Clinical Drug Experience Knowledgebase

Criteria

Inclusion Criteria:

Male and female patients ≥ 12 months and ≤ 18 years of age at the time of inclusion in the study.
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.
Women with reproductive capacity must have a negative pregnancy test at the time of inclusion and must agree to use highly effective contraceptive methods (diaphragms plus spermicide or male condom plus spermicide, oral contraceptive combined with a second method contraceptive implant, contraceptive injectable, permanent intrauterine device, sexual abstinence or partner with vasectomy) during their participation in the study until 30 days after the last visit.
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.

Exclusion Criteria:

Subjects who for medical reasons can not be moved to the University Hospital La Paz in Madrid.
Subjects who have received immunotherapy including oral corticosteroids (> 15 mg / day) for more than 1 week (excluding inhaled and ophthalmic preparations) or chemotherapy 8 days prior to inclusion in the study. The inclusion of the patient is understood from the signing of the informed consent.
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 signs of active systemic infection at the time of inclusion in the study. In any case, according to the researcher's criteria, the patient can be reevaluated for inclusion in the study after improvement of the infectious pathology.
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.
Pregnant women at the time of inclusion or women of childbearing age who do not practice abstinence or employ acceptable means of contraception, as determined by the investigator during the trial.
Biochemical abnormalities at the time of inclusion: albumin <2.5 g / dL, Hemoglobin <7.5 g / dL.
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.