[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"trial:NCT05866419":3,"trial-entities:NCT05866419":330,"trial-summary:NCT05866419":334},{"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":23,"primary_purpose":24,"phases":25,"enrollment_info":27,"interventions":30,"primary_outcomes":37,"secondary_outcomes":44,"sex":56,"minimum_age":57,"maximum_age":58,"healthy_volunteers":59,"eligibility_criteria":60,"std_ages":64,"locations":68,"central_contacts":318,"overall_officials":323,"references":324,"see_also_links":329},"NCT05866419","QT-0179","Study of an Intrathecal Port and Catheter System for Subjects With Spinal Muscular Atrophy","Safety and Performance of the ThecaFlex DRx™ System Port and Catheter for Chronic Intrathecal Access, Cerebrospinal Fluid (CSF) Aspiration, and DElivery of Nusinersen in Spinal Muscular Atrophy (SMA) Patients Resistant to Lumbar PunctuRE Trial (PIERRE)","RECRUITING","2030-07","2026-08","2026-08-19","2023-11-27","Alcyone Therapeutics, Inc","INDUSTRY",true,"The primary objective of the clinical investigation is to demonstrate successful clinical use of the ThecaFlex DRx™ System in delivering nusinersen in subjects with spinal muscular atrophy (SMA).\n\nAll enrolled subjects will undergo implantation of the investigational device (ThecaFlex DRx™ System) and will be followed for 12 months after receiving the implant. The 12-month data will be used to assess the primary endpoint support a Pre-Market Approval (PMA) application.","This is a multicenter, multi-national, prospective, non-randomized, single arm, group sequential design, pivotal clinical investigation (device exemption study) to assess the safety and performance of the ThecaFlex DRx™ System in subjects with SMA.\n\nThe subject population will include subjects aged ≥ 3 years with SMA who are considered candidates for intrathecal port and catheter implantation because they require chronic, bolus intrathecal administration of necessary therapy, and who meet all the inclusion\u002Fexclusion criteria for the clinical investigation.\n\nAfter a screening period of up to 200 days, all enrolled subjects will undergo implantation of the investigational device (ThecaFlex DRx™ System) and will be followed for 12 months after receiving the implant.\n\nIn agreement with the nusinersen dosing information, the treatment frequency will be adapted for non-naïve and naïve subjects:\n\nSubjects who are not naïve to nusinersen should have nusinersen maintenance doses administered via the implant every 4 months (i.e., 4, 8, and 12 months after their last nusinersen dose, before enrollment in the clinical investigation).\n\nSubjects who are naïve to nusinersen will have their loading and maintenance doses administered via the device according to the drug labeling.\n\nA DMC and Clinical Events Committee (CEC), independent of each other and the Sponsor, will be used to perform ongoing consistent adjudication of events related to the safety throughout the entire duration of the clinical investigation and to periodically review data that relate to the safety of the study.\n\nThe clinical investigation will enroll 90 subjects. The clinical investigation incorporates a lead-in phase which consists of 10 subjects enrolled, implanted, and followed for 30-days. Enrollment for the lead-in phase is expected to take approximately 6 to 9 months, at which point the Data Monitoring Committee (DMC) will review the data for safety and approve continuation of enrollment. The second phase of enrollment is expected to take approximately 12 to 15 months.\n\nIndividual subjects are anticipated to be enrolled in the clinical investigation for approximately 37 months. The total duration of this clinical investigation is estimated to be approximately 60 months.",[19,20,21],"Spinal Muscular Atrophy","Spine Deformity","Scoliosis",[],"INTERVENTIONAL","TREATMENT",[26],"NA",{"count":28,"type":29},90,"ESTIMATED",[31],{"type":32,"name":33,"description":34,"armGroupLabels":35},"DEVICE","ThecaFlex DRx System","The ThecaFlex DRx™ System consists of an implantable intrathecal catheter, and an implantable subcutaneous port, designed specifically to meet the implantation, Cerebrospinal Fluid (CSF) aspiration, and intrathecal delivery and dosing needs of patients (aged ≥ 3 years) requiring repeated\u002Fon-going intermittent chronic bolus intrathecal treatment for life threatening, debilitating Central Nervous System (CNS) disorders who are precluded from Lumbar Puncture (LP) due to complex spine conditions, or who are resistant to LP for therapy administration or CSF aspiration.",[36],"ThecaFlex DRx Port and Catheter System",[38,42],{"measure":39,"description":40,"timeFrame":41},"The proportion of subjects with successful Implantation of the ThecaFlex DRx™ Port and Catheter","The primary outcome measures the proportion of subjects with successful implantation of the ThecaFlex DRx™ Port and Catheter System who are able to receive successful intrathecal therapy infusion(s).","12 Months",{"measure":43,"timeFrame":41},"The proportion of subjects with successful Intrathecal Therapy Infusion(s) through 12 months",[45,48,50,52,54],{"measure":46,"timeFrame":47},"Proportion of subjects with reduced anesthesia and radiation exposure compared to repeat LP for nusinersen infusion in SMA subjects.","12 months",{"measure":49,"timeFrame":47},"Incidence of device-related adverse events (AEs)\u002Fcomplications through 12 months",{"measure":51,"timeFrame":47},"Incidence of procedural complications through 12 months",{"measure":53,"timeFrame":47},"Incidence of nusinersen-related AEs",{"measure":55,"timeFrame":47},"Mean duration of radiation exposure from implant through 12-month follow-up","ALL","3 Years",null,false,{"inclusion":61,"exclusion":62,"raw_text":63},[],[],"Inclusion Criteria:\n\n1. Subject is 3 years or older\n2. Subject is diagnosed with SMA and is a candidate for Spinraza treatment as indicated in country-specific label\n3. Subject resistant to lumbar puncture (LP), where resistance is defined as:\n\n   1. Subject with respiratory issues or other comorbidities who is at an increased risk for complications due to the need for repeat anesthesia and imaging radiation exposure to safely perform LP; or\n   2. Subjects for whom the treating physician determines implantation of the ThecaFlex DRx™ System is otherwise in the subject's best interest\n4. Subject has been prescribed nusinersen via chronic intrathecal bolus administration in accordance with the country-specific drug labeling\n5. Subject, per Investigator discretion, is able to undergo a percutaneous or open surgical procedure for index port and catheter implantation where at least 7 cm of the ThecaFlex DRx system catheter will be placed intrathecally\n6. Subject (or subject's legally authorized representative) is willing and able to provide written informed consent; and\n7. Subject and parent\u002Fcaregiver able and willing to complete all clinical investigation procedures, measurements, and visits.\n\nExclusion Criteria:\n\n1. Subject meets any of the contraindications for use of the ThecaFlex DRx System as outlined in the ThecaFlex DRx System Instructions for Use\n2. Presence or history of (\\\u003C 6 months prior to the procedure): an implanted microinfusion pump, intrathecal catheter, or other intrathecal drug delivery devices\u002Fcomponent, or implanted shunt for drainage of cerebrospinal fluid (CSF), or reservoir in the CSF space (note: any ThecaFlex Port and Catheter System must not be placed in the same location as a previous indwelling port and catheter)\n3. Subject is pregnant or nursing or plans to become pregnant during the course of the clinical investigation, or is a woman of childbearing potential or a man of reproductive age who is unwilling or unable to practice contraception, as described in Section 14.10.\n4. Any condition or event, which in the opinion of the Investigator may adversely affect the safety and effective implantation and use of the ThecaFlex DRx system or confound the clinical investigation, including:\n\n   1. Severe structural impediment that may preclude safe implantation of the catheter and port\n   2. Major medical events (as defined per investigator's discretion) within 60 days prior to screening; or\n   3. Relevant surgeries (e.g., head and neck, back, etc.) within 60 days prior to screening or planned during the duration of the clinical investigation\n   4. Space-occupying lesion with mass effect\n   5. Posterior fossa mass\n   6. Arnold-Chiari malformation\n   7. Coagulation abnormalities and\u002For thrombocytopenia\n   8. Insufficient or inadequate skin or underlying fat to adequately protect the port or enough surface area to allow the skin to close safely\n5. Subject is contraindicated for administration of nusinersen per its approved country-specific labeling\n6. History of intrathecal granuloma formation\n7. History of bacterial meningitis or aseptic meningitis within 6 months of screening\n8. History of tumors or other spinal abnormalities documented by magnetic resonance imaging (MRI) or computed tomography (CT) that would interfere with the catheter implantation procedures or CSF circulation\n9. History of hydrocephalus\n10. Diagnosed degenerative muscular disease other than SMA\n11. History of depression, cognitive impairment, or another psycho-behavioral problem that in the opinion of the Investigator may preclude safe participation in the clinical investigation and\n12. Serious medical condition that, in the opinion of the investigator, may lead to reduced life expectancy beyond 12 months.\n13. Subject is involved in another Investigation Device Exemption Study.\n14. Subject has clinically meaningful immunosuppression, in the opinion of the investigator, either from therapy or a comorbid condition, which, in the opinion of the investigator, makes them a poor surgical candidate due to increased risk of infection.",[65,66,67],"CHILD","ADULT","OLDER_ADULT",[69,87,99,110,121,133,146,158,170,182,194,206,217,229,242,253,264,275,285,296,307],{"facility":70,"status":8,"city":71,"state":72,"zip":73,"country":74,"contacts":75,"geoPoint":84},"Barrow Neurological Institute","Phoenix","Arizona","85013","United States",[76,81],{"name":77,"role":78,"phone":79,"email":80},"Kathleen Nagaran Hildebrand","CONTACT","(602) 406 7031","kathleen.nagaranhildebrand900@commonspirit.org",{"name":82,"role":83},"Shafeeq Ladha, MD","PRINCIPAL_INVESTIGATOR",{"lat":85,"lon":86},33.44838,-112.07404,{"facility":88,"status":8,"city":89,"state":90,"zip":91,"country":74,"contacts":92,"geoPoint":96},"Children's Hospital Orange County","Orange","California","92868",[93],{"name":94,"role":78,"email":95},"Melina Thaxton","MelinaThaxton@choc.org",{"lat":97,"lon":98},33.78779,-117.85311,{"facility":100,"status":8,"city":101,"state":90,"zip":102,"country":74,"contacts":103,"geoPoint":107},"Stanford Medical Center","Palo Alto","94301",[104],{"name":105,"role":78,"email":106},"Habib Mofakham","neuromuscularresearch@stanford.edu",{"lat":108,"lon":109},37.44188,-122.14302,{"facility":111,"status":8,"city":112,"state":90,"zip":113,"country":74,"contacts":114,"geoPoint":118},"Rady Children's Hospital","San Diego","92037",[115],{"name":116,"role":78,"email":117},"Elizabeth Moreno","e4moreno@health.ucsd.edu",{"lat":119,"lon":120},32.71571,-117.16472,{"facility":122,"status":8,"city":123,"state":124,"zip":125,"country":74,"contacts":126,"geoPoint":130},"Nemours Children's Hospital, Florida","Orlando","Florida","32827",[127],{"name":128,"role":78,"email":129},"Debbie Berry","debbie.berry@nemours.org",{"lat":131,"lon":132},28.53834,-81.37924,{"facility":134,"status":8,"city":135,"state":136,"zip":137,"country":74,"contacts":138,"geoPoint":143},"Lurie Childrens Hospital","Chicago","Illinois","60611",[139,141],{"name":140,"role":78},"Raven HIll",{"role":78,"email":142},"rhill@luriechildrens.org",{"lat":144,"lon":145},41.85003,-87.65005,{"facility":147,"status":8,"city":148,"state":149,"zip":150,"country":74,"contacts":151,"geoPoint":155},"Boston Children's Hospital","Boston","Massachusetts","02115",[152],{"name":153,"role":78,"email":154},"Adam Porter","adam.porter@childrens.harvard.edu",{"lat":156,"lon":157},42.35843,-71.05977,{"facility":159,"status":8,"city":160,"state":161,"zip":162,"country":74,"contacts":163,"geoPoint":167},"Helen DeVos Children's Hospital","Grand Rapids","Michigan","49503",[164],{"name":165,"role":78,"email":166},"Natasha Schafer","natasha.schafer@corewellhealth.org",{"lat":168,"lon":169},42.96336,-85.66809,{"facility":171,"status":8,"city":172,"state":172,"zip":173,"country":74,"contacts":174,"geoPoint":179},"Columbia University Irving Medical Center\u002FNewYork Presbyterian Hospital","New York","10032",[175],{"name":176,"role":78,"phone":177,"email":178},"Kimbery Berry","212-342-3038","KB2996@cumc.columbia.ediu",{"lat":180,"lon":181},40.71427,-74.00597,{"facility":183,"status":8,"city":184,"state":185,"zip":186,"country":74,"contacts":187,"geoPoint":191},"UH Rainbow Babies and Children's Hospital","Cleveland","Ohio","44106",[188],{"name":189,"role":78,"email":190},"Abigail Schlosser","Abigail.Schlosser@UHhospitals.org",{"lat":192,"lon":193},41.4995,-81.69541,{"facility":195,"status":8,"city":196,"state":197,"zip":198,"country":74,"contacts":199,"geoPoint":203},"Penn State Milton S. Hershey Medical Center","Hershey","Pennsylvania","17033",[200],{"name":201,"role":78,"email":202},"Chelsea Emrick","awinters2@pennstatehealth.psu.edu",{"lat":204,"lon":205},40.28592,-76.65025,{"facility":207,"status":8,"city":208,"state":197,"zip":209,"country":74,"contacts":210,"geoPoint":214},"Children's Hospital Philadelphia","Philadelphia","19104",[211],{"name":212,"role":78,"email":213},"Samantha Gogel","Gogels@chop.edu",{"lat":215,"lon":216},39.95238,-75.16362,{"facility":218,"status":8,"city":219,"state":220,"zip":221,"country":74,"contacts":222,"geoPoint":226},"Texas Children's Hospital","Houston","Texas","77030",[223],{"name":224,"role":78,"email":225},"Sarah Wisor","sxwisor@texaschildrens.org",{"lat":227,"lon":228},29.76328,-95.36327,{"facility":230,"status":8,"city":231,"state":232,"zip":233,"country":74,"contacts":234,"geoPoint":239},"University of Virginia","Charlottesville","Virginia","22903",[235,237],{"name":236,"role":78},"Chelsea Masterson",{"role":78,"email":238},"CDM5FA@uvahealth.org",{"lat":240,"lon":241},38.02931,-78.47668,{"facility":243,"status":8,"city":244,"state":232,"zip":245,"country":74,"contacts":246,"geoPoint":250},"Childrens Hospital of the King's Daughters","Norfolk","23510",[247],{"name":248,"role":78,"email":249},"Erika Paradiso","Erika.Paradiso@chkd.org",{"lat":251,"lon":252},36.84681,-76.28522,{"facility":254,"status":8,"city":255,"country":256,"contacts":257,"geoPoint":261},"Universitätsklinikum Essen","Essen","Germany",[258],{"name":259,"role":78,"email":260},"Jaqueline Lipka","jaqueline.lipka@uk-essen.de",{"lat":262,"lon":263},51.45657,7.01228,{"facility":265,"status":8,"city":266,"country":267,"contacts":268,"geoPoint":272},"Specialised Hospital Ludwika Rydygiera","Krakow","Poland",[269],{"name":270,"role":78,"email":271},"Ryszard Nowak","badaniakliniczne@rydygierkrakow.pl",{"lat":273,"lon":274},50.06143,19.93658,{"facility":276,"status":8,"city":277,"country":267,"contacts":278,"geoPoint":282},"Research Institute of Polish Mother's Memorial Hospital","Lodz",[279],{"name":280,"role":78,"email":281},"Lukasz Przyslo","lukasz.przyslo@iczmp.edu.pl",{"lat":283,"lon":284},51.77058,19.47395,{"facility":286,"status":8,"city":287,"country":288,"contacts":289,"geoPoint":293},"Hospital Universitario La Paz","Madrid","Spain",[290],{"name":291,"role":78,"email":292},"María del Mar García Romero","mmar.garcia.romero@salud.madrid.org",{"lat":294,"lon":295},40.4165,-3.70256,{"facility":297,"status":8,"city":298,"zip":299,"country":288,"contacts":300,"geoPoint":304},"Hospital Universitario y Politecnico la Fe de Valencia","Valencia","46026",[301],{"name":302,"role":78,"email":303},"Nancy Ñungo Garzon","carolina_nungo@iislafe.es",{"lat":305,"lon":306},39.47391,-0.37966,{"facility":308,"status":8,"city":309,"country":310,"contacts":311,"geoPoint":315},"Sheffield Teaching Hospitals NHS Foundation Trust","Sheffield","United Kingdom",[312],{"name":313,"role":78,"email":314},"Lee Tuddenham","lee.tuddenham@nhs.net",{"lat":316,"lon":317},53.38297,-1.4659,[319],{"name":320,"role":78,"phone":321,"email":322},"Janelle Arrambide","857-500-7526","janelle.arrambide@biogen.com",[],[325],{"pmid":326,"type":327,"citation":328},"41830093","DERIVED","Mesina-Estarron I, Abruzzo M, Gomes FC, Thaxton M, Yengo-Kahn A, Stone S, Northam W, Snyder B, Cahill P, Bauer D, Muhonen M. Clinical evaluation of ThecaFlex DRx, a novel implantable catheter-port for intrathecal nusinersen delivery in spinal muscular atrophy: Initial results from the PIERRE-IDE study. J Neuromuscul Dis. 2026 Mar 13:22143602261432303. doi: 10.1177\u002F22143602261432303. Online ahead of print.",[],{"nct_id":4,"conditions":331,"biomarkers":333},[21,332,19],"Spinal Deformity",[],{"nct_id":4,"found":15,"summary":335,"prompt_version":345},{"design":336,"status":337,"heading":338,"summary":339,"follow_up":340,"word_count":341,"commitments":342,"compensation":343,"drugs_mentioned":344},"This is a single-arm study, meaning all 90 participants will receive the ThecaFlex DRx System. It is a pivotal clinical investigation to assess the safety and performance of the device.","completed","Study of ThecaFlex DRx System for Spinal Muscular Atrophy","This study is testing a device called the ThecaFlex DRx System for people with Spinal Muscular Atrophy (SMA) who need repeated doses of nusinersen (Spinraza). The ThecaFlex DRx System includes an implantable port and catheter designed to deliver nusinersen directly into the fluid around the spinal cord. You might be eligible if you are 3 years or older, have SMA, and have difficulty with standard lumbar punctures (spinal taps) due to other health issues. The main goals are to see how many people have the device successfully implanted and how many successfully receive nusinersen through it over 12 months. This study aims to show the device can be used effectively to deliver nusinersen.","Participants will be followed for 12 months after receiving the implant to assess the primary endpoints.",113,"You would undergo implantation of the ThecaFlex DRx System and be followed for 12 months. Nusinersen doses would be administered via the implant, with frequency depending on whether you have received nusinersen before.","Not stated in the trial record.",[33],"v2"]