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

Description

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

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Study Overview

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Study Details

Study overview

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.

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)

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

Condition
Interstitial Lung Disease
Intervention / Treatment

-

Contacts and Locations

Los Angeles

aTyr Investigative Site, Los Angeles, California, United States, 90024

San Diego

aTyr Investigative Site, San Diego, California, United States, 92093

Miami

aTyr Investigative Site, Miami, Florida, United States, 33146

Chicago

aTyr Investigative Site, Chicago, Illinois, United States, 60153

Chicago

aTyr Investigative Site, Chicago, Illinois, United States, 60611

Chicago

aTyr Investigative Site, Chicago, Illinois, United States, 60612

New Orleans

aTyr Investigative Site, New Orleans, Louisiana, United States, 70115

New York

aTyr Investigative Site, New York, New York, United States, 10027

Cleveland

aTyr Investigative Site, Cleveland, Ohio, United States, 44195

Oklahoma City

aTyr Investigative Site, Oklahoma City, Oklahoma, United States, 73104

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.

For general information about clinical research, read Learn About Studies.

Eligibility 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 OR
  • 1. Any 1 laboratory marker for active disease:
  • * C-reactive protein ≥ 0.6 mg/dL (≥ 6 mg/L)
  • * Erythrocyte sedimentation rate ≥ 28 mm/hr
  • * Platelet count ≥ 330 × 10e9/L (330,000/μL) OR
  • 2. Clinically significant decline in FVC % predicted (%pred) based on ≥ 5% relative decline over the preceding one year OR
  • 3. An increase ≥ 3 in the mRSS over 6 months or less
  • 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. MMF of ≥ 2 gm/day (or equivalent doses of other mycophenolate based compounds) for 6 months OR When documented intolerance to mycophenolates, treatment with adequate doses and duration of an alternate immunosuppressant with a stable dose for the 4 weeks prior to baseline. The use of an alternate immunosuppressant must be discussed with the Medical Monitor.
  • 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. Treatment with more than 1 immunosuppressant (e.g., MMF, methotrexate \[MTX\], azathioprine \[AZA\], or leflunomide)
  • 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

Ages Eligible for Study

18 Years to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

aTyr Pharma, Inc.,

Lisa Carey, STUDY_DIRECTOR, aTyr Pharma, Inc.

Study Record Dates

2024-12