Roche and Ionis halt GENERATION – HD1 trial early
The Huntington’s Disease Association received the news last night (22 March 2021) that Roche and Ionis are halting their phase III Tominersen huntingtin-lowering trial, GENERATION – HD1, early. This was not due to new safety concerns.
What was the GENERATION HD1 study?
The GENERATION HD1 study evaluates the efficacy and safety of the investigational antisense medicine, Tominersen (previously IONIS-HTTRx or RG604), in people showing Huntington’s disease symptoms. The aim of this study was to see if Tominersen was effective in lowering the huntingtin protein and improved symptoms in people with Huntington’s disease.
Why has it been halted?
Roche have stated the trial has been halted on the advice of the Independent Data Monitoring Committee. This is a group of completely independent experts whose sole job is to monitor and decide whether the trial should continue. They generally monitor two things – is it safe and does it work?
What information do we have?
At the moment, the information we have is limited. We know the decision to pause the trial was not because of any additional safety concerns. Roche have stated they had halted the trial because of the “investigational therapy’s potential benefit/risk profile”.
We don’t really know at this point what that means but it could be that the drug either makes symptoms worse or does not improve them. It is really important to understand that no one has the answers to those questions now. When answers become available, we will inform our community as quickly as we can.
What happens now?
Moving forward, lessons learnt from this trial show that the huntingtin protein can be lowered in people with Huntington’s disease – which means future trials can start from a much more advanced point. There are several other trials that are looking at different methods of doing this, so whilst this is really disappointing news there is still much to be hopeful about.
If you or a family member have been involved in the study and you have further questions we recommend that you contact your Site Investigator. If you would like support and someone to talk to our Specialist Huntington's Disease Advisers are here for you.
HD Buzz have written an article about this announcement, you can read more here.
The Huntington’s Disease Association received the news last night (22 March 2021) that Roche and Ionis are halting their phase III Tominersen huntingtin-lowering trial, GENERATION – HD1, early. This was not due to new safety concerns.
What was the GENERATION HD1 study?
The GENERATION HD1 study evaluates the efficacy and safety of the investigational antisense medicine, Tominersen (previously IONIS-HTTRx or RG604), in people showing Huntington’s disease symptoms. The aim of this study was to see if Tominersen was effective in lowering the huntingtin protein and improved symptoms in people with Huntington’s disease.
Why has it been halted?
Roche have stated the trial has been halted on the advice of the Independent Data Monitoring Committee. This is a group of completely independent experts whose sole job is to monitor and decide whether the trial should continue. They generally monitor two things – is it safe and does it work?
What information do we have?
At the moment, the information we have is limited. We know the decision to pause the trial was not because of any additional safety concerns. Roche have stated they had halted the trial because of the “investigational therapy’s potential benefit/risk profile”.
We don’t really know at this point what that means but it could be that the drug either makes symptoms worse or does not improve them. It is really important to understand that no one has the answers to those questions now. When answers become available, we will inform our community as quickly as we can.
What happens now?
Moving forward, lessons learnt from this trial show that the huntingtin protein can be lowered in people with Huntington’s disease – which means future trials can start from a much more advanced point. There are several other trials that are looking at different methods of doing this, so whilst this is really disappointing news there is still much to be hopeful about.
If you or a family member have been involved in the study and you have further questions we recommend that you contact your Site Investigator. If you would like support and someone to talk to our Specialist Huntington's Disease Advisers are here for you.
HD Buzz have written an article about this announcement, you can read more here.