Dose-Ranging Efficacy Study of rapamycin cream applied topically in subjects diagnosed with port wine stains

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Aft Pharmaceuticals Limited

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years

Gender

Male and Female

Therapeutic area

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

Trial duration

2 Jan 2024 → ongoing

Decision date (initial)

2025-01-27

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

External identifiers

EU CT number

2025-520821-19-00

EudraCT number

2021-003604-40

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy, Dose response, Safety, Efficacy

To probe that the two strengths (0.5% and 1.0%) of Rapamycin cream, topical will improve the appearance of PWS following PDL therapy compared to placebo following PDL therapy over a 12 week period. It is also expected that both strengths will be well tolerated.

Secondary objectives 4

1. 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.
2. Subjective improvement rating of PWS from baseline after 0, 4, 8 and 12 weeks of treatment
3. Clinic Objective improvement rating in PWS from baseline after 0, 4, 8 and 12 weeks of treatment
4. Photographic Objective improvement rating in PWS from baseline after 0, 4, 8 and 12 weeks of treatment

Conditions and MedDRA coding

Subjects diagnosed with port wine stains

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 7

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

Exclusion criteria 12

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

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. 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.

Secondary endpoints 4

1. 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.
2. Subjective improvement rating of PWS from baseline after 0, 4, 8 and 12 weeks of treatment
3. Clinic Objective improvement rating in PWS from baseline after 0, 4, 8 and 12 weeks of treatment
4. Photographic Objective improvement rating in PWS from baseline after 0, 4, 8 and 12 weeks of treatment

Investigational products

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

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Aft Pharmaceuticals Limited

Sponsor organisation

Aft Pharmaceuticals Limited

Address

Level 1, Nielsen Centre, 129 Hurstmere Road, Takapuna Nielsen Centre 129 Hurstmere Road

City

Auckland

Postcode

0622

Country

New Zealand

Scientific contact point

Organisation

AFT Pharmaceuticals Ltd

Contact name

Ioana Stanescu

Public contact point

Organisation

AFT Pharmaceuticals Ltd

Contact name

Ioana Stanescu

Locations

1 EU/EEA country · 1 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 11 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