CompletedPhase 1/2

RAFT - Clinical Trial of RAFT for Aniridia Related Keratopathy

United Kingdom9 participantsStarted 2021-07-28

Plain-language 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 can participate

Age range18 Years

SexALL

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Inclusion criteria

✓. Confirmed diagnosis of congenital aniridia
✓. Confirmed diagnosis of advanced aniridia related keratopathy with corneal surface conjunctivalisation, vascularisation and increasing opacity with worsening vision loss, glare \& ocular surface pain
✓. Patients aged 18 years and over
✓. Participants must use acceptable contraception from enrolment up to 6 weeks for female participants and 90 days for male participants, after stopping immunosuppression therapy
✓. Negative viral screen for, HIV, syphilis, hepatitis B \& C and Human T-cell Leukaemia Virus (HTLV)
✓. Negative urine pregnancy test.

Exclusion criteria

✕. Poor tear production, as assessed by a Schirmer's test type 1
✕. Lid malposition (entropion, ectropion, fornix shortening, symblepharon)
✕. Current corneal infection

What they're measuring

Primary safety outcome defined as the number of adverse events related to the intervention

Timeframe: 12 months

primary efficacy outcome defined as Improvement in the corneal surface at 3months post intervention.

Timeframe: 3 months

Trial details

NCT IDNCT05044598

SponsorUniversity College, London

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2025-04-17

View on ClinicalTrials.gov More Aniridia trials