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

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

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.

Study Overview

• Status: Terminated
• Conditions: Cardiomyopathy, Hypertrophic Obstructive
• Intervention / Treatment: Drug: Placebo; Drug: CT-G20

• Study Type: Interventional
• Enrollment (Actual): 23
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Boram Lee; Phone Number: +82-2-3403-9659; Email: boram.lee@celltrion.com

Study Locations

• Korea, Republic of
  • Gwangju, Korea, Republic of, 61469
    • Chonnam National University Hospital
  • Seoul, Korea, Republic of, 03080
    • Seoul National University Hospital
  • Seoul, Korea, Republic of, 03722
    • Severance Hospital
  • Seoul, Korea, Republic of, 06973
    • Chung-Ang University Hospital
  • Seoul, Korea, Republic of, 06351
    • Samsung Medical Center
  • Gyeonggi-do
    • Seongnam, Gyeonggi-do, Korea, Republic of, 13620
      • Seoul National University Bundang Hospital
• Poland
  • Mazowieckie
    • Warsaw, Mazowieckie, Poland, 02097
      • Independent Public Central Clinical Hospital
    • Warsaw, Mazowieckie, Poland, 04628
      • Institute of Cardiology in Warsaw
• United States
  • California
    • Los Angeles, California, United States, 90048
      • Cedars-Sinai Medical Center
  • Massachusetts
    • Boston, Massachusetts, United States, 02111
      • Tufts Medical Center
  • New York
    • New York, New York, United States, 10016
      • New York University Langone Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 70 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. Men or women aged 18 to 70 years, inclusive at Screening
2. 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
3. Has LVOT gradient ≥30 mmHg at rest or LVOT gradient ≥50 mmHg with Valsalva maneuver, due to SAM

Exclusion Criteria:

1. Known infiltrative, genetic or storage disorder causing cardiac hypertrophy that mimics HCM, such as Fabry disease, amyloidosis or Noonan syndrome with LV hypertrophy
2. History of persistent atrial fibrillation prior to Screening or Baseline
3. History of paroxysmal atrial fibrillation requiring treatment (e.g., anti-coagulation and/or antiarrhythmic therapy) within 3 months prior to Screening
4. 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
5. Systolic heart failure with ejection fraction <55% or heart failure symptoms of NYHA Class IV
6. QTcF >480 msec at Screening or Baseline

7. Presence of diseases classified as significant by the Investigator at Screening or Baseline, including the following but not limited to:

   1. Diabetes mellitus requiring treatment
   2. Moderate or severe renal insufficiency or renal insufficiency with estimated glomerular filtration rate <70 mL/min/1.73m2
8. Concomitant use of Disopyramide or Ranolazine within at least 7 days or 5 half-lives (whichever is longer) prior to Screening

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Sequential Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Placebo	Drug: Placebo; oral tablet
Experimental: CT-G20	Drug: CT-G20; oral tablet

What is the study measuring?

Primary Outcome Measures

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

Collaborators and Investigators

• Sponsor: Celltrion

Study record dates

Study Major Dates

• Study Start (Actual): October 23, 2020
• Primary Completion (Actual): November 22, 2022
• Study Completion (Actual): November 22, 2022

Study Registration Dates

• First Submitted: May 28, 2020
• First Submitted That Met QC Criteria: June 1, 2020
• First Posted (Actual): June 5, 2020

Study Record Updates

• Last Update Posted (Actual): March 2, 2023
• Last Update Submitted That Met QC Criteria: February 28, 2023
• Last Verified: August 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Heart Diseases; Cardiovascular Diseases; Pathological Conditions, Anatomical; Aortic Valve Disease; Heart Valve Diseases; Aortic Stenosis, Subvalvular; Aortic Valve Stenosis; Hypertrophy; Cardiomyopathies; Cardiomyopathy, Hypertrophic
• Other Study ID Numbers: CT-G20 1.2

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No