Study to Evaluate the Efficacy, Safety, and Tolerability of Efzofitimod in Patients With Systemic Sclerosis (SSc)-Related Interstitial Lung Disease (ILD) (SSc-ILD)
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: efzofitimod 450 mg, efzofitimod 270 mg, Placebo.
- Who it may be relevant to
- Registry conditions: Interstitial Lung Disease. Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
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)
Overview
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.
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]
Eligibility criteria
Inclusion criteria
- Diagnosis of SSc based on ACR/ EULAR criteria (2013)
- Overall duration of SSc < 84 months from the first non-Raynaud symptom manifestation prior to Day 1
- 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
- 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 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 Day 1
- Treatment with more than 1 immunosuppressant (e.g., MMF, methotrexate \[MTX\], azathioprine \[AZA\], or leflunomide)
- 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)
- 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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Parallel assignment
- Masking
- Triple blind
- Primary purpose
- Treatment
Study locations
United States · 15 centers
- aTyr Investigative Site — Los Angeles
- aTyr Investigative Site — San Diego
- aTyr Investigative Site — Miami
- aTyr Investigative Site — Chicago
- aTyr Investigative Site — Chicago
- aTyr Investigative Site — Chicago
- aTyr Investigative Site — New Orleans
- aTyr Investigative Site — New York
- … and 7 more centers
Identifiers
NCT: NCT05892614 · ATYR1923-C-005