Obstructive Hypertrophic Cardiomyopathy
Conditions
Brief summary
This is an open-label extension study of China cohort in the phase 3 study (CY 6031) of aficamten for the treatment of obstructive HCM (oHCM) to collect long-term safety and tolerability data, including assessments of cardiac function and steady-state Pharmacokinetics (PK) during chronic dosing with aficamten.
Interventions
DRUGAficamten
5-20mg
Sponsors
Corxel Pharmaceuticals
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 85 Years
Healthy volunteers
No
Inclusion criteria
1. Completion of a Cytokinetics trial investigating CK-3773274 2. LVEF ≥55% at the Screening Visit
Exclusion criteria
1. Has participated in another investigational device or drug study or received an investigational device or drug \<1 month (or 5 half-lives for drugs, whichever is longer) prior to screening. Other investigational procedures while participating in this study are not permitted. 2. Since completion of a previous study of aficamten has:Developed new-onset paroxysmal or permanent atrial fibrillation (AF) requiring rhythm restoring treatment (e.g., direct-current cardioversion, ablation procedure, or antiarrhythmic therapy) \<30 days prior to screening. Patient may rescreen for JX01003 after 30 days if heart rate (HR) \<100 bpm and/or rhythm is stable \>30 days. 3. Undergone septal reduction therapy (surgical myectomy or transcatheter alcohol ablation). 4. Had a confirmed LVEF \<40% with an associated dose interruption during participation in a prior study with aficamten. 5. History of appropriate implantable cardioverter defibrillator (ICD) shock within 30 days prior to screening. 6. Has received treatment with mavacamten.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Patient incidence of reported adverse events (AEs) | Baseline to End of study, up to 2 years |
| Patient incidence of reported serious adverse events (SAEs ) | Baseline to End of study, up to 2 years |
| Patient incidence of LVEF<50% & LVEF <40% | Baseline to End of study, up to 2 years |
Secondary
| Measure | Time frame |
|---|---|
| Proportion of patients with resting LVOT-G <50 mmHg | Change from baseline values to week 48 at 12-week intervals; |
| Proportion of patients with resting LVOT-G <30 mmHg | Change from baseline values to week 48 at 12-week intervals; |
| Proportion of patients with post-Valsalva LVOT-G <50 mmHg | Change from baseline values to week 48 at 12-week intervals; |
| Proportion of patients with post-Valsalva LVOT-G <30 mmHg | Change from baseline values to week 48 at 12-week intervals; |
| Proportion of patients with LVEF ≥50%, resting LVOT-G <30 mmHg, and post-Valsalva LVOT-G <50 mmHg | Change from baseline values to week 48 at 12-week intervals; |
| First resting LVOT-G <50 mmHg | Time to the following event through last follow-up, up to 2 years. |
| First resting LVOT-G <30 mmHg | Time to the following event through last follow-up, up to 2 years. |
| First post-Valsalva LVOT-G <50 mmHg | Time to the following event through last follow-up, up to 2 years. |
| First LVEF ≥50%, resting LVOT-G <30 mmHg, and post-Valsalva LVOT-G <50 mmHg | Time to the following event through last follow-up, up to 2 years. |
| First post-Valsalva LVOT-G <30 mmHg | Time to the following event through last follow-up, up to 2 years. |
| Peak LVOT-G at rest and with Valsalva provocation | Change from baseline values to week 48 at 12-week intervals; |
Countries
China
Contacts
PRINCIPAL_INVESTIGATORYuhui Zhang, MD
Chinese Academy of Medical Sciences, Fuwai Hospital
Outcome results
None listed