Overview
Sponsor-declared trial summary
Phase
Therapeutic use (Phase IV)
Status
Authorised, recruitment pending
Participants planned
30
Countries
1
Sites
2
dVIN caused by Lichen Sclerosus
Determine the effectiveness of MTX in prevention of recurrence in dVIN by the time-to recurrence before and after the use of MTX in women with therapy resistant lichen sclerosus and recurrent dVIN lesions.
Key facts
- Sponsor
- Amsterdam UMC Stichting
- Participant type
- Patients
- Age range
- 18-64 years, 65+ years
- Gender
- Female
- Therapeutic area
- Diseases [C] - Skin and Connective Tissue Diseases [C17], Diseases [C] - Female Urogenital Diseases and Pregnancy Complications [C13]
- Decision date (initial)
- 2026-04-28
- Transition trial
- No
- Low-intervention
- Yes
- Rare-disease indication
- No
- Vulnerable population
- Yes
- Funding sources
- Catharina Onderzoeksfonds
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Safety, Dose response, Therapy, Efficacy
Determine the effectiveness of MTX in prevention of recurrence in dVIN by the time-to recurrence before and after the use of MTX in women with therapy resistant lichen sclerosus and recurrent dVIN lesions.
Secondary objectives 5
- Safety and tolerability of side effects with MTX use and foliac acid.
- Reduction of vulvar complaints since MTX use.
- Reduction of topical corticosteroid application since MTX introduction.
- Patient satisfaction with the use of MTX.
- Tumor microenvironment in recurrent dVIN lesions.
Conditions and MedDRA coding
dVIN caused by Lichen Sclerosus
Study design 2 periods
| # | Title | Allocation | Blinding | Roles blinded | Arms |
|---|---|---|---|---|---|
| 1 | Treatment Determine the effectiveness of MTX in prevention of recurrence in dVIN by the time-to recurrence before and after the use of MTX in women with lichen sclerosus and recurrent dVIN lesions.
|
Not Applicable | None | ||
| 2 | Follow up Follow up after treatment
|
Not Applicable | None |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 3
- Age of 18 years or older.
- Failure to standard therapy with intensive clobetasol ointment either or not combined with tacrolimus 0,1% ointment. Failure to standard therapy is defined by complaints of lichen sclerosus which are insufficiently controlled by the topical therapy.
- Recurrent dVIN lesions, at least 1 previous dVIN, for which surgical treatment has been performed.
Exclusion criteria 13
- Concomitant diagnoses of vulvar cancer
- Severe decreased liver function with a serum bilirubin value >85,5umol/l or >5mg/dl
- Severe decreased kidney function with creatinine clearance of <30ml/min
- Bone marrow diseases (including leukopenia, thrombocytopenia, anemia, bone marrow hypoplasia)
- Acute or chronic infections (including HIV/AIDS, tuberculosis)
- Immunodeficiency or immunosuppressive medication
- Severe pulmonary disease
- Pregnancy or current pregnancy wish
- Legal incapability
- Insufficient knowledge of the Dutch or English language
- Alcohol abuse.
- Oral or gastro-intestinal ulcera or stomatitis
- Recently received a live attenuated vaccine or when in need of receiving a live attenuated vaccine.
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 2
- To assess effectiveness of MTX in prevention of recurrence in dVIN, recurrence rates with or without MTX will be compared within this prospective self-controlled pilot study, as each patient functions as its own control.
- Time-to recurrence of dVIN in women with lichen sclerosus and recurrent dVIN lesions with- and without the use of MTX will also be assessed by determination of clinical responses at 1-3-6-9-12-15-18 months and will be identified as stable disease (SD) or progressive disease (PD).
Secondary endpoints 2
- Safety and tolerability of side effects, reduction of vulvar complaints, reduction of topical corticosteroid application and patient satisfaction with the use of MTX will be evaluated by patient reported use and documentation of side effects using a questionnaire. Severity of side effects will be evaluated according to the CTCEA version 5.0 2017.
- Pre-treatment and, when performed, current- or post-treatment biopsies will be identified and central blinded pathology revision will be performed. To explore microenvironment in recurrent dVIN with and without the use of MTX immunohistochemistry staining, including p53 will be performed.
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
Methotrexaat Sandoz 2,5 mg, tabletten
PRD12020963 · Product
- Active substance
- Methotrexate
- Pharmaceutical form
- TABLET
- Route of administration
- ORAL USE
- Max daily dose
- 2.57 mg milligram(s)
- Max total dose
- 1560 mg milligram(s)
- Max treatment duration
- 78 Week(s)
- Authorisation status
- Authorised
- ATC code
- L01BA01 — METHOTREXATE
- Marketing authorisation
- RVG 28636
- MA holder
- SANDOZ B.V.
- MA country
- Netherlands
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Amsterdam UMC Stichting
- Sponsor organisation
- Amsterdam UMC Stichting
- Address
- De Boelelaan 1117
- City
- Amsterdam
- Postcode
- 1081 HV
- Country
- Netherlands
Scientific contact point
- Organisation
- Amsterdam UMC Stichting
- Contact name
- Annemijn Aarts
Public contact point
- Organisation
- Amsterdam UMC Stichting
- Contact name
- Annemijn Aarts
Locations
1 EU/EEA country · 2 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Netherlands | Authorised, recruitment pending | 30 | 2 |
| Rest of world | — | 0 | — |
Investigational sites
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 6 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_Protocol_2025-524373-17-00 | 4 |
| Protocol (for publication) | D4_Patient facing documents deelnemersdagboekje | 1 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements | 2 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF | 3 |
| Summary of Product Characteristics (SmPC) (for publication) | E2_SmPC_Methotrexaat Sandoz | 1 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis The Netherlands | 1 |
Application history
1 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2026-01-02 | Netherlands | Acceptable 2026-04-20
|
2026-04-28 |