Study Stopped
Early termination following Trial Steering Committee recommendation
Investigation of the Safety and Feasibility of AAV1/SERCA2a Gene Transfer in Patients With Chronic Heart Failure
SERCA-LVAD
2 other identifiers
interventional
5
1 country
2
Brief Summary
The aim of the study is to determine the safety and feasibility of giving an adeno-associated viral vector expressing the sarcoplasmic reticulum calcium ATPase (SERCA2a), driven by the CMV promoter (AAV1-CMV-SERCA2a), to heart failure patients that have received a left ventricular assist device (LVAD) for an accepted clinical indication.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
Started Jul 2014
Shorter than P25 for phase_2
2 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
September 24, 2007
CompletedFirst Posted
Study publicly available on registry
September 26, 2007
CompletedStudy Start
First participant enrolled
July 1, 2014
CompletedPrimary Completion
Last participant's last visit for primary outcome
September 1, 2015
CompletedStudy Completion
Last participant's last visit for all outcomes
September 1, 2015
CompletedResults Posted
Study results publicly available
February 25, 2020
CompletedFebruary 8, 2023
January 1, 2023
1.2 years
September 24, 2007
September 17, 2018
January 6, 2023
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Overall Safety and Feasibility of Administering AAV1/SERCA2a to LVAD Patients
Safety is defined as the incidence of patients experiencing death and major adverse cardiovascular events, and out of range laboratory values. Both AAV1/SERCA2a treated cohorts (NAb+ and NAb-) will be compared to the placebo group.
6 months
Secondary Outcomes (5)
Number of Participants With Exogenous Viral Vector Genome in the Myocardium Measured by qPCR for the Viral DNA
6 months
Left Ventricular Function (LVEF)
6 months
Levels of SERCA2a Protein
6 months
Other Relevant Proteins e.g. Phospholamban, the Sarcoplasmic Reticulum Calcium Release Channel, the Na+/Ca2+-Exchanger.
6 months
Function of Isolated Myocytes
6 months
Study Arms (2)
AAV1/SERCA2A
ACTIVE COMPARATORSERCA gene therapy
Placebo
PLACEBO COMPARATORPlacebo (saline solution)
Interventions
AAV1/SERCA2a will be delivered by a percutaneous method in the catheter laboratory. Dose: 1x 10\^13 DRP (DNase resistant particles)
Placebo aliquots will be of the same composition as the investigational medicinal product with the absence of the active ingredient and will be visually indistinguishable from the medicinal product. Placebo is prepared and handled exactly as above in a blinded fashion.
Eligibility Criteria
You may qualify if:
- Patients that have had a left ventricular assist device (LVAD) implanted for chronic heart failure, where chronic heart failure is defined as at least 6 months
- Patients are clinically stable in the opinion of the clinical team looking after the patient
- Written informed consent
You may not qualify if:
- \<18 or \>70 years of age at the time of consent
- Pregnancy or within 6 months of giving birth
- Women of child-bearing potential not using an effective method of contraception
- Men not using an effective method of contraception
- Suspected or active viral, fungal or parasitic infection within 48 hours prior to administration of IMP, in the opinion of the investigator\*.
- Patients at a high risk of thrombosis in the opinion of the investigator
- Patients with a previous episode of LVAD thrombosis
- Patients with persistently raised lactate dehydrogenase (LDH \>2.5 ULN)
- Patients requiring triple anticoagulation i.e. warfarin and dual anti-platelet
- Patients participating in another clinical trial
- Patients unable to comply with the protocol mandated procedures for social or other reasons, in the opinion of the investigator and primary care physician
- Eligible, enrolled and randomised patients who develop an infection will have study treatment delayed until 7 or more days after the time point when infection is no longer clinically evident.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Imperial College Londonlead
- British Heart Foundationcollaborator
- Leducq Foundationcollaborator
- Celladon Corporationcollaborator
Study Sites (2)
Papworth Hospital
Cambridge, CB23 3RE, United Kingdom
Harefield Hospital, Royal Brompton and Harefiled NHS Trust
Middlesex, UB9 6JH, United Kingdom
Results Point of Contact
- Title
- Dr Alexander Lyon
- Organization
- Imperial College London
Study Officials
- PRINCIPAL INVESTIGATOR
Sian Harding
Imperial College London
- PRINCIPAL INVESTIGATOR
Alexander Lyon
Imperial College London
Publication Agreements
- PI is Sponsor Employee
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
September 24, 2007
First Posted
September 26, 2007
Study Start
July 1, 2014
Primary Completion
September 1, 2015
Study Completion
September 1, 2015
Last Updated
February 8, 2023
Results First Posted
February 25, 2020
Record last verified: 2023-01
Data Sharing
- IPD Sharing
- Will not share