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.
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
Active, untreated bacterial, mycobacterial or fungal infection
Active herpes zoster infection
Currently requiring extracorporeal membrane oxygenation (ECMO)
Participant refusal to participate in the study
Single group
Emapalumab
DrugEmapalumab 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.
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.
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.
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
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.
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.
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.
New infections during treatment
Number of participants who experience at least one new infection during the study period, as identified through clinical assessment.
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University of Miami
Lead sponsor
Swedish Orphan Biovitrum
Collaborator
