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

NCT05892614 | Recruiting Phase 2 FDA Drug

• 1 other identifier: ATYR1923-C-005
• Study Type: interventional
• Target: 25
• Locations: 1 country
• Sites: 15

Brief 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.

Conditions

Interstitial Lung Disease

Keywords

ILD; SSc-ILD; Interstitial Lung Disease; lung inflammation; fibrosis; pulmonary function; efzofitimod; systemic sclerosis

Outcome Measures

Primary Outcomes (4)

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

  24 weeks

• Annual rate of decline in FVC in mL

  24 weeks

• Annual rate of decline in FVC in percent predicted

  24 weeks

• Change in HRCT fibrosis score

  Baseline to Week 24

Study Arms (3)

efzofitimod 450 mg

EXPERIMENTAL

Administered IV infusion

Drug: efzofitimod 450 mg

efzofitimod 270 mg

EXPERIMENTAL

Administered IV infusion

Drug: efzofitimod 270 mg

Placebo

PLACEBO COMPARATOR

Administered IV infusion

Drug: Placebo

Interventions

efzofitimod 450 mg DRUG

IV infusion over approximately 60 minutes every 4 weeks

efzofitimod 450 mg

efzofitimod 270 mg DRUG

IV infusion over approximately 60 minutes every 4 weeks

efzofitimod 270 mg

Placebo DRUG

IV infusion over approximately 60 minutes every 4 weeks

Placebo

Eligibility Criteria

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

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• aTyr Pharma, Inc.: lead

Study Sites (15)

aTyr Investigative Site

Los Angeles, California, 90024, United States

RECRUITING

aTyr Investigative Site

San Diego, California, 92093, United States

RECRUITING

aTyr Investigative Site

Miami, Florida, 33146, United States

RECRUITING

aTyr Investigative Site

Chicago, Illinois, 60153, United States

RECRUITING

aTyr Investigative Site

Chicago, Illinois, 60611, United States

RECRUITING

aTyr Investigative Site

Chicago, Illinois, 60612, United States

RECRUITING

aTyr Investigative Site

New Orleans, Louisiana, 70115, United States

RECRUITING

aTyr Investigative Site

New York, New York, 10027, United States

RECRUITING

aTyr Investigative Site

Cleveland, Ohio, 44195, United States

RECRUITING

aTyr Investigative Site

Oklahoma City, Oklahoma, 73104, United States

RECRUITING

aTyr Investigative Site

Charleston, South Carolina, 29425, United States

RECRUITING

aTyr Investigative Site

Dallas, Texas, 75204, United States

RECRUITING

aTyr Investigative Site

Houston, Texas, 77204, United States

RECRUITING

aTyr Investigative Site

Salt Lake City, Utah, 84112, United States

RECRUITING

aTyr Investigative Site

Richmond, Virginia, 23284, United States

RECRUITING

MeSH Terms

Conditions

Lung Diseases, Interstitial; Pneumonia; Fibrosis; Scleroderma, Systemic

Condition Hierarchy (Ancestors)

Lung Diseases; Respiratory Tract Diseases; Respiratory Tract Infections; Infections; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases

Study Officials

• Lisa Carey

  aTyr Pharma, Inc.

  STUDY DIRECTOR

Central Study Contacts

aTyr Pharma Clinical Research

CONTACT

877-215-5731; clinicaltrials@atyrpharma.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 12, 2023
• First Posted: June 7, 2023
• Study Start: October 26, 2023
• Primary Completion: April 1, 2026
• Study Completion: April 1, 2026
• Last Updated: May 7, 2025

Record last verified: 2025-05

Locations

US (15)