A Phase I Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of CT-G20 in Subjects With Obstructive Hypertrophic Cardiomyopathy

Study Overview
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Brief Summary
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This is a randomized, double-blind, placebo-controlled, sequential, 5-day treatment, ascending dose study in subjects with obstructive HCM aged 18-70 years. The purpose of this study is to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of CT-G20.

Official Title
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A Randomized, Double-Blind, Placebo-Controlled, Sequential, Phase I Study to Evaluate Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of CT-G20 in Subjects With Obstructive Hypertrophic Cardiomyopathy

Conditions
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Cardiomyopathy, Hypertrophic Obstructive

Intervention / Treatment
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* Drug: CT-G20
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Men or women aged 18 to 70 years, inclusive at Screening Has established diagnosis of HCM defined by standard criteria as a maximal left ventricular wall thickness ≥15 mm at initial diagnosis in the absence of other causative loading abnormalities capable of producing the magnitude of hypertrophy observed or ≥13 mm if the subject has a family history of HCM Has LVOT gradient ≥30 mmHg at rest or LVOT gradient ≥50 mmHg with Valsalva maneuver, due to SAM Exclusion Criteria: Known infiltrative, genetic or storage disorder causing cardiac hypertrophy that mimics HCM, such as Fabry disease, amyloidosis or Noonan syndrome with LV hypertrophy History of persistent atrial fibrillation prior to Screening or Baseline History of paroxysmal atrial fibrillation requiring treatment (e.g., anti-coagulation and/or antiarrhythmic therapy) within 3 months prior to Screening Recently treated with invasive septal reduction (surgical myectomy or percutaneous alcohol septal ablation) within 6 months prior to Screening or plans to have either of these treatments during the study Systolic heart failure with ejection fraction <55% or heart failure symptoms of NYHA Class IV QTcF >480 msec at Screening or Baseline Presence of diseases classified as significant by the Investigator at Screening or Baseline, including the following but not limited to: Diabetes mellitus requiring treatment Moderate or severe renal insufficiency or renal insufficiency with estimated glomerular filtration rate <70 mL/min/1.73m2 Concomitant use of Disopyramide or Ranolazine within at least 7 days or 5 half-lives (whichever is longer) prior to Screening

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 70 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: CT-G20<br> | Drug: CT-G20<br>* oral tablet<br>|
| Placebo Comparator: Placebo<br> | Drug: Placebo<br>* oral tablet<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence and severity of treatment-emergent adverse events and their relationship to the investigational product |  | 12 days |