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NCT Number: NCT05892614

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

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.

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Key information

Conditions

Interstitial Lung Disease Connective Tissue Diseases Fibrosis Infections Lung Diseases Lung Diseases, Interstitial Pathologic Processes Pathological Conditions, Signs and Symptoms Pneumonia Respiratory Tract Diseases Respiratory Tract Infections Scleroderma, Systemic Skin Diseases Skin and Connective Tissue Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

aTyr Investigative Site, Los Angeles, California, United States

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Diagnosis of SSc based on ACR/ EULAR criteria (2013)
  • Overall duration of SSc < 84 months from the first non-Raynaud symptom manifestation prior to Day 1
  • HRCT obtained at the Screening Visit or within the 3 months prior to Screening consistent with SSc-ILD (adjudicated by a central reader) AND with pulmonary involvement > 10%
  • Clinical presentation at Screening consistent with lcSSc (up to 40% of patients) or dcSSc
  • MMF of ≥ 2 gm/day (or equivalent doses of other mycophenolate based compounds) for 3 months prior to Day 1 OR When documented intolerance to mycophenolates (in discussion with the Medical Monitor): treatment with maximum tolerated dose of MMF is acceptable, if < 2 gm/day, provided the cumulative duration of dosing has exceeded 3 months, OR An adequate dose and duration of an alternate immunosuppressant with a stable dose for the 4 weeks prior to baseline is also allowed.

Exclusion criteria

  • Pulmonary disease with FVC %pred ≤ 45% OR DLco %pred ≤ 30%; FEV1/FVC ratio < 0.7
  • Participants with pulmonary artery hypertension on parenteral therapy or with clinical evidence of right heart failure
  • HRCT obtained in the 3 months prior to Screening consistent with other confounding pathology.
  • Treatment with corticosteroids (> 10 mg/day of prednisone or equivalent) within 2 weeks prior to Day 1
  • Treatment with more than 1 immunosuppressant (e.g., MMF, methotrexate [MTX], azathioprine [AZA], or leflunomide)
  • 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)
  • Rheumatic autoimmune disease other than SSc, Is an active, heavy smoker of tobacco/nicotine-containing products
  • History of (anti-Jo-1) anti-synthetase syndrome or Jo-1 positive at Screening

Treatment and study plan

efzofitimod 450 mg

Drug

IV infusion over approximately 60 minutes every 4 weeks

efzofitimod 270 mg

Drug

IV infusion over approximately 60 minutes every 4 weeks

Placebo

Drug

IV infusion over approximately 60 minutes every 4 weeks

Primary outcomes

  1. Absolute change from baseline in forced vital capacity (FVC) in mL

    Time frame: 24 weeks

  2. Annual rate of decline in FVC in mL

    Time frame: 24 weeks

  3. Annual rate of decline in FVC in percent predicted

    Time frame: 24 weeks

  4. Change in HRCT fibrosis score

    Time frame: Baseline to Week 24

Study contacts

Contact information is provided by the study sponsor or research team.

aTyr Pharma Clinical Research

CONTACT

[email protected]

877-215-5731

Sponsors and collaborators

Lead sponsor

aTyr Pharma, Inc.

Industry

Registry information

Official study title

Randomized, Double-blind, Placebo-controlled Proof-of-Concept (PoC) Study to Evaluate the Efficacy, Safety, and Tolerability of Efzofitimod in Patients With Systemic Sclerosis (SSc)-Related Interstitial Lung Disease (ILD) (SSc-ILD)

Important dates

Study start
2023
Primary completion
2026
Study completion
2026
First posted
Jun 7, 2023
Registry last updated
May 7, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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