Emapalumab for Autoimmune Enteropathy

Overseen By Michail S Lionakis, M.D.

Age: Any Age

Sex: Any

Trial Phase: Phase 2

Sponsor: National Institute of Allergy and Infectious Diseases (NIAID)

Must be taking: Acyclovir, Valacyclovir, Entecavir

Must not be taking: Investigational drugs

Disqualifiers: HIV, Hepatitis B/C, Alcohol/drug abuse, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 4

Duration: 6 months

10 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests emapalumab, a drug designed to help people with enteritis, an inflammation of the small intestine caused by a rare condition known as APECED. The researchers will evaluate the effectiveness and safety of emapalumab in reducing the immune system's attack on the small intestine. Participants will receive the drug intravenously and attend several study visits over 18 months. This trial suits individuals diagnosed with APECED who have had enteritis for over six months and have not responded to other treatments. As a Phase 2 trial, this research measures how well the treatment works in an initial, smaller group of people, offering participants a chance to contribute to important medical advancements.

Do I have to stop taking my current medications for the trial?

The trial information does not specify if you need to stop taking your current medications. However, you cannot participate if you have used any investigational drug recently. It's best to discuss your specific medications with the trial team.

Is there any evidence suggesting that emapalumab is likely to be safe for humans?

Research has shown that emapalumab has been tested in people before and is already approved for treating primary hemophagocytic lymphohistiocytosis (HLH), a rare immune condition. In these earlier studies, 53% of patients experienced serious side effects, with common issues including infections and gastrointestinal bleeding.

Although these findings indicate significant side effects, emapalumab has undergone human trials, providing doctors with some understanding of its effects. This trial, in an early phase, aims to assess the safety of emapalumab for treating enteritis in APECED patients. Participants will receive close monitoring for any side effects. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Emapalumab is unique because it targets the interferon-gamma pathway, which plays a crucial role in autoimmune diseases like autoimmune enteropathy. Unlike standard treatments that often involve broad immunosuppression, such as steroids or other immunosuppressive drugs, emapalumab specifically inhibits interferon-gamma, potentially reducing side effects and improving precision in targeting the disease. Researchers are excited about this treatment because it offers a more targeted approach, which could lead to better management of symptoms and improved quality of life for patients with autoimmune enteropathy.

What evidence suggests that emapalumab might be an effective treatment for enteritis caused by APECED?

Research has shown that emapalumab may help treat conditions with an overactive immune system. In studies on other immune-related diseases, many patients responded well to the treatment. For example, 82.4% of patients noticed symptom improvement within eight weeks. Another study found a significant decrease in signs of inflammation among patients. This trial will evaluate emapalumab for autoimmune enteropathy. These results suggest that emapalumab might help calm the immune system in people with enteritis caused by APECED, possibly reducing symptoms and improving overall health. ^{2 6 7 8 9}

Who Is on the Research Team?

Michail S Lionakis, M.D.

Principal Investigator

National Institute of Allergy and Infectious Diseases (NIAID)

Are You a Good Fit for This Trial?

This trial is for individuals with Autoimmune Polyglandular Syndrome and Autoimmune Enteropathy, specifically APECED enteritis. Participants will receive the drug emapalumab through IV infusions over several months, with follow-up virtual and in-person visits to monitor their condition.

Inclusion Criteria

Proficient in written English

I am willing to take medication to prevent herpes reactivation.

I am between 2 and 75 years old.

Exclusion Criteria

I am expecting to undergo a major surgery during the study.

Known history of hypersensitivity to emapalumab

Current or recent use of any investigational drug (within 3 months or 5 half-lives, whichever is longer, prior to screening)

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive emapalumab in 2 intravenous infusions 3 days apart, followed by 5 additional monthly IV infusions

6 months

1 inpatient visit (up to 10 days), 5 monthly visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment via virtual visits every 2 months and an in-person visit at the end of the 6-month period

6 months

3 virtual visits, 1 in-person visit

What Are the Treatments Tested in This Trial?

Interventions

• Emapalumab

Trial Overview The study tests the effectiveness of emapalumab, an anti-interferon-gamma antibody, on gastrointestinal symptoms in patients with APECED enteritis. It's a phase 2 trial where participants start treatment at different times and are monitored for changes in their symptoms and overall health.

How Is the Trial Designed?

Group I: Group 4 Experimental Treatment 1 Intervention

6 month observation period prior to start of study drug.

Group II: Group 3 Experimental Treatment 1 Intervention

4 month observation period prior to start of study drug.

Group III: Group 2 Experimental Treatment 1 Intervention

2 month observation period prior to start of study drug.

Group IV: Group 1 Experimental Treatment 1 Intervention

No observation period prior to start of study drug.

Who Is Running the Clinical Trial?

National Institute of Allergy and Infectious Diseases (NIAID)

Lead Sponsor

Trials

3,361

Recruited

5,516,000+

Citations

1. withpower.com withpower.com/trial/phase-2-polyendocrinopathies-autoimmune-9-2025-fe3d8

Emapalumab for Autoimmune Enteropathy It's a phase 2 trial where participants start treatment at different times and are monitored for changes in their symptoms and overall health.

2. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/38429096/

Real-world treatment patterns and outcomes in patients ... Emapalumab treatment resulted in normalization of key laboratory parameters, including chemokine ligand 9 (24/33, 72.7%), ferritin (20/45, 44.4 ...

3. sobi.com sobi.com/en/press-releases/new-data-emapalumab-treatment-macrophage-activation-syndrome-be-presented-acr-conference-2275523

New data for emapalumab in the treatment of macrophage ... Overall response (OR) by week 8: 32 patients (82.4%) achieved an overall response by week 8. · Clinical MAS remission: · Survival rate: ...

4. frontiersin.org frontiersin.org/journals/immunology/articles/10.3389/fimmu.2020.608492/full

Novel Therapeutic Approaches to Familial HLH ... Among previously treated patients, 26% achieved CR, 30% PR, and 7% had improvement in measures of hemophagocytic lymphohistiocytosis, while 37% had no response.

5. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10314091/

Efficacy and safety of emapalumab in macrophage ... By week 8, 13 patients (93%) achieved MAS remission at a median time of 25 days after emapalumab initiation, the earliest at day 9 (figure 2). At week 8, 2 of ...

6. gamifant.com gamifant.com/phlh/safety

Safety Data | Gamifant® (emapalumab-lzsg) For HCPs Serious adverse reactions were reported in 53% of patients. The most common serious adverse reactions (≥3%) included infections, gastrointestinal hemorrhage ...

7. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC7793976/

Novel Therapeutic Approaches to Familial HLH (Emapalumab ... Available data support the use of emapalumab for treatment of pHLH patients with refractory, recurrent or progressive disease, or intolerance to conventional ...

8. accessdata.fda.gov accessdata.fda.gov/drugsatfda_docs/label/2018/761107lbl.pdf

GAMIFANT (emapalumab-lzsg) injection, - accessdata.fda.gov The safety data described in this section reflect exposure to GAMIFANT in which 34 patients with untreated primary HLH and previously treated patients with ...

9. clinicaltrials.gov clinicaltrials.gov/study/NCT05001737?term=emapalumab&rank=4

Evaluate Efficacy, Safety and Tolerability, PK and PD of ... A group of independent scientists who monitor the safety and scientific integrity of a clinical trial. The DMC can recommend to the sponsor that the trial be ...