Clinical Trials
Clinical trials are research studies that test new treatments, new combinations of treatments, or new ways of managing ITP. They help researchers understand how well a treatment works, what side effects may occur, and whether it could become an option for people living with ITP in the future. For some people, clinical trials offer access to emerging therapies that are not yet widely available.
Why Clinical Trials Matter in ITP
Many people respond well to current ITP treatments, but others may have ongoing symptoms or side effects. Clinical trials play an important role in improving care by:
- Exploring new targeted therapies
- Testing treatments used in other autoimmune conditions
- Understanding which treatments work best for different types of ITP
- Expanding future treatment options
Participating in a trial also helps researchers learn more about ITP and develop better treatments for the community.
Types of Clinical Trials
Clinical trials progress through different phases, each with a specific purpose:
- Phase 1 – Safety and dose-finding Tests a new treatment in a small group to understand safety, side effects, and the best dose range. This phase helps determine how much of the treatment should be given.
- Phase 2 – Early effectiveness Looks at whether the treatment improves platelet counts or reduces bleeding, while continuing to monitor safety.
- Phase 3 – Larger comparison studies Compares the new treatment to existing treatments in a larger group to confirm how well it works and how safe it is.
- Phase 4 – After approval Studies long-term safety and real-world use once a treatment is already available.
Understanding these phases helps you know what to expect and why a particular study may be recommended.
What Clinical Trials Involve
Every clinical trial is different, but most include:
- Regular visits with a study team
- Monitoring of platelet counts and symptoms
- Blood tests and safety checks
- Clear information about risks and benefits before joining
Some trials test medicines already used for other conditions, while others evaluate completely new therapies. Your healthcare team will help you understand what to expect.
What to Expect if you Participate in a Clinical Trial
Before joining a trial, you will receive detailed information about:
- The purpose of the study
- Possible risks and benefits
- How often you will need to attend appointments
- What tests or procedures are involved
- Your rights as a participant
Clinical trials in Australia must be approved by a Human Research Ethics Committee (HREC), ensuring they meet national safety and ethical standards. You can withdraw at any time.
Clinical Trials in Australia and New Zealand
Australia regularly participates in ITP clinical trials, including studies of new immune-modulating medicines and targeted therapies. Trial sites may be available in major cities across several states. Your haematologist can help you understand whether a clinical trial may be suitable for you and how to find current studies.
New Zealand has limited Clinical Trial options, this is something that ITPANZ is looking to address with the various government bodies.
International Clinical Trials
Many ITP trials are also running internationally. These include studies of:
- FcRn blockers
- BTK inhibitors
- SYK inhibitors
- Combination therapies
- New approaches for early-phase and chronic ITP
You can explore global trials through international clinical trial registries.
Is a Clinical Trial Right for Me?
Joining a clinical trial is a personal decision. It may be worth considering if:
- Current treatments are not working well
- You are experiencing significant side effects
- You want access to emerging therapies
- You are interested in contributing to ITP research
Your haematologist can help you weigh the potential benefits and risks.
Support During Clinical Trials
Deciding whether to join a clinical trial can feel overwhelming. Support is available to help you understand your options, prepare questions for your doctor, and connect with others who have participated in research.