This is a Phase 2 open-label extension study to evaluate the long-term safety, tolerability, and clinical activity of AT-02. AT-02 is an investigational medicinal product being developed to treat systemic amyloidosis.
The study will enroll subjects with systemic amyloidosis who have participated in AT02-001 study and will also directly enroll AL participants with renal disease who did not participate in study AT02-001. The study includes screening period (56 days), treatment period (week 104), follow up (week 112). The total duration of participant in study is up to 120 weeks. A Safety Review Committee (SRC) will periodically convene and review all available clinical and laboratory data during the study. A single SRC will monitor safety across all AT-02 studies to ensure that safety signals are assessed in aggregate.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
120
Dosage Form: Solution for injection/infusion Dosage level: Different dose levels of AT02 Route of Administration: Intravenous use
Kansas City
Kansas City, Kansas, United States
Cleveland Clinic
Cleveland, Ohio, United States
OHSU (Oregon Health & Science University)
Portland, Oregon, United States
Penn Presbyterian Medical Center
Philadelphia, Pennsylvania, United States
Incidence, frequency, and severity of Treatment-emergent adverse events (TEAEs) as assessed National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE version 5.0)
Time frame: Up to 112 weeks
To assess the safety and tolerability of AT-02 through change from baseline in clinical laboratory results
Time frame: Up to 112 weeks
To assess PK of AT-02 during long-term administration
Parameter: maximum observed concentration of AT-02 (Cmax)
Time frame: Up to 112 weeks
To assess PK of AT-02 during long-term administration
Parameter: time to maximum observed AT-02 concentration (Tmax)
Time frame: Up to 112 weeks
To assess PK of AT-02 during long-term administration
Parameter: AUClast
Time frame: Up to 112 weeks
To assess PK of AT-02 during long-term administration
Parameter: AUCinf
Time frame: Up to 112 weeks
To assess PK of AT-02 during long-term administration
Parameter: volume of distribution at steady state (Vss)
Time frame: Up to 112 weeks
To assess PK of AT-02 during long-term administration
Parameter: total body clearance (CL) of AT-02
Time frame: Up to 112 weeks
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To assess PK of AT-02 during long-term administration
Parameter: AT-02 half-life (t½)
Time frame: Up to 112 weeks
Incidence of treatment-emergent Anti-drug antibodies (ADAs)
The number and percentage of subjects who develop detectable ADA will be summarized by dose cohort.
Time frame: Up to 112 weeks
To evaluate the clinical efficacy of AT-02 during long-term administration through change from baseline in biomarkers
Biomarkers include serum N-terminal prohormone of brain natriuretic peptide (NT-proBNP)
Time frame: Up to 112 weeks
To evaluate the clinical efficacy of AT-02 during long-term administration through change from baseline in biomarkers
Biomarkers include serum High-sensitivity cardiac troponin T (hsTnT)
Time frame: Up to 112 weeks
To evaluate the clinical efficacy of AT-02 during long-term administration through change from baseline in biomarkers
Biomarkers include serum Urine albumin creatinine ratio (UACR)
Time frame: Up to 112 weeks
Serial cardiac magnetic resonance assessments of systemic amyloidosis
Time frame: Up to 112 weeks