Summary
Upadacitinib 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 3 registered trials linking Upadacitinib to Vitiligo: 1 is currently recruiting; 1 is active or not yet recruiting; 1 has completed. The most advanced is Phase 3 (NCT06118411). It plans or enrolled 614 participants. Registration activity spans 2021 to 2024. The literature layer holds 6 publications for this pair: 3 Cochrane reviews, 1 meta-analysis, 1 systematic review and 1 clinical trial publication. 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 | 45.5 (trials 11.5, literature 24.0, tier 10, approved bonus 0.0) |
| Registered trials | 3 total: 1 recruiting, 1 active / not yet recruiting, 1 completed, 0 other |
| Linked publications | 6 (3 Cochrane reviews, 1 meta-analysis, 1 systematic review, 1 clinical trial publication) |
| 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trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5).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 |
|---|---|---|---|---|
| NCT06118411 | A Study To Assess Adverse Events and Effectiveness of Upadacitinib Oral Tablets in Adult and Adolescent Participants With Vitiligo 2023 | active, not recruiting | Phase 3 | 614 |
| NCT04927975 | Study to Evaluate Adverse Events and Change in Disease Activity With Oral Tablets of Upadacitinib in Adult Participants With Non-Segmental Vitiligo 2021 | completed | Phase 2 | 185 |
| NCT06454461 | Efficacy of Upadacitinib After NECS in Vitiligo 2024 | recruiting | Not applicable | 60 |
Published literature
- Cochrane review Large-scale meta-analysis of infection risk with JAK-STAT inhibitors in 29,000 patientsKrebs M, Gulyás L, Fazekas KK et al. · Journal of the European Academy of Dermatology and Venereology : JEADV · 2026 · PMID 42187316
- Clinical trial publication Psychometric Properties and Meaningful Change Thresholds of the Vitiligo Area Scoring IndexEzzedine K, Soliman AM, Camp HS et al. · JAMA dermatology · 2025 · PMID 39475960
- Cochrane review Upadacitinib Therapy in Adolescent Severe Alopecia Areata: A Case Series and Narrative ReviewGao Y, Zhu C, Jin H · Clinical, cosmetic and investigational dermatology · 2025 · PMID 40922723
- Meta-analysis Relative Impact of Monotherapies for Vitiligo: A Network Meta-Analysis StudyGupta AK, Bamimore MA, Seneschal J et al. · Journal of cosmetic dermatology · 2025 · PMID 40013382
- Systematic review Off-label use of JAK1 inhibitor upadacitinib in dermatologyZhang TY, Zeng YP · Archives of dermatological research · 2025 · PMID 39920358
- Cochrane review Protocol for a systematic review and meta-analysis on Janus kinase inhibitors in the management of vitiligoPranić S, Pulumati A, Vuković D · Systematic reviews · 2024 · PMID 38641831
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 Upadacitinib approved for Vitiligo?
Upadacitinib 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 Upadacitinib in clinical trials for Vitiligo?
ClinicalTrials.gov lists 3 registered trials linking Upadacitinib to Vitiligo: 1 is currently recruiting; 1 is active or not yet recruiting; 1 has completed. The most advanced is Phase 3 (NCT06118411). It plans or enrolled 614 participants. Registration activity spans 2021 to 2024.
What does the evidence show for Upadacitinib in Vitiligo?
Upadacitinib 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.