Clinical trial NCT06594393: A Phase 2 Study of TCP-25 Gel in Patients with Epidermolysis Bullosa, STEP-study
Official title: 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
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: PHASE2
Conditions: Epidermolysis Bullosa (EB)
Lead sponsor: Xinnate AB
Sponsor class: INDUSTRY
First posted: 2024-09-19
Enrollment: 26
Sex: ALL
Minimum age (years): 4
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Intra-subject control
Primary purpose: TREATMENT

Brief summary: This is a Phase 2, double-blind, randomized, vehicle-controlled study designed to evaluate efficacy, safety, and tolerability of topically applied TCP-25 gel in patients with confirmed DEB or JEB. The study will implement intrasubject randomization, ie, pairs of matching index wounds will be randomly assigned to be treated with a local application of either TCP 25 gel or vehicle gel.

Detailed description: The study will include a Screening Period, a Treatment Period (Days 1 to 56, with study visits conducted every 2 weeks, including the baseline \[Day 1\], Day 14, Day 28, Day 42, and Day 56 visits) and a Safety Follow-up conducted via a telephone/video call at approximately 14 days after the last dose of the IMP. Patients will undergo efficacy, safety, tolerability, and PK assessments at scheduled visits. For evaluation of the primary efficacy endpoint, a trained clinician will take the images of the index wounds at all scheduled visits from baseline through Day 56 (Visit 6) using 3D digital photography and software for wound measurement.

Eligibility criteria: Inclusion Criteria:

* 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.

Exclusion Criteria:

* 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 \&gt;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 or current malignancy over the index wound, eg, basal cell carcinoma or squamous cell carcinoma.

Primary outcomes:
measure: Open wound area
timeFrame: 56 days
Secondary outcomes:
measure: Frequency, intensity, and seriousness of Adverse Events in all treated patients until end of treatment
timeFrame: 56 days
measure: Change in procedural pain using the Wong Baker FACES® Pain Rating Scale (0 min -10 max)
timeFrame: 56 days
measure: Change from baseline in clinical impression of overall treatment area
timeFrame: 56 days
measure: TCP-25 plasma concentrations
timeFrame: 56 days
measure: Number of participants with treatment-related local tolerability assessment at the area of IMP application assessed on a 4-point scale
timeFrame: 56 days
measure: Clinically significant changes from baseline in blood pressure until end of treatment
timeFrame: 56 days
measure: Number of patients with clinically significant changes from baseline in pulse rate until end of treatment
timeFrame: 56 days
measure: Number of patients with clinically significant changes from baseline in physical examination findings until end of treatment
timeFrame: 56 days
Other outcomes:
Locations:
city: Lund
country: Sweden
facility: University hospital
state: Skane
Study officials:
References:
citation: Lundgren S, Wallblom K, Fisher J, Erdmann S, Schmidtchen A, Saleh K. Study protocol for a phase 1, randomised, double-blind, placebo-controlled study to investigate the safety, tolerability and pharmacokinetics of ascending topical doses of TCP-25 applied to epidermal suction blister wounds in healthy male and female volunteers. BMJ Open. 2023 Feb 22;13(2):e064866. doi: 10.1136/bmjopen-2022-064866.