The cardiology service at the University of Florida is looking to assess efficacy of therapeutic gene delivery using a virus vector. The study is placebo controlled and double blinded; some dogs will receive the treatment and some will not. However, if there is a benefit to gene delivery, the placebo treated dogs will be treated with gene delivery once a benefit is recognized. To qualify for this study, the following criteria must be met:
- Inclusion Criteria:
- A diagnosis of DCM and previous evidence of congestive heart failure which has been controlled with medical management
- A circulating neutralizing antibody titer to the virus vector of less than 1:20
- Clear of extra-cardiac disease, congenital heart disease or primary mitral valvular disease
- Owner agreement to authorize participation with informed consent and collection of the heart following euthanasia or death from any cause.
- Additional Information:
Eligibility for enrollment criteria: Procedures covered by the study include bloodwork, ECH, and ECG. Enrolled patients also receive repeat screenings at no cost. The value of the study procedure is estimated at $5,000. The cardiology exam ($200) is NOT covered by the study, nor is the cost of thoracic radiographs if not provided by referring veterinarian.
- Contact: Contact the Cardiology Service at the Small Animal Hospital 352-392-2235
- PI: Dr. Meg Sleeper, VMD, DACVIM (cardiology). Prior to setting an appointment, you are STRONGLY encouraged to email Dr. Sleeper that she can help pre-screen potential candidates and to clarify any fees you might incur.
At enrollment, an antibody titer, hematology and biochemistry panel will be used for screening purposes. Dogs will undergo a 3-minute electrocardiogram (ECG), a complete echocardiogram (ECHO) and the owners will complete a quality of life questionnaire. In order to participate in the study, dogs fulfilling the requirements will be randomly assigned to one of two groups: the placebo arm (cardiac injection with saline) or the gene therapy group (cardiac injection with the virus vector: AAV2/6-ARC-s100a1). Standard medical management for DCM and congestive heart failure will continue throughout the study in all dogs (pimobendan, angiotensin converting enzyme inhibitor and diuretic therapy). Saline instead of empty virus will be used as the placebo so that control dogs can undergo gene delivery if the treatment group demonstrates a significant improvement compared to the placebo group. Owners will need to commit to re-evaluations at 2, 4, 6, 9, and 12 months at the University of Florida following therapy.