A Phase 2, Open-label Extension Study to Evaluate the Long-term Safety and Tolerability of AT-02

Attralus, Inc.4 sites in 1 country120 target enrollmentSeptember 21, 2023

ConditionsAmyloidosis; Systemic

InterventionsAT02

DrugsAT02

Overview

Phase: Phase 2
Intervention: AT02
Conditions: Amyloidosis; Systemic
Sponsor: Attralus, Inc.
Enrollment: 120
Locations: 4
Primary Endpoint: 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)
Status: Recruiting
Last Updated: 8 months ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

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 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.

Registry

clinicaltrials.gov

Start Date

September 21, 2023

End Date

February 28, 2026

Last Updated

8 months ago

Study Type

Interventional

Study Design

Single Group

Sex

All

Investigators

Sponsor

Attralus, Inc.

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•Subject understands the study procedures and can give signed informed consent.
•Subject is willing and able to comply with this protocol and will be available for the entire duration of the study.
•Has a confirmed diagnosis of AL amyloidosis and meets the criteria below:
•Histologic confirmation with a biopsy containing deposits of apple-green birefringent, Congophilic material or other amyloid staining (i.e., thioflavin T or sulfated alcian blue) with confirmatory immunohistochemistry or mass spectrometry, AND
•May be receiving maintenance daratumumab and must have achieved and maintained a hematologic very good partial response (VGPR) or complete response (CR), have completed chemotherapy therapy (ie, melphalan, bortezomib, thalidomide, lenalidomide, or cyclophosphamide) and be at least 6 months from first hematologic response (CR or VGPR), AND
•Screening eGFR ≥20 and ≤75 mL/min/1.73m2 based on CKD-EPI equation, OR
•Proteinuria that is not improving (e.g., \<25% reduction in urine protein creatinine ratio (UPCR) in the last 12 months or since hematologic response, whichever is shorter) with screening urine albumin creatinine ratio (UACR) \>700 mg/g based on central lab assessment of first morning void urine collected at screening and confirmed by a separate Screening 24-hr urine protein \>1.0gm/day; 24-hr urine protein may be repeated once during Screening.
•Note: Participants may meet both eGFR and proteinuria criteria
•Women of childbearing potential (WOCBP):
•WOCBP must have a negative serum or urine pregnancy test within 24 hours prior to the start of study treatment.

Exclusion Criteria

•Receiving hemodialysis or peritoneal dialysis.
•Myocardial infarction within 3 months of Screening.
•New York Heart Association Class IV heart failure.
•Kidney disease not caused by AL amyloidosis.
•Respiratory insufficiency requiring oxygen therapy.
•Currently receiving: melphalan, bortezomib, thalidomide, lenalidomide, or cyclophosphamide.
•Currently receiving unfractionated heparin or heparin analogs (e.g., enoxaparin, dalteparin).
•Active malignancy with exception of basal cell carcinoma of the skin, curatively resected squamous cell carcinoma of the skin, curatively treated in situ cervical cancer, non-metastatic prostate adenocarcinoma stably managed on hormonal therapy by medical oncologist or for which appropriate management is observation alone.
•Uncontrolled or active infection.
•Autoimmune disease requiring treatment with immunosuppressive/modulating treatment in the last year.

Arms & Interventions

A (AT-02)

Subjects will receive AT-02 via intravenous infusion once every two or 4 weeks for 104 weeks (52 total AT-02 administrations).

Intervention: AT02

Outcomes

Primary Outcomes

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

Secondary Outcomes

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)
To evaluate the clinical efficacy of AT-02 during long-term administration through change from baseline in biomarkers(Up to 112 weeks)
Incidence of treatment-emergent Anti-drug antibodies (ADAs)(Up to 112 weeks)