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

Amyloidosis; Systemic Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05951049
• Other study ID #: AT02-003
• Status: Recruiting
• Phase: Phase 2
• Start date: September 21, 2023
• Est. completion date: February 28, 2026

Study information

• Verified date: January 2024
• Source: Attralus, Inc.
• Contact: Scott Stephens
• Phone: +1-321-228-7400
• Email: sstephens[@]attralus.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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 information / eligibility

• Status: Recruiting
• Enrollment: 120
• Est. completion date: February 28, 2026
• Est. primary completion date: February 28, 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 85 Years

Eligibility

Inclusion Criteria:

1. Subject understands the study procedures and can give signed informed consent.

2. Subject is willing and able to comply with this protocol and will be available for the entire duration of the study.

3. Subject must have a confirmed diagnosis of SA per the diagnostic criteria specified in the parent study protocol.

4. Subject must have participated in the study AT01-001 and wishes to receive open-label AT-02.

5. 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.

6. 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.

7. Must continue to satisfy the eligibility criteria in the parent study protocol for WOCBP, WONCBP, or male participants

Exclusion Criteria:

1. Is pregnant, breastfeeding, or is planning to become pregnant or breastfeed during this study and follow-up period.

2. Is mentally or legally incapacitated, has significant emotional problems at the time of the study, or has a history of psychosis.

3. Has acquired any new, clinically significant underlying illness since enrollment in the parent study.

4. 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.

5. Estimated glomerular filtration (eGFR) =30 mL/min/1.73 m2.

6. Currently using any prohibited concomitant medications.

7. Any contraindication to MRI or MRI contrast.

8. 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.

Related Conditions & MeSH terms

• Amyloidosis
• Amyloidosis; Systemic

Intervention

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

Locations

Country	Name	City	State
Australia	Flinders Medical Centre	Bedford Park	South Australia
Australia	Box Hill Hospital	Box Hill	Victoria
Australia	Royal Perth Hospital	Perth	Western Australia
Australia	Princess Alexandra Hospital	Woolloongabba	Queensland
United Kingdom	Royal Free London Nhs Foundation Trust Royal Free Hospital	London
United States	Johns Hopkins	Baltimore	Maryland
United States	Cleveland Clinic	Cleveland	Ohio
United States	Midwest Heart and Vascular	Overland Park	Kansas
United States	Penn Presbyterian Medical Center	Philadelphia	Pennsylvania
United States	OHSU (Oregon Health & Science University)	Portland	Oregon

Sponsors (2)

Lead Sponsor	Collaborator
Attralus, Inc.	Novotech (Australia) Pty Limited

Countries where clinical trial is conducted

United States,  Australia,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	To assess the safety and tolerability of AT-02 through change from baseline in clinical laboratory results		Up to 112 weeks
Secondary	To assess PK of AT-02 during long-term administration	Parameter: maximum observed concentration of AT-02 (Cmax)	Up to 112 weeks
Secondary	To assess PK of AT-02 during long-term administration	Parameter: time to maximum observed AT-02 concentration (Tmax)	Up to 112 weeks
Secondary	To assess PK of AT-02 during long-term administration	Parameter: AUClast	Up to 112 weeks
Secondary	To assess PK of AT-02 during long-term administration	Parameter: AUCinf	Up to 112 weeks
Secondary	To assess PK of AT-02 during long-term administration	Parameter: volume of distribution at steady state (Vss)	Up to 112 weeks
Secondary	To assess PK of AT-02 during long-term administration	Parameter: total body clearance (CL) of AT-02	Up to 112 weeks
Secondary	To assess PK of AT-02 during long-term administration	Parameter: AT-02 half-life (t½)	Up to 112 weeks
Secondary	Incidence of treatment-emergent Anti-drug antibodies (ADAs)	The number and percentage of subjects who develop detectable ADA will be summarized by dose cohort.	Up to 112 weeks
Secondary	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)	Up to 112 weeks
Secondary	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)	Up to 112 weeks
Secondary	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)	Up to 112 weeks
Secondary	Serial cardiac magnetic resonance assessments of systemic amyloidosis		Up to 112 weeks