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

Phase 4

Completed

• Conditions: 10030046; Corneal lesions with associated moderate to severe limbal stem cell deficiency due to ocular burns

• Registration Number: NL-OMON50240
• Lead Sponsor: Holostem Terapie Avenzate S.r.l

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 23 April 2024

Recruitment & Eligibility

• Status: Completed
• Sex: Not specified
• Target Recruitment: 5

Inclusion Criteria

1. Written informed consent prior to any study-related procedures;, 2. Adult
male and female patients (>=18 years old) - (Five Paediatric patients aged 2 to
17 years will be also enrolled for safety purposes only);, 3.LSCD secondary to
unilateral or bilateral physical or chemical ocular burns*, with at least 1-2
mm2 of undamaged limbus to harvest stem cells for expansion in culture. LSCD
will be considered for inclusion in presence of superficial neo-vascularization
invading at least two corneal quadrants with evidence of central corneal
(central 6 mm diameter) involvement (including corneal neo-vascularisation and
corneal opacity) according to the independent assessors;, 4.Stability of LSCD,
defined by a duration of disease of at least 24 months (12 months for minors)
at the time of the Screening Visit and as presence of continuum epithelium as
per fluorescein staining scored as none or trace; , 5. Presence of severe
impairment in visual acuity defined by a score after best correction (i.e. Best
Corrected Visual Acuity) equal or below 1/10 (or 20/200) at the Snellen chart
(legal blindness); , 6. Absence of other clinical contraindications to ACLSC
transplantation based upon investigator*s judgment;, 7. A cooperative attitude
to follow up the study procedures (Caregivers in case of minors).

Exclusion Criteria

1. LSCD of mild degree (i.e. below 2 quadrants of vascularization invasion),
due to a recent burn (less than 24 months before screening for adults and less
than 12 months before screening for minors), or secondary to medical conditions
other than burns (i.e.radiotherapy);, 2. Severe ocular inflammation according
to the Efron Grading Scale for Contact Lens Complications. Patient can be
re-screened after appropriate treatment; , 3. Presence of eyelids malposition;,
4. Conjunctival scarring with fornix shortening;, 5. Severe tear secretion
deficiency, determined by Schirmer*s test type 1 (<5 mm/ 5 min);, 6. Corneal
anaesthesia and conjunctival anaesthesia or severe hypoesthesia;, 7. Active
local or systemic infections at the time of screening. Patient can be
re-screened after appropriate treatment;, 8. Diagnosis of local or systemic
neoplastic disease;, 9. Congenital diseases (i.e., Aniridia);, 10. Bilateral
inflammatory diseases (i.e. Stevens-Johnson syndrome, phemphigoid);, 11. A
pre-existing blindness precluding a functional recovery;, 12. Female subjects:
pregnant or lactating women and all women physiologically capable of becoming
pregnant (i.e. women of childbearing potential) UNLESS are willing to use one
or more reliable methods of contraception (e.g. oral contraceptives, IUD, tubal
ligature). Reliable contraception should be maintained throughout the study.
A pregnancy test in urine will be performed at screening in all women of
childbearing potential, and repeated before biopsy and treatment. Any
postmenopausal women (physiologic menopause defined as *12 consecutive months
of amenorrhea*) or women permanently sterilized (e.g. tubal occlusion,
hysterectomy or bilateral salpingectomy) may be enrolled in the study
Parental control will be applied for the paediatric population when needed;,
13. Allergy, sensitivity or intolerance to concomitant drugs or excipients
(Hypersensitivity to any of the excipients listed in section 6.1 or to bovine
serum and murine 3T3-J2 cells);, 14; Contraindications to the local or systemic
antibiotics and/ or corticosteroids foreseen by the protocol;, 15.
Contraindications to the surgical procedure;, 16. Clinically significant or
unstable concurrent disease or other clinical contraindications to stem cell
transplantation based upon investigator*s judgment or other concomitant medical
conditions affecting grafting procedure;, 17. Patients (or parents in case of
paediatric subject) unlikely to comply with the study protocol or unable to
understand the nature and scope of the study or the possible benefits or
unwanted effects of the study procedures and treatments;, 18. Participation in
another clinical trial where investigational drug was received less than 4
weeks prior to screening visit.

Study & Design

• Study Type: Interventional
• Study Design: Not specified