Heart failure & cardiomyopathies
REALM-DCM: A Phase 3, Multinational, Randomized, Placebo-Controlled Trial of ARRY-371797 in Patients With Symptomatic LMNA -Related Dilated Cardiomyopathy
- terminated after planned interim analysis suggested futility
- Terminated after a planned interim analysis suggested futility
Authors' conclusion
Findings from REALM-DCM demonstrated futility without safety concerns.
Study context
- Population
- Symptomatic LMNA-related dilated cardiomyopathy: NYHA class II/III, LVEF ≤50%, ICD, reduced 6-minute walk distanceaged 23–72 years
- Participants
- 77 participants
- Comparison
- ARRY-371797 400 mg twice daily vs placebo
- ARRY-371797n = 40
- Placebon = 37reference
- Primary endpoint
- Change from baseline in 6-minute walk test distance
- Follow-up
- Week 24
Methodological detailsphase 3, multinational, double-blind, placebo-controlled, Enrolment April 2018 to October 2022
- Phase 3
- Multinational
- Double-blind
- Placebo-controlled
- Enrolment April 2018 to October 2022
Primary result
As reported in the abstract
Change in 6-minute walk test distance
No significant difference between groups; median difference
4.9 m
95% CI −24.2 to 34.1; P=0.82
Secondary outcomes
Change in KCCQ physical limitation score · week 24
- No significant difference between groups
- 2.4 (95% CI −6.4 to 11.2; P=0.54)
Change in KCCQ total symptom score · week 24
- No significant difference between groups
- 5.3 (95% CI −4.3 to 14.9; P=0.48)
Change in NT-proBNP concentration · week 24
- No significant difference between groups
- −339.4 pg/mL (95% CI −1131.6 to 452.7; P=0.17)
Composite of worsening HF or all-cause mortality
- Similar between groups; HR
- 0.43 (95% CI 0.11-1.74; P=0.23)
Overall survival
- Similar between groups; HR
- 1.19 (95% CI 0.23-6.02; P=0.84)
Original publication
REALM-DCM: A Phase 3, Multinational, Randomized, Placebo-Controlled Trial of ARRY-371797 in Patients With Symptomatic LMNA -Related Dilated Cardiomyopathy. Circ Heart Fail. Published 9 July 2024. doi:10.
Summarised from the published abstract; every number is checked against it