Tofacitinib Cream for Cutaneous T-Cell Lymphoma

Overseen By Julia Dai, MD

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: M.D. Anderson Cancer Center

Must not be taking: Topical corticosteroids, Systemic corticosteroids

Disqualifiers: Active malignancy, Uncontrolled illness, Pregnancy, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

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: 4

Duration: 12 weeks

20 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a cream called tofacitinib to determine its safety and effectiveness for people with early-stage cutaneous T-cell lymphoma (CTCL), a type of skin cancer. Participants will apply the cream to up to five skin lesions twice a day. The trial targets individuals diagnosed with early-stage CTCL who have at least two skin lesions unresponsive to previous treatment and no significant blood involvement. As a Phase 2 trial, the research focuses on evaluating the treatment's effectiveness in an initial, smaller group, providing participants an opportunity to contribute to important findings.

Will I have to stop taking my current medications?

The trial requires that you stop using certain treatments before starting, such as topical corticosteroids, chemotherapy, and other therapies within specific timeframes. It's best to discuss your current medications with the trial team to see if they need to be paused.

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

Research has shown that tofacitinib cream has been tested on people and is approved for use in some other countries, indicating a certain level of safety. However, risks exist. Some individuals using tofacitinib have experienced serious infections, sometimes requiring hospital treatment or posing life-threatening situations.

A separate study found that tofacitinib may increase the risk of heart problems and certain types of cancer. While tofacitinib might offer benefits, it can also have serious side effects. Considering these risks is crucial when deciding whether to join a trial. Discuss with a doctor what this could mean personally. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Most treatments for cutaneous T-cell lymphoma, like phototherapy and systemic therapies, can be invasive or have significant side effects. Unlike these standard options, tofacitinib cream is applied topically, directly to the skin lesions, which may offer a more localized treatment with potentially fewer systemic side effects. Tofacitinib works as a Janus kinase (JAK) inhibitor, which is a different mechanism than most current therapies. Researchers are excited about this treatment because it targets specific pathways involved in the disease, offering a promising alternative that could improve patient outcomes with greater convenience and potentially fewer side effects.

What evidence suggests that tofacitinib cream might be an effective treatment for CTCL?

Research has shown that tofacitinib cream holds promise for treating various inflammatory and autoimmune conditions. It has proven effective for conditions like psoriatic arthritis and rheumatoid arthritis. In earlier studies, tofacitinib also benefited people with moderate-to-severe plaque psoriasis, with many patients experiencing significant skin improvements. These results suggest that tofacitinib cream, which participants in this trial will apply, might help treat early-stage cutaneous T-cell lymphoma (CTCL) by targeting similar inflammation pathways. While specific data for CTCL is still being gathered, the treatment's success in related skin conditions offers hope for its effectiveness. ^{3 4 6 7 8}

Who Is on the Research Team?

Julia Dai, MD

Principal Investigator

M.D. Anderson Cancer Center

Are You a Good Fit for This Trial?

This trial is for individuals with early-stage Cutaneous T-cell Lymphoma (CTCL), specifically stages IA, IB, and IIA. Participants should have a confirmed diagnosis of CTCL to be eligible.

Inclusion Criteria

Women of child-bearing potential and men must use adequate contraception.

I agree to use contraception.

My cancer is at stage IIA and has not spread to many lymph nodes.

Exclusion Criteria

I do not have any serious health issues that could affect the study's outcome.

I still have side effects from cancer treatment, except for hair loss.

Pregnant or breastfeeding women are excluded.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants apply tofacitinib 2% cream twice daily on up to 5 treatment-eligible skin lesions

12 weeks

Visits at baseline, 4 weeks, 8 weeks, and 12 weeks

Extension

Participants may choose to extend treatment with assessments at weeks 20, 28, 36, 44, and 52

40 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Tofacitinib

Trial Overview The trial is testing the safety and effectiveness of a topical medication called tofacitinib 2% cream in treating patients with early-stage CTCL.

How Is the Trial Designed?

Group I: Tofacitinib 2% Experimental Treatment 1 Intervention

Participants will apply a thin layer of tofacitinib 2% cream 2 times a day on up to 5 treatment-eligible skin lesions.

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

Approved in United States as Xeljanz for:

• Rheumatoid Arthritis
• Psoriatic Arthritis
• Ulcerative Colitis
• Ankylosing Spondylitis
• Polyarticular Course Juvenile Idiopathic Arthritis

Approved in European Union as Xeljanz for:

• Rheumatoid Arthritis
• Psoriatic Arthritis
• Ulcerative Colitis
• Ankylosing Spondylitis

Who Is Running the Clinical Trial?

M.D. Anderson Cancer Center

Lead Sponsor

Trials

3,107

Recruited

1,813,000+

Published Research Related to This Trial

Tofacitinib, an oral Janus kinase inhibitor, was found to be safe and well-tolerated in a study involving 95 healthy participants, with only mild adverse events like nausea and headache reported.

The pharmacokinetics of tofacitinib showed rapid absorption and elimination, indicating that it reaches peak serum concentration quickly and is eliminated from the body efficiently, which is important for its potential therapeutic use.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Safety, tolerability, and pharmacokinetics of single oral doses of tofacitinib, a Janus kinase inhibitor, in healthy volunteers. Krishnaswami, S., Boy, M., Chow, V., et al.[2022]

Tofacitinib (5 mg twice daily) significantly improves key symptoms of active psoriatic arthritis (PsA) and related conditions after 3 months of treatment, with benefits maintained for up to 30 months in ongoing studies.

The drug has an acceptable safety profile, showing low rates of serious infections, malignancies, and other adverse events over 36 months, indicating it is a viable option for patients who have not responded to previous therapies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Tofacitinib: A Review in Psoriatic Arthritis. Paik, J., Deeks, ED.[2020]

Tofacitinib, a selective Janus kinase inhibitor, shows promising efficacy in treating various dermatological conditions such as psoriasis, alopecia areata, vitiligo, and atopic dermatitis, based on a review of 43 studies.

Despite its potential benefits, there are safety concerns regarding tofacitinib's overall profile, indicating that further research is needed to establish a favorable risk-to-benefit ratio for its use in dermatology.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Tofacitinib, an Oral Janus Kinase Inhibitor: Perspectives in Dermatology. Kostovic, K., Gulin, SJ., Mokos, ZB., et al.[2018]

Tofacitinib, an oral JAK inhibitor, demonstrated bioequivalence to Xeljanz® in a phase I trial involving 32 healthy Chinese subjects, with pharmacokinetic parameters such as peak concentration and area under the curve falling within the acceptable range of 80-125%.

The safety profile of tofacitinib was found to be comparable to that of Xeljanz®, supporting its potential for clinical use in treating inflammatory and autoimmune diseases.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A randomized, crossover, phase I clinical study to evaluate bioequivalence and safety of tofacitinib and Xeljanz® in Chinese healthy subjects. Xu, Z., Wang, Y., Liu, Z., et al.[2022]

Tofacitinib, a pan Janus kinase inhibitor, has shown efficacy as a standalone treatment for patients new to methotrexate and those who did not respond fully to it, as well as in combination with other drugs for patients who failed TNF inhibitors, leading to its approval for use in many countries.

The clinical trials demonstrated a reasonable safety profile for tofacitinib, with ongoing assessments in long-term studies and postmarketing reports confirming its safety in diverse patient populations.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Tofacitinib in the treatment of active rheumatoid arthritis in adults. Fleischmann, R.[2018]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC6035868/

JAK inhibitors in dermatology: the promise of a new drug class The efficacy of tofacitinib in moderate-to-severe plaque psoriasis was shown in phase 3 randomized controlled trials. In one of the studies, the PASI 75 ...

2. cancer.gov cancer.gov/research/participate/clinical-trials-search/v?id=NCI-2024-09674

Tofacitinib 2% Cream for the Treatment of Stage IA, IB, and ... This phase II trial studies how well tofacitinib 2% cream works in treating stage IA, IB, and IIA cutaneous T-cell lymphoma (CTCL).

3. ema.europa.eu ema.europa.eu/en/documents/product-information/xeljanz-epar-product-information_en.pdf

Xeljanz, INN-tofacitinib citrate - European Medicines Agency Tofacitinib is indicated for the treatment of adult patients with moderately to severely active ulcerative colitis (UC) who have had an inadequate response, ...

4. accessdata.fda.gov accessdata.fda.gov/drugsatfda_docs/label/2018/203214s018lbl.pdf

XELJANZ (tofacitinib) - accessdata.fda.gov SERIOUS INFECTIONS. Patients treated with XELJANZ/XELJANZ XR are at increased risk for developing serious infections that may lead to hospitalization or death [ ...

5. withpower.com withpower.com/trial/phase-2-lymphoma-4-2025-b57a0

Tofacitinib Cream for Cutaneous T-Cell Lymphoma Tofacitinib has been shown to be effective in treating other inflammatory and autoimmune conditions like psoriatic arthritis and rheumatoid arthritis, where it ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT06698822

Study Details | NCT06698822 | A Phase 2 Trial to Assess ... A Phase 2 Trial to Assess Safety and Efficacy of Tofacitinib 2% Cream in the Treatment of Cutaneous T-cell Lymphoma (CTCL), Stages IA, IB, and IIA.

7. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11853177/

Therapeutic Targeting of the Janus Kinase/Signal Transducer ... A phase 1/2 study of tofacitinib combined with chidamide (a HDAC inhibitor) in treatment of EN/NK/T-cell lymphoma (NCT03598959) and a phase 2 ...

8. nejm.org nejm.org/doi/full/10.1056/NEJMoa2109927

Cardiovascular and Cancer Risk with Tofacitinib in ... Risks of MACE and cancers were higher with tofacitinib and did not meet noninferiority criteria. Several adverse events were more common with tofacitinib.