A clinical trial currently recruiting in Australia is SELECT-HD. SELECT-HD is a Phase 1b/2a global, multicenter, randomised trial. The aim of the trial is to study the drug (WVE-003). The study includes up to 36 adults worldwide. Patients with early-manifest HD who carry a specific polymorphism (SNP3) can take part.

SNPs are normal variations in DNA. A blood test determines if you have SNP3. The presence of SNP3 enables WVE-003 to target the toxic huntingtin protein. While this targeting occurs, the drug leaves the healthy huntingtin protein relatively intact. Participants receive the study drug by means of a lumbar puncture. A lumbar puncture is an injection into the spinal canal.

For every participant that receives the placebo, two participants will receive the active drug. Neither the doctor nor the participant will know whether they have received the active drug or the placebo.

The study will measure:

  • safety
  • tolerability
  • manner in which the medicine moves through the body
  • manner in which the medicine impacts the body

The study uses an adaptive design. This design means that there is a committee that reviews the data throughout the trial. The committee decides what the next dose will be and how often the drug should be administered.

Keep in mind that the trial only includes 36 people from across the world. Only a few people from Australia will be eligible. To be considered for this trial you must live near one of the Australian sites.

Australian sites taking part in this trial include:

  • The Perron Institute (WA)
  • Westmead (NSW)
  • Royal Melbourne Hospital (VIC)
  • Monash Health (VIC)

Update from WAVE Life Sciences: 3 August 2023