Trial Search Results
A Study of ARRY-371797 (PF-07265803) in Patients With Symptomatic Dilated Cardiomyopathy Due to a Lamin A/C Gene Mutation
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.
Stanford is currently not accepting patients for this trial.
- Drug: ARRY-371797 (PF-07265803)
- Other: Placebo
Selected Key Inclusion Criteria:
- Patients with symptomatic lamin A/C protein (LMNA)-related cardiomyopathy Class
II/III/ or Class IV defined as:
- Gene positive for a pathogenic, likely pathogenic, or VUS mutation in the LMNA gene as
determined by an accredited clinical laboratory.
- Evidence of cardiac impairment in LVEF <= 50%
- Patient will have an implantable cardioverter defibrillator/cardiac resynchronization
therapy defibrillator (ICD/CRT-D). ICD implanted at least 4 weeks prior to initiation
of study treatment or CRT-D initiated at least 6 months prior to initiation of study
treatment and defibrillation function activated at least 4 weeks prior to initiation
of study treatment.
- Class II/III patients must have objective functional impairment evidenced by a
reduction in 6-minute walk test (6MWT); a. Screening: 6MWT distance >100 m but ≤450 m,
AND b. Day -1 visit: 6MWT distance >100 m but ≤485 m, AND c. Baseline visit (Day 1):
6MWT distance >100 m but ≤485
- Class II/III patients must be stable for at least 3 months
- Stable medical and/or device therapy consistent with regional American Heart
Association (AHA) / American College of Cardiology (ACC) or European Society of
Cardiology (ESC) guidelines at the investigator discretion, without change in heart
failure drug(s) dose in the past 1 month.
- Patients must meet acceptable hematology, hepatic and renal laboratory values within
35 days prior to Day 1 as specified in the protocol.
Selected Key Exclusion Criteria:
- Presence of other form(s) of cardiomyopathy contributing to HF (eg, inflammatory or
infiltrative cardiomyopathy), clinically significant cardiac anatomic abnormality
(eg,LV aneurysm), clinically significant coronary artery disease (eg, coronary
revascularization, exercise induced angina) or uncorrected, hemodynamically
significant (ie, moderate-severe) primary structural valvular disease not due to HF,
per investigator judgment.
- Currently receiving intermittent or continuous IV inotrope infusion, or presence of a
ventricular assist device, or history of prior heart transplantation. Participants
listed for cardiac transplantation may be enrolled provided transplantation is not
likely to occur in the next 6 months.
- Myocardial infarction, cardiac surgical procedures (other than for pacemaker/ICD/CRT-D
implantation or replacement), acute coronary syndrome, serious systemic infection with
evidence of septicemia, or any major surgical procedure requiring general anesthesia
within 3 months prior to screening.
- Currently receiving or deemed at high risk of requiring chronic renal replacement
therapy (eg, hemodialysis or peritoneal dialysis) within 6 months.
- Initiation of CRT within 6 months prior to screening.
- Treatment with any investigational agent(s) for HF within 35 days prior to Day 1.
- Malignancy that is active or has been diagnosed within 3 years prior to screening,
except surgically curatively resected in situ malignancies or surgically cured early
breast cancer, prostate cancer, skin cancer (basal cell carcinoma, squamous cell
carcinoma), thyroid cancer, or cervical cancer, or, with prior review by the medical
monitor, other early stage surgically curatively resected malignancies with less than
a 20% expected 2 year recurrence rate.
- Non-cardiac condition that limits lifespan to < 1 year.
- Serum positive for hepatitis B surface antigen, viremic hepatitis C, or human
immunodeficiency virus (HIV) at screening.
Ages Eligible for Study
18 Years - N/A
Genders Eligible for Study