Phosphatase Inhibition by Intracoronary Gene Therapy in Subjects With Non-Ischemic NYHA Class III Heart Failure (GenePHIT)
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ClinicalTrials.gov Identifier: NCT05598333 |
Recruitment Status :
Recruiting
First Posted : October 28, 2022
Last Update Posted : May 6, 2024
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Tracking Information | |||||||
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First Submitted Date ICMJE | October 25, 2022 | ||||||
First Posted Date ICMJE | October 28, 2022 | ||||||
Last Update Posted Date | May 6, 2024 | ||||||
Actual Study Start Date ICMJE | October 20, 2023 | ||||||
Estimated Primary Completion Date | December 2030 (Final data collection date for primary outcome measure) | ||||||
Current Primary Outcome Measures ICMJE |
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Original Primary Outcome Measures ICMJE | Same as current | ||||||
Change History | |||||||
Current Secondary Outcome Measures ICMJE |
Heart Failure Related Hospitalizations [ Time Frame: 52 weeks ] Number of hospitalizations attributed to heart failure
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Original Secondary Outcome Measures ICMJE | Same as current | ||||||
Current Other Pre-specified Outcome Measures | Not Provided | ||||||
Original Other Pre-specified Outcome Measures | Not Provided | ||||||
Descriptive Information | |||||||
Brief Title ICMJE | Phosphatase Inhibition by Intracoronary Gene Therapy in Subjects With Non-Ischemic NYHA Class III Heart Failure | ||||||
Official Title ICMJE | A Phase 2, Adaptive, Double-blinded, Placebo Controlled, Randomized, Multi-center Trial to Evaluate the Efficacy, Safety and Tolerability of Intracoronary Infusion of AB-1002 in Adult Subjects With New York Heart Association (NYHA) Class III Heart Failure and Non-ischemic Cardiomyopathy | ||||||
Brief Summary | This is a Phase 2 adaptive, double-blinded, placebo-controlled, randomized, multi-center trial study to evaluate the safety and efficacy of a single dose of AB-1002, administered via antegrade intracoronary artery infusion, in males and females age >18 years with non-ischemic cardiomyopathy and NYHA Class III symptoms of HF. Subjects will be randomized into one of three treatment groups in a 1:1:1 |
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Detailed Description | Subjects will be randomized into one of three treatment groups in a 1:1:1 fashion to either 3.25E13vg (n=30-50), 6.5E13 (n=30-50) or placebo group (n=30-50). Approximately 90 to 150 subjects will be randomly assigned to study intervention Placebo Study duration until the primary analysis at 52 week will be approximately 37 months including 25 months of recruitment and 52-week Observation Period after dosing. Once all the subjects complete the 52 weeks Observation Period, the treatment groups will be unblinded and primary analysis performed. Study participation duration: The study will last 52 weeks from dosing, with another 4 years of long-term follow-up for a total of 5 years. During the 4 year long-term follow up sites will contact subjects twice a year for two years, then once a year for the remaining two years for safety, efficacy assessments, and survival |
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Study Type ICMJE | Interventional | ||||||
Study Phase ICMJE | Phase 2 | ||||||
Study Design ICMJE | Allocation: Randomized Intervention Model: Parallel Assignment Intervention Model Description: Adaptive, double blinded, placebo controlled, randomized Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)Masking Description: The IWRS will be programmed with blind-breaking instructions. In case of an emergency, the investigator has the responsibility for determining if unblinding of a subject's intervention assignment is warranted. If the investigator is unavailable, and a treating physician not associated with the study requests emergency unblinding, the emergency unblinding requests are forwarded to the emergency medical advice 24 hours/7 day service. Primary Purpose: Treatment
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Condition ICMJE | Congestive Heart Failure | ||||||
Intervention ICMJE | Drug: AB-1002
Intracoronary Infusion of AB-1002 or placebo
Other Names:
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Study Arms ICMJE |
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Publications * | Not Provided | ||||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |||||||
Recruitment Status ICMJE | Recruiting | ||||||
Estimated Enrollment ICMJE |
150 | ||||||
Original Estimated Enrollment ICMJE | Same as current | ||||||
Estimated Study Completion Date ICMJE | December 2030 | ||||||
Estimated Primary Completion Date | December 2030 (Final data collection date for primary outcome measure) | ||||||
Eligibility Criteria ICMJE | Inclusion Criteria:
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Sex/Gender ICMJE |
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Ages ICMJE | 18 Years and older (Adult, Older Adult) | ||||||
Accepts Healthy Volunteers ICMJE | No | ||||||
Contacts ICMJE |
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Listed Location Countries ICMJE | United States | ||||||
Removed Location Countries | |||||||
Administrative Information | |||||||
NCT Number ICMJE | NCT05598333 | ||||||
Other Study ID Numbers ICMJE | ASK-CHF2-CS201 | ||||||
Has Data Monitoring Committee | Yes | ||||||
U.S. FDA-regulated Product |
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IPD Sharing Statement ICMJE |
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Current Responsible Party | Asklepios Biopharmaceutical, Inc. | ||||||
Original Responsible Party | Same as current | ||||||
Current Study Sponsor ICMJE | Asklepios Biopharmaceutical, Inc. | ||||||
Original Study Sponsor ICMJE | Same as current | ||||||
Collaborators ICMJE | Not Provided | ||||||
Investigators ICMJE | Not Provided | ||||||
PRS Account | Asklepios Biopharmaceutical, Inc. | ||||||
Verification Date | May 2024 | ||||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |