Overview
Sponsor-declared trial summary
Phase
Therapeutic exploratory (Phase II)
Status
Ended
Participants planned
14
Countries
2
Sites
4
Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT)
To assess the safety and tolerability of CRD-4730 when administered to participants with Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT)
Key facts
- Sponsor
- Cardurion Pharmaceuticals Inc.
- Participant type
- Patients
- Age range
- 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Cardiovascular Diseases [C14]
- Trial duration
- 15 Apr 2024 → 31 May 2025
- Decision date (initial)
- 2024-03-15
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- Yes
- Funding sources
- Cardurion Pharmaceuticals Inc.
External identifiers
- EU CT number
- 2022-502845-83-00
- ClinicalTrials.gov
- NCT06005428
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Pharmacokinetic, Others, Safety, Pharmacodynamic
To assess the safety and tolerability of CRD-4730 when administered to participants with Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT)
Secondary objectives 2
- To assess the effect of CRD-4730 on the inducibility of ventricular arrythmia(s) (VA) during the exercise stress test (EST), when administered to participants with CPVT
- To assess the pharmacokinetics (PK) of CRD-4730 when administered to participants with CPVT
Conditions and MedDRA coding
Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT)
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 25.1 | LLT | 10036095 | Polymorphic ventricular tachycardia | 10007541 |
Study design 3 periods
| # | Title | Allocation | Blinding | Roles blinded | Arms |
|---|---|---|---|---|---|
| 1 | Screening Eligibility criteria and associated screening study assessments must be confirmed during the screening period; this must take place after a patient has provided informed consent, but before receiving study drug.
|
Not Applicable | None | ||
| 2 | Treatment Participants will be randomly assigned on Day 1 to 1 of 2 sequences in which they will receive 2 dose levels of CRD-4730 and 1 dose of matching placebo. Randomization to the 2 treatment sequences will be in a 1:1 ratio. Participants will receive a single dose of CRD-4730 or matching placebo on Days 1, 8, and 15.
|
Randomised Controlled | Double | [{"id":101085,"code":1,"name":"Subject"},{"id":101086,"code":2,"name":"Investigator"}] | CRD-4750: Patients will receive single oral doses of CRD-4730 on Days 1, 8, and 15 of the study Placebo: Patients will receive single oral doses of placebo on Days 1, 8, and 15 of the study. The placebo will be identical in appearance to the CRD-4730 capsules but will not contain active drug. |
| 3 | Follow-up Participants will return to the site on Day 22 for a follow-up visit.
|
Not Applicable | None |
Regulatory references
- Scientific advice from competent authorities
- Medicines Evaluation Board, National Agency For The Safety Of Medicine And Health Products
- Plan to share IPD
- No
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 5
- The participant is male or female ≥18 years of age
- The participant has a confirmed CPVT diagnosis, based on genetic screening for a known RyR2 mutation and a clinical phenotype consistent with CPVT at Screening. Previous CPVT genetic testing documented in medical history is acceptable if confirmed by the investigator and documented in the study source records.
- The participant can perform an EST during which frequent premature ventricular contractions (PVCs) (≥10 per minute), ventricular bigeminy; or higher-grade VA (equivalent to a VA score ≥2) are identified by the investigator. Please refer to the complete VA scoring system, as described in Section 8.4.2.
- The participant has been on a stable dose of any anti-arrhythmic medication, except amiodarone, for 4 weeks prior to Screening.
- The patient must be willing to adhere to all contraceptive requirements.
Exclusion criteria 5
- The participant has clinically significant structural heart disease, diagnosis of heart failure, or clinically significant coronary artery disease.
- The participant has a history of a myocardial infarction, cerebrovascular accident, or transient ischemic attack within 3 months of Screening.
- The participant has a history of malignancy within the past 5 years at Screening, with the exception of successfully treated basal cell carcinoma or nonmetastatic squamous cell carcinoma of the skin or cervical carcinoma in situ.
- The female participant is pregnant, lactating/breastfeeding, or has plans to become pregnant during the study or within 3 months following the last study drug administration.
- Use of amiodarone with 3 months prior to screening. NOTE: other protocol defined Inclusion/ Exclusion criteria may apply.
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- Adverse events, clinical laboratory test results (hematology, serum chemistry, and urinalysis), vital sign measurements, and 12-lead electrocardiogram (ECG) results, and physical examination findings
Secondary endpoints 2
- Change in ventricular arrhythmia (VA) score from baseline to Day 1, from baseline to Day 8, and from baseline to Day 15
- Plasma concentrations of CRD-4730 over time for each treatment period
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
PRD10743920 · Product
- Active substance
- CRD-4730
- Pharmaceutical form
- CAPSULE
- Route of administration
- ORAL
- Max daily dose
- 600 mg milligram(s)
- Max total dose
- 900 mg milligram(s)
- Max treatment duration
- 22 Day(s)
- Authorisation status
- Not Authorised
- MA holder
- CARDURION PHARMACEUTICALS INC.
- Paediatric formulation
- No
- Orphan designation
- No
Placebo 1
N/A · Product
- Other product name
- N/A
- Pharmaceutical form
- N/A
- ATC code
- N/A — N/A
- Marketing authorisation
- N/A
- MA holder
- N/A
- MA country
- Iceland
- Paediatric formulation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Cardurion Pharmaceuticals Inc.
- Sponsor organisation
- Cardurion Pharmaceuticals Inc.
- Address
- 78 Blanchard Road Suite 200
- City
- Burlington
- Postcode
- 01803-6014
- Country
- United States
Scientific contact point
- Organisation
- Cardurion Pharmaceuticals Inc.
- Contact name
- Lauren Melton
Public contact point
- Organisation
- Cardurion Pharmaceuticals Inc.
- Contact name
- Lauren Melton
Third parties 5
| Organisation | City, country | Duties |
|---|---|---|
| Eresearchtechnology Inc. ORG-100013039
|
Philadelphia, United States | Other |
| PPD Development LP ORG-100011560
|
Wilmington, United States | On site monitoring, Code 10, Code 11, Code 12, Code 13, Other, Code 2, Code 5, Data management, E-data capture, Code 8, Code 9 |
| Sumika Chemical Analysis Service Ltd. ORG-100012379
|
Osaka, Japan | Other |
| PPD Global Central Labs ORG-100046496
|
Zaventem, Belgium | Other, Laboratory analysis |
| Bbk Worldwide LLC ORG-100044633
|
Needham, United States | Other |
Locations
2 EU/EEA countries · 4 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| France | Ended | 3 | 3 |
| Italy | Ended | 2 | 1 |
| Rest of world
United States, Canada
|
— | 9 | — |
Investigational sites
Assistance Publique Hopitaux De Paris
Cardiology, 46 Rue Henri Huchard, 75877, Paris Cedex 18
Centre Hospitalier Universitaire De Nantes
Service de cardiologie / Institut du Thorax Hôpital Nord Laennec, Boulevard Du Professeur Jacques Monod, 44800, Saint Herblain
Hospices Civils De Lyon
Centre de référence des troubles du rythme cardiaque héréditaire Hopital cardiologique Louis Pradel, 28 Avenue Du Doyen Jean Lepine, 69500, Bron
Country notifications
Trial-start, recruitment-start, end and early-termination notifications submitted per Member State
| Country | Trial start | Trial end | Recruitment start | Recruitment end | Early termination |
|---|---|---|---|---|---|
| France | 2024-04-15 | 2024-06-17 | 2025-05-31 | ||
| Italy | 2024-05-07 | 2025-01-20 | 2025-05-31 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Summary of results Art. 37(4) CTR
| Title | Submission date | Status | Type |
|---|---|---|---|
| Results summary SUM-136345
|
2026-05-29T11:13:07 | Submitted | Summary of Results |
Layperson summary Annex V
| Title | Submission date | Status | Type |
|---|---|---|---|
| Lay language summaries | 2026-05-29T11:13:13 | Submitted | Laypersons Summary of Results |
Documents 23 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Laypersons summary of results (for publication) | CRD-4730-201_Plain Language Summary_Final_10Feb2026_Fra | 1 |
| Laypersons summary of results (for publication) | CRD-4730-201_Plain Language Summary_Final_10Feb2026_Ita | 1 |
| Protocol (for publication) | D1_Cardurion_CRD-4730-201_JustificationCollectionEthnicityInfo_ 2022-502845-83-00_Public | N/A |
| Protocol (for publication) | D1_Cardurion_CRD-4730-201_Protocol Clarification Memo 2_ 2022-502845-83-00_Public | n/a |
| Protocol (for publication) | D1_Cardurion_CRD-4730-201_Protocol Clarification Memo_ 2022-502845-83-00_Public | N/A |
| Protocol (for publication) | D1_Cardurion_CRD-4730-201_Protocol_ 2022-502845-83-00_Public | 4.1 |
| Recruitment arrangements (for publication) | K1_CRD-4730-201_EU CTR_Consent_Recruitment_Template_Public | 1.0 |
| Recruitment arrangements (for publication) | K1_CRD-4730-201_Recruitment-Arrangements_FR_French__Public | NA |
| Recruitment arrangements (for publication) | K2_CRD-4730-201_Additional-Document_FR_French_Public | NA |
| Recruitment arrangements (for publication) | K2_CRD-4730-201_GP letter_IT_Italian_Public | 1.0 |
| Subject information and informed consent form (for publication) | L1_CRD-4730-201_Main ICF_IT_Italian_Public | 7.0 |
| Subject information and informed consent form (for publication) | L1_CRD-4730-201_Main-ICF_FR_French_Public | 7.0 |
| Subject information and informed consent form (for publication) | L1_CRD-4730-201_PP ICF_IT_Italian_Public | 3.0 |
| Subject information and informed consent form (for publication) | L1_CRD-4730-201_Pre-Screening Travel ICF_IT_Italian_Public | 2.0 |
| Subject information and informed consent form (for publication) | L1_CRD-4730-201_Pregnant-Partner-or-Participant-ICF_FR_French__Public | 3.0 |
| Subject information and informed consent form (for publication) | L1_CRD-4730-201_Privacy Addendum ICF_IT_Italian_Public | 4.0 |
| Subject information and informed consent form (for publication) | L2_CRD-4730-201_Study Expense Reimbursement Service_IT_Italian_Public | NA |
| Subject information and informed consent form (for publication) | L2_CRD-4730-201_Study Meal Service_FR_French_Public | 1 |
| Subject information and informed consent form (for publication) | L2_CRD-4730-201_Study Meal Service_IT_Italian_Public | NA |
| Subject information and informed consent form (for publication) | L2_CRD-4730-201_Travel Assistance Service_IT_Italian_Public | NA |
| Summary of results (for publication) | CRD-4730-201_Results summary_REDACTED | 1 |
| Synopsis of the protocol (for publication) | D1_Cardurion_CRD-4730-201_Protocol synopsis_2022-502845-83-00_FRE_Public | 4.1 |
| Synopsis of the protocol (for publication) | D1_Cardurion_CRD-4730-201_Protocol synopsis_2022-502845-83-00_ITA_Public | 4.1 |
Application history
3 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2023-11-22 | France | Acceptable 2024-03-11
|
2024-03-14 |
| 2 | SUBSTANTIAL MODIFICATION | SM-1 | 2024-06-17 | France | Acceptable 2024-09-02
|
2024-09-03 |
| 3 | SUBSTANTIAL MODIFICATION | SM-2 | 2024-12-20 | France | Acceptable 2025-03-18
|
2025-03-20 |