A Study of AT-02 in Subjects With Systemic Amyloidosis.

NCT05951049 | Active Not Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: AT02-003
• Study Type: interventional
• Target: 120
• Locations: 1 country
• Sites: 4

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.

Conditions

Amyloidosis; Systemic

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (12)

• To assess PK of AT-02 during long-term administration

  Up to 112 weeks

• To assess PK of AT-02 during long-term administration

  Up to 112 weeks

• To assess PK of AT-02 during long-term administration

  Up to 112 weeks

• To assess PK of AT-02 during long-term administration

  Up to 112 weeks

• To assess PK of AT-02 during long-term administration

  Up to 112 weeks

Study Arms (1)

A (AT-02)

EXPERIMENTAL

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

Drug: AT02

Interventions

AT02 DRUG

Dosage Form: Solution for injection/infusion Dosage level: Different dose levels of AT02 Route of Administration: Intravenous use

A (AT-02)

Eligibility Criteria

• Age: 18 Years - 85 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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
• Either
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• 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.
• Must not be breastfeeding, lactating, or planning a pregnancy during the study period.
• WOCBP who are not exclusively in same-sex relationships must agree to remain abstinent (complete avoidance of heterosexual intercourse) or use adequate contraceptive methods, defined as use of a condom by the male partner combined with use of a highly effective method of contraception by the female partner, during the treatment period and for at least 105 days after the last dose of study intervention.
• Women of non-childbearing potential (WONCBP), and female participants with vasectomized male partners:

You may not qualify if:

• 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.
• History of solid organ transplantation.
• Suspected or known substance abuse disorder(s), serious psychiatric or any other medical condition, which, in the opinion of the Investigator, makes the participant unsuitable.
• Has any concurrent illness that, in the opinion of the Investigator, might confound the results of the study or pose additional risk to the participant.
• Screening alanine transaminase (ALT) or aspartate transaminase (AST) is \>2.5x upper limit of normal (ULN).
• Screening estimated creatinine clearance/eGFR using the CKD-EPI equation is \<20 mL/min/1.73 m2.

Sponsors & Collaborators

• Attralus, Inc.: lead
• Novotech (Australia) Pty Limited: collaborator

Study Sites (4)

Kansas City

Kansas City, Kansas, 64111, United States

Location

Cleveland Clinic

Cleveland, Ohio, 44195, United States

Location

OHSU (Oregon Health & Science University)

Portland, Oregon, 97239, United States

Location

Penn Presbyterian Medical Center

Philadelphia, Pennsylvania, 19104, United States

Location

MeSH Terms

Conditions

Immunoglobulin Light-chain Amyloidosis

Condition Hierarchy (Ancestors)

Neoplasms, Plasma Cell; Neoplasms by Histologic Type; Neoplasms; Amyloidosis; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases; Lymphoproliferative Disorders; Immunoproliferative Disorders; Immune System Diseases; Paraproteinemias

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 29, 2023
• First Posted: July 18, 2023
• Study Start: September 21, 2023
• Primary Completion (Estimated): September 28, 2026
• Study Completion (Estimated): September 28, 2026
• Last Updated: May 6, 2026

Record last verified: 2026-05

Locations

US (4)