DermatomyositisPhase 2NCT07486869
Emapalumab MDA5 Rapidly Progressive Interstitial Lung Disease (RP-ILD) Study
A Phase 2 study testing Emapalumab in people with Dermatomyositis and Dermatomyositis Sine Myositis, run by University of Miami at 1 site in United States.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Dermatomyositis, Dermatomyositis Sine Myositis, Dermatomyositis With Myopathy
- What is being tested?
- Emapalumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteUnited States
- Study dates
- 23 Jul 2026to Jul 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
University of Miami Hospital and Clinics
Miami, Florida, 33136
Kelly Corbitt, D.O., 305-243-7545, KKC51@miami.edu
About this study
Official title: Emapalumab for the Treatment of Anti-MDA5 Antibody Positive Rapidly Progressive Interstitial Lung Disease
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
- Being tested
- Emapalumab
- Medical subject headings
- Dermatomyositis, Amyopathic dermatomyositis, Lung Diseases, Interstitial
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EmapalumabDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: Emapalumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 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
Measured: 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.
Measured: Baseline, 4 weeks, 12 weeks
Secondary outcomes (3)
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.
Measured: Baseline, 4 weeks, 12 weeks
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.
Measured: 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.
Measured: Baseline, 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Mar 2026
- Study start23 Jul 2026
- Primary completionJul 2027 (estimated)
- Study completionJul 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for DermatomyositisStudies of the same condition, open and under way.
- More trials run by University of MiamiThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Miami
- Sponsor type
- University, hospital or other
- Collaborators
- Swedish Orphan Biovitrum
- Responsible party
- Principal investigator, Kelly Corbitt, University of Miami
- Investigators
- Kelly Corbitt, D.O., Principal investigator, University of Miami
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07486869
- Sponsor's protocol ID
- 20241227
- Status last verified
- Aug 2026
- First submitted
- 16 Mar 2026
- Last updated
- 25 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Emapalumab for the Treatment of Anti-MDA5 Antibody Positive Rapidly Progressive Interstitial Lung Disease
- ClinicalTrials.gov ID
- NCT07486869
- Acronym / sponsor ID
- 20241227
- Conditions
- Dermatomyositis, Dermatomyositis Sine Myositis, Dermatomyositis With Myopathy, Dermatomyositis With Respiratory Involvement, Dermatomyositis With Organ Involvement, Interstitial Lung Disease
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 5 participants (estimated)
- Lead sponsor
- University of MiamiUniversity, hospital or other
- Collaborators
- Swedish Orphan Biovitrum
- Study start
- 23 Jul 2026 (actual)
- Primary completion
- Jul 2027 (estimated)
- Study completion
- Jul 2027 (estimated)
- First posted
- 23 Mar 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Emapalumab | Experimental | Drug: EmapalumabParticipants 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- 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
Key exclusion criteria (6 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| EmapalumabEmapalumab 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. | Drug | Emapalumab |
Primary outcome
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.
3 secondary outcomes
Change in Ferritin level
Time frame: Baseline, 4 weeks, 12 weeks
Change in MDA5 antibody level
Time frame: Baseline, 12 weeks
New infections during treatment
Time frame: Baseline, 12 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Aug 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Kelly Corbitt, D.O. principal investigator
University of Miami
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Emapalumab in people with Dermatomyositis and Dermatomyositis Sine Myositis, run by University of Miami at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 25 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Kelly Corbitt, D.O. (305-243-7545, KKC51@miami.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in oxygen requirement (time frame: Baseline, 4 weeks, 12 weeks). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Aug 2026.