Frequently Asked Questions

List of Abbreviations

  PsA Pseudomonas aeruginosa
  CF Cystic Fibrosis
  IV Intravenous
     

What is TORPEDO-CF?

What is PsA and what does it mean for me / my child?

PsA is a germ which is common in the environment. PsA lung infection can be treated if it is picked up early but it becomes impossible to eradicate once it has gained a foothold. In the long term PsA can cause lasting damage to the lungs.

What is TORPEDO-CF about?

TORPEDO-CF is a clinical trial which compares two ways of getting rid of PsA lung infection in people with CF. One group of participants will receive antibiotics given intravenously (by a tube into the vein) and the other group of participants will receive antibiotics given orally (by mouth). People in both groups (treatment arms) will also receive nebulised (inhaled as a mist directly into the lungs) antibiotics.

Why is TORPEDO-CF being conducted?

We know that both treatment types work well at getting rid of PsA but we don't know if one is better than the other.

Why don't we already know the best way to eradicate PSA?

Because no one has done a clinical trial (a fair test) to compare the two types of treatment yet.

Do all doctors agree or do some think they know the answer?

So far over 700 CF patients around the world have taken part in high quality trials of Pseudomonas eradication. However, a proper trial of intravenous versus oral eradication treatment has never been done so no-one knows the answer yet.

I thought doctors were supposed to know all the answers. How do you expect me to have any confidence in you if you say you don't know the best treatment options?

We know that many of the treatments we use in CF are effective. There are good trials of Dornase alfa, inhaled tobramycin and azithromycin for example and we can be confident that these drugs are safe and effective. In previous years, doctors would have been reluctant to tell you when they were not sure about the best treatment option. Now days we are happier to say when we don’t know and to design trials which will find the answer.

Who is funding the trial and why?

The Health Technology Assessment (HTA) programme is funding the trial. They are part of the UK National Institute for Health Research (NIHR) which is funded through the Department of Health with the aim of improving the health and wealth of the nation through research.

Why take part?

Don't we already have treatments for PsA? If both oral and IV antibiotics work, why does it matter?

We think both treatments work well however each treatment type has a risk of side effects and intravenous treatment sometimes requires a hospital stay so it is important to know if one treatment type is better than the other to ensure the benefits of choosing one particular treatment over another outweigh any potential drawbacks.

Will this trial benefit me?

In general, patients who take part in trials do better than those who do not. This is true even if trial patients get a dummy medication (“placebo”) although we don’t use a placebo in TORPEDO-CF! We do not know why trial patients do better. If you take part in TORPEDO-CF you will get one of two treatments – both of which we think are effective. The main benefit for you will be knowing you will be helping doctors make the right decisions when they see patients in future. 

How will this trial benefit people with CF now and in the future?

People with CF will benefit from better, safer treatment.

Have studies like this changed practice and led to improvements in care in the past?

There are plenty of examples of how clinical trials in CF have improved care. A previous trial called TOPIC showed that giving intravenous antibiotic tobramycin once a day minimised harmful effects on the kidneys. The once a day option has now replaced the usual 3 times a day dose in standard care. This allows patients to have a more convenient dosing option, with fewer side effects. However we could only prove this conclusively with the help of 244 CF patients who helped us find the answer by taking part in the trial. In contrast, if we accept new treatments uncritically we may waste time and money on ineffective or harmful treatments.

Can I take part?

Who is eligible for TORPEDO-CF?

Most CF patients over 4 weeks of age can take part, if a new growth of PsA has been detected in the last few days or weeks. There are very few patients who cannot take part – for example patients with chronic PsA infection or patients who have had antibiotic treatment for PsA recently. Your CF doctor can go through a full checklist with you. 

How can I find out if my centre is participating?

There is a map of participating centres here

You can also ask your CF doctor if your CF centre is participating. 

My CF team hasn't mentioned it to me, does that mean I'm not eligible?

Not necessarily – why not ask them?

What can I do if my centre is not participating?

Ask them why not. It is most important to have a new PsA infection treated promptly, so do not delay treatment and discuss the best option with your CF team. The TORPEDO-CF team are also very happy to talk to your CF centre (or to you) about whether the TORPEDO-CF trial is an option. If you or your CF doctor would like to discuss the trial further you can contact the TORPEDO-CF team here

Surely this is aimed at children. Why are you trying to recruit adults?

Our latest UK CF Registry data show that more than half of adult patients are free of chronic PsA. They also have a right to receive the safest and most effective treatment to eradicate PsA. There is evidence that eradication also works well in adults and in future it will be used more and more in adult clinics.

How can I find out more?

Ask you CF doctor or contact us here

What will happen if I decide to take part?

What is involved?

If you or your child take part, we will decide at random whether intravenous or oral treatment is given. Intravenous treatment is for 2 weeks and usually means an admission to hospital. Oral treatment lasts for 3 months. Everyone also gets a nebulised antibiotic called colistin for 3 months. After that, the trial team will see you at the end of the 3 months of treatment to take a sample, looking for Pseudomonas. The trial team will then see you every 3 months as a minimum, these study visits will usually be at the same time as your routine clinics. There are no extra blood tests.

How long will it take?

The first visit will take longer than your usual clinic visits because we have to ask you to provide written consent, then the CF team at your centre will do the randomisation and ensure the medications are dispensed. After that the important visits are at 3 months and 15 months (after the first visit)  – to take samples looking for Pseudomonas. These visits should not take any longer than your usual clinic.

Will it have a detrimental effect on my health if I take part?

No 

What is randomisation and why is it necessary?

We decide treatment at random. This is just like tossing a coin – although we use a computer which produces random numbers. If treatment was not decided at random then doctors might choose the treatment they thought might work best for the patients they felt might have the best chance of successful PsA eradication. If that happened, the results of the trial would be worse than useless. We know it is asking a lot for you to consent to a trial where there is a 50:50 chance of a hospital admission. However, it is the only way to find out if routine intravenous treatment is justified. If we don’t do the trial intravenous treatment may be used more and more frequently, just because people feel it “ought” to be “stronger”.

Will I have to stop any of my existing medication?

No

What if I get selected for orals? Everyone knows IV’s are better.

We think both options work well, we don’t know which one is better.

If I get randomised to IVs can I do them at home?

That depends on whether your CF centre can support home IV treatment and if think you can manage it at home.

If we’re selected for IVs what do we need to bring in with us? Do we have to stay in all the time or are we allowed out?

You should pack changes of clothes and toiletries as usual. However, many CF centres encourage patients to take some time off the ward during a 2 week admission. It is important you or your child are there for there antibiotics and physio sessions though!

If I get randomised to IVs and have to come into hospital for 2 weeks, what should I tell my employer?

You are entitled to sick leave in the usual way. 

If I do the study, will I still be able to go on holiday and get travel insurance?

The treatments we are comparing are both given routinely in the NHS and so taking part will not affect travel insurance. Holidays are not a problem unless they conflict with the 3 month and 15 month study visits.

If I do the study, will I be reimbursed for expenses like petrol, car parking, loss of earnings? Will I get paid?

We cannot reimburse loss of earnings or pay if you participate however where possible we have tried to ensure that study visits occur at the same time as normal clinic visits. Our grant for the study does not contain money for travel expenses but we would not wish this to be a barrier to taking part in the study which has been designed to match normal hospital practice as closely as possible. Please discuss this with your CF team if this is a particular issue for you.

What happens if I grow pseudomonas again, will I have to go through the randomisation process again to see how it will be treated? 

You can only take part in TORPEDO once. If Pseudomonas shows up again in samples from your lungs, then you and your CF doctor should discuss the best treatment option for you.

If I do the study, will it make me ineligible for other clinical trials that might come up?

That depends. Usually you cannot do two drug trials at the same time, in case the trials conflict with one another. However, TORPEDO treatment will be finished after 3 months and you will be eligible for other drug trials (if you want to take part) shortly after that. 

What will happen to the results of the study?

How are you going to judge which treatment works best?

The key outcome is to see whether or not PsA has gone after the end of treatment at 3 months and whether it stays away for a year; we will compare the two treatment groups to see if there is a significant difference.

When will you have the results?

We need 280 patients to take part in the study. Once we have collected data from all 280 patients their data will be analysed and the results will then be produced.

Why do you need 280 patients to take part?

If TORPEDO-CF recruits a smaller number of patients, the trial may not show a difference between the two treatments because the trial is too small. This is because with small numbers there is a lot of “background noise” due to random variation in PsA rates in the two arms. We have carefully calculated the number we need to get a clear result and this is 280. Unless we get a clear result, we will not change way doctors make their decisions. Doctors are very stubborn!

What happens if you don't reach the recruitment target?

We believe we will reach our target – otherwise the outcome will be inconclusive and the efforts made by those who have taken part already will be wasted.

How will the results of Torpedo-CF be shared?

We will tell CF teams what we have found by talking about the trial at medical conferences, we will publish the results in medical journals and we will also share the results with everyone who has taken part in the trial via their CF doctor.

Will you share the results with the wider CF community?

We also plan to communicate with the wider patient community through the mass media, the web and through social media.