Non-obstructive Hypertrophic Cardiomyopathy
Conditions
Keywords
nHCM, non-obstructive HCM, non-obstructive hypertrophic cardiomyopathy, HCM, hypertrophic cardiomyopathy
Brief summary
FORTITUDE-HCM is a global, multicenter, double-blind, parallel-group, placebo-controlled Phase 2b study that will assess the efficacy and safety of ninerafaxstat compared to placebo on top of Standard of Care in patients with symptomatic nHCM
Interventions
DRUGNinerafaxstat 200mg MR
Ninerafaxstat 200mg Modified Release tablet administered BID
DRUGPlacebo
Matching placebo tablet administered BID
Sponsors
Imbria Pharmaceuticals, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Caregiver, Investigator)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Select Inclusion Criteria: * Has a clinical diagnosis of HCM consistent with current American College of Cardiology/American Heart Association and European Society of Cardiology Guideline definitions * Has had confirmation of nHCM by the echocardiography core laboratory based on screening rest and exercise stress echocardiography * New York Heart Association (NYHA) functional Class II or III at screening * Functional limitation as defined by a screening CPET Select
Exclusion criteria
* Has a known or suspected infiltrative, genetic, or storage disorder causing cardiac hypertrophy that mimics nHCM * Has any other condition judged by the investigator to be the primary cause of dyspnea, exercise intolerance, and/or angina * Has an inability to exercise on a treadmill or bicycle (eg, orthopedic limitations) * Has any medical condition that precludes upright exercise stress testing Other protocol-defined inclusion and
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CSS) | Baseline to Week 12 | The Clinical Summary Score is a merging of symptom frequency and symptom burden domains into a total symptom score combined with the physical limitation domain. Scoring ranges from 0 to 100, with higher scores indicating better health status. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in ventilatory efficiency (VE/VCO2 slope) during cardiopulmonary exercise testing (CPET) | Baseline to Week 12 | — |
| Change in the Kansas City Cardiomyopathy Questionnaire Total Symptom Score (KCCQ-TSS) | Baseline to Week 12 | The Total Symptom Score is a merging of symptom frequency and symptom burden domains. Scoring ranges from 0 to 100, with higher scores indicating better health status. |
| Change in the Kansas City Cardiomyopathy Questionnaire Physical Limitations Score (KCCQ-PLS) | Baseline to Week 12 | The Physical Limitations Score is a single-domain score. Scoring ranges from 0 to 100, with higher scores indicating better health status. |
| Change in the Kansas City Cardiomyopathy Questionnaire Overall Summary Score (KCCQ-OSS) | Baseline to Week 12 | The Overall Summary Score is a merging of symptom, physical limitations, social limitations, and quality of life domains. Scoring ranges from 0 to 100, with higher scores indicating better health status. |
Countries
Austria, Belgium, France, Germany, Italy, Netherlands, Poland, Portugal, Spain, United Kingdom, United States
Contacts
CONTACTMedical Monitor
Outcome results
None listed