Clinical trial update – 19th October 2018

In light of very recent events, we would like to provide some important information about upcoming clinical trials in Alpha-1 in the UK.

Currently, there are three trials in AATD planned to commence by pharmaceutical/biotech companies (Mereo Biopharma, Arrowhead Pharmaceuticals and CSL Behring). These trials will be undertaken in several different centres with Alpha-1 expertise across the UK.

It has come to our attention that Dr Turner from UH Birmingham implied in a recent email to an AATD patient that these trials will be conducted in Birmingham only and that patients should get in touch with her team now if they want to be considered for participation in any of these trials. This is misleading and FACTUALLY INCORRECT. Patients have no advantage for being chosen to participate in a clinical trial whatsoever if they provide their contact details to Birmingham or to any of the prospective trial centres at this point; in fact, there are several reasons why it may be counter-productive to do so.

The general process for patient recruitment is always the same and as follows: Once a trial has been formally approved, the company conducting the trial will publicly announce more detailed information about the trial, the trial centres and contact information at each trial centre. Once this information is released, patients interested in participating in the trial can then contact the trial centre of their choice (or several different centres if they wish). For logistical reasons, most patients tend to choose a trial centre that is geographically close to where they live, as most clinical trials require regular visits to the trial centre – however, the choice of centre lies entirely with the patient.

A few important points to keep in mind when you think about which trial centre to choose:

• Every clinical trial needs to follow a “trial protocol” which sets out how the trial is conducted, how often patients need to visit the centre, which tests are being performed etc. Each trial centre needs to strictly comply with this protocol. Patients will therefore have the exact same experience throughout the clinical trial, irrespective of the trial centre they choose.
• Every centre where the trial is conducted will have undergone a detailed screening process to ensure that the clinical staff have relevant and sufficient expertise and competence in AATD and that the technical, administrative and ethical requirements are met by the centre.
• A clinical study is less likely to be successful if the majority of patients attend a single trial centre. The more evenly distributed the patient numbers are across different centres, the better. There are several reasons why it is advantageous for the trial if patients choose a trial site geographically convenient for them: if patients have to travel a long way to the trial centre, they are more likely to miss study visits or drop out of the trial.
• The better the patient community advertises AATD trials, the more likely it is that the trials are successful, because we reach a wide range of patients, rather than only those who ‘register’ with a specific centre, as Dr Turner misleadingly suggested in her email to the Alpha-1 patient. All trial centres will offer the opportunity to discuss the trial in detail with each patient, once patient recruitment is allowed to start. 
• The AATD patient community has campaigned for many years to increase disease awareness and clinical expertise across the UK. As a result of this campaign, we now have several very experienced AATD centres across England, with two additional satellite centres currently being set up in the South and the North of England. It is crucially important for patients to utilise all centres, in order to help them gain more expertise and to ensure that all centres remain sustainable.
• Please note that patients who participate in clinical trials do not need to move their routine specialist NHS care to this trial site (even if this is suggested by the trial centre). It is entirely up to the patient to choose which doctor’s care they wish to be under.

We are in regular and frequent communication with all companies who are planning to conduct trials in AATD in the UK. As soon as they are in a position to publish specific information on these trials, including details about the trial centres and contact details, this will be available on our website. For current information on upcoming trials:
• Read the update in our recent newsletter (pages 2 and 3,…/…/51/A1UK_Newsletter2018.pdf),
• Watch the presentations that Mereo Biopharma, Arrowhead Pharmaceuticals and CSL Behring gave on their respective clinical trial programme at our recent annual patient group meeting – fast-forward to minute 9 on video 2 This video includes a list of all prospective UK trial centres for the upcoming Mereo Biopharma trial – fast-forward to minute 23 of video 2.
• As soon as we have any update on any trials relevant in the UK, we will post it in this group.

Karen O’Hara, on behalf on the Alpha-1 UK Support Group, supporting all Alphas in the UK