Safety Study and Preliminary Efficacy of Infusion Haploidentical Mesenchymal Stem Cells Derived From Bone Marrow for Treating Recessive Dystrophic Epidermolysis Bullosa

NCT04153630 | Unknown Phase 1

• 1 other identifier: MesenSistem-EB
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 1

Brief Summary

Phase I / II pilot clinical trial, to evaluate the safety and preliminary efficacy of the systemic infusion of mesenchymal stem cells derived from bone marrow (BM-MSCs) from a haploidentical donor to improve the healing process and / or the mucocutaneous fragility phenotype associated with EBDR.

Conditions

Epidermolysis Bullosa Dystrophica, Recessive

Outcome Measures

Primary Outcomes (1)

• Safety evaluation: Incidence of Treatment-Emergent Adverse Events as assessed by protocol.

  To evaluate the safety of haploidentical MSCs derived from bone marrow administered by intravenous injection with a dose of 2-3x106 cells / Kg in 3 infusions separated by 21 days each for the treatment of patients with RDEB: All adverse events will be registered for 1 year from first infusion of cells as assessed by grade: mild, moderate or severe (according to protocol)

  1 year after infusion

Secondary Outcomes (17)

• Cutaneous mechanical resistance

  2 year after infusion

• Skin surface affected

  2 year after infusion

• Number of blisters

  2 year after infusion

• Non-specific general markers of systemic inflammation: white blood cell count

  2 year after infusion

• Non-specific general markers of systemic inflammation: Negative acute phase reactant (albumin)

  2 year after infusion

Study Arms (1)

Haploidentical MSCs derived from bone marrow

EXPERIMENTAL

Haploidentical MSCs derived from bone marrow administered by intravenous injection with a dose of 2-3x106 cells / Kg

Biological: mesenchymal stem cells derived from bone marrow (BM-MSCs)

Interventions

mesenchymal stem cells derived from bone marrow (BM-MSCs) BIOLOGICAL

Procedure: Haploidentical MSCs derived from bone marrow administered by intravenous injection with a dose of 2-3x106 cells / Kg

Haploidentical MSCs derived from bone marrow

Eligibility Criteria

• Age: 12 Months - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Patients with a clinical, molecular and genetic diagnosis of EBDR.
• Patients with presence of the NC-1 domain of type VII collagen, in skin biopsies and/or Western-Blot, detected with a battery of specific antibodies.
• Patients with a haploidentical donor.
• Subjects with a severity score\> 20 according to "The Birmingham Epidermolysis Bullosa Severity Score".
• Minor subjects whose representative / legal guardian has voluntarily signed the informed consent before the first intervention of the study.
• In the case of mature minors (12-17 years of age), in addition to the consent signed by the legal guardian, the consent of the minor will be obtained.
• Males should agree to use a double-barrier contraceptive method (condom plus spermicide or diaphragm plus spermicide) during their participation in the study and up to 30 days after the last dose of the study drug, or the male patient or his Female partners must be surgically sterilized or the female partner must be postmenopausal.
• The patient must be able to attend all study visits and comply with all study procedures.

You may not qualify if:

• Subjects who for medical reasons can not be moved to the University Hospital La Paz in Madrid.
• Subjects with a known allergy to any of the components of the investigational product (including penicillin and streptomycin), or who can not receive treatment with antihistamines and/or corticosteroids.
• Subjects with a history or signs of malignancy, including cutaneous squamous cell carcinoma.
• Subjects with circulating anti-C7 antibodies and anti-C7 antibodies deposited in the dermo-epidermal junction detected in skin biopsies by indirect immunofluorescence.
• Subjects to whom other investigational drugs have been administered in the 90 days prior to the treatment phase.
• Subjects who are unable to understand the information sheet and unable to sign the informed consent.

Sponsors & Collaborators

• Instituto de Investigación Hospital Universitario La Paz: lead
• Universidad Carlos III Madrid (TERMeG): collaborator
• St John's Institute of Dermatology Kings College London: collaborator
• Instituto de Salud Carlos III: collaborator
• DEBRA: collaborator
• CIBER Enfermedades raras: collaborator

Study Sites (1)

Hospital Universitario La Paz

Madrid, 28046, Spain

Location

MeSH Terms

Conditions

Epidermolysis Bullosa Dystrophica

Condition Hierarchy (Ancestors)

Epidermolysis Bullosa; Skin Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases, Genetic; Genetic Diseases, Inborn; Collagen Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases; Skin Diseases, Vesiculobullous

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 6, 2019
• First Posted: November 6, 2019
• Study Start: May 17, 2018
• Primary Completion: March 1, 2021
• Study Completion: March 1, 2021
• Last Updated: November 8, 2019

Record last verified: 2019-04

Locations

ES (1)