An Interventional, Phase II, Non Randomized, Mono-centric Study on the Clinical Efficacy and Safety of the Medical Device PAGETEX® as a Photodynamic Therapy Device in the Treatment of Extra-Mammary Paget's Disease of the Vulva (EMPV)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Centre Hospitalier Universitaire De Lille

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Female

Therapeutic area

Diseases [C] - Neoplasms [C04]

Trial duration

27 Aug 2019 → ongoing

Decision date (initial)

2024-09-20

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Lille University Hospital Center (France · LILLE Inserm U1189 ONCO THAI Bâtiment ONCOLILLE, Boulevard du Pr. Jules Leclerc, 59000 Lille Cedex · GALDERMA S.A. Global R&D Rue d'Entre-deux-Villes 10-12 1014 La Tour-de Peilz, SWITZERLAND

External identifiers

EU CT number

2024-514093-41-00

EudraCT number

2018-002604-13

ClinicalTrials.gov

NCT03713203

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Therapy, Efficacy

The main objective of the study will be to define whether the PAGETEX device makes it possible to obtain a disease control rate at 3 months (stability, partial response or total response) in 30% of the patients included, considering the extent of lesion.

Secondary objectives 9

1. Evaluate the clinical response at 6 months to PDT treatment with the PAGETEX medical device on vulvar extra-mammary Paget's disease.
2. Evaluator, by an independent committee of doctors, the clinical response to PDT treatment at 3 and 6 months
3. Evaluate the change in the patient's quality of life at 3 and 6 months of PDT treatment
4. Assess related pain during each PDT session
5. Evaluate the histological evolution of the lesions at 3 and 6 months of PDT treatment
6. Evaluate the clinical evolution of erythema at 3 and 6 months of PDT treatment
7. Evaluate the residual PpIX after each PDT session using the Fotofinder® Dermoscope
8. Evaluate patient satisfaction at 6 months of PDT treatment
9. Evaluate the tolerance/tolerability of PDT treatment

Conditions and MedDRA coding

Vulvar Extra Mammary Paget's Disease confirmed by biopsy

Study design 1 period

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 7

1. Non-invasive, primary or recurrent vulvar Paget's disease after surgical resection
2. Biopsy confirming the diagnosis less than one year old
3. Woman aged ≥18 years
4. Patient capable of understanding and voluntarily forming informed consent
5. Patient capable of adhering to the study visit schedule and other protocol requirements
6. Women of childbearing age must benefit from effective contraception
7. Status of social insured or entitled to social security

Exclusion criteria 16

1. Invasive Paget's disease
2. Underlying adenocarcinoma
3. Prone to reaction/photosensitive disorders
4. Treatment with Imiquimod/Aldara 5% cream in the last 3 months
5. Photodynamic Therapy used to treat MPV lesions within the last 3 months
6. Uses of photosensitive agents in the last 3 months
7. Treatment with an investigational drug within 30 days before the start of the study
8. Allergic or hypersensitivity to methyl aminolevulinate or any of the other ingredients contained in this medicine (propyl p-hydroxybenzoate, cetostearyl alcohol, methyl p-hydroxybenzoate)
9. Allergic or hypersensitive to peanut or soy due to the presence of peanut oil in Metvixia®
10. Patient suffering from Porphyria
11. Patient already treated with topical corticosteroids on the injured area in the last 3 months
12. Patients with immune disorders (HIV, transplantation)
13. Clinical follow-up impossible for psychological, family, social or geographical reasons
14. Legal incapacity (persons deprived of liberty or under guardianship or curatorship)
15. Pregnant or breastfeeding woman
16. Refusal to participate or sign study consent

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. disease control rate defined by the proportion of patients having a clinical response (stability, partial response or complete response to treatment compared to patients who are in progressive disease (increase in size of the lesion, progression to invasive disease and/or adenocarcinoma). The clinical response will be obtained by the investigating doctor during the 3-month visit using the photograph of the lesion at inclusion as a reference.

Secondary endpoints 9

1. The disease control rate at 6 months, assessed by the investigating doctor
2. The disease control rate at 3 and 6 months evaluated by an independent committee of doctors (same definition and grading as by the investigating doctor) based on standardized photographs taken during the inclusion visits, at 3 months and at 6 months
3. The level of quality of life, quality of sexual life, anxiety and depression of the patient assessed from the analysis of the scores of questionnaires validated for dermatology: DLQI, HADS scale, FSFI, SF36 completed at the inclusion visit, at 3 months and at 6 months
4. The level of pain felt by the patient during each PDT session assessed using a visual analog scale (VAS) graded from 0 (no pain) to 10 (unbearable pain). The level of pain will be classified into 4 levels: 0 = no pain/reaction 1 to 3 = pain/weak reaction 4 to 6 = moderate pain/reaction 7 to 10 = severe pain/reaction
5. The presence of Paget cells in the thickness of the epidermis after staining of biopsies using the hematoxylin and eosin staining technique (H&E staining). A vulvar biopsy will be performed for each suspicious lesion, whether or not it is symptomatic at the 3-month and 6-month visit
6. The severity of the erythema at the inclusion visit, at 3 months and at 6 months noted by: The investigating doctor on the basis of a 4-point scale: 0 = no erythema, 1 = mild erythema or pruritus, 2 = moderate erythema (macules, papules, dry desquamation), 3 = severe erythema (vesicles, ulceration, desquamation oozing, necrotic) A quantitative measurement, carried out by colorimetry of an area of damaged skin/or mucosa, defined at inclusion, which will be carried out on 10 to 20 distinct
7. The presence of PpIX in cancer cells after each PDT session effectively using the Fotofinder® dermoscope. The presence or absence of fluorescence on the images will confirm the presence or absence of residual PpIX and therefore the complete or partial destruction of Paget's cells after the PDT session
8. The patient's overall level of satisfaction measured at 6 months of PDT treatment (final visit) by the overall score of a self-questionnaire collecting the patient's impressions in terms of practicality of use, comfort, size, duration of treatment and improvement to be made to the device.
9. Collection of all adverse events (AEs and SAEs) from the start of PDT treatment until the last study visit (according to the drift criteria of the National Cancer Institute (NCI-CTCAE version 4.0) after each PDT session and at each follow-up visit (3 months and 6 months => see paragraph 11.3 for details).

Investigational products

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

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Centre Hospitalier Universitaire De Lille

Sponsor organisation

Centre Hospitalier Universitaire De Lille

Address

2 Avenue Oscar Lambret, Cs 70001 Cs 70001

City

Lille Cedex

Postcode

59037

Country

France

Scientific contact point

Organisation

Centre Hospitalier Universitaire De Lille

Contact name

Pr MORTIER Laurent

Public contact point

Organisation

Centre Hospitalier Universitaire De Lille

Contact name

Pr MORTIER Laurent

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 7 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