Clinical trial NCT02351856: A Rollover Study of ARRY-371797 in Patients With LMNA-Related Dilated Cardiomyopathy
Official title: An Open-label Rollover Study of ARRY-371797 in Patients With Symptomatic Genetic Dilated Cardiomyopathy Due to a Lamin A/C Gene Mutation
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: LMNA-Related Dilated Cardiomyopathy
Keywords: laminopathy
ARRY-797
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2015-01-30
Enrollment: 8
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This is a rollover study designed to investigate the safety and effectiveness of investigational study drug ARRY-371797 in patients who previously received ARRY-371797 in a study for patients with LMNA-related dilated cardiomyopathy sponsored by Array BioPharma and may, in the Investigator's opinion, derive benefit from continued treatment.


Eligibility criteria: Key Inclusion Criteria:

* Received ARRY-371797 as treatment for a genetic dilated cardiomyopathy secondary to LMNA mutations in a clinical study sponsored by Array BioPharma.
* May, in the opinion of the Investigator, benefit from continued ARRY-371797 treatment.
* Additional criteria exist.

Key Exclusion Criteria:

* Discontinued treatment in the parent study for any reason other than study completion or Sponsor termination of the study.
* Additional criteria exist.

Primary outcomes:
measure: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
timeFrame: Baseline up to 30 days after last dose of study drug (i.e., maximum up to 282 weeks)
measure: Number of Participants With Change From Baseline Value in Clinical Laboratory Hematology Test Results to Worst Value
timeFrame: Baseline, Post-Baseline (anytime from first dose of study drug to maximum duration of up to 282 weeks)
measure: Number of Participants With Change From Baseline Value in Clinical Laboratory Chemistry Test Results to Worst Value
timeFrame: Baseline, Post-Baseline (anytime from first dose of study drug to maximum duration of up to 282 weeks)
measure: Number of Participants With Abnormal Physical Examination Findings
timeFrame: Baseline up to 30 days after last dose of study drug (i.e., maximum up to 282 weeks)
measure: Number of Participants With Abnormalities in Vital Signs
timeFrame: Baseline up to 30 days after last dose of study drug (i.e., maximum up to 282 weeks)
measure: Number of Participants With Abnormal 12-lead Electrocardiogram (ECG) Findings
timeFrame: Baseline up to 30 days after last dose of study drug (i.e., maximum up to 282 weeks)
Secondary outcomes:
measure: Change From Baseline in Six Minute Walk Test (6MWT) Distance at Day 1, Weeks 24, 48, 72, 96 and Early Termination Visit
timeFrame: Baseline, Day 1, Weeks 24, 48, 72, 96 and early termination visit (anytime within the maximum duration of up to 282 weeks)
measure: Change From Baseline in Left Ventricular End Systolic Index (LVESVI) and Left Ventricular End Diastolic Volume Index (LVEDVI) at Day 1, Weeks 24, 48, 72, 96 and Early Termination Visit
timeFrame: Baseline, Day 1, Weeks 24, 48, 72, 96 and early termination visit (anytime within the maximum duration of up to 282 weeks)
measure: Change From Baseline in Left Ventricular Mass (LVM) at Day 1, Weeks 24, 48, 72, 96 and Early Termination Visit
timeFrame: Baseline, Day 1, Weeks 24, 48, 72, 96 and early termination visit (anytime within the maximum duration of up to 282 weeks)
measure: Change From Baseline in Left Ventricular Ejection Fraction (LVEF) at Day 1, Weeks 24, 48, 72, 96 and Early Termination Visit
timeFrame: Baseline, Day 1, Weeks 24, 48, 72, 96 and early termination visit (anytime within the maximum duration of up to 282 weeks)
measure: Change From Baseline in Left Ventricular Mass (LVM) to Volume Ratio at Day 1, Weeks 24, 48, 72, 96 and Early Termination Visit
timeFrame: Baseline, Day 1, Weeks 24, 48, 72, 96 and early termination visit (anytime within the maximum duration of up to 282 weeks)
measure: Change From Baseline in Right Ventricular End Diastolic Diameter at Day 1, Weeks 24, 48, 72, 96 and Early Termination Visit
timeFrame: Baseline, Day 1, Weeks 24, 48, 72, 96 and early termination visit (anytime within the maximum duration of up to 282 weeks)
measure: Change From Baseline in Right Ventricular Fractional Area at Day 1, Weeks 24, 48, 72, 96 and Early Termination Visit
timeFrame: Baseline, Day 1, Weeks 24, 48, 72, 96 and early termination visit (anytime within the maximum duration of up to 282 weeks)
measure: Change From Baseline in 36-Item Short-Form (SF-36) Health Survey Score at Day 1, Weeks 24, 48, 72, 96 and Early Termination Visit
timeFrame: Baseline, Day 1, Weeks 24, 48, 72, 96 and early termination visit (anytime within the maximum duration of up to 282 weeks)
measure: Change From Baseline in Quality of Life by Kansas City Cardiomyopathy Questionnaire (KCCQ) Scores at Day 1, Weeks 24, 48, 72, 96 and Early Termination Visit
timeFrame: Baseline, Day 1, Weeks 24, 48, 72, 96 and early termination visit (anytime within the maximum duration of up to 282 weeks)
measure: Mean Plasma Concentration of ARRY-371797 and Metabolites (AR00420643, AR00428028, AR00486705)
timeFrame: Pre dose and post dose on Day 1, Weeks 12 and 24
Other outcomes:
Locations:
city: Aurora
country: United States
facility: University of Colorado Hospital
state: Colorado
city: Baltimore
country: United States
facility: Johns Hopkins Hospital
state: Maryland
city: Boston
country: United States
facility: Brigham & Women's Hospital
state: Massachusetts
city: Madison
country: United States
facility: Meriter Wisconsin Heart
state: Wisconsin
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References:
citation: Judge DP, Lakdawala NK, Taylor MRG, Mestroni L, Li H, Oliver C, Angeli FS, Lee PA, MacRae CA. Long-Term Efficacy and Safety of ARRY-371797 (PF-07265803) in Patients With Lamin A/C-Related Dilated Cardiomyopathy. Am J Cardiol. 2022 Nov 15;183:93-98. doi: 10.1016/j.amjcard.2022.08.001. Epub 2022 Sep 13.