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Randomized Stepped Wedge Study of Emapalumab in APECED Enteritis

NCT07202598 · Status: RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2025-12-11

No results posted yet for this study

Summary

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.

...

Conditions

  • Autoimmune Polyendocrinopathy Candidiasis Ectodermal Dystrophy Enteritis

Interventions

DRUG

Emapalumab

Emapalumab will be administered via IV infusion once a month. The initial dose of emapalumab is 3 mg/kg followed by a second dose of 3 mg/kg after 3 days. Subsequently, the dose is 3 mg/kg once a month for five additional doses.

Sponsors & Collaborators

  • National Institute of Allergy and Infectious Diseases (NIAID)

    lead NIH

Principal Investigators

  • Michail S Lionakis, M.D. · National Institute of Allergy and Infectious Diseases (NIAID)

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
2 Years
Max Age
75 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-11-12
Primary Completion
2031-03-31
Completion
2031-09-01
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07202598 on ClinicalTrials.gov