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

Sponsor

aTyr Pharma, Inc. (Industry)

Overall Status

Not yet recruiting

CT.gov ID

NCT05892614

Collaborator

(none)

Enrollment

Arms

Anticipated Duration (Months)

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Interstitial Lung Disease	Drug: efzofitimod 450 mg Drug: efzofitimod 270 mg Drug: Placebo	Phase 2

Study Design

Study Type:

Interventional

Anticipated Enrollment :

25 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Masking:

Triple (Participant, Care Provider, Investigator)

Primary Purpose:

Treatment

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)

Anticipated Study Start Date :

Sep 1, 2023

Anticipated Primary Completion Date :

Dec 1, 2024

Anticipated Study Completion Date :

Dec 1, 2024

Arms and Interventions

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

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

Outcome Measures

Primary Outcome Measures

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]
Proportion of patients with > 5% and ≥ 10% decline in absolute FVC [Baseline to week 24]
Proportion of patients with > 5% and ≥ 10% decline in percent predicted FVC [Baseline to Week 24]
Change in HRCT fibrosis score [Baseline to Week 24]

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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 in 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 dcSSc (distribution on trunk and proximal to knee, elbow)
1. Presence of at least: Any 1 laboratory marker for active disease

Any 1 criteria for progressive pulmonary fibrosis over the preceding 1 year

mRSS of > 10
MMF of ≥ 2 gm/day for 6 months

Exclusion Criteria:

Pulmonary disease with FVC %pred ≤ 45% OR DLco %pred ≤ 30%
Participants with pulmonary artery hypertension on parenteral therapy or with clinical evidence of right heart failure
Participants requiring > 2 L/min of supplemental oxygen by nasal cannula
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), nintedanib, 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