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Clinical trials for gene therapy

What is my role in the clinical trial?

When you decide to participate in a clinical trial, you are agreeing to take part in a carefully controlled study that is seeking answers to specific questions. The study will have a plan (protocol) with very particular requirements that need to be followed to provide the evidence to prove these answers. To play your part in the study, you are agreeing to complete all of the requirements so that the researchers can demonstrate that the protocol was followed. 

The current gene therapy trials in haemophilia are investigating important questions about gene therapy:

  • How well does this gene therapy treatment approach work?
  • How safe is it?
  • What are the effects of the treatment? Are there side-effects?
  • How long does the effect last?

If you agree to participate in one of these gene therapy trials, you will make a commitment to follow the protocol and take part in intensive monitoring, often face-to-face, for more than 12 months.

What is involved for a participant?

People participating in gene therapy clinical trials are making a commitment to frequent study visits over the first 12 months, with ongoing visits for the long-term follow-up, which is often around 5 years.

The first step in the trial is the selection process. Each gene therapy clinical trial will have its own inclusion and exclusion criteria. You will need to fit all the criteria. To check this, you may need to undergo tests or produce previous test results and will need to provide comprehensive records of your treatment history.

The second step is being available to start. Gene therapy clinical trials for haemophilia in Australia are part of worldwide studies which are administered by experienced experts in gene therapy often working with local Haemophilia Treatment Centres (HTCs) and clinics. Places in the trial are offered internationally to all the sites when they become available. There are often small numbers of places and you would need to be ready to start the trial and have the flexibility to fit with its schedule.

The third step is to start the trial and follow the protocol for the next 12 months. The pharmaceutical company responsible for the gene therapy provides the trial protocol and those conducting the clinical trial arrange for its delivery. The principal investigator of the study is accountable to the company for making sure the protocol is followed. With the current AAV vector gene therapy trials, the protocol may involve:

  • Multiple visits to the clinic in preparation
  • A visit to the clinic for the gene therapy infusion, followed by a period of monitoring in the hospital to check for any negative (adverse) effects. You may need to stay overnight
  • Then regular visits to the HTC over the next 12 months for testing and monitoring – at times weekly, at other times monthly or quarterly
  • This may mean organising regular travel to the clinic and overnight accommodation
  • Tests may include providing blood, faeces and other body fluid samples, such as semen and saliva
  • Some trials may require you to abstain from alcohol or limit physical exercise
  • Providing comprehensive and real-time electronic records of bleeds and treatments
  • Committing to ensuring that a pregnancy is not possible, which may involve double contraception (eg, condom and contraceptive pill) for both you and your partner.

What follow-up is involved?

The long-term effect of gene therapy is an important question and participants will be followed for some years. This may require visits to the HTC every 3 or 6 months for testing and monitoring for several years – on average 5 years.

What support services are available on a clinical trial?

Support services such as psychological or social support would be provided through the clinic, and may be provided by a referral to an external service.

Date last reviewed: 12 October 2019

Important Note: This information was developed by Haemophilia Foundation Australia for education and information purposes only and does not replace advice from a treating health professional. Always see your health care provider for assessment and advice about your individual health before taking action or relying on published information. This information may be printed or photocopied for educational purposes.

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