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The early termination of the clinical trial is due to an internal administrative and business decision, the study is not being terminated for safety reasons.
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Dose 1 | Experimental | CRD-4730 Dose 1 capsule |
|
| Dose 2 | Experimental | CRD-4730 Dose 2 capsule |
|
| Dose 3 | Placebo Comparator | Placebo capsule to match CRD-4730 |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| CRD-4730 | Drug | Oral CRD-4730 in capsule form |
| |
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | Baseline to Day 1 |
| Change in Ventricular Arrhythmia (VA) score during Exercise Stress Test (EST) |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Jason Homsy, M.D., Ph.D. | Executive Medical Director | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | United States | ||
| Cleveland Clinic Children's Hospital |
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3-period randomized 2-sequence study
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Investigator and Subject Blinded, Sponsor Unblinded; Placebo-controlled
| Placebo |
| Drug |
Placebo to match CRD-4730 in capsule form |
|
| 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 |
The VA score is a physician administered score on a scale of 0-5 with higher numbers being worse. |
| 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. | Baseline to Day 15 |
| Assessment of PK effect | Plasma concentrations of CRD-4730 over time for each treatment period | Baseline through Day 15 |
| Cleveland |
| Ohio |
| 44195 |
| United States |
| Baylor College of Medicine | Houston | Texas | 77030 | United States |
| Stollery Children's Hospital University of Alberta | Edmonton | Alberta | T6G 2R7 | Canada |
| University of British Columbia (UBC) Hospital | Vancouver | British Columbia | V5Z 1M9 | Canada |
| British Columbia Children's Hospital | Vancouver | British Columbia | V6H 3N1 | Canada |
| University of Western Ontario | London | Ontario | N6A 5A5 | Canada |
| Hôptal Nord Laennec | Nantes | Loire-Atlantique | 44805 | France |
| Hôpital Louis Pradel | Bron | 69677 | France |
| Groupe Hospitalier Bichat Claude Bernard | Paris | 75018 | France |
| IRCCS Pavia Istituti Clinici Scientifici Maugeri Spa Società Benefit | Pavia | Lombardy | 27100 | Italy |
| ID | Term |
|---|---|
| D006330 | Heart Defects, Congenital |
| D006331 | Heart Diseases |
| D017180 | Tachycardia, Ventricular |
| ID | Term |
|---|---|
| D018376 | Cardiovascular Abnormalities |
| D002318 | Cardiovascular Diseases |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D013610 | Tachycardia |
| D001145 | Arrhythmias, Cardiac |
| D000075224 | Cardiac Conduction System Disease |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
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