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

Phase 2

Recruiting

• Conditions: Interstitial Lung Disease
• Interventions: Drug: efzofitimod 450 mg; Drug: efzofitimod 270 mg; Drug: Placebo

• Registration Number: NCT05892614
• Lead Sponsor: aTyr Pharma, Inc.

Brief 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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2023-05-12
• Study First Submitted QC: 2023-06-05
• Study First Posted: 2023-06-07
• Study Start: 2023-10-26
• Status Verified: 2024-07
• Last Update Submitted: 2024-07-30
• Last Update Posted: 2024-07-31
• Primary Completion: 2024-12
• Study Completion: 2024-12

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 25

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 OR

   If overall duration of SSc > 48 and < 72 months from the first non-Raynaud symptom manifestation, then with presence of any of the following:

   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.

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Exclusion Criteria

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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
efzofitimod 450 mg	efzofitimod 450 mg	Administered IV infusion
efzofitimod 270 mg	efzofitimod 270 mg	Administered IV infusion
Placebo	Placebo	Administered IV infusion

Primary Outcome Measures

Name	Time	Method
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
Change in HRCT fibrosis score	Baseline to Week 24
Proportion of patients with > 5% and ≥ 10% decline in percent predicted FVC	Baseline to Week 24
Absolute change from baseline in forced vital capacity (FVC) in mL	24 weeks

Trial Locations

Locations (1)

aTyr Investigative Site; 🇺🇸 Richmond, Virginia, United States