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Trial record 4 of 24 for:    epidermolysis bullosa | Recruiting, Not yet recruiting, Available Studies

Allogeneic ABCB5-positive Stem Cells for Treatment of Epidermolysis Bullosa

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ClinicalTrials.gov Identifier: NCT03529877
Recruitment Status : Not yet recruiting
First Posted : May 18, 2018
Last Update Posted : May 18, 2018
Sponsor:
Collaborators:
FGK Clinical Research GmbH
Granzer Regulatory Consulting & Services
Ticeba GmbH
Information provided by (Responsible Party):
RHEACELL GmbH & Co. KG

Brief Summary:
The aim of this clinical trial is to investigate the efficacy (by monitoring overall improvement of EB symptoms) and safety (by monitoring adverse events) of three doses of allo-APZ2-EB administered intravenously to patients with recessive dystrophic epidermolysis bullosa (RDEB).

Condition or disease Intervention/treatment Phase
Recessive Dystrophic Epidermolysis Bullosa Biological: allo-APZ2-EB Phase 1 Phase 2

Detailed Description:

This is an interventional, single arm, non-randomized, open label, phase I/IIa clinical trial to investigate the efficacy and safety of the IMP allo-APZ2-EB in patients with RDEB.

Patients will undergo treatment with the IMP (three repeated intravenous applications) and will be followed up for efficacy for 12 weeks. To assess long-term safety of allo-APZ2-EB one follow-up visit at Month 12 post IMP applications is included.

Determination of the EB linked symptoms and quality of life will be assessed by using the EBDASI score, the iscorEB, the change in pain and itch perception, and patient's quality of life in EB. The wound healing process will be documented by photography.


Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 18 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official 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)
Estimated Study Start Date : October 2018
Estimated Primary Completion Date : October 2019
Estimated Study Completion Date : October 2020


Arm Intervention/treatment
Experimental: allo-APZ2-EB
intravenous infusion, three doses of allo-APZ2-EB (2 x 10^6 cells/kg)
Biological: allo-APZ2-EB
intravenous infusion of allo-APZ2-EB
Other Name: allogeneic ABCB5-positive mesenchymal stem cells




Primary Outcome Measures :
  1. Overall improvement of EB symptoms after 12 weeks (measured by percentage change of a patient's (EBDASI score) [ Time Frame: Week 12 post baseline, or last available post-baseline measurement if the Week 12 measurement is missing (last observation carried forward [LOCF]) ]
    EBDASI: epidermolysis bullosa disease activity and scarring index; measured in percentage change to baseline score

  2. Assessment of adverse event (AE) occurrenc [ Time Frame: Up to 12 months ]
    All AEs occurring during the clinical trial will be registered, documented and evaluated.


Secondary Outcome Measures :
  1. 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) ]
    EBDASI: epidermolysis bullosa disease activity and scarring index; measured in percentage change to baseline score

  2. Overall improvement of EB symptoms after 12 weeks (measured by percentage change of patient's iscorEB) [ Time Frame: Week 12 post baseline, or last available post-baseline measurement if the Week 12 measurement is missing ]
    iscorEB: instrument for scoring clinical outcome of research for epidermolysis bullosa; measured in percentage change to baseline score

  3. 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) ]
    iscorEB: instrument for scoring clinical outcome of research for epidermolysis bullosa; measured in percentage change to baseline score

  4. 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 ]
    EBDASI: epidermolysis bullosa disease activity and scarring index; measured in percentage change to baseline score

  5. 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 ]
    iscorEB: instrument for scoring clinical outcome of research for epidermolysis bullosa; measured in percentage change to baseline score

  6. 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 ]
    EBDASI: epidermolysis bullosa disease activity and scarring index; measured in percentage change to baseline score

  7. 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 ]
    iscorEB: instrument for scoring clinical outcome of research for epidermolysis bullosa; measured in percentage change to baseline score

  8. Inflammation (measured by panel of inflammation markers) [ Time Frame: between baseline and day 17, day 35 and week 12 post baseline ]
  9. Pain assessment as per NRS [ Time Frame: between baseline and day 17, day 35 and week 12 post baseline ]
    Pain assessment as per numerical rating scale (NRS) will be evaluated.

  10. Itch assessment as per NRS [ Time Frame: between baseline and day 17, day 35 and week 12 post baseline ]
    Itch assessment as per numerical rating scale (NRS) will be evaluated.

  11. Differences in patient's quality of life in EB [ Time Frame: between baseline and day 17, day 35 and week 12 post baseline ]
    Assessment of quality of life data using an EB-specific quality of life questionnaire

  12. Physical examination [ Time Frame: At Screening, baseline, day 17, day 35 and week 12 ]
    A full physical examination will be performed and abnormal physical examination results will be evaluated and reported as AEs.

  13. Vital signs: Body temperature [ Time Frame: At Screening, baseline, day 17, day 35 and week 12 ]
    Body temperature will be evaluated at Screening, baseline, day 17, day 35 and week 12

  14. Vital signs: Blood pressure [ Time Frame: At Screening, baseline, day 17, day 35 and week 12 ]
    Blood pressure will be evaluated at Screening, baseline, day 17, day 35 and week 12

  15. Vital signs: Heart rate [ Time Frame: At Screening, baseline, day 17, day 35 and week 12 ]
    Heart rate will be evaluated at Screening, baseline, day 17, day 35 and week 12

  16. Overall survival at month 12 [ Time Frame: month 12 post baseline ]


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Ages Eligible for Study:   12 Months to 55 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. a) Male or female patients aged between ≥18 years and ≤55 years; b) After positive safety assessment of 3 treated patients (≥18 years and ≤55 years): male or female patients aged between ≥12 months and ≤55 years;
  2. Diagnosed with RDEB;
  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.

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. Clinically significant or unstable concurrent disease or other clinical contraindications (based upon investigator's judgment);
  6. Patient/legal representative anticipated to be unwilling or unable to comply with the requirements of the protocol;
  7. Pregnant or lactating women;
  8. Current or previous (within 30 days of enrollment) treatment with another IMP, or participation and/or under follow-up in another clinical trial;
  9. Previous participation in this clinical trial (except for screening failures due to an exclusion criterion);
  10. Known abuse of alcohol, drugs, or medicinal products;
  11. Employees of the sponsor, or employees or relatives of the investigator.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03529877


Contacts
Contact: Christoph Ganss, Dr. +49 6221 71833 ext 0 office@rheacell.com
Contact: Kathrin Dieter +49 6221 71833 ext 0 office@rheacell.com

Locations
United States, Minnesota
University of Minnesota, Masonic Cancer Center and Medical Center
Minneapolis, Minnesota, United States, 55455
Germany
Department of Dermatology, Medical Center-University of Freiburg
Freiburg, Germany, 79104
Sponsors and Collaborators
RHEACELL GmbH & Co. KG
FGK Clinical Research GmbH
Granzer Regulatory Consulting & Services
Ticeba GmbH

Responsible Party: RHEACELL GmbH & Co. KG
ClinicalTrials.gov Identifier: NCT03529877     History of Changes
Other Study ID Numbers: allo-APZ2-EB-II-01
First Posted: May 18, 2018    Key Record Dates
Last Update Posted: May 18, 2018
Last Verified: May 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by RHEACELL GmbH & Co. KG:
Epidermolysis Bullosa
Epidermolysis Bullosa Dystrophica
Skin Abnormalities
Congenital Abnormalities
Skin Diseases, Genetic
Somatic Cell Therapy
Mesenchymal Stem Cells
ABCB5
Allogeneic

Additional relevant MeSH terms:
Epidermolysis Bullosa
Epidermolysis Bullosa Dystrophica
Skin Abnormalities
Congenital Abnormalities
Skin Diseases, Genetic
Genetic Diseases, Inborn
Skin Diseases
Skin Diseases, Vesiculobullous
Collagen Diseases
Connective Tissue Diseases