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

Interstitial Lung Disease Clinical Trial

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)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05892614
• Other study ID #: ATYR1923-C-005
• Status: Recruiting
• Phase: Phase 2
• Start date: October 26, 2023
• Est. completion date: December 2024

Study information

• Verified date: April 2024
• Source: aTyr Pharma, Inc.
• Contact: aTyr Pharma Clinical Research
• Phone: 877-215-5731
• Email: clinicaltrials[@]atyrpharma.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 25
• Est. completion date: December 2024
• Est. primary completion date: December 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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 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. 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)

6. MMF of = 2 gm/day (or equivalent doses of other mycophenolate based compounds) for 6 months

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. 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), 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

Related Conditions & MeSH terms

• Interstitial Lung Disease
• Lung Diseases
• Lung Diseases, Interstitial
• Scleroderma, Diffuse
• Scleroderma, Systemic
• Sclerosis

Intervention

Drug:
• efzofitimod 450 mg
IV infusion over approximately 60 minutes every 4 weeks
• efzofitimod 270 mg
IV infusion over approximately 60 minutes every 4 weeks
• Placebo
IV infusion over approximately 60 minutes every 4 weeks

Locations

Country	Name	City	State
United States	aTyr Investigative Site	Charleston	South Carolina
United States	aTyr Investigative Site	Chicago	Illinois
United States	aTyr Investigative Site	Chicago	Illinois
United States	aTyr Investigative Site	Chicago	Illinois
United States	aTyr Investigative Site	Cleveland	Ohio
United States	aTyr Investigative Site	Dallas	Texas
United States	aTyr Investigative Site	Houston	Texas
United States	aTyr Investigative Site	Los Angeles	California
United States	aTyr Investigative Site	Miami	Florida
United States	aTyr Investigative Site	New Orleans	Louisiana
United States	aTyr Investigative Site	New York	New York
United States	aTyr Investigative Site	Oklahoma City	Oklahoma
United States	aTyr Investigative Site	Richmond	Virginia
United States	aTyr Investigative Site	Salt Lake City	Utah
United States	aTyr Investigative Site	San Diego	California

Sponsors (1)

Lead Sponsor	Collaborator
aTyr Pharma, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Absolute change from baseline in forced vital capacity (FVC) in mL		24 weeks
Primary	Annual rate of decline in FVC in mL		24 weeks
Primary	Annual rate of decline in FVC in percent predicted		24 weeks
Primary	Proportion of patients with > 5% and = 10% decline in absolute FVC		Baseline to week 24
Primary	Proportion of patients with > 5% and = 10% decline in percent predicted FVC		Baseline to Week 24
Primary	Change in HRCT fibrosis score		Baseline to Week 24