Stem cell transplantation with cord blood and mesenchymal stem cells after reduced intensity conditioning for severe forms of the blistering disease epidermolysis bullosa

Phase 1

• Conditions: The source population consists of patients referred to or within the UMC Groningen because they have diagnosed clinically and genetically severe generalized RDEB.; Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]

• Registration Number: EUCTR2012-000605-72-NL
• Lead Sponsor: niversitair Medisch Centrum Utrecht

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2013-08-12
• Last Update Posted: 10 September 2018

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 11

Inclusion Criteria

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

Exclusion Criteria

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

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified