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

NCT07486869 | Not Yet Recruiting Phase 2 FDA Drug

• 1 other identifier: 20241227
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Dermatomyositis; Dermatomyositis Sine Myositis; Dermatomyositis With Myopathy; Dermatomyositis With Respiratory Involvement; Dermatomyositis With Organ Involvement; Interstitial Lung Disease; Interstitial Lung Disease Due to Connective Tissue Disease (Disorder)

Outcome Measures

Primary Outcomes (4)

• 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

• 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

• 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

Secondary Outcomes (3)

• Change in Ferritin level

  Baseline, 4 weeks, 12 weeks

• Change in MDA5 antibody level

  Baseline, 12 weeks

• New infections during treatment

  Baseline, 12 weeks

Study Arms (1)

Emapalumab

EXPERIMENTAL

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

Interventions

Emapalumab DRUG

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.

Emapalumab

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Progressive interstitial lung disease as determined by at least 2/4 of the following:
• worsening respiratory symptoms;
• worsening or new oxygen requirement;
• worsening disease on CT chest;
• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• University of Miami: lead
• Swedish Orphan Biovitrum: collaborator

Study Sites (1)

University of Miami Hospital and Clinics

Miami, Florida, 33136, United States

Location

MeSH Terms

Conditions

Dermatomyositis; Amyopathic dermatomyositis; Lung Diseases, Interstitial

Interventions

Emapalumab

Condition Hierarchy (Ancestors)

Polymyositis; Myositis; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases; Lung Diseases; Respiratory Tract Diseases

Study Officials

• Kelly Corbitt, D.O.

  University of Miami

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Kelly Corbitt, D.O.

CONTACT

305-243-7545; KKC51@miami.edu

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Assistant Professor of Clinical Medicine

Study Record Dates

• First Submitted: March 16, 2026
• First Posted: March 23, 2026
• Study Start: July 1, 2026
• Primary Completion (Estimated): May 1, 2027
• Study Completion (Estimated): May 1, 2027
• Last Updated: June 18, 2026

Record last verified: 2026-06

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)