Clinical trialNCT07202598RecruitingPhase 2Interventional

Randomized Stepped Wedge Study of Emapalumab in APECED Enteritis

SponsorNational Institute of Allergy and Infectious Diseases (NIAID)Enrollment30Autoimmune Polyendocrinopathy Candidiasis Ectodermal Dystrophy Enteritis

View the registry record on ClinicalTrials.gov

Summary

The lay summary the sponsor registered on ClinicalTrials.gov.

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. Objective: To test a drug (emapalumab) in people with enteritis caused by APECED. Eligibility: People aged 2 to 75 years with APECED and enteritis. They must also be enrolled in protocol 11-I-0187. Design: Participants will have 10-13 study visits in an 18-month period. Participants will be screened. They will have a physical exam with blood tests. These tests will be repeated at every study visit. They will have a test of their heart function. This will be at screening and prior to drug administration. Other tests are optional: Participants may have imaging exams and a test of lung function. They may have an endoscopy, which is an exam of their digestive tract. Participants may provide samples of urine, stool, nail clippings, saliva, vaginal fluid, or skin. Photos may be taken of their skin or scalp. These tests may be repeated at some visits. Emapalumab is given through a tube attached to a needle inserted into a vein. All participants will receive 7 doses: 2 on their first study visit; then 1 each at 30-day intervals. Some participants will have an observation period before they begin taking the drug; in those situations, they will either be seen in person or via video visit every 2 months before starting emapalumab to see how their symptoms change over time. Participants will have a follow-up visit 1 month after their last dose. Then they will have 2 telehealth visits at 30-day intervals. They will have a final clinic visit 1 year after their first dose. ...

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Investigators

The overall officials registered on this study. Names that resolve to a FindMyKOL profile link to it; the rest are shown as registered.

Michail S Lionakis, M.D.
National Institute of Allergy and Infectious Diseases (NIAID)
Principal Investigator
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A profile link is shown only when the registered name resolves to a person record with a confident, namesake-checked match; ambiguous names stay unlinked.

Sites

1 registered facility, with the per-site recruitment status where the registry publishes one. ClinicalTrials.gov does not publish per-site enrollment counts.

United States

National Institutes of Health Clinical Center
Bethesda, Maryland, United States
Recruiting

Site investigator names are published by ClinicalTrials.gov only while a study recruits, so completed studies list sites without them.

Drugs studied

The drug and biological interventions registered on this study, normalized to their RxNorm ingredient where possible.

Emapalumab →

Linked drugs open the directory filtered to KOLs working with them.clinicaltrials.gov ↗

Registration & key dates

Registry attributes as recorded on the ClinicalTrials.gov study record.

Start: 12 Nov 2025
Primary completion: 31 Mar 2031
Completion: 1 Sept 2031
Last update posted: 18 Aug 2026
Registered enrollment: 30
Registry id: NCT07202598

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Source: ClinicalTrials.gov, via the CTTI AACT database, data as of 24 Aug 2026. Registry facts are shown as registered by the study sponsor; FindMyKOL does not interpret them.