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

Phase 1

Active, not recruiting

• Conditions: Chronic Conjunctivitis; Stevens-Johnson Syndrome; Lyell Syndrome; Pemphigoid
• Interventions: Drug: Single Dose; Drug: Double Dose

• Registration Number: NCT05520086
• Lead Sponsor: Instituto de Investigación Sanitaria de la Fundación Jiménez Díaz

Brief 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

Detailed 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.

Study Timeline

• Study First Submitted: 2022-08-22
• Study First Submitted QC: 2022-08-26
• Study First Posted: 2022-08-29
• Study Start: 2022-11-11
• Status Verified: 2023-11
• Primary Completion: 2024-06-11
• Last Update Submitted: 2024-07-05
• Last Update Posted: 2024-07-08
• Study Completion: 2024-11-11

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 20

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
Single dose Allogenic Adiposse derived mesenchimal stem cells	Single Dose	Allogenic Adiposse derived mesenchimal stem cells (MSC) Dose: 12,5 million cells
Double dose Allogenic Adiposse derived mesenchimal stem cells	Double Dose	Allogenic Adiposse derived mesenchimal stem cells (MSC) Dose: 12,5 million cells at day 0 and 12,5 million cells at day 14

Primary Outcome Measures

Name	Time	Method
Percentage of complications	Through study completion, an average of 3 years	Percentage of complications related to anesthesia, administration of investigational medicinal product and postoperative period.

Secondary Outcome Measures

Name	Time	Method
changes in quality of life	At 7 days (and 21 days in the 2-dose group), 4 weeks, 12 weeks, 24 weeks and 52 weeks	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
Signs improvement	At 7 days (and 21 days in the 2-dose group), 4 weeks, 12 weeks, 24 weeks and 52 weeks	Changes from baseline in scarring conjunctivitis rating scale
Symptom improvement	At 7 days (and 21 days in the 2-dose group), 4 weeks, 12 weeks, 24 weeks and 52 weeks	Changes from baseline in Ocular Surface Disease Index (OSDI) questionnaire (0-48). The lower the rate obtained in the score, better the outcome.
Visual acuity improvement	At 7 days (and 21 days in the 2-dose group), 4 weeks, 12 weeks, 24 weeks and 52 weeks	Changes from baseline in Early Treatment Diabetic Retinopathy Study (ETDRS) (0-1,2).; The higher the rate obtained in the score, better the outcome.
evolution of the conjunctival flora	At 7 days (and 21 days in the 2-dose group), 4 weeks, 12 weeks, 24 weeks and 52 weeks	variation in the number and type of microorganisms in the conjunctival exudates, performing cultures and PCR herpes viridae

Trial Locations

Locations (8)

Hospital General Universitario Gregorio Marañon; 🇪🇸 Madrid, Spain

Hospital Universitario de Cruces; 🇪🇸 Barakaldo, Bizkaia, Spain

Instituto Oftalmológico Fernández - Vega; 🇪🇸 Oviedo, Asturias, Spain

Hospital General La Mancha Centro; 🇪🇸 Alcázar De San Juan, Ciudad Real, Spain

Hospital La Arruzafa; 🇪🇸 Córdoba, Spain

Hospital Universitario La Paz; 🇪🇸 Madrid, Spain

Hospital Clínico Universitario de Valladolid; 🇪🇸 Valladolid, Spain

Hospital Universitario Fundación Jiménez Diaz; 🇪🇸 Madrid, Spain