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

Completed

• Conditions: Limbal Stem Cell Deficiency Due to Ocular Burn
• Interventions: Procedure: Ophthalmologic examinations; Other: Digital pictures; Other: QoL Questionnaires

• Registration Number: NCT03288844
• Lead Sponsor: Holostem Terapie Avanzate s.r.l.

Brief Summary

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.

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 Timeline

• Study First Submitted: 2017-09-18
• Study First Submitted QC: 2017-09-19
• Study First Posted: 2017-09-20
• Study Start: 2017-12-13
• Primary Completion: 2023-03-31
• Study Completion: 2023-03-31
• Status Verified: 2023-05
• Last Update Submitted: 2023-05-25
• Last Update Posted: 2023-05-26

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 47

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 & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
HOLOCORE Patients	Digital pictures	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
HOLOCORE Patients	QoL Questionnaires	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
HOLOCORE Patients	Ophthalmologic examinations	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

Primary Outcome Measures

Name	Time	Method
Success of transplantation	1 year	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.

Trial Locations

Locations (1)

Hospital San Raffaele; 🇮🇹 Milan, Italy