A Study of AT-02 in Subjects With Systemic Amyloidosis.
- Registration Number
- NCT05951049
- Lead Sponsor
- Attralus, Inc.
- Brief Summary
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.
- Detailed Description
The study will enroll subjects with systemic amyloidosis who have participated in AT02-001 study.
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.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 120
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Subject understands the study procedures and can give signed informed consent.
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Subject is willing and able to comply with this protocol and will be available for the entire duration of the study.
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Subject must have a confirmed diagnosis of SA per the diagnostic criteria specified in the parent study protocol.
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Subject must have participated in the study AT01-001 and wishes to receive open-label AT-02.
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AT02-001 Part 2:
a. Subjects must have completed the last follow-up visit in AT02-001 Part 2 without significant adverse events, as determined by the Investigator.
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AT02-001 Part 3:
a. Subjects must have completed the post-treatment imaging studies in AT02-001Part 3 (e.g., CMR, echocardiogram) without significant AEs in the parent study as determined by the Investigator.
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Must continue to satisfy the eligibility criteria in the parent study protocol for WOCBP, WONCBP, or male participants
- Is pregnant, breastfeeding, or is planning to become pregnant or breastfeed during this study and follow-up period.
- Is mentally or legally incapacitated, has significant emotional problems at the time of the study, or has a history of psychosis.
- Has acquired any new, clinically significant underlying illness since enrollment in the parent study.
- Has any clinically significant worsening of organ function associated with underlying SA or clinically significant change in concomitant medications for the treatment of SA since enrollment in the parent study.
- Estimated glomerular filtration (eGFR) ≤30 mL/min/1.73 m2.
- Currently using any prohibited concomitant medications.
- Any contraindication to MRI or MRI contrast.
- Is currently participating in an interventional clinical study or has participated in another clinical study (other than AT02-001) within the last four (4) weeks or within five (5) half-lives of the prior study treatment, whichever is longer.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description A (AT-02) AT02 Subjects will receive AT-02 via intravenous infusion once every two or 4 weeks for 104 weeks (52 total AT-02 administrations).
- Primary Outcome Measures
Name Time Method 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) Up to 112 weeks To assess the safety and tolerability of AT-02 through change from baseline in clinical laboratory results Up to 112 weeks
- Secondary Outcome Measures
Name Time Method Serial cardiac magnetic resonance assessments of systemic amyloidosis Up to 112 weeks To assess PK of AT-02 during long-term administration Up to 112 weeks Parameter: AT-02 half-life (t½)
To evaluate the clinical efficacy of AT-02 during long-term administration through change from baseline in biomarkers Up to 112 weeks Biomarkers include serum Urine albumin creatinine ratio (UACR)
Incidence of treatment-emergent Anti-drug antibodies (ADAs) Up to 112 weeks The number and percentage of subjects who develop detectable ADA will be summarized by dose cohort.
Trial Locations
- Locations (10)
Johns Hopkins
🇺🇸Baltimore, Maryland, 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
Princess Alexandra Hospital
🇦🇺Woolloongabba, Queensland, Australia
Flinders Medical Centre
🇦🇺Bedford Park, South Australia, Australia
Box Hill Hospital
🇦🇺Box Hill, Victoria, Australia
Royal Perth Hospital
🇦🇺Perth, Western Australia, Australia
Midwest Heart and Vascular
🇺🇸Overland Park, Kansas, United States
Royal Free London Nhs Foundation Trust Royal Free Hospital
🇬🇧London, United Kingdom