NCT07486869 · University of Miami
Emapalumab MDA5 Rapidly Progressive Interstitial Lung Disease (RP-ILD) Study
What this study is about
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.
View original scientific description
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
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.
Primary outcome measures
Change in oxygen requirement
Time frame: 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 Vital Capacity (FVC)
Time frame: Baseline, 12 weeks
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)
Time frame: 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
Time frame: 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.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- Progressive interstitial lung disease as determined by at least 2/4 of the following:
- 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
- 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
Where
- Miami, Florida
Collaborators
Swedish Orphan Biovitrum
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Aug 25, 2026 · Source of record for eligibility and locations