Patient Information

PATIENT INFORMATION

The following Patient information sheets are available from your local clinic if you would like more information about the study:

● Children ages 2 to 6 years

● Children aged 7 to 11 years

● Children ages 12 to 15 years

● Adults from 16 years

● Legal Guardian

If you would like to take part in the study, the possible benefits are:

It might mean that you will no longer need to take the Eculizumab treatment and face any potential risks or side effects associated with treatment.

Patients are about a thousand times more likely to develop a serious, potentially life threatening infection with meningococcus, a bug that causes meningitis or sepsis.

Vaccination, and even antibiotics, do not give complete protection from this.

Other serious problems have also been reported in patients taking Eculizumab but we cannot be certain that Eculizumab was the cause of these problems.

Being in the study will mean that you will no longer need bi-weekly infusions and will not have to continue taking additional antibiotics to prevent infection.

However, it is possible that you could need to restart and continue Eculizumab if a relapse was to occur.

The possible risks of taking part in the study:

The withdrawal of Eculizumab treatment could lead to a relapse of atypical HUS and relapse associated complications.

When a relapse is diagnosed, your Eculizumab treatment will be restarted within 24 hours of presentation.

It is essential that you present to a hospital with your patient card as soon as you begin to feel unwell or the home urine test shows an increase in the level of blood.

This is to ensure that the Eculizumab is re-started as soon as possible to reduce the likelihood of kidney damage and associated complications.

If your treatment is re-started, you will receive your first infusion in the hospital.

You will also have the antibiotics restarted to protect you from infection.

If you need to be put back on to Eculizumab treatment you can decide to receive your infusions in hospital or at home.

This can be arranged with your clinical team if you relapse while in the study.

If a relapse occurs this could lead to a drop in kidney function or other problems related to the disease which can affect organs such as the pancreas or nervous system.

The evidence that is available at the moment suggests that if Eculizumab is reintroduced quickly these problems can be avoided.

This is not proven and this study will test whether this is true.

Overseas Travel:

If, during the trial, you decide to travel outside of the country, we ask that you only travel to countries where Eculizumab is available so treatment can be re-started immediately as required.

You MUST first inform your hospital to check whether Eculizumab is available in the country that you plan to travel to and inform them of the dates that you will be outside of the country.

While the cost of the Eculizumab will be covered by NHS England you must ensure that you have appropriate travel insurance and inform your insurance company that you are taking part in the trial.

This is to ensure that all other treatment costs and hospital stays abroad are covered by your own insurance should you relapse.

We have developed a travel guide for you should you decide to travel outside of the country while you are participating in the withdrawal study.

Inclusion and Exclusion Criteria: To minimise the risks of taking part in the study, we will only include you in the study if you:

● have been on Eculizumab treatment for at least 6 months;

● are in remission;

● have a stable kidney function;

● are willing to attend for safety monitoring assessments;

● are willing to travel only to countries that can supply Eculizumab (to be confirmed with co-ordinating centre prior to travel);

● are able to perform and record self-monitoring urinalysis;

● sexually active females of child bearing age must have a negative pregnancy test at screening and be using an effective contraception for the duration of the study (please ask your doctor/nurse) OR be post-menopausal or have undergone surgical sterilisation.

You will not be able to take part if you:

● have lost a previous transplant kidney to recurrent aHUS;

● are currently or are planning pregnancy;

● are unable to comply with safety monitoring assessments;

● are currently participating in another clinical trial (not including participation in aHUS registries)

Please contact your local clinical team if you would like to discuss the study in more detail.