RAFT

Open for recruitment:  18th August 2021
RECRUITMENT TARGET = 21 participants

EudraCT: 2019-002371-34                                     
Clinicaltrials.gov: NCT05044598
REC: 21/SC/0020
IRAS: 269184
ISRCTN11428712

Trial Information

Brief Summary
The RAFT trial is a first in human trial of a novel cellular therapy called RAFT-OS (Real Architecture for 3D Tissues Ocular Surface) developed and manufactured by Cells for Sight Stem Cell Therapy Research Unit at UCL institute of Ophthalmology. 

The aim of this seamless phase I/II single-dose, single-arm trial is to investigate if RAFT-OS is a safe and effective alternative treatment for patients with aniridia related keratopathy (ARK) in 21 patients. 

ARK is a complication of aniridia, which is a genetic eye condition present from birth. 

RAFT-OS is an artificial tissue, populated with limbal epithelial cells and stromal cells. The source of the adult limbal and stromal cells is from donated human corneas from the NHS blood and Transplant, Tissue and Eye services in Liverpool. 

Following a Screening visit, participants will commence 10-weeks of immune suppression therapy to prepare for the transplantation of RAFT-OS.

The RAFT-OS will be transplanted into the participants worst affected eye. Following surgery, each participant will be assessed at days 1, 7, 14, 21, and 1-month for major or intermediate safety events. Participants will continue to be followed up to 12 months after transplantation and will be required to stay on the immune suppression therapy for the duration of the trial.

The trial is conducted at Moorfields Eye Hospital NHS Foundation Trust (MEH), London in the United Kingdom (UK). MEH is a leading provider of eye health services in the UK and is a world-class centre of excellence for ophthalmic research and education. 

All trial medical assessments and procedures will be performed in an appropriate clinical setting by suitability qualified staff.

 

Who is organising and funding the research? 
This research is being funded by Medical Research Council (Grant Code: MR/S018883/1).

The trial is being sponsored and co-ordinated by Comprehensive Clinical Trials Unit at University College London. 

 

Eligibility criteria

Please find the eligibility criteria for RAFT below:

Participant Inclusion Criteria

1. Confirmed diagnosis of congenital aniridia. 
2. Confirmed diagnosis of advanced aniridia related keratopathy with corneal surface conjunctivalisation, vascularisation and opacification with visual 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.

Participant Exclusion Criteria

1. Poor tear production, as assessed by a Schirmer’s test type 1.
2. Lid malposition (entropion, ectropion, fornix shortening, symblepharon).
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 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.

 

Outcomes

Primary outcomes

1. The primary safety endpoint is occurrence of either any major safety event (non-ocular SUSAR; corneal melting; death) or any intermediate safety event (persisting epithelial defect before progression to corneal ulcer; persisting ocular inflammation; loss of corneal clarity; ocular SAEs related to the RAFT-OS and not surgery alone, conjunctival or lid swelling considered related to the ATIMP (RAFT-OS) and not surgery alone).
2. The primary efficacy endpoint is corneal surface normalisation (i.e., persistence of normal corneal epithelium and absence of vascularisation, epithelial defect and conjunctivalisation) at 3 months post-RAFT-OS transplantation using a validated ocular surface scoring system. 

 

Secondary outcomes

1. Visual improvement assessed by distance EDTRS visual acuity (refractive best corrected) at 3- & 12-months post RAFT-OS transplantation.
2. Improvement in quality of life at 3- & 12-months post RAFT-OS transplantation as assessed by NEI-VFQ-25 patient questionnaire and RAND 36-Item Health Survey.

 

Participating Site / Research contacts

Status: Recruiting
Please contact the local trial team for more information.

Moorfields Eye Hospital, London

• Principal Investigator: Mr Sajjad Ahmad
  Email: sajjad.ahmad3@nhs.net 
  Tel: 0207 566 2320
• Trial Clinical Fellow: Dr Abigail Kaye
  Email: Abigail.kaye2@nhs.net
  Tel: 0207 566 2320

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Meet the RAFT Team
Mr Sajjad Ahmad
Chief Investigator

Prof Julie Daniels
Scientific Coordinator

Dr Abigail Kaye
Trial Clinical Fellow

Liz Deane
Clinical Project Manager

Amalia Ndoutoumou 
Clinical Trial Manager

Katherine Chan
Data Manager

 

Trial Additional Information

Additional information about our trial can be found on the service of the U.S. National Institutes of Health here: beta.clinicaltrials.gov/study/NCT05044598?distance=50&cond=NCT05044598.

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