Allogeneic ABCB5-positive Stem Cells for Treatment of Epidermolysis Bullosa

• Register: ClinicalTrials.gov
• Last refreshed on: 17 October 2022
• Main ID: NCT03529877
• Date of registration: 24/04/2018
• Prospective Registration: Yes
• Primary sponsor: RHEACELL GmbH & Co. KG
• Public title: Allogeneic ABCB5-positive Stem Cells for Treatment of Epidermolysis Bullosa
• Scientific title: An Interventional, Multicenter, Single Arm, Phase I/IIa Clinical Trial to Investigate the Efficacy and Safety of Allo-APZ2-EB on Epidermolysis Bullosa (EB)
• Date of first enrolment: February 16, 2019
• Target sample size: 16
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03529877
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1/Phase 2

Countries of recruitment

Austria, France, Germany, Italy, United Kingdom, United States

Contacts

• Name: Jakub Tolar, MD, PhD
• Affiliation: University of Minnesota, Masonic Cancer Center and Medical Center

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Male or female patients aged between 0 and =55 years;

Staggered design for patient enrollment:

1. at least 3 adult patients (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;

5. patients 0 to <12 months (only in the UK);

2. Diagnosed with RDEB (combined diagnosis 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;

US only:

Patient is eligible to participate in this clinical trial based on general health condition
assessed by specific lab values (Hematology: Absolute neutrophil count >1000/mm3 and
platelet count >150,000/mcL; Coagulation: PT and PTT <2x the upper limit of normal for age;
Hepatic: AST and ALT <2x the upper limit of normal for age; Renal: Creatinine <2x the upper
limit of normal for age; Pulmonary: Oxygen saturation >92% on room air and without
supplemental oxygen requirement);

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.

Exclusion Criteria:

1. Tumor diseases or history of tumor disease;

2. Known positive result for human immunodeficiency virus 1 and/or 2;

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. History of prior thrombosis or patients at risk for thrombosis;

6. Clinically significant or unstable concurrent disease or other clinical
contraindications (based upon investigator's judgment);

7. Patient/legal representative anticipated to be unwilling or unable to comply with the
requirements of the protocol;

8. Pregnant or lactating women;

9. Current or previous (within 30 days of enrollment) treatment with another IMP, or
participation and/or under follow-up in another clinical trial;

10. Previous participation in this clinical trial (except for screening failures due to an
exclusion criterion);

11. Known abuse of alcohol, drugs, or medicinal products;

12. Employees of the sponsor, or employees or relatives of the investigator.



Age minimum: 0 Years
Age maximum: 55 Years
Gender: All

Health Condition(s) or Problem(s) studied

Recessive Dystrophic Epidermolysis Bullosa

Intervention(s)

Biological: allo-APZ2-EB

Primary Outcome(s)

Assessment of adverse event (AE) occurrence [Time Frame: Up to 24 months]

Overall improvement of EB symptoms after 12 weeks (measured by percentage change of a patient's EBDASI score), score), or last available post-baseline measurement if the Week 12 measurement is missing [Time Frame: Week 12 post baseline, or last available post-baseline measurement if the Week 12 measurement is missing (last observation carried forward [LOCF])]

Secondary Outcome(s)

Differences in patient's quality of life in EB [Time Frame: between baseline and day 17, day 35 and week 12 post baseline]

Itch assessment as per NRS [Time Frame: between baseline and day 17, day 35 and week 12 post baseline]

Vital signs: Blood pressure until Week 12; [Time Frame: At Screening, baseline, day 17, day 35 and week 12]

Overall improvement of EB symptoms after 12 weeks (measured by percentage change of patient's iscorEB) [Time Frame: between baseline and week 12 post baseline (without LOCF)]

Overall improvement of EB symptoms at Day 17 (measured by percentage change of a patient's EBDASI score) [Time Frame: between baseline and day 17 post baseline]

Overall improvement of EB symptoms at Day 17 (measured by percentage change of a patient's iscorEB) [Time Frame: between baseline and day 17 post baseline]

Overall survival at month 24 [Time Frame: month 24 post baseline]

Pain assessment as per NRS [Time Frame: between baseline and day 17, day 35 and week 12 post baseline]

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 [Time Frame: Week 12 post baseline, or last available post-baseline measurement if the Week 12 measurement is missing (LOCF);]

Vital signs: Heart rate until Week 12; [Time Frame: At Screening, baseline, day 17, day 35 and week 12]

Overall improvement of EB symptoms after 12 weeks (measured by percentage change of a patient's EBDASI score) [Time Frame: between baseline and week 12 post baseline (without LOCF)]

Inflammation (measured by panel of inflammation markers) [Time Frame: between baseline and day 17, day 35 and week 12 post baseline]

Vital signs: Body temperature until Week 12; [Time Frame: At Screening, baseline, day 17, day 35 and week 12]

Overall improvement of EB symptoms at Day 35 (measured by percentage change of a patient's EBDASI score) [Time Frame: between baseline and day 35 post baseline]

Overall improvement of EB symptoms at Day 35 (measured by percentage change of a patient's iscorEB) [Time Frame: between baseline and day 35 post baseline]

Physical examination until Week 12; [Time Frame: At Screening, baseline, day 17, day 35 and week 12]

Secondary ID(s)

allo-APZ2-EB-II-01

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

FGK Clinical Research GmbH

Granzer Regulatory Consulting & Services

Ticeba GmbH