Right here in Australia, we have had several clinical trials aiming to treat the root cause of HD, which is the production of the abnormal HD protein.
On this page we provide information and links to three trials (2 from Wave, and 1 from Roche).
If you want additional information about trials, you may wish to contact staff at your nearest HD clinic.
Wave Clinical Trials
Wave Life Sciences conducted two Phase 1b/2a clinical trials called PRECISION-HD1 and PRECISION-HD2. A third trial PRECISION-HD3, will be underway soon in Australia.
These trials aimed to specifically target and reduce the amount of mutant huntingtin protein produced, whilst leaving the healthy huntingtin protein untouched.
The treatment for the Wave trials was administered into the spinal canal via a lumbar puncture. The main goal of these trials was to examine the safety and tolerability of each drug. Researchers were also interested to understand how each drug affects the levels of huntingtin protein produced, and their effectiveness against the clinical symptoms of HD. A full examination of the treatment effects will only be possible in the next stage of study, a Phase III trial, which has more participants so that it can examine the full benefits and side effects for patients.
Both trials involved the same procedures, however participants in PRECISION-HD1 were administered a drug called WVE-120101, whilst those participating in PRECISION-HD2 received the drug WVE-120102.
There were five clinical trial sites in Australia investigating this drug. These sites are located in Melbourne, Sydney, Brisbane and Perth.
Roche Clinical Trials
Sponsored by Roche, the GENERATION HD1 study administered eligible HD patients with a drug that has been shown to reduce the production of the huntingtin protein. This protein is responsible for HD symptoms.
The purpose of this phase III trial was to study whether treatment with this drug slows the progression of symptoms.
This study also aimed to:
- better understand whether any benefits observed during the trial are due to the drug, and not to other factors, and
- determine how often the drug should be administered to provide any observed patient benefits.
All participants attended study visits every 2 months for either drug or placebo injection via a lumbar puncture. Participants were randomised to one of three groups in which they receive either:
- study drug once every 2 months,
- study drug once every 4 months, or
- study drug once every 4 months and placebo every 2 months.
The Roche trial enrolled more than 660 people with HD across 80-90 clinics in 15 countries around the world, including Australia. In Australia we had three sites involved in the trial, which are located in Melbourne and Sydney.
For more information about Wave Life Sciences, you can access their website here.
For more information about Roche, you can access their website here.