Actively Recruiting
Evaluate the Efficacy and Safety of Ruxolitinib on Hair Regrowth in Patients With Autoimmune Polyendocrinopathy Candidiasis Ectodermal Dystrophy (APECED)-Associated Alopecia Areata
Led by National Institute of Allergy and Infectious Diseases (NIAID) · Updated on 2025-12-01
70
Participants Needed
1
Research Sites
254 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Background: Autoimmune polyendocrinopathy candidiasis ectodermal dystrophy (APECED) is a problem of the immune system. In people with APECED, the immune system makes a mistake and attacks the body. Some people with APECED have a type of hair loss called alopecia areata (AA). No drugs are approved to treat AA. Objective: To see if a study drug (ruxolitinib) can help hair regrowth in people with APECED-associated AA and if it can improve other symptoms caused by the immune system s attack to the body. Eligibility: People aged 12 to 65 years with APECED and severe AA. Design: Participants will be in this study for up to 10 months. They will have 5 in-person visits and 6 televisits, each about 4 weeks apart. One in-person visit may be up to a 10-day stay in the hospital. The first in-person visit will include screening. Participants will have a physical exam. They will have blood tests. Photographs may be taken of their skin. They will answer questions about their quality of life. Participants will begin taking the study drug during their hospital stay. They will take the pills by mouth twice a day for 8 months. Researchers may take tissue samples from participants scalp, gums, and lower lip. Participants may provide samples of urine, stool, nail clippings, and saliva. They may have an eye exam and an ultrasound exam of their abdomen. Some tests may be repeated in subsequent in-person visits. In telehealth visits, participants will answer questions about how they are feeling. They will describe and send photos of hair regrowth. They will be asked to have blood drawn and the results sent to the researchers.
CONDITIONS
Official Title
Evaluate the Efficacy and Safety of Ruxolitinib on Hair Regrowth in Patients With Autoimmune Polyendocrinopathy Candidiasis Ectodermal Dystrophy (APECED)-Associated Alopecia Areata
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Able to understand and provide informed consent
- Aged 12 to 75 years
- Diagnosed with APECED and severe alopecia areata with at least 50% scalp hair loss
- Hair loss lasting more than 6 months
- No current evidence of hair regrowth
- Naive or unresponsive to other alopecia areata treatments
- No alopecia treatment in the 2 months before enrollment
- Willing to use valacyclovir to prevent herpes virus reactivation
- Vaccinations up to date per CDC guidelines before starting ruxolitinib
- Proficient in written English
- Agree to use highly effective contraception from screening until 12 weeks after last dose if able to get pregnant or impregnate
You will not qualify if you...
- Known allergy to ruxolitinib or other JAK inhibitors
- Active scalp skin diseases other than alopecia areata
- Uncertain alopecia areata diagnosis or hair loss pattern making assessment difficult
- Use of certain alopecia treatments or immunosuppressive drugs recently (within 2-3 months)
- Participation in other investigational drug studies recently or planned
- High dose systemic steroids or steroid bursts near screening
- History of alcohol or drug abuse within 6 months
- Significant abnormal lab results (liver enzymes, blood counts, kidney function)
- Planned major surgery during the study
- Planned live vaccinations within 1 month before first dose
- Known immunodeficiency other than APECED
- History of serious or recurrent infections
- Untreated latent tuberculosis or infections with HIV, hepatitis B or C
- Recent serious bacterial infections or chronic bacterial infections
- History of blood clots, stroke, heart attack, or heart failure
- History of certain infections resolved less than 2 months ago
- History of certain cancers without sufficient remission time
- Use of prohibited medications or procedures during the study
- Current pregnancy or breastfeeding
- Other medical problems posing risks or interfering with study participation as judged by investigators
AI-Screening
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Trial Site Locations
Total: 1 location
1
National Institutes of Health Clinical Center
Bethesda, Maryland, United States, 20892
Actively Recruiting
Research Team
M
Michail S Lionakis, M.D.
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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