NCT02577861

Efficacy and Safety of Autologous Cultivated Limbal Stem Cells Transplantation (ACLSCT) for Restoration of Corneal Epithelium in Patients With Limbal Stem Cell Deficiency

Multinational, Multicentre, Prospective, Open-label, Uncontrolled Clinical Trial to Assess the Efficacy and Safety of Autologous Cultivated Limbal Stem Cells Transplantation (ACLSCT) for Restoration of Corneal Epithelium in Patients With Limbal Stem Cell Deficiency Due to Ocular Burns (HOLOCORE)

• Status: Completed
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 70 (estimated)
• Sponsor: Holostem s.r.l. · Industry
• Sex: All
• Age: 2 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this trial is to confirm the efficacy of Holoclar at one year after the first treatment in patients suffering from moderate to severe LSCD (Limbal Stem Cell Deficiency) secondary to ocular burns. In case of failure of the treatment and upon clinical indication of the Principal Investigator, a second transplant with Holoclar will be offered if eligibility will be confirmed. The efficacy of two treatments with Holoclar at one year after the last treatment will be also evaluated. All patients will be followed-up for 12 months after each implant to determine the treatment efficacy by an Independent Assessor.

Detailed description

This is a multinational, multicentre, prospective, open label, uncontrolled clinical trial. Patients will be screened according to the Study Inclusion and Exclusion criteria and will be candidates for the ACLSCT if all eligibility criteria are met. Patients then will undergo limbal biopsy for the collection of limbal stem cells for product manufacturing. The confirmation of the eligibility criteria is followed by a roll-in phase of approximately 6 months. At the end of the roll-in period, Holoclar will be implanted through a specific surgical procedure. After ACLSCT, efficacy assessments will be performed at 1, 3, 6, 9 and 12 months for both first and second treatment (the latter only when applicable). One year after the transplantation, efficacy will be adjudicated by two independent assessors (primary and key secondary endpoints) and the study completion will be reached when 1 year of follow-up after the last transplant in the last patient will be accomplished.

Conditions

• Limbal Stem Cells Deficiency

Interventions

Type	Name	Description
PROCEDURE	Biopsy from donor eye	Corneal biopsy from undamaged limbus
PROCEDURE	Implant of Holoclar	Implant of Holoclar into the eye to be treated after scraping of the fibrovascular pannus
PROCEDURE	Ophtalmologic examination	The following assessments are performed: Epithelial defects assessment by fluorescein staining; superficial corneal neo-vascularization assessment; Best-Corrected Visual Acuity; ocular tonometry; slit lamp examination (both corneal endothelium and anterior chamber); conjunctival inflammation (both bulbar and limbal) assessment; corneal sensitivity and involvement assessment; Schirmer's test type I; evaluation of the following symptoms: pain, burning, photophobia.
PROCEDURE	Blood sample collection	Standard haematology, biochemistry and Infectious profile (Virology) are assessed
PROCEDURE	Digital pictures	At least 4 digital corneal slit lamp photos without fluorescein and at least 4 digital corneal slit lamp photos with fluorescein are taken using digital camera
PROCEDURE	ECG	A 12-lead ECG measurement in single recording at screening for safety reason and before each transplantation.
BEHAVIORAL	Questionnaires	Quality of Life assessment through the NEI VFQ 25and EQ-5D-3L questionnaire (EQ-5D-Y in paediatric population)
PROCEDURE	Physical examination and vital signs	A complete physical examination and vital signs (Systolic and Diastolic Blood Pressure (SBP, DBP) and Pulse Rate (PR) must be assessed at each visit (but not at pre-screening and at visit 6)

Timeline

• Start date: 2015-10-01
• Primary completion: 2020-12-01
• Completion: 2022-03-11
• First posted: 2015-10-16
• Last updated: 2022-04-01

Locations

1 site across 1 country: Italy