Rung 03 of 06 · From Australian Clinical Trials

Gene therapy clinical trials, and how to find one

The Australian Government’s clinical trials website lets anyone search trials listed in the Australian New Zealand Clinical Trials Registry (ANZCTR), which includes trials with an Australian recruitment site registered there or on ClinicalTrials.gov, the US registry. Each listing includes contact details, so you can ask about that trial, including who can take part. The government does not refer people to individual trials, so the site suggests talking it through with the health professionals involved in your care, support groups for a condition, and people who have taken part in trials.

General information, not medical advice. Whether a trial suits you is for you and your own doctor to work out. The Australian Clinical Trials website is the official place to search and to read more.

Search
australianclinicaltrials.gov.au
Registry behind it
ANZCTR
Taking part
Voluntary, and you can leave at any time
Ethics approval
Required for every clinical trial

Why trials matter for gene therapy

Healthdirect says gene therapy is mostly still in research and clinical trials, and is not generally available in Australia. The Australian Clinical Trials website lists experimental drugs, cells and other biological products among the kinds of intervention that trials study, and it is clear about the trade-off: there is no guarantee that a participant will benefit directly from taking part.

Close-up of a laboratory microscope’s objective lenses, marked 10x, 20x and 63x, above the stage, in cool blue light
A microscope’s objective lenses above the stage. Photo by PublicDomainPictures on Pixabay

The phases a trial moves through

Trials of biomedical interventions usually run in four phases, starting with a few people and growing.

  1. Before phase 1Exploratory studies

    Sometimes called phase 0 or pilot studies: very few people, small doses given once or for a short time, to see how the body responds.

  2. Phase 120 to 80 people

    The first test of a new intervention, to find a safe dose range and identify side effects.

  3. Phase 2For example, several hundred

    Whether it works as intended, with more safety evaluation.

  4. Phase 3Several hundred to several thousand

    Compared with standard care, another intervention or a placebo, while adverse effects are watched.

  5. Phase 4After it is in use

    How well it works in the general population, and adverse effects over longer periods.

Most trials compare groups: one receives the new intervention, another an existing one, standard care or a placebo. Researchers do not use a placebo where going without an effective treatment would put a patient at risk, and must say before a person agrees if a placebo will be used.

  1. Start at the national searchThe Australian Clinical Trials search takes a keyword, and lets you choose a broad health condition, one of which is “Human Genetics and Inherited Disorders”, then a specific condition.
  2. Read the listingEach listing includes contact details for the trial, so you can ask about it, including who can take part.
  3. Look wider if you need toThe International Clinical Trials Registry Platform aims to give a single point of access to trial information from registries around the world, ANZCTR among them, and some Australian organisations list trials in their own fields.
  4. Talk it throughThe site suggests your GP, specialists, nurses or allied health professionals, condition support groups, and others who have been in trials.

Who can take part

Every trial has eligibility criteria: inclusion criteria a person must meet, and exclusion criteria that rule them out. They can turn on age, gender, the type and stage of a condition, earlier treatment and other medical conditions, and a trial may simply have enough people in a category already. Tests, such as a blood test, may be needed before joining.

Taking part is voluntary, and a participant can withdraw at any time without any negative effect on the quality of their medical treatment or their relationship with their doctor. For children, a parent or guardian must give legal consent for a child under 16, and the child may be asked for their own agreement, called assent, in a way that suits their age.

Weighing it up

The site lists possible benefits, such as access to new interventions before they are widely available and closer monitoring of a condition, and possible risks: the intervention may not work, may be less effective than standard care, or may cause unpleasant, serious or even life-threatening side effects, and limited testing in humans can bring risks no one has foreseen. Researchers must describe a trial’s risks in detail as part of informed consent.

The site also publishes its own list of questions to ask about a trial, grouped by the trial, your involvement, risks, costs and what happens afterwards. Rung 06 sets out questions for the doctor about a genetic condition and its treatment more generally.

Who signs off a gene therapy trial

A trial that gives people a genetically modified product can need more than one approval. The OGTR does not require them in any particular order, and says they can run in parallel with its own assessment.

  • An ethics committee. All clinical trial research must be approved by an ethics committee, in Australia usually a Human Research Ethics Committee (HREC). See rung 04.
  • The TGA. A trial using an unapproved therapeutic good must be notified to the TGA or approved by it, and approval under the CTA scheme is mandatory for a Class 4 biological, the class that includes genetically modified human cells, unless one of two exceptions applies. See rung 02.
  • The Gene Technology Regulator. Putting a GMO into a person needs the Regulator’s licence, unless it is a modified human somatic cell that meets specific conditions. See rung 05.

If something worries you during a trial

The Australian Clinical Trials website sets out where to take a concern, roughly in this order:

  1. The researchersFor any undesirable experience with a trial drug, device or procedure, report it to the lead researcher as soon as possible. Their contact details are on the consent form signed before joining.
  2. The ethics committee that approved the trialIf the trial is not following its approved protocol, or is not consistent with ethical research, contact the HREC’s ethics office. An HREC can suspend a trial or withdraw its approval.
  3. The institution running the trialIf the HREC’s response is not satisfactory, or you cannot find out which HREC approved the trial.
  4. Your state or territory’s health complaints bodyEach has formal powers to look into complaints about health services and registered practitioners; the site lists one for each state and territory.

The NHMRC can give advice to someone who does not want to approach the trial site, but it does not investigate research misconduct, which is the research institution’s job. A trial with significant safety concerns about a therapeutic good can be reported to the TGA, but side effects themselves go to the researcher, not the TGA.

Leaving is always open. A participant can stop at any time, may be asked to sign a withdrawal of consent form, and leaving does not affect their routine care or their relationship with their doctor.