Pulmonary SarcoidosisPhase 3NCT05415137

Efficacy and Safety of Intravenous Efzofitimod in Patients With Pulmonary Sarcoidosis

A Phase 3 study testing Efzofitimod 3 mg/kg and Efzofitimod 5 mg/kg in people with Pulmonary Sarcoidosis, run by aTyr Pharma, Inc. at 91 sites in 9 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

Search all trials

Trial summary

Who is it for?: Ages 18 years and older
Conditions the study is for: Pulmonary Sarcoidosis
What is being tested?: Efzofitimod 3 mg/kg and Efzofitimod 5 mg/kg
Type of treatment: Injection or infusion
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 268 participants
Where: 91 sitesin 9 countries
Study dates: 15 Sep 2022to 14 Jul 2025
Phase: Phase 3
Are there results?: None posted in the registry

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

Documented history of pulmonary sarcoidosis for at least 6 months, supported by the following evidence: documented histologically proven diagnosis of sarcoidosis by tissue biopsy and documented evidence of parenchymal lung involvement by historical radiological evidence
Evidence of symptomatic pulmonary sarcoidosis, as demonstrated by the following criteria: Modified Medical Research Council (MRC) dyspnea scale grade of at least 1 and KSQ-Lung score ≤70
Patients must be receiving treatment with OCS of ≥ 3 months at Day 1 with a starting dose between ≥ 7.5 and ≤ 25 mg/day ≥ 4 weeks prior to Day 1.
Body weight ≥ 40 kg and < 160 kg

Who cannot join

Treatment with > 1 immunosuppressant therapy
Treatment with biological immunomodulators, such as tumor necrosis factor-alpha (TNF-α) inhibitors or antifibrotics or interleukin inhibitors
Likelihood of significant pulmonary fibrosis as shown by any 1 or more of the following: High resolution CT fibrosis > 20% within the last 12 months; FVC percent predicted (FVCPP) < 50% and KSQ-Lung score < 30
In the opinion of the investigator, clinically significant pulmonary hypertension
Patients with active cardiac, neuro, or renal sarcoidosis requiring organ-specific therapy in the past 2 years
Patients with cutaneous or ocular sarcoidosis, which in the opinion of the Investigator, are at risk for exacerbation, necessitating OCS rescue or other systemic therapy

Show all 10Show fewer

History of Addisonian symptoms that precluded previous OCS taper attempts
Is an active, heavy smoker of tobacco/nicotine-containing products
History of anti-synthetase syndrome or Jo-1 positive at Screening
Patients with active tuberculosis or those currently undergoing treatment for tuberculosis

Trial locations and contacts

91 sites in 9 countries.

Country

City, postcode or hospital

91 sites in 9 countries

United States39 sites

aTyr Investigative Site
Ada, Michigan, 49301
aTyr Investigative Site
Albany, New York, 12208
aTyr Investigative Site
Atlanta, Georgia, 30308
aTyr Investigative Site
Baltimore, Maryland, 21224
aTyr Investigative Site
Birmingham, Alabama, 35294
aTyr Investigative Site
Boston, Massachusetts, 02114
aTyr Investigative Site
Charleston, South Carolina, 29425-0630
aTyr Investigative Site
Chicago, Illinois, 60611
aTyr Investigative Site
Chicago, Illinois, 60612
aTyr Investigative Site
Cincinnati, Ohio, 45219
aTyr Investigative Site
Cleveland, Ohio, 44195
aTyr Investigative Site
Dallas, Texas, 75246
aTyr Investigative Site
Dallas, Texas, 75390
aTyr Investigative Site
Denver, Colorado, 80206
aTyr Investigative Site
Detroit, Michigan, 48202
aTyr Investigative Site
Dickson, Tennessee, 37055
aTyr Investigative Site
Durham, North Carolina, 27710
aTyr Investigative Site
Falls Church, Virginia, 22042
aTyr Investigative Site
Gainesville, Florida, 32610
aTyr Investigative Site
Greenville, North Carolina, 27834
aTyr Investigative Site
Houston, Texas, 77030
aTyr Investigative Site
Jackson, Mississippi, 39216
aTyr Investigative Site
Los Angeles, California, 90033
aTyr Investigative Site
Louisville, Kentucky, 40202
aTyr Investigative Site
Middletown, New York, 10941
aTyr Investigative Site
Minneapolis, Minnesota, 55455
aTyr Investigative Site
New Orleans, Louisiana, 70115
aTyr Investigative Site
Oklahoma City, Oklahoma, 73104
aTyr Investigative Site
Philadelphia, Pennsylvania, 19140
aTyr Investigative Site
Phoenix, Arizona, 85027
aTyr Investigative Site
Portland, Oregon, 97220
aTyr Investigative Site
Richmond, Virginia, 23298
aTyr Investigative Site
Rochester, Minnesota, 55905
aTyr Investigative Site
Sacramento, California, 95817
aTyr Investigative Site
Salt Lake City, Utah, 84132
aTyr Investigative Site
St Louis, Missouri, 63110
aTyr Investigative Site
Tampa, Florida, 33606
aTyr Investigative Site
Washington D.C., District of Columbia, 20060
aTyr Investigative Site
Weston, Florida, 33331

Other trials in United States

Japan16 sites

Kyorin Investigative Site
Aomori, 036-8563
Kyorin Investigative Site
Fukuoka, 814-0180
Kyorin Investigative Site
Fukushima, 960-1295
Kyorin Investigative Site
Hokkaido, 060-0033
Kyorin Investigative Site
Hokkaido, 060-8648
Kyorin Investigative Site
Hyōgo, 653-0013
Kyorin Investigative Site
Kanagawa, 236-0004
Kyorin Investigative Site
Kanagawa, 236-0051
aTyr Investigative Site
Kumamoto, 860-8556
Kyorin Investigative Site
Miyagi, 980-8574
Kyorin Investigative Site
Okayama, 700-8558
Kyorin Investigative Site
Osaka, 591-8025
Kyorin Investigative Site
Shimane, 693-8501
Kyorin Investigative Site
Shizuoka, 431-3192
Kyorin Investigative Site
Tokyo, 150-8935
Kyorin Investigative Site
Tokyo, 151-8528

Other trials in Japan

Italy9 sites

aTyr Investigative Site
Catania, 95124
aTyr Investigative Site
Florence, 50134
aTyr Investigative Site
Forlì, 47121
aTyr Investigative Site
Milan, 20123
aTyr Investigative Site
Naples, 80131
aTyr Investigative Site
Padua, 35128
aTyr Investigative Site
Rome, 00168
aTyr Investigative Site
Siena, 53100
aTyr Investigative Site
Trieste, 34149

Other trials in Italy

Spain6 sites

aTyr Investigative Site
Barcelona, 08036
aTyr Investigative Site
Barcelona, 08907
aTyr Investigative Site
Madrid, 28006
aTyr Investigative Site
Madrid, 28031
aTyr Investigative Site
Santander, 39008
aTyr Investigative Site
Valencia, 46010

Other trials in Spain

United Kingdom6 sites

aTyr Investigative Site
Birmingham, B15 2TH
aTyr Investigative Site
Cambridge, CB233RE
aTyr Investigative Site
Cottingham, HU16 5JQ
aTyr Investigative Site
Coventry, CV2 2DX
aTyr Investigative Site
London, SE59RS
aTyr Investigative Site
London, SW3 6NP

Other trials in United Kingdom

Brazil5 sites

aTyr Investigative Site
Porto Alegre, Rio Grande do Sul, 90020-090
aTyr Investigative Site
Salvador, Estado de Bahia, 40220-005
aTyr Investigative Site
São Bernardo do Campo, 09780-000
aTyr Investigative Site
São Paulo, 04039-901
aTyr Investigative Site
São Paulo, 05403-900

Other trials in Brazil

France5 sites

aTyr Investigative Site
Bobigny, 93000
aTyr Investigative Site
La Tronche, 38700
aTyr Investigative Site
Montpellier, 34295
aTyr Investigative Site
Paris, 75015
aTyr Investigative Site
Toulouse, 31000

Other trials in France

Germany4 sites

aTyr Investigative Site
Essen, 45122
aTyr Investigative Site
Freiburg im Breisgau, 79106
aTyr Investigative Site
Hanover, 30625
aTyr Investigative Site
Heidelberg, 69126

Other trials in Germany

Netherlands1 site

aTyr Investigative Site
Nieuwegein, 3435CM

Other trials in Netherlands

About this study

Official title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Intravenous Efzofitimod in Patients With Pulmonary Sarcoidosis

This is a multicenter, randomized, double-blind, placebo-controlled, study comparing the efficacy and safety of intravenous (IV) efzofitimod 3 mg/kg and 5 mg/kg versus placebo after 48 weeks of treatment. This study will enroll adults with histologically confirmed pulmonary sarcoidosis receiving stable treatment with oral corticosteroid (OCS), with or without immunosuppressant therapy.

Conditions: Pulmonary Sarcoidosis
Being tested: Efzofitimod 3 mg/kg, Efzofitimod 5 mg/kg, Placebo
Keywords: Pulmonary Sarcoidosis, Sarcoidosis, Granuloma, Inflammation, Lymphoproliferative Disorders, Interstitial Lung Disease, Neuropilin-2, Steroids, Oral corticosteroids, Immunomodulatory, tRNA Synthetase, ATYR1923, KRP-R120, Efzofitimod, Fibrosis
Medical subject headings: Sarcoidosis, Pulmonary, Sarcoidosis, Granuloma, Inflammation, Lymphoproliferative Disorders, Lung Diseases, Interstitial, Fibrosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Efzofitimod 3 mg/kgDrug
Also called ATYR1923, KRP-R120
Efzofitimod IV infusion every 4 weeks for a total of 12 doses
Efzofitimod 5 mg/kgDrug
Also called ATYR1923, KRP-R120
Efzofitimod IV infusion every 4 weeks for a total of 12 doses
PlaceboDrug
Placebo IV infusion every 4 weeks for a total of 12 doses

Study groups in NCT05415137
Study group	Role	What they receive
Efzofitimod 3 mg/kg	Experimental	Not described Receives: Drug: Efzofitimod 3 mg/kg
Efzofitimod 5 mg/kg	Experimental	Not described Receives: Drug: Efzofitimod 5 mg/kg
Placebo	Placebo comparator	Not described Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

Change from baseline in mean daily oral corticosteroid (OCS) dose at Week 48
Measured: Baseline to Week 48

Secondary outcomes (3)

Change from baseline in KSQ-Lung score at Week 48
Measured: Baseline to Week 48
Steroid withdrawal rate
Measured: Baseline to Week 48
Change from baseline in absolute value of FVC at Week 48
Measured: Baseline to Week 48

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

15 Sep 202214 Jul 2025

First posted10 Jun 2022
Study start15 Sep 2022
Primary completion25 Jun 2025
Study completion14 Jul 2025

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Publications (2)Papers the sponsor has linked to this study.
Other trials for Pulmonary SarcoidosisStudies of the same condition, open and under way.
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 3
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind
Who is masked: Participant, Care provider, Investigator, Outcomes assessor
Enrolment: 268 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT05415137
Sponsor's protocol ID: ATYR1923-C-004
Status last verified: Jun 2026
First submitted: 8 Jun 2022
Last updated: 4 Jun 2026
Results posted: No
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: Undecided

Publications

Culver DA, Bonella F, Gupta R, Spagnolo P, Veltkamp M, Cameli P, Restrepo CI, Frye BC, Kameda M, Cifrian-Martinez J, Ali H, Aryal S, Carmona EM, Nunes H, Obi ON, Hart SP, Sellares J, Azuma A, Grutters JC, Kinnersley N, Niranjan V, Baughman RP. Efzofitimod versus placebo in patients with pulmonary sarcoidosis (EFZO-FIT): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet Respir Med. 2026 Oct 26:S2213-2600(26)00283-3. doi: 10.1016/S2213-2600(26)00283-3. Online ahead of print.
PubMed 42933455
Forster S, Chong YE, Siefker D, Becker Y, Bao R, Escobedo E, Qing Y, Rauch K, Burman L, Burkart C, Kainz P, Cubitt A, Muders M, Nangle LA. Development and Characterization of a Novel Neuropilin-2 Antibody for Immunohistochemical Staining of Cancer and Sarcoidosis Tissue Samples. Monoclon Antib Immunodiagn Immunother. 2023 Oct;42(5):157-165. doi: 10.1089/mab.2023.0007.
PubMed 37902990

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingObservational study
Role of Genetic Factors in the Development of Lung Disease
Cystic Fibrosis, Pulmonary Fibrosis
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An Efficacy Study of XTMAB-16 in Patients With Pulmonary Sarcoidosis With or Without Extrapulmonary Manifestations
Pulmonary Sarcoidosis
Not yet recruitingPhase 2
LAM-001 for the Treatment of Sarcoidosis Associated Pulmonary Hypertension (SAPH)
Sarcoidosis Associated Pulmonary Hypertension
Not yet recruitingPhase 2/3
Evaluation of the Efficacy and Safety of Compound Betamethasone in Pulmonary Sarcoidosis
Sarcoidosis, Pulmonary
Not yet recruitingPhase 2
A Clinical Trial of Tulisokibart (MK-7240) in Participants With Pulmonary Sarcoidosis (MK-7240-016)
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All Pulmonary Sarcoidosis trials

Study at a glance

From the registry record, last updated by the sponsor on 4 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Intravenous Efzofitimod in Patients With Pulmonary Sarcoidosis
ClinicalTrials.gov ID: NCT05415137
Acronym / sponsor ID: ATYR1923-C-004
Conditions: Pulmonary Sarcoidosis
Keywords: Pulmonary Sarcoidosis, Sarcoidosis, Granuloma, Inflammation, Lymphoproliferative Disorders, Interstitial Lung Disease, Neuropilin-2, Steroids
Phase: Phase 3
Study type: Interventional
Recruitment status: Completed
Enrolment: 268 participants (actual)
Lead sponsor: aTyr Pharma, Inc.Industry (pharma, biotech, devices)
Collaborators: Kyorin Pharmaceutical Co.,Ltd
Study start: 15 Sep 2022 (actual)
Primary completion: 25 Jun 2025 (actual)
Study completion: 14 Jul 2025 (actual)
First posted: 10 Jun 2022 (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.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind (participant, care provider, investigator, outcomes assessor)
Number of arms: 3

Arm	Type	Receives
Efzofitimod 3 mg/kg	Experimental	Drug: Efzofitimod 3 mg/kg
Efzofitimod 5 mg/kg	Experimental	Drug: Efzofitimod 5 mg/kg
Placebo	Placebo comparator	Drug: Placebo

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (4 listed)

Documented history of pulmonary sarcoidosis for at least 6 months, supported by the following evidence: documented histologically proven diagnosis of sarcoidosis by tissue biopsy and documented evidence of parenchymal lung involvement by historical radiological evidence
Evidence of symptomatic pulmonary sarcoidosis, as demonstrated by the following criteria: Modified Medical Research Council (MRC) dyspnea scale grade of at least 1 and KSQ-Lung score ≤70
Patients must be receiving treatment with OCS of ≥ 3 months at Day 1 with a starting dose between ≥ 7.5 and ≤ 25 mg/day ≥ 4 weeks prior to Day 1.
Body weight ≥ 40 kg and < 160 kg

Key exclusion criteria (10 listed)

Treatment with > 1 immunosuppressant therapy
Treatment with biological immunomodulators, such as tumor necrosis factor-alpha (TNF-α) inhibitors or antifibrotics or interleukin inhibitors
Likelihood of significant pulmonary fibrosis as shown by any 1 or more of the following: High resolution CT fibrosis > 20% within the last 12 months; FVC percent predicted (FVCPP) < 50% and KSQ-Lung score < 30
In the opinion of the investigator, clinically significant pulmonary hypertension
Patients with active cardiac, neuro, or renal sarcoidosis requiring organ-specific therapy in the past 2 years

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
Efzofitimod 3 mg/kgAlso: ATYR1923, KRP-R120Efzofitimod IV infusion every 4 weeks for a total of 12 doses	Drug	Efzofitimod 3 mg/kg
Efzofitimod 5 mg/kgAlso: ATYR1923, KRP-R120Efzofitimod IV infusion every 4 weeks for a total of 12 doses	Drug	Efzofitimod 5 mg/kg
PlaceboPlacebo IV infusion every 4 weeks for a total of 12 doses	Drug	Placebo

Primary outcome

Change from baseline in mean daily oral corticosteroid (OCS) dose at Week 48
Time frame: Baseline to Week 48

3 secondary outcomes

Change from baseline in KSQ-Lung score at Week 48
Time frame: Baseline to Week 48
Steroid withdrawal rate
Time frame: Baseline to Week 48
Change from baseline in absolute value of FVC at Week 48
Time frame: Baseline to Week 48

Results and safety

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 4 Jun 2026.
Derived from this study: PubMed 42933455Culver DA, Bonella F, Gupta R, Spagnolo P, Veltkamp M, Cameli P, Restrepo CI, Frye BC, Kameda M, Cifrian-Martinez J, Ali H, Aryal S, Carmona EM, Nunes H, Obi ON, Hart SP, Sellares J, Azuma A, Grutters JC, Kinnersley N, Niranjan V, Baughman RP. Efzofitimod versus placebo in patients with pulmonary sarcoidosis (EFZO-FIT): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet Respir Med. 2026 Oct 26:S2213-2600(26)00283-3. doi: 10.1016/S2213-2600(26)00283-3. Online ahead of print.
Derived from this study: PubMed 37902990Forster S, Chong YE, Siefker D, Becker Y, Bao R, Escobedo E, Qing Y, Rauch K, Burman L, Burkart C, Kainz P, Cubitt A, Muders M, Nangle LA. Development and Characterization of a Novel Neuropilin-2 Antibody for Immunohistochemical Staining of Cancer and Sarcoidosis Tissue Samples. Monoclon Antib Immunodiagn Immunother. 2023 Oct;42(5):157-165. doi: 10.1089/mab.2023.0007.
Individual participant dataSharing planned: Undecided

Related trials for Pulmonary Sarcoidosis

RecruitingObservational study
Role of Genetic Factors in the Development of Lung Disease
Cystic Fibrosis, Pulmonary Fibrosis
Not yet recruitingPhase 2
An Efficacy Study of XTMAB-16 in Patients With Pulmonary Sarcoidosis With or Without Extrapulmonary Manifestations
Pulmonary Sarcoidosis
Not yet recruitingPhase 2
LAM-001 for the Treatment of Sarcoidosis Associated Pulmonary Hypertension (SAPH)
Sarcoidosis Associated Pulmonary Hypertension
Not yet recruitingPhase 2/3
Evaluation of the Efficacy and Safety of Compound Betamethasone in Pulmonary Sarcoidosis
Sarcoidosis, Pulmonary
Not yet recruitingPhase 2
A Clinical Trial of Tulisokibart (MK-7240) in Participants With Pulmonary Sarcoidosis (MK-7240-016)
Pulmonary Sarcoidosis

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. This study is not recruiting at the moment.

Lisa Carey study director

aTyr Pharma, Inc.

91 sites in 9 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Efzofitimod 3 mg/kg and Efzofitimod 5 mg/kg in people with Pulmonary Sarcoidosis, run by aTyr Pharma, Inc. at 91 sites in 9 countries.

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 4 inclusion and 10 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: finished, as of the sponsor's last update on 4 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 91 sites in United States, Japan, Italy, Spain, United Kingdom and 4 more countries. 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. Some participants in this study may receive a placebo. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change from baseline in mean daily oral corticosteroid (OCS) dose at Week 48 (time frame: Baseline to Week 48). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05415137, which also lists 2 linked publications.

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 4 Jun 2026.