Clinical trial NCT05520086: Clinical Trial to Evaluate Safety and Efficacy of Cell Therapy in Patients With Cicatricial Conjuntivitis.
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.
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE1
PHASE2
Conditions: Chronic Conjunctivitis
Stevens-Johnson Syndrome
Lyell Syndrome
Pemphigoid
Lead sponsor: Instituto de Investigación Sanitaria de la Fundación Jiménez Díaz
Sponsor class: OTHER
Collaborators: name: Effice Servicios Para la Investigacion S.L.
First posted: 2022-08-29
Enrollment: 20
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SEQUENTIAL
Intervention model description: 2 consecutive cohorts will be included in order of recruitment. the first 10 patients will receive 1 single dose and the second 10 patients will receive 2 doses separated by 15 days
Primary purpose: TREATMENT

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.

Eligibility criteria: 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

Primary outcomes:
measure: Percentage of complications
timeFrame: Through study completion, an average of 3 years
Secondary outcomes:
measure: Signs improvement
timeFrame: At 7 days (and 21 days in the 2-dose group), 4 weeks, 12 weeks, 24 weeks and 52 weeks
measure: Symptom improvement
timeFrame: At 7 days (and 21 days in the 2-dose group), 4 weeks, 12 weeks, 24 weeks and 52 weeks
measure: Visual acuity improvement
timeFrame: At 7 days (and 21 days in the 2-dose group), 4 weeks, 12 weeks, 24 weeks and 52 weeks
measure: changes in quality of life
timeFrame: At 7 days (and 21 days in the 2-dose group), 4 weeks, 12 weeks, 24 weeks and 52 weeks
measure: evolution of the conjunctival flora
timeFrame: At 7 days (and 21 days in the 2-dose group), 4 weeks, 12 weeks, 24 weeks and 52 weeks
Other outcomes:
Locations:
city: Oviedo
country: Spain
facility: Instituto Oftalmológico Fernández - Vega
state: Asturias
city: Barakaldo
country: Spain
facility: Hospital Universitario de Cruces
state: Bizkaia
city: Alcázar De San Juan
country: Spain
facility: Hospital General La Mancha Centro
state: Ciudad Real
city: Córdoba
country: Spain
facility: Hospital La Arruzafa
city: Madrid
country: Spain
facility: Hospital General Universitario Gregorio Marañon
city: Madrid
country: Spain
facility: Hospital Universitario Fundación Jiménez Diaz
city: Madrid
country: Spain
facility: Hospital Universitario La Paz
city: Valladolid
country: Spain
facility: Hospital Clínico Universitario de Valladolid
Study officials:
affiliation: Hospital Fundación Jiménez Diaz
name: Nicolás Alejandre Alba
role: PRINCIPAL_INVESTIGATOR
References: