Heart failure & cardiomyopathies
CRISPR-Cas9 Gene Editing with Nexiguran Ziclumeran for ATTR Cardiomyopathy
Authors' conclusion
In this phase 1 study involving patients with ATTR-CM, treatment with a single dose of nex-z was associated with transient infusion-related reactions and consistent, rapid, and durable reductions in serum TTR levels.
Study context
- Population
- Patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM)≥12 months follow-up
- Participants
- 36 patients
- Outcome
- Safety and pharmacodynamics, including serum TTR level
Methodological detailssingle-group
- Single-group
Main finding
As reported in the abstract
Mean % change in serum TTR at 28 days
−89%
95% CI −92 to −87
Mean % change in serum TTR at 12 months
−90%
95% CI −93 to −87
Secondary outcomes
NT-proBNP, geometric mean factor change from baseline · month 12
- Geometric mean factor change in NT-proBNP
- 1.02 (95% CI 0.88 to 1.17)
High-sensitivity cardiac troponin T, geometric mean factor change from baseline · month 12
- Geometric mean factor change in hs-cTnT
- 0.95 (95% CI 0.89 to 1.01)
6-minute walk distance, median change from baseline · month 12
- Median change in 6-minute walk distance (IQR −33 to 49)
- 5 m
New York Heart Association (NYHA) class
- Improvement or no change in NYHA class
- 92%
Safety
- Patients with adverse events
- 34
- Transient infusion-related reactions
- 5
- Transient liver-enzyme elevations, treatment-related
- 2
- Patients with serious adverse events (most consistent with ATTR-CM)
- 14
Original publication
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