Limbal Stem Cell Deficiency Due to Ocular BurnHOLOCORE-FUNCT03288844
Follow-up Study After ACLSCT for Restoration of Corneal Epithelium in Patients With LSCD Due to Ocular Burns
An observational study of Limbal Stem Cell Deficiency Due to Ocular Burn, run by Holostem s.r.l. at 1 site in Italy.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Limbal Stem Cell Deficiency Due to Ocular Burn
- What is being tested?
- Ophthalmologic examinations, Digital pictures and QoL Questionnaires
- Type of treatment
- Surgery or procedure
- How many people
- 47 participants
- Where
- 1 siteItaly
- Study dates
- 13 Dec 2017to 31 Mar 2023
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
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.
Who may be able to join
- 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.
Who cannot join
- No specific exclusion criterion is considered for this study, except for patients dropping out from the HOLOCORE study or withdrawing consent.
Trial locations and contacts
1 site in 1 country.
About this study
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
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.
Read the 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.
- Being tested
- Ophthalmologic examinations, Digital pictures, QoL Questionnaires
- Keywords
- LSCD, Holoclar, follow-up
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Ophthalmologic examinationsProcedure or surgery
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.
Digital picturesOther
Digital pictures 2D to be taken at each visit
QoL QuestionnairesOther
Quality of Life (NEI VFQ 25 and EQ-5D-3L/Y) questionnaires will be submitted at each visit
| Study group | Role | What they receive |
|---|---|---|
| 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 Receives: Procedure: Ophthalmologic examinations; Other: Digital pictures; Other: QoL Questionnaires |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Sep 2017
- Study start13 Dec 2017
- Primary completion31 Mar 2023
- Study completion31 Mar 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Limbal Stem Cell Deficiency Due to Ocular BurnStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 47 participants (actual)
Sponsor and investigators
- Lead sponsor
- Holostem s.r.l.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Cromsource
- Responsible party
- Sponsor
- Investigators
- Graziella Pellegrini, Professor, Study director, Holostem s.r.l.Paolo Rama, MD, Study chair, Fondazione I.R.C.C.S. Policlinico San Matteo, Pavia, Italy
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03288844
- Sponsor's protocol ID
- CCD-GPLSCD01-03-FU
- Other IDs
- 2015-001344-11, 2015
- Acronym
- HOLOCORE-FU
- Status last verified
- May 2023
- First submitted
- 18 Sep 2017
- Last updated
- 26 May 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 May 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT03288844
- Acronym / sponsor ID
- HOLOCORE-FU / CCD-GPLSCD01-03-FU
- Keywords
- LSCD, Holoclar, follow-up
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 47 participants (actual)
- Lead sponsor
- Holostem s.r.l.Industry (pharma, biotech, devices)
- Collaborators
- Cromsource
- Study start
- 13 Dec 2017 (actual)
- Primary completion
- 31 Mar 2023 (actual)
- Study completion
- 31 Mar 2023 (actual)
- First posted
- 20 Sep 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| HOLOCORE Patients | – | Procedure: Ophthalmologic examinations, Other: Digital pictures, Other: QoL QuestionnairesPatients 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 |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- 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.
Key inclusion criteria (1 listed)
- 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.
Key exclusion criteria (1 listed)
- No specific exclusion criterion is considered for this study, except for patients dropping out from the HOLOCORE study or withdrawing consent.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Ophthalmologic examinationsepithelial 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. | Procedure or surgery | HOLOCORE Patients |
| Digital picturesDigital pictures 2D to be taken at each visit | Other | HOLOCORE Patients |
| QoL QuestionnairesQuality of Life (NEI VFQ 25 and EQ-5D-3L/Y) questionnaires will be submitted at each visit | Other | HOLOCORE Patients |
Primary outcome
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.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 May 2023.
- Individual participant dataSharing planned: No
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Graziella Pellegrini, Professor study director
Holostem s.r.l.
Paolo Rama, MD study chair
Fondazione I.R.C.C.S. Policlinico San Matteo, Pavia, Italy
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Limbal Stem Cell Deficiency Due to Ocular Burn, run by Holostem s.r.l. at 1 site in Italy.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 26 May 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Italy. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Success of transplantation (time frame: 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 May 2023.