Gene Therapy for Dobermans with Dilated Cardiomyopathy

The cardiology service at the University of Florida is looking to assess efficacy of therapeutic gene delivery using a virus vector.  The study is open label, so all dogs will be treated. To qualify for this study, the following criteria must be met:

Enrollment Status: Currently Enrolling

• Inclusion Criteria:
  • Must be a Doberman
  • Diagnosed with dilated cardiomyopathy, with or without congestive heart failure (early or late stage disease)
  • 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.
  • An initial screening visit to determine eligibility
• Additional Information: 

  You will not receive any monetary compensation for this study. The study will pay for all expenses related to delivery of the gene therapy and monitoring of cardiac function for one year, including:   

  • Scheduled physical examinations   
  • Scheduled ECG and echocardiography   
  • Baseline laboratory testing (CBC, blood chemistries, urinalysis, antibody screening)   
  • Anesthesia   
  • Cardiac catheterization and injection of the study drug  
• Contact:  Contact the Cardiology Service at the Small Animal Hospital 352-392-2235.  If you have further questions or want to see if your dog qualifies, please complete the Cardiology Study Interest Form.
• 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.

Study Details:

At enrollment, for a CBC (complete blood count), blood chemistries (organ function panel), and testing for antibodies against the gene therapy vectors 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 receive the gene therapy (cardiac injection with the virus vector: AAV2/6-ARC-s100a1). Standard medical management for DCM, and congestive heart failure, if applicable, will continue throughout the study in all dogs (pimobendan, angiotensin converting enzyme inhibitor and diuretic therapy). Owners will need to commit to re-evaluations at 2, 4, 6, 9, and 12 months at the University of Florida following therapy.

Funding for the research is provided through the efforts and generosity of the AKC Canine Health Foundation.