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

About

Brief Summary

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.

Primary Purpose
Treatment
Study Type
Interventional
Phase
Phase II

Eligibility

Gender
All
Healthy Volunteers
No
Minimum Age
18 Years
Maximum Age
N/A

Inclusion Criteria:

  • Diagnosis of SSc based on ACR/ EULAR criteria (2013)
  • Overall duration of SSc < 48 months from the first non-Raynaud symptom manifestation
  • 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
  • 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)
  • MMF of ≥ 2 gm/day (or equivalent doses of other mycophenolate based compounds) for 6 months

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 baseline
  • SSc-ILD treatments other than MMF OR MMF < 2 gm/day
  • Any previous treatment 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

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Study Stats
Protocol No.
23-5154
Category
Autoimmune Disorders
Contact
Tory Ham
Location
  • UCLA Westwood
For Providers
NCT No.
NCT05892614
For detailed technical eligibility, visit ClinicalTrials.gov.