HDYO-NZ members attended an update of the clinical trial which is testing the safety and effectiveness of drug RG6042 (Genentech/Roche) for patients with Huntington’s disease. Associate Professor Richard Roxburgh provided a summary of what the trial would look like for New Zealand, and what it means to be involved in this ground-breaking trial. To learn more about this international trial, please check out the following link https://en.hdbuzz.net/265
In a public meeting at Auckland Hospital, Associate Prof Roxburgh summarised the Kiwi-arm of the international study:
· 21 HD patients showing symptoms in New Zealand will be selected as candidates to be part of an international trial of 660 patients. 9 candidates will come from Auckland, 3 from Wellington and 9 from Christchurch.
· The trial will involve clinic visits every 8 weeks for 25 months, with lumbar punctures (spinal tap) at each visit (13 lumber punctures).
· Out of the 660 patients in the study, 220 will receive placebo (an injection of saline) every 8 weeks, 220 will receive RG6042 every 8 weeks, and 220 visit the clinic every 8 weeks but will receive RG6042 every 16 weeks, alternated with placebo at the other visits.
· In addition to the lumber punctures, other measurements including neurological tests, MRI scans, daily activity monitoring (using smartwatch technology) will be carried out.
· To be eligible for the trial, there are several criteria which need to be met based on neurological assessment. (1) Patients need to be between the ages of 25 – 65 at the start of the study (2) have a stable medical, psychiatric, and neurological status for at least 12 weeks prior to the trial screening and enrolment, and (3) have a CAP score greater than 400.
· What is a CAP score? CAP stands for CAG Age Product, a very basic mathematical formula that can be used in part to estimate a person’s age of HD onset, based on how long they have lived with their mutation. CAP score = (length of CAG repeat – 33.66) x age at start of the study. The purpose of the CAP score limit is to make it more likely that a difference between the active drug and placebo is measurable.
· The participants in this study ARE UNLIKELY TO be eligible for other clinical trials that emerge.
· This trial is a huge commitment! In the words of Associate Prof Roxburgh, the chosen participants will be the “All Blacks” of HD patients because they will be the best possible candidates for the trial and just like the All Blacks, the time investment is considerable.
To learn more about the trial and further eligibility criteria in New Zealand, please email Christina Buchanan, study coordinator CBuchanan@adhb.govt.nz