Clinical trial NCT04418297: A Phase I Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of CT-G20 in Subjects With Obstructive Hypertrophic Cardiomyopathy
Official title: 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
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE1
Conditions: Cardiomyopathy, Hypertrophic Obstructive
Lead sponsor: Celltrion
Sponsor class: INDUSTRY
First posted: 2020-06-05
Enrollment: 23
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

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.


Eligibility criteria: 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

Primary outcomes:
measure: Incidence and severity of treatment-emergent adverse events and their relationship to the investigational product
timeFrame: 12 days
Secondary outcomes:
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Cedars-Sinai Medical Center
state: California
city: Boston
country: United States
facility: Tufts Medical Center
state: Massachusetts
city: New York
country: United States
facility: New York University Langone Medical Center
state: New York
city: Seongnam
country: Korea, Republic of
facility: Seoul National University Bundang Hospital
state: Gyeonggi-do
city: Gwangju
country: Korea, Republic of
facility: Chonnam National University Hospital
city: Seoul
country: Korea, Republic of
facility: Seoul National University Hospital
city: Seoul
country: Korea, Republic of
facility: Severance Hospital
city: Seoul
country: Korea, Republic of
facility: Samsung Medical Center
city: Seoul
country: Korea, Republic of
facility: Chung-Ang University Hospital
city: Warsaw
country: Poland
facility: Independent Public Central Clinical Hospital
state: Mazowieckie
city: Warsaw
country: Poland
facility: Institute of Cardiology in Warsaw
state: Mazowieckie
Study officials:
References: