Clinical Trials

It is an exciting time in Huntington’s research with studies being conducted around the world to try and find ways to slow down and even reverse the impacts of the disease.

Clinical trials are a vital part of these efforts, allowing researchers the opportunity to test new drugs and treatments with volunteers in a controlled environment.

These staged trials ensure that any new medical interventions work as intended, are safe and have no harmful side effects.

It is only after the successful completion of clinical trials that companies can seek approval to market and sell a new drug or treatment.

On this page we will post the latest information available on the progress of major trials and opportunities for people who have HD to get involved.

Q and A: Find out more about clinical trials and what’s involved for participants.

You can also search for clinical trials in Australia and worldwide.


GENERATION HD1 clinical trial


The largest and most important HD clinical trial taking place right now is Roche’s GENERATION HD1.

This phase III trial involves almost 800 people in 18 countries with Huntington’s who are showing the early symptoms of the disease.

A phase III trial is the last stage before a drug can be submitted to regulatory authorities for approval to be sold and distributed.

In Australia the trial sites are Westmead Hospital in Sydney and the Royal Melbourne Hospital in Melbourne.

The DNA-based drug being tested is called Tominersen and aims to reduce the production of the mutant Huntingtin protein which causes the brain damage that people with HD experience.

Recruitment for the trial was completed in April 2020. Participants in the 25 month trial are receiving the drug either once every two months or once every four months via a spinal injection. Half the participants receive a placebo (dummy drug).

There is much cause for optimism around this drug due to the results of an earlier phase I/II trial involving 46 volunteers.

From that trial it was reported that there were reductions of 40 to 60 per cent in the amount of the mutant protein in the cerebrospinal fluid of participants and the drug was safe and well-tolerated.

However, it remains unclear whether reductions of the protein in the cerebrospinal fluid, which circulates along the spine and bathes the brain, means the progression of the disease has been slowed.

More work is being done to assess the impact of the drug on the cognitive, functional and motor skills of the participants.

Even though there are some disruptions due to COVID 19, findings are expected to be released sometime in 2022.




Two other exciting clinical trials that are underway right now are being conducted by biomedical company Wave Life Sciences.

The company is testing two DNA-based drugs in these phase 1b/2a trials – WVE-120102 and WVE-120101.

These drugs are designed to lower the mutant level of huntingtin protein in people who are showing the early symptoms of the disease.

The difference with the Roche trial is these drugs are designed to only reduce the mutant protein not the healthy huntingtin protein which everybody has in their body.

Due to the specific way the drugs are targeted within the DNA, only 70% of people with HD can be treated with them.

In Australia the trial sites are Westmead Hospital in Sydney, Royal Brisbane and Women’s Hospital, Royal Melbourne Hospital and the Perron Institute in Perth.

Three quarters of the participants will receive one of the drugs and the other one quarter will receive a placebo (dummy treatment).

Wave released some preliminary results from the trial of the WVE-120102 drug in December 2019.

Those results showed the drug was generally safe and well tolerated and reductions of up to 12.4% in the mutant huntingtin protein were found in the cerebrospinal fluid of volunteers.

Compared to the Roche result, that was seen as disappointing news by investors but researchers working in the HD space welcomed the result saying it was a cause for optimism and the doses of the drug tested were far lower than in the Roche trial.

Higher doses of the drug are now being tested and it is hoped results from the trial will be available late in 2020.