A Study of ARRY-371797 (PF-07265803) in Patients With Symptomatic Dilated Cardiomyopathy Due to a Lamin A/C Gene Mutation

Summary

This is a randomized, double-blind, placebo-controlled study in patients with dilated cardiomyopathy (DCM) due to a mutation of the gene encoding the lamin A/C protein (LMNA). The study will further evaluate a dose level of study drug (ARRY-371797) that has shown preliminary efficacy and safety in this patient population. After the primary analysis has been performed, eligible patients may receive open-label treatment with ARRY-371797.

Timeline

Start

2018-04-17

Primary completion

2022-10-13

Completion

2022-10-13

Publications

• Garcia-Pavia P, Cai X, Di J, Li H, Demanuele C, Bruno J, Hinnershitz TM, Angeli FS, MacRae CA. Actigraphy-Quantified Physical Activity Measurements in Patients with Symptomatic LMNA-Related Dilated Cardiomyopathy in REALM-Dilated Cardiomyopathy. Digit Biomark. 2026 Jan 6;10(1):32-40. doi: 10.1159/000550259. eCollection 2026 Jan-Dec.
• Garcia-Pavia P, Lakdawala NK, Sinagra G, Ripoll-Vera T, Afshar K, Priori SG, Ware JS, Owens A, Li H, Angeli FS, Elliott P, MacRae CA, Judge DP. Characterization and natural history of patients with LMNA-related dilated cardiomyopathy in the phase 3 REALM-DCM trial. ESC Heart Fail. 2024 Dec;11(6):4201-4208. doi: 10.1002/ehf2.14955. Epub 2024 Aug 15.
• Garcia-Pavia P, Palomares JFR, Sinagra G, Barriales-Villa R, Lakdawala NK, Gottlieb RL, Goldberg RI, Elliott P, Lee P, Li H, Angeli FS, Judge DP, MacRae CA; REALM-DCM Investigators. REALM-DCM: A Phase 3, Multinational, Randomized, Placebo-Controlled Trial of ARRY-371797 in Patients With Symptomatic LMNA-Related Dilated Cardiomyopathy. Circ Heart Fail. 2024 Jul;17(7):e011548. doi: 10.1161/CIRCHEARTFAILURE.123.011548. Epub 2024 Jul 9.
• Judge DP, Taylor MR, Li H, Oliver C, Angeli FS, Lee PA, MacRae CA. Long-term effectiveness of ARRY-371797 in people with dilated cardiomyopathy and a faulty LMNA gene: a plain language summary. Future Cardiol. 2023 Mar;19(3):117-126. doi: 10.2217/fca-2022-0125. Epub 2023 Apr 3.
• MacRae CA, Taylor MR, Mestroni L, Moses J, Ashley EA, Wheeler MT, Lakdawala NK, Hershberger RE, Sandor V, Saunders ME, Oliver C, Lee PA, Judge DP. Plain Language Summary of Publication of the safety and efficacy of ARRY-371797 in people with dilated cardiomyopathy and a faulty LMNA gene. Future Cardiol. 2023 Feb;19(2):55-63. doi: 10.2217/fca-2022-0099. Epub 2023 Jan 31.

Drugs

Indications