Study to investigate the efficacy and safety of allo-APZ2-EB on wound healing of epidermolysis bullosa (EB)

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 16

Inclusion Criteria

1. Male or female patients aged between =12 months and =55 years,
Staggered design for patient enrollment:
1.) at least 3 adult patients =18 to =55 years (safety assessment 2 weeks after last treatment of third patient),
2.) at least 3 patients =12 to <18 years (safety assessment 2 weeks after first treatment of third patient),
3.) at least 3 patients =5 to <12 years (safety assessment 2 weeks after first treatment of third patient) and
4.) at least 3 patients =12 months to <5 years (safety assessment 2 weeks after first treatment of third patient);
2. Diagnosed with RDEB (combined diagnoses by genotype assessment [mutation analysis] and correlating phenotype assessment [wound assessment]), patients must have a negative immunofluorescence test result on salt-split skin against proteins of the basement membrane at Visit 1 (existing test results will be accepted);
3. Patient is eligible to participate in this clinical trial based on general health condition at the investigator’s discretion;
4. Patient/legal representative understands the nature of the procedure and are providing written informed consent prior to any clinical trial procedure;
5. Women of childbearing potential must have a negative urine pregnancy test at Visit 1;
6. Women of childbearing potential and their partner must be willing to use highly effective contraceptive methods during the course of the clinical trial.
Are the trial subjects under 18? yes
Number of subjects for this age range: 9
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 7
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Tumor diseases or history of tumor disease;
2. Positive test result for human immunodeficiency virus 1 and/or 2 at Screening;
3. Any known allergies to components of the IMP;
4. Evidence of any other medical conditions (such as psychiatric illness or active infection) based on physical examination, or laboratory findings that may interfere with the planned treatment, affect the patient’s compliance, or place the patient at high risk of complications related to the treatment; at investigators discretion;
5. Inadequate pulmonary function with evidence of chronic obstructive or severe restrictive pulmonary disease;
6. Inadequate cardiac function with evidence of uncontrolled high blood pressure, congestive heart failure, angina pectoris, acute myocardial infarction within 6 months prior to treatment;
7. History of prior thrombosis or patients at risk for thrombosis
8. Clinically significant or unstable concurrent disease or other clinical contraindications to IMP application (based upon investigator’s judgment);
9. Patient/legal representative anticipated to be unwilling or unable to comply with the requirements of the protocol;
10. Pregnant or lactating women;
11. Current or previous (within 30 days of enrollment) treatment with another IMP, or participation and/or under follow-up in another clinical trial;
12. Previous participation in this clinical trial (except for screening failures due to an exclusion criterion);
13. Known abuse of alcohol, drugs, or medicinal products;
14. Employees of the sponsor, or employees or relatives of the investigator.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

Updated 12/4/2018

Study to investigate the efficacy and safety of allo-APZ2-EB on wound healing of epidermolysis bullosa (EB)

Recruitment & Eligibility

Study & Design

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