What side effects are associated with this type of intervention?

Common treatments for Alopecia Areata, particularly JAK inhibitors like Ruxolitinib and Tofacitinib, are associated with several side effects. These can include an increased risk of infections, including serious infections, and potential development of malignancies. Laboratory abnormalities such as elevated liver enzymes and changes in blood counts have also been reported. Other immunosuppressants like Methotrexate and Cyclosporine can cause liver toxicity, bone marrow suppression, and increased susceptibility to infections. Close monitoring is essential to manage these potential adverse effects.

What prior FDA approvals exist?

As of now, there are no FDA-approved treatments specifically for alopecia areata. However, several JAK inhibitors, including ruxolitinib, are being studied for their potential efficacy in treating this condition. These drugs work by inhibiting the Janus kinase pathways, which are involved in the immune response that leads to hair loss in alopecia areata. Clinical trials and studies are ongoing to evaluate their safety and effectiveness.

What other types of research exist?

Promising alternatives to Ruxolitinib for Alopecia Areata include Tofacitinib and Dupilumab. Tofacitinib, another JAK inhibitor, has shown significant hair regrowth in both adults and children with Alopecia Areata, Alopecia Totalis, and Alopecia Universalis. Dupilumab, an IL-4 receptor alpha antagonist, has demonstrated potential benefits in clinical trials, with some patients achieving notable improvements in hair regrowth, especially with longer treatment durations.

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Janus Kinase (JAK) Inhibitor

Ruxolitinib for Alopecia Areata

Phase 2

Recruiting

Led By Michail S Lionakis, M.D.

Research Sponsored by National Institute of Allergy and Infectious Diseases (NIAID)

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Patients with APECED (genetic or clinical diagnosis) and severe AA (defined as having >=50% total scalp loss at screening per the SALT score).

No present evidence of hair regrowth.

Must not have

Use of systemic immunosuppressive or immune-modulating agents within 3 months prior to screening, except systemic steroids 10 mg of prednisone equivalent per day

History of or active skin disease on the scalp other than AA, such as psoriasis or seborrheic dermatitis

Timeline

Screening 3 weeks

Treatment Varies

Follow Up week 32

Awards & highlights

Summary

This trial tests if ruxolitinib, a pill that calms the immune system, can help people with APECED and severe hair loss. The medication works by blocking overactive immune signals, which may reduce body attacks and promote hair regrowth. Ruxolitinib has shown promise in treating severe alopecia areata by inducing hair regrowth in patients.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

While not explicitly listed here, potential side effects of Ruxolitinib may include infection risk due to immune system suppression (since it's an immunosuppressant drug), liver function changes, high cholesterol levels; headache; weight gain; dizziness; or difficulty sleeping.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

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

Select...

I have not noticed any new hair growth.

Select...

I have not been treated for AA, or treatments did not work.

Select...

I have APECED and have lost more than half of my scalp hair.

Select...

I have not noticed any new hair growth.

Select...

I am between 12 and 65 years old.

Select...

I understand the study details and can give my consent.

Select...

My alopecia areata hasn't improved with other treatments.

Select...

I have been experiencing hair loss for more than 6 months.

Select...

I am using an intrauterine device for birth control.

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I am between 12 and 65 years old.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

I haven't taken immune-suppressing drugs in the last 3 months, except for low-dose steroids.

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I have or had a skin condition on my scalp other than alopecia areata.

Select...

I am HIV positive.

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I am taking more than 10 mg of steroids daily or had a short course of high-dose steroids recently.

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I have had a stroke, heart attack, heart failure, or serious infection recently.

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I have untreated latent TB.

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I have had serious infections that were not treated.

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I have an immune system disorder, but it's not APECED.

Select...

I expect to undergo a major surgery during the study.

Select...

I have an untreated hepatitis B or C infection.

Select...

I have had cancer before, but it depends on the type and when.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ week 32

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~week 32

This trial's timeline: 3 weeks for screening, Varies for treatment, and week 32 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Response defined as a 30% improvement from baseline in the Severity of Alopecia Tool (SALT) score at 32 weeks.

Secondary study objectives

16. Achievement of ClinRO measure for EB hair loss 0 or 1 with =2-point improvement from baseline (among participants with ClinRO measure for EB hair loss =2 at baseline) at week 32.

Achievement of ClinRO measure for EL hair loss 0 or 1 with =2-point improvement from baseline (among participants with ClinRO measure for EL hair loss =2 at baseline) at week 32.

Achievement of SALT30 at week 16.

+14 more

Side effects data

From 2020 Phase 3 trial • 149 Patients • NCT02038036

33%

Anaemia

19%

Hypertension

17%

Nasopharyngitis

16%

Weight increased

14%

Herpes zoster

14%

Constipation

14%

Abdominal pain

14%

Headache

12%

Pruritus

12%

Back pain

12%

Epistaxis

12%

Pyrexia

12%

Dizziness

10%

Asthenia

10%

Fatigue

10%

Cough

10%

Oedema peripheral

10%

Arthralgia

Thrombocytosis

Upper respiratory tract infection

Hypercholesterolaemia

Haematoma

Dyslipidaemia

Pain in extremity

Abdominal discomfort

Diarrhoea

Dyspepsia

Vomiting

Blood lactate dehydrogenase increased

Memory impairment

Dyspnoea

Tinnitus

Osteoarthritis

Leukocytosis

Thrombocytopenia

Flatulence

Nausea

Sinusitis

Basal cell carcinoma

Neuropathy peripheral

Hyperuricaemia

Cystitis

Blood creatine phosphokinase increased

Bronchitis

Paraesthesia

Skin ulcer

Abdominal pain upper

Pulmonary embolism

Pneumonia

Influenza

Myalgia

Urinary tract infection

Depression

Night sweats

Peripheral artery thrombosis

Vertigo

Intervertebral disc protrusion

Urethral stenosis

Acute pulmonary oedema

Ureterolithiasis

Localised infection

Pericardial effusion

Acute myocardial infarction

Syncope

Gastrooesophageal reflux disease

General physical health deterioration

Atrial fibrillation

Cardiac disorder

Mitral valve incompetence

Vertigo positional

Retinal artery occlusion

Visual acuity reduced

Gastrointestinal haemorrhage

Oesophageal varices haemorrhage

Lower respiratory tract infection

Pyelonephritis

Respiratory tract infection

Sepsis

Tendon rupture

Ulna fracture

Weight decreased

Decreased appetite

Hyponatraemia

Blast cell crisis

Bone marrow tumour cell infiltration

Lung adenocarcinoma

Metastases to spine

Myelofibrosis

Prostatic adenoma

Squamous cell carcinoma of skin

Nephrolithiasis

Gamma-glutamyltransferase increased

Haematocrit increased

Musculoskeletal pain

Ischaemic stroke

Diabetes mellitus

100%

80%

60%

40%

20%

Study treatment Arm

All Crossover Patients

Best Available Therapy

Ruxolitinib

Trial Design

1Treatment groups

Experimental Treatment

Group I: Ruxolitinib/APECED-AA PatientsExperimental Treatment1 Intervention

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

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Ruxolitinib

2018

Completed Phase 3

~1140

0 / 22Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Alopecia Areata include JAK inhibitors like Ruxolitinib and Tofacitinib, which work by interfering with the JAK-STAT signaling pathways. This pathway is involved in the immune response that leads to hair follicle destruction in Alopecia Areata. By inhibiting JAK enzymes, these treatments reduce inflammation and immune activity against hair follicles, promoting hair regrowth. This mechanism is crucial for patients as it directly targets the underlying autoimmune process, offering a potential for more effective and sustained hair regrowth compared to traditional therapies.

Case Report: Successful Treatment of Alopecia Universalis With Tofacitinib and Increased Cytokine Levels: Normal Therapeutic Reaction or Danger Signal?