RAFT – Clinical Trial of RAFT for Aniridia Related Keratopathy

Brief Title

RAFT - Clinical Trial of RAFT for Aniridia Related Keratopathy

Official Title

RAFT - First in Human Phase I/II Clinical Trial of RAFT for Aniridia Related Keratopathy

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.
    


Study Phase

Phase 1/Phase 2

Study Type

Interventional


Primary Outcome

Safety

Secondary Outcome

 Visual Improvement

Condition

Aniridia

Intervention

RAFT-OS


Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Device

Estimated Enrollment

21

Start Date

July 28, 2021

Completion Date

November 2022

Primary Completion Date

November 2022

Eligibility Criteria

        Inclusion Criteria:

          1. Confirmed diagnosis of congenital aniridia

          2. Confirmed diagnosis of advanced aniridia related keratopathy with corneal surface
             conjunctivalisation, vascularisation and increasing opacity with worsening vision
             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.

        Exclusion Criteria:

          1. Poor tear production, as assessed by a Schirmer's test type 1

          2. Lid malposition

          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 & moxifloxacin

         10. Patients who are participating in any concurrent 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.
      

Gender

All

Ages

18 Years - N/A

Accepts Healthy Volunteers

No

Contacts

, 020 7907 4674, [email protected]

Location Countries

United Kingdom

Location Countries

United Kingdom

Administrative Informations


NCT ID

NCT05044598

Organization ID

CTU/2017/307


Responsible Party

Sponsor

Study Sponsor

University College, London

Collaborators

 Medical Research Council

Study Sponsor

, , 


Verification Date

September 2021