AGANIRSEN for Corneal neovascularization in aniridia-associated keratopathy

Inclusion criteria

• {"criterion_text":"- Pathologically vascularized cornea due to aniridia-associated keratopathy\n- Male and female patients ≥ 12 years old\n- Written informed consent from the patients and parents and/or legal guardian has been obtained prior to any trial-related procedures\n- Patient has confirmed diagnosis of aniridia and progressive corneal neovascularization (confirmed either through 2 slit-lamp photographs with at least 4 weeks apart or through clinical situation) and/or diagnosis of Stage 2 aniridia-associated keratopathy\n- At least 2 mm diameter centrally clear cornea without vessels\n- Absence of other clinical contraindications to any part or product of the treatment plan\n- A cooperative attitude to follow up the trial procedures\n- In case of bilateral disease only one eye will be included"}

Exclusion criteria

• {"criterion_text":"- Active or suspected intraocular inflammation\n- Pregnant women and nursing mothers as trial medication has not been tested in these subjects before\n- Persons held in an institution by legal or official order\n- Use of autologous or allogenous serum eye drops during the trial period (have to be stopped 14 days ahead of enrolment into the trial)\n- Failure to use highly-effective contraceptive methods (for women of childbearing potential and males who have a partner of childbearing potential) during the study period.. The following contraceptive methods with a Pearl Index lower than 1% are regarded as highly-effective: Oral hormonal contraception (‘pill’), Dermal hormonal contraception, Vaginal hormonal contraception (NuvaRing®), Contraceptive plaster, Long-acting injectable contraceptives, Implants that release progesterone (Implanon®), Tubal ligation (female sterilisation), Intrauterine devices that release hormones (hormone spiral), Avoiding sexual activities that could lead to pregnancy\n- Active corneal ulceration\n- Clinically significant or unstable concurrent disease or other medical condition. The possibility of a previously undiagnosed nephroblastoma (Wilms tumour) in schoolchildren and adolescents must already have been ruled out.\n- Rheumatic diseases that require systemic treatment\n- Patients unlikely to comply with the trial protocol or unable to understand the nature and scope of the trial or the possible benefits or unwanted effects of the trial procedures and treatments\n- Participation in another clinical trial where an investigational drug was received less than 4 weeks prior to screening visit\n- Known abuse of alcohol, drugs, or medicinal products\n- Evidence of any other medical conditions (such as psychiatric illness, physical examination, or laboratory findings) that may interfere with the planned treatment, affect the subject’s compliance, or place the subject at high risk of complications related to the treatment\n- Employees of the sponsor, or employees or relatives of the investigator"}

Primary endpoints

• {"endpoint_text":"- Relative change of corneal area covered by CoNV assessed by digital standardized slit lamp images and an independent reading center.","definition_or_measurement_approach":"Assessed by digital standardized slit lamp images and evaluation by an independent reading center (relative change of corneal area covered by corneal neovascularization)."}

Secondary endpoints

• {"endpoint_text":"- Visual acuity (BCVA) measured by ETDRS charts (transformed to logMAR)\n- Glare visual acuity measured by ETDRS charts (transformed to logMAR)\n- Vision related quality of life assessed by overall score of NEI-VFQ25 and overall score of Aniridia-Net questionnaire\n- Intraocular pressure measured by I Care or Goldmann applanation tonometry, reported as mmHg\n- Ocular Surface Staining measured by Oxford Grading Scale (Grades 0-5)\n- Corneal Sensation measured by length of the nylon monofilament of Cochet-Bonnet esthesiometer when the applied pressure is noticed by the participant\n- Central Corneal Thickness measured by Scheimpflug Corneal Tomography (Pentacam) or Anterior Segment Optical Coherence Tomography (Casia-2)\n- Vessel Morphometry measured by vessel thickness, vessel length, vessel regression, vessel recurrence\n- Quality of Life (Ocular Pain) assessed by overall score of Ocular Pain Asessment Survey (OPAS)\n- Aniridia-Associated Keratopathy Grading measured by Lagali Aniridia Associated Kerathopathy Grading\n- Retinal adverse events (Optical Coherence Tomography)\n- Active infectious keratitis or corneal ulceration assessed by slit lamp examination","definition_or_measurement_approach":"As stated for each measure: BCVA by ETDRS charts transformed to logMAR; Glare VA by ETDRS; QoL by NEI-VFQ25 and Aniridia-Net questionnaire overall scores; IOP by I Care or Goldmann applanation tonometry (mmHg); Ocular surface staining by Oxford Grading Scale (0-5); Corneal sensation by Cochet-Bonnet esthesiometer filament length; CCT by Pentacam or Anterior Segment OCT (Casia-2); Vessel morphometry by vessel thickness/length/regression/recurrence assessments; Ocular pain QoL by OPAS overall score; AAK grading by Lagali grading; Retinal AEs by OCT; Infectious keratitis/corneal ulceration by slit lamp examination."}

Germany

Earliest CTIS Part Ii Submission Date

14-01-2026

Latest Decision Or Authorization Date

05-05-2026

Processing Time Days

111

Number Of Sites

1

Number Of Participants

20

Primary sponsor

Full Name

University Of Cologne

Organisation Type

Educational Institution

Country Of Registered Address

Germany

Third parties

• {"country":"United States","full_name":"Nitto Denko Avecia Inc.","duties_or_roles":"Manufacture and release of API","organisation_type":"Pharmaceutical company"}
• {"country":"France","full_name":"Creapharm Clinical Supplies","duties_or_roles":"Clinical Packager and Batch Certification","organisation_type":"Pharmaceutical company"}
• {"country":"France","full_name":"Laboratoires Kol","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
• {"country":"Germany","full_name":"Universitaetsklinikum Koeln AöR","duties_or_roles":"","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"Germany","full_name":"Universitaetsklinikum Koeln AöR","duties_or_roles":"Analysis of CoNV area and vessel morphometry through digital standardized slit-lamp images","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"Germany","full_name":"Universitaetsklinikum Koeln AöR","duties_or_roles":"","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"France","full_name":"Laboratoire Unither","duties_or_roles":"Manufacture of finished product (preparation of solution and aseptic filling), primary and secondary packaging, control, stability studies and release","organisation_type":"Pharmaceutical company"}