Are you currently living with BAG3 Cardiomyopathy?
If so, you may be interested in participating in the study of RP-A701, an investigational gene therapy being developed specifically for people with dilated cardiomyopathy caused by a BAG3 mutation.
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What is the RP-A701 clinical trial?
RP-A701 is an investigational gene therapy being developed for people with dilated cardiomyopathy (DCM) caused by a mutation in the BAG3 gene. The investigational therapy is designed deliver a working copy of the BAG3 gene to heart muscle cells, with the goal of helping those cells produce the BAG3 protein at more typical levels.
By addressing the underlying genetic cause of the disease, this investigational approach is intended to support healthier heart cell function and slow or stabilize disease progression.
This clinical trial is an early-phase study. Its primary purpose is to evaluate the safety of RP-A701 and to understand how the therapy behaves in the body. The study will also explore potential effects on heart function and other disease-related measures.
Participants will receive a single intravenous dose of RP-A701. Medications will be given before, during, and after the infusion to help manage the body’s immune response. Participants will be followed closely for approximately two years after treatment, with additional long-term follow-up lasting up to 3-5 years, as required for gene therapy studies.
Participation is subject to eligibility criteria determined during screening. RP-A701 is investigational and has not been approved by regulatory authorities.
All study related costs, including travel and lodging, will be provided by the study sponsor.
This study is being conducted by Rocket Pharmaceuticals Inc.
Who can join the RP-A701 clinical trial?
Male or female between 18 and 65 years of age at the time of enrollment
Documentation of a pathogenic or likely pathogenic variant of BAG3 by a CLIA-certified laboratory
Clinical diagnosis of DCM requiring each of the following:
- Mild to moderate systolic dysfunction as defined in the study protocol.
- Absence of severe coronary artery disease as defined in the study protocol.
- Absence of uncontrolled hypertension, significant cardiac valve disease, or systemic disease known to cause cardiomyopathy.
- History of implantable cardioverter-defibrillator (ICD) implantation at least 3 months prior to clinical trial screening.
- NYHA Class II or III symptoms.
What are the key exclusion criteria?
- Previous participation in a study of gene transfer or gene editing.
- Gene testing indicating that the cardiovascular disease may be related to a genetic cause other than a BAG3 pathogenic variant.
- Severe Cardiovascular disease as defined by study protocol and assessed by Principal Investigator.
Note: This is not a complete list of inclusion and exclusion criteria. The Principal Investigator at the study site will discuss the details of criteria with potential study participants.
What is involved in participating?
Clinical Trial – Currently Enrolling
Screening
Takes place over 2-3 visits, includes medical history and assessments, and occurs approximately 2 months prior to gene therapy.
Outpatient Pre-Treatment
Prepares the immune system and occurs in the 2 weeks leading up to the gene therapy.
Gene Therapy Treatment
Inpatient at a hospital for approximately 4-14 days with daily monitoring for safety.
Follow-up
Weekly monitoring in the initial month, bi-weekly in the second month with additional follow-up at months 3, 6, 9, 12, 18 and 24.
Clinical Trial Ends
Study Clinic Locations
Dr. Barry Greenberg
Principal Investigator
(Coming Soon)
Dr. John Giudicessi
Principal Investigator
(Coming Soon)
Dr. Daniel Judge
Principal Investigator
Inquiry Form
Complete the form below to learn more about the RP-A701 clinical trial. After you have submitted the form, a patient advocacy expert from our partners at the SADS Foundation will contact you to discuss the trial, answer your questions, and — if the trial is right for you — help connect you directly with a participating study site.
About BAG3 Dilated Cardiomyopathy
Dilated cardiomyopathy (DCM) is a condition in which the heart becomes enlarged and weakened, making it harder to pump blood to the rest of the body. In some people, DCM is caused by changes in certain genes, which are commonly referred to as variants or mutations.
One of these genes is called BAG3, which helps heart muscle cells stay healthy and function properly. When the BAG3 gene does not work as it should, heart muscle cells can weaken over time. This can lead to heart failure symptoms, abnormal heart rhythms, and an increased risk of hospitalization or heart transplantation.
BAG3-related DCM is a rare, inherited form of heart disease that can affect people from adolescence through adulthood. Currently, there are no treatments approved specifically for DCM caused by BAG3 gene mutations.
Most patients are treated with standard heart failure medications and, in some cases, implanted devices that help manage symptoms and reduce complications. While these treatments can be lifesaving, they do not address the underlying genetic cause of the disease. For some individuals, heart transplantation may eventually be required, highlighting the need to study investigational treatments that target the root cause of BAG3-related DCM.
More Information
A more detailed description of the trial can be found at: