Upadacitinib for Vitiligo

(Viti-Up Trial)

Recruiting at 159 trial locations

Overseen By ABBVIE CALL CENTER

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: AbbVie

Disqualifiers: Segmental vitiligo, Active skin disease, Leukotrichia, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

614 Participants Needed

Trial Summary

What is the purpose of this trial?

Vitiligo is a common chronic autoimmune disease that causes the body's immune system to attack its own pigment producing skin cells. This study is to evaluate how safe and effective upadacitinib is in participants with non-segmental vitiligo (NSV). Adverse effects and change in disease activity will be assessed. Upadacitinib is an approved drug for various immune-mediated inflammatory diseases and is currently being investigated for the treatment of NSV. There will be 2 replicate studies running at the same time (Study 1 and Study 2 with periods A and B) and an optional exploratory Narrow-Band Ultraviolet B (NB-UVB) phototherapy study (Study 3). In Period A, participants are placed in 1 of 2 groups called treatment arms. Each group receives a different treatment. There is a 1 in 3 chance that participants will receive placebo and 2 in 3 chance participants will receive upadacitinib. In Period B, all participants will receive upadacitinib. Approximately 270 adult and adolescent participants with NSV will be enrolled in each main study ((Study 1 and Study 2, 540 subjects total) at approximately 90 sites worldwide with an option for adult participants who completed Period A of either study and did not achieve T-VASI 90 at week 48 while on study drug, to enter Study 3. In Studies 1 and 2: Period A, participants will receive oral tablets of upadacitinib or placebo once a day for 48 weeks. In Period B, participants will receive oral tablets of upadacitinib 15 mg once a day for 112 weeks. Participants will be followed up for 30 days. Study 3 participants will receive upadacitinib monotherapy or upadacitinib with NB-UBV phototherapy for at least 24 weeks followed by upadacitinib alone. There may be higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular visits during the study at a hospital or clinic. The effect of the treatment will be checked by medical assessments, blood tests, checking for side effects and completing questionnaires.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

What data supports the effectiveness of the drug Upadacitinib for treating vitiligo?

While there is no direct evidence for Upadacitinib in vitiligo, similar drugs like ruxolitinib, which also target immune pathways, have shown promise in repigmenting vitiligo patches. This suggests that Upadacitinib might also be effective due to its similar action on the immune system. ^{1 2 3 4 5}

How does the drug Upadacitinib differ from other treatments for vitiligo?

Upadacitinib is a unique treatment option for vitiligo as it is a JAK inhibitor, which works by blocking specific enzymes (proteins that speed up chemical reactions in the body) involved in the immune response that leads to skin depigmentation. Unlike other treatments like topical creams or light therapy, Upadacitinib is taken orally, offering a different approach to managing the condition. ^{1 2 3 4 5}

Research Team

ABBVIE INC.

Principal Investigator

AbbVie

Eligibility Criteria

Adults and adolescents with non-segmental vitiligo (NSV) who have tried at least one topical treatment without success or show signs of actively worsening vitiligo can join. They must have a certain amount of skin affected but not too much, as measured by specific scales.

Inclusion Criteria

I have been diagnosed with non-segmented vitiligo.

I have vitiligo that hasn't improved with certain creams or is getting worse.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment Period A

Participants receive oral tablets of upadacitinib or placebo once a day

48 weeks

Regular visits at a hospital or clinic

Treatment Period B

All participants receive upadacitinib 15 mg once a day

112 weeks

Regular visits at a hospital or clinic

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Optional Study 3

Participants receive upadacitinib with optional NB-UVB phototherapy

24-28 weeks

Treatment Details

Interventions

• Placebo
• Upadacitinib

Trial Overview The trial is testing Upadacitinib, an oral tablet for autoimmune diseases, against a placebo to see if it's safe and effective for NSV. Participants are randomly assigned to get either the drug or placebo in Period A for 48 weeks; everyone gets Upadacitinib in Period B for 112 weeks.

Participant Groups

Group I: Study 2, Period B: Group 2 Open-Label Extension Period Experimental Treatment 1 Intervention

Participants that were randomized to receive placebo in Period A Group 2, will receive upadacitinib 15 mg once daily for 112 weeks in Period B.

Group II: Study 2, Period B: Group 1 Open-Label Extension Period Experimental Treatment 1 Intervention

Participants that were randomized to receive upadacitinib in Period A Group 1, will continue to receive upadacitinib 15 mg once daily for 112 weeks in Period B.

Group III: Study 2, Period A: Group 1 Experimental Treatment 1 Intervention

Participants will receive upadacitinib 15 mg once daily for 48 weeks.

Group IV: Study 1, Period B: Group 2 Open-Label Extension Period Experimental Treatment 1 Intervention

Participants that were randomized to receive placebo in Period A Group 2, will receive upadacitinib 15 mg once daily for 112 weeks in Period B.

Group V: Study 1, Period B: Group 1 Open-Label Extension Period Experimental Treatment 1 Intervention

Participants that were randomized to receive upadacitinib in Period A Group 1, will continue to receive upadacitinib 15 mg once daily for 112 weeks in Period B.

Group VI: Study 1, Period A: Group 1 Experimental Treatment 1 Intervention

Participants will receive upadacitinib 15 mg once daily for 48 weeks.

Group VII: (Optional) Study 3: Experimental Treatment 2 Interventions

Participants will receive 15 mg upadacitinib once daily for 112 weeks and NB-UVB for up to 28 weeks

Group VIII: (Optional) Study 3, Period B: Open Label Upadacitinib Experimental Treatment 2 Interventions

Open Label Upadacitinib Participants will receive upadacitinib 15 mg once daily for 112 weeks

Group IX: Study 2, Period A: Group 2 Placebo Group 1 Intervention

Participants will receive placebo once daily for 48 weeks.

Group X: Study 1, Period A: Group 2 Placebo Group 1 Intervention

Participants will receive placebo once daily for 48 weeks.

Who Is Running the Clinical Trial?

AbbVie

Lead Sponsor

Trials

1,079

Recruited

535,000+

Founded

2013

Headquarters

North Chicago, USA

Known For

Immunology treatments

Top Products

Humira (adalimumab), Skyrizi (risankizumab), Rinvoq (upadacitinib)

Dr. Roopal Thakkar

AbbVie

Chief Medical Officer since 2023

MD from Wayne State University School of Medicine

Robert A. Michael

AbbVie

Chief Executive Officer

Bachelor's degree in Finance from the University of Illinois

Findings from Research

Combining low-dose tofacitinib with 308-nm excimer laser treatment shows promise as an effective therapy for patients with nonsegmental vitiligo who have not responded to standard treatments.

This combination therapy could offer a new option for patients struggling with vitiligo, potentially improving their skin condition when other therapies have failed.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Low-dose tofacitinib with 308-nm excimer therapy successfully induced repigmentation in patients with refractory vitiligo. Fang, WC., Lin, SY., Huang, SM., et al.[2022]

In a phase 2 trial involving patients with vitiligo, those using 1.5% ruxolitinib cream showed a significant improvement in facial Vitiligo Area Scoring Index scores, with a mean improvement of 79.9% at week 24 compared to only 1.1% in those who did not respond.

Proteomic analysis revealed distinct inflammatory biomarker profiles between patients who achieved at least a 50% improvement and those who did not, suggesting that these biomarkers could help identify which patients are more likely to benefit from ruxolitinib treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Baseline Levels of Circulating Inflammatory Biomarkers Stratify Patients with Vitiligo Who Significantly Repigment after Treatment with Ruxolitinib Cream. Howell, MD., Kuo, FI., Rumberger, B., et al.[2023]

The combination of apremilast and narrowband UVB therapy significantly reduces immune cell markers (like CD8+ T cells) and enhances melanogenesis markers in vitiligo skin, suggesting a more effective treatment approach than narrowband UVB alone.

This study supports the clinical observation that apremilast can enhance the repigmentation effects of narrowband UVB therapy in patients with generalized vitiligo, particularly in skin types IV-VI, although it was limited by a small sample size and high dropout rate.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Apremilast and narrowband ultraviolet B combination therapy suppresses Th17 axis and promotes melanogenesis in vitiligo skin: a randomized, split-body, pilot study in skin types IV-VI. Kim, HJ., Del Duca, E., Pavel, AB., et al.[2023]

In two 52-week phase 3 trials, 52% of patients treated with ruxolitinib 1.5% cream experienced at least a 75% improvement in their vitiligo, demonstrating its efficacy as a treatment.

Ruxolitinib is the first FDA-approved topical medication for repigmentation in vitiligo patients, showing it is both safe and effective, although its cost may limit accessibility for some patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A Review on the Use of Topical Ruxolitinib for the Treatment of Vitiligo. Grossmann, MC., Haidari, W., Feldman, SR.[2023]

In a phase 2 study involving 157 adults with vitiligo, ruxolitinib cream demonstrated significant efficacy, with 45% of patients using the 1.5% twice daily dose achieving at least a 50% improvement in facial vitiligo area by week 24, compared to only 3% in the control group.

The treatment was well tolerated, with most adverse events being mild to moderate, suggesting that ruxolitinib cream could be a safe and effective option for repigmentation in vitiligo patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Ruxolitinib cream for treatment of vitiligo: a randomised, controlled, phase 2 trial. Rosmarin, D., Pandya, AG., Lebwohl, M., et al.[2021]

References

1. England pubmed.ncbi.nlm.nih.gov

Low-dose tofacitinib with 308-nm excimer therapy successfully induced repigmentation in patients with refractory vitiligo. [2022]

2. Netherlands pubmed.ncbi.nlm.nih.gov

Baseline Levels of Circulating Inflammatory Biomarkers Stratify Patients with Vitiligo Who Significantly Repigment after Treatment with Ruxolitinib Cream. [2023]

3. Germany pubmed.ncbi.nlm.nih.gov

Apremilast and narrowband ultraviolet B combination therapy suppresses Th17 axis and promotes melanogenesis in vitiligo skin: a randomized, split-body, pilot study in skin types IV-VI. [2023]

4. United States pubmed.ncbi.nlm.nih.gov

A Review on the Use of Topical Ruxolitinib for the Treatment of Vitiligo. [2023]

5. England pubmed.ncbi.nlm.nih.gov

Ruxolitinib cream for treatment of vitiligo: a randomised, controlled, phase 2 trial. [2021]