A first-in-human trial assessing the safety and efficacy of the RAFT transplant in the treatment of aniridia-related keratopathy
- Conditions
- Damage to the cornea due to a complete or partial absence of the coloured part of the eye (aniridia-related keratopathy)Eye DiseasesAniridia-related keratopathy
- Registration Number
- ISRCTN11428712
- Lead Sponsor
- niversity College London
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Stopped
- Sex
- All
- Target Recruitment
- 9
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
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
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method 1. Occurrence of serious adverse events within the 12 months after surgery measured using patient records<br>2. Health of the surface of the cornea using a validated ocular surface scoring system at baseline and 3 months after surgery
- Secondary Outcome Measures
Name Time Method 1. Vision using the distance EDTRS visual acuity at 3 & 12 months after surgery<br>2. Quality of life using the NEI-VFQ-25 patient questionnaire and RAND 36-Item Health Survey at 3 & 12 months after surgery