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Clinical Trial Report
Gene Therapy Trial Report
Summary
AC6 Gene Transfer for CHF
NCTID
NCT00787059
(View at clinicaltrials.gov)
Description
This research study is designed to determine: 1) whether gene transfer using an agent called Ad5.hAC6 (adenovirus-5 encoding human adenylyl cyclase type 6) can be given safely to patients with congestive heart failure (CHF) and 2) whether this agent may be of benefit in heart failure. Gene transfer is a process by which genes are introduced into cells and the cells then produce the specific protein that the gene directs, in this case, a protein known as adenylyl cyclase type 6 (AC6). The gene is carried into the heart cells by a modified virus. The virus that is modified is an adenovirus (Ad5), a virus that sometimes causes a brief cold. In extensive animal experiments, it was found that increased amounts of AC6 protein in heart cells appeared to make the heart pump more vigorously.
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Development Status
Active
Indication
Congestive Heart Failure
Disease Ontology Term
DOID:9651
Compound Name
RT-100
Compound Description
Ad5.CMV.hAC6
Sponsor
Hammond, H. Kirk, M.D.
Funder Type
Indiv
Recruitment Status
Completed
Enrollment Count
56
Results Posted
Not Available
Therapy Information
Target Gene/Variant
ADCY6
Therapy Type
Gene transfer
Therapy Route
In-vivo
Mechanism of Action
Overexpression of protective allele/gene
Route of Administration
Intracoronary
Drug Product Type
Viral vector
Target Tissue/Cell
Myocardium
Delivery System
Viral transduction
Vector Type
Ad5
Editor Type
Dose 1
3.2E9 vp (n=6)
Dose 2
3.2E10 vp (n=6)
Dose 3
1E10 vp (n=6)
Dose 4
3.2E11 vp (n=12)
Dose 5
1E12 vp (n=12)
Study Record Dates
Current Stage
Phase1, Phase2
Submit Date
2008-11-06
Completion Date
2017-11-16
Last Update
2018-02-09
Participation Criteria
Eligible Age
18 Years - 80 Years
Standard Ages
Adult, Older adult
Sexes Eligible for Study
ALL
Locations
No.of Trial Sites
7
Locations
United States
Regulatory Information
Has US IND
True
FDA Designations
Fast Track
Recent Updates
Company is preparing to submit a plan to the FDA for the Phase 2b/3 study
Resources/Links
Clinical Publications
Intracoronary Gene Transfer of Adenylyl Cyclase 6 in Patients With Heart Failure: A Randomized Clinical Trial
Design of a Phase 3 trial of intracoronary administration of human adenovirus 5 encoding human adenylyl cyclase type 6 (RT-100) gene transfer in patients with heart failure with reduced left ventricular ejection fraction: The FLOURISH Clinical Trial
News and Press Releases
Renova Therapeutics Announces Strategic Focus and Roadmap Prioritization for Its Gene Therapies to Treat Heart Failure and Type 2 Diabetes Mellitus
RT-100 (AC6 gene transfer)
US FDA grants Fast Track designation to Renova Therapeutic's RT-100 AC6 gene transfer for the treatment of heart failure.
Preclinical Publications
Increased expression of adenylylcyclase type VI proportionately increases beta-adrenergic receptor-stimulated production of cAMP in neonatal rat cardiac myocytes
Adenylylcyclase increases responsiveness to catecholamine stimulation in transgenic mice
Cardiac-directed adenylyl cyclase expression improves heart function in murine cardiomyopathy
Intracoronary delivery of adenovirus encoding adenylyl cyclase VI increases left ventricular function and cAMP-generating capacity
Adenylyl cyclase increases survival in cardiomyopathy
Indirect intracoronary delivery of adenovirus encoding adenylyl cyclase increases left ventricular contractile function in mice
Adenylyl cyclase type VI gene transfer reduces phospholamban expression in cardiac myocytes via activating transcription factor 3
Intracoronary adenovirus encoding adenylyl cyclase VI increases left ventricular function in heart failure
Nitroprusside increases gene transfer associated with intracoronary delivery of adenovirus
Adenylylcyclase gene transfer increases function of the failing heart
Intracoronary Gene Transfer of Adenylyl Cyclase 6 in Patients With Heart Failure: A Randomized Clinical Trial