RAFT - Clinical Trial of RAFT for Aniridia Related Keratopathy
Study Details
Study Description
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 the 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 Liverpool. Participants will commence on 3 months of immune suppression therapy to prepare for the transplantation of RAFTOS.
The product will then be transplanted into the participants worst affected eye. Following surgery participants will be allowed to go home the following day, after the day 1 eye examination. Participants will then be followed up closely for the first 4 weeks following surgery for signs of infection or other complications. If there are no serious events, the next participant will be treated with RAFT-OS. The trial team will regularly follow participants after transplantation for 12 months, with detailed review of symptoms, investigations such as digital photography of the eye and examinations of the surface of the eye.
Participants will be required to stay on the immune suppression therapy for the duration of the trial.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 1/Phase 2 |
Study Design
Outcome Measures
Primary Outcome Measures
- Primary safety outcome defined as the number of adverse events related to the intervention [12 months]
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).
- primary efficacy outcome defined as Improvement in the corneal surface at 3months post intervention. [3 months]
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
Secondary Outcome Measures
- Change in visual acuity [3 & 12 months post RAFT transplantation]
1. Visual improvement assessed by distance EDTRS visual acuity (refractive best corrected) at 3 & 12 months post RAFT-OS transplantation.
- changes in quality of life scores [at 3 & 12 months post RAFT transplantation]
as assessed by NEI-VFQ-25 patient questionnaire
- Changes in quality of Life scores [3 &12 months post RAFT transplantation.]
RAND 36-Item Health Survey.
Eligibility Criteria
Criteria
Inclusion Criteria:
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Confirmed diagnosis of congenital aniridia
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Confirmed diagnosis of advanced aniridia related keratopathy with corneal surface conjunctivalisation, vascularisation and increasing opacity with worsening vision loss, glare & ocular surface pain
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Patients aged 18 years and over
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Participants must use acceptable contraception from enrolment up to 6 weeks for female participants and 90 days for male participants, after stopping immunosuppression therapy
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Negative viral screen for, HIV, syphilis, hepatitis B & C and Human T-cell Leukaemia Virus (HTLV)
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Negative urine pregnancy test.
Exclusion Criteria:
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Poor tear production, as assessed by a Schirmer's test type 1
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Lid malposition
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Current corneal infection
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Uncontrolled glaucoma (defined as uncontrolled eye pressure, changes to medication, recent surgery in the last 3 months or being considered for surgical treatment)
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Must not be NPL (no light perception) in one or both eyes
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Patients who refuse to consent to the site informing their GP of their participation
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Patients who lack capacity to give full informed consent to participate
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Pregnant or lactating women
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Patients with known contraindications to any of the following non-investigational medicinal products; mycophenolate, prednisolone, omeprazole, doxycycline, dexamethasone & moxifloxacin
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Patients who are participating in any concurrent trial involving an investigational medical product, device or surgical intervention within the last 12 months
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Known albumin or egg allergy
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Known penicillin allergy
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Known hydrocortisone allergy
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Inability to lie flat for surgical procedure.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Moorfields Eye Hospital | London | United Kingdom | EC1V 2PD | |
| 2 | Comprehensive Clinical Trials Unit | London | United Kingdom | WC1V 6LJ |
Sponsors and Collaborators
- University College, London
- Medical Research Council
- Moorfields Eye Hospital NHS Foundation Trust
Investigators
None specified.Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- CTU/2017/307