Recessive dystrophic epidermolysis bullosa (RDEB) MedDRA version: 20.0 Level: PT Classification code 10014989 Term: Epidermolysis bullosa System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Product Name: allo-APZ2-EB
Product Code: allo-APZ2-EB
Pharmaceutical Form: Suspension for injection
Current Sponsor code: T202-3
Other descriptive name: Allogeneic skin-derived ABCB5-positive mesenchy
Sponsors
RHEACELL GmbH & Co. KG
Eligibility
Sex/Gender
All
Inclusion criteria
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 =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The aim of this clinical trial is to investigate the efficacy (by monitoring overall improvement of EB symptoms measured by epidermolysis bullosa disease activity and scarring index [EBDASI] score and instrument for scoring clinical outcome of research for epidermolysis bullosa [iscorEB], wound healing assessed by photo documentation, and patients quality of life in EB [QOLEB] assessment) and safety (by monitoring adverse events [AEs]) of the IMP allo APZ2 EB administered intravenously to patients with EB in three applications (Day 0, Day 17 ±3, Day 35 ±3).;Secondary Objective: Not applicable.;Primary end point(s): 1. Efficacy endpoint: Overall improvement of EB symptoms after 12 weeks (measured by percentage change of a patient’s EBDASI score), or last available post-baseline measurement if the Week 12 measurement is missing (last observation carried forward [LOCF]). 2. Safety endpoint: Adverse events. ;Timepoint(s) of evaluation of this end point: 1. Week 12, after first IMP application or timepoint of last observation carried forward. 2. During all patient visits except screening. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Secondary efficacy endpoints: 1. Overall improvement of EB symptoms after 12 weeks (measured by percentage change of a patient’s EBDASI score, without LOCF); 2. Overall improvement of EB symptoms after 12 weeks (measured by percentage change of patient’s iscorEB), or last available post-baseline measurement if the Week 12 measurement is missing (LOCF); 3. Overall improvement of EB symptoms after 12 weeks (measured by percentage change of patient’s iscorEB, without LOCF); 4. Overall improvement of EB symptoms at Day 17 (measured by percentage change of a patient’s EBDASI score and patient’s iscorEB); 5. Overall improvement of EB symptoms at Day 35 (measured by percentage change of a patient’s EBDASI score and patient’s iscorEB); 6. Inflammation (measured by panel of inflammation markers); 7. Pain assessment as per numerical rating scale (NRS); 8. Itch assessment as per NRS; 9. Assessment of QOLEB. Secondary safety endpoint: 10. Physical examination and vital signs until Week 12; 11. Overall survival at month 12.;Timepoint(s) of evaluation of this end point: 1. Week 12, after first IMP application; 2. Week 12, after first IMP application or timepoint of last observation carried forward; 3. Week 12, after first IMP application; 4. Day 17, after first IMP application; 5. Day 35, after first IMP application; 6. Day 0, 17 and 35 and week 12 after first IMP application; 7. Day 17 and 35 and week 12, after first IMP application; 8. Day 17 and 35 and week 12, after first IMP application; 9. Day 17 and 35 and week 12, after first IMP application; 10. Day 17, 35 and week 12 after first IMP application; 11. Month 12, after first IMP application; | — |
Countries
Austria, France, Germany, Italy, United Kingdom, United States
Contacts
Public ContactInformation Office
RHEACELL GmbH & Co. KG
Outcome results
None listed