A Phase 2 Study of TCP-25 Gel in Patients With Epidermolysis Bullosa, STEP-study

Официальное название

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

Обзор

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, a pair of matching index wounds will be randomly assigned to be treated with a local application of either TCP 25 gel or vehicle gel.

Подробное описание

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.

Вмешательства

• Препарат TCP-25 gel
  Topical gel
• Препарат Vehicle (placebo)
  Placebo gel

Первичные конечные точки

• Open wound area [Срок оценки: 56 days]

Вторичные конечные точки (8)

• Frequency, intensity, and seriousness of Adverse Events in all treated patients until end of treatment [Срок оценки: 56 days]
• Change in procedural pain using the Wong Baker FACES® Pain Rating Scale (0 min -10 max) [Срок оценки: 56 days]
• Change from baseline in clinical impression of overall treatment area [Срок оценки: 56 days]
• TCP-25 plasma concentrations [Срок оценки: 56 days]
• Number of participants with treatment-related local tolerability assessment at the area of IMP application assessed on a 4-point scale [Срок оценки: 56 days]
• Clinically significant changes from baseline in blood pressure until end of treatment [Срок оценки: 56 days]
• Number of patients with clinically significant changes from baseline in pulse rate until end of treatment [Срок оценки: 56 days]
• Number of patients with clinically significant changes from baseline in physical examination findings until end of treatment [Срок оценки: 56 days]

Критерии участия

Критерии включения

• 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.

Критерии исключения

• 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 or current malignancy over the index wound, eg, basal cell carcinoma or squamous cell carcinoma.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Рандомизированное

Модель

Параллельные группы

Маскирование

Четверное слепое

Основная цель

Лечение

Центры проведения

Испания · 3 центра

• San Joan de Deu, SJD Children's hospital Barcelona — Barcelona
• Hospital Clinic de Barcelona — Barcelona
• Hospital Univ La Paz — Madrid

Греция · 2 центра

• Andreas Syngros Hospital of Veneral & Dermatological Diseases — Athens
• Hospital Of Skin And Venereal Diseases of Thessaloniki — Thessaloniki

Швеция · 2 центра

• University hospital — Lund
• Akademiska sjukhuset — Uppsala

Франция · 1 центр

• Hopital St Louis — Paris

Италия · 1 центр

• IDI Istituto Dermopatico dell'Immacolata IRCCS — Rome

Публикации

• 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 PMID 36813496

Идентификаторы

NCT: NCT06594393 · TCP25-002

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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