Kristen Hsu, ARC’s Executive Director of Research, shared the latest updates on ATTR (transthyretin amyloidosis) clinical trials and what these developments mean for patients and families. There was also an opportunity for questions and answers.
Original Presentation Date:
July 22, 2026
0:00 — Welcome
2:21 — Introduction
4:59 — What is a Clinical Trial?
6:07 — Approaches to Treating ATTR Amyloidosis
12:23 — ATTR Amyloidosis Clinical Trials
13:16 — Changes Since January 2026
14:21 — CARDIO-TTRansform (gene silencer; ATTR-CM)
21:40 — Trials Currently Recruiting in 2026
22:10 — ACT-EARLY (TTR stabilizer; asymptomatic carriers of TTR mutations)
28:11 — TRITON-CM (gene silencer; ATTR-CM)
32:33 — TRITON-PN (gene silencer; ATTR-PN)
35:25 — MAGNITUDE (gene editing; ATTR-CM)
40:17 — MAGNITUDE-2 (gene editing; hereditary ATTR-PN)
42:30 — CLEOPATTRA (anti-amyloid fibril; ATTR-CM)
46:02 — Upcoming Milestones for ATTR Therapies
46:37 — ASCEND-ATTR (TTR stabilizer; ATTR-CM)
48:26 — DepleTTR-CM (amyloid depleter; ATTR-CM)
52:42 — Other Recruiting and Planned Studies
54:48 — How to Find Clinical Trials
57:04 — Q&A Session
57:40 — What is a Phase 3 trial?
1:00:02 — Do people still receive the drug at the end of a study?
1:01:42 — Why would standard of care vary from place to place?
1:03:20 — Why are studies in CM or PN when ATTR is one disease?
1:05:58 — What is considered first symptoms for the ACT-EARLY study?
1:07:02 — What’s the difference between “gene positive” and having hereditary ATTR?
1:08:40 — Why is a high level of NTpro-BNP required for studies trying to give treatment early?
1:10:45 — If NTpro-BNP levels change can you be re-screened for at trial?
1:11:24 — Is there a reason I might not feel better even if I’m on a treatment?
1:12:48 — Is there evidence for one treatment being better than another? Should I consider being on different types of treatments?
1:15:46 — Conclusion
Contact ARC:
Call +1 (617) 467-5170
Email ARC at support@arci.org
