[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"trial:NCT06789445":3,"trial-entities:NCT06789445":169,"trial-summary:NCT06789445":174},{"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":28,"study_type":38,"primary_purpose":39,"phases":40,"enrollment_info":43,"interventions":46,"primary_outcomes":59,"secondary_outcomes":63,"sex":67,"minimum_age":68,"maximum_age":69,"healthy_volunteers":70,"eligibility_criteria":71,"std_ages":89,"locations":92,"central_contacts":160,"overall_officials":163,"references":164,"see_also_links":165},"NCT06789445","OpCT-001-101","A Study to Investigate the Safety of OpCT-001 in Adults Who Have Primary Photoreceptor Disease (CLARICO)","A Phase 1\u002F2a Study of Subretinal Administration of OpCT-001 Photoreceptor Precursor Cells Derived From iPSCs in Patients With Primary Photoreceptor Disease","RECRUITING","2030-10","2026-08","2026-08-19","2025-03-10","BlueRock Therapeutics","INDUSTRY",true,"Study OpCT-001-101 is a Phase 1\u002F2a first-in-human, multisite, 2-part interventional study to evaluate the safety, tolerability, and the effect on clinical outcomes of OpCT-001 in approximately 54 adults with primary photoreceptor (PR) disease. Phase 1 focuses on safety and features a dose-escalation design. Phase 2 is designed to gather additional safety data and assess the effect of OpCT-001 on measures of visual function, functional vision, and anatomic measures of engraftment in different clinical subgroups.","Phase 1 of the study includes up to 4 planned dose levels to be administered across up to 4 cohorts. Participants are assigned to receive OpCT-001 in an open-label manner.\n\nDose escalation in Phase 1 is being conducted using a standard 3+3 scheme in which a total of up to 24 legally blind participants (\\~3 to 6 per cohort) will receive OpCT-001. Phase 2 is planned to enroll a maximum of 15 participants per cohort in 2 cohorts to evaluate 2 dose levels of OpCT-001 that will be selected based on Phase 1 safety and tolerability data. Phase 2 participants will be randomized 1:1 to either dose-level cohort. Phase 2 participants and investigators and study site personnel outside of the surgical team will be masked to the Phase 2 OpCT-001 dose-level assignments.",[19,20,21,22,23,24,25,26,27],"Primary Photoreceptor Disease","Retinitis Pigmentosa (RP)","Usher Syndrome","Inherited Retinal Disease (IRD)","Rod-Cone Dystrophy","Rod-Cone Disease","Retinal Degeneration","Cone-Rod Disease (C-RD)","Cone-Rod Dystrophy",[29,30,31,32,21,33,34,35,36,37],"CLARICO","Photoreceptor cells","Cell Therapy","Cellular Therapy","Retinitis Pigmentosa","Inherited retinal disease (IRD)","Primary Photoreceptor disease (PPD)","Rod-Con Disease (R-CD)","Cone-Rose disease (C-RD)","INTERVENTIONAL","TREATMENT",[41,42],"PHASE1","PHASE2",{"count":44,"type":45},54,"ESTIMATED",[47,53],{"type":48,"name":49,"description":50,"armGroupLabels":51},"BIOLOGICAL","OpCT-001","Cell therapy product composed of photoreceptor precursor cells derived from human induced pluripotent stem cells (iPSCs).",[52],"Cohort 1",{"type":48,"name":49,"description":54,"armGroupLabels":55},"Cell therapy product composed of photoreceptor precursor cells derived from human iPSCs.",[56,57,58],"Cohort 2","Cohort 3 (optional)","Cohort 4",[60],{"measure":61,"timeFrame":62},"Incidence and severity of ocular and non-ocular treatment-emergent adverse events (TEAEs) through the Week 52 visit","From OpCT-001 administration through the Week 52 visit",[64],{"measure":65,"timeFrame":66},"Change from baseline over time through the Week 52 visit in the treated portion of the study eye (SE) retina in outer retinal layer thickness as measured by spectral domain optical coherence tomography (SD-OCT)","From baseline through the Week 52 visit","ALL","18 Years",null,false,{"inclusion":72,"exclusion":76,"raw_text":88},[73,74,75],"Confirmed genetic diagnosis of primary photoreceptor (PR) disease","Best corrected visual acuity (BCVA) in the study eye at Screening for Phase 1: Logmarithm of the minimum angle of resolution (LogMAR) 3.9 to LogMAR 1.3. BCVA at Screening for Phase 2: ETDRS letter score between 20 to 60, inclusive.","Retinal structure examination in the study eye demonstrating regions suitable for cell administration.",[77,78,79,80,81,82,83,84,85,86,87],"Clinically relevant, active ocular inflammation or infection","Glaucoma or other significant optic neuropathy","Diabetic macular edema or diabetic retinopathy","Clinically significant cystoid macular edema","In phakic participants: Spherical equivalent refractive error of greater than 8.00 diopters myopia","Ocular surgery ≤3 months before Screening","Monocular vision (ie, no light perception in the fellow eye)","Currently active malignancy, or history of malignancy within 5 years before OpCT-001 administration. Exception: Basal cell carcinoma that has been definitively treated.","Any current and active infection (bacterial\u002Fviral\u002Ffungal) that could put the participant at risk from immunosuppression","History of any cell therapy, gene therapy, or retinal implant at any time","Previously received a bone marrow or solid organ transplant","Key Inclusion Criteria:\n\n* Confirmed genetic diagnosis of primary photoreceptor (PR) disease\n* Best corrected visual acuity (BCVA) in the study eye at Screening for Phase 1: Logmarithm of the minimum angle of resolution (LogMAR) 3.9 to LogMAR 1.3. BCVA at Screening for Phase 2: ETDRS letter score between 20 to 60, inclusive.\n* Retinal structure examination in the study eye demonstrating regions suitable for cell administration.\n\nKey Exclusion Criteria:\n\n* Clinically relevant, active ocular inflammation or infection\n* Glaucoma or other significant optic neuropathy\n* Diabetic macular edema or diabetic retinopathy\n* Clinically significant cystoid macular edema\n* In phakic participants: Spherical equivalent refractive error of greater than 8.00 diopters myopia\n* Ocular surgery ≤3 months before Screening\n* Monocular vision (ie, no light perception in the fellow eye)\n* Currently active malignancy, or history of malignancy within 5 years before OpCT-001 administration. Exception: Basal cell carcinoma that has been definitively treated.\n* Any current and active infection (bacterial\u002Fviral\u002Ffungal) that could put the participant at risk from immunosuppression\n* History of any cell therapy, gene therapy, or retinal implant at any time\n* Previously received a bone marrow or solid organ transplant",[90,91],"ADULT","OLDER_ADULT",[93,111,121,135,150],{"facility":94,"status":8,"city":95,"state":96,"zip":97,"country":98,"contacts":99,"geoPoint":108},"University of Southern California \u002FChildren's Hospital of Los Angeles","Los Angeles","California","90007","United States",[100,105],{"name":101,"role":102,"phone":103,"email":104},"Patient Engagement","CONTACT","877-380-3931","clinicaltrials@bluerocktx.com",{"name":106,"role":102,"email":107},"Hilary Schwartz-Gale","hschwartz@chla.usc.edu",{"lat":109,"lon":110},34.05223,-118.24368,{"facility":112,"status":8,"city":113,"state":114,"zip":115,"country":98,"contacts":116,"geoPoint":118},"University of Miami, Bascom Palmer Eye Institute","Miami","Florida","33136",[117],{"name":101,"role":102,"phone":103,"email":104},{"lat":119,"lon":120},25.77427,-80.19366,{"facility":122,"status":8,"city":123,"state":124,"zip":125,"country":98,"contacts":126,"geoPoint":132},"Mid Atlantic Retina\u002F Wills Eye","Philadelphia","Pennsylvania","19107",[127,128],{"name":101,"role":102,"phone":103,"email":104},{"name":129,"role":102,"phone":130,"email":131},"Study Coordinator","215-928-3092","alemus@chla.usc.edu",{"lat":133,"lon":134},39.95238,-75.16362,{"facility":136,"status":8,"city":137,"state":138,"zip":139,"country":98,"contacts":140,"geoPoint":147},"Retina Foundation of the Southwest","Dallas","Texas","75231",[141,142],{"name":101,"role":102,"phone":103,"email":104},{"name":143,"role":102,"phone":144,"phoneExt":145,"email":146},"Jennifer Herrera","214-363-3911","135","jherrera@retinafoundation.org",{"lat":148,"lon":149},32.78306,-96.80667,{"facility":151,"status":8,"city":152,"state":153,"zip":154,"country":98,"contacts":155,"geoPoint":157},"University of Wisconsin","Madison","Wisconsin","53705",[156],{"name":101,"role":102,"phone":103,"email":104},{"lat":158,"lon":159},43.07305,-89.40123,[161],{"name":101,"role":102,"phone":162,"email":104},"1-877-380-3931",[],[],[166],{"label":167,"url":168},"Related Info","https:\u002F\u002Fclaricostudy.com\u002F",{"nct_id":4,"conditions":170,"biomarkers":173},[171,172,19,33,23,21],"Cone-Rod Dystrophies","Hereditary nephropathy",[],{"nct_id":4,"found":15,"summary":175,"prompt_version":185},{"design":176,"status":177,"heading":178,"summary":179,"follow_up":180,"word_count":181,"commitments":182,"compensation":183,"drugs_mentioned":184},"This is a Phase 1\u002F2a study with two parts. Phase 1 is open-label, meaning everyone knows they are receiving OpCT-001, and involves increasing doses in small groups. Phase 2 will involve up to 30 participants, where some will be masked to the dose level they receive.","completed","CLARICO Study: OpCT-001 for Primary Photoreceptor Disease","This study, called CLARICO, is testing a new cell therapy called OpCT-001 for adults with primary photoreceptor disease, including conditions like Retinitis Pigmentosa (RP) and Usher Syndrome. OpCT-001 is made of special photoreceptor precursor cells grown from human stem cells. The main goal of the study is to see how safe OpCT-001 is and if you experience any side effects over 52 weeks. Later parts of the study will also look at how it affects your vision. You may be able to join if you are an adult with a confirmed genetic diagnosis of primary photoreceptor disease and meet specific vision requirements. The study is currently recruiting about 54 participants.","You will be followed for safety and side effects for 52 weeks after receiving OpCT-001.",110,"You would receive OpCT-001 and be monitored for side effects through weekly visits for 52 weeks after treatment.","Not stated in the trial record.",[49],"v2"]