A Phase 2 Randomized, Double-blind, Placebo-Controlled Study of the Safety and Efficacy of MTX-474 in Participants with Diffuse Cutaneous Systemic Sclerosis (dcSSc)
Participants with dcSSc who meet the study's inclusion and exclusion criteria will be randomly assigned in a 3:2 ratio to receive MTX-474 or a matching placebo by intravenous (IV) infusion. Concomitant use of one of the approved dcSSc therapies (immunosuppressive therapy, systemic glucocorticoids or other antifibrotic agents) is permitted under certain criteria. Participants randomized to the MTX-474 arm of the study will receive an IV infusion every 4 weeks, beginning at Day 0 and ending at Week 20. The End of Treatment Visit will occur at Week 24, and a Safety Follow-Up Visit will occur at Week 28, 8 weeks after the final infusion. mRSS assessments will occur at Screening, Baseline, and at all subsequent treatment visits up to and including Week 24. Spirometry will be performed at screening and Weeks 12. HRCT will be performed at screening and week 24. DLCO will be performed at Screening. Skin biopsies will be performed at Baseline and Week 12. Participants will have blood drawn for safety assessment and to assess Ephrin receptor levels at Screening, Baseline and every 4 weeks until Week 28. Blood will be drawn for serum PK analyses relative to the first and last doses of MTX-474.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
85
Dosage level: 4 mg/kg Unit dose strength: 50mg/ml MTX-474 is a human immunoglobulin G1 (IgG1) monoclonal antibody that binds the human EphrinB2 with high specificity and high affinity. MTX-474 is being developed as a therapy for patients with systemic sclerosis (SSc).
Placebo
EncompaSSc site in Newport Beach, CA 92663
Newport Beach, California, United States
EncompaSSc site in Clearwater, FL 33765
Clearwater, Florida, United States
Change from baseline in Modified Rodnan Skin Score (mRSS)
Mean change from baseline to week 24 in the modified Rodnan Skin Score, a clinician-assessed measure of skin thickness across 17 body areas. Unit of measure: Score (range 0-51)
Time frame: Baseline to week 24
Proportion of participants with a gene signature response in skin biopsy
Proportion of participants in each arm (MTX-474 and placebo) demonstrating a dcSSc gene expression signature response on skin biopsy at Week 12. Unit of Measure: Participants (%)
Time frame: 12 weeks
Number of participants with clinically significant findings on physical examination, vital signs, and clinical laboratory tests
Number of participants with clinically significant findings on physical examination, vital signs, and clinical laboratory tests Unit of Measure: Participants (%)
Time frame: Baseline through end of study (week 28)
Number of participants with dose interruptions or treatment discontinuations due to adverse events
Number of participants with dose interruptions or treatment discontinuations due to adverse events Unit of Measure: Participants (%)
Time frame: Baseline through end of study (week 28)
Safety and tolerability of MTX-474 in participants with dcSSc: Number of participants with treatment-emergent adverse events (TEAEs) and treatment-related adverse events (TRAEs)
Incidence, seriousness, and severity of TEAEs and TRAEs as determined by investigator assessment. Unit of measure: Participants (%)
Time frame: Baseline through end of study (week 28)
Serum concentration of MTX-474 at sparse PK time points
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Serum MTX-474 concentrations collected at predefined sparse PK time points to support population pharmacokinetic (popPK) analysis Unit of Measure: ng/mL
Time frame: Baseline to week 28
Clearance (CL) of MTX-474
Clearance of MTX-474 calculated from serial PK sampling using noncompartmental analysis Unit of Measure: L/hour
Time frame: Baseline to week 28
Terminal elimination half-life (t½) of MTX-474
Terminal elimination half-life of MTX-474 derived from serial PK sampling. Unit of Measure: Hours
Time frame: Baseline to week 28
Area under the plasma concentration-time curve (AUC) of MTX-474
AUC of MTX-474 calculated from serial PK sampling using noncompartmental analysis. Unit of Measure: ng·h/mL
Time frame: Baseline to week 28