Effectiveness of CRD-4730 in Participants With Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT)

NCT06005428 | Terminated Phase 2 DMC FDA Drug

• 1 other identifier: CRD-4730-201
• Study Type: interventional
• Target: 7
• Locations: 4 countries
• Sites: 11

Brief Summary

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.

Conditions

CPVT1; Heart Defects, Congenital; Heart Diseases; Ventricular Tachycardia

Keywords

CaMKII; Catecholaminergic polymorphic VT; Ventricular Tachycardia; CRD-4730

Outcome Measures

Primary Outcomes (8)

• 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.

  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.

  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

  Baseline to Day 15

• Changes in Vital Signs Measurement: Pulse Rate

  Percent change from Baseline to post Baseline will be assessed for pulse rate

  Baseline to Day 15

• Changes in Vital Signs Measurement: Respiratory Rate

  Percent change from Baseline to post Baseline will be assessed for respiratory rate

  Baseline to Day 15

• Changes in Vital Signs Measurement: Body Temperature

  Percent change from Baseline to post Baseline will be assessed for body temperature

  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

  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

  Baseline to Day 22

Secondary Outcomes (4)

• Change in Ventricular Arrhythmia (VA) score during Exercise Stress Test (EST)

  Baseline to Day 1

• Change in Ventricular Arrhythmia (VA) score during Exercise Stress Test (EST)

  Baseline to Day 8

• Change in Ventricular Arrhythmia (VA) score during Exercise Stress Test (EST)

  Baseline to Day 15

• Assessment of PK effect

  Baseline through Day 15

Study Arms (3)

Dose 1

EXPERIMENTAL

CRD-4730 Dose 1 capsule

Drug: CRD-4730

Dose 2

EXPERIMENTAL

CRD-4730 Dose 2 capsule

Drug: CRD-4730

Dose 3

PLACEBO COMPARATOR

Placebo capsule to match CRD-4730

Drug: Placebo

Interventions

CRD-4730 DRUG

Oral CRD-4730 in capsule form

Dose 1; Dose 2

Placebo DRUG

Placebo to match CRD-4730 in capsule form

Dose 3

Eligibility Criteria

• Age: 18 Years - 99 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Males or Females ≥18 years of age, at screening.
• Confirmed CPVT diagnosis, based on genetic screening for a known RyR2 mutation and a clinical phenotype consistent with CPVT at screening.
• The participant can perform an EST during which frequent premature ventricular contraction (PVCs) (≥10 per minute), ventricular bigeminy, or higher-grade VA (equivalent to a VA score ≥2) are identified by the investigator.
• Stable doses of any anti-arrhythmic medication, except amiodarone, for 4 weeks prior to screening.
• Adhere to all contraceptive criteria.

You may not qualify if:

• Clinically significant structural heart disease, diagnosis of heart failure, or clinically significant coronary artery disease.
• History of a myocardial infarction, cerebrovascular accident, or transient ischemic attack within 3 months of screening.
• History of malignancy within the past 5 years at screening (except successfully treated basal cell carcinoma or non-metastatic squamous cell carcinoma of the skin or cervical carcinoma in situ).
• Female participant that is pregnant or lactating/ breastfeeding, or has plans to do so during the study or within 3 months following last dose of study drug.

Sponsors & Collaborators

• Cardurion Pharmaceuticals, Inc.: lead

Study Sites (11)

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Cleveland Clinic Children's Hospital

Cleveland, Ohio, 44195, United States

Location

Baylor College of Medicine

Houston, Texas, 77030, United States

Location

Stollery Children's Hospital University of Alberta

Edmonton, Alberta, T6G 2R7, Canada

Location

University of British Columbia (UBC) Hospital

Vancouver, British Columbia, V5Z 1M9, Canada

Location

British Columbia Children's Hospital

Vancouver, British Columbia, V6H 3N1, Canada

Location

University of Western Ontario

London, Ontario, N6A 5A5, Canada

Location

Hôptal Nord Laennec

Nantes, Loire-Atlantique, 44805, France

Location

Hôpital Louis Pradel

Bron, 69677, France

Location

Groupe Hospitalier Bichat Claude Bernard

Paris, 75018, France

Location

IRCCS Pavia Istituti Clinici Scientifici Maugeri Spa Società Benefit

Pavia, Lombardy, 27100, Italy

Location

MeSH Terms

Conditions

Heart Defects, Congenital; Heart Diseases; Tachycardia, Ventricular

Condition Hierarchy (Ancestors)

Cardiovascular Abnormalities; Cardiovascular Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Tachycardia; Arrhythmias, Cardiac; Cardiac Conduction System Disease; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Jason Homsy, M.D., Ph.D.

  Executive Medical Director

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Investigator and Subject Blinded, Sponsor Unblinded; Placebo-controlled
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 3-period randomized 2-sequence study

Study Record Dates

• First Submitted: August 8, 2023
• First Posted: August 22, 2023
• Study Start: November 7, 2023
• Primary Completion: May 31, 2025
• Study Completion: May 31, 2025
• Last Updated: July 14, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will not share

Locations

IT (1); US (3); FR (3); CA (4)