A Study of ARRY-371797 in Patients With LMNA-Related Dilated Cardiomyopathy

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.

Stanford is currently accepting patients for this trial.

Intervention(s):

  • drug: ARRY-371797, p38 inhibitor; oral

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.

Ages Eligible for Study

18 Years - N/A

Genders Eligible for Study

All

Now accepting new patients

Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305
Recruiting

Our research team includes physicians, residents, medical students, research assistants, and volunteers. Our research topics include medical imaging, device validation,  mobile application development, and pharmaceutical trials.  

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