Clinical Trial to Assess Safety and Efficacy of Autologous Cultured Epidermal Grafts Containing Epidermal Stem Cells Genetically Modified in Patients With JEB (HOLOGENE17)

• Register: ClinicalTrials.gov
• Last refreshed on: 24 August 2021
• Main ID: NCT03490331
• Date of registration: 18/03/2018
• Prospective Registration: Yes
• Primary sponsor: Holostem Terapie Avanzate s.r.l.
• Public title: Clinical Trial to Assess Safety and Efficacy of Autologous Cultured Epidermal Grafts Containing Epidermal Stem Cells Genetically Modified in Patients With JEB (HOLOGENE17) HOLOGENE17
• Scientific title: Prospective, Open-label, Uncontrolled Clinical Trial to Assess the Safety and Efficacy of Autologous Cultured Epidermal Grafts Containing Epidermal Stem Cells Genetically Modified With a Gamma-retroviral (rv) Vector Carrying COL17A1 cDNA for Restoration of Epidermis in Patients With Junctional Epidermolysis Bullosa
• Date of first enrolment: March 19, 2018
• Target sample size: 1
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/show/NCT03490331
• 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

Contacts

• Name: Johann W. Bauer, MD
• Affiliation: Paracelsus Medical University - EB House

• Name: Michele De Luca, MD/Professor
• Affiliation: Holostem Terapie Avanzate s.r.l.

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Signed and dated informed consent prior to any study-related procedures;

2. Male and female patients between 6 years old to 54 years old;

3. JEB molecular characterization by mutation analysis;

4. NC16A antibody immunofluorescence or positive staining in Western Blot analysis with
polyclonal antibody produced against a synthetic peptide corresponding to amino acids
131-145 of human COL17A1;

5. Presence of chronic (persistent or recurrent for more than 3 months) large wounds (>10
cm2) and/or persistent or recurrent erosions;

6. A cooperative attitude to follow up the study procedures (Caregivers in case of
minors).

Exclusion Criteria:

1. Known or suspected intolerances against anaesthesia;

2. Bad general condition (ECOG index >1);

3. Unresectable metastasizing Squamous Cell Carcinomas (SCCs);

4. Antibodies to type XVII collagen associated antigens demonstrated on indirect
immunofluorescence;

5. Clinical and/or laboratory signs of acute systemic infections at the time of
screening. Patient can be re-screened after appropriate treatment;

6. Severe systemic diseases (i.e. uncompensated diabetes mellitus);

7. Female subjects: pregnant or lactating women and all women physiologically capable of
becoming pregnant (i.e. women of childbearing potential) UNLESS they are willing to
use one or more reliable methods of contraception with a Pearl index =1. Reliable
contraception should be maintained throughout the study.

8. Allergy, sensitivity or intolerance to drugs, excipients or other material (as per
Investigator's brochure):

- Transport medium (Dulbecco's Modified Eagles Medium supplemented with
L-glutamine)

- Fibrin support

- Povidone iodine

9. Contraindications to the local or systemic antibiotics and/ or corticosteroids
foreseen by the protocol;

10. Contraindications to undergo extensive surgical procedures;

11. Clinically significant or unstable concurrent disease or other clinical
contraindications to stem cell transplantation based upon investigator's judgment or
other concomitant medical conditions affecting grafting procedure;

12. Patients (or parents in case of paediatric subject) unlikely to comply with the study
protocol or unable to understand the nature and scope of the study or the possible
benefits or unwanted effects of the study procedures and treatments;

13. Participation in another clinical trial where investigational drug was received less
than 6 months prior to screening Visit.



Age minimum: 6 Years
Age maximum: 54 Years
Gender: All

Health Condition(s) or Problem(s) studied

Junctional Epidermolysis Bullosa

Intervention(s)

Other: Transplantation surgery of genetically corrected cultured epidermal autograft (ATMP)

Primary Outcome(s)

Safety events (ADRs and SAEs) related to the study treatment (tolerability) [Time Frame: 3 months after treatment]

Safety events (ADRs and SAEs) related to the study treatment (tolerability) [Time Frame: 12 months after treatment]

Secondary Outcome(s)

Skin functionality in the short term (Treatment efficacy) [Time Frame: 3 months after transplantation]

Skin stability in the short term (Treatment efficacy) [Time Frame: 3 months after transplantation]

Skin functionality in the long term (Treatment efficacy) [Time Frame: 12 months after transplantation]

Skin stability in the long term (Treatment efficacy) [Time Frame: 12 months after transplantation]

Secondary ID(s)

HTA-HG17-01

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Paracelsus Medical University