Limbal Stem Cell DeficiencyPhase 3NCT01489501
Multicenter Study of CAOMECS Transplantation to Patients With Total Limbal Stem Cell Deficiency
A Phase 3 study testing Surgical transplantation of CAOMECS to the ocular surface in people with Limbal Stem Cell Deficiency, run by CellSeed France S.A.R.L. at 1 site in Germany.
Trial summary
This clinical study is a scientific study on patients who do not have any limbal stem cells. In this clinical study tissue is taken from the inside of the mouth, and cells from that tissue (epithelial cells) will be grown to form a multilayered cell-sheet, called CAOMECS, which is then transplanted onto the cornea. Read more about this study
- Who is it for?
- Ages 2 to 74 years
- Conditions the study is for
- Limbal Stem Cell Deficiency
- What is being tested?
- Surgical transplantation of CAOMECS to the ocular surface
- Type of treatment
- Pill or oral medicine, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteGermany
- Study dates
- Start not givento not given
- Phase
- Phase 3
- 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 2 to 74 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Age ≥2 years to 74 years
- Males or females with bilateral or unilateral total LSCD due to one of the following causes:
- Chemical burns
- Thermal burns
- Contact lens wear
- Surgery of the ocular surface
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- Stevens-Johnson syndrome and other inflammatory disease under stable condition
- Aniridia
- Documented conjunctivalization of the corneal surface, measured by fluorescein staining
- Stable disease, i.e. history of LSCD for at least 6 months
- Clinical signs indicative of conjunctivalisation:
- Superficial blood vessels on the corneal surface
- Loss of epithelial transparency or persistent epithelial defect
- Healthy oral mucosa
- Absence from tobacco and alcohol (7 days before the biopsy)
- Regular tooth brushing (at least twice daily)
- Ability to comply with the protocol
- Covered by a social security system
- Signed informed consent form, ability to understand the study procedures, and contractual capability. Applicable to patient or legal carer (including parent)
- Special inclusion criteria for patients between 18 and 74 years of age (adults):
- Multiple surgeries in the limbal region
Who cannot join
- Acute systemic infection
- Acute ocular inflammation in the previous 6 months
- Previous neoplastic/cancer disease
- Severe dry eye confirmed by a Schirmer test
- Lyell-Syndrome, epidermolysis bullosa
- Total symblepharon
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- Medical history of hypersensitivity or allergy to bovine or murine derived materials
- Pregnant and lactating patients, positive urine pregnancy test in women of childbearing potential
- Any systemic infectious disease as diagnosed by serology tests such as syphilis, HIV-1, HIV-2, hepatitis B or C, or HTLV-1 infection at screening and at the day of the biopsy
- Current or previous (within 30 days of enrolment) treatment with another investigational drug or participation in another clinical study
- Previous participation of the patient in this study
- Evidence of any other medical conditions (such as psychiatric illness, physical examination or laboratory findings) that may interfere with the planned treatment, affect patient compliance or place the patient at high risk of treatment-related complications
- Employees of the sponsor or patients who are employees or relatives of the investigator
- Genetic conditions such as ectodermal dysplasia or multiple endocrine neoplasia
- Special exclusion criteria for patients ≥2 and <18 years of age (children):
- Multiple surgeries in the limbal region
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Multicenter Study of Cultured Autologous Oral Mucosal Epithelial Cell-Sheet (CAOMECS) Transplantation to Patients With Total Limbal Stem Cell Deficiency
This clinical study is a scientific study on patients who do not have any limbal stem cells. In this clinical study tissue is taken from the inside of the mouth, and cells from that tissue (epithelial cells) will be grown to form a multilayered cell-sheet, called CAOMECS, which is then transplanted onto the cornea. This transplantation method should repair the damage of the cornea.
The aim of this study is to see if the transplantation of CAOMECS renews the surface of the eye, by preventing the growth of the conjunctiva over the cornea and stopping new small blood vessels forming.
Read the detailed description
This is an open clinical study without a control group, which means that all patients will be given the study treatment (CAOMECS).
Adults as well as minor patients can participate in this clinical trial.
CAOMECS is undergoing a clinical trial, which means that it has not yet been officially approved for the treatment of this disease. Up to now it was clinically tested in France, on 26 people with limbal stem cell loss.
Study objective
The objective of this study is to restore the ocular surface epithelium of patients with total LSCD by preventing or reducing recurrent conjunctivalization and neovascularization up to 12 months post-transplantation.
Methodology
This is a prospective, open, multi-center study with CAOMECS in patients with total LSCD. Stem cells will be isolated from an oral mucosa biopsy and will be grown in cell culture. The resultant epithelial cell-sheets will then be transplanted to the ocular surface of the respective patients under general anesthesia. After transplantation patients may be hospitalized according to routine clinical practice.
Overall, the study will consist of a screening visit where eligibility will be evaluated, the day of the biopsy, the day before the transplantation where baseline data will be evaluated, the transplantation (day 0), and a follow-up period consisting of nine visits at days 1, 5, and 10 and months 1, 3, 6, 12, 24, and 36. Other visits as clinically indicated may also occur.
- Conditions
- Limbal Stem Cell Deficiency
- Being tested
- Surgical transplantation of CAOMECS to the ocular surface
- Keywords
- Caomecs, Transplantation, Limbal stem cell deficiency,
- Medical subject headings
- Limbal Stem Cell Deficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Surgical transplantation of CAOMECS to the ocular surfaceProcedure or surgery
Surgical transplantation of oral mucosa derived CAOMECS sheet onto eye cornea.
| Study group | Role | What they receive |
|---|---|---|
| only one arm available | Experimental | Caomecs transplantation on eye cornea. Receives: Procedure: Surgical transplantation of CAOMECS to the ocular surface |
What is being measured
The primary outcome is the main question the study is designed to answer.
Presence of a stable epithelium on cornea and absence of conjunctivalization in the visual axis
This is assessed by delayed fluorescein staining and impression cytology.
Measured: month 12
Extent of neovascularization
Measurement of the area of corneal neovascularisation as documented by photographs.
Measured: month 12
Secondary outcomes (2)
Presence of a stable epithelium on cornea and absence of conjunctivalization in the visual axis
This is assessed by delayed fluorescein staining and impression cytology.
Measured: month 24 and 36
Extent of neovascularization
Measurement of the area of corneal neovascularisation as documented by photographs.
Measured: month 24 and 36
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Dec 2011 (estimated)
Why it stopped: Change of strategic priorities in the sponsor's clinical development sector.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Limbal Stem Cell DeficiencyStudies 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
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- CellSeed France S.A.R.L.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- FGK Clinical Research GmbH
- Responsible party
- Sponsor
- Investigators
- Friedrich Kruse, Prof. Dr., Principal investigator, Medizinische Fakultät der Friedrich-Alexander-Universität Erlangen-Nürnberg
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01489501
- Sponsor's protocol ID
- CS001-EU01
- Other IDs
- 2011-000598-30
- Status last verified
- Mar 2015
- First submitted
- 8 Dec 2011
- Last updated
- 25 Mar 2015
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Mar 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multicenter Study of Cultured Autologous Oral Mucosal Epithelial Cell-Sheet (CAOMECS) Transplantation to Patients With Total Limbal Stem Cell Deficiency
- ClinicalTrials.gov ID
- NCT01489501
- Acronym / sponsor ID
- CS001-EU01
- Conditions
- Limbal Stem Cell Deficiency
- Keywords
- Caomecs, Transplantation, Limbal stem cell deficiency,
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Change of strategic priorities in the sponsor's clinical development sector.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- CellSeed France S.A.R.L.Industry (pharma, biotech, devices)
- Collaborators
- FGK Clinical Research GmbH
- First posted
- 9 Dec 2011 (estimated)
- 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 clinical study is a scientific study on patients who do not have any limbal stem cells. In this clinical study tissue is taken from the inside of the mouth, and cells from that tissue (epithelial cells) will be grown to form a multilayered cell-sheet, called CAOMECS, which is then transplanted onto the cornea. This transplantation method should repair the damage of the cornea.
The aim of this study is to see if the transplantation of CAOMECS renews the surface of the eye, by preventing the growth of the conjunctiva over the cornea and stopping new small blood vessels forming.
Detailed description
This is an open clinical study without a control group, which means that all patients will be given the study treatment (CAOMECS).
Adults as well as minor patients can participate in this clinical trial.
CAOMECS is undergoing a clinical trial, which means that it has not yet been officially approved for the treatment of this disease. Up to now it was clinically tested in France, on 26 people with limbal stem cell loss.
Study objective
The objective of this study is to restore the ocular surface epithelium of patients with total LSCD by preventing or reducing recurrent conjunctivalization and neovascularization up to 12 months post-transplantation.
Methodology
This is a prospective, open, multi-center study with CAOMECS in patients with total LSCD. Stem cells will be isolated from an oral mucosa biopsy and will be grown in cell culture. The resultant epithelial cell-sheets will then be transplanted to the ocular surface of the respective patients under general anesthesia. After transplantation patients may be hospitalized according to routine clinical practice.
Overall, the study will consist of a screening visit where eligibility will be evaluated, the day of the biopsy, the day before the transplantation where baseline data will be evaluated, the transplantation (day 0), and a follow-up period consisting of nine visits at days 1, 5, and 10 and months 1, 3, 6, 12, 24, and 36. Other visits as clinically indicated may also occur.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| only one arm available | Experimental | Procedure: Surgical transplantation of CAOMECS to the ocular surfaceCaomecs transplantation on eye cornea. |
Who may qualify
- Ages
- 2 to 74 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (21 listed)
- Age ≥2 years to 74 years
- Males or females with bilateral or unilateral total LSCD due to one of the following causes:
- Documented conjunctivalization of the corneal surface, measured by fluorescein staining
- Stable disease, i.e. history of LSCD for at least 6 months
- Clinical signs indicative of conjunctivalisation:
Key exclusion criteria (16 listed)
- Acute systemic infection
- Acute ocular inflammation in the previous 6 months
- Previous neoplastic/cancer disease
- Severe dry eye confirmed by a Schirmer test
- Lyell-Syndrome, epidermolysis bullosa
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 |
|---|---|---|
| Surgical transplantation of CAOMECS to the ocular surfaceSurgical transplantation of oral mucosa derived CAOMECS sheet onto eye cornea. | Procedure or surgery | only one arm available |
Primary outcome
Presence of a stable epithelium on cornea and absence of conjunctivalization in the visual axis
Time frame: month 12
This is assessed by delayed fluorescein staining and impression cytology.
Extent of neovascularization
Time frame: month 12
Measurement of the area of corneal neovascularisation as documented by photographs.
2 secondary outcomes
Presence of a stable epithelium on cornea and absence of conjunctivalization in the visual axis
Time frame: month 24 and 36
Extent of neovascularization
Time frame: month 24 and 36
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Mar 2015.
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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.
Friedrich Kruse, Prof. Dr. principal investigator
Medizinische Fakultät der Friedrich-Alexander-Universität Erlangen-Nürnberg
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Surgical transplantation of CAOMECS to the ocular surface in people with Limbal Stem Cell Deficiency, run by CellSeed France S.A.R.L. at 1 site in Germany.
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 21 inclusion and 16 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: withdrawn, as of the sponsor's last update on 25 Mar 2015. 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 Germany. 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.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
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?
Presence of a stable epithelium on cornea and absence of conjunctivalization in the visual axis (time frame: month 12). The study lists 2 primary outcomes in all. 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 25 Mar 2015.