Active — Not RecruitingPhase 2

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

United States120 participantsStarted 2023-09-21

Plain-language 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.

Who can participate

Age range18 Years – 85 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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
✓. Either
✓. 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.

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.

What they're measuring

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)

Timeframe: Up to 112 weeks

To assess the safety and tolerability of AT-02 through change from baseline in clinical laboratory results

Timeframe: Up to 112 weeks

Trial details

NCT IDNCT05951049

SponsorAttralus, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2026-09-28

View on ClinicalTrials.gov More Amyloidosis; Systemic trials

Who can participate

Age range18 Years – 85 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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
✓. Either
✓. 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.

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.

What they're measuring

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)

Timeframe: Up to 112 weeks

To assess the safety and tolerability of AT-02 through change from baseline in clinical laboratory results

Timeframe: Up to 112 weeks