Key Takeaways
- In TRuE-V1, topical ruxolitinib 1.5% cream showed clinically meaningful facial repigmentation versus vehicle by week 24, with continued gains through longer treatment.
- Primary endpoint centered on F-VASI improvement; secondary endpoints included T-VASI and global assessments.
- Safety profile was favorable: mostly mild local reactions (application-site acne/erythema/pruritus); systemic exposure remained low for a topical agent.
- Responses concentrated on the face/neck; acral areas remained comparatively refractory and may benefit from adjunct phototherapy.
Abstract
TRuE-V1 was a phase 3, randomized, double-blind study evaluating ruxolitinib 1.5% cream in adolescents and adults with nonsegmental vitiligo. The trial used face-specific (F-VASI) and total-body (T-VASI) endpoints during a vehicle-controlled period followed by extended active treatment. This article summarizes the study design, dosing practices, key efficacy outcomes (with emphasis on facial response), safety findings, and practical considerations for clinical use.
Trial Design
| Element | Details |
|---|---|
| Phase & masking | Phase 3; randomized, double-blind, vehicle-controlled |
| Duration | Vehicle-controlled period (~24 weeks) with extended treatment thereafter |
| Population | Adolescents & adults with nonsegmental vitiligo; facial involvement required for F-VASI endpoints |
| Assessments | Standardized photography; blinded central review where applicable |
Participants
| Category | Criteria/Notes |
|---|---|
| Inclusion | Nonsegmental vitiligo; minimum facial depigmentation threshold (F-VASI); age ≥12 years |
| Exclusion | Recent systemic immunosuppressants; uncontrolled dermatoses; photosensitivity |
| Baseline | Mixed phototypes; facial and non-facial lesions; acral lesions in subset |
Interventions & Dosing
| Arm | Regimen | Notes |
|---|---|---|
| Ruxolitinib 1.5% cream | Thin layer to affected areas, typically BID | Avoid occlusion; apply after emollient if irritation |
| Vehicle | Matched schedule | Eligible for switch to active in extension |
Endpoints
- Primary: Facial repigmentation by F-VASI at end of vehicle-controlled period (e.g., proportion achieving a predefined response threshold).
- Secondary: T-VASI change, patient/physician global assessments, time to visible perifollicular islands, durability on extension.
- Exploratory: QoL instruments (e.g., DLQI/VitiQoL), photographic composites, hair follicle-associated repigmentation patterns.
Efficacy Results
Ruxolitinib cream demonstrated superior facial repigmentation versus vehicle at ~24 weeks, with progressive improvement during continued use. Non-facial areas improved more slowly; acral sites remained the most resistant.
| Outcome | Ruxolitinib cream | Vehicle | Interpretation |
|---|---|---|---|
| F-VASI responders (week ~24) | Higher proportion | Lower proportion | Meets primary endpoint |
| T-VASI % change | Greater reduction | Smaller reduction | Consistent secondary benefit |
| Time to first visible islands | Earlier | Later | Predicts stronger endpoint |
| Durability (extension) | Maintained/improved | — | Supports ongoing therapy |
Safety
| Event | Ruxolitinib cream | Vehicle | Notes |
|---|---|---|---|
| Application-site acne | More frequent | Less frequent | Usually mild; manage with spacing/cleansers |
| Erythema/pruritus | Occasional | Occasional | Often transient; emollients/helpful |
| Headache/URTI | Similar to vehicle | Similar | Non-differentiating |
Topical administration yields low systemic exposure; class warnings for JAK inhibitors remain part of labeling considerations. Clinical judgment is advised in patients with significant infection/malignancy risk factors.
Pharmacokinetics/Exposure
Measured plasma levels after topical dosing are generally low. No signal suggestive of systemic JAK inhibition was evident in routine labs within the trial framework; ongoing vigilance is recommended in real-world use.
Limitations
Facial endpoints may not fully capture whole-body disease activity; acral and long-standing lesions respond less robustly; access/cost and maintenance strategies influence real-world effectiveness.
References
- Phase 3 TRuE-V1 publication reporting facial (F-VASI) and total (T-VASI) outcomes with ruxolitinib 1.5% cream versus vehicle.
- Supplementary extension data describing durability and safety with continued topical JAK inhibition.
- Methodology resources on VASI scoring and photographic assessment in vitiligo trials.