FAQs

Frequently Asked Questions

 

What is a placebo?

A placebo is a non-active ‘dummy’ treatment, which looks like the real treatment. It is used in clinical trials as a comparator with the active treatment being tested to determine statistically if the active treatment actually works. In a well designed clinical trial both the participants and the investigators are ‘blind’ to the allocation of active treatment and placebo until the trial is finished and the ‘code’ is broken. This rules out any ‘placebo effect’ and bias and is the only way that new treatments are scientifically proven and accepted by regulatory authorities.

Will I be informed if I was taking the study medication (insulin) or the placebo?

You will be informed by the researchers at the end of the trial, when the the code has been broken.

What is the aim of the trial?

The primary aim of the trial is to determine if type 1 diabetes can be prevented. Participants need to be at high risk of developing type 1 diabetes. This is determined by the free screening blood test for antibodies.

Who is the sponsor of the trial?

Melbourne Health is the trial sponsor.

Who is funding the trial?

The trial is being funded by the National health and Medical Research Council of Australia (NHMRC) and the Juvenile Diabetes Research Foundation (JDRF).

What is the treatment being used in the trial?

The treatment is insulin, administered via a nasal spray.

How does nasal insulin work?

Nasal insulin works like a vaccine to stimulate a protective type of immune response to insulin. This has been shown to suppress the immune attack on the pancreatic beta cells which causes type 1 diabetes.

Has nasal insulin been tested before?

Yes, nasal insulin has been tested in an earlier clinical trial, in which it was shown to be safe and to induce immune effects.

Does the nasal insulin cause hypoglycaemia?

No, insulin inhaled through the nose does not enter the blood stream so it does not cause hypoglycaemia.

Was the treatment initially tested on animals?

Yes. It was shown to be safe and to stimulate a protective immune response to insulin which suppressed the development of diabetes, in mice that are prone to type 1 diabetes. It is a legal requirement that every treatment is tested in animals before being tested in a clinical trial in humans. Strict guidelines ensure that animals involved in scientific testing are treated humanely.

How many people will participate in the trial?

Almost 10,000 people across Australia and New Zealand who have a relative with type 1 diabetes participated by having the initial screening blood test. However, only approximately 2% (1 in 50) were eligible to participate in the trial because they have the antibodies in their blood indicating a high risk of developing type 1 diabetes.

Who can participate?

People aged between 4-30 years who have a first-degree (mother, father, brother, sister, son, daughter) or second-degree (aunt, uncle, grandmother, grandfather, half-brother, half-sister) relative with type 1 diabetes are participating in the trial.

My child is younger than 4. Can they still be involved in this trial?

No. The lower age limit for participation in this trial is 4.

How often will I need to take the nasal spray? How often will I need to visit the hospital?

If eligible, you will self-administer a nasal spray each morning for one week and then once a week for 12 months. You will return for a check-up every 3 months during the treatment year, then every 6 months for a further 4 years.

Why do I have to have a blood test?

Participants must be at risk of developing type 1 diabetes. The blood test measures antibodies that indicate the risk of getting type 1 diabetes.

My blood test result is negative. Does this mean I will never get diabetes?

No. Rarely, people who don’t show antibodies in the blood test can develop diabetes, or they may become positive for antibodies later. Overall, however, being negative for antibodies means your risk is low.

How long is the trial?

The treatment phase is 1 year. After this, participants are then followed up for a further 4 years.

Will I be paid to participate?

No. You will not be paid for your participation in the trial. However, you will be reimbursed for travel expenses (see below).

Will it cost me anything?

No. There is no charge to you for consultations, examinations and blood tests related to the trial.

Will my travel expenses be reimbursed?

Reasonable travel expenses will be reimbursed (e.g. taxi fare/reimbursement for using own car).

What if I don’t live near a trial site – can I still participate?

Yes. The screening blood test can be done at a pathology centre close to you or your local GP. If you are required to visit a distant trial site, you can be reimbursed for any travel costs you incur.

What are the possible benefits of the trial to me?

The trial is designed to determine if the onset of type 1 diabetes can be delayed or prevented by a nasal insulin vaccine. Therefore, if the trial was successful it would benefit people at risk for type 1 diabetes by forestalling or preventing the development of type 1 diabetes in them.

If you have the screening blood test for the trial there is a very high (98%) chance that you won’t test positive for antibodies. Therefore, this would be reassuring for you.

There is only a small (2% or 1 in 50) chance of having antibodies in the blood indicating a high risk (40-50% over 5 years) of developing type 1 diabetes. In this case, you would be offered participation in the trial. In addition, you would be offered advice to minimise symptoms and optimise treatment should you ever develop diabetes. In our experience, people who know they are at risk have fewer difficulties if they develop diabetes.

What are the possible risks of the trial to me?

If you have the screening blood test and are one of the minority with a positive test this can cause you anxiety. In our experience this anxiety is temporary. As with all medications, there is a small possibility that you may have side effects, but with the nasal spray these are usually very minor and transient. As with any blood test, there is a possibility of discomfort and local bruising, but these are usually minor.

Will I be compensated if something goes wrong?

In the unlikely event that you suffer an injury as a result of participation in this research trial, hospital care and treatment will be provided by the public health system at no extra cost to you.

Can I drop out at any time?

Yes. If you wish to stop participating in the trial you may do so at any time. You are under no obligation to finish the trial, but we hope you will. If you decide not to continue, your decision will not affect your future medical care in any way.

When you send me information, does that mean I am participating in the trial?

No. In the screening phase we first determine if you are eligible to participate in the trial.

ONLY about 2% or 1 in 50 people who have the blood test will have levels of antibodies that will make them eligible for the trial – this is why it is critical to encourage as many family members as possible to have the free blood test.

Can my mother, father, partner or friend take part?

If you mother, father, partner or friend is aged between 4 and 30 and has a relative with type 1 diabetes then they can participate in the trial.

Why can’t I take part?

The trial has strict inclusion and exclusion criteria that you need to meet in order to participate. In order to participate, you must have type 1 diabetes in the family but not have type 1 diabetes yourself (unless it is your child you are enquiring about). Your relative with type 1 diabetes needs to have been diagnosed before the age of 40. If your relative with type 1 diabetes is your grandmother, grandfather, aunt, uncle, niece, nephew, half-sister or half-brother, i.e. a second degree relative, you need to be aged 4-20 years to participate.

Will participation in this trial affect my ability to get life/health insurance?

Insurance companies are primarily interested in genetic risk of disease. In the trial we do not measure genes for increased diabetes risk. However, the trial is clearly for individuals who may be at increased risk because they have a relative with type 1 diabetes. Furthermore, the presence of antibodies in the blood to components of the insulin-producing cells indicates increased risk for type 1 diabetes. As most participants in the trial are children it is unlikely they would be applying for insurance during the trial. If they did apply, it would be for rated life insurance and not income protection insurance.

If the trial worked, the risk of diabetes would be reduced. If diabetes developed after the trial, the usual conditions on insurance would apply as for someone with a pre-existing condition.

A person has a duty under both the common law and statute (in Australia, the Insurance Contracts Act 1984) to disclose to an insurer every matter that he/she knows could be relevant to the decision of the insurer whether to accept the risk.

If an insurer asks a direct question about the results of tests that person may have had, then the person has an obligation to answer the question truthfully. Failure to answer truthfully could result in, amongst other things, the policy being voided by the insurer.

When will I find out about the outcome of the trial?

You will be informed of the outcome when the code is broken and the results are analysed and ready for publication in the scientific medical literature.

Will I be informed of any interim outcome of the trial?

There will be no interim outcome unless the trial is concluded due to a pre-planned interim analysis. In this case, you will be informed of the outcome when the code is broken and the results are analysed and ready for publication in the scientific medical literature.

Will this trial contribute to finding a cure for type 1 diabetes?

Yes, because there will be no cure without prevention. A cure for type 1 diabetes will involve replacing or regenerating lost insulin-producing beta cells. Insulin itself and other components of the beta cells are targets of the destructive immune response. The immune system has a memory so as soon as insulin-producing cells are replaced or regenerated they will again be attacked by the same autoimmune process that destroyed them in the first place. Therefore, approaches to preventing type 1 diabetes will also be critical for attempts to cure type 1 diabetes, by replacing or regenerating insulin-producing cells.

If you have more questions, please call 1300 138 712.