Ritlecitinib for Vitiligo

(Tranquillo Trial)

Not currently recruiting at 160 trial locations

Overseen By Pfizer CT.gov Call Center

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: Pfizer

Must not be taking: Antibiotics, Antivirals, Antifungals, others

Disqualifiers: Psychiatric conditions, Skin disorders, Infections, 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

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 52 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the effectiveness of Ritlecitinib, an oral medication, for treating nonsegmental vitiligo, a condition that causes skin to lose its pigment. Researchers aim to determine if Ritlecitinib can restore skin color over a year compared to a placebo (inactive pill). Individuals with nonsegmental vitiligo for at least three months and visible patches on their body and face might be suitable for this study. Participants must not use other vitiligo treatments during the trial. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to a potentially groundbreaking treatment.

Do I need to stop my current medications for the trial?

You can continue taking your current medications as long as they are not for vitiligo and your regimen is stable, meaning no new drugs or dosage changes within 7 days or 5 half-lives before the trial starts. You must stop all other treatments for vitiligo during the trial.

Will I have to stop taking my current medications?

The trial requires that you stop all treatments for vitiligo, but you can continue taking other medications as long as you stay on a stable regimen (no changes in dosage or new medications) during the study.

Is there any evidence suggesting that Ritlecitinib is likely to be safe for humans?

Previous studies have shown that ritlecitinib works well and is safe for patients with nonsegmental vitiligo. Over 48 weeks, most patients tolerated the treatment well, with few serious side effects. One study found that less than 0.1% of participants experienced a significant drop in white blood cell count, a very rare occurrence. Age might influence some side effects, particularly in participants aged 65 and older. Overall, earlier trials have demonstrated a strong safety record for the treatment. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for vitiligo?

Ritlecitinib is unique because it works by targeting Janus kinase 3 (JAK3), which plays a key role in the immune response involved in vitiligo. Unlike standard treatments like topical corticosteroids or calcineurin inhibitors, which primarily manage inflammation, Ritlecitinib aims to directly interrupt the signaling pathways that lead to skin depigmentation. Researchers are excited about this approach because it could offer a more targeted and possibly more effective way to restore skin pigmentation for people with vitiligo.

What evidence suggests that Ritlecitinib might be an effective treatment for vitiligo?

Research has shown that ritlecitinib, which participants in this trial may receive, effectively treats nonsegmental vitiligo, a condition causing loss of skin color. Studies have found that patients using ritlecitinib experienced noticeable skin color return over 48 weeks. One study showed that combining ritlecitinib with another treatment improved facial skin color by 69.6%, compared to 55.1% with ritlecitinib alone. These results suggest that ritlecitinib can help restore skin color for people with vitiligo and appears to be a promising treatment option. ^{1 2 3 4 6}

Who Is on the Research Team?

Pfizer CT.gov Call Center

Principal Investigator

Pfizer

Are You a Good Fit for This Trial?

Adults and adolescents with vitiligo, both active (with at least one active lesion) and stable (no signs of disease progression), can join this study. Participants must have a certain amount of facial involvement and overall body surface area affected by vitiligo. Those under 18 may join in some regions if approved. People with skin conditions that could affect the trial or severe psychiatric issues, recent infections requiring hospitalization, or other health risks are excluded.

Inclusion Criteria

My vitiligo is either not changing or slowly getting worse.

I have trichrome lesions.

I have at least one active vitiligo lesion.

Exclusion Criteria

You have a medical or psychiatric condition that could make participating in the study potentially harmful for you or make you unsuitable for the study.

I have vitiligo or another skin condition.

I have been hospitalized or treated for a serious infection recently.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive ritlecitinib or placebo for 52 weeks to assess efficacy, safety, and tolerability

52 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Placebo
• Ritlecitinib

Trial Overview The trial is testing Ritlecitinib oral capsules for treating vitiligo over a period of 52 weeks against a placebo (a pill without any medication). The goal is to see if Ritlecitinib helps restore skin color in patients with either active spots where pigment is lost or stable conditions where there hasn't been any change.

How Is the Trial Designed?

Group I: Ritlecitinib 50 mg Experimental Treatment 1 Intervention

Ritlecitinib 50 mg QD (ritilecitinib 50 mg QD arm; approximately 400 participants)

Group II: Placebo Placebo Group 1 Intervention

Placebo (placebo arm; approximately 200 participants)

Ritlecitinib is already approved in European Union, United States for the following indications:

Approved in European Union as Litfulo for:

• Severe alopecia areata

Approved in United States as Litfulo for:

• Severe alopecia areata

Who Is Running the Clinical Trial?

Pfizer

Lead Sponsor

Trials

4,712

Recruited

50,980,000+

Known For

Vaccine Innovations

Top Products

Viagra, Zoloft, Lipitor, Prevnar 13

Albert Bourla

Pfizer

Chief Executive Officer since 2019

PhD in Biotechnology of Reproduction, Aristotle University of Thessaloniki

Patrizia Cavazzoni

Pfizer

Chief Medical Officer

MD from McGill University

Published Research Related to This Trial

Topical tofacitinib, a Janus kinase (JAK) inhibitor, was used in combination with phototherapy for 9 months to treat an adolescent patient with long-standing and treatment-resistant vitiligo.

The treatment resulted in near complete repigmentation of the skin, suggesting that JAK inhibitors may be a promising therapy for vitiligo.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Topical Tofacitinib: A Janus Kinase Inhibitor for the Treatment of Vitiligo in an Adolescent Patient. Berbert Ferreira, S., Berbert Ferreira, R., Neves Neto, AC., et al.[2021]

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 preliminary study involving four patients with progressing vitiligo, treatment with oral baricitinib for 12 weeks resulted in significant re-pigmentation without major side effects, indicating its efficacy and safety.

In laboratory tests, baricitinib enhanced tyrosinase activity and melanin production in damaged melanocytes, suggesting a mechanism of action that could help restore pigmentation in vitiligo patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Baricitinib is Effective in Treating Progressing Vitiligo in vivo and in vitro. Dong, J., Huang, X., Ma, LP., et al.[2022]

Ritlecitinib (LITFULOTM) is an effective treatment for severe alopecia areata, achieving a significant reduction in hair loss as measured by the Severity of Alopecia Tool (SALT) score, with 31% of participants on the 200 mg dose achieving a SALT score of ≤20 after 24 weeks.

The treatment was generally well-tolerated, with mild to moderate side effects such as headache and upper respiratory infections, indicating a favorable safety profile for patients aged 12 and older.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

LITFULOTM (Ritlecitinib) Capsules: A Janus Kinase 3 Inhibitor for the Treatment of Severe Alopecia Areata. Gupta, AK., Ravi, SP., Vincent, K., et al.[2023]

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]

Ritlecitinib, an oral kinase inhibitor developed by Pfizer, was approved in the USA on June 23, 2023, for treating severe alopecia areata in adults and adolescents aged 12 and older.

The drug has also received approval in Japan for intractable cases of alopecia areata and is undergoing regulatory review in the EU, UK, and China, indicating its potential as a significant treatment option for this condition.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Ritlecitinib: First Approval. Blair, HA.[2023]

Ritlecitinib, an oral Janus kinase inhibitor, is being developed for multiple conditions including alopecia areata and rheumatoid arthritis, with data from 12 clinical trials involving healthy participants and patients collected between 2014 and 2021.

The development of a population pharmacokinetic model for ritlecitinib involved three iterations, leading to a final model that effectively addressed clinical drug development questions and informed the product label, indicating its potential efficacy and safety in various conditions.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Evolution of Ritlecitinib Population Pharmacokinetic Models During Clinical Drug Development. Wojciechowski, J., S Purohit, V., Huh, Y., et al.[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]

Topical ruxolitinib cream has been shown to significantly improve vitiligo symptoms, with 58% of patients achieving a 50% improvement in Vitiligo Area Scoring Index (VASI) after 52 weeks of treatment, making it an effective option for repigmentation.

The treatment has a favorable safety profile, with only mild adverse effects reported, such as erythema and pruritus, and it offers significant clinical improvements compared to traditional therapies, which may have more severe side effects.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Review of Ruxolitinib for Treatment of Non-Segmental Vitiligo. Hwang, JR., Driscoll, MS.[2023]

Citations

1. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/36370907/

Efficacy and safety of oral ritlecitinib for the treatment ... Conclusions: Oral ritlecitinib was effective and well tolerated over 48 weeks in patients with active nonsegmental vitiligo. Keywords: JAK inhibitor; JAK/STAT ...

2. sciencedirect.com sciencedirect.com/science/article/pii/S0190962222029899

Efficacy and safety of oral ritlecitinib for the treatment ... Conclusions. Oral ritlecitinib was effective and well tolerated over 48 weeks in patients with active nonsegmental vitiligo.

3. clinicaltrials.gov clinicaltrials.gov/study/NCT06163326

NCT06163326 | A 52-Week Study to Learn About the ... This study is to evaluate how safe and effective ritlecitinib is in participants with non-segmental vitiligo (NSV).

4. dermatologytimes.com dermatologytimes.com/view/oral-ritlecitinib-plus-nbuv-b-accelerates-repigmentation-in-nonsegmental-vitiligo

Oral Ritlecitinib Plus nbUV-B Accelerates Repigmentation ... Specifically, combination therapy led to a 69.6% mean improvement in facial repigmentation vs 55.1% with ritlecitinib monotherapy. Moreover, the ...

5. pfizerclinicaltrials.com pfizerclinicaltrials.com/find-a-trial/nct06163326-vitiligo-trial

Clinical Trial for Vitiligo. | Pfizer This study is to evaluate how safe and effective ritlecitinib is in participants with non-segmental vitiligo (NSV).

6. litfulo.pfizerpro.com litfulo.pfizerpro.com/safety/pooled-safety-data

Pooled Safety Data Confirmed ALC <500/mm 3 occurred in 1 (<0.1%) subject treated with LITFULO 50 mg · Age appeared to be a risk factor in patients ≥65 years of age.