Efficacy and Safety of Autologous Cultivated Limbal Stem Cells Transplantation (ACLSCT) for Restoration of Corneal Epithelium in Patients With Limbal Stem Cell Deficiency (HOLOCORE)
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|ClinicalTrials.gov Identifier: NCT02577861|
Recruitment Status : Active, not recruiting
First Posted : October 16, 2015
Last Update Posted : August 3, 2020
|Condition or disease||Intervention/treatment||Phase|
|Limbal Stem Cells Deficiency||Procedure: Biopsy from donor eye Procedure: Implant of Holoclar Procedure: Ophtalmologic examination Procedure: Blood sample collection Procedure: Digital pictures Procedure: ECG Behavioral: Questionnaires Procedure: Physical examination and vital signs||Phase 4|
This is a multinational, multicentre, prospective, open label, uncontrolled clinical trial.
Patients will be screened according to the Study Inclusion and Exclusion criteria and will be candidates for the ACLSCT if all eligibility criteria are met.
Patients then will undergo limbal biopsy for the collection of limbal stem cells for product manufacturing. The confirmation of the eligibility criteria is followed by a roll-in phase of approximately 6 months. At the end of the roll-in period, Holoclar will be implanted through a specific surgical procedure.
After ACLSCT, efficacy assessments will be performed at 1, 3, 6, 9 and 12 months for both first and second treatment (the latter only when applicable). One year after the transplantation, efficacy will be adjudicated by two independent assessors (primary and key secondary endpoints) and the study completion will be reached when 1 year of follow-up after the last transplant in the last patient will be accomplished.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||70 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Multinational, Multicentre, Prospective, Open-label, Uncontrolled Clinical Trial to Assess the Efficacy and Safety of Autologous Cultivated Limbal Stem Cells Transplantation (ACLSCT) for Restoration of Corneal Epithelium in Patients With Limbal Stem Cell Deficiency Due to Ocular Burns (HOLOCORE)|
|Actual Study Start Date :||October 2015|
|Estimated Primary Completion Date :||September 2020|
|Estimated Study Completion Date :||December 2021|
Treatment with Holoclar (medicinal product), including biopsy, graft production and implantation of the graft containing stem cell
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
Other Name: ACLSCT
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
A 12-lead ECG measurement in single recording at screening for safety reason and before each transplantation.
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)
- Success of transplantation [ Time Frame: 12 months after transplantation ]The percentage of patients with a success of first transplantation at approximately 12 months from the first Holoclar treatment. Transplantation success is defined on the basis of the degree of "superficial corneal neo-vascularization" (CNV) and "epithelial defects".
- Success of one or two transplantation(s) [ Time Frame: 12 months after the last transplantation ]
The percentage of patients with clinical success after one or two ACLSCTs assessed at 12 months after the last treatment with Holoclar.
Transplantation will be considered successful on the basis of the degree of "superficial corneal neo-vascularization" and "epithelial defects" according to the same definition as for the primary efficacy endpoint assessment.
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 ClinicalTrials.gov identifier (NCT number): NCT02577861
|Hospital San Raffaele|
|Milan, Italy, 20100|
|Study Director:||Graziella Pellegrini, Professor||Holostem Terapie Avanzate s.r.l.|
|Principal Investigator:||Paolo Rama, MD||Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ospedale San Raffaele|