Summary
Methotrexate has reached Phase III as its most advanced recorded development stage across any indication, and the database holds no approval for Vitiligo. ClinicalTrials.gov lists 8 registered trials linking Methotrexate to Vitiligo: 4 are active or not yet recruiting; 3 have completed; 1 is terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 4 (NCT05008887). The largest enrolment is 20000 participants (NCT07336641). Registration activity spans 2019 to 2026. The literature layer holds 2 publications for this pair: 1 Cochrane review and 1 systematic review. Publication years run from 2024 to 2026. Because at least one synthesis-level source exists (systematic review, meta-analysis or Cochrane review), this pair has been assessed beyond single studies, although the synthesis may concern a different indication.
The RepurpOS disease-intelligence file for Vitiligo ranks 50 candidate therapeutics from Open Targets, ChEMBL, DGIdb and PubMed; only a minority carry direct clinical evidence, and this page covers one of those. Spontaneous remission context recorded for the condition: Spontaneous complete repigmentation extremely rare (<1%); partial spontaneous repigmentation possible in small lesions; progressive without treatment in most patients.
Evidence table
| Evidence tier | B · Moderate |
|---|---|
| Evidence score | 42.5 (trials 24.5, literature 8.0, tier 10, approved bonus 0.0) |
| Registered trials | 8 total: 0 recruiting, 4 active / not yet recruiting, 3 completed, 1 other |
| Linked publications | 2 (1 Cochrane review, 1 systematic review) |
| Agent type | Drug (Small molecule) |
| Development stage (any indication) | Phase III |
| Mechanism / class | Not recorded |
| Data sources | Open Targets |
How the evidence score is calculated
- Each registered trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5). The trial component is capped at 30.
- Each literature item scores by design (Cochrane review 5, meta-analysis 4, systematic review 3, RCT 3, clinical trial publication 2, curated reference 1.5, other PubMed record 1). The literature component is capped at 30.
- The existing evidence tier adds 15 (A / Strong), 10 (B / Moderate), 5 (C / Preliminary) or 0 (D / Anecdotal).
- Agents approved for any indication (max clinical phase 4) add 5, because an approved agent has an established safety profile that lowers the barrier to repurposing trials.
- The score ranks what has been studied, not what works. It does not read effect sizes or directions of effect.
Registered clinical trials
| NCT ID | Title | Status | Phase | Enrolment |
|---|---|---|---|---|
| NCT07352293 | The Value of Methotrexate in NCES for Stable Vitiligo 2026 | active, not recruiting | Phase 4 | 20 |
| NCT05008887 | Fractional CO2 Laser-assisted Cutaneous Delivery of Methotrexate Versus 5-fluorouracil in Stable Non-segmental Vitiligo 2022 | status unknown | Phase 4 | 50 |
| NCT04942860 | The Evaluation of Vitiligous Lesions Repigmentation After Topical Administration of Methotrexate in Patients With Active Vitiligo 2019 | completed | Phase 3 | 25 |
| NCT04237103 | Combination of Methotrexate and Phototherapy Versus Phototherapy in Adults With Progressive Vitiligo 2023 | completed | Phase 2 | 32 |
| NCT05467839 | Evaluation of Efficacy of Microneedling and Topical Methotrexate Versus Microneedling and Topical 5-flourouracil in Treatment of Vitillgo Patients 2022 | completed | Not applicable | 30 |
| NCT07336641 | LANDSCAPE: Demographics and Treatment Patterns of Patients With Immune-Mediated Inflammatory Skin Diseases in Italian Clinical Practice 2026 | not yet recruiting | Not applicable | 20,000 |
| NCT07208890 | Methotrexate Iontophoresis Versus Methotrexate 1% Gel on Depigmentation in Vitiligo Patients 2025 | not yet recruiting | Not applicable | 30 |
| NCT06877455 | Topical Methotrexate with Fractional CO2 Laser in Treatment of Non Segmental Vitiligo 2026 | not yet recruiting | Not applicable | 50 |
Published literature
- Cochrane review Shining Light on Treatments: A Systematic Review of Existing Therapies for Pediatric Segmental VitiligoGhani H, Hoffman V, Shah A et al. · Pediatric dermatology · 2026 · PMID 41936454
- Systematic review A systematic review of case series and clinical trials investigating systemic oral or injectable therapies for the treatment of vitiligoJafarzadeh A, Pour Mohammad A, Khosravi M et al. · Skin research and technology : official journal of International Society for Bioengineering and the Skin (ISBS) [and] International Society for Digital Imaging of Skin (ISDIS) [and] International Society for Skin Imaging (ISSI) · 2024 · PMID 38454597
Mechanism and notes
No mechanism of action is recorded for this pair in the source databases.
OSMF's existing evidence tier for this pair is B (Moderate), derived from the strength of the disease association recorded in Open Targets. The tier describes how well the drug-disease link is documented, not how well the drug works.
Frequently asked questions
Is Methotrexate approved for Vitiligo?
Methotrexate has reached Phase III as its most advanced recorded development stage across any indication, and the database holds no approval for Vitiligo. Approval status for the specific indication should always be confirmed with the relevant regulator and prescribing information.
Is Methotrexate in clinical trials for Vitiligo?
ClinicalTrials.gov lists 8 registered trials linking Methotrexate to Vitiligo: 4 are active or not yet recruiting; 3 have completed; 1 is terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 4 (NCT05008887). The largest enrolment is 20000 participants (NCT07336641). Registration activity spans 2019 to 2026.
What does the evidence show for Methotrexate in Vitiligo?
Methotrexate has both completed registered trials and synthesis-level publications linked to Vitiligo. That is the strongest profile in this database, but the summaries here do not extract effect sizes, so read the linked reviews for direction and magnitude of benefit. OSMF's existing evidence tier for this pair is B (Moderate), derived from the strength of the disease association recorded in Open Targets. The tier describes how well the drug-disease link is documented, not how well the drug works.
Cite this page
Data: ClinicalTrials.gov, PubMed, Open Targets, ChEMBL, DGIdb and the OSMF therapeutic agent database. Last updated 2026-07-06. Page built 2026-10-07.