Dysautonomia International

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

Purpose

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)

Eligibility

Eligible Ages: Over 18 Years
Eligible Sex: All
Accepts Healthy Volunteers: No

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 Design

Phase: Phase 2
Study Type: Interventional
Allocation: N/A
Intervention Model: Single Group Assignment
Primary Purpose: Treatment
Masking: None (Open Label)

Arm Groups

Arm	Description	Assigned Intervention
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.

Recruiting Locations

University of Miami Hospital and Clinics
Miami, Florida 33136

Contact:
Kelly Corbitt, D.O.
305-243-7545
KKC51@miami.edu

More Details

Status: Recruiting
Sponsor: University of Miami

Study Contact

Kelly Corbitt, D.O.
305-243-7545
KKC51@miami.edu

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.