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

a study on Interstitial Lung Disease Systemic Sclerosis Diffuse Scleroderma

Summary

Eligibility
for people ages 18 years and up (full criteria)
Location
at UCLA
Dates
study started
study ends around
Principal Investigator
by Elizabeth R. Volkmann, MD, MS (ucla)
Headshot of Elizabeth R. Volkmann
Elizabeth R. Volkmann

Description

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.

Official 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)

Keywords

Interstitial Lung Disease, ILD, SSc-ILD, lung inflammation, fibrosis, pulmonary function, efzofitimod, systemic sclerosis, Lung Diseases, Interstitial Lung Diseases, Systemic Scleroderma, Diffuse Scleroderma, Sclerosis, efzofitimod 450 mg, efzofitimod 270 mg

Eligibility

You can join if…

Open to people ages 18 years and up

  1. Diagnosis of SSc based on ACR/ EULAR criteria (2013)
  2. Overall duration of SSc < 84 months from the first non-Raynaud symptom manifestation prior to Day 1
  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 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 CAN'T join if...

  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 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 of tobacco/nicotine-containing products
  8. History of (anti-Jo-1) anti-synthetase syndrome or Jo-1 positive at Screening

Locations

  • aTyr Investigative Site accepting new patients
    Los Angeles California 90024 United States
  • aTyr Investigative Site accepting new patients
    San Diego California 92093 United States

Lead Scientist at University of California Health

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
aTyr Pharma, Inc.
ID
NCT05892614
Phase
Phase 2 research study
Study Type
Interventional
Participants
Expecting 25 study participants
Last Updated