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

Phase 1

Terminated

• Conditions: Cardiomyopathy, Hypertrophic Obstructive
• Interventions: Drug: Placebo; Drug: CT-G20

• Registration Number: NCT04418297
• Lead Sponsor: Celltrion

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2020-05-28
• Study First Submitted QC: 2020-06-01
• Study First Posted: 2020-06-05
• Study Start: 2020-10-23
• Status Verified: 2022-08
• Primary Completion: 2022-11-22
• Study Completion: 2022-11-22
• Last Update Submitted: 2023-02-28
• Last Update Posted: 2023-03-02

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 23

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
Placebo	Placebo	-
CT-G20	CT-G20	-

Primary Outcome Measures

Name	Time	Method
Incidence and severity of treatment-emergent adverse events and their relationship to the investigational product	12 days

Trial Locations

Locations (11)

Cedars-Sinai Medical Center; 🇺🇸 Los Angeles, California, United States

Tufts Medical Center; 🇺🇸 Boston, Massachusetts, United States

Institute of Cardiology in Warsaw; 🇵🇱 Warsaw, Mazowieckie, Poland

Seoul National University Hospital; 🇰🇷 Seoul, Korea, Republic of

Severance Hospital; 🇰🇷 Seoul, Korea, Republic of

New York University Langone Medical Center; 🇺🇸 New York, New York, United States

Seoul National University Bundang Hospital; 🇰🇷 Seongnam, Gyeonggi-do, Korea, Republic of

Chonnam National University Hospital; 🇰🇷 Gwangju, Korea, Republic of

Samsung Medical Center; 🇰🇷 Seoul, Korea, Republic of

Independent Public Central Clinical Hospital; 🇵🇱 Warsaw, Mazowieckie, Poland

Chung-Ang University Hospital; 🇰🇷 Seoul, Korea, Republic of