NCT05892614 · aTyr Pharma, Inc.
Study to Evaluate the Efficacy, Safety, and Tolerability of Efzofitimod in Patients With Systemic Sclerosis (SSc)-Related Interstitial Lung Disease (ILD) (SSc-ILD)
What this study is about
This is a 2-Part study with Part A, a where neither patients nor doctors know which treatment is given, randomly assigned, compared against an inactive treatment, PoC study to evaluate the effectiveness, safety, and tolerability of efzofitimod in patients with SSc-ILD. The primary objective of the study is to evaluate the PoC for effectiveness in a population with SSc-ILD.
View original scientific description
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.
Interventions
DRUG
efzofitimod 450 mg
IV infusion over approximately 60 minutes every 4 weeks
DRUG
efzofitimod 270 mg
IV infusion over approximately 60 minutes every 4 weeks
DRUG
Placebo
IV infusion over approximately 60 minutes every 4 weeks
Primary outcome measures
Absolute change from baseline in forced vital capacity (FVC) in mL
Time frame: 24 weeks
Annual rate of decline in FVC in mL
Time frame: 24 weeks
Annual rate of decline in FVC in percent predicted
Time frame: 24 weeks
Change in HRCT fibrosis score
Time frame: Baseline to Week 24
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- 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
- Pulmonary disease with FVC %pred ≤ 45% OR D
Where
- Los Angeles, California
- San Diego, California
- Miami, Florida
- Chicago, Illinois
- New Orleans, Louisiana
- New York, New York
- Cleveland, Ohio
- Oklahoma City, Oklahoma
- Charleston, South Carolina
- Dallas, Texas
- Houston, Texas
- Salt Lake City, Utah
And 1 more location — see the full list below.
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced May 7, 2025 · Source of record for eligibility and locations