A Phase 1/2 interventional study of RAFT-OS in Aniridia, sponsored by University College, London. Completed at 1 site in United Kingdom. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2025-04-30.
Sponsored by University College, London · Phase 1/2, Interventional, and Treatment
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.
23 studies on the registry are indexed under Aniridia; 8 are open to participants now.
Browse Aniridia studies →University College, London is the lead sponsor of 632 studies on the registry; 145 are open to participants now.
Of its 6 completed or terminated interventional studies of FDA-regulated products, 2 (33%) have results posted.
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Exclusion Criteria:
Single-arm trial is to investigate if RAFT-OS ((Real Architecture for 3D Tissues Ocular Surface) is a safe and effective alternative treatment for patients with aniridia related keratopathy (ARK) in 21 patients. 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.
Other: RAFT-OS
RAFT-OS (Real Architecture for 3D Tissues Ocular Surface) is an artificial tissue, populated with limbal epithelial cells and stromal cells.
Primary safety outcome defined as the number of adverse events related to the intervention
defined as either major adverse event (non-ocular SUSAR; corneal melting; death) or any intermediate adverse event (persisting epithelial defect; 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).
Time frame: 12 months
primary efficacy outcome defined as Improvement in the corneal surface at 3months post intervention.
Efficacy will be based on 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
Time frame: 3 months
Change in visual acuity
1. Visual improvement assessed by distance EDTRS visual acuity (refractive best corrected) at 3 \& 12 months post RAFT-OS transplantation.
Time frame: 3 & 12 months post RAFT transplantation
changes in quality of life scores
as assessed by NEI-VFQ-25 patient questionnaire
Time frame: at 3 & 12 months post RAFT transplantation
Changes in quality of Life scores
RAND 36-Item Health Survey.
Time frame: 3 &12 months post RAFT transplantation.
Plan to share: No
No publications or documents are linked to this record.
This study is completed, as verified in Apr 2025. You cannot join it, but the record below documents what was studied.
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University College, London