HCM Awareness Day

February 9, 2024 – Blog for Patient Community (WomenHeart)

Participating in clinical trials can help advance therapies for cardiovascular diseases
such as HCM. Please read below about what clinical trials (also called research
studies) are and about the phases of clinical trials.

First, what are clinical trials and how do they work?

Clinical trials are research studies in human participants that explore how an
investigational drug affects the body and a disease. An “investigational drug” is a drug
that is being studied and is not yet approved by health authorities for doctors to
prescribe to the general public.

What are the phases of a clinical trial?

Clinical trials are carried out in a series of steps, known as phases, to study whether the
investigational drug is safe and effective for people to use.

A typical series of steps starts from early, small Phase 1 studies to late-stage, larger,
Phase 3 studies.

Phase 1
During Phase 1, usually the shortest phase, researchers test an investigational drug for
the first time in humans by giving it to a small number (20-80) of healthy volunteers to
evaluate how the drug works in humans and identify side effects. The information from
Phase 1 studies is important to the design of Phase 2 studies. In general, approximately
70% of drugs move to the next phase.

Phase 2
During Phase 2, the investigational drug is given to a larger number of people (100-300)
who have a particular disease or condition to further evaluate the side effects and
whether the drug works as treatment for a specific disease.

Phase 3
During Phase 3, the investigational drug is studied in people with the target disease or
condition to further evaluate side effects and how well the drug works. Sometimes
known as pivotal studies, these studies involve hundreds to thousands of participants
and can last several years. Phase 3 studies provide most the data required by health
authorities to assess the safety and effectiveness of the drug.

After Phase 3, health authorities, for example, the FDA in the US, thoroughly examine
all submitted data on the investigational drug and decide to approve it or not to approve
it.

As you can see, there are many required steps for a new drug to be developed and
approved. This process takes 10-15 years on average.

Phase 4
After an investigational drug is approved and available by prescription, phase 4 clinical
trials are sometimes conducted. Phase 4 studies are designed to find out more about
side effects and long-term risks and benefits.

About Hypertrophic Cardiomyopathy (HCM)
Cardiomyopathy is a family of diseases that affect the heart muscle and its ability to
pump blood through the body. Cardiomyopathy can affect anyone of any age or race. It
is not well known – it’s often misdiagnosed or undetected.

One of the most common types is called hypertrophic cardiomyopathy or HCM. In
HCM, the heart muscle thickens. As a result, the heart can’t pump blood effectively to
the rest of the body.

Please click here to visit BMS Study Connect to learn more about clinical trials in
patients with HCM and take a simple 8-question pre-screener to see if you may pre-
qualify for HCM clinical trials.

For more information, please call 855-907-3286 or email clinical.trials@bms.com.

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