Sirolimus (rapamycin) for Port-wine stain

Inclusion criteria

• {"criterion_text":"- Male and female patients aged ≥ 3 months and ≤ 18 years on the day informed consent is obtained\n- Patients presenting either facial, neck, trunk or upper arm port-wine stains\n- Port wine stain size of at least 36 cm2 in size (ideally at least 6 cm x 6 cm)\n- Patients (or their legal representatives) capable of understanding the explanation of the clinical trial, and who give written informed consent for participation\n- Patients (or their legal representatives) able to maintain patient diaries following the instructions of the investigator or sub-investigator\n- Patients are willing and able to follow instructions to only apply cream to part of their port-wine stain\n- Skin type classified as either I, II, III or IV on the Fitzpatrick scale"}

Exclusion criteria

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

Primary endpoints

• {"endpoint_text":"- The primary efficacy endpoint is the change in extent and intensity of PWS colour from baseline to week 12 (V3), or at last visit if early withdrawal/discontinuation occurs, measured using a colorimetry system through standardized clinical photographs.","definition_or_measurement_approach":"Change in extent and intensity of PWS colour from baseline to week 12 (V3) measured using a colorimetry system through standardized clinical photographs (or at last visit if early withdrawal/discontinuation occurs)."}

Secondary endpoints

• {"endpoint_text":"- Change in extent and intensity of PWS colour from baseline at weeks 0, 4, 8 and follow-up/last assessment, measured using a colorimetry system through standardized clinical photographs.","definition_or_measurement_approach":"Measured using a colorimetry system through standardized clinical photographs at weeks 0, 4, 8 and follow-up/last assessment."}
• {"endpoint_text":"- Subjective improvement rating of PWS from baseline after 0, 4, 8 and 12 weeks of treatment","definition_or_measurement_approach":"Subjective improvement rating assessed at weeks 0, 4, 8 and 12 (method as per trial assessments; timing specified)."}
• {"endpoint_text":"- Clinic Objective improvement rating in PWS from baseline after 0, 4, 8 and 12 weeks of treatment","definition_or_measurement_approach":"Clinic objective improvement ratings assessed at weeks 0, 4, 8 and 12."}
• {"endpoint_text":"- Photographic Objective improvement rating in PWS from baseline after 0, 4, 8 and 12 weeks of treatment","definition_or_measurement_approach":"Photographic objective improvement ratings assessed via standardized clinical photographs at weeks 0, 4, 8 and 12."}

Spain

Earliest CTIS Part Ii Submission Date

24-01-2025

Latest Decision Or Authorization Date

27-01-2025

Processing Time Days

3

Number Of Sites

1

Number Of Participants

30

Primary sponsor

Full Name

Aft Pharmaceuticals Limited

Organisation Type

Pharmaceutical company

Country Of Registered Address

New Zealand