Olisens-Aniridia

Overview

Sponsor-declared trial summary

Key facts

Sponsor

University Of Cologne

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Eye Diseases [C11]

Decision date (initial)

2026-01-23

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

EUROPEAN HEALTH AND DIGITAL EXECUTIVE AGENCY (HADEA) - Project 101080611 — RESTORE VISION

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Efficacy

Primary objective is to prove the efficacy of Olisens, an antisense oligonucleotide eye drop formulation against IRS-1 in halting/reducing CoNV in eyes with CoNV in AAK as measured in reduction of percentage of vascularized corneal area.

Secondary objectives 12

1. Visual acuity
2. Glare visual acuity
3. Vision-related quality of life (NEI-VFQ25 and Aniridia-Net Questionnaire)
4. Intraocular pressure
5. Ocular Surface Staining according to Oxford Grading Scale
6. Corneal Sensation
7. Central Corneal Thickness
8. Vessel morphometry
9. Ocular Pain related QoL Questionnaire (OPAS)
10. Aniridia-Associated Keratopathy Grading
11. Safety
12. Active infectious keratitis or corneal ulceration

Conditions and MedDRA coding

Corneal Neovascularization in Aniridia-associated Keratopathy

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 8

1. Pathologically vascularized cornea due to aniridia-associated keratopathy
2. Male and female patients ≥ 12 years old
3. Written informed consent from the patients and parents and/or legal guardian has been obtained prior to any trial-related procedures
4. 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
5. At least 2 mm diameter centrally clear cornea without vessels
6. Absence of other clinical contraindications to any part or product of the treatment plan
7. A cooperative attitude to follow up the trial procedures
8. In case of bilateral disease only one eye will be included

Exclusion criteria 13

1. Active or suspected intraocular inflammation
2. Active corneal ulceration
3. 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.
4. Rheumatic diseases that require systemic treatment
5. 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
6. Participation in another clinical trial where an investigational drug was received less than 4 weeks prior to screening visit
7. Known abuse of alcohol, drugs, or medicinal products
8. 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
9. Employees of the sponsor, or employees or relatives of the investigator
10. Pregnant women and nursing mothers as trial medication has not been tested in these subjects before
11. Persons held in an institution by legal or official order
12. Use of autologous or allogenous serum eye drops during the trial period (have to be stopped 14 days ahead of enrolment into the trial)
13. 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

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Relative change of corneal area covered by CoNV assessed by digital standardized slit lamp images and an independent reading center.

Secondary endpoints 12

1. Visual acuity (BCVA) measured by ETDRS charts (transformed to logMAR)
2. Glare visual acuity measured by ETDRS charts (transformed to logMAR)
3. Vision related quality of life assessed by overall score of NEI-VFQ25 and overall score of Aniridia-Net questionnaire
4. Intraocular pressure measured by I Care or Goldmann applanation tonometry, reported as mmHg
5. Ocular Surface Staining measured by Oxford Grading Scale (Grades 0-5)
6. Corneal Sensation measured by length of the nylon monofilament of Cochet-Bonnet esthesiometer when the applied pressure is noticed by the participant
7. Central Corneal Thickness measured by Scheimpflug Corneal Tomography (Pentacam) or Anterior Segment Optical Coherence Tomography (Casia-2)
8. Vessel Morphometry measured by vessel thickness, vessel length, vessel regression, vessel recurrence
9. Quality of Life (Ocular Pain) assessed by overall score of Ocular Pain Asessment Survey (OPAS)
10. Aniridia-Associated Keratopathy Grading measured by Lagali Aniridia Associated Kerathopathy Grading
11. Retinal adverse events (Optical Coherence Tomography)
12. Active infectious keratitis or corneal ulceration assessed by slit lamp examination

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

University Of Cologne

Sponsor organisation

University Of Cologne

Address

Albertus-Magnus-Platz 1

City

Cologne

Postcode

50923

Country

Germany

Scientific contact point

Organisation

University Of Cologne

Contact name

Claus Cursiefen

Public contact point

Organisation

University Of Cologne

Contact name

Claus Cursiefen

Third parties 7

Locations

1 EU/EEA country · 1 investigational sites

By country

Investigational sites

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 11 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

2 submissions — initial application plus substantial / non-substantial modifications