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

• Status: Not yet recruiting
• Trial end date: 2024-12-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: aTyr Pharma, Inc.

Criteria

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 in 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 dcSSc (distribution on trunk and proximal to knee, elbow)

5. 1. Presence of at least: Any 1 laboratory marker for active disease

OR

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

6. mRSS of > 10

7. MMF of ≥ 2 gm/day for 6 months

Exclusion Criteria:

1. Pulmonary disease with FVC %pred ≤ 45% OR DLco %pred ≤ 30%

2. Participants with pulmonary artery hypertension on parenteral therapy or with clinical evidence of right heart failure

3. Participants requiring > 2 L/min of supplemental oxygen by nasal cannula

4. 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

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), nintedanib, 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