Clinical Trials

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HeartWorks Clinical Trials for CHD

HeartWorks is leading the world’s largest effort to develop new treatment options for CHD patients. Establishing new therapies is a resource-intensive process and requires a coordinated effort between product developers, clinicians, medical institutions, funding mechanisms, and, most importantly, patients. Patients engaged and willing to participate are essential to conduct the studies to generate the data required by regulatory agencies and clinicians to approve and adopt new therapies into clinical practice.

HeartWorks Sponsored research activities and participation locations are listed below, along with definitions related to clinical research and resources to identify additional clinical research opportunities.

The Co-op at HeartWorks

A database of members medical journey data to inform future clinical innovation and design of clinical trials which address the needs of the members.

Clinical Readiness Skin Punch Biopsy Sample Collection

We’re collecting a small skin biopsy sample to create patient-specific stem cells. These cells are made for promising regenerative cardiac therapies which you may be eligible for in the future.

Bioengineered Cardiac Tissue

Skin cells that are manufactured into contracting cardiac lineage to be delivered into the heart muscle to determine if it will strengthen the heart.

Sponsor: Heartworks

The Co-op at HeartWorks

Purpose: This study aims to create a database of members medical journey data to inform future clinical innovation and design of clinical trials which address the needs of the members. The knowledge generated from this study will help advance the care of CHD patients through the deliberate action of The Co-Op @ HeartWorks members.

Protocol Summary: This protocol is a research study involving human subjects diagnosed with Congenital Heart Defects/Disease (CHD). The Co-Op @ HeartWorks is a cooperative between the research platform at HeartWorks and members of the CHD community. Individuals choosing to participate will be referred to as ‘members’ of the co-op. Unlike a traditional disease registry, the members of The Co-Op @ HeartWorks will actively inform and contribute to the future studies affecting their health. Members will provide their data on a schedule of their choosing. There are no scheduled visit windows.

Eligibility Criteria: Potential members will self-declare their eligibility as a CHD patient (or caregiver of a CHD minor) and provide diagnoses upon enrollment.

Learn More / Become a Member	Site Contact Info
The Co-op at HeartWorks	Clint Hagen / clint@webuildhearts.org

Sponsor: Heartworks | site: mayo clinic

Clinical Readiness Skin Punch Biopsy Sample Collection

Purpose: Generate autologous iPSC in a manner compliant with requirements for future autologous clinical use. This may reduce manufacturing time when patients are identified as eligible to receive autologous product under a separate interventional treatment protocol.

Protocol Summary: A skin biopsy will be collected and used to complete a portion of cellular manufacturing.

Eligibility Criteria:

Individuals who meet all of the following criteria are eligible for enrollment as study participants:
1. Age 18 and older
2. Congenital heart disease

Individuals who meet any of these criteria are not eligible for enrollment as study participants:
1. Previous cardiac transplantation
2. Unable or unwilling to consent
3. HIV diagnosis
4. Hepatitis diagnosis

Site	Location	Study Coordinator	Contact Information
Mayo Clinic	Rochester, MN	Lori Riess	hlhs@mayo.edu

Sponsor: Heartworks | Site: mayo clinic

Bioengineered Cardiac Tissue Phase I First in Human

Purpose: The purpose of this study to test the feasibility and safety for autologous (from your own body) skin cells that are manufactured into stem cells of cardiac lineage to be delivered into the heart muscle to determine if those stem cells will strengthen the heart muscle and can be used as an additional treatment for the management of congenital heart disease.

Protocol Summary: Autologous (self) cardiac lineage cells will be delivered into the heart of enrolled subjects during a single administration episode. Subjects will be followed for the remainder of their life, assessing both cardiac function and formation of tumors anywhere in the body.

Eligibility Criteria: Not limited to information below. Coordinator will provide more criteria.

Individuals who meet all of the following criteria are eligible for enrollment as study participants:
1. Age 18 and older
2. Congenital heart disease
3. End-stage systolic heart failure, defined as Class IV according to New York Heart Association (NYHA) with abnormal visually estimated ejection fraction below 40%.
4. Prognosis of 1 to 1.5 years survival at time of skin biopsy.
5. The patient falls into one of the following categories:
-Currently listed for heart transplantation at an accredited program in the US but has an expected waiting time for a suitable organ that is likely longer than anticipated life-expectancy;
– Has been denied access to a heart transplantation at an accredited US institution;
– Is currently on or planning to be on mechanical support as destination therapy.
– All guideline directed therapy available to the subject has been maximized, for a minimum of 3 months prior to enrollment.

Individuals who meet any of these criteria are not eligible for enrollment as study participants:
1. Previous cardiac transplantation
2. Unable or unwilling to consent
3. HIV diagnosis
4. Hepatitis diagnosis

Site	Location	Study Coordinator	Contact Information
Mayo Clinic	Rochester, MN	Lori Riess	hlhs@mayo.edu

Changing the trajectory of CHD treatment options and outcomes

HeartWorks Clinical Research Education

Clinical Research

Clinical Research includes all research involving human participants. It does not include secondary studies using existing biological specimens or data collected without identifiers or data that are publicly available. Clinical research is necessary for new discoveries and treatments options to become available to patients on a routine basis (become standard of care), with the goal of improving outcomes.

Clinical Trials

Clinical Trials are a type of clinical research study involving human participants assigned to an intervention in which the study is designed to evaluate the effect(s) of the intervention on the participant and the effect being evaluated is a health-related biomedical or behavioral outcome. This is synonymous with an Interventional Study by NIH definition.

Observational study

Observational study are research studies in which researchers simply collect information (called data) from participants or look at data that was already collected. Often, researchers use observational studies to look at (observe) the different ways people behave and how it affects their health. Some observational studies use patient registries. A patient registry is an organized collection of data that patients agree to give. Researchers can use a patient registry to quickly access data provided by hundreds, or thousands, of similar patients.

Clinical Trial Sponsor

A clinical trial sponsor is an individual, company, or organization that manages and funds a clinical trial. The sponsor is responsible for the trial’s design, conduct, and outcome. HeartWorks is the “Sponsor” of several clinical trials, listed above.

Clinical Trial Site

A clinical trial site (or clinical site) refers to a physical location, such as a hospital, research center, or medical facility, where the clinical trial activities are conducted. HeartWorks has established a international consortium which includes several of the top pediatric CHD care centers in North America, and plans are underway to identify similar partner medical centers to be sites for our adult CHD clinical trials.

Resources

There are a number of ways to identify clinical trials or research studies. Results may include both studies currently open to enrollment, not yet enrolling, or completed.

• https://clinicaltrials.gov/
• https://www.mayo.edu/research/clinical-trials

Who We Are

Our Work

Get Involved

Clinical Trials

HeartWorks Clinical Trials for CHD

The Co-op at HeartWorks

Clinical Readiness Skin Punch Biopsy Sample Collection

Bioengineered Cardiac Tissue

Sponsor: Heartworks

The Co-op at HeartWorks

Sponsor: Heartworks | site: mayo clinic

Clinical Readiness Skin Punch Biopsy Sample Collection

Sponsor: Heartworks | Site: mayo clinic

Bioengineered Cardiac Tissue Phase I First in Human

HeartWorks Clinical Research Education

Clinical Research

Clinical Trials

Observational study

Clinical Trial Sponsor

Clinical Trial Site

Resources

Who We Are

Our Work

Get Involved

Clinical Trials

HeartWorks Clinical Trials for CHD

The Co-op at HeartWorks

Clinical Readiness Skin Punch Biopsy Sample Collection

Bioengineered Cardiac Tissue

Sponsor: Heartworks

The Co-op at HeartWorks

Sponsor: Heartworks | site: mayo clinic

Clinical Readiness Skin Punch Biopsy Sample Collection

Sponsor: Heartworks | Site: mayo clinic

Bioengineered Cardiac Tissue Phase I First in Human

HeartWorks Clinical Research Education

Clinical Research

Clinical Trials

Observational study

Clinical Trial Sponsor

Clinical Trial Site

Resources

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