Ruxolitinib for Alopecia Areata

Overseen By Michail S Lionakis, M.D.

Age: Any Age

Sex: Any

Trial Phase: Phase 2

Sponsor: National Institute of Allergy and Infectious Diseases (NIAID)

Must be taking: Valacyclovir

Must not be taking: Steroids, Immunosuppressives

Disqualifiers: HIV, Hepatitis B/C, Heart failure, 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: 6

Duration: 32 weeks

70 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores whether ruxolitinib, a study drug, can aid hair regrowth in individuals with alopecia areata linked to APECED, a rare immune system disorder. The researchers aim to determine if it can also improve other symptoms caused by the immune system mistakenly attacking the body. Suitable candidates have APECED, have experienced significant hair loss for over six months, and have not seen any hair regrowth. Participants will take the study drug for eight months, with several in-person and telehealth check-ins throughout the trial. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.

Will I have to stop taking my current medications?

The trial requires that you have not used certain treatments for alopecia in the past 2 months and that you do not use systemic immunosuppressive or immune-modulating agents within 3 months prior to screening. If you are currently on these medications, you may need to stop them before joining the trial.

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

Research has shown that ruxolitinib, a JAK inhibitor, has been tested for safety in treating conditions like alopecia areata (AA), which causes hair loss. In one study, 75% of people with AA experienced significant hair regrowth after using ruxolitinib, suggesting it could be an effective treatment.

Regarding safety, studies indicate that ruxolitinib is generally well-tolerated, with most people not experiencing serious side effects. However, some might encounter mild side effects, such as headaches or minor stomach issues. Discussing any concerns with a healthcare provider is important.

In summary, ruxolitinib has shown promise in aiding hair regrowth in AA and is generally safe for most people. Always consult a doctor to understand how it might work for individual needs. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Ruxolitinib is unique because it targets the Janus kinase (JAK) pathway, which is a new approach for treating alopecia areata. While current treatments like corticosteroids and topical immunotherapies mainly focus on reducing inflammation or stimulating hair growth, ruxolitinib directly modulates the immune response by inhibiting specific pathways involved in the autoimmune attack on hair follicles. Researchers are excited about ruxolitinib because it offers the potential for more precise control of the immune system, which could lead to more effective and faster hair regrowth compared to existing options.

What evidence suggests that ruxolitinib might be an effective treatment for alopecia areata?

Research has shown that ruxolitinib may help treat alopecia areata (AA), a type of hair loss. This trial will evaluate the effectiveness of ruxolitinib in participants. Studies have found that similar treatments have led to significant hair regrowth. Specifically, one study showed that many patients experienced at least a 30% improvement in their hair condition. Ruxolitinib blocks certain proteins in the body that can cause inflammation and hair loss. Early results suggest that this drug could benefit people with AA, particularly those with APECED, by promoting hair regrowth and reducing other immune system issues. ^{1 2 3 6 7}

Who Is on the Research Team?

Michail S Lionakis, M.D.

Principal Investigator

National Institute of Allergy and Infectious Diseases (NIAID)

Are You a Good Fit for This Trial?

This trial is for people aged 12-65 with APECED and severe alopecia areata, meaning they've lost at least half their scalp hair. They haven't seen any hair regrowth recently, haven't treated their alopecia in the last two months, and have had hair loss for over six months. Participants must be up-to-date on vaccinations, willing to use herpes prevention medication and contraception if necessary, and able to give informed consent.

Inclusion Criteria

I am willing to take medication to prevent herpes reactivation.

Participants who can get pregnant or impregnate their partner must agree to use at least one highly effective method of contraception when engaging in sexual activities that can result in pregnancy, starting at screening until 12 weeks after the last dose

I have APECED and severe alopecia with over 50% scalp hair loss.

Exclusion Criteria

Current or recent use of any investigational drug

My hair loss diagnosis is uncertain or hard to measure.

You have had a bad reaction to ruxolitinib or similar medications in the past.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

1 visit (in-person)

Observation

Participants undergo an 8-week observation period to assess baseline disease state

8 weeks

Treatment

Participants receive oral ruxolitinib twice daily for 8 months with dose adjustments at weeks 8 and 16

32 weeks

5 visits (in-person), 6 visits (virtual)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Ruxolitinib

Trial Overview The study tests Ruxolitinib's effectiveness in promoting hair regrowth in patients with autoimmune-related severe alopecia areata. Over a period of up to 10 months, participants will take Ruxolitinib orally twice daily for eight months while undergoing various assessments including physical exams, blood tests, tissue samples from the scalp and other areas, as well as telehealth check-ins.

How Is the Trial Designed?

Group I: Ruxolitinib/APECED-AA Patients Experimental Treatment 1 Intervention

All participants receiving IP through this protocol (APECED-AA patients)will receive ruxolitinib.

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

Approved in United States as Jakafi for:

• Intermediate or high-risk myelofibrosis
• Polycythemia vera
• Steroid-refractory acute graft-versus-host disease
• Chronic graft-versus-host disease
• Vitiligo

Approved in European Union as Jakavi for:

• Intermediate or high-risk myelofibrosis
• Polycythemia vera
• Steroid-refractory acute graft-versus-host disease
• Chronic graft-versus-host disease
• Non-segmental vitiligo

Who Is Running the Clinical Trial?

National Institute of Allergy and Infectious Diseases (NIAID)

Lead Sponsor

Trials

3,361

Recruited

5,516,000+

Published Research Related to This Trial

In a pilot study involving 12 patients with moderate-to-severe alopecia areata, 75% experienced significant hair regrowth (average of 92%) after treatment with the oral JAK1/2 inhibitor ruxolitinib for 3-6 months.

The treatment was well-tolerated with no serious adverse effects reported, and gene expression analysis indicated a reduction in inflammatory markers associated with the disease, suggesting a mechanism of action that targets the autoimmune response.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Oral ruxolitinib induces hair regrowth in patients with moderate-to-severe alopecia areata. Mackay-Wiggan, J., Jabbari, A., Nguyen, N., et al.[2023]

Oral JAK inhibitors have shown a significantly higher response rate in treating alopecia areata compared to control groups, with a response rate of 6.86 times greater in randomized controlled trials involving 1845 patients.

Topical and sublingual JAK inhibitors were found to be ineffective, and there is a high recurrence rate of 54% after stopping treatment, suggesting that continuous administration may be necessary to maintain benefits.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The efficacy and safety of JAK inhibitors for alopecia areata: A systematic review and meta-analysis of prospective studies. Yan, D., Fan, H., Chen, M., et al.[2022]

Ruxolitinib, a JAK1/2 inhibitor, effectively modulated inflammatory changes in human dermal papilla cells (hDPCs) associated with alopecia areata by blocking the JAK-STAT pathway, which may help in restoring hair growth.

The treatment also stimulated key growth factors and activated the Wnt/β-catenin signaling pathway, promoting signals necessary for hair follicle re-entry into the growth phase, while preserving immune privilege in hair follicles.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The Effect of JAK Inhibitor on the Survival, Anagen Re-Entry, and Hair Follicle Immune Privilege Restoration in Human Dermal Papilla Cells. Kim, JE., Lee, YJ., Park, HR., et al.[2021]

In two phase III trials involving 855 adults with severe alopecia areata, baricitinib treatment over 52 weeks led to significant hair regrowth, with 40.9% of patients achieving a SALT score ≤ 20 in one trial and 36.8% in the other.

The treatment was generally well-tolerated, with no new safety concerns identified, although common side effects included upper respiratory infections and headaches, suggesting that long-term use of baricitinib can be safe and effective for managing severe alopecia areata.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy and Safety of Baricitinib in Patients with Severe Alopecia Areata over 52 Weeks of Continuous Therapy in Two Phase III Trials (BRAVE-AA1 and BRAVE-AA2). Kwon, O., Senna, MM., Sinclair, R., et al.[2023]

Oral tofacitinib and ruxolitinib show a 66% success rate in achieving significant improvement in alopecia areata, based on a systematic review of 12 studies involving 346 patients.

Common adverse events include infections and laboratory abnormalities, with a notable increase in lab issues for patients treated longer than six months, and a high recurrence rate of 74% within three months after stopping treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Treatment outcome of oral tofacitinib and ruxolitinib in patients with alopecia areata: A systematic review and meta-analysis. Yu, DA., Kim, YE., Kwon, O., et al.[2022]

A systematic review of 30 studies involving 289 cases found that 72.4% of patients with alopecia areata responded to JAK inhibitors, with a mean time to initial hair growth of about 2.2 months and complete regrowth in approximately 6.7 months.

Oral administration of JAK inhibitors was significantly more effective than topical treatments, and recurrences of hair loss were noted primarily after treatment was stopped, highlighting the need for ongoing therapy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

JAK inhibitors for alopecia areata: a systematic review and meta-analysis. Phan, K., Sebaratnam, DF.[2019]

In a long-term study involving two patients with alopecia areata, oral ruxolitinib led to sustained, near-complete hair regrowth over a year without any significant side effects.

The results suggest that ruxolitinib is a safe and effective treatment option for individuals with alopecia who do not respond to other therapies, warranting its consideration in community dermatology settings.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Two cases of alopecia areata treated with ruxolitinib: a discussion of ideal dosing and laboratory monitoring. Vandiver, A., Girardi, N., Alhariri, J., et al.[2021]

JAK inhibitors represent a significant advancement in the treatment of moderate-to-severe alopecia areata (AA), a condition that previously had limited treatment options.

Phase 3 clinical trial data supports the efficacy of JAK inhibitors in treating AA, highlighting the importance of understanding the differences among various JAK inhibitors for optimal patient selection and safety considerations.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Janus kinase inhibitors for alopecia areata. King, BA., Craiglow, BG.[2023]

Ruxolitinib, a Janus kinase inhibitor approved for myelofibrosis and polycythemia vera, shows promising efficacy in treating dermatologic conditions like atopic dermatitis, psoriasis, and vitiligo, based on data from clinical trials.

The evidence for ruxolitinib's effectiveness in alopecia areata is mixed, varying between topical and oral formulations, indicating the need for further research to clarify its role in treating various skin disorders.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Off-label Studies on the Use of Ruxolitinib in Dermatology. Tegtmeyer, K., Ravi, M., Zhao, J., et al.[2021]

A systematic review of 28 studies involving 1719 patients found that JAK-inhibitors for alopecia areata are generally well-tolerated, with most adverse events being mild and a low withdrawal rate due to side effects (1.6% compared to 2.2% for placebo).

Common adverse events included laboratory abnormalities (40.1%), respiratory issues (20.8%), and skin reactions (17.2%), but no fatal outcomes were reported, indicating a favorable safety profile for these treatments.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Adverse events in patients treated with Jak-inhibitors for alopecia areata: A systematic review. Sechi, A., Song, J., Dell'Antonia, M., et al.[2023]

Citations

1. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/40794245/

Evaluating Current and Emergent JAK Inhibitors for ... Ritlecitinib similarly reported 23% of patients achieving a SALT score ≤ 20 at week 24. Deuruxolitinib has also shown efficacy with 31% of ...

2. jamanetwork.com jamanetwork.com/journals/jamanetworkopen/fullarticle/2806418

Janus Kinase Inhibitors for Alopecia Areata: A Systematic ... Main Outcomes and Measures The primary outcomes of interest were (1) proportion of patients who achieved 30%, 50%, and 90% improvement in ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT05398809

Study Details | NCT05398809 | Evaluate the Efficacy and ... Response defined as a 30% improvement from baseline in the Severity of Alopecia Tool (SALT) score at 32 weeks. To evaluate the efficacy of ruxolitinib on hair ...

4. sciencedirect.com sciencedirect.com/science/article/pii/S2352512625001730

Topical ruxolitinib in combination with oral minoxidil results ... Case report. Topical ruxolitinib in combination with oral minoxidil results in hair regrowth in a patient with central centrifugal cicatricial alopecia and ...

5. link.springer.com link.springer.com/article/10.1007/s13555-025-01517-9

Evaluating Current and Emergent JAK Inhibitors for ... In the latest interim efficacy results, ritlecitinib continues to demonstrate successful scalp hair regrowth, increasing from 40–45% of patients ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12454744/

Evaluating Current and Emergent JAK Inhibitors for ... Efficacy and safety of baricitinib in patients with severe alopecia areata over 52 weeks of continuous therapy in two phase III trials ...

7. insight.jci.org insight.jci.org/articles/view/89790

Oral ruxolitinib induces hair regrowth in patients with ... In this pilot study, 9 of 12 patients (75%) treated with ruxolitinib showed significant scalp hair regrowth and improvement of AA.