Autoimmune Polyendocrinopathy Candidiasis Ectodermal Dystrophy study: Ruxolitinib
Official title Evaluate the Efficacy and Safety of Ruxolitinib on Hair Regrowth in Patients With Autoimmune Polyendocrinopathy Candidiasis Ectodermal Dystrophy (APECED)-Associated Alopecia Areata
ClinicalTrials.gov ID: NCT05398809
What this study is testing
What is Ruxolitinib?
This is an investigational pill being studied to see if it can help hair regrow in people with APECED-associated alopecia areata. It is designed to block certain signals in the immune system that cause inflammation.
Also referred to as Jakafi.
Plain-language explanation of the investigational treatment. It is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This study aims to see if ruxolitinib can help hair regrow and improve other symptoms caused by the immune system attacking the body.
- Who it's for
- The study is looking for people aged 12 to 65 who have a rare immune disorder called APECED and severe patchy hair loss.
- What's involved
- Participants will take a pill twice a day for 8 months. They will have physical exams, blood tests, and may provide skin, urine, or saliva samples.
- Time commitment
- The study lasts up to 10 months and includes 5 in-person visits and 6 video visits, with one visit that may require a 10-day hospital stay.
- Phase 2: a mid-size study of how well it works
- Time commitment: about 10 months
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 12 to 75. Healthy volunteers may be eligible.
You may be able to join if
- Participant must be able to understand and provide informed consent.
- Aged \>=12 to \<=75 years.
- Patients with APECED (genetic or clinical diagnosis) and severe AA (defined as having \>=50% total scalp loss at screening per the SALT score).
- Duration of hair loss greater than 6 months.
- No present evidence of hair regrowth.
You likely can't join if
- 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...
- Treated within the last 2 months with intralesional steroids, systemic steroids, anthralin, squaric acid, diphenylcyclopropenone, tacrolimus...
- 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.
See the full eligibility criteria
- Participant must be able to understand and provide informed consent.
- Aged \>=12 to \<=75 years.
- Patients with APECED (genetic or clinical diagnosis) and severe AA (defined as having \>=50% total scalp loss at screening per the SALT score).
- Duration of hair loss greater than 6 months.
- No present evidence of hair regrowth.
- Is na(SqrRoot) ve or unresponsive to other treatments for AA.
- No treatment for alopecia in the past 2 months prior to study enrollment.
- Willingness to use valacyclovir prophylaxis for the prevention of herpes viral reactivation.
- Vaccinations should be up to date in agreement with current CDC immunization guidelines prior to start of ruxolitinib.
- Proficient in written English.
- 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...
- Intrauterine device; intrauterine hormone-releasing system.
- Two barrier methods (eg, condom with spermicide, diaphragm with spermicide, or cervical cap and spermicide). Internal and external condoms may not be used together.
- Aged \>=18 years.
- Lives with the participant.
- Proficient in written English.
- 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...
- 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.
- 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...
- Untreated latent tuberculosis infection.
- Infection with HIV.
- Untreated infection with hepatitis B or C.
- 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).
- History of unprovoked DVT, PE, arterial thrombosis, or other thrombotic events.
- History of stroke, heart attack, or heart failure.
- History of herpes zoster or cytomegalovirus infection that resolved within 2 months prior to screening.
- 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...
- History of other malignancies, unless the participant is in remission and curative therapy was completed at least 5 years prior to screening.
- Planned or anticipated use of any prohibited medications and procedures during the study.
- Current pregnancy or breastfeeding.
- 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 team makes the final eligibility decision.
Where it is enrolling
- Bethesda, Maryland, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part, but confirm specifics with the study team.
How long does this study last?
The study runs about 10 months per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling all sexes, 12 years to 75 years. Healthy volunteers may be eligible. The study team makes the final eligibility decision.
Where is this trial taking place?
This study is enrolling now at Bethesda, Maryland, United States. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.