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

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.

Conditions

• Limbal Stem Cells Deficiency

Interventions

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)

Sponsors & Collaborators

• Holostem s.r.l.

  lead INDUSTRY

Principal Investigators

• Graziella Pellegrini, Professor · Holostem s.r.l.

• Paolo Rama, MD · Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ospedale San Raffaele

Study Design

Allocation

NA

Purpose

TREATMENT

Masking

NONE

Model

SINGLE_GROUP

Eligibility

Min Age

2 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2015-10-31

Primary Completion

2020-12-31

Completion

2022-03-11

Countries

• Italy

Study Locations