All our current treatments start out as investigational therapies. We rely on the results from clinical trials to bring these treatments to market. This is only possible with the help of patients who volunteer to take part in clinical trials. The more people in our clinical trials, the faster new treatments can become available to the community.

What is a clinical trial?

Clinical trials are medical studies that involve regular people like you and me. We use clinical trial results to develop new treatments, interventions or tests to detect, treat or manage various diseases and medical conditions.

Some trials examine how people respond to a new treatment, and identify possible side effects. This helps us determine how a new treatment works, whether it’s safe, and if it’s better than treatments already available.

Clinical trials might also compare existing treatments, test new ways to use or combine existing treatments, or observe how people respond to other factors that might affect their health (such as dietary changes).

Other trials look at ways to diagnose diseases earlier, or how to prevent the onset of particular diseases and medical conditions.
 

The World Health Organisation (WHO) defines a clinical trial as:

‘Any research study that prospectively assigns human participants or groups of humans to one or more health-related interventions to evaluate the effects on health outcomes’.

Clinical trial treatments include (but are not restricted to):

  • medications
  • medical devices
  • vaccines
  • surgical and other medical treatments and procedures
  • cellular and other biological products
  • psychotherapeutic and behavioural therapies
  • health service changes
  • preventive care strategies and
  • educational interventions.

Treatments begin as ‘experimental’ and move to ‘standard’ along a clinical pathway that’s checked and regulated at every stage. In the early stages, the new treatment is tested in a small number of people to assess safety and effectiveness. If the results are promising, the treatment moves to later stages of testing where we collect more information on effectiveness and possible side effects by increasing the number of people in the clinical trial.

Summary of clinical trial phases for medicines and biologicals (like vaccines):

Indicative number of participants

10-15
Testing includes a limited number of people with a limited range of doses, for a limited period of time

Objectives

Assess pharmacokinetics

New treatments are tested to gather data on how drugs move around the body and are eliminated from the body.

Indicative number of participants

10-100
This phase may involve the first administration to people , usually small numbers of healthy volunteers or patients

Objectives

Safety and tolerance

Tests identify a safe dose and how best to administer it, and they uncover any side effects for new treatments, by gathering data on how drugs move around and are eliminated from the body.

Indicative number of participants

100-300
Tests include a larger group of patients (up to several hundred)

Objectives

Efficacy and safety

New treatments are tested to demonstrate their effectiveness, optimal dose and safety.

Indicative number of participants

300-3000
Testing usually involves a large group of patients (from several hundred to several thousand)

Objectives

Safety, efficacy or effectiveness

New treatments are tested to demonstrate their effectiveness and safety in the patient group the treatment is intended to benefit.

Indicative number of participants

1000s

Testing involves thousands of people

Objectives

Post-marketing surveillance or resolution of treatment uncertainties

Approved treatments are tested after they’ve been marketed. These studies are designed to monitor the effectiveness of a treatment in the general population, and to collect information about any side effects associated with widespread use over longer periods of time.

They may also be used to investigate the potential use of the treatment in a different condition or in combination with other treatments.

Indicative number of participants

10-15
Testing includes a limited number of people with a limited range of doses, for a limited period of time

Objectives

Assess pharmacokinetics

New treatments are tested to gather data on how drugs move around the body and are eliminated from the body.

Indicative number of participants

10-100
This phase may involve the first administration to people , usually small numbers of healthy volunteers or patients

Objectives

Safety and tolerance

Tests identify a safe dose and how best to administer it, and they uncover any side effects for new treatments, by gathering data on how drugs move around and are eliminated from the body.

Indicative number of participants

100-300
Tests include a larger group of patients (up to several hundred)

Objectives

Efficacy and safety

New treatments are tested to demonstrate their effectiveness, optimal dose and safety.

Indicative number of participants

300-3000
Testing usually involves a large group of patients (from several hundred to several thousand)

Objectives

Safety, efficacy or effectiveness

New treatments are tested to demonstrate their effectiveness and safety in the patient group the treatment is intended to benefit.

Indicative number of participants

1000s

Testing involves thousands of people

Objectives

Post-marketing surveillance or resolution of treatment uncertainties

Approved treatments are tested after they’ve been marketed. These studies are designed to monitor the effectiveness of a treatment in the general population, and to collect information about any side effects associated with widespread use over longer periods of time.

They may also be used to investigate the potential use of the treatment in a different condition or in combination with other treatments.

Summary of clinical trial stages for medical devices:

Indicative number of participants

10-30

Testing usually involves a small group of patients

Objectives

Exploratory

New devices are tested to gather data on safety and performance, and to see if modifications to the design are necessary.

Indicative number of participants

100s

Testing involves hundreds of people

Objectives

Confirmatory

New devices are tested to determine their safety and performance for their intended use.

Indicative number of participants

1000s

Testing involves thousands of people

Objectives

Confirmatory or Observational

New devices are tested to determine safety and performance in broader populations.
OR
New devices are tested to better understand safety, long-term outcomes and health economics.

Indicative number of participants

10-30

Testing usually involves a small group of patients

Objectives

Exploratory

New devices are tested to gather data on safety and performance, and to see if modifications to the design are necessary.

Indicative number of participants

100s

Testing involves hundreds of people

Objectives

Confirmatory

New devices are tested to determine their safety and performance for their intended use.

Indicative number of participants

1000s

Testing involves thousands of people

Objectives

Confirmatory or Observational

New devices are tested to determine safety and performance in broader populations.
OR
New devices are tested to better understand safety, long-term outcomes and health economics.

Current clinical trials

TBC

 

Why do we need clinical trials?

Most modern medical treatments are a direct result of clinical research. New treatments for all diseases and conditions — including cancer, heart disease, high blood pressure and asthma — have been developed through clinical research.

Computer simulation and animal testing can only tell us so much. Without clinical trials, we can’t determine whether new treatments are effective or safe, or whether a diagnostic test works properly. If we want to understand how a new medical treatment or diagnostic test works on the human body, there’s no substitute for working with actual humans.

Clinical trials also make it possible to test and monitor the effects of a treatment on large numbers of people. This ensures that any improvement is a result of the treatment for many people, and not a random effect for one person.

Clinical trials often lead to new treatments that help people live longer and have less pain or disability. Clinical trials also help improve health care services by raising standards of treatment. Doctors and medical staff involved in clinical trials are continually training to provide best practice medical care.

Why take part in a clinical trial?

New treatments that help people live longer, healthier lives are only possible because of the willingness of volunteers to take part in clinical trials. We need volunteers to help find new ways to diagnose, prevent, treat or cure disease and disability. If more people are involved in clinical trials, it may reduce the time it takes for new treatments to become available to the general population.
 

People participate in clinical trials for a variety of reasons:

  • To contribute to the advancement of scientific knowledge
  • To help researchers find better treatments for others in the future
  • To potentially receive a benefit from the treatment for their condition
  • To gain access to new treatments before they become widely available
  • To receive additional care and support by clinical trial staff at no extra cost.

Why GenesisCare?

GenesisCare conducts internationally-recognised, high-quality clinical trials. Our doctors and research staff have a wealth of knowledge and expertise that is helping to improve health care in Australia and around the world. Our clinicians believe that new treatments should only be adopted once adequate assessment takes place to confirm their effectiveness, safety and cost.

All research conducted by GenesisCare conforms to the Ethical Principles of the Declaration of Helsinki and to international Good Clinical Practice guidelines. Before research can go ahead, it’s approved by an independent ethics committee that operates according to the guidelines issued by the National Health and Medical Research Council (NHMRC Guidelines).

You will never be ‘out of pocket’ for participating in a trial. Every patient gets the standard of care treatment – alone or combined with a new treatment. Our job is to work with you to make sure that new treatments live up to their claims. Together, we can improve future health outcomes for all Australians and the wider global community.

It’s important you know that you are not obliged in any way to take part in a clinical trial. If you do take part, you can withdraw at any time without it affecting your ongoing medical care.

What is Transcatheter Alcohol ablation of Septal Hypertrophy (TASH) for Hypertrophic (obstructive) Cardiomyopathy (HCM / HOCM)?

 

Hypertrophic (obstructive) cardiomyopathy (HCM / HOCM) is a condition where, usually due to genetic factors, the muscle that makes up the heart’s pumping chambers grows too thick (see Figure 1). This thickening can occur so as to block the flow of blood out from the heart’s main pumping chamber into the rest of the body. When thickening occurs of this kind the word obstructive is added to the description of the condition.
 
The abbreviations HCM (or if obstruction is present HOCM) are commonly used. One potential treatment option for HOCM, where medications are no longer effective to control symptoms, is Transcatheter Alcohol ablation of Septal Hypertrophy (TASH). The discussion that follows below is general medical information only, and does not replace the need for specific advice from your treating doctors.

 

Figure 1: Hypertrophic (obstructive) cardiomyopathy (HCM / HOCM)

heart

 
 
 
 

The Transcatheter Alcohol ablation of Septal Hypertrophy (TASH) procedure

The Transcatheter Alcohol ablation of Septal Hypertrophy (TASH) procedure involves the following:

  • General anaesthetic (fully put to sleep) with a breathing machine to support breathing
  • An ultrasound probe (transoesophageal echocardiogram probe) is placed down your food pipe to visualise the heart.
  • Tubes are also inserted in the veins in the neck and or leg in addition to a tube in the artery in the arm / wrist or leg (groin). These tubes are called sheaths.
  • A temporary pacemaker may be inserted via the tubes in the veins.
  • A tube called a catheter is advanced up to the heart under x-ray steered to the heart artery that goes down the front of the heart:
    • – Through this a wire is positioned in to a small artery called the septal artery.
      – A balloon is steered down this wire to block the artery and allow for near 98 % alcohol (ethanol) to be injected into the artery (and nowhere else).
      – See Figure 2.
  • This causes the heart muscle in this area to die (a type of permanent heart attack) reducing the amount of thickened muscle.

 

Figure 2: Transcatheter Alcohol ablation of Septal Hypertrophy (TASH) procedure

fig2

Are there any risks with Transcatheter Alcohol ablation of Septal Hypertrophy (TASH) for Hypertrophic (obstructive) Cardiomyopathy (HCM / HOCM)?

The procedure has many potential risks. The information supplied here is for general reference only based on the published data around the procedure from across the world. Risks specific to your condition and overall situation need to be discussed with your treating doctors. Potential risks of TASH include:
 
 
 

5% or greater risk of:

  • Requiring a permanent pacemaker to be implanted after the procedure.
  • Failure of the procedure to successfully modify the heart enough to improve symptoms (and may require repeat procedures).
  • If the arm / wrist artery is used, 5 – 10% risk of that pulse blocking / disappearing permanently.
  • Bruising and pain, sometimes with a large bruise (called a haematoma) at the area where the tubes go in that can take several weeks to resolve.

1 – 2% risk of:

  • Heart attack leading to heart failure, need for heart transplantation, need for stenting of heart arteries or death.
  • Stroke or embolism (resulting in permanent disability, blindness, limb loss or death).
  • Vascular access complication (problem with the artery or vein leading to the heart) needing an operation or medication injection (thrombin) to fix and or requiring a blood transfusion and uncommonly resulting in permanent nerve damage or loss of limb (rare).
  • Post procedure infection, blood clot (DVT) and pain.

Uncommon / rare events:

  • Adverse reaction to the general anaesthetic leading to permanent disability or death (<1/10,000).

5% or greater risk of:

  • Requiring a permanent pacemaker to be implanted after the procedure.
  • Failure of the procedure to successfully modify the heart enough to improve symptoms (and may require repeat procedures).
  • If the arm / wrist artery is used, 5 – 10% risk of that pulse blocking / disappearing permanently.
  • Bruising and pain, sometimes with a large bruise (called a haematoma) at the area where the tubes go in that can take several weeks to resolve.

1 – 2% risk of:

  • Heart attack leading to heart failure, need for heart transplantation, need for stenting of heart arteries or death.
  • Stroke or embolism (resulting in permanent disability, blindness, limb loss or death).
  • Vascular access complication (problem with the artery or vein leading to the heart) needing an operation or medication injection (thrombin) to fix and or requiring a blood transfusion and uncommonly resulting in permanent nerve damage or loss of limb (rare).
  • Post procedure infection, blood clot (DVT) and pain.

Uncommon / rare events:

  • Adverse reaction to the general anaesthetic leading to permanent disability or death (<1/10,000).