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3 days ago · Your Guide to Reading Scientific Papers in HLHS


Scientific papers, also called research papers or journal articles, are a way for scientists to communicate knowledge publicly. Because researchers often write these papers to share their work with others in that field, they aren’t always easy for non-scientists to understand.

But finding and reading scientific papers is easier than you might think! Part III of our Demystifying Clinical Trials series will teach you how you to find, read, understand, and think critically about scientific papers.

Journals, Articles, and How to Find Them

A journal is a periodical that regularly publishes a collection of scientific articles. For example, the New England Journal of Medicine publishes articles on general medical topics, Nature publishes on general science, the American Journal of Cardiology focuses specifically on cardiology, and many other journals publish on cancer, pediatrics, etc. Many journals require a subscription, but most also provide articles for free after a few months. (For more on the process of how articles are written and accepted for publication, you can read our earlier blog post here.)

The American Association for Cancer Research has a great review of the most common sites to search for journal articles. These sites, along with Google Scholar, allow easy searching by keyword, disease, author, title, or journal. In addition to these sites, many doctors have profile pages on their hospital’s or clinic’s website that include a list of their recent publications. This is often provided as a citation list, which is a standard format listing the author’s names, year of publication, title, journal of publication for each article. Information from the citation can be used to find a paper on one of the common journal article search websites.

Parts of a paper

Newspapers have the same regularly occurring sections in the same order (major news, local, sports, weather, etc.). Similarly, scientific papers also have the same regular sections in the same order to ensure that information is presented consistently across all journals. Most scientific papers have the following sections:

  • Title and authors: The general focus of the paper, who wrote it, and where they work
  • Abstract: Spoiler alert – this is an executive summary of the entire paper, including the findings and conclusion. Abstracts are always provided for free to help readers determine if they want to keep reading (or possibly pay for) the whole article.
  • Introduction/Background: Reviews previous research, why this study was done, and the hypotheses the researchers had before starting.
  • Methods: Explains how the research was done. This section includes details on the sample (animals, humans, adults, kids, etc.), size of the sample, new drug or treatment procedures, data collected, and more. The section is very detailed so that other scientists can replicate the study to confirm the findings, and so people know exactly what was done in case there are questions.
  • Results: Extremely detailed scientific results. This section presents information on data analysis, technical facts and figures, and usually presents large amounts of statistical results in tables and graphs.
  • Discussion/Conclusion: This is the reader-friendly explanation of the results section. It also usually includes discussion of any weaknesses or limitations of the study and ideas for future research.
  • References/Bibliography: The citation list of previous research that informed this paper.

HLHS blog - ucb phase i paper

Most papers also include tables, graphs, and figures (pictures) to visually share important information. Journal articles are strictly limited to only a few thousand words – and a picture is worth a thousand words! In addition, it’s often easier for readers to see a picture demonstrating something like umbilical cord blood collection than to read several paragraphs and try to imagine the process.


How to Read a Scientific Journal Article

There are three main reasons people read scientific papers: 1) interest in a particular topic, 2) to gain information needed for treating patients, or 3) to inform new scientific research. With so many papers being published in so many places now, even professionals can use tips on how to efficiently read journal articles. The best suggestion comes from Dr. Subramanyam (2013):

The cardinal rule when reading papers is: Never start reading an article from the beginning to the end. It’s better to begin by identifying the conclusions of the study by reading the title and abstract.… After reading the abstract and conclusions, if the reader deems it interesting or useful, then the entire article can be read.

Journal articles are not novels that must be read from start to finish. Start by reading the abstract to get an overview of the sample, what was done in the study, and what the researchers learned at the end. Then, if you want to know more or still have questions, you can read the Introduction and Discussion sections. (The Methods and Results section share extremely technical data that is most useful to other scientists in that field.)

Thinking Critically about Journal Articles

As important as knowing the sections and how to read them is thinking critically about the paper you’re reading. The article may have been peer-reviewed by experts, but there are still important questions to keep in mind.

For instance, think about the sample. How many people did the researchers collect data on? Is the paper reviewing data from thousands of people across multiple countries, or only a few patients from one hospital? Where were they from? When was the data collected (e.g., recently or more than five years ago)? If the topic or condition is rare there will be fewer people available to collect data on, but looking at the sample size is always key when thinking about the bigger picture.

Another question to keep in mind is whether the authors discussed limitations to their study. All research has strengths and weaknesses, and the authors themselves should point out not only what might have hampered their work, but also how future research can resolve the problem. Additionally, most journals require authors to submit financial disclosure statements on how the study was funded and whether or not the authors receive money from an organization that presents a conflict of interest in their work.

The HLHS Program Presents – #ResearchRecap

Finding and reading scientific papers isn’t as overwhelming, or time-consuming, as it used to be. The internet now makes it easy for scientists share their work, and for the public to find and compare results from many different studies. There’s also a new movement among scientists to better communicate their work to the public in ways that are easy to understand. Many researchers now share their work and even answer questions from the public on their social media pages.

The Todd and Karen Wanek Family Program for HLH fully supports this initiative and is excited to announce a new regularly-occurring series – #ResearchRecap! Beginning in September, our blog and social media pages will regularly provide a brief, reader-friendly overview of new scientific papers on HLHS or related congenital heart defects. We’ll try to include papers that are available to read for free, but will always bring you the most recent and most exciting research being published. Stay tuned!




The Todd and Karen Wanek Family Program for Hypoplastic Left Heart Syndrome (HLHS) is a collaborative network of specialists bonded by the vision of finding solutions for individuals affected by congenital heart defects including HLHS. Our specialized team is addressing the various aspects of these defects by using research and clinical strategies ranging from basic science to diagnostic imaging to regenerative therapies. Email us at to learn more.

Fri, Aug 9 9:44am · Register Now for Feel the Beat 2019! in HLHS

            You are invited to the 7th Annual Feel the Beat event on

                                     Saturday, October 12, 2019!



Our annual Feel the Beat event is a family-friendly event for anyone and everyone interested in learning more about hypoplastic left heart syndrome (HLHS) and the work the Todd and Karen Wanek Family Program for HLHS is doing.  It’s also a great opportunity to connect with other heart-warrior families!

Feel the Beat was created in 2013 as team members from the HLHS program were looking for ways to bring HLHS families together, and to thank research participants for all that they’ve done for the program. None of the program’s research work would be possible without support and participation of families.

Now in its seventh year, Feel the Beat has grown and continues to bring together families, advocates, researchers, and physicians to transform the future of HLHS. The theme this year is “Elevate” — raising awareness and celebrating how research enhances the lives of individuals with HLHS.  We’ll be highlighting the role of Clinical Research Coordinators on this year’s panel.  We have coordinators and families from several HLHS Consortium sites who will share their experiences and describe how impactful this positive relationship is to newly-diagnosed families.

We’ll also have a very inspiring special guest, Naomi Babcock, who is a 17-year old, pre-professional ballerina with HLHS.  Naomi and her mother will walk us through their journey from being diagnosed after birth, to becoming a student who is training full-time in New York.  Naomi will also give a special performance and meet-and-greet to end the day!

Please join us on October 12, 2019 from 8:00 a.m. until 2:00 p.m. in Rochester, MN.

Register for Feel the Beat today!

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The Todd and Karen Wanek Family Program for Hypoplastic Left Heart Syndrome (HLHS) is a collaborative network of specialists bonded by the vision of delaying or preventing heart failure for individuals affected by congenital heart defects including HLHS. The specialized team is addressing the various aspects of these defects by using research and clinical strategies ranging from basic science to diagnostic imaging to regenerative therapies. To learn more or to participate in the research, email and follow the HLHS program on Facebook, Twitter and Instagram.

Mon, Jul 22 10:55am · Phase I Stem Cell Trial Results Published! in HLHS


We’re proud to announce the publication of results from our Phase I stem cell delivery trial!  This paper is the first report of a first-in-infant FDA-monitored trial of a new stem cell therapy in the treatment of hypoplastic left heart syndrome (HLHS).  The paper was published in the Journal of Thoracic and Cardiovascular Surgery and is available to read free-of-charge here.



Stem cells were first discovered by Russian-American scientist Alexander Maksimov in 1908. Sixty years later, the University of Minnesota performed the world’s first successful bone marrow transplant, which is now the standard treatment for blood cancers like leukemia.  In 1978, stem cells were discovered in umbilical cord blood (UCB), and in 2006, scientists created induced pluripotent stem cells which are cells that have been “reprogrammed” from one part of the body into a stem cell-like state. In 2010, scientists in Germany reported using stem cells from bone marrow to treat an 11-month old with HLHS who was in heart failure.  The National Institutes of Health has a great primer on stem cells here.

According to the International Society for Stem Cell Research, “Stem cells are the foundation for every organ and tissue in your body.”  Transplanting stem cells either from the same person (autologous) or from a donor (allogeneic) has been shown to be safe.  However, there are many different kinds of stem cells from all parts of the body that each have different purposes, so scientists first had to do preclinical research to determine which cells would be best to use in regenerating heart muscle.

Researchers found that UCB contains mononuclear stem cells (MNCs), which interact with whatever environment they’re in by releasing cytokines or growth factors.  When put in an environment like the heart, those growth factors act like fertilizer and can stimulate new heart muscle to grow bigger.


Phase I Clinical Trial

Decades of research has been done using stem cells to treat heart disease, but Paper - UCBalmost all of the research has been in adults, not in children or people with congenital heart defects.  The first step in testing any new treatment or drug is to start with a small group of subjects in a Phase I, or pilot study. The goal of a Phase I study is very simple – make sure the new treatment is safe.

Because the heart muscle is more susceptible to regeneration at younger ages, and because autologous MNCs have the lowest rate of complication, researchers on this study decided to collect MNCs from an HLHS-diagnosed baby and inject them into the baby’s own during a planned surgery at three months of age (e.g., the Glenn procedure).

MNCs are also found in bone marrow, but harvesting bone marrow from an infant is challenging and risky.  So researchers decided to collect UCB from the baby at birth and harvest the cells from there.  Because of this novel approach to collecting and processing UCB, this Phase I study also had an additional goal of making sure the entire process was feasible.

There were initial challenges with UCB collection in the early stages of the study.  Collecting UCB is generally done in the delivery or operating room, which is a busy place with many people taking care of the new baby and mother.  Some early collections didn’t have enough cord blood to harvest enough MNCs.  New training was provided to emphasize the importance of collecting as much cord blood as possible, and the collection rates increased significantly.

The first stem cell delivery on this Phase I study was performed in 2015 and our paper reports on the first 10 patients treated at the Children’s Hospital at OU Medicine, Mayo Clinic, and Children’s Hospital of Philadelphia.  A total of 23 patients have now been treated on the trial at additional hospitals in the HLHS Consortium including the Children’s Hospital of Los Angeles, Children’s Hospital Colorado, and Children’s Minnesota.

We are proud to share that the results published in this first paper demonstrate that collecting UCB from an HLHS-diagnosed infant and injecting the MNCs into the heart during the Glenn is both safe and feasible.  There were no operative deaths, and none of the children had any significant safety concerns over the six months after surgery.  According to Dr. Tim Nelson, MD, PhD, Director of the Todd and Karen Wanek Family Program for HLHS at Mayo Clinic:

“The infrastructure is now in place to collect and process stem cells with this method for any HLHS baby born in the United States.”

Phase IIb Clinical Trial

The Phase I trial showed that the procedure is safe and feasible.  However, there weren’t enough patients treated to determine its efficacy (e.g., how effective it is).  The promising results suggesting improved growth rates and right ventricular ejection fraction (e.g., the amount of blood being pumped out of the right side of the heart and into the lungs to get oxygen) have led to the development of a Phase IIb trial.  A Phase IIb, or pivotal trial, is designed to evaluate the efficacy of a new procedure by treating more patients.

Paper - MNC deliveryThe new study is now open and treating patients at HLHS Consortium hospitals across the country.  It includes both a treatment arm for infants whose UCB has been collected and a control arm for patients diagnosed after birth or who did not collected cord blood for use in the study.

“We’re excited to keep working with the physicians and research teams at these sites, and to continue expanding the consortium with new Centers of Excellence” says Dr. Nelson.  “Individuals with HLHS will have more access to better treatments and groundbreaking clinical trials.”

You can read more about the results of our Phase I paper on the Mayo Clinic News Network.





The Todd and Karen Wanek Family Program for Hypoplastic Left Heart Syndrome (HLHS) is a collaborative network of specialists bonded by the vision of finding solutions for individuals affected by congenital heart defects including HLHS. The specialized team is addressing the various aspects of these defects by using research and clinical strategies ranging from basic science to diagnostic imaging to regenerative therapies. Email the program at to learn more.

Fri, Jul 19 9:42am · HLHS Consortium Builds Team Approach for Surgical Care in HLHS

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Mayo Clinic is a world-renowned, non-profit hospital with a history of pioneering groundbreaking medical practices for congenital heart disease, including development of the first successful heart-lung machine and open-heart surgeries.  Over the past five years, Mayo Clinic has been focused on building a nationwide, research-focused Hypoplastic Left Heart Syndrome (HLHS) Consortium to develop the next generation of optimal treatments for all HLHS patients.

Collaboration among care providers, researchers, and educators helps improve outcomes for individuals with HLHS.  Each member brings strengths to the consortium to create a rigorous and strategically-balanced partnership that helps families across the country access the high-quality resources they need.

As of July 1, Mayo Clinic has decided to collaborate with Children’s Minnesota for the initial surgical care of HLHS patients whose families wish to receive care in Minnesota.  Combining our current volume of patients with those of Children’s Minnesota can be expected to improve outcomes for infants undergoing this complex surgery.

Mayo Clinic will work with Children’s Minnesota for cell-based therapy in neonatal surgery, and will continue to provide diagnostic, subsequent, and ongoing clinical care for patients with HLHS at which Mayo excels.  Mayo Clinic will also continue to pioneer cell-based therapy research through the HLHS Consortium.


Finding the Right Hospital for Your HLHS Needs

The medical care for HLHS is complex and requires several surgeries after birth and in early childhood.  As children with HLHS age, their medical and surgical needs change, and the types of procedures they may need also change.  For example, older patients may need valve replacement, re-replacement, or heart transplant.

Although choosing a hospital or provider may be impacted by insurance or finances, overwhelming research has shown that centers which perform a high-volume of procedures have significantly better outcomes than low-volume centers.  Your provider should be willing to have an open, honest discussion with you about the care your child needs and the center’s outcomes – and you should advocate to get the information you need to feel comfortable to make an informed decision.

Please contact us at for information about Mayo Clinic’s expertise as well as options within the HLHS Consortium.




Tue, Jun 25 2:15pm · New Phase II Clinical Trial Now Open in HLHS

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The Todd and Karen Wanek Family Program for Hypoplastic Left Heart Syndrome (HLHS) at Mayo Clinic is excited to announce a new Phase II clinical trial for infants diagnosed with HLHS before birth. This clinical trial uses stem cells collected from the baby’s own umbilical cord blood (UCB) at birth and delivers them into the heart to help strengthen the heart muscle. There are two parts to this study: a Treatment Arm and a Control Arm.

Treatment Arm

Babies with HLHS standardly undergo a Stage I (Norwood) surgery shortly after birth, followed by a Stage II surgery (Glenn) a few months later. In this study, a baby’s own stem cells from the UCB are injected into the right side of the heart during the Glenn surgery. The stem cells release growth factors that act like fertilizer to help the heart muscle grow bigger and stronger.

A Phase I study was completed in 2018 and showed that the procedure is safe. This Phase II study will help researchers determine if the procedure helps the heart muscle recover after surgery and get stronger in the future.

To participate in the Treatment Arm and be eligible to receive the stem cell delivery, a baby’s UCB must be stored with the Todd and Karen Wanek Family Program for HLHS at birth. There is no cost to collect and store UCB with the program. For more information on UCB banking options, you can read our earlier blog post.

Control Arm

This trial is also open to infants with HLHS who did not have UCB collected. This might happen if a baby was not diagnosed before birth or the collection attempt was not successful. Infants with HLHS can still participate in this groundbreaking clinical trial by enrolling in the Control Arm before the Glenn surgery.

Infants on the Control Arm will not have stem cells delivered during surgery. However, their medical data will provide important information to compare and understand how babies’ hearts are affected by surgery with and without the stem cell delivery. Babies on both arms of this study will also undergo an additional MRI scan after surgery to get an in-depth look at how the heart is functioning. The cost of the MRI will be covered by the study.


Trial Open at HLHS Consortium Sites

The HLHS Consortium was created to bring innovative research opportunities to more patients across the country. UCB can be collected at birth from any hospital and stored with the Program. However, the stem cell delivery and Glenn surgery must be performed at one of the following hospitals in the HLHS Consortium: Children’s Minnesota (Minneapolis, MN), Children’s Hospital of Philadelphia (Philadelphia, PA), Children’s Hospital of Los Angeles (Los Angeles, CA), Children’s Hospital Colorado (Aurora, CO), or The Children’s Hospital at OU Medicine.

“We’re excited to join forces with the physicians and research teams at these sites,” says Tim Nelson, MD, PhD, director of the HLHS Program at Mayo Clinic. “It means that individuals with HLHS will now have more access to participate in groundbreaking clinical trials.”

To learn more about this study, contact or visit our website.


The Todd and Karen Wanek Family Program for Hypoplastic Left Heart Syndrome (HLHS) is a collaborative network of specialists bonded by the vision of finding solutions for individuals affected by congenital heart defects including HLHS. The specialized team is addressing the various aspects of these defects by using research and clinical strategies ranging from basic science to diagnostic imaging to regenerative therapies. Email the program at to learn more.

Mon, Jun 17 11:46am · Ochsner Hospital for Children Joins HLHS Consortium in HLHS

HLHS Consortium Ochsner

NEW ORLEANS — Mayo Clinic’s Todd and Karen Wanek Family Program for Hypoplastic Left Heart Syndrome (HLHS) and Ochsner Hospital for Children are proud to announce their collaboration to prevent heart failure in HLHS, a rare and complex form of congenital heart disease.

Ochsner’s Pediatric and Adult Congenital Cardiac Surgery Program is led by Benjamin Peeler, M.D. who joined Ochsner’s team in 2016 and has brought decades of invaluable experience and leadership to the team.  “Dr. Peeler has been building a team of dedicated professionals, and their work has resulted in a 100% survival rate of the complex Norwood surgery every year since 2017,” says Tim Nelson, M.D., Ph.D., director of the HLHS Program at Mayo Clinic.  “They are passionate about ensuring that all families in Louisiana have access to the best level of surgical care which is why we’re so proud to welcome them to the HLHS Consortium.”

Ochsner is now the eighth member of the HLHS Consortium, which includes six other hospitals: Mayo Clinic, Children’s Hospital of Philadelphia, Children’s Hospital Los Angeles, Children’s Minnesota, Children’s Hospital Colorado, and The Children’s Hospital at OU Medicine.  The HLHS Consortium also includes founding member Sisters by Heart, an advocacy group for parents and families.

Read more about this announcement on the Mayo Clinic News Network.


The Todd and Karen Wanek Family Program for Hypoplastic Left Heart Syndrome (HLHS) is a collaborative network of specialists bonded by the vision of finding solutions for individuals affected by congenital heart defects including HLHS. The specialized team is addressing the various aspects of these defects by using research and clinical strategies ranging from basic science to diagnostic imaging to regenerative therapies. Email the program at to learn more.

Fri, Jun 7 11:04am · Your Guide to Understanding Common Research Terms in HLHS

HLHS Mayo - doctor and patient

                             Demystifying Clinical Trials — Part 2

The clinical research world can sometimes seem confusing. Research teams have many people in different roles, and they may use words that are unfamiliar to people outside of research work.

The guide below defines some commonly used words and phrases. Let us know in the comments below or on our Facebook, Twitter, or Instagram pages if you’d like definitions of other words or parts of the research process!


Accrual – the number of subjects who have completed or are actively in the process of completing a study. The accrual goal is how many subjects are needed to finish the study (2).

Adverse event (AE) – a negative symptom or experience encountered by an subject during the course of a clinical trial. Adverse events can be expected or unexpected.

Assent – a minor child’s affirmative agreement to participate in a clinical trial. Failure to object may not be taken as assent.

Clinical research coordinator – a study team member who manages the day-to-day study tasks as directed by the principal investigator. (3)

Consent form – a document explaining all relevant study information to assist the study subject in understanding the expectations and requirements of participating in the trial. This document is presented to and signed by the study subject.

Control arm/group – a comparison group of study subjects who are not treated with the investigational agent. The subjects in this group have the same disease or condition under study, but receive either a different treatment, no treatment, or a placebo.

Data – the objective information gathered during a research study that is used to determine the results of the study.

De-identification – the process of removing identifiers (personal names, dates, social security numbers, etc.) that directly or indirectly point to a person, and removing those identifiers from the data. De-identification of protected health information is essential for protecting patient privacy (4).

Enroll/Enrollment – the process of an eligible participant signing a consent form and voluntarily agreeing to participate in a research study (2).

Ethics committee – an independent group of both medical and non-medical professionals who are responsible for verifying the integrity of a study and ensuring the safety, integrity, and human rights of the study participants.

Food and Drug Administration (FDA) – the agency within the Department of Health and Human Services (DHHS) that enforces public health laws related to research conduct.

Greater than minimal risk – the research involves more than minimal risk to subjects (2).

Health Insurance Portability and Accountability Act of 1996 (HIPAA) – required the Department of Health & Human Services to develop regulations protecting the privacy and security of certain health information (5). The HIPAA Privacy Rule established the conditions under which health information may be used or disclosed by approved entities for research purposes (6).

Hypothesis – a specific, clear, and testable proposition or prediction about the possible outcome of a scientific research study (7).

Informed consent – the process of discussing a clinical trial that goes beyond signing the consent form. The discussion should provide sufficient information so that a subject can make an informed decision about whether or not to enroll in a study, or continue participation in a study. Informed consent is a voluntary agreement to participate in research, and should be an ongoing conversation throughout a subject’s entire time in the study (8).

Investigational New Drug Application (IND) – the process through which an investigator requests the FDA to allow human testing of a new drug.

Institutional Review Board (IRB) – an independent group of professionals designated to review and approve the clinical protocol, informed consent forms, study advertisements, and patient brochures to ensure that the study is safe for human participation. It is also the IRB’s responsibility to ensure that the study adheres to the FDA’s regulations.

Minimal risk – the probability that harm or discomfort anticipated in the research study are not greater than those encountered in daily life or during routine physical examinations (2).

National Institutes of Health (NIH) – agency within DHHS that provides funding for research, conducts studies, and funds multi-site national studies.

Protected Health Information (PHI) – individually identifiable health information. HIPAA provides federal protections for personal health information and gives patients more control over their health information. It also sets boundaries for how entities and institutions can use and release health records (9).

Placebo – an inactive substance designed to resemble the drug being tested. It is used as a control to rule out any psychological effects testing may present. Most advanced clinical trials include a control group that is unknowingly taking a placebo.

Principal Investigator – the primary individual responsible for conducting a clinical trial and adhering to federal regulations, institutional policies, and IRB regulations (2).

Protocol – a detailed plan that sets out the objectives, study design, and methodology for a clinical trial. A study protocol must be approved by an IRB before research may begin on human subjects.

Randomization – study participants are assigned to groups in such a way that each participant has an equal chance of being assigned to each treatment or control group. Since randomization ensures that no specific criteria are used to assign any patients to a particular group, all the groups will be equally comparable.

Research – systematic investigation designed to develop or contribute to generalizable knowledge.

Standard treatment/Standard of care – the currently accepted treatment or intervention considered to be effective in the treatment of a specific disease or condition.

Statistical significance – the probability that an event or difference was occurred by chance alone. In clinical trials, the level of statistical significance depends on the number or participants studied and the observations made, as well as the magnitude of differences observed.

Subject/Participant – a patient or healthy individual participating in a research study.

Treatment arm/group – a group of study subjects who are treated with the investigational agent.

Visit schedule/Test schedule – the number, frequency, and type of exams, tests, and procedures that research subjects will be expected to undergo during the study. Some visits may be the same as normal clinical care visits, while others may be required just for the purpose of collecting data for the research study.


Definitions taken from unless otherwise cited.










The Todd and Karen Wanek Family Program for Hypoplastic Left Heart Syndrome (HLHS) is a collaborative network of specialists bonded by the vision of finding solutions for individuals affected by congenital heart defects including HLHS. The specialized team is addressing the various aspects of these defects by using research and clinical strategies ranging from basic science to diagnostic imaging to regenerative therapies. Email the program at to learn more.

Mon, May 20 2:30pm · Demystifying Clinical Trials – Part 1 in HLHS

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In honor of Clinical Trials day on May 20th, we’re kicking off a series that’s designed to shed some light on the world of clinical research. Follow us on Facebook, Twitter, and Instagram and stay tuned for posts over the coming months about commonly confusing terms, questions to ask before enrolling in a clinical trial, where to find research results, interviews with members of our research team about their roles, and more!

What is a clinical research trial?

A clinical research trial, also called a research study, is a process designed to test whether a new treatment or drug is safe and effective. There are strict regulations and guidelines, from both the Food & Drug Administration (FDA) and the institution conducting the research, which all researchers must follow to ensure that a trial is conducted safely and reliably.

Why is clinical research important?

As researchers learn more about science and health, they develop ideas, called hypotheses, about new treatments that might help people. However, before these new treatments can be used for everyone, they need to be tested.

The process to test a new drug or treatment starts by giving it to a small number of people and watching closely to make sure it’s safe. The next step is to give it to more people and compare them to people who didn’t get the new treatment. If a clinical trial shows that a new treatment is both safe and effective, researchers share their results publically so doctors know they can use the new treatment regularly.

Who conducts clinical research?

Most major hospitals and universities around the world participate in clinical trial research. Health care device and pharmaceutical companies also conduct trials. All researchers in the US must follow strict rules designed by the National Institutes of Health, FDA, and the institution conducting the research to make sure that participants are kept safe and that their data is kept confidential.

A research team composed of a lead investigator, several co-investigators, research coordinators, statisticians, and sometimes laboratory staff work together to conduct the trial. One of the ways patients are kept safe is by having the research teams follow clear guidelines for how the study will be conducted. In addition, having pre-determined criteria for which patients can enroll ensures everyone is healthy enough to participate.

When should I consider participating in research?

Most clinical trial research involves testing a new procedure or drug that’s intended to treat a specific medical condition. Some studies are observational and are only designed to naturally watch what happens to help researchers better understand a condition. Your doctor may discuss clinical trials that are available if you have been diagnosed with a specific disease or disorder and are healthy enough to participate.

You may also consider participating in a research study if you are healthy and want to help researchers compare data between healthy and unhealthy groups of people. Studies looking for healthy volunteers are often advertised around hospital and university campuses or online.

How is clinical research conducted?

Today’s clinical trials follow steps that are based on the Scientific Method that was developed in the 17th century.  Let’s walk through the process using a new cholesterol-lowering drug as an example.

Hypothesis – Dr. Jane Smith studied the results of other scientists, as well as her own previous research, and developed a theory about a new drug that might lower cholesterol.

Protocol – Dr. Smith writes a long, detailed plan called a protocol. This document explains why she thinks this drug will work better than the current ones, which people will be able to enroll in the study, exactly how and when data will be collected to test the drug, how the data will be measured, and how she will know for sure if it’s safe and effective.

Data Collection and Analysis – Dr. Smith and her research team tell patients with high cholesterol about the new drug and why it might be better than current ones. Those who are eligible and want to enroll are treated according to the plan in the protocol. The research team collects data on their health and outcomes, sometimes over several years, to ensure everyone is safe. Once the data has been collected, the team analyzes the results to see whether or not the drug lowered cholesterol better than the standard.

Publication – The research team then writes a brief paper (usually 6-10 pages) explaining the details of their hypothesis, how they conducted the study, and their results. Just like there are many magazines on a variety of specialized topics, there are many scientific journals that publish papers written by research teams. Dr. Smith would choose a journal related to the topic of their study and submit it for review. All reliable journals have what’s called a “peer-review process” in which experts in the field volunteer to review papers scientifically (both the research team and reviewers are anonymous). If a group of reviewers all agree that Dr. Smith’s study was conducted well and the results are valid, the paper will be published in the journal. Others in the field can then read about it and design similar studies to confirm or contradict Dr. Smith’s results.

Changing Clinical Practice – Once several studies have found that the new drug is safe and effective, doctors everywhere can start using it in regular clinical practice. This means that all patients can get it without needing to enroll on a clinical trial at a particular hospital. The point of clinical research is to continually find safer and more effective treatments for all patients.

Where can I find clinical trials and learn more?

Many local hospitals and universities also have information on their websites about available trials and who to contact.



The Todd and Karen Wanek Family Program for Hypoplastic Left Heart Syndrome (HLHS) is a collaborative network of specialists bonded by the vision of finding solutions for individuals affected by congenital heart defects including HLHS. The specialized team is addressing the various aspects of these defects by using research and clinical strategies ranging from basic science to diagnostic imaging to regenerative therapies. Email the program at to learn more.