Emapalumab MDA5 Rapidly Progressive Interstitial Lung Disease (RP-ILD) Study

Emapalumab for the Treatment of Anti-MDA5 Antibody Positive Rapidly Progressive Interstitial Lung Disease

This is a proof of concept study to determine if Emapalumab appears effective for the treatment of anti-MDA5 antibody positive rapidly progressive interstitial lung disease (MDA5 RP-ILD). Emapalumab is a medication that is currently used for a severe problem with the immune system, called macrophage activation syndrome, and this disease shares some similar features with MDA5 RP-ILD.

Study Overview

• Status: Not yet recruiting
• Conditions: Interstitial Lung Disease; Dermatomyositis; Interstitial Lung Disease Due to Connective Tissue Disease (Disorder); Dermatomyositis Sine Myositis; Dermatomyositis With Myopathy; Dermatomyositis With Respiratory Involvement; Dermatomyositis With Organ Involvement
• Intervention / Treatment: Drug: Emapalumab

• Study Type: Interventional
• Enrollment (Estimated): 5
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Kelly Corbitt, D.O.; Phone Number: 305-243-7545; Email: KKC51@miami.edu

Study Locations

• United States
  • Florida
    • Miami, Florida, United States, 33136
      • University of Miami Hospital and Clinics
      • Contact: Kelly Corbitt, D.O.; Phone Number: 305-243-7545; Email: KKC51@miami.edu

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Progressive interstitial lung disease as determined by at least 2/4 of the following:

  1. worsening respiratory symptoms;
  2. worsening or new oxygen requirement;
  3. worsening disease on CT chest;
  4. worsening Forced Vital Capacity (FVC1) or Diffusing Capacity for Carbon Monoxide (DLCO) on pulmonary function tests
• MDA5 antibodies present
• Elevated ferritin above the upper limit of normal (ULN)
• Participant at least 18 years old

Exclusion Criteria:

• Active, untreated bacterial, mycobacterial or fungal infection
• Active herpes zoster infection
• Currently requiring extracorporeal membrane oxygenation (ECMO)
• Participant refusal to participate in the study
• Pregnant women
• Prisoners

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Emapalumab; Participants will receive emapalumab administered intravenously at a dose of 6 mg/kg on Day 1, followed by 3 mg/kg every 3 days for 2 weeks, and then 3 mg/kg twice weekly for an additional 2 weeks. Total treatment duration is 12 weeks.	Drug: Emapalumab; Emapalumab administered intravenously according to the following dosing regimen: 6 mg/kg on Day 1, followed by 3 mg/kg every 3 days for 2 weeks, and then 3 mg/kg twice weekly for 2 weeks.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in oxygen requirement	Supplemental oxygen requirement assessed as the oxygen flow rate (reported in liters per minute [L/min]) while the participant is at rest and, when feasible, while the participant is ambulating during the 6-Minute Walk Test (6MWT). Values are compared with baseline to assess change over time.	Baseline, 4 weeks, 12 weeks
Diffusing capacity of the lungs for carbon monoxide (DLCO)	Pulmonary gas exchange capacity assessed using diffusing capacity of the lungs for carbon monoxide (DLCO) measured by standard pulmonary function testing. The outcome is expressed as percent change from baseline (%) in DLCO	Baseline, 12 weeks
Change in chest computed tomography (CT) consolidations	Pulmonary consolidations assessed on chest computed tomography (CT) scans. CT images are reviewed by a radiologist and pulmonologist, and percentage of lung affected by consolidations will be documented. Percentage change of affected lung will be evaluated.	Baseline, 4 weeks, 12 weeks
Forced Vital Capacity (FVC)	Pulmonary function assessed using forced vital capacity (FVC) measured by standard pulmonary function testing (spirometry). The outcome is expressed as percent change from baseline (%) in FVC.	Baseline, 12 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in Ferritin level	Serum ferritin concentration measured using standard clinical laboratory testing and reported in nanograms per milliliter (ng/mL). Values obtained at baseline and during post-baseline assessments are used to assess change from baseline in ferritin level.	Baseline, 4 weeks, 12 weeks
Change in MDA5 antibody level	Serum anti-melanoma differentiation-associated protein 5 (anti-MDA5) antibody level measured as a quantitative serum laboratory value using a validated clinical assay. Values obtained at baseline and during post-baseline assessments are used to assess change from baseline in serum anti-MDA5 antibody level.	Baseline, 12 weeks
New infections during treatment	Number of participants who experience at least one new infection during the study period, as identified through clinical assessment.	Baseline, 12 weeks

Collaborators and Investigators

• Sponsor: University of Miami
• Collaborators: Swedish Orphan Biovitrum AB
• Investigators: Principal Investigator: Kelly Corbitt, D.O., University of Miami

Study record dates

Study Major Dates

• Study Start (Estimated): May 1, 2026
• Primary Completion (Estimated): May 1, 2027
• Study Completion (Estimated): May 1, 2027

Study Registration Dates

• First Submitted: March 16, 2026
• First Submitted That Met QC Criteria: March 16, 2026
• First Posted (Actual): March 23, 2026

Study Record Updates

• Last Update Posted (Actual): April 9, 2026
• Last Update Submitted That Met QC Criteria: April 4, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Musculoskeletal Diseases; Nervous System Diseases; Muscular Diseases; Neuromuscular Diseases; Connective Tissue Diseases; Respiratory Tract Diseases; Lung Diseases; Skin Diseases; Myositis; Polymyositis; Skin and Connective Tissue Diseases; Lung Diseases, Interstitial; Dermatomyositis; Amyopathic dermatomyositis; Emapalumab
• Other Study ID Numbers: 20241227

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No