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| Name | Class |
|---|---|
| Swedish Orphan Biovitrum | INDUSTRY |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| 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. |
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | Baseline, 4 weeks, 12 weeks |
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Inclusion Criteria:
Progressive interstitial lung disease as determined by at least 2/4 of the following:
MDA5 antibodies present
Elevated ferritin above the upper limit of normal (ULN)
Participant at least 18 years old
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Kelly Corbitt, D.O. | Contact | 305-243-7545 | KKC51@miami.edu |
| Name | Affiliation | Role |
|---|---|---|
| Kelly Corbitt, D.O. | University of Miami | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Miami Hospital and Clinics | Miami | Florida | 33136 | United States |
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| 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. |
| Baseline, 12 weeks |
| New infections during treatment | Number of participants who experience at least one new infection during the study period, as identified through clinical assessment. | Baseline, 12 weeks |
| ID | Term |
|---|---|
| D003882 | Dermatomyositis |
| C538250 | Amyopathic dermatomyositis |
| D017563 | Lung Diseases, Interstitial |
| ID | Term |
|---|---|
| D017285 | Polymyositis |
| D009220 | Myositis |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D012871 | Skin Diseases |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
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| ID | Term |
|---|---|
| C000644327 | Emapalumab |
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