Clinical trial NCT03288844: Follow-up Study After ACLSCT for Restoration of Corneal Epithelium in Patients With LSCD Due to Ocular Burns
Official title: 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
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Limbal Stem Cell Deficiency Due to Ocular Burn
Keywords: LSCD, Holoclar, follow-up
Lead sponsor: Holostem s.r.l.
Sponsor class: INDUSTRY
Collaborators: name: Cromsource
First posted: 2017-09-20
Enrollment: 47
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
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.

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Success of transplantation
timeFrame: 1 year
Secondary outcomes:
Other outcomes:
Locations:
city: Milan
country: Italy
facility: Hospital San Raffaele
Study officials:
affiliation: Holostem s.r.l.
name: Graziella Pellegrini, Professor
role: STUDY_DIRECTOR
affiliation: Fondazione I.R.C.C.S. Policlinico San Matteo, Pavia, Italy
name: Paolo Rama, MD
role: STUDY_CHAIR
References: