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

April 19, 2024 updated by: aTyr Pharma, Inc.

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)

This is 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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

25

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • United States
    • California
      • Los Angeles, California, United States, 90024
        • Not yet recruiting
        • aTyr Investigative Site
        • Contact:
      • San Diego, California, United States, 92093
        • Not yet recruiting
        • aTyr Investigative Site
        • Contact:
    • Florida
      • Miami, Florida, United States, 33146
        • Not yet recruiting
        • aTyr Investigative Site
        • Contact:
    • Illinois
      • Chicago, Illinois, United States, 60611
      • Chicago, Illinois, United States, 60612
      • Chicago, Illinois, United States, 60153
        • Not yet recruiting
        • aTyr Investigative Site
        • Contact:
    • Louisiana
      • New Orleans, Louisiana, United States, 70115
    • New York
      • New York, New York, United States, 10027
    • Ohio
      • Cleveland, Ohio, United States, 44195
    • Oklahoma
      • Oklahoma City, Oklahoma, United States, 73104
    • South Carolina
      • Charleston, South Carolina, United States, 29425
    • Texas
      • Dallas, Texas, United States, 75204
      • Houston, Texas, United States, 77204
    • Utah
      • Salt Lake City, Utah, United States, 84112
    • Virginia
      • Richmond, Virginia, United States, 23284

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Diagnosis of SSc based on ACR/ EULAR criteria (2013)
  2. Overall duration of SSc < 48 months from the first non-Raynaud symptom manifestation
  3. 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%
  4. Clinical presentation at Screening consistent with lcSSc (up to 40% of patients) or dcSSc

  5. Presence of at least:

    Any 1 laboratory marker for active disease

    OR

    Clinically significant decline in FVC % predicted (%pred) based on ≥ 105% relative decline over the preceding one year (two readings from the same pulmonary function laboratory)

  6. MMF of ≥ 2 gm/day (or equivalent doses of other mycophenolate based compounds) for 6 months

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 with clinical evidence of right heart failure
  3. HRCT obtained in the 3 months prior to Screening consistent with other confounding pathology.
  4. Treatment with corticosteroids (> 10 mg/day of prednisone or equivalent) within 2 weeks prior to baseline
  5. SSc-ILD treatments other than MMF OR MMF < 2 gm/day
  6. Any previous treatment 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 of tobacco/nicotine-containing products
  8. History of (anti-Jo-1) anti-synthetase syndrome or Jo-1 positive at Screening

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Triple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: efzofitimod 450 mg
Administered IV infusion
Drug: efzofitimod 450 mg
IV infusion over approximately 60 minutes every 4 weeks
Experimental: efzofitimod 270 mg
Administered IV infusion
Drug: efzofitimod 270 mg
IV infusion over approximately 60 minutes every 4 weeks
Placebo Comparator: Placebo
Administered IV infusion
Drug: Placebo
IV infusion over approximately 60 minutes every 4 weeks

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Absolute change from baseline in forced vital capacity (FVC) in mL
Time Frame: 24 weeks
24 weeks
Annual rate of decline in FVC in mL
Time Frame: 24 weeks
24 weeks
Annual rate of decline in FVC in percent predicted
Time Frame: 24 weeks
24 weeks
Proportion of patients with > 5% and ≥ 10% decline in absolute FVC
Time Frame: Baseline to week 24
Baseline to week 24
Proportion of patients with > 5% and ≥ 10% decline in percent predicted FVC
Time Frame: Baseline to Week 24
Baseline to Week 24
Change in HRCT fibrosis score
Time Frame: Baseline to Week 24
Baseline to Week 24

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

aTyr Pharma, Inc.

Investigators

  • Study Director: Lisa Carey, aTyr Pharma, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

October 26, 2023

Primary Completion (Estimated)

December 1, 2024

Study Completion (Estimated)

December 1, 2024

Study Registration Dates

First Submitted

May 12, 2023

First Submitted That Met QC Criteria

June 5, 2023

First Posted (Actual)

June 7, 2023

Study Record Updates

Last Update Posted (Actual)

April 22, 2024

Last Update Submitted That Met QC Criteria

April 19, 2024

Last Verified

April 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ATYR1923-C-005

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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