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New treatment option for Autoimmune Polyendocrinopathy Candidiasis Ectodermal Dystrophy...
Official title Randomized Stepped Wedge Study of Emapalumab in APECED Enteritis
ClinicalTrials.gov ID: NCT07202598
What this study is testing
What is Emapalumab?
Emapalumab is an investigational medicine, given as a monthly infusion into a vein, being studied as a potential treatment for autoimmune polyendocrinopathy candidiasis ectodermal dystrophy enteritis.
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
- Background: Autoimmune polyendocrinopathy-candidiasis-ectodermal dystrophy (APECED), also known as Autoimmune polyendocrine syndrome type-1 (APS-1), is a disease that causes the immune system to attack parts of a person s body. In some people, APECED attacks the small intestine; this causes an illness called enteritis.
- Phase 2: a mid-size study of how well it works
- Which group you join is decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 2 to 75
You may be able to join if
- 1. Participant must be able to understand and provide informed consent.
- Aged \>= 2 to \<= 75 years.
- Currently co-enrolled on NIH protocol 11-I-0187, "The Natural History and Pathogenesis of Human Fungal Infections."
- Patients with APECED (genetic or clinical diagnosis) and enteritis (with APECED ES \> 50 at screening).
- Duration of enteritis greater than 6 months.
You likely can't join if
- Known history of hypersensitivity to emapalumab.
- History of active intestinal disease other than enteritis such as inflammatory bowel disease.
- 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.
- History of alcohol or drug abuse within 6 months prior to screening.
- Presence of one or more of the following clinically significant laboratory abnormalities:
See the full eligibility criteria
- 1. Participant must be able to understand and provide informed consent.
- Aged \>= 2 to \<= 75 years.
- Currently co-enrolled on NIH protocol 11-I-0187, "The Natural History and Pathogenesis of Human Fungal Infections."
- Patients with APECED (genetic or clinical diagnosis) and enteritis (with APECED ES \> 50 at screening).
- Duration of enteritis greater than 6 months.
- Is naive or unresponsive to other treatments for enteritis.
- Willingness to use acyclovir or valacyclovir prophylaxis for the prevention of herpes viral reactivation.
- Willingness to use entecavir prophylaxis against hepatitis B virus reactivation, if applicable.
- Vaccinations should be up to date in agreement with current Centers for Disease Control and Prevention immunization guidelines prior to start of emapalumab.
- Proficient in written English.
- 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...
- 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 (IUD); intrauterine hormone-releasing system.
- Two barrier methods (e.g., condom with spermicide, diaphragm with spermicide, or cervical cap and spermicide). Internal and external condoms may not be used together.
- 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...
- Known history of hypersensitivity to emapalumab.
- History of active intestinal disease other than enteritis such as inflammatory bowel disease.
- 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.
- 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.
- Serum creatinine \>= 2 times ULN.
- Planned or anticipated major surgical procedure during the study.
- Plans to receive any live or live attenuated vaccines within 1 month of the anticipated first dose of emapalumab.
- Known or suspected immunodeficiency disorder besides APECED.
- History of untreated invasive opportunistic infections (e.g., 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 (e.g., chronic pyelonephritis, osteomyelitis).
- 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's taking place
- Bethesda, Maryland, United States
Compensation & support
Compensation mentioned.
ClinicalTrials.gov doesn't provide a reliable structured field for payment or travel support - confirm details with the study team.
Questions & answers
Do participants get paid in this trial?
This study's listing includes signals that participants may be compensated or receive a stipend. Amounts vary and are set by the study team - confirm the details with them.
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 - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 2 years to 75 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Bethesda, Maryland, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.