AniridiaPhase 1/2RAFTNCT05044598
RAFT - Clinical Trial of RAFT for Aniridia Related Keratopathy
A Phase 1/2 study testing RAFT-OS in people with Aniridia, run by University College, London at 1 site in United Kingdom.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Aniridia
- What is being tested?
- RAFT-OS
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- 28 Jul 2021to 17 Apr 2025
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- Poor tear production, as assessed by a Schirmer's test type 1
- Lid malposition (entropion, ectropion, fornix shortening, symblepharon)
- Current corneal infection
- Uncontrolled glaucoma (defined as uncontrolled eye pressure, changes to medication, recent surgery in the last 3 months or being considered for surgical treatment)
- Must not be NPL (no light perception) in one or both eyes
- Patients who refuse to consent to the site informing their GP of their participation
Show all 14Show fewer
- Patients who lack capacity to give full informed consent to participate
- Pregnant or lactating women
- Patients with known contraindications to any of the following non-investigational medicinal products; mycophenolate, prednisolone, omeprazole, doxycycline, dexamethasone \& moxifloxacin or excipients according to the relevant SmPCs
- Patients who are participating in any concurrent trial involving an investigational medical product, device or surgical intervention within the last 12 months
- Known albumin or egg allergy
- Known penicillin allergy
- Known hydrocortisone allergy
- Inability to lie flat for surgical procedure.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: RAFT - First in Human Phase I/II Clinical Trial of RAFT for Aniridia Related Keratopathy
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.
Read more
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.
- Conditions
- Aniridia
- Being tested
- RAFT-OS
- Medical subject headings
- Aniridia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RAFT-OSOther
RAFT-OS (Real Architecture for 3D Tissues Ocular Surface) is an artificial tissue, populated with limbal epithelial cells and stromal cells.
| Study group | Role | What they receive |
|---|---|---|
| RAFT-OS | Other | 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. Receives: Other: RAFT-OS |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: 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
Measured: 3 months
Secondary outcomes (3)
Change in visual acuity
- Visual improvement assessed by distance EDTRS visual acuity (refractive best corrected) at 3 \& 12 months post RAFT-OS transplantation.
Measured: 3 & 12 months post RAFT transplantation
changes in quality of life scores
as assessed by NEI-VFQ-25 patient questionnaire
Measured: at 3 & 12 months post RAFT transplantation
Changes in quality of Life scores
RAND 36-Item Health Survey.
Measured: 3 &12 months post RAFT transplantation.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Sep 2021
- Study start28 Jul 2021
- Primary completion17 Apr 2025
- Study completion17 Apr 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for AniridiaStudies of the same condition, open and under way.
- More trials run by University College, LondonThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- University College, London
- Sponsor type
- University, hospital or other
- Collaborators
- Medical Research Council, Moorfields Eye Hospital NHS Foundation Trust
- Responsible party
- Sponsor
- Investigators
- Sajjad Ahmad, Principal investigator, Moorfields Eye Hospital, LondonJulie Daniels, Study chair, University College, London
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05044598
- Sponsor's protocol ID
- CTU/2017/307
- Acronym
- RAFT
- Status last verified
- Apr 2025
- First submitted
- 19 May 2021
- Last updated
- 30 Apr 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Kaye AE, Morgan L, Shah R, Vernon AJ, Embleton-Thirsk A, Dehbi HM, Sibley D, Oreaba A, Daniels JT, Ahmad S. Combined Limbal Epithelial and Stromal Cell Transplant for Aniridia-Related Keratopathy: A Nonrandomized Clinical Trial. JAMA Ophthalmol. 2026 Jul 23:e262701. doi: 10.1001/jamaophthalmol.2026.2701. Online ahead of print.
PubMed 42490097
Study at a glance
From the registry record, last updated by the sponsor on 30 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- RAFT - First in Human Phase I/II Clinical Trial of RAFT for Aniridia Related Keratopathy
- ClinicalTrials.gov ID
- NCT05044598
- Acronym / sponsor ID
- RAFT / CTU/2017/307
- Conditions
- Aniridia
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 9 participants (actual)
- Lead sponsor
- University College, LondonUniversity, hospital or other
- Collaborators
- Medical Research Council, Moorfields Eye Hospital NHS Foundation Trust
- Study start
- 28 Jul 2021 (actual)
- Primary completion
- 17 Apr 2025 (actual)
- Study completion
- 17 Apr 2025 (actual)
- First posted
- 16 Sep 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| RAFT-OS | Other | Other: RAFT-OSSingle-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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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)
Key exclusion criteria (14 listed)
- Poor tear production, as assessed by a Schirmer's test type 1
- Lid malposition (entropion, ectropion, fornix shortening, symblepharon)
- Current corneal infection
- Uncontrolled glaucoma (defined as uncontrolled eye pressure, changes to medication, recent surgery in the last 3 months or being considered for surgical treatment)
- Must not be NPL (no light perception) in one or both eyes
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RAFT-OSRAFT-OS (Real Architecture for 3D Tissues Ocular Surface) is an artificial tissue, populated with limbal epithelial cells and stromal cells. | Other | RAFT-OS |
Primary outcome
Primary safety outcome defined as the number of adverse events related to the intervention
Time frame: 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.
Time frame: 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
3 secondary outcomes
Change in visual acuity
Time frame: 3 & 12 months post RAFT transplantation
changes in quality of life scores
Time frame: at 3 & 12 months post RAFT transplantation
Changes in quality of Life scores
Time frame: 3 &12 months post RAFT transplantation.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Apr 2025.
- Derived from this study: PubMed 42490097Kaye AE, Morgan L, Shah R, Vernon AJ, Embleton-Thirsk A, Dehbi HM, Sibley D, Oreaba A, Daniels JT, Ahmad S. Combined Limbal Epithelial and Stromal Cell Transplant for Aniridia-Related Keratopathy: A Nonrandomized Clinical Trial. JAMA Ophthalmol. 2026 Jul 23:e262701. doi: 10.1001/jamaophthalmol.2026.2701. Online ahead of print.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Sajjad Ahmad principal investigator
Moorfields Eye Hospital, London
Julie Daniels study chair
University College, London
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing RAFT-OS in people with Aniridia, run by University College, London at 1 site in United Kingdom.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 30 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Primary safety outcome defined as the number of adverse events related to the intervention (time frame: 12 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05044598, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Apr 2025.