NCT04153630Unknown1 期
Safety Study and Preliminary Efficacy of Infusion Haploidentical Mesenchymal Stem Cells Derived From Bone Marrow for Treating Recessive Dystrophic Epidermolysis Bullosa
Instituto de Investigación Hospital Universitario La Paz2 个研究点 分布在 1 个国家目标入组 9 人开始时间: 2018年5月17日最近更新:
适应症
试验速览
- 阶段
- 1 期
- 入组人数
- 9
- 试验地点
- 2
- 主要终点
- Safety evaluation: Incidence of Treatment-Emergent Adverse Events as assessed by protocol.
研究概览
简要总结
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.
详细描述
The Main Objective is to evaluate the safety and therapeutic efficacy of haploidentical MSCs derived from bone marrow administered by intravenous injection for the treatment of patients with RDBS. The assessment of the symptomatic improvement of the treated patients will be made regarding the baseline situation and the response to treatment at the biochemical, histological and molecular level.
Secondary Objectives:
Describe the clinical and molecular phenotype of the mucocutaneous involvement of patients, including the characterization of the mutations responsible for the disease.
Study drug: Allogenic mesenchymal cells (haploidentical) derived from bone marrow and expanded.
Method of administration: Systemic / Intravenous Administration dose: 2-3x10e6 BM-MSC / Kg. Weekly dose for three consecutive weeks
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 12 Months 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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.
结局指标
主要结局
Safety evaluation: Incidence of Treatment-Emergent Adverse Events as assessed by protocol.
时间窗: 1 year after infusion
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)
次要结局
- Number of blisters(2 year after infusion)
- Skin surface affected(2 year after infusion)
- Non-specific general markers of systemic inflammation: Negative acute phase reactant (albumin)(2 year after infusion)
- Severity index according to "The Birmingham Epidermolysis Bullosa Severity Score" before and after treatment(2 year after infusion)
- Severity index according to "The Epidermolysis Bullosa Disease Activity and Scarring Index (EBDASI) before and after treatment(2 year after infusion)
- Variation of pain respect to baseline status: Visual Analog Scale(2 year after infusion)
- Evaluation of circulating anti-C7 antibodies(2 year after infusion)
- Cutaneous mechanical resistance(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 reactants (pre-albumin, transferrin and retinol-binding protein)(2 year after infusion)
- Non-specific general markers of systemic inflammation: Positive acute phase reactants (c-reactive protein and fibrinogen)(2 year after infusion)
- Non-specific general markers of systemic inflammation: Positive acute phase reactants (ferritin)(2 year after infusion)
- Expression of Collagen VII (C7) in skin biopsy(2 year after infusion)
- Analysis of anchoring fibrils(2 year after infusion)
- Change in general condition of the patient(2 year after infusion)
- Modification in itching perceived(2 year after infusion)
- Change in quality of life of patient: European Quality of Life-5 Dimensions-5 level (EUROQL-5D)(2 year after infusion)
研究者
研究点 (2)
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