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ARC Executive Director of Research Kristen Hsu presented the most current updates for the clinical trial landscape, with a focus on ATTR amyloidosis. Kristen will discussed the direction of future research.

The webinar concluded with a Q&A session.

Original Presentation Date:
January 22, 2025

Download the Slides →

My Amyloidosis Pathfinder →

0:00 — Introduction

2:42 — Webinar Overview

5:26 — What is a Clinical Trial? What’s involved?

11:55 — What makes a study a good fit for me?

16:25 — Investigative Approaches to Treating ATTR Amyloidosis

23:46 — ATTR Amyloidosis Clinical Trial Overview

25:36 — ACT-EARLY (acoramidis; asymptomatic carriers of TTR mutations)

32:58 — MAGNITUDE (Nex-Z/NTLA-2001; ATTR-CM wildtype or hereditary)

37:52 — MAGNITUDE-2 (Nex-Z/NTLA-2001; ATTR-PN hereditary only)

39:31 — DepleTTR-CM (ALXN2220; ATTR-CM wild type or hereditary)

42:21 — Upcoming Milestones for Novel ATTR Therapies

43:05 — HELIOS-B (vutrisiran; ATTR-CM wildtype or hereditary)

44:29 — CARDIO-TTRansform (eplontersen; ATTR-CM wildtype or hereditary)

45:45 — Coramitug (NNC6019-0001; ATTR-CM wildtype or hereditary)

47:02 — AT-02 (Cardiac AL and ATTR amyloidosis)

47:53 — Future Trials for Novel ATTR Therapies

49:37 — Other Recruiting and Planned Clinical Trials

50:29 — REVEAL (I-124 Evuzamitide; suspected cardiac amyloidosis)

52:47 — CArdiag ([18F]Florbetaben; suspected cardiac amyloidosis)

54:36 — How to Find Clinical Trials

56:12 — Q&A Session

57:01 — What’s t he difference between neuropathy with wild type vs hereditary ATTR amyloidosis?

59:48 — What does “age of onset” mean with hereditary ATTR?

1:00:37 — Can wild type patients participate in the polyneuropathy studies?

1:01:22 — Why do symptoms have to be so significant to participate in trials?

1:04:15 — Why isn’t ATTR Wild Type included in the ACT-EARLY study?

1:05:17 — Are Carpal Tunnel and Spinal Stenosis caused by infiltration of the nerve or deposition surrounding the nerve?

1:06:41 — How do I enroll in a clinical trial? Are these trials available in Canada?

1:09:02 — Are there preventative criteria to trials (e.g. heart transplants, age of patients)?

1:11:12 — Conclusion

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