Efficacy and Safety of Intravenous Efzofitimod in Patients With Pulmonary Sarcoidosis

NCT ID: NCT05415137

Last Updated: 2025-05-20

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE3

Total Enrollment

268 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-09-15

Study Completion Date

2025-08-31

Brief Summary

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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.

Detailed Description

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Conditions

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Pulmonary Sarcoidosis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Efzofitimod 3 mg/kg

Group Type EXPERIMENTAL

Efzofitimod 3 mg/kg

Intervention Type DRUG

EfzofitimodIV infusion every 4 weeks for a total of 12 doses

Efzofitimod 5 mg/kg

Group Type EXPERIMENTAL

Efzofitimod 5 mg/kg

Intervention Type DRUG

EfzofitimodIV infusion every 4 weeks for a total of 12 doses

Placebo

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Placebo IV infusion every 4 weeks for a total of 12 doses

Interventions

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Efzofitimod 3 mg/kg

EfzofitimodIV infusion every 4 weeks for a total of 12 doses

Intervention Type DRUG

Efzofitimod 5 mg/kg

EfzofitimodIV infusion every 4 weeks for a total of 12 doses

Intervention Type DRUG

Placebo

Placebo IV infusion every 4 weeks for a total of 12 doses

Intervention Type DRUG

Other Intervention Names

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ATYR1923 KRP-R120 ATYR1923 KRP-R120

Eligibility Criteria

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Inclusion Criteria

* 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

Exclusion Criteria

* 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
* 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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Kyorin Pharmaceutical Co.,Ltd

INDUSTRY

Sponsor Role collaborator

aTyr Pharma, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Lisa Carey

Role: STUDY_DIRECTOR

aTyr Pharma, Inc.

Locations

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aTyr Investigative Site

Birmingham, Alabama, United States

Site Status

aTyr Investigative Site

Phoenix, Arizona, United States

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aTyr Investigative Site

Los Angeles, California, United States

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aTyr Investigative Site

Sacramento, California, United States

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aTyr Investigative Site

Denver, Colorado, United States

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aTyr Investigative Site

Washington D.C., District of Columbia, United States

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aTyr Investigative Site

Gainesville, Florida, United States

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aTyr Investigative Site

Tampa, Florida, United States

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aTyr Investigative Site

Weston, Florida, United States

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aTyr Investigative Site

Atlanta, Georgia, United States

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aTyr Investigative Site

Chicago, Illinois, United States

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aTyr Investigative Site

Chicago, Illinois, United States

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aTyr Investigative Site

Louisville, Kentucky, United States

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aTyr Investigative Site

New Orleans, Louisiana, United States

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aTyr Investigative Site

Baltimore, Maryland, United States

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aTyr Investigative Site

Boston, Massachusetts, United States

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aTyr Investigative Site

Ada, Michigan, United States

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aTyr Investigative Site

Detroit, Michigan, United States

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aTyr Investigative Site

Minneapolis, Minnesota, United States

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aTyr Investigative Site

Rochester, Minnesota, United States

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aTyr Investigative Site

Jackson, Mississippi, United States

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aTyr Investigative Site

St Louis, Missouri, United States

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aTyr Investigative Site

Albany, New York, United States

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aTyr Investigative Site

Middletown, New York, United States

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aTyr Investigative Site

Durham, North Carolina, United States

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aTyr Investigative Site

Greenville, North Carolina, United States

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aTyr Investigative Site

Cincinnati, Ohio, United States

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aTyr Investigative Site

Cleveland, Ohio, United States

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aTyr Investigative Site

Oklahoma City, Oklahoma, United States

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aTyr Investigative Site

Portland, Oregon, United States

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aTyr Investigative Site

Philadelphia, Pennsylvania, United States

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aTyr Investigative Site

Charleston, South Carolina, United States

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aTyr Investigative Site

Dickson, Tennessee, United States

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aTyr Investigative Site

Dallas, Texas, United States

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aTyr Investigative Site

Dallas, Texas, United States

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aTyr Investigative Site

Houston, Texas, United States

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aTyr Investigative Site

Salt Lake City, Utah, United States

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aTyr Investigative Site

Falls Church, Virginia, United States

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aTyr Investigative Site

Richmond, Virginia, United States

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aTyr Investigative Site

Salvador, Estado de Bahia, Brazil

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aTyr Investigative Site

Porto Alegre, Rio Grande do Sul, Brazil

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aTyr Investigative Site

São Bernardo do Campo, , Brazil

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aTyr Investigative Site

São Paulo, , Brazil

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aTyr Investigative Site

São Paulo, , Brazil

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aTyr Investigative Site

Bobigny, , France

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aTyr Investigative Site

La Tronche, , France

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aTyr Investigative Site

Montpellier, , France

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aTyr Investigative Site

Paris, , France

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aTyr Investigative Site

Toulouse, , France

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aTyr Investigative Site

Essen, , Germany

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aTyr Investigative Site

Freiburg im Breisgau, , Germany

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aTyr Investigative Site

Hanover, , Germany

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aTyr Investigative Site

Heidelberg, , Germany

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aTyr Investigative Site

Catania, , Italy

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aTyr Investigative Site

Florence, , Italy

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aTyr Investigative Site

Forlì, , Italy

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aTyr Investigative Site

Milan, , Italy

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aTyr Investigative Site

Napoli, , Italy

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aTyr Investigative Site

Padua, , Italy

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aTyr Investigative Site

Rome, , Italy

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aTyr Investigative Site

Siena, , Italy

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aTyr Investigative Site

Trieste, , Italy

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Kyorin Investigative Site

Aomori, , Japan

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Kyorin Investigative Site

Fukuoka, , Japan

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Kyorin Investigative Site

Fukushima, , Japan

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Kyorin Investigative Site

Hokkaido, , Japan

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Kyorin Investigative Site

Hokkaido, , Japan

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Kyorin Investigative Site

Hyōgo, , Japan

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Kyorin Investigative Site

Kanagawa, , Japan

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Kyorin Investigative Site

Kanagawa, , Japan

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aTyr Investigative Site

Kumamoto, , Japan

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Kyorin Investigative Site

Miyagi, , Japan

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Kyorin Investigative Site

Okayama, , Japan

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Kyorin Investigative Site

Osaka, , Japan

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Kyorin Investigative Site

Shimane, , Japan

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Kyorin Investigative Site

Shizuoka, , Japan

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Kyorin Investigative Site

Tokyo, , Japan

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Kyorin Investigative Site

Tokyo, , Japan

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aTyr Investigative Site

Nieuwegein, , Netherlands

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aTyr Investigative Site

Barcelona, , Spain

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aTyr Investigative Site

Barcelona, , Spain

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aTyr Investigative Site

Madrid, , Spain

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aTyr Investigative Site

Madrid, , Spain

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aTyr Investigative Site

Santander, , Spain

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aTyr Investigative Site

Valencia, , Spain

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aTyr Investigative Site

Birmingham, , United Kingdom

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aTyr Investigative Site

Cambridge, , United Kingdom

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aTyr Investigative Site

Cottingham, , United Kingdom

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aTyr Investigative Site

Coventry, , United Kingdom

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aTyr Investigative Site

London, , United Kingdom

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aTyr Investigative Site

London, , United Kingdom

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Countries

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United States Brazil France Germany Italy Japan Netherlands Spain United Kingdom

References

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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.

Reference Type DERIVED
PMID: 37902990 (View on PubMed)

Other Identifiers

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ATYR1923-C-004

Identifier Type: -

Identifier Source: org_study_id

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