This is a Phase 2, multicenter, double-blind, sponsor unblinded, placebo-controlled, single-dose clinical study of CRD-4730 to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of CRD-4730 when administered as single oral doses to participants with Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT). The study will have 2 cohorts in which participants with CPVT will participate in a 3-period, randomized 2-sequence study. Each participant will receive 2 different doses of CRD-4730 and 1 dose of matching placebo, with each study drug administered as a single dose.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
7
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
Cleveland Clinic Children's Hospital
Cleveland, Ohio, United States
Baylor College of Medicine
Houston, Texas, United States
Stollery Children's Hospital University of Alberta
Edmonton, Alberta, Canada
University of British Columbia (UBC) Hospital
Vancouver, British Columbia, Canada
British Columbia Children's Hospital
Vancouver, British Columbia, Canada
University of Western Ontario
London, Ontario, Canada
Hôptal Nord Laennec
Nantes, Loire-Atlantique, France
Hôpital Louis Pradel
Bron, France
Groupe Hospitalier Bichat Claude Bernard
Paris, France
...and 1 more locations
Treatment Emergent Adverse Events (TEAEs)
The number of participants with TEAEs including drug-related AEs, serious AEs (SAEs), and AEs leading to study drug discontinuation will be assessed.
Time frame: Baseline to Day 22
Changes in Laboratory Assessments
The number of participants who have normal/ abnormal values at Baseline compared to normal/ abnormal values post-Baseline will be assessed for hematology, serum chemistry and urinalysis.
Time frame: Baseline to Day 15
Changes in Vital Signs Measurement: Systolic and Diastolic blood pressure
Percent change from Baseline to post Baseline will be assessed for systolic and diastolic blood pressure
Time frame: Baseline to Day 15
Changes in Vital Signs Measurement: Pulse Rate
Percent change from Baseline to post Baseline will be assessed for pulse rate
Time frame: Baseline to Day 15
Changes in Vital Signs Measurement: Respiratory Rate
Percent change from Baseline to post Baseline will be assessed for respiratory rate
Time frame: Baseline to Day 15
Changes in Vital Signs Measurement: Body Temperature
Percent change from Baseline to post Baseline will be assessed for body temperature
Time frame: Baseline to Day 15
Changes in Physical Exam
General physical exams will be carried out to detect any abnormalities in the cardiovascular, respiratory and other body systems
Time frame: Baseline to Day 22
Changes in Electrocardiogram (ECG) Measurements
Number of participants who have normal/abnormal ECG measurements at Baseline will be compared to normal/abnormal ECG measurement post Baseline
Time frame: Baseline to Day 22
Change in Ventricular Arrhythmia (VA) score during Exercise Stress Test (EST)
The VA score is a physician administered score on a scale of 0-5 with higher numbers being worse.
Time frame: Baseline to Day 1
Change in Ventricular Arrhythmia (VA) score during Exercise Stress Test (EST)
The VA score is a physician administered score on a scale of 0-5 with higher numbers being worse.
Time frame: Baseline to Day 8
Change in Ventricular Arrhythmia (VA) score during Exercise Stress Test (EST)
The VA score is a physician administered score on a scale of 0-5 with higher numbers being worse.
Time frame: Baseline to Day 15
Assessment of PK effect
Plasma concentrations of CRD-4730 over time for each treatment period
Time frame: Baseline through Day 15
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