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. |
|
Recruiting Locations
University of Miami Hospital and Clinics
Miami, Florida 33136
Miami, Florida 33136
More Details
- Status
- Recruiting
- Sponsor
- University of Miami