Follow-up Study After ACLSCT for Restoration of Corneal Epithelium in Patients With LSCD Due to Ocular Burns (HOLOCORE-FU)

Multinational, Multicenter, Prospective, Long-term Safety and Efficacy Follow-up Study After Autologous Cultivated Limbal Stem Cells Transplantation (ACLSCT) for Restoration of Corneal Epithelium in Patients With Limbal Stem Cell Deficiency Due to Ocular Burns

This is a multinational, multicentre, prospective, non-pharmacological follow-up study of the clinical trial HOLOCORE. All patients transplanted with Holoclar in the HOLOCORE clinical trial who consent to participate will be enrolled in this prospective study and observed for at least 12 months.

Study Overview

• Status: Completed
• Conditions: Limbal Stem Cell Deficiency Due to Ocular Burn
• Intervention / Treatment: Procedure: Ophthalmologic examinations; Other: Digital pictures; Other: QoL Questionnaires

Detailed Description

HOLOCORE FOLLOW-UP prospective study offers the opportunity to collect long-term efficacy and safety data after one or two treatments with Holoclar. Moreover, since Holoclar (study drug used in the main HOLOCORE interventional clinical trial) is suspected to improve the success of keratoplasty in those patients candidate to receive this treatment but no data were collected in the three previous retrospective studies (HLSTM01, HLSTM02 and HLSTM04) on Holoclar, this follow-up study has been thought to observe the progress of the disease and to collect data on keratoplasty and evaluate the benefits of Holoclar in subsequent keratoplasty in patients treated once or two times with Holoclar.

All patients treated in the HOLOCORE clinical trial who consent to roll over to the present extension study at the end of the HOLOCORE will be observed for a follow-up period which may vary from a minimum of 12 month for the last patient to a maximum of 57 months for the first patient entered.

A total of approximately 70 patients is expected (all patients who completed the HOLOCORE study).

In case the last enrolled patient undergoes to Keratoplasty at the Last Visit of the Follow-up study, this will consequently lead to a prolongation of the study up to further 12 months for all patients.

The subjects will be enrolled and observed at the same investigational centers where they were recruited for the HOLOCORE clinical trial.

No drug will be administered for the scope of this study. Ophtalmological visits including pictures collection and quality of life evaluations will be performed every 6 months.

• Study Type: Observational
• Enrollment (Actual): 47

Contacts and Locations

Study Locations

• Italy
  • Milan, Italy, 20100
    • Hospital San Raffaele

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

All eligible patients who entered the core HOLOCORE study will be asked consent to roll over into this extension study before their inclusion. Approximately 70 patients will be enrolled in this study.

Safety population: all patients entering the study. The adult safety population will be used to explore efficacy. Efficacy data collected on paediatric population will be only listed.

Description

Inclusion Criteria:

• All patients (adults and pediatrics) who completed the HOLOCORE core study and who consent to roll over to the present extension study at the end of the HOLOCORE follow-up.

Exclusion Criteria:

• No specific exclusion criterion is considered for this study, except for patients dropping out from the HOLOCORE study or withdrawing consent.

Study Plan

How is the study designed?

Design Details

• Observational Models: Case-Only
• Time Perspectives: Prospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort

Intervention / Treatment

HOLOCORE Patients; Patients who completed the main HOLOCORE clinical trial will undergo to Ophthalmologic examinations, Digital pictures collection and QoL questionnaires (NEI VFQ 25 and EQ-5D-3L/Y) will be performed/administered at each study visit

Procedure: Ophthalmologic examinations; epithelial defects assessment by fluorescein staining; superficial corneal neo-vascularization assessment; ocular tonometry; slit lamp examination; conjunctival (both bulbar and limbal) inflammation assessment; corneal sensitivity; central cornea involvement (for opacity or central CNV); Best-Corrected Visual Acuity; evaluation of symptoms.; Other: Digital pictures; Digital pictures 2D to be taken at each visit; Other: QoL Questionnaires; Quality of Life (NEI VFQ 25 and EQ-5D-3L/Y) questionnaires will be submitted at each visit

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Success of transplantation	Transplantation will be considered with a clinically-relevant success if the severity of superficial corneal neo-vascularization will be judged as not exceeding one quadrant of invasion (without central cornea involvement) by the investigator and the degree of Epithelial defect as 'None' or 'Trace', respectively.	1 year

Collaborators and Investigators

• Sponsor: Holostem Terapie Avanzate s.r.l.
• Collaborators: Cromsource
• Investigators: Study Director: Graziella Pellegrini, Professor, Holostem Terapie Avanzate s.r.l.; Study Chair: Paolo Rama, MD, Fondazione I.R.C.C.S. Policlinico San Matteo, Pavia, Italy

Study record dates

Study Major Dates

• Study Start (Actual): December 13, 2017
• Primary Completion (Actual): March 31, 2023
• Study Completion (Actual): March 31, 2023

Study Registration Dates

• First Submitted: September 18, 2017
• First Submitted That Met QC Criteria: September 19, 2017
• First Posted (Actual): September 20, 2017

Study Record Updates

• Last Update Posted (Actual): May 26, 2023
• Last Update Submitted That Met QC Criteria: May 25, 2023
• Last Verified: May 1, 2023

More Information

Terms related to this study

• Keywords: LSCD, Holoclar, follow-up
• Additional Relevant MeSH Terms: Eye Diseases; Wounds and Injuries; Corneal Diseases; Burns; Limbal Stem Cell Deficiency
• Other Study ID Numbers: CCD-GPLSCD01-03-FU; 2015-001344-11 (EudraCT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No