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

NCT ID: NCT05892614

Last Updated: 2025-05-07

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE2

Total Enrollment

25 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-10-26

Study Completion Date

2026-04-30

Brief Summary

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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

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Conditions

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Interstitial Lung Disease

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators

Study Groups

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efzofitimod 450 mg

Administered IV infusion

Group Type EXPERIMENTAL

efzofitimod 450 mg

Intervention Type DRUG

IV infusion over approximately 60 minutes every 4 weeks

efzofitimod 270 mg

Administered IV infusion

Group Type EXPERIMENTAL

efzofitimod 270 mg

Intervention Type DRUG

IV infusion over approximately 60 minutes every 4 weeks

Placebo

Administered IV infusion

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

IV infusion over approximately 60 minutes every 4 weeks

Interventions

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efzofitimod 450 mg

IV infusion over approximately 60 minutes every 4 weeks

Intervention Type DRUG

efzofitimod 270 mg

IV infusion over approximately 60 minutes every 4 weeks

Intervention Type DRUG

Placebo

IV infusion over approximately 60 minutes every 4 weeks

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

1. Diagnosis of SSc based on ACR/ EULAR criteria (2013)
2. Overall duration of SSc \< 84 months from the first non-Raynaud symptom manifestation prior to Day 1
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 3 months prior to Day 1 OR When documented intolerance to mycophenolates (in discussion with the Medical Monitor): treatment with maximum tolerated dose of MMF is acceptable, if \< 2 gm/day, provided the cumulative duration of dosing has exceeded 3 months, OR An adequate dose and duration of an alternate immunosuppressant with a stable dose for the 4 weeks prior to baseline is also allowed.

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 Day 1
5. Treatment with more than 1 immunosuppressant (e.g., MMF, methotrexate \[MTX\], azathioprine \[AZA\], or leflunomide)
6. Any treatment in the 12 months prior to Day 1 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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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aTyr Pharma, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Lisa Carey

Role: STUDY_DIRECTOR

aTyr Pharma, Inc.

Locations

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aTyr Investigative Site

Los Angeles, California, United States

Site Status RECRUITING

aTyr Investigative Site

San Diego, California, United States

Site Status RECRUITING

aTyr Investigative Site

Miami, Florida, United States

Site Status RECRUITING

aTyr Investigative Site

Chicago, Illinois, United States

Site Status RECRUITING

aTyr Investigative Site

Chicago, Illinois, United States

Site Status RECRUITING

aTyr Investigative Site

Chicago, Illinois, United States

Site Status RECRUITING

aTyr Investigative Site

New Orleans, Louisiana, United States

Site Status RECRUITING

aTyr Investigative Site

New York, New York, United States

Site Status RECRUITING

aTyr Investigative Site

Cleveland, Ohio, United States

Site Status RECRUITING

aTyr Investigative Site

Oklahoma City, Oklahoma, United States

Site Status RECRUITING

aTyr Investigative Site

Charleston, South Carolina, United States

Site Status RECRUITING

aTyr Investigative Site

Dallas, Texas, United States

Site Status RECRUITING

aTyr Investigative Site

Houston, Texas, United States

Site Status RECRUITING

aTyr Investigative Site

Salt Lake City, Utah, United States

Site Status RECRUITING

aTyr Investigative Site

Richmond, Virginia, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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aTyr Pharma Clinical Research

Role: CONTACT

877-215-5731

Facility Contacts

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Role: primary

877-689-4494

Role: primary

877-689-4494

Role: primary

877-689-4494

Role: primary

877-689-4494

Role: primary

877-689-4494

Role: primary

877-689-4494

Role: primary

877-689-4494

Role: primary

877-689-4494

Role: primary

877-689-4494

Role: primary

877-689-4494

Role: primary

877-689-4494

Role: primary

877-689-4494

Role: primary

877-689-4494

Role: primary

877-689-4494

Role: primary

877-689-4494

Other Identifiers

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ATYR1923-C-005

Identifier Type: -

Identifier Source: org_study_id

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