Efficacy and Safety of Intravenous Efzofitimod in Patients With Pulmonary Sarcoidosis

NCT05415137 | Active Not Recruiting Phase 3 DMC FDA Drug

• 1 other identifier: ATYR1923-C-004
• Study Type: interventional
• Target: 268
• Locations: 9 countries
• Sites: 91

Brief Summary

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

Keywords

Pulmonary Sarcoidosis; Sarcoidosis; Granuloma; Inflammation; Lymphoproliferative Disorders; Interstitial Lung Disease; Neuropilin-2; Steroids; Oral corticosteroids; Immunomodulatory; tRNA Synthetase; ATYR1923; KRP-R120; Efzofitimod; Fibrosis

Outcome Measures

Primary Outcomes (1)

• Change from baseline in mean daily oral corticosteroid (OCS) dose at Week 48

  Baseline to Week 48

Secondary Outcomes (3)

• Change from baseline in KSQ-Lung score at Week 48

  Baseline to Week 48

• Steroid withdrawal rate

  Baseline to Week 48

• Change from baseline in absolute value of FVC at Week 48

  Baseline to Week 48

Study Arms (3)

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

Interventions

Efzofitimod 3 mg/kg DRUG

EfzofitimodIV infusion every 4 weeks for a total of 12 doses

Also known as: ATYR1923, KRP-R120

Efzofitimod 3 mg/kg

Efzofitimod 5 mg/kg DRUG

EfzofitimodIV infusion every 4 weeks for a total of 12 doses

Also known as: ATYR1923, KRP-R120

Efzofitimod 5 mg/kg

Placebo DRUG

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

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• aTyr Pharma, Inc.: lead
• Kyorin Pharmaceutical Co.,Ltd: collaborator

Study Sites (91)

aTyr Investigative Site

Birmingham, Alabama, 35294, United States

Location

aTyr Investigative Site

Phoenix, Arizona, 85027, United States

Location

aTyr Investigative Site

Los Angeles, California, 90033, United States

Location

aTyr Investigative Site

Sacramento, California, 95817, United States

Location

aTyr Investigative Site

Denver, Colorado, 80206, United States

Location

aTyr Investigative Site

Washington D.C., District of Columbia, 20060, United States

Location

aTyr Investigative Site

Gainesville, Florida, 32610, United States

Location

aTyr Investigative Site

Tampa, Florida, 33606, United States

Location

aTyr Investigative Site

Weston, Florida, 33331, United States

Location

aTyr Investigative Site

Atlanta, Georgia, 30308, United States

Location

aTyr Investigative Site

Chicago, Illinois, 60611, United States

Location

aTyr Investigative Site

Chicago, Illinois, 60612, United States

Location

aTyr Investigative Site

Louisville, Kentucky, 40202, United States

Location

aTyr Investigative Site

New Orleans, Louisiana, 70115, United States

Location

aTyr Investigative Site

Baltimore, Maryland, 21224, United States

Location

aTyr Investigative Site

Boston, Massachusetts, 02114, United States

Location

aTyr Investigative Site

Ada, Michigan, 49301, United States

Location

aTyr Investigative Site

Detroit, Michigan, 48202, United States

Location

aTyr Investigative Site

Minneapolis, Minnesota, 55455, United States

Location

aTyr Investigative Site

Rochester, Minnesota, 55905, United States

Location

aTyr Investigative Site

Jackson, Mississippi, 39216, United States

Location

aTyr Investigative Site

St Louis, Missouri, 63110, United States

Location

aTyr Investigative Site

Albany, New York, 12208, United States

Location

aTyr Investigative Site

Middletown, New York, 10941, United States

Location

aTyr Investigative Site

Durham, North Carolina, 27710, United States

Location

aTyr Investigative Site

Greenville, North Carolina, 27834, United States

Location

aTyr Investigative Site

Cincinnati, Ohio, 45219, United States

Location

aTyr Investigative Site

Cleveland, Ohio, 44195, United States

Location

aTyr Investigative Site

Oklahoma City, Oklahoma, 73104, United States

Location

aTyr Investigative Site

Portland, Oregon, 97220, United States

Location

aTyr Investigative Site

Philadelphia, Pennsylvania, 19140, United States

Location

aTyr Investigative Site

Charleston, South Carolina, 29425-0630, United States

Location

aTyr Investigative Site

Dickson, Tennessee, 37055, United States

Location

aTyr Investigative Site

Dallas, Texas, 75246, United States

Location

aTyr Investigative Site

Dallas, Texas, 75390, United States

Location

aTyr Investigative Site

Houston, Texas, 77030, United States

Location

aTyr Investigative Site

Salt Lake City, Utah, 84132, United States

Location

aTyr Investigative Site

Falls Church, Virginia, 22042, United States

Location

aTyr Investigative Site

Richmond, Virginia, 23298, United States

Location

aTyr Investigative Site

Salvador, Estado de Bahia, 40220-005, Brazil

Location

aTyr Investigative Site

Porto Alegre, Rio Grande do Sul, 90020-090, Brazil

Location

aTyr Investigative Site

São Bernardo do Campo, 09780-000, Brazil

Location

aTyr Investigative Site

São Paulo, 04039-901, Brazil

Location

aTyr Investigative Site

São Paulo, 05403-900, Brazil

Location

aTyr Investigative Site

Bobigny, 93000, France

Location

aTyr Investigative Site

La Tronche, 38700, France

Location

aTyr Investigative Site

Montpellier, 34295, France

Location

aTyr Investigative Site

Paris, 75015, France

Location

aTyr Investigative Site

Toulouse, 31000, France

Location

aTyr Investigative Site

Essen, 45122, Germany

Location

aTyr Investigative Site

Freiburg im Breisgau, 79106, Germany

Location

aTyr Investigative Site

Hanover, 30625, Germany

Location

aTyr Investigative Site

Heidelberg, 69126, Germany

Location

aTyr Investigative Site

Catania, 95124, Italy

Location

aTyr Investigative Site

Florence, 50134, Italy

Location

aTyr Investigative Site

Forlì, 47121, Italy

Location

aTyr Investigative Site

Milan, 20123, Italy

Location

aTyr Investigative Site

Napoli, 80131, Italy

Location

aTyr Investigative Site

Padua, 35128, Italy

Location

aTyr Investigative Site

Rome, 00168, Italy

Location

aTyr Investigative Site

Siena, 53100, Italy

Location

aTyr Investigative Site

Trieste, 34149, Italy

Location

Kyorin Investigative Site

Aomori, 036-8563, Japan

Location

Kyorin Investigative Site

Fukuoka, 814-0180, Japan

Location

Kyorin Investigative Site

Fukushima, 960-1295, Japan

Location

Kyorin Investigative Site

Hokkaido, 060-0033, Japan

Location

Kyorin Investigative Site

Hokkaido, 060-8648, Japan

Location

Kyorin Investigative Site

Hyōgo, 653-0013, Japan

Location

Kyorin Investigative Site

Kanagawa, 236-0004, Japan

Location

Kyorin Investigative Site

Kanagawa, 236-0051, Japan

Location

aTyr Investigative Site

Kumamoto, 860-8556, Japan

Location

Kyorin Investigative Site

Miyagi, 980-8574, Japan

Location

Kyorin Investigative Site

Okayama, 700-8558, Japan

Location

Kyorin Investigative Site

Osaka, 591-8025, Japan

Location

Kyorin Investigative Site

Shimane, 693-8501, Japan

Location

Kyorin Investigative Site

Shizuoka, 431-3192, Japan

Location

Kyorin Investigative Site

Tokyo, 150-8935, Japan

Location

Kyorin Investigative Site

Tokyo, 151-8528, Japan

Location

aTyr Investigative Site

Nieuwegein, 3435CM, Netherlands

Location

aTyr Investigative Site

Barcelona, 08036, Spain

Location

aTyr Investigative Site

Barcelona, 08907, Spain

Location

aTyr Investigative Site

Madrid, 28006, Spain

Location

aTyr Investigative Site

Madrid, 28031, Spain

Location

aTyr Investigative Site

Santander, 39008, Spain

Location

aTyr Investigative Site

Valencia, 46010, Spain

Location

aTyr Investigative Site

Birmingham, B15 2TH, United Kingdom

Location

aTyr Investigative Site

Cambridge, CB233RE, United Kingdom

Location

aTyr Investigative Site

Cottingham, HU16 5JQ, United Kingdom

Location

aTyr Investigative Site

Coventry, CV2 2DX, United Kingdom

Location

aTyr Investigative Site

London, SE59RS, United Kingdom

Location

aTyr Investigative Site

London, SW3 6NP, United Kingdom

Location

Related Publications (1)

• 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.

  PMID: 37902990 DERIVED

MeSH Terms

Conditions

Sarcoidosis, Pulmonary; Sarcoidosis; Granuloma; Inflammation; Lymphoproliferative Disorders; Lung Diseases, Interstitial; Fibrosis

Condition Hierarchy (Ancestors)

Lung Diseases; Respiratory Tract Diseases; Lymphatic Diseases; Hemic and Lymphatic Diseases; Hypersensitivity, Delayed; Hypersensitivity; Immune System Diseases; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Immunoproliferative Disorders

Study Officials

• Lisa Carey

  aTyr Pharma, Inc.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 8, 2022
• First Posted: June 10, 2022
• Study Start: September 15, 2022
• Primary Completion: July 31, 2025
• Study Completion: August 31, 2025
• Last Updated: May 20, 2025

Record last verified: 2025-05

Locations

IT (9); US (39); FR (5); ES (6); GB (6); BR (5); JP (16); DE (4); NL (1)