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A Study of ARRY-371797 in Patients With LMNA-Related Dilated Cardiomyopathy
Recruiting
Trial ID: NCT02057341
Purpose
This is a Phase 2 pilot study, involving a 48-week treatment period, designed to test the
effectiveness of investigational study drug ARRY-371797 in treating patients with symptomatic
genetic dilated cardiomyopathy due to a lamin A/C gene mutation, and to further evaluate the
drug's safety. Approximately 12 patients from the US will be enrolled in this study.
Eligibility
Key Inclusion Criteria:
- Patients with idiopathic dilated cardiomyopathy and stable New York Heart Association
(NYHA) Class II - IIIa congestive heart failure (CHF).
- Stable, guidelines-based medical and device therapy, without any CHF hospitalizations
or change in heart failure drug dose with ≥ 50% reduction in dose or ≥ 100% increase
in dose in the past 3 months.
- Left ventricular (LV) end diastolic diameter by trans-thoracic echocardiography of >
3.3 cm/m2 (for females) or 3.4 cm/m2 (for males) and/or LV ejection fraction ≤ 45%.
- Gene positive for a pathogenic mutation in the LMNA gene, as determined by a
CLIA-certified clinical laboratory (mutations including but not limited to:
splice-site, non-sense, deletion mutations, a mis-sense mutation in a highly conserved
codon, a mis-sense mutation involving a major charge change, a mis-sense mutation
previously associated with genetic dilated cardiomyopathy).
- Within 3 weeks prior to first dose of study drug, completed distance during six minute
walk test of ≥ 100 m and ≤ 350 m AND/OR ≥ 100 m and ≤ 450 m AND ≤ 60% predicted
distance AND patient is symptomatic for dilated cardiomyopathy per Investigator
judgment.
- On the day before and day of first dose of study drug, completed distance during six
minute walk test of ≥ 100 m and ≤ 400 m (with the greater value within 10% of the
lesser value) AND/OR ≥ 100 m and ≤ 475 m (with the greater value within 10% of the
lesser value) AND patient is symptomatic for dilated cardiomyopathy per Investigator
judgment.
- Acceptable hematology, hepatic and renal function laboratory values within 3 weeks
prior to first dose of study drug.
- Additional criteria exist.
Key Exclusion Criteria:
- Unstable clinical cardiac symptoms requiring unscheduled hospitalization within 60
days prior to study start.
- Clinically significant coronary artery disease, as per Investigator judgment.
- Currently receiving continuous intravenous (IV) inotrope infusion, or presence of a
ventricular assist device, or history of prior heart transplantation.
- Any of the following within 60 days prior to study start: Myocardial infarction,
cardiac surgical procedures, acute coronary syndrome, hemodynamically destabilizing
cardiac arrhythmia, serious systemic infection with evidence of septicemia, any major
surgical procedure requiring general anesthesia.
- Uncorrected, hemodynamically significant primary valvular disease.
- Initiation of cardiac resynchronization therapy within 180 days prior to study start.
- Likelihood, in the Investigator's opinion, of undergoing cardiac transplantation, left
ventricular assist device or other device implantation, or other cardiac surgery
within the next 6 months; or of requiring continuous IV inotropic treatment, or
referral for hospice or end-of-life treatment.
- Active malignancy (except surgically-curative basal cell carcinoma, squamous cell
carcinoma, or cervical carcinoma).
- Receiving chronic immunosuppressant therapy.
- Known positive serology for the human immunodeficiency virus (HIV), active hepatitis B
and/or hepatitis C.
- Participation in any other investigational study of drugs or devices within 30 days
prior to study start.
- Additional criteria exist.
Intervention(s):
drug: ARRY-371797, p38 inhibitor; oral
Recruiting
Contact Information
Stanford University
School of Medicine
300 Pasteur Drive
Stanford,
CA
94305