Study to Evaluate the Efficacy, Safety, and Tolerability of Efzofitimod in Patients With Systemic Sclerosis (SSc)-Related Interstitial Lung Disease (ILD) (SSc-ILD)

Last updated: May 5, 2025
Sponsor: aTyr Pharma, Inc.
Overall Status: Active - Recruiting

Phase

2

Condition

Lung Disease

Scar Tissue

Treatment

efzofitimod 450 mg

efzofitimod 270 mg

Placebo

Clinical Study ID

NCT05892614
ATYR1923-C-005
  • Ages > 18
  • All Genders

Study Summary

This is a 2-Part study with Part A, a double-blind, randomized, placebo-controlled, PoC study to evaluate the efficacy, safety, and tolerability of efzofitimod in patients with SSc-ILD. The primary objective of the study is to evaluate the PoC for efficacy in a population with SSc-ILD. While improvement of ILD is the outcome of interest, the study will also evaluate changes in the skin. After initial screening (up to 4 weeks), approximately 25 eligible participants will be randomized 2:2:1 to 1 of 2 active (experimental) dose arms or placebo, administered every 4 weeks up to and including Week 20. Part B is an optional open-label extension to Part A in which participants can receive 450 mg efzofitimod every 4 weeks for 6 doses.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  1. Diagnosis of SSc based on ACR/ EULAR criteria (2013)

  2. Overall duration of SSc < 84 months from the first non-Raynaud symptom manifestationprior to Day 1

  3. HRCT obtained at the Screening Visit or within the 3 months prior to Screeningconsistent with SSc-ILD (adjudicated by a central reader) AND with pulmonaryinvolvement > 10%

  4. Clinical presentation at Screening consistent with lcSSc (up to 40% of patients) ordcSSc

  5. MMF of ≥ 2 gm/day (or equivalent doses of other mycophenolate based compounds) for 3months prior to Day 1 OR When documented intolerance to mycophenolates (indiscussion with the Medical Monitor): treatment with maximum tolerated dose of MMFis acceptable, if < 2 gm/day, provided the cumulative duration of dosing hasexceeded 3 months, OR An adequate dose and duration of an alternateimmunosuppressant with a stable dose for the 4 weeks prior to baseline is alsoallowed.

Exclusion

Exclusion Criteria:

  1. Pulmonary disease with FVC %pred ≤ 45% OR DLco %pred ≤ 30%; FEV1/FVC ratio < 0.7

  2. Participants with pulmonary artery hypertension on parenteral therapy or withclinical evidence of right heart failure

  3. HRCT obtained in the 3 months prior to Screening consistent with other confoundingpathology.

  4. Treatment with corticosteroids (> 10 mg/day of prednisone or equivalent) within 2weeks prior to Day 1

  5. Treatment with more than 1 immunosuppressant (e.g., MMF, methotrexate [MTX],azathioprine [AZA], or leflunomide)

  6. Any treatment in the 12 months prior to Day 1 with any of the following: rituximab,intravenous immune globulin (IVIG), tocilizumab, cyclophosphamide, pirfenidone,tyrosine-kinase inhibitors (e.g., imatinib, nilotinib, dasatinib)

  7. Rheumatic autoimmune disease other than SSc, Is an active, heavy smoker oftobacco/nicotine-containing products

  8. History of (anti-Jo-1) anti-synthetase syndrome or Jo-1 positive at Screening

Study Design

Total Participants: 25
Treatment Group(s): 3
Primary Treatment: efzofitimod 450 mg
Phase: 2
Study Start date:
October 26, 2023
Estimated Completion Date:
April 30, 2026

Connect with a study center

  • aTyr Investigative Site

    Los Angeles, California 90024
    United States

    Active - Recruiting

  • aTyr Investigative Site

    San Diego, California 92093
    United States

    Active - Recruiting

  • aTyr Investigative Site

    Miami, Florida 33146
    United States

    Active - Recruiting

  • aTyr Investigative Site

    Chicago, Illinois 60612
    United States

    Active - Recruiting

  • aTyr Investigative Site

    New Orleans, Louisiana 70115
    United States

    Active - Recruiting

  • aTyr Investigative Site

    New York, New York 10027
    United States

    Active - Recruiting

  • aTyr Investigative Site

    Cleveland, Ohio 44195
    United States

    Active - Recruiting

  • aTyr Investigative Site

    Oklahoma City, Oklahoma 73104
    United States

    Active - Recruiting

  • aTyr Investigative Site

    Charleston, South Carolina 29425
    United States

    Active - Recruiting

  • aTyr Investigative Site

    Dallas, Texas 75204
    United States

    Active - Recruiting

  • aTyr Investigative Site

    Houston, Texas 77204
    United States

    Active - Recruiting

  • aTyr Investigative Site

    Salt Lake City, Utah 84112
    United States

    Active - Recruiting

  • aTyr Investigative Site

    Richmond, Virginia 23284
    United States

    Active - Recruiting

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