children's Hospital","Vancouver","British Columbia",[268],{"name":269,"role":122,"email":270},"Gabe Massarotto","gabe.massarotto@cw.bc.ca",{"lat":272,"lon":273},49.24966,-123.11934,{"facility":275,"status":8,"city":276,"state":277,"country":255,"contacts":278,"geoPoint":282},"London Health Sciences Centre","London","Ontario",[279],{"name":280,"role":122,"email":281},"Rhiannon Hicks","rhiannon.hicks@lhsc.on.ca",{"lat":283,"lon":284},42.98339,-81.23304,{"facility":286,"status":239,"city":287,"state":288,"zip":289,"country":290,"geoPoint":291},"Fukui Prefectural Hospital","Fukui-shi","Fukui","910-8526","Japan",{"lat":292,"lon":293},36.06443,136.22257,{"facility":295,"status":8,"city":296,"state":297,"zip":298,"country":290,"contacts":299,"geoPoint":303},"National Hospital Organization Nagara Medical Center","Nagara","Gifu-shi, Gifu","502-8558",[300],{"name":301,"role":122,"email":302},"Clinical Trial Management Office","yonekura.kei.wu@mail.hosp.go.jp",{"lat":304,"lon":305},28.25,129.25,{"facility":307,"status":239,"city":308,"state":309,"zip":310,"country":290,"contacts":311,"geoPoint":315},"NHO Osaka Toneyama Medical Center","Toyonaka","Osaka","560-8552",[312],{"name":313,"role":122,"email":314},"Clinical Trial Office","410-chiken@mail.hosp.go.jp",{"lat":316,"lon":317},34.78244,135.46932,{"facility":319,"status":239,"city":320,"state":321,"zip":322,"country":290,"contacts":323},"Shiga General Hospital","Moriyama-shi","Shiga","524-8524",[324],{"name":325,"role":122,"email":326},"Children's Building","kenji.s.address@gmail.com",{"facility":328,"status":239,"city":329,"state":330,"zip":331,"country":290,"geoPoint":332},"National Center of Neurology and Psychiatry","Kodaira","Tokyo","187-8551",{"lat":333,"lon":334},35.72603,139.48508,{"facility":336,"status":239,"city":337,"zip":338,"country":339,"contacts":340,"geoPoint":344},"Starship Children's Hospital","Auckland","1023","New Zealand",[341],{"name":342,"role":122,"email":343},"Margaret Joppa","mjoppa@adhb.govt.nz",{"lat":345,"lon":346},-36.84853,174.76349,{"facility":348,"status":239,"city":349,"state":350,"country":351,"contacts":352,"geoPoint":356},"Pusan National University Yangsan Hospital","Yangsan","Gyeongsangnam","South Korea",[353],{"name":354,"role":122,"email":355},"JaeYoon Kim","lusterwuzz@naver.com",{"lat":357,"lon":358},35.34199,129.03358,{"facility":360,"status":239,"city":361,"country":351,"contacts":362,"geoPoint":366},"Seoul National University Hospital","Seoul",[363],{"name":364,"role":122,"email":365},"Hyeon Ju Kwon","hjkwon@snuh.org; 49041@snuh.org",{"lat":367,"lon":368},37.566,126.9784,{"facility":370,"status":239,"city":371,"zip":372,"country":373,"contacts":374,"geoPoint":378},"Ankara Bilkent City Hospital","Ankara","06800","Turkey (Türkiye)",[375],{"name":376,"role":122,"email":377},"Gozde Akgun","gozde.akgun@medex-smo.com",{"lat":379,"lon":380},39.91987,32.85427,{"facility":382,"status":239,"city":383,"zip":384,"country":373,"contacts":385,"geoPoint":389},"Yeditepe University Kosuyolu Hospital","Istanbul","34718",[386],{"name":387,"role":122,"email":388},"Onur Güner","onurguner@ascotscience.com",{"lat":390,"lon":391},41.01384,28.94966,{"facility":393,"status":239,"city":394,"zip":395,"country":373,"contacts":396,"geoPoint":400},"S.B.U. Dr. Behcet uz Pediatric Diseases and Surgery Training and Research Hospital","Izmir","11794",[397],{"name":398,"role":122,"email":399},"Zeynep Erturk","zeyneperturk@ascotscience.com",{"lat":401,"lon":402},38.41273,27.13838,[404],{"name":405,"role":122,"phone":406,"email":407},"Trial info","1-866-677-6276","trialinfo@nspharma.com",[],[],[411],{"label":412,"url":413},"Please follow this link for additional information on how to participate","https:\u002F\u002Fmytomorrows.com\u002Ftrials\u002Fdiscover-ns-089\u002Fen-us?utm_source=website&utm_medium=affiliate&utm_campaign=161_usa_en_web_organic_cnv_afi_link-for-ct-gov&utm_content=161_link-for-ct-gov_txt_link-for-ct-gov",{"nct_id":4,"conditions":415,"biomarkers":416},[19],[417],"DMD Gene",{"nct_id":4,"found":15,"summary":419,"prompt_version":429},{"design":420,"status":421,"heading":422,"summary":423,"follow_up":424,"word_count":425,"commitments":426,"compensation":427,"drugs_mentioned":428},"This is a Phase 2, open-label study, meaning both you and the researchers will know which treatment you are receiving. It involves 20 participants and is conducted across multiple centers.","completed","Phase 2 Study of NS-089\u002FNCNP-02 for Duchenne Muscular Dystrophy","This Phase 2 study is testing a drug called NS-089\u002FNCNP-02 in boys with Duchenne Muscular Dystrophy (DMD) who have a specific genetic change (mutation) that could benefit from \"exon 44 skipping.\" This means the drug aims to help the body make more of a protein called dystrophin, which is missing or faulty in DMD. The study is open to boys aged 4 to 14 years who can walk independently. Researchers will be looking at how safe the drug is and how the body processes it (pharmacokinetics). The study plans to enroll 20 participants, but its current status is unclear.","Participants will be followed through study completion, which includes a follow-up phone call for Part 2 of the study.",99,"Participants will receive NS-089\u002FNCNP-02 as a weekly intravenous (IV) infusion. The study has two parts, with treatment phases lasting 4 weeks at different dose levels and then a 24-week treatment phase.","Not stated in the trial record.",[35],"v2"]