Safety and efficacy of allogenic adipose tissue-derived mesenchymal stromal cells in patients with epidermolysis bullosa: Clinical trial phase I/II

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Masarykova Univerzita

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Trial duration

12 Oct 2022 → 22 Jul 2025

Decision date (initial)

2024-11-11

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Masarykova univerzita · DEBRA ČR, z. ú.

External identifiers

EU CT number

2024-516614-37-00

EudraCT number

2020-002936-55

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Efficacy

The primary objective of the study is to assess the safety of human allogeneic mesenchymal stromal cells administered intradermally.

Secondary objectives 1

1. The secondary objective of the study is to obtain pilot data on the efficacy of the treatment

Conditions and MedDRA coding

Epidermolysis bullosa congenita

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

1. Age > 2 years (incl.)
2. Clinical diagnosis of hereditary EB supported by histological diagnosis (electron microscopy, immunofluorescence antigen mapping) and/or DNA analysis
3. The extent of the disease is at least 10% of the body surface
4. Participants in a clinical trial of childbearing potential must agree to the use of prescribed contraceptive methods for the duration of the clinical study and for at least 6 months following the last dose of the study medication: a) Women - Proper use of a highly reliable method of contraception, ie combined hormonal contraception (in oral, vaginal or transdermal dosage form), gestagen hormonal contraception associated with ovulation inhibition (in oral or injectable dosage form), non-hormonal intrauterine device or intrauterine device releasing hormones, or the presence of bilateral tubal occlusion, a partner's vasectomy, or adherence to sexual abstinence as part of the patient's normal lifestyle. b) Men - Adherence to sexual abstinence or the use of an adequate contraceptive method (ie condom) in case of sexual intercourse.
5. Willingness and ability to adhere to restrictions in the care of study lesions and to adhere to the schedule of visits and examinations during the course of the study.

Exclusion criteria 15

1. Systemic infection or sepsis 7 days before MSC administration
2. Treatment with blood transfusion (erythrocyte concentrate or whole blood transfusion) 7 days before MSC administration
3. Clinical signs of infection in the study lesions 7 days before MSC administration
4. Inability to tolerate repeated skin injections (especially in pediatric patients)
5. History of basal cell or squamous cell carcinoma of the skin in the last 5 years
6. History of other malignancies of any type at any time during life
7. Severe lung disease that requires home oxygenation
8. Severe kidney or liver disease
9. Dilatation cardiomyopathy
10. Life expectancy less than 90 days
11. Severe immune response to allogeneic human cells
12. Other severe somatic or psychiatric illnesses that are not adequately controlled and, in the opinion of the investigator, would not allow the study protocol to be followed
13. Previous administration of a medicinal product in a clinical trial in the 30 days or 5 elimination half-lives (whichever is longer) before the enrolment to this trial (applies to clinical investigations of medical devices, as well)
14. Pregnancy or breastfeeding
15. Allergy/hypersensitivity to any component of Hypotermosol solution

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Incidence of adverse events of special interest (AESI): severe local reaction at the injection site other than blistering, severe systemic allergic reaction at any time after MSC administration, severe systemic or local infectious complications at any time after MSC administration (combined primary safety endpoint)

Secondary endpoints 4

1. Incidence, type, seriousness, intensity of AEs and their relation to study medication
2. Percent change in the non-healed area of the area marked in the study active and control lesions from baseline
3. Estimation of the total area of lesions on the patient's body (% of body surface area)
4. Patient-reported outcomes (itching, ease of wound dressing change)

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Masarykova Univerzita

Sponsor organisation

Masarykova Univerzita

Address

Zerotinovo Namesti 617/9, Brno-Mesto Brno-Mesto

City

Brno

Postcode

602 00

Country

Czechia

Scientific contact point

Organisation

Masarykova Univerzita

Contact name

Regina Demlová

Public contact point

Organisation

Masarykova Univerzita

Contact name

Regina Demlová

Locations

1 EU/EEA country · 2 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 14 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

3 submissions — initial application plus substantial / non-substantial modifications