nrelated cord blood transplantation after reduced toxicity conditioning with mesenchymal stromal cell co-infusion in patients with severe epidermolysis bullosa - CB+MSCforEB

niversitair Medisch Centrum Utrecht0 个研究点目标入组 11 人开始时间: 2013年8月12日最近更新: 2018年9月10日

适应症

试验速览

暂无简介。

•1)Diagnosed recessive dystrophic EB, severe generalized type (complete loss of functional type VII collagen by immunofluorescence microscopy and confirmed by molecular analysis).
•2)having a single matching (= 5/6) umbilical CB unit available with sufficient cell count total NC count > 3 * E7/kg
•3)Lansky / Karnofsky > 40
•4)Age <18 *( *= Karnofs364 days)
•5)Signed Informed Consent
•6)Approval by International Expert Advisory Panel (Annex 7) & UMCU kernteam-HCT
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 2
•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

•1)Creatinine clearance < 40 ml/min
•2)Cardiac dysfunction (SF < 30%) (Ejection fraction < 45%), unstable angina, or unstable cardiac arrhythmias
•3)Pulmonary function test (for those who can do: age dependent) VC, FEV1 and/ or DOC< 50%
•4)Subjects with medical history of evidence of malignancy, including cutaneous squamous cell carcinoma
•5)Patients with positive auto-antibodies to type VII collagen Determined with blood sample and salt split skin test (Immunodermatology lab UMCG)
•6)Allergy to any of the known constituents found in the investigational products (reduced toxicity chemotherapy drugs, CB and MSC)
•7)HIV seropositive infection

发起方

niversitair Medisch Centrum Utrecht