EUCTR2017-000606-37-ESActive, not recruitingPhase 1
Safety and preliminary efficacy study of infusing mesenchymal stem cells derived from bone marrow for treating Recessive Dystrophic Epidermolysis Bullosa.
Fundación para la Investigación Biomédica Hospital Universitario La Paz0 sites9 target enrollmentStarted: September 18, 2017Last updated:
Conditions
Trial Snapshot
- Phase
- Phase 1
- Status
- Active, not recruiting
- Sponsor
- Enrollment
- 9
Study Overview
Brief Summary
No summary available.
Study Design
- Study Type
- Interventional clinical trial of medicinal product
Eligibility Criteria
- Sex
- All
Inclusion Criteria
- •1. Male or female patients = 4 and = 18 years old at the time of inclusion in the study.
- •2. Patients with clinical, molecular and genetic diagnosis of EBDR.
- •3. Patients with presence of the NC-1 domain of type VII collagen in skin and/or western blot biopsies detected with a battery of specific antibodies
- •4. Patients with haploidentical donor
- •5. Subjects with a severity score of> 20 according to The Birmingham Epidermolysis Bullosa Severity Score.
- •6. Minors whose legal representative / guardian has voluntarily signed informed consent prior to the first study intervention.
- •7. In the case of mature minors (12-17 years of age), in addition to the consent signed by the legal guardian, the child's consent will be obtained.
- •8. Women with reproductive capacity should have a negative pregnancy test at the time of inclusion and must have access to highly effective contraceptive methods (diaphragms plus spermicide or male condom plus spermicide, oral contraceptive combined with a second contraceptive implant method, contraceptive Injecting, permanent intrauterine device, sexual abstinence or partner with vasectomy) during their participation in the study and in the 30 following the last visit.
- •9. Males should agree to use a double barrier contraceptive method (condom plus spermicide or diaphragm plus spermicide) during their participation in the study and 30 days after the last dose of the study drug, or the male patient or their Female couple should be surgically sterilized or the female partner should be postmenopausal.
- •10. The patient should be able to attend all study visits and follow all study procedures.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 9
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
Exclusion Criteria
- •1. Subjects that for medical reasons can not be transferred to La Paz University Hospital in Madrid.
- •2. Subjects who have received immunotherapy, including oral corticosteroids (> 15 mg / day) for more than 1 week (except for intranasal and topical preparations) or chemotherapy within 8 days prior to inclusion in the study. Inclusion of the patient is understood from the signing of informed consent.
- •3. Subjects with a known allergy to any component of the product under investigation (including penicillin and streptomycin), or who can not receive antihistamine and / or corticoid treatment.
- •4. Subjects with signs of active systemic infection at the time of inclusion in the study.
- •5. Subjects with a history or signs of malignancy, including cutaneous squamous cell carcinoma.
- •6. Subjects with circulating anti-C7 antibodies and anti-C7 antibodies deposited at the dermoepidermal junction detected in skin biopsies by indirect immunofluorescence.
- •7. Pregnant women at the time of inclusion or women of childbearing age who do not practice abstinence or use acceptable means of contraception, as determined by the investigator during the trial.
- •8. Biochemical abnormalities at the time of inclusion: albumin <2.5 g / dL, Hemoglobin <7.5 g / dL.
- •9. Subjects who have been given other investigational drugs within 90 days prior to the treatment phase.
- •10. Subjects who are not able to understand the information sheet and are unable to sign informed consent.
Investigators
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