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)
Conditions
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.
Interventions
DRUGEmapalumab
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.
Sponsors
University of Miami
Swedish Orphan Biovitrum AB
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in oxygen requirement | Baseline, 4 weeks, 12 weeks | 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 Expiratory Volume in 1 Second (FEV₁) | Baseline, 12 weeks | Pulmonary function assessed using forced expiratory volume in 1 second (FEV₁) measured by standard pulmonary function testing (spirometry). The outcome is expressed as percent change from baseline (%) in FEV₁. |
| Diffusing capacity of the lungs for carbon monoxide (DLCO) | Baseline, 12 weeks | 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 | Baseline, 4 weeks, 12 weeks | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in Ferritin level | Baseline, 4 weeks, 12 weeks | 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 | Baseline, 12 weeks | 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 | Baseline, 12 weeks | Number of participants who experience at least one new infection during the study period, as identified through clinical assessment. |
Countries
United States
Contacts
CONTACTKelly Corbitt, D.O.
PRINCIPAL_INVESTIGATORKelly Corbitt, D.O.
University of Miami
Outcome results
None listed