We’re Looking at DCM in a New Light
If you have dilated cardiomyopathy (DCM) and are willing to undergo genetic testing to see if it may be caused by a BAG3 gene mutation, you can learn more about the DCMRestore Study. Joining a research study is your choice. You can decide not to join or to leave the study at any time.
Learn more about the studyThe DCMRestore Study at a Glance
We are enrolling about 30 adults with BAG3-associated DCM at study sites in approximately nine countries. Enrollment depends on the site capacity and eligibility.
Eligible participants must:
- Be 18 to 70 years of age
- Have a diagnosis of DCM that is or may be caused by mutation of the BAG3 gene
- Testing for BAG3 gene mutations will be performed at screening
- Have diagnosed chronic heart failure for at least three months before the study
There are additional requirements, which the study team will discuss with interested individuals.
About the DCMRestore Study
The purpose of this study is to find out how safe an investigational medication gene therapy is and how well it works to stabilize disease and prevent further disease progression for adults who have BAG3-associated DCM. An investigational medication gene therapy is one that has not been approved for use and is considered experimental for this study. The investigational medication gene therapy is given once as a single intravenous (IV) infusion as part of the study. It is designed to deliver a working BAG3 gene to the heart tissue. The study will look at safety and whether it may help stabilize disease or slow it down.
There are two parts to this study: Part A and Part B.
- Part A: Researchers will aim to determine the best dose of the investigational medication gene therapy for adults with BAG3-associated DCM.
- Part B: Researchers will evaluate how well that dose may work to stabilize DCM and prevent further disease progression.
Study Participation
Participation in the DCMRestore Study will include:
Screening
Interested individuals will be evaluated to see if the study is a good match for them. If they are eligible and decide to join, they will attend additional visits and may need to take medications (such as medications to lower their immune response) to prepare for the infusion of investigational medication gene therapy.
Dosing and Inpatient Stay
Participants will be admitted to the hospital to receive the investigational medication gene therapy infusion and be closely monitored. Blood sample collections will be done before and after the infusion. After the infusion, participants will begin taking antibiotics. The investigational medication gene therapy can cause side effects. The study team will explain the risks and what to do if you have symptoms.
Short-Term Follow-Up
After participants are discharged from the hospital, they will attend regular follow-up visits so that the study team can check on their health.
Long-Term Follow-Up
Participants will have occasional check-ins so that the study team can evaluate their health over a long period of time after receiving the investigational medication gene therapy.
Throughout the study, participants will undergo regular tests and assessments, such as echocardiograms and electrocardiograms (ECG). They will also be asked to complete some tasks at home, including taking their study medications and using wearable medical devices at certain points.
These tests, along with pre-treatment immunotherapy, the investigational medication gene therapy, and the ongoing monitoring of your health, will be provided at no cost. No insurance is needed to join this study. There may be costs for medical care that is not part of the study.
About BAG3-Associated Dilated Cardiomyopathy
Dilated cardiomyopathy, or DCM, is a condition where the heart’s main pumping chamber (left ventricle) becomes enlarged and weak, making it hard for the heart to pump blood effectively. DCM can be a result of many conditions, such as viral infections, heart disease, and overconsumption of alcohol, but it may also be caused by genetics.Footnote 1
BAG3-associated DCM is an inherited condition, and occurs when the BAG3 gene, which normally creates a protein that helps maintain heart health, is faulty. If the proteins aren’t made correctly, heart cells can be left vulnerable and the muscle may weaken over time, leading to DCM.Footnote 2 Individuals with DCM may undergo genetic testing to see if their condition is caused by a faulty BAG3 gene.
Symptoms of DCM may include:
- Shortness of breath
- Tiredness
- Swelling of the feet, ankles, or abdomen
- Chest pain
- Irregular heartbeatFootnote 1
Since there is no cure for BAG3-associated DCM, standard treatments currently focus on managing symptoms and slowing the progression of the disease.
About Gene Therapy
Genes are the building blocks that determine how the body functions. When genes are faulty or damaged, they may function improperly, causing or leaving certain parts of the body susceptible to disease.
Standard treatments for diseases caused by faulty genes typically focus on managing symptoms and slowing disease progression, but different therapies are beginning to emerge that may address these diseases at their root cause.
Gene therapy is an emerging approach to treating diseases caused by genes that are faulty or damaged. It involves introducing, correcting, or replacing genes within an individual’s cells with the goal of restoring normal function.
The following resources can provide additional information about gene therapy:
- American Society of Gene and Cell Therapy – patienteducation.asgct.org
- National Organization for Rare Disorders – rarediseases.org/gene-therapy
What You Should Know About Clinical Research Studies
Clinical research studies look at an investigational medication, medical device, or procedure to see if it is safe, how it affects the body, and whether it may work to treat a disease. Clinical research studies are conducted by doctors who are responsible for the study participants’ study-related care.
In most countries, the regulatory health authority requires that several phases of clinical research be performed to better understand the safety and effectiveness of investigational medical gene therapies and certain medical devices and procedures.
Clinical research studies must be reviewed by an institutional review board (IRB) or ethics committee (EC). An IRB/EC is an independent group that is responsible for helping to protect the rights and well-being of study participants. In addition, every study participant is monitored with study-related medical tests and exams before, during, and sometimes even after the study.
Participation in any clinical research study is completely voluntary, and you may choose to withdraw from the study at any time for any reason. If you would like to leave the study, you should discuss this with your study doctor, who will give you information about how to do this safely.
Throughout the study, the study team will perform tests and procedures to check on your health. These tests and procedures include vital sign measurements (such as blood pressure and temperature), physical exams, height and weight measurements, blood and urine sample collections for lab tests, heart tests such as electrocardiograms (ECGs), questionnaires on how you are feeling, and other assessments.
Participation in the DCMRestore Study includes a screening period, a dosing and inpatient period, a short-term follow-up period, and a long-term follow-up period.