Long-term evaluation of AT-02 for systemic amyloidosis

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

This study is testing the long-term safety and effectiveness of a treatment called AT-02 for people with systemic amyloidosis who have already taken part in a previous study.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 120 (estimated)
• Ages: 18 Years to 85 Years
• Sex: All
• Sponsor: Attralus, Inc. (Industry-sponsored)
• Locations: 10 sites (Overland Park, Kansas and 9 other locations)
• Trial ID: NCT05951049 on ClinicalTrials.gov

What this trial studies

This Phase 2 open-label extension study aims to assess the long-term safety, tolerability, and clinical activity of AT-02 in subjects with systemic amyloidosis who have previously participated in the AT02-001 study. Participants will undergo a screening period of 56 days, followed by a treatment period lasting up to 104 weeks, and a follow-up period extending to week 112. A Safety Review Committee will monitor safety signals throughout the study to ensure participant well-being.

Who should consider this trial

Good fit: Ideal candidates are individuals with a confirmed diagnosis of systemic amyloidosis who have previously participated in the AT02-001 study.

Not a fit: Patients who have not participated in the AT02-001 study or those with significant adverse events during that study may not benefit from this trial.

Why it matters

Potential benefit: If successful, this study could provide a new treatment option for patients suffering from systemic amyloidosis.

How similar studies have performed: While this approach is part of an ongoing investigation, previous studies involving AT-02 have shown promise in treating systemic amyloidosis.

Eligibility criteria

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.

Where this trial is running

Overland Park, Kansas and 9 other locations

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

Study contacts

• Study coordinator: Scott Stephens

Conditions Amyloidosis; Systemic