Could a cancer drug regrow hair in rare autoimmune disease?
NCT ID NCT05398809
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This phase 2 trial tests whether ruxolitinib, a drug already approved for certain blood disorders, can help regrow hair in people with APECED, a rare immune condition that often causes severe alopecia areata. About 70 participants aged 12 to 75 will take the pill twice daily for 8 months, with doctors tracking hair regrowth and safety. The study is open-label, meaning everyone knows they are getting the drug, and it is currently recruiting.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- ruxolitinib (a JAK inhibitor pill)
- What this could lead to
- If successful, this could offer a treatment option for hair regrowth in people with APECED-related alopecia areata, a condition with no approved therapies.
- What could go wrong
- This is an early-phase, open-label trial with no placebo group, so results may be less reliable. Ruxolitinib can cause side effects like infection or blood changes, and hair regrowth may not be sustained.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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About 70 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Jan 2023
- Expected to finish
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Dec 2027
An estimate. End dates often move.
- Lead sponsor
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A government research agency
The lead sponsor is the US National Institutes of Health.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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12 to 75 years
- Sex
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Anyone
- Healthy volunteers
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Accepted
You do not need to have the condition being studied to take part.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
* INCLUSION CRITERIA: For participants with APECED-associated AA: 1. Participant must be able to understand and provide informed consent. 2. Aged \>=12 to \<=75 years. 3. Patients with APECED (genetic or clinical diagnosis) and severe AA (defined as having \>=50% total scalp loss at screening per the SALT score). 4. Duration of hair loss greater than 6 months. 5. No present evidence of hair regrowth. 6. Is na(SqrRoot) ve or unresponsive to other treatments for AA. 7. No treatment for alopecia in the past 2 months prior to study enrollment. 8. Willingness to use valacyclovir prophylaxis for the prevention of herpes viral reactivation. 9. Vaccinations should be up to date in agreement with current CDC immunization guidelines prior to start of ruxolitinib. 10. Proficient in written English. 11. 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. Highly effective contraceptive measures include: 1. Stable use of combined (estrogen- and progestogen-containing) hormonal contraception (oral, intravaginal, transdermal) or progestogen-only hormonal contraception (oral, injectable, implantable) starting 1 month prior to screening. 2. Intrauterine device; intrauterine hormone-releasing system. 3. Two barrier methods (eg, condom with spermicide, diaphragm with spermicide, or cervical cap and spermicide). Internal and external condoms may not be used together. 4. Bilateral tubal ligation. Periodic abstinence (calendar, symptothermal, and post-ovulation methods), withdrawal (coitus interruptus), spermicides only, and lactational amenorrhea method are not acceptable methods of contraception. For family members or household contacts: 1. Aged \>=18 years. 2. Lives with the participant. 3. Proficient in written English. EXCLUSION CRITERIA: For participants with APECED-associated AA: 1. Known history of hypersensitivity to ruxolitinib or other JAK inhibitors. 2. History of or active skin disease on the scalp other than AA, such as psoriasis or seborrheic dermatitis. 3. 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. 4. 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. 5. Current or recent use of any investigational drug (within 3 months or 5 half-lives, whichever is longer, prior to screening). 6. Scheduled to participate in another clinical study involving an investigational drug during the course of this study. 7. Use of systemic immunosuppressive or immune-modulating agents within 3 months prior to screening, except systemic steroids 10 mg of prednisone equivalent per day. 8. 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. 9. History of alcohol or drug abuse within 6 months prior to screening. 10. Presence of one or more of the following clinically significant laboratory abnormalities: 1. Serum ALT\>=3 times upper limit of normal (ULN). 2. Serum total bilirubin \>=2 times ULN. 3. ANC\<=1000 cells/microliter. 4. Hemoglobin \<=9.0 g/dL. 5. Platelet count \<=100,000/microliter. 6. Serum creatinine \>=2 times ULN. 11. Planned or anticipated major surgical procedure during the study. 12. Plans to receive any live vaccines within 1 month of the anticipated first dose of ruxolitinib. 13. Known or suspected immunodeficiency disorder besides APECED. 14. 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. 15. Untreated latent tuberculosis infection. 16. Infection with HIV. 17. Untreated infection with hepatitis B or C. 18. History of serious bacterial infection within the last 3 months prior to screening, unless treated and resolved with antibiotics, or any chronic bacterial infection (eg, chronic pyelonephritis, osteomyelitis). 19. History of unprovoked DVT, PE, arterial thrombosis, or other thrombotic events. 20. History of stroke, heart attack, or heart failure. 21. History of herpes zoster or cytomegalovirus infection that resolved within 2 months prior to screening. 22. History of basal cell carcinoma, localized squamous cell carcinoma of the skin, or in situ carcinoma of the cervix, unless the participant is in remission and curative therapy was completed at least 12 months prior to screening. 23. History of other malignancies, unless the participant is in remission and curative therapy was completed at least 5 years prior to screening. 24. Planned or anticipated use of any prohibited medications and procedures during the study. 25. Current pregnancy or breastfeeding. 26. Past or current medical problems or findings from physical examination, EKG, or laboratory testing that are not listed above, which, in the opinion of the investigator, may pose additional risks from participation in the study, may interfere with the participant s ability to comply with study requirements or that may impact the quality or interpretation of the data obtained from the study. There are no exclusion criteria for family members or household contacts.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The study's own enquiry address
This study publishes an address for enquiries. See it below .
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The places running it
1 site. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Show contact details
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Genom att skicka in godkänner du våra Användarvillkor
Study contacts
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Contact
Email: •••••@•••••
Locations
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National Institutes of Health Clinical Center
RECRUITINGBethesda, Maryland, 20892, United States
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