A Randomized, Double-blind, Vehicle-controlled Phase 2 Study to Evaluate Efficacy, Safety, and Tolerability of Topically Applied TCP-25 in Patients with Epidermolysis Bullosa

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Xinnate AB

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Skin and Connective Tissue Diseases [C17]

Trial duration

23 Dec 2025 → ongoing

Decision date (initial)

2024-12-16

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Xinnate AB

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Therapy, Pharmacokinetic

Evaluate efficacy of TCP-25 gel in promoting wound healing and improving EB symptoms in patients with DEB or JEB
Safety Objective: Evaluate safety and tolerability, including local tolerability of TCP-25 gel in patients with DEB or JEB

Secondary objectives 1

1. Secondary Pharmacokinetics Objective: Evaluate the systemic exposure to TCP-25 in patients with DEB or JEB

Conditions and MedDRA coding

Epidermolysis Bullosa

Study design 3 periods

Regulatory references

Scientific advice from competent authorities

European Medicines Agency

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 1

1. • Male or female patients with documented diagnosis of DEB or JEB, confirmed by genetic testing and/or by a skin biopsy with immunofluorescence mapping. • Patients ≥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. • The 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 1

1. • The patient has any subtype of EB other than DEB or JEB. • The 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. • Use 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. • The 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. • The patient has a history of or current malignancy over any study treatment area, eg, basal cell carcinoma or squamous cell carcinoma.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Efficacy of TCP-25 gel as compared with the vehicle in terms of open index wound area until Day 56 relative to the baseline.

Secondary endpoints 3

1. 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.
2. 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
3. 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

Investigational products

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

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Xinnate AB

Sponsor organisation

Xinnate AB

Address

Scheelevagen 2

City

Lund

Postcode

223 63

Country

Sweden

Scientific contact point

Organisation

Xinnate AB

Contact name

Matilda Hugerth

Public contact point

Organisation

Xinnate AB

Contact name

Matilda Hugerth

Third parties 8

Locations

5 EU/EEA countries · 10 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 78 files

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

Application history

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