Latest News from the Clinical Trial
Our patients are very important to us and without them our clinical research could not happen. In this section of our website we would like to keep patients as up to date as possible with news from the clinical trial.
We thank all patients for their ongoing commitment and patience and for giving up their time to take part in our studies.
Update on the trial from Professor Alton - March 2013^Top
I am delighted to say that the Wave 1 trial is proceeding well.
We now have approximately 70 CF subjects who have received at least one dose of gene therapy, whilst some are up to the tenth of their allocated twelve monthly doses. The trial has been, as planned, reviewed twice so far by the Safety Committee who had no concerns about progress.
We still have room for anyone wishing to participate; please contact either Clare Saunders in London or Maria Dewar in Edinburgh for details. Much as we would like to know about how people are doing on the trial, no analysis of the data is allowed until we reach the end of the study. At present the last patient's data should become available to us around June next year.
Patient Identification Centres - March 2013 ^Top
The Multi-dose gene therapy trial is proceeding well. Some patients have already completed 10 out of the proposed 12 monthly doses. Read more here.
Multi-dose clinical trial funding press release - March 2012 ^Top
University of Edinburgh & NHS Lothian Clinical Research Facility (CRF) Newsletter - June 2011 ^Top
The Edinburgh & NHS Lothian Clinical Research Facility Newsletter for June 2011 contains information on the new gene therapy facilities at the University of Edinburgh & NHS Lothian CRF.
Message to Run-in Volunteers and their Families - March 2011^Top
You may already be aware of an announcement made by the CF Trust on their website (http://www.cftrust.org.uk/) regarding our Gene Therapy programme. As a volunteer or parent of a child who has helped us with the Run-in study, we wish to provide you with some further information and expand on this.
The CF Trust has been fully committed to supporting the UK CF Gene Therapy Consortium for the last 10 years in its mission to develop clinically beneficial gene therapy for cystic fibrosis. The CF Trust has generously funded the preclinical development which allowed us to identify the best, currently-available gene therapy product to take forward. Their funding has also allowed us to conduct a number of clinical studies, which we believe have been genuinely world leading, not only for gene therapy, but also for other future CF research.
As you know, the Run-In study was conceived to allow us to design the best multi-dose trial, choosing optimal outcome measures and most suitable patients. We will very shortly be contacting those of you still waiting to hear whether, based on your Run-In data, you are in the optimal group to go forward. In addition to this, we have also conducted a single-dose safety trial (Pilot study) of this product, which is nearing completion, and which has allowed us to choose the dose for the multi-dose trial.
Based on the above we had been anticipating contacting everyone regarding a September start date for the multi-dose trial, for which everything is now in place. However, related to the economic downturn, the CF Trust has experienced a severe reduction in income which we have recently become aware of. This has resulted in their recent decision to take stock of their current financial situation and to ask us to revise our programme. Both the CF Trust and the Consortium intend the multi-dose trial to continue, and we are working closely together on this, but as you will realise, this inevitably will lead to some delay. The focus of the trial will remain as before, looking to produce improvements in lung disease. If successful, the Wave 1 product would then move rapidly into further trials as previously planned.
We will be in a much clearer position within the next few weeks and will keep closely in touch with you.
We wish to take this opportunity to thank you very much for your participation in the gene therapy research programme; without volunteers such as you and your children, we would have been completely unable to make any of this progress.
Status of Run-in Patients - 2010 ^Top
The upcoming multi-dose clinical trial will determine if gene therapy can improve CF lung disease. This is a very important proof-of-principle study and it is, therefore, important to select the most appropriate patients to answer this question. The multi-dose trial will have the best chance of success if we select patients that (a) have healthy enough lungs to ensure that the gene is delivered to most of the airways, but (b) also have sufficient lung disease to allow us to measure an improvement after gene therapy.
Based on the results of the Run-in study we are able to assign patients to one of three groups below. You will have been contacted by the consortium to let you know which group you are in.
Group 1: patients asked to continue on the Run-in with a view to participate in the Multi-dose trial
These patients have healthy enough lungs to allow efficient delivery of the gene, but also have sufficient lung disease to allow us to assess improvement after gene delivery.
These patients have largely been seen for a visit five and will be contacted in the furture regarding further Run-in visits.
Group 2: Patients not suitable for progression into the Multi-dose trial
There are two reasons why patients may not be suitable for the multi-dose trial:
Patients who are extremely well and have very healthy lungs are not suitable because although we would be able to deliver the gene to the airways of these patients efficiently, we would not be able to measure an improvement in lung disease after gene therapy, because the lungs are already very healthy.
Patients who have quite advanced lung disease are not suitable because the comparatively advanced lung disease in these patients would make it very difficult to deliver the gene and, therefore, reduce the chance of seeing an improvement in lung disease severity. In addition, we are concerned that the treatment may be less well tolerated in patients with severe lung disease, because gene delivery may cause a transient drop in lung function.
These patients have been invited to attend a visit five to exit the Run-in study.
Group 3: Patients for whom we cannot make a decision yet, but hope to do so shortly
There are two reasons why we may not be able to make a decision yet:
Patients who are quite well and have only limited lung disease. Although we would be able to deliver the gene to the airways of these patients, we may not be able to measure an improvement in lung disease after gene therapy, because the lungs are fairly healthy. Patients may have abnormal results in some, but not all of the tests we plan to include in the multi-dose trial. Further detailed analysis of our data will help us decide if patients in this group may be suitable for taking part in the trial.
Patients whose average FEV1 has been lower than 50% during the Run-in study. We anticipate that we would be able to deliver the gene to the airways of these patients, and would also be able to measure an improvement after gene therapy. However, we are concerned that the treatment may be less well tolerated because gene delivery may cause a transient drop in lung function. Over the next few months we will be looking at ways to prevent the transient decrease in lung function after gene therapy. If this is successful, patients in this group may be suitable for inclusion in the multi-dose trial.
Information for Patients on Our Clinical Trial
Consortium Members' Internal Website
Medical Futures Innovation Award 2011