Набор скоро начнётся NCT07436611

Rapamycin Dose-Ranging Efficacy Study in Port Wine Stains

Фаза II С лечением Port Wine Stains

Ориентир для пациента и семьи

Простыми словами

Автоматическая сводка по структурированным данным реестра. Она помогает сориентироваться, но не заменяет официальный протокол или оценку врача.

Что изучают: В протоколе указаны: Rapamycin cream, Pulsed dyed laser.
Кому может быть актуально: Состояния в реестре: Port Wine Stains. Базовые параметры: до 10 лет · Все.
Что важно проверить: Возраст, диагноз и пол — только базовые ориентиры. Предыдущее лечение, анализы и другие обязательные условия указаны ниже в критериях участия.
Где проводится: США, Испания
Следующий шаг: Сохраните исследование, покажите его лечащему врачу и уточните актуальный статус у исследовательского центра. Расходы, документы и поездка →

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Официальное название

Rapamycin Dose-Ranging Efficacy Study: A Phase II, Proof of Concept, Double-blind, Placebo-controlled, Randomized, Parallel-group, Dose-response Comparison of the Effects of Different Strengths of Rapamycin Cream Applied Topically in Subjects Diagnosed With Port Wine Stains

Обзор

The goal of this clinical trial is to learn if the use of rapamycin cream can be used as a treatment together with pulsed dyed laser in treating port wine stain birthmarks. The main question it aims to answer is: Will rapamycin cream and laser treatment show a greater improvement in appearance of port wine birthmarks, compared to treatment with placebo cream and laser? Researchers will compare two concentrations of rapamycin cream (0.6% or 1.0%) with placebo treatment to see if appearance is improved following 12 weeks of treatment. Participants will receive laser treatment of their port wine birthmark and then apply the rapamycin or placebo cream daily for 12 weeks. Patients will visit the clinic every 4 weeks for checkups and tests.

Подробное описание

This is a single-centre, double-blind, randomized withdrawal design, parallel group, placebo-controlled, dose-response clinical study investigating the efficacy and safety of two concentrations of topical rapamycin cream in the treatment of port wine stain as an adjunct to pulsed dyed laser (PDL) in paediatric patients. 30 participants will be enrolled into the study.

The study hypothesis is that topical rapamycin, as an adjunct to PDL, will show a dose-dependent increase in the blanching of port wine birthmarks, compared to PDL with placebo after 12 weeks.

Eligible participants will undergo PDL treatment of their entire port wine birthmark. Patients that respond positively to PDL within 5 days will be enrolled and randomized, before the first application of the study drug. Participants will be randomly assigned to one of three possible treatments in a 1:1:1 ratio:

* Rapamycin cream, topical (0.6% rapamycin) * Rapamycin cream, topical (1.0% rapamycin) * Placebo The stain will be divided into two parts. One part will be treated only with PDL and the other will be treated with both PDL and the assigned cream. Cream will be applied once daily for 12 weeks.

During this phase of the study, four clinical visits will occur, one every 4 weeks for 12 weeks. Assessment of the extent and severity of birthmark size and colour will be performed at randomization, at each clinical visit, and at follow-up using photography and percentage improvement ratings. Safety will be assessed by recording all adverse events experienced and testing blood rapamycin concentration at prior to treatment and at the last clinical visit.

Follow-up will be performed 28 days after the last cream application. An extra PDL session will be optionally provided at the follow-up appointment. If PDL treatment occurs at the follow-up visit, a further follow-up phone call will occur 7±2 days after the second PDL session to assess concomitant medications and adverse events.

Вмешательства

Препарат Rapamycin cream
Application of rapamycin cream to port wine birthmark
Процедура Pulsed dyed laser
Pulsed dyed laser (PDL) treatment of port wine birthmark

Первичные конечные точки

Clinic Objective improvement rating at week 12 [Срок оценки: From before laser treatment to the end of treatment after 12 weeks of cream application]

Вторичные конечные точки (3)

Subjective improvement rating (all timepoints) [Срок оценки: From before laser treatment to the end of treatment after 12 weeks of cream application]
Clinic Objective improvement rating (all timepoints) [Срок оценки: From before laser treatment to the end of treatment after 12 weeks of cream application]
Photographic Objective improvement rating (all timepoints) [Срок оценки: From before laser treatment to the end of treatment after 12 weeks of cream application]

Критерии участия

Критерии включения

Male and female patients aged ≤ 10 years on the day informed consent is obtained
Patients presenting either facial, neck, or upper trunk port-wine stains
Port wine stain size of at least 5 cm tall x 2.5 cm wide (2x1 inches)
Patients (or their legal representatives) capable of understanding the explanation of the clinical trial, and who give written informed consent for participation
Patients (or their legal representatives) able to maintain patient diaries following the instructions of the investigator or sub-investigator
Patients (or their guardians) are willing and able to follow instructions to only apply cream to part of their port-wine stain
Skin type classified as either I, II, III or IV on the Fitzpatrick scale
Patients who are indicated for PDL treatment and accept this treatment
Patients who respond to PDL with purpura or blanching

Критерии исключения

Skin type classified as either V or VI on the Fitzpatrick scale
Patients with PWS in extremities and/or acral areas
Patients unwilling or unable to carry out the treatment plan or follow-up assessment
Patients with serious skin lesions such as erosions or ulcers
Patients with known hypersensitivity to any component of the study product
Patients who have received rapamycin/sirolimus, everolimus, or temsirolimus within 3 months of enrolment
Patients who have received laser therapy or surgical therapy to treat PWS within 3 months prior to trial enrolment
Patients who have received 5 or more PDL treatments previously
Patients who participated in any other clinical trial within 3 months prior to the day of enrolment
Patients judged unsuitable for this clinical trial by the investigator or sub-investigator
Pregnant or lactating females
Sexually active females of childbearing potential not using adequate contraception and sexually active males not using adequate contraception\*
Patients with immune dysfunction or receiving any form of immunosuppression

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

Здоровые добровольцы: Нет