Clinical Trial to Evaluate Safety and Efficacy of Cell Therapy in Patients With Cicatricial Conjuntivitis.

Stevens-Johnson Syndrome Clinical Trial

— CELOPHIN  

Official title:

Phase IIa Multicenter Clinical Trial to Determine the Feasibility and Safety of the Use of Adipose-derived Mesenchymal Stem Cells (ASC) in the Treatment of Patients With Cicatricial Conjunctivitis Associated With Lyell's Syndrome, Stevens-Johnson Syndrome and Pemphigoid of the Mucous Membranes With Ocular Involvement.

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT05520086
• Other study ID #: CELOPHIN
• Status: Active, not recruiting
• Phase: Phase 1/Phase 2
• Start date: November 11, 2022
• Est. completion date: February 2024

Study information

• Verified date: November 2023
• Source: Instituto de Investigación Sanitaria de la Fundación Jiménez Díaz
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

A phase IIa, open label, non controlled clinical trial to assess the feasibility and safety of allogeneic adipose-derived mesenchymal stem cells (ASC) in the treatment of cicatricial conjunctivitis associated with Lyell's syndrome, Stevens-Johnson's syndrome and mucous membrane pemphigoid with ocular involvement

Description:

A multicentre, nationwide clinical trial will be performed. 20 patients fulfilling eligibility criteria will be included in two different dose level cohorts: the first 10 patients included will receive a single cell application and the remainder 10 will be administered 2 cell applications separated by 15 days.

The investigational drug consists of locally administered, expanded, allogeneic adipose-derived adult mesenchymal stem cells (ASCs), at a dose of 5 million cells per ml (0,5 ml to be infused per quadrant)

Study cronogram: recruitment is estimated to take 8 months, and follow-up period will be 12 months.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 20
• Est. completion date: February 2024
• Est. primary completion date: February 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

1. Men and women over 18 years of age.

2. Diagnosis of ocular pemphigoid in Foster stages I-IIcIIIb (2) or diagnosis of recurrent chronic or episodic inflammation accompanied by cicatricial conjunctivitis of the mucous membranes with ocular involvement after the acute phase of Stevens-Johnson Syndrome or Lyell Syndrome with insufficient disease control or inaceptable toxicity or impossibility to administer usual care treatments (according to physician or patient criteria)

3. In the case of women of childbearing age, who are willing to use an effective contraceptive method during the period of participation in the study

4. Consent to participate and signature of the informed consent

Exclusion Criteria:

1. Signs of active infection on the ocular surface.

2. History of neoplasms in the last 5 years. except for epithelial basal or squamous cell carcinoma

3. Allergy to local anesthetics

4. Patients who have participated in another clinical trial with medication during the 90 days prior to signing the IC

5. Medical or psychiatric illness of any kind that, in the opinion of the investigator, may be a reason for exclusion from the study.

6. Congenital or acquired immunodeficiencies.

7. Major surgery or serious trauma of the subject in the semester prior to signing the IC.

8. Pregnant or lactating women.

9. Impossibility or refusal to carry out the follow-up required in the study by the patient

Related Conditions & MeSH terms

• Conjunctivitis
• Lyell Syndrome
• Pemphigoid
• Pemphigoid, Bullous
• Stevens-Johnson Syndrome
• Syndrome

Intervention

Drug:
• Single Dose
Single Dose: Administration of 12,5 million allogeneic mesenchymal stem cells derived from adipose tissue by subcnjunctival injection and topical conjunctival injection.
• Double Dose
Repetead Dose: Administration of 2 doses, separated by 14 days, of 12,5 million allogeneic mesenchymal stem cells derived from adipose tissue by subcnjunctival injection and topical conjunctival injection.

Locations

Country	Name	City	State
Spain	Hospital General La Mancha Centro	Alcázar De San Juan	Ciudad Real
Spain	Hospital Universitario de Cruces	Barakaldo	Bizkaia
Spain	Hospital La Arruzafa	Córdoba
Spain	Hospital General Universitario Gregorio Marañon	Madrid
Spain	Hospital Universitario Fundación Jiménez Diaz	Madrid
Spain	Hospital Universitario La Paz	Madrid
Spain	Instituto Oftalmológico Fernández - Vega	Oviedo	Asturias
Spain	Hospital Clínico Universitario de Valladolid	Valladolid

Sponsors (2)

Lead Sponsor	Collaborator
Instituto de Investigación Sanitaria de la Fundación Jiménez Díaz	Effice Servicios Para la Investigacion S.L.

Country where clinical trial is conducted

Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Percentage of complications	Percentage of complications related to anesthesia, administration of investigational medicinal product and postoperative period.	Through study completion, an average of 3 years
Secondary	Signs improvement	Changes from baseline in scarring conjunctivitis rating scale	At 7 days (and 21 days in the 2-dose group), 4 weeks, 12 weeks, 24 weeks and 52 weeks
Secondary	Symptom improvement	Changes from baseline in Ocular Surface Disease Index (OSDI) questionnaire (0-48). The lower the rate obtained in the score, better the outcome.	At 7 days (and 21 days in the 2-dose group), 4 weeks, 12 weeks, 24 weeks and 52 weeks
Secondary	Visual acuity improvement	Changes from baseline in Early Treatment Diabetic Retinopathy Study (ETDRS) (0-1,2).; The higher the rate obtained in the score, better the outcome.	At 7 days (and 21 days in the 2-dose group), 4 weeks, 12 weeks, 24 weeks and 52 weeks
Secondary	changes in quality of life	Changes from baseline using the specific National Eye Institute-Visual Function Questionnaire 25 (NEI-VFQ-25) (0-100). The value of 0 is the lowest and 100 the best possible	At 7 days (and 21 days in the 2-dose group), 4 weeks, 12 weeks, 24 weeks and 52 weeks
Secondary	evolution of the conjunctival flora	variation in the number and type of microorganisms in the conjunctival exudates, performing cultures and PCR herpes viridae	At 7 days (and 21 days in the 2-dose group), 4 weeks, 12 weeks, 24 weeks and 52 weeks