Damage to the cornea due to a complete or partial absence of the coloured part of the eye (aniridia-related keratopathy) Eye Diseases Aniridia-related keratopathy
Conditions
Interventions
This is a seamless phase I/II single-dose, single-arm safety and efficacy evaluation of RAFT-OS in patients with ARK.
A two-stage approach to in evaluating safety and efficacy will be taken.
Stage 1
sequential recruitment as, cohorts of two, full parallel recruitment, or stopping the trial. The recommendation will be based on pre-specified stopping and adaptation rules outlined in this document a
Sponsors
University College London
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Confirmed diagnosis of congenital aniridia 2. Confirmed diagnosis of advanced aniridia related keratopathy with corneal surface conjunctivalisation, vascularisation and increasing opacity with worsening vision loss, glare & ocular surface pain 3. Patients aged 18 years and over 4. Participants must use acceptable contraception from enrolment up to 6 weeks for female participants and 90 days for male participants, after stopping immunosuppression therapy 5. Negative viral screen for, HIV, syphilis, hepatitis B & C and Human T-cell Leukaemia Virus (HTLV) 6. Negative urine pregnancy test
Exclusion criteria
Exclusion criteria: 1. Poor tear production, as assessed by a Schirmer’s test type 1 2. Lid malposition 3. Current corneal infection 4. Uncontrolled glaucoma (defined as uncontrolled eye pressure, changes to medication, recent surgery in the last 3 months or being considered for surgical treatment) 5. Must not be NPL (no light perception) in one or both eyes 6. Patients who refuse to consent to the site informing their GP of their participation 7. Patients who lack capacity to give full informed consent to participate 8. Pregnant or lactating women 9. Patients with known contraindications to any of the following non-investigational medicinal products; mycophenolate, prednisolone, omeprazole, doxycycline, dexamethasone or moxifloxacin or excipients according to the relevant SmPCs 10. Patients who are participating in any concurrent trial involving an investigational medical product, device or surgical intervention within the last 12 months 11. Known albumin or egg allergy 12. Known penicillin allergy 13. Known hydrocortisone allergy 14. Inability to lie flat for surgical procedure
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 1. Occurrence of serious adverse events within the 12 months after surgery measured using patient records 2. Health of the surface of the cornea using a validated ocular surface scoring system at baseline and 3 months after surgery | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. Vision using the distance EDTRS visual acuity at 3 & 12 months after surgery 2. Quality of life using the NEI-VFQ-25 patient questionnaire and RAND 36-Item Health Survey at 3 & 12 months after surgery | — |
Countries
England, United Kingdom
Contacts
Public ContactRAFT Trial Manager
Outcome results
None listed