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TRUE-V1 and TRUE-V2 clinical trial design

Opzelura® is indicated for the treatment of non-segmental vitiligo with facial involvement in adults and adolescents from 12 years of age1

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Opzelura® monotherapy has been studied in two large, randomised, pivotal studies: TRuE-V1 and TRuE-V22

Opzelura® (ruxolitinib) was studied in two identical double-blind, vehicle-controlled, randomised, Phase III clinical trials, TRuE-V1 and TRuE-V2 (Topical Ruxolitinib Evaluation in Vitiligo Study 1 and 2). 674 adult and adolescent patients from 12 years of age with non-segmental vitiligo with facial involvement were enrolled in the two trials which investigated ruxolitinib, a Janus kinase (JAK) inhibitor for the treatment of non-segmental vitiligo.2

Study design

Image showing baseline characteristics of participants in the TRuE-V1 and TRuE-V2 clinical trials for vitiligo.

Adapted from Rosmarin et al. 2022.

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Study design of TRuE-V1 and TRuE-V2: Supporting context

TRuE-V1 and TRuE-V2 were multinational, randomised Phase III clinical trials assessing ruxolitinib in vitiligo, which took place in over 101 centres in countries including the United States, Canada and Europe (Bulgaria, France, Germany, Italy, Netherlands, Poland and Spain). 2

In TRuE-V1 and TRuE-V2, patients were randomised in a 2:1 ratio to Opzelura® treatment or vehicle cream. 450 patients were assigned to Opzelura® treatment, whilst 224 were assigned the vehicle cream, twice daily (BID) for 24 weeks. After Week 24, there was a 28-week open-label extension, where patients originally assigned to the vehicle could switch to Opzelura®.2

TRuE-V1 and TRuE-V2 enrolled adults and adolescents with non-segmental vitiligo2

Key eligibility criteria for TRuE-V1 and TRuE-V2 participants included:2

  • A confirmed diagnosis of non-segmental vitiligo with depigmented areas of vitiligo covering ≤10% total body surface area (T-BSA), ≥0.5% BSA on the face (F-BSA) and ≥3% non-facial BSA
  • Facial Vitiligo Area Scoring Index (F-VASI) scores of ≥0.5
  • Scores of ≥3 on the Total Vitiligo Area Scoring Index (T-VASI)

Key exclusion criteria for TRuE-V1 and TRuE-V2 participants included:2

  • Complete leukotrichia within any facial lesion
  • Previously received JAK inhibitor therapy
  • Biologic or experimental therapies within 12 weeks (or 5 half-lives), phototherapy within 8 weeks, immunomodulating treatments within 4 weeks, or topical treatments within 1 week
  • Dermatologic disease confounding vitiligo assessment
  • Patients <12 years of age

TRuE-V1 and TRuE-V2 assessed improvement in depigmented skin in participants with vitiligo2

The primary and secondary endpoints in the TRuE-V1 and TRUE-V2 clinical trials used the VASI scoring index to assess improvement in depigmented skin in participants with non-segmental vitiligo.

TRuE-V1 and TRuE-V2 primary endpoint2

  • F-VASI75 – the proportion of participants achieving at least 75% improvement in F-VASI from baseline at Week 24

TRuE-V1 and TRuE-V2 key secondary endpoints2

  • F-VASI90 – the proportion of participants achieving at least 90% improvement in F-VASI from baseline at Week 24
  • F-VASI50 – the proportion of participants achieving at least 50% improvement in F-VASI from baseline at Week 24
  • T-VASI50 – the proportion of participants achieving at least 50% improvement in T-VASI from baseline at Week 24
  • F-BSA – the percentage change from baseline at Week 24
  • Proportion of patients achieving a Vitiligo Noticeability Score (VNS) response*

Other prespecified secondary endpoints include rates of treatment-emergent adverse events (TEAEs)

VASI score

The VASI score is calculated by (surface area of vitiligo lesions in hand units) X (degree of depigmentation of lesions).3,4

Image showing vitiligo lesions and VASI score.

Participants were required to have:

  • F-VASI vitiligo scores from 0–3, with higher scores representing a greater area of facial depigmentation across the forehead to original hairline, cheeks to jawline vertically and laterally from corner of mouth to tragus, nose and eyelids2
  • Scores of 3 or higher on the T-VASI range, 0–100, with higher scores indicating a greater area of total body depigmentation across the head or neck, including the scalp; trunk, including genitalia; upper limbs, including axillae; hands; lower limbs, including buttocks; and feet2

In TRuE-V1 and TRuE-V2 Opzelura® was studied in a range of patients including those with established disease and facial involvement2

The TRuE-V1 and TRuE-V2 trials included all Fitzpatrick skin types, with 89% of participants having types II–IV. The mean age of patients in the safety populations was 40.2 years (adult age group) and 15.9 years (adolescent age group) in TRuE-V1 and 38.9 years (adult age group) and 14.3 years (adolescent age group) years in TRuE-V2.2,5

A total of 10.9% of the patients in TRuE-V1 and 10.5% of those in TRuE-V2 were 12–17 years of age, and 54.5% and 57.1% respectively, of the patients were 40 years of age or younger; 56.4% and 50.1% respectively, of the patients were girls or women.2

Baseline characteristics of participants in TRuE-V12

Graphs showing percentage of participants in the TRuE-V1 clinical trial who have previously received vitiligo treatment.

Adapted from Rosmarin et al. 2022.

Additional baseline characteristics from TRuE-V12

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Vehicle cream
(n=109) 		Opzelura
(n=221)
Race or ethnic group, n (%)‡
White 96 (88.1) 180 (81.4)
Black 4 (3.7) 11 (5.0)
Asian 4 (3.7) 5 (2.3)
Other 2 (1.8) 9 (4.1)
Not reported 3 (2.8) 16 (7.2)
Geographic region, n (%)
North America 73 (67.0) 147 (66.5)
Europe 36 (33.0) 74 (33.5)
F-VASI, mean±SD§ 1.0±0.6 0.9±0.6
T-VASI, mean±SD 6.4±1.9 6.5±2.0
Facial BSA affected by vitiligo, %** 1.2±0.7 1.1±0.7
Total BSA affected by vitiligo, % 7.2±2.0 7.3±2.0
Received diagnosis in childhood, n (%) 34 (31.2) 72 (32.6)
Other autoimmune disorders, n (%)†† 18 (16.5) 53 (24.0)
Thyroid disorders 17 (15.6) 50 (22.6)
Juvenile diabetes mellitus 1 (0.9) 0
Pernicious anaemia 0 1 (0.5)
Other 1 (0.9) 5 (2.3)
Previous therapy, n (%)‡‡ 61 (56.0) 131 (59.3)
Topical calcineurin inhibitors 31 (28.4) 72 (32.6)
Topical glucocorticoids 28 (25.7) 67 (30.3)
NB-UVB phototherapy 20 (18.3) 41 (18.6)
Excimer laser therapy 8 (7.3) 18 (8.1)
PUVA photochemotherapy 4 (3.7) 8 (3.6)
Vitamin D derivatives 2 (1.8) 4 (1.8)
Other 11 (10.1) 24 (10.9)

Adapted from Rosmarin et al. 2022.

Additional baseline characteristics in TRuE-V22

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Baseline characteristics for TRuE-V2
Vehicle cream
(n=115) 		Opzelura
(n=228)
Age (years) mean±SD 39.8±12.1 38.4±15.2
Age group, n (%)
12–17 6 (5.2) 30 (13.2)
18–64 106 (92.2) 186 (81.6)
≥65 3 (2.6) 12 (5.3)
Female sex, n (%) 60 (52.2) 112 (49.1)
Race or ethnic group, n (%)‡
White 93 (80.9) 182 (79.8)
Black 5 (4.3) 12 (5.3)
Asian 7 (6.1) 12 (5.3)
Other 7 (6.1) 19 (8.3)
Not reported 3 (2.6) 3 (1.3)
Fitzpatrick skin type, n (%)§§
I 1 (0.9) 2 (0.9)
II 32 (27.8) 57 (25.0)
III 45 (39.1) 89 (39.0)
IV 25 (21.7) 55 (24.1)
V 10 (8.7) 17 (7.5)
VI 2 (1.7) 8 (3.5)
Geographic region, n (%)
North America 83 (72.2) 160 (70.2)
Europe 32 (27.8) 68 (29.8)
F-VASI, mean±SD§ 0.8±0.5 0.9±0.5
T-VASI, mean±SD 7.0±2.2 6.8±2.1
Facial BSA affected by vitiligo, %** 0.9±0.6 1.0±0.6
Total BSA affected by vitiligo, % 7.7±2.0 7.4±2.0
Duration of disease (years) 16.0±11.6 15.9±12.1
Received diagnosis in childhood, n (%) 43 (37.4) 96 (42.1)
Disease stability, n (%)†
Stable 88 (76.5) 166 (72.8)
Progressive 27 (23.5) 62 (27.2)
Other autoimmune disorders, n (%)††
Thyroid disorders 15 (13.0) 35 (15.4)
Juvenile diabetes mellitus 0 0
Pernicious anaemia 0 0
Other 6 (5.2) 5 (2.2)
Previous therapy, n (%)‡‡ 76 (66.1) 143 (62.7)
Topical calcineurin inhibitors 37 (32.2) 74 (32.5)
Topical glucocorticoids 28 (24.3) 66 (28.9)
NB-UVB phototherapy 27 (23.5) 52 (22.8)
Excimer laser therapy 14 (12.2) 16 (7.0)
PUVA photochemotherapy 8 (7.0) 15 (6.6)
Vitamin D derivatives 1 (0.9) 0
Other 14 (12.2) 34 (14.9)

Adapted from Rosmarin et al. 2022.

*A rating of “a lot less noticeable” or “no longer noticeable”.

†Determination of disease stability was based on investigator judgement.

‡Race and ethnic group were reported by the patient. “Other” includes American Indian or Alaska Native, Native Hawaiian or Pacific Islander and other.

§Scores on the F-VASI range from 0–3, with higher scores indicating a greater area of facial depigmentation.

Scores on the T-VASI range from 0–100, with higher scores indicating a greater area of total body depigmentation.

**Shown is the percentage of total BSA.

††Patients could report multiple autoimmune disorders.

‡‡Patients could have used multiple previous lines of therapy. “Other” includes other types of phototherapy, oral glucocorticoids, surgical techniques and other.

§§Fitzpatrick skin types range from I to VI: type I indicates pale white; type II, white; type III, light brown; type IV, moderate brown; type V, dark brown; and type VI, deeply pigmented brown to black.

Abbreviations

BID, twice daily; BSA, body surface area; F-BSA, facial body surface area; F-VASI, Facial Vitiligo Area Scoring Index; F-VASI50, ≥50% reduction from baseline in Facial Vitiligo Area Scoring Index; F-VASI75, ≥75% reduction from baseline in Facial Vitiligo Area Scoring Index; F-VASI90, ≥90% reduction from baseline in Facial Vitiligo Area Scoring Index; JAK, Janus kinase; NB-UVB, narrowband ultraviolet B; PUVA, psoralen ultraviolet A; SD, standard deviation; T-BSA, total body surface area; TCI, topical calcineurin inhibitor; TCS, topical corticosteroid; TEAE, treatment-emergent adverse event; TRuE-V, topical ruxolitinib evaluation in vitiligo study; T-VASI, Total Vitiligo Area Scoring Index; T-VASI50, ≥50% reduction from baseline in Total Vitiligo Area Scoring Index; VASI, Vitiligo Area Scoring Index; VNS, Vitiligo Noticeability Scale.

References

  1. Opzelura® (ruxolitinib) cream Summary of Product Characteristics. Incyte Biosciences UK Ltd. November 2023.
  2. Rosmarin D, et al. N Engl J Med. 2022;387(16):1445–1455.
  3. Rosmarin D, et al. Efficacy and safety of ruxolitinib cream for the treatment of vitiligo: week 24 pooled analysis of the TRuE-V phase 3 studies. Presented at American Academy of Dermatology 2022 Annual Meeting; March 25–29; Boston, MA. Abstract 34789.
  4. Hamzavi I, et al. Arch Dermatol. 2004;140(6):677–683.
  5. Bae JM, et al. J Am Acad Dermatol. 2022;86(2):387–393.

UNITED KINGDOM
Adverse events should be reported. Reporting forms and information can be found at: www.mhra.gov.uk/yellowcard or search for MHRA Yellow Card in the Google Play or Apple App Store. Adverse events should also be reported to Incyte immediately by calling 03301003677 (Great Britain) 00-800-0002-7423 (United Kingdom (Northern Ireland)).

REPUBLIC OF IRELAND
Adverse events should be reported. Reporting forms and information can be found at: HPRA Pharmacovigilance website: www.hpra.ie Adverse events should also be reported to Incyte by calling 00-800-0002-7423.