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RecruitingNCT05398809Updated Nov 6, 2026

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

A Phase 2 interventional study of Ruxolitinib in Autoimmune Polyendocrinopathy Candidiasis Ectodermal Dystrophy (Apeced) and Alopecia Areata, sponsored by National Institute of Allergy and Infectious Diseases (NIAID). Recruiting at 1 site in United States. Open to participants aged 12 Years to 75 Years, including healthy volunteers. Per ClinicalTrials.gov, last updated 2026-11-06.

Sponsored by National Institute of Allergy and Infectious Diseases (NIAID) · Phase 2, Interventional, and Treatment

From the registry’s dates

  • Started Jan 2023; still recruiting 3 years 9 months later.
Phase
Phase 2
Study type
Interventional
Enrollment
70
Allocation
Not applicable
Ages
12 Years to 75 Years
Sex
All
01

Study summary

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.

Read the detailed description

Study Description:

This is a phase 2 open-label study to evaluate the efficacy and safety of the Janus-associated kinase (JAK) inhibitor, ruxolitinib, in severe APECED-associated alopecia areata (AA). Following an 8-week observation period to assess baseline disease state, qualifying participants may be admitted for up to 10 days for an inpatient visit to start receiving twice daily dosing of oral ruxolitinib. The dose will be increased at weeks 8 and 16, but may be maintained or reduced if the participant experiences certain adverse events (AEs). The total duration of the ruxolitinib regimen is 8 months. At in-person study visits, participants will be monitored for AEs/reactions and will complete patient-reported outcome (PRO) metrics to track their symptoms and general wellbeing. Participants will also have televisits during the months when there is no in-person visit, for AE assessment and local blood draws for safety labs.

Family members or household contacts of participants will also be enrolled to complete a quality-of-life survey, for comparison with a corresponding patient survey.

Primary Objective:

To evaluate the efficacy of ruxolitinib on hair regrowth in participants with APECED-associated AA.

Secondary Objectives:

  1. To evaluate the safety of ruxolitinib in patients with APECED-associated AA.
  2. To assess additional measures of efficacy related to hair regrowth in response to ruxolitinib treatment.
  3. To investigate the effect of ruxolitinib on PROs related to hair regrowth.
  4. To evaluate the effect of ruxolitinib on histologic and immunologic features of the skin.

Exploratory Objectives:

  1. To investigate the effect of ruxolitinib on improving clinical symptoms and associated laboratory, histologic, and immunologic abnormalities in affected organs and tissues beyond the skin.
  2. To investigate the effect of ruxolitinib on PROs of affected organs and tissues beyond the skin.
  3. To investigate the effect of ruxolitinib on prevention of development of new APECED associated complications during the 32-week treatment period.
  4. To examine the effects of ruxolitinib on ameliorating immunologic readouts of excessive interferon (IFN)- >=/JAK/STAT signaling in peripheral blood and affected organs and tissues beyond the skin.
  5. To determine whether ruxolitinib treatment affects the composition of microbial communities at the mucocutaneous barriers.

Primary Endpoint:

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

Secondary Endpoints:

  1. Incidence of SAEs, AEs requiring study drug discontinuation, and other AEs.
  2. Achievement of 30 percent improvement of SALT score (SALT30) at week 16.
  3. Achievement of 50 percent improvement of SALT score (SALT50) at week 32.
  4. Achievement of SALT score \<=20 at week 32.
  5. Time for participants to achieve SALT score \<=20.
  6. Time for participants to achieve SALT30 and SALT50.
  7. Percentage of hair regrowth at week 32.
  8. Improvement of Alopecia Areata Symptom Impact Scale (AASIS) score from baseline at week 32.
  9. Improvement of Dermatology Life Quality Index (DLQI) score (adults) from baseline at week 32.
  10. Improvement of Children s Dermatology Life Quality Index (CDLQI) score (pediatrics) from baseline at week 32.
  11. Improvement of Family Dermatology Life Quality Index (FDLQI) score (family members or household contacts) from baseline at week 32.
  12. Improvement of the 36-Item Short Form Survey (SF-36) score (adults) from baseline at week 32.
  13. Improvement of the Pediatric Quality of Life Inventory (PedsQL) score (pediatrics) from baseline at week 32.
  14. PRO for Scalp Hair Assessment Score of 0 or 1 with a >=2-point improvement from baseline among participants with a score of >=3 at baseline at week 32.
  15. Improvement of AA investigator global assessment (IGA) measure from baseline at week 32.
  16. Achievement of clinician-reported outcome (ClinRO) measure for eyebrow (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.
  17. Achievement of ClinRO measure for eyelash (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.
  18. Change from baseline in Skindex-16 AA symptoms, emotions, and functioning domain scores at week 32.
  19. Change from baseline in histologic and immunologic abnormalities of scalp tissue at week 32.

Exploratory Endpoints:

  1. Change from baseline in histologic or immunologic abnormalities of salivary gland tissue in participants with Sj(SqrRoot)(Delta)gren s-like syndrome at week 32.
  2. Change from baseline in histologic or immunologic abnormalities of oral mucosal tissue at week 32.
  3. Change from baseline in histologic or immunologic abnormalities of gastric tissue in participants with gastritis at week 32.
  4. Change from baseline in histologic or immunologic abnormalities of duodenal tissue in participants with intestinal dysfunction at week 32.
  5. Time to development of next APECED-associated manifestation(s) over the 32-week treatment period.
  6. Number of APECED-associated manifestations developed over the 32-week treatment period.
  7. Development of new APECED-associated manifestation(s) over the 32-week treatment period.
  8. Change from baseline in irritable bowel syndrome (IBS) quality of life (QOL) and small intestinal bacterial overgrowth (SIBO) questionnaire scores in participants with intestinal dysfunction at week 32.
  9. Change from baseline in salivary flow rate in participants with Sj(SqrRoot)(Delta)gren s-like syndrome at week 32.
  10. Change from baseline in corticosteroid dose requirements, adrenocorticotropic hormone, cortisol, electrolyte, and renin levels in participants with adrenal insufficiency at week 32.
  11. Change from baseline in thyroid hormone replacement dose requirements, thyroid-stimulating hormone (TSH), free and total thyroxine, anti-thyroglobulin, and thyroid peroxidase antibody in participants with hypothyroidism at week 32.
  12. Change from baseline in calcium replacement requirements, intact parathyroid hormone, and calcium levels in participants with hypoparathyroidism.
  13. Change from baseline in insulin requirements, hemoglobin A1C (HbA1c), fasting glucose, insulin, c-peptide, urine ketone, and glucose levels in participants with type 1 diabetes at week 32.
  14. Change from baseline in levels of follicle-stimulating hormone, estradiol, Anti-Mullerian hormone, total and free testosterone, and sex hormone binding globulin in participants with hypogonadism at week 32.
  15. Change from baseline in vitamin B12 and intrinsic factor antibody levels in participants with pernicious anemia at week 32.
  16. Change from baseline in Vitiligo Area Scoring Index (VASI) in participants with vitiligo at week 32.
  17. Change from baseline in Ocular Surface Disease Index score (OSDI) in participants with dry eyes at week 32.
  18. Change from baseline measure corneal fluorescein staining (grading scale), tear breakup time (TBUT, in seconds), automated keratography TBUT, and tear meniscus assessments in participants with keratoconjunctivitis at week 32.
  19. Change from baseline grade of conjunctival redness in participants with keratoconjunctivitis at week 32.
  20. Change from baseline in tear production as assessed by Schirmer s test in participants with Sj(SqrRoot)(Delta)gren s-like syndrome at week 32.
  21. Change from baseline in frequency and severity for oral, vaginal, and/or nail candidiasis during the 32-week treatment period.
  22. Change from baseline in spleen volume as measured by ultrasound at week 32.
  23. Change from baseline in nail appearance in participants with nail dystrophy at week 32.
  24. Improvement in ClinRO PRO for nail appearance from baseline at 32 weeks.
  25. Change from baseline in cytokine- and tissue-specific autoantibodies over the 32-week treatment period.
  26. Change from baseline of levels of phosphorylated STAT molecules in peripheral blood mononuclear cells (PBMCs) over the 32-week treatment period.
  27. Change from baseline in serum levels of IFN-Y, CXCL9, CXCL10, and/or other immune-based biomarkers over the 32-week treatment period.
  28. Change from baseline in transcriptional profile in whole blood and tissue biopsies over the 32-week treatment period.
  29. Change from baseline in the oral, vaginal, stool, and/or skin microbiome over the 32-week treatment period.
02

Conditions studied

  • Autoimmune Polyendocrinopathy Candidiasis Ectodermal Dystrophy (Apeced)
  • Alopecia Areata

Keywords

  • Aps-1
  • Aire
  • Jak
  • Autiommunity
  • Hair Loss
03

In context

Alopecia Areata

220 studies on the registry are indexed under Alopecia Areata; 61 are open to participants now.

This study's planned enrollment of 70 is above the median of 50 across 171 interventional studies indexed under Alopecia Areata.

Browse Alopecia Areata studies →

Lead sponsor

National Institute of Allergy and Infectious Diseases (NIAID) is the lead sponsor of 2,446 studies on the registry; 179 are open to participants now.

Of its 405 completed or terminated interventional studies of FDA-regulated products, 302 (75%) have results posted.

Browse National Institute of Allergy and Infectious Diseases (NIAID) studies →

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
12 Years to 75 Years
Sexes eligible
All
Accepts healthy volunteers
Yes

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

For participants with APECED-associated AA:

  1. Participant must be able to understand and provide .
  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 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

EXCLUSION CRITERIA:

For participants with APECED-associated AA:

  1. Known history of 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 , 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 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 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 laboratory abnormalities:

    1. Serum >=3 times (ULN).
    2. Serum total >=2 times ULN.
    3. ANC\<=1000 cells/microliter.
    4. \<=9.0 g/dL.
    5. 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 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 .
  17. Untreated infection with 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 , 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, , 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.

Terms in these criteria, in plain language(17)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Informed consent

Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.

You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.

Contraception

Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.

Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.

Hypersensitivity

An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.

Corticosteroids (steroids)

Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.

Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.

Investigational drug or device

A drug or device being studied in a clinical trial.

It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.

Immunosuppression

A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.

Read whether the line is about a condition or about medicines, and which medicines and doses it means.

Clinically significant

Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.

ALT and AST (liver enzymes)

Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.

Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.

ULN (upper limit of normal)

The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.

“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.

Bilirubin

A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.

Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.

Hemoglobin (Hb)

The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).

Platelets

Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.

Live vaccines

Vaccines made with a weakened form of the germ, such as measles–mumps–rubella (MMR), chickenpox and yellow fever. They can be risky when the immune system is weakened. Flu injections are not live; the nasal flu spray is.

HIV

The human immunodeficiency virus, which weakens the immune system.

Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.

Hepatitis B tests

Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.

Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.

Cancer (malignancy)

Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.

Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.

ECG (electrocardiogram)

A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.

05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
70 participants (estimated)

Study arms

  • Experimental
    Ruxolitinib/APECED-AA Patients

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

    Drug: Ruxolitinib

Interventions

  • DrugRuxolitinib

    Ruxolitinib (Jakafi) is approved by the FDA for the treatment of intermediate or high-risk myelofibrosis in adults, polycythemia vera in adults who have had an inadequate response to or are intolerant of hydroxyurea, and acute and chronic graft-versus-host disease in adult and pediatric patients (aged =12 years).

06

What researchers measure

Primary outcomes

  1. 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 regrowth in participants with APECED-associated AA

    Time frame: 32 weeks

Secondary outcomes

  1. Improvement of AA-IGA measure from baseline at week 32.

    1\. To evaluate the safety of ruxolitinib in patients with APECED associated AA 2. Assess additional measures of efficacy related to hair regrowth in response to ruxolitinib treatment. 3. Investigate the effect of ruxolitinib on PROs related to hair regrowth. 4. Evaluate the effect of ruxolitinib on the histologic and immunologic features of the skin.

    Time frame: Week 32

  2. Improvement of DLQI score (adults) from baseline at week 32.

    1\. To evaluate the safety of ruxolitinib in patients with APECED associated AA 2. Assess additional measures of efficacy related to hair regrowth in response to ruxolitinib treatment. 3. Investigate the effect of ruxolitinib on PROs related to hair regrowth. 4. Evaluate the effect of ruxolitinib on the histologic and immunologic features of the skin.

    Time frame: Week 32

  3. Improvement of AASIS score from baseline at week 32.

    1\. To evaluate the safety of ruxolitinib in patients with APECED associated AA 2. Assess additional measures of efficacy related to hair regrowth in response to ruxolitinib treatment. 3. Investigate the effect of ruxolitinib on PROs related to hair regrowth. 4. Evaluate the effect of ruxolitinib on the histologic and immunologic features of the skin.

    Time frame: Week 32

  4. Percentage of hair regrowth at week 32.

    1\. To evaluate the safety of ruxolitinib in patients with APECED associated AA 2. Assess additional measures of efficacy related to hair regrowth in response to ruxolitinib treatment. 3. Investigate the effect of ruxolitinib on PROs related to hair regrowth. 4. Evaluate the effect of ruxolitinib on the histologic and immunologic features of the skin.

    Time frame: Week 32

  5. Time for participants to achieve SALT30 and SALT50.

    1\. To evaluate the safety of ruxolitinib in patients with APECED associated AA 2. Assess additional measures of efficacy related to hair regrowth in response to ruxolitinib treatment. 3. Investigate the effect of ruxolitinib on PROs related to hair regrowth. 4. Evaluate the effect of ruxolitinib on the histologic and immunologic features of the skin.

    Time frame: Variable

  6. Achievement of SALT50 at week 32.

    1\. To evaluate the safety of ruxolitinib in patients with APECED associated AA 2. Assess additional measures of efficacy related to hair regrowth in response to ruxolitinib treatment. 3. Investigate the effect of ruxolitinib on PROs related to hair regrowth. 4. Evaluate the effect of ruxolitinib on the histologic and immunologic features of the skin.

    Time frame: Week 32

  7. Achievement of SALT30 at week 16.

    1\. To evaluate the safety of ruxolitinib in patients with APECED associated AA 2. Assess additional measures of efficacy related to hair regrowth in response to ruxolitinib treatment. 3. Investigate the effect of ruxolitinib on PROs related to hair regrowth. 4. Evaluate the effect of ruxolitinib on the histologic and immunologic features of the skin.

    Time frame: Week 16

  8. Change from baseline in histologic and immunologic abnormalities of scalp tissue at week 32.

    1\. To evaluate the safety of ruxolitinib in patients with APECED associated AA 2. Assess additional measures of efficacy related to hair regrowth in response to ruxolitinib treatment. 3. Investigate the effect of ruxolitinib on PROs related to hair regrowth. 4. Evaluate the effect of ruxolitinib on the histologic and immunologic features of the skin.

    Time frame: Week 32

  9. Change from baseline in Skindex-16 AA symptoms, emotions, and functioning domain scores at week 32.

    1\. To evaluate the safety of ruxolitinib in patients with APECED associated AA 2. Assess additional measures of efficacy related to hair regrowth in response to ruxolitinib treatment. 3. Investigate the effect of ruxolitinib on PROs related to hair regrowth. 4. Evaluate the effect of ruxolitinib on the histologic and immunologic features of the skin.

    Time frame: Week 32

  10. 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.

    1\. To evaluate the safety of ruxolitinib in patients with APECED associated AA 2. Assess additional measures of efficacy related to hair regrowth in response to ruxolitinib treatment. 3. Investigate the effect of ruxolitinib on PROs related to hair regrowth. 4. Evaluate the effect of ruxolitinib on the histologic and immunologic features of the skin.

    Time frame: Week 32

  11. 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.

    1\. To evaluate the safety of ruxolitinib in patients with APECED associated AA 2. Assess additional measures of efficacy related to hair regrowth in response to ruxolitinib treatment. 3. Investigate the effect of ruxolitinib on PROs related to hair regrowth. 4. Evaluate the effect of ruxolitinib on the histologic and immunologic features of the skin.

    Time frame: Week 32

  12. PRO for Scalp Hair Assessment Score of 0 or 1 with a =2-point improvement from baseline among participants with a score of =3 at baseline at week 32.

    1\. To evaluate the safety of ruxolitinib in patients with APECED associated AA 2. Assess additional measures of efficacy related to hair regrowth in response to ruxolitinib treatment. 3. Investigate the effect of ruxolitinib on PROs related to hair regrowth. 4. Evaluate the effect of ruxolitinib on the histologic and immunologic features of the skin.

    Time frame: Week 32

  13. Improvement of PedsQL score (pediatrics) from baseline at week 32.

    1\. To evaluate the safety of ruxolitinib in patients with APECED associated AA 2. Assess additional measures of efficacy related to hair regrowth in response to ruxolitinib treatment. 3. Investigate the effect of ruxolitinib on PROs related to hair regrowth. 4. Evaluate the effect of ruxolitinib on the histologic and immunologic features of the skin.

    Time frame: Week 32

  14. Improvement of SF-36 score (adults) from baseline at week 32.

    1\. To evaluate the safety of ruxolitinib in patients with APECED associated AA 2. Assess additional measures of efficacy related to hair regrowth in response to ruxolitinib treatment. 3. Investigate the effect of ruxolitinib on PROs related to hair regrowth. 4. Evaluate the effect of ruxolitinib on the histologic and immunologic features of the skin.

    Time frame: Week 32

  15. Improvement of FDLQI score (family members or household contacts) from baseline at week 32.

    1\. To evaluate the safety of ruxolitinib in patients with APECED associated AA 2. Assess additional measures of efficacy related to hair regrowth in response to ruxolitinib treatment. 3. Investigate the effect of ruxolitinib on PROs related to hair regrowth. 4. Evaluate the effect of ruxolitinib on the histologic and immunologic features of the skin.

    Time frame: Week 32

  16. Improvement of CDLQI score (pediatrics) from baseline at week 32.

    1\. To evaluate the safety of ruxolitinib in patients with APECED associated AA 2. Assess additional measures of efficacy related to hair regrowth in response to ruxolitinib treatment. 3. Investigate the effect of ruxolitinib on PROs related to hair regrowth. 4. Evaluate the effect of ruxolitinib on the histologic and immunologic features of the skin.

    Time frame: Week 32

  17. Incidence of SAEs, AEs requiring study drug discontinuation, and other AEs.

    1\. To evaluate the safety of ruxolitinib in patients with APECED associated AA 2. Assess additional measures of efficacy related to hair regrowth in response to ruxolitinib treatment. 3. Investigate the effect of ruxolitinib on PROs related to hair regrowth. 4. Evaluate the effect of ruxolitinib on the histologic and immunologic features of the skin.

    Time frame: End of Study

07

Study locations

1 of 1 sites recruiting
  • National Institutes of Health Clinical Center
    Bethesda, Maryland 20892, United States
    Recruiting
    • For more information at the NIH Clinical Center contact Office of Patient Recruitment (OPR) · Contactccopr@nih.gov · 800-411-1222
08

References and documents

09

Timeline

3 registry updates since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reachedSimilar studies: half by the dot; the band is the middle halfHalf of similar studies reached it by the dot; the band is the middle half, from a quarter to three quarters

Started Jan 2023 (actual). Primary completion is estimated for Dec 2027, 58 months after the start.

Of 30 phase 2 interventional studies under Alopecia Areata, half reached primary completion within 24 months of starting (the median), counting those still running; a quarter within 15 and three quarters within 35. This study's planned 58 months is above that.

How these figures are worked out, and which studies are left out of them.

Registered
May 26, 2022
First posted
Jun 1, 2022
StartActual
Jan 18, 2023
Primary completionEst.
Dec 1, 2027
CompletionEst.
Dec 1, 2027
Last update
Nov 6, 2026
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
Minor edits
Nothing that changes what the study is or who can join. Edited: verification date
3 updates, last Nov 6, 2026
Show all 3 updates
  1. Nov 6, 2026
    Minor edits only
    + 1 other change: verification date
  2. Nov 2, 2026
    Minor edits only
    + 1 other change: verification date
  3. Oct 23, 2026
    Minor edits only
    + 1 other change: verification date

From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗

10

Registry details

Key details

Study ID
NCT05398809
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
Responsible party
Sponsor
First posted
Jun 1, 2022
Start date
Jan 18, 2023
Primary completion
Dec 1, 2027 (estimated)
Completion
Dec 1, 2027 (estimated)
Last update
Nov 6, 2026

Study contacts

Michail S Lionakis, M.D.
Contact
RepAIREStudyTeam@mail.nih.gov
Not Listed
Michail S Lionakis, M.D.
principal investigator · National Institute of Allergy and Infectious Diseases (NIAID)

Oversight

FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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Studies of the drug tested here: Ruxolitinib

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