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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)

Primary Purpose

Interstitial Lung Disease

Status
Recruiting
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
efzofitimod 450 mg
efzofitimod 270 mg
Placebo
Sponsored by
aTyr Pharma, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Interstitial Lung Disease focused on measuring ILD, SSc-ILD, Interstitial Lung Disease, lung inflammation, fibrosis, pulmonary function, efzofitimod, systemic sclerosis

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept 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 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), 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

Sites / Locations

  • aTyr Investigative SiteRecruiting
  • aTyr Investigative Site

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Experimental

Placebo Comparator

Arm Label

efzofitimod 450 mg

efzofitimod 270 mg

Placebo

Arm Description

Administered IV infusion

Administered IV infusion

Administered IV infusion

Outcomes

Primary Outcome Measures

Absolute change from baseline in forced vital capacity (FVC) in mL
Annual rate of decline in FVC in mL
Annual rate of decline in FVC in percent predicted
Proportion of patients with > 5% and ≥ 10% decline in absolute FVC
Proportion of patients with > 5% and ≥ 10% decline in percent predicted FVC
Change in HRCT fibrosis score

Secondary Outcome Measures

Full Information

First Posted
May 12, 2023
Last Updated
September 26, 2023
Sponsor
aTyr Pharma, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT05892614
Brief Title
Study to Evaluate the Efficacy, Safety, and Tolerability of Efzofitimod in Patients With Systemic Sclerosis (SSc)-Related Interstitial Lung Disease (ILD) (SSc-ILD)
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)
Study Type
Interventional

2. Study Status

Record Verification Date
September 2023
Overall Recruitment Status
Recruiting
Study Start Date
September 2023 (Anticipated)
Primary Completion Date
December 2024 (Anticipated)
Study Completion Date
December 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
aTyr Pharma, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Interstitial Lung Disease
Keywords
ILD, SSc-ILD, Interstitial Lung Disease, lung inflammation, fibrosis, pulmonary function, efzofitimod, systemic sclerosis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigator
Allocation
Randomized
Enrollment
25 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
efzofitimod 450 mg
Arm Type
Experimental
Arm Description
Administered IV infusion
Arm Title
efzofitimod 270 mg
Arm Type
Experimental
Arm Description
Administered IV infusion
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Administered IV infusion
Intervention Type
Drug
Intervention Name(s)
efzofitimod 450 mg
Intervention Description
IV infusion over approximately 60 minutes every 4 weeks
Intervention Type
Drug
Intervention Name(s)
efzofitimod 270 mg
Intervention Description
IV infusion over approximately 60 minutes every 4 weeks
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
IV infusion over approximately 60 minutes every 4 weeks
Primary Outcome Measure Information:
Title
Absolute change from baseline in forced vital capacity (FVC) in mL
Time Frame
24 weeks
Title
Annual rate of decline in FVC in mL
Time Frame
24 weeks
Title
Annual rate of decline in FVC in percent predicted
Time Frame
24 weeks
Title
Proportion of patients with > 5% and ≥ 10% decline in absolute FVC
Time Frame
Baseline to week 24
Title
Proportion of patients with > 5% and ≥ 10% decline in percent predicted FVC
Time Frame
Baseline to Week 24
Title
Change in HRCT fibrosis score
Time Frame
Baseline to Week 24

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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), 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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
aTyr Pharma Clinical Research
Phone
877-215-5731
Email
clinicaltrials@atyrpharma.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Lisa Carey
Organizational Affiliation
aTyr Pharma, Inc.
Official's Role
Study Director
Facility Information:
Facility Name
aTyr Investigative Site
City
New Orleans
State/Province
Louisiana
ZIP/Postal Code
70115
Country
United States
Individual Site Status
Recruiting
Facility Contact:
Phone
877-689-4494
Email
SScILD@cssienroll.com
Facility Name
aTyr Investigative Site
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44195
Country
United States
Individual Site Status
Not yet recruiting
Facility Contact:
Phone
877-689-4494
Email
SScILD@cssienroll.com

12. IPD Sharing Statement

Learn more about this trial

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

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