Evaluate the Efficacy and Safety of Ruxolitinib on Hair Regrowth in Patients With Autoimmune Polyendocrinopathy Candidiasis Ectodermal Dystrophy (APECED)-Associated Alopecia Areata

Recruiting now · Phase 2

Conditions studied: Autoimmune Polyendocrinopathy Candidiasis Ectodermal Dystrophy (Apeced), Alopecia Areata

In brief

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.

Key facts

Study ID

NCT05398809

Run by

National Institute of Allergy and Infectious Diseases (NIAID)

People needed

70

Starts

2023-01-18

Expected to finish

2027-12-01

Last updated by the study team

2026-06-22

Who can join

Age: 12 and older, up to 75. Sex: any. Healthy volunteers: accepted.

You may not qualify if…

• For participants with APECED-associated AA:
• Known history of hypersensitivity to ruxolitinib or other JAK inhibitors.
• History of or active skin disease on the scalp other than AA, such as psoriasis or seborrheic dermatitis.
• Diagnosis of AA is in question or the pattern of hair loss is such that quantification of hair loss and assessment of regrowth is difficult, eg, patients with androgenic alopecia.
• Treated within the last 2 months with intralesional steroids, systemic steroids, anthralin, squaric acid, diphenylcyclopropenone, tacrolimus, minoxidil, or other medication that, in the opinion of the investigator, may affect hair regrowth.
• Current or recent use of any investigational drug (within 3 months or 5 half-lives, whichever is longer, prior to screening).
• Scheduled to participate in another clinical study involving an investigational drug during the course of this study.
• Use of systemic immunosuppressive or immune-modulating agents within 3 months prior to screening, except systemic steroids 10 mg of prednisone equivalent per day.
• Current use of systemic steroids with daily dose >10 mg of prednisone equivalent for any reason or steroid burst for >3 days within 1 month of screening.
• History of alcohol or drug abuse within 6 months prior to screening.
• Presence of one or more of the following clinically significant laboratory abnormalities:
• Serum ALT>=3 times upper limit of normal (ULN).
• Serum total bilirubin >=2 times ULN.
• ANC<=1000 cells/microliter.
• Hemoglobin <=9.0 g/dL.
• Platelet count <=100,000/microliter.
• Serum creatinine >=2 times ULN.
• Planned or anticipated major surgical procedure during the study.
• Plans to receive any live vaccines within 1 month of the anticipated first dose of ruxolitinib.
• Known or suspected immunodeficiency disorder besides APECED.
• History of untreated invasive opportunistic infections (eg, tuberculosis, non-tuberculous mycobacterial infections, histoplasmosis, listeriosis, coccidioidomycosis, pneumocystis pneumonia, aspergillosis) despite infection resolution or otherwise recurrent infections of
• abnormal frequency or prolonged infections suggesting an immune-compromised status as judged by the investigator.
• Untreated latent tuberculosis infection.
• Infection with HIV.
• Untreated infection with hepatitis B or C.

Where it is running

• National Institutes of Health Clinical Center — Bethesda, Maryland, United States (enrolling)

Full record on ClinicalTrials.gov

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