H-GLY-LYS-TYR-GLY-PHE-TYR-THR-HIS-VAL-PHE-ARG-LEU-LYS-LYS-TRP-ILE-GLN-LYS-VAL-ILE-ASP-GLN-PHE-GLY-GLU-OH for Epidermolysis bullosa|Dystrophic epidermolysis bullosa (DEB)|Junctional epidermolysis bullosa (JEB)

Inclusion criteria

• {"criterion_text":"- •\tMale or female patients with documented diagnosis of DEB or JEB, confirmed by genetic testing and/or by a skin biopsy with immunofluorescence mapping.\n- •\tPatients ≥4 years old. Note: Initially, patients ≥12 years will be enrolled. Enrollment will be open to 4 to 11 year old pediatric patients (both inclusive), after a DMC reviews and provides a positive opinion regarding the safety and tolerability of the IMP in at least 3 patients 12 to 18 years old who have completed at least 4 weeks of IMP use.\n- •\tThe patient has the presence of a well-matched index wound pair which will be included in two respective treatment areas, with a minimum distance of at least 5 cm (2 inches) between the treatment areas."}

Exclusion criteria

• {"criterion_text":"- •\tThe patient has any subtype of EB other than DEB or JEB.\n- •\tThe patient is currently being treated or planned to be treated with systemic antibiotics. Note: Use of preventive and/or anti-inflammatory antibiotic treatment, including doxycycline, on an established treatment regimen (stable dose for ≥6 weeks before the Baseline Visit) is permitted. Use of topical antibiotics on the index wounds within 7 days before the Baseline Visit is prohibited.\n- •\tUse of systemic corticosteroids >0.2 mg/kg prednisone dose equivalent per day within 30 days or use of topical corticosteroids on index wounds within 7 days before the Screening Visit. Note: Corticosteroids for inhalation, ophthalmic, or intranasal use are permitted.\n- •\tThe patient has a history of any of the following treatments: a. Local topical Filsuvez on the study treatment areas within 1 month before the Screening Visit, b. Local topical gene therapy with Vyjuvek on the study treatment areas within 3 months before the Screening Visit, c. Systemic mesenchymal stem cell treatment, including Ebesenar, within 6 months before the Screening Visit, or d. Cell-gene (graft) therapy, Zevaskyn, for the treatment of EB affecting the treatment areas any time before the Screening Visit.\n- • The patient has a history of or current malignancy over any study treatment area, eg, basal cell carcinoma or squamous cell carcinoma."}

Primary endpoints

• {"endpoint_text":"- Efficacy of TCP-25 gel as compared with the vehicle in terms of open index wound area until Day 56 relative to the baseline.","definition_or_measurement_approach":"Change in open index wound area until Day 56 relative to baseline; described in translations as area under the curve (AUC) up to Day 56 relative to baseline (reported AUC / rAUC of open index wound area)."}

Secondary endpoints

• {"endpoint_text":"- Key secondary point: Efficacy of TCP-25 gel as compared with the vehicle in terms of proportions of index wounds achieving complete closure of index wound (defined as 100% re-epithelialization without drainage), within Day 56.","definition_or_measurement_approach":"Proportion of index wounds achieving complete closure by Day 56; complete closure defined as 100% re-epithelialization without drainage."}
• {"endpoint_text":"- Change in procedural pain using the Wong Baker FACES® Pain Rating Scale Change from baseline in clinical impression of the index wound using the CGI-S scale and the CGI-C score","definition_or_measurement_approach":"Procedural pain measured by Wong-Baker FACES® Pain Rating Scale. Clinical Global Impression scales (CGI-S and CGI-C) used to assess clinical impression change from baseline for index wound."}
• {"endpoint_text":"- TCP-25 plasma concentrations Frequency, intensity, and seriousness of Adverse Events in all treated patients Local tolerability assessment at the area of IMP application Clinically significant changes from baseline in vital signs (systolic and diastolic blood pressure and pulse rate) and physical examination","definition_or_measurement_approach":"Measurement of TCP-25 plasma concentrations (PK). Safety endpoints include frequency, intensity and seriousness of AEs in all treated patients; local tolerability assessment at application site; clinically significant changes in vital signs and physical examination compared with baseline."}

Methods

• Web and social media advertisements (country-specific adverts present: e.g., 'advertisement for web page or social media' documents for SWE, FRA, GRC, SPA, ITA).
• Patient-facing presentations and study introduction materials (country-specific presentation documents listed).
• GP/primary care communications (e.g., GP-letter_IT for Italy).
• Study-specific patient adverts on web pages and social media targeted to patients with epidermolysis bullosa and HCPs in each participating country.
• Local site recruitment via dermatology clinics and hospital departments listed at each site.

Sweden

Earliest CTIS Part Ii Submission Date

18-11-2024

Latest Decision Or Authorization Date

29-10-2025

Processing Time Days

345

Number Of Sites

2

Number Of Participants

5

France

Earliest CTIS Part Ii Submission Date

18-10-2024

Latest Decision Or Authorization Date

29-10-2025

Processing Time Days

376

Number Of Sites

1

Number Of Participants

6

Greece

Earliest CTIS Part Ii Submission Date

13-09-2024

Latest Decision Or Authorization Date

30-10-2025

Processing Time Days

412

Number Of Sites

2

Number Of Participants

5

Spain

Earliest CTIS Part Ii Submission Date

18-10-2024

Latest Decision Or Authorization Date

03-11-2025

Processing Time Days

381

Number Of Sites

3

Number Of Participants

5

Italy

Earliest CTIS Part Ii Submission Date

13-11-2024

Latest Decision Or Authorization Date

11-11-2025

Processing Time Days

363

Number Of Sites

2

Number Of Participants

5

Primary sponsor

Full Name

Xinnate AB

Organisation Type

Pharmaceutical company

Country Of Registered Address

Sweden

Contract research organisations

Name

PPD Global Limited

Responsibilities

Multiple sponsor duties across study operations (codes: [1,2,5,6,7,8,9,10,11,12,13,14] as listed in CTIS third-party entries)

Name

Klifo A/S

Responsibilities

IMP release and clinical supplies for sites (sponsorDuties codes: [14,15]; noted value: 'IMP release, clinical supplies for sites')

Third parties

• {"country":"Sweden","full_name":"Lund University","duties_or_roles":"sponsorDuties codes: [4]","organisation_type":"Educational Institution"}
• {"country":"Greece","full_name":"PPD Global Ltd.","duties_or_roles":"sponsorDuties codes: [1,2]","organisation_type":"Pharmaceutical company"}
• {"country":"Austria","full_name":"Pharm-Analyt Labor Ges.m.b.H.","duties_or_roles":"sponsorDuties codes: [4]","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"United States","full_name":"Veeva Systems Inc.","duties_or_roles":"sponsorDuties codes: [3,7]","organisation_type":"Non-Pharmaceutical company"}
• {"country":"Denmark","full_name":"Klifo A/S","duties_or_roles":"sponsorDuties codes: [14,15]; value: 'IMP release, clinical supplies for sites'","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Scout Clinical","duties_or_roles":"sponsorDuties codes: [15]; value: 'Travel management and travel reimbursement'","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"United States","full_name":"Canfield Scientific Inc.","duties_or_roles":"sponsorDuties codes: [15]; value: 'Imaging management (supplies shipment, imaging monitoring ; 2D and 3D imaging analysis)'","organisation_type":"Pharmaceutical company"}
• {"country":"United Kingdom","full_name":"PPD Global Limited","duties_or_roles":"sponsorDuties codes: [1,2,5,6,7,8,9,10,11,12,13,14] (multiple operational and study management roles as listed)","organisation_type":"Pharmaceutical company"}