Clinical trial NCT05892614: 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
Overall status: RECRUITING
Phase: PHASE2
Conditions: Interstitial Lung Disease
Keywords: ILD
SSc-ILD
Interstitial Lung Disease
lung inflammation
fibrosis
pulmonary function
efzofitimod
systemic sclerosis
Lead sponsor: aTyr Pharma, Inc.
Sponsor class: INDUSTRY
First posted: 2023-06-07
Enrollment: 25
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility criteria: 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

Primary outcomes:
measure: Absolute change from baseline in forced vital capacity (FVC) in mL
timeFrame: 24 weeks
measure: Annual rate of decline in FVC in mL
timeFrame: 24 weeks
measure: Annual rate of decline in FVC in percent predicted
timeFrame: 24 weeks
measure: Change in HRCT fibrosis score
timeFrame: Baseline to Week 24
Secondary outcomes:
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: aTyr Investigative Site
state: California
city: San Diego
country: United States
facility: aTyr Investigative Site
state: California
city: Miami
country: United States
facility: aTyr Investigative Site
state: Florida
city: Chicago
country: United States
facility: aTyr Investigative Site
state: Illinois
city: Chicago
country: United States
facility: aTyr Investigative Site
state: Illinois
city: Chicago
country: United States
facility: aTyr Investigative Site
state: Illinois
city: New Orleans
country: United States
facility: aTyr Investigative Site
state: Louisiana
city: New York
country: United States
facility: aTyr Investigative Site
state: New York
city: Cleveland
country: United States
facility: aTyr Investigative Site
state: Ohio
city: Oklahoma City
country: United States
facility: aTyr Investigative Site
state: Oklahoma
city: Charleston
country: United States
facility: aTyr Investigative Site
state: South Carolina
city: Dallas
country: United States
facility: aTyr Investigative Site
state: Texas
city: Houston
country: United States
facility: aTyr Investigative Site
state: Texas
city: Salt Lake City
country: United States
facility: aTyr Investigative Site
state: Utah
city: Richmond
country: United States
facility: aTyr Investigative Site
state: Virginia
Study officials:
affiliation: aTyr Pharma, Inc.
name: Lisa Carey
role: STUDY_DIRECTOR
References: