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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03288844
Recruitment Status : Recruiting
First Posted : September 20, 2017
Last Update Posted : August 3, 2020
Information provided by (Responsible Party):
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.

Condition or disease Intervention/treatment
Limbal Stem Cell Deficiency Due to Ocular Burn 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.

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Study Type : Observational
Estimated Enrollment : 70 participants
Observational Model: Case-Only
Time Perspective: Prospective
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
Actual Study Start Date : December 13, 2017
Estimated Primary Completion Date : December 2022
Estimated Study Completion Date : December 2022

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Burns

Group/Cohort Intervention/treatment
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

Primary Outcome Measures :
  1. Success of transplantation [ Time Frame: 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.

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
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.


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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03288844

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Contact: Clinical Trials Info +39 059 2058070

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Hospital San Raffaele Recruiting
Milan, Italy, 20100
Contact: Paolo Rama, MD         
Sponsors and Collaborators
Holostem Terapie Avanzate s.r.l.
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Study Director: Graziella Pellegrini, Professor Holostem Terapie Avanzate s.r.l.
Principal Investigator: Paolo Rama, MD San Raffaele Hospital
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Responsible Party: Holostem Terapie Avanzate s.r.l. Identifier: NCT03288844    
Other Study ID Numbers: CCD-GPLSCD01-03-FU
2015-001344-11 ( EudraCT Number )
First Posted: September 20, 2017    Key Record Dates
Last Update Posted: August 3, 2020
Last Verified: July 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Holostem Terapie Avanzate s.r.l.:
LSCD, Holoclar, follow-up
Additional relevant MeSH terms:
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Wounds and Injuries