Skip Content
 
Page Banner Image

RG6042 Huntingtin-Lowering Trial Information

For more information on this trial click here.

Roche announces that the first patients have been enrolled in the HD Natural History and Phase III GENERATION HD1 clinical studies

January 28, 2019

Today, Roche Pharmaceuticals sent a letter to the global HD community to provide an update on the progress of their HD clinical trials. You can read the full letter from Roche here.

We have also included the FAQ from Roche below.

  • HD Natural History Study
    • This observational study for early manifest HD will run in the USA, Canada, Germany and the UK; planned study sites were announced in late 2018.
    • Initial sites have opened and the first patients have enrolled; the Roche team continues to work to open recruitment at all study sites as quickly as possible.
    • In Canada the HD Natural History Study will take place in:
      • Vancouver, British Columbia at the University of British Columbia Centre for Huntington disease
      • Toronto, Ontario at the Centre for Movement Disorders
  • Phase III GENERATION HD1 Study
    • Roche has received health authority approvals in the USA and Canada to start this pivotal study for manifest HD.
    • Planned study sites for those countries were announced at the end of 2018 and the first patient has now enrolled.
    • This study will run in approximately 15 countries; Roche is diligently working to set up study infrastructure and receive approvals in the remaining countries.
    • In Canada, the expected GENERATION HD1 study sites are:
      • Vancouver, British Columbia at the University of British Columbia Centre for Huntington disease
      • Edmonton, Alberta at the University of Alberta
      • Toronto, Ontario at the Centre for Movement Disorders
      • Ottawa, Ontario at the Ottawa Hospital
      • Montreal, Quebec at the Centre Hospitalier de l’Université de Montréal
      • Halifax, Nova Scotia at True North Clinical Research

Up-to-date information about these trials as well as additional HD clinical trials can be found on our website at www.huntingtonsociety.ca/hd-clinical-trials.

We applaud the volunteers who have stepped up and continue to step up to advance research. Their bravery pushes us closer to meaningful treatments. We have many reasons to be hopeful.  This update from Roche is another positive step forward.

For our family members, if you have specific questions about participation in clinical trials we recommend that you contact your local neurologist, Movement Disorders or Huntington Disease Clinic.  We will continue to stay in touch and keep you updated.

Robin Markowitz

CEO, Huntington Society of Canada

Frequently asked questions and answers from Roche

What is the HD Natural History study?

This 15-month observational study aims to further understand the role of mutant huntingtin protein in disease progression, including how levels of mHTT change over time in the absence of any drug treatment. There is no drug treatment in this study. This study will include up to 100 participants with early manifest (Stage I and II) HD. For all patients who complete the HD Natural History study, an open-label extension study with the option of receiving RG6042 (no placebo control) is planned, pending eligibility, approval by Authorities and Ethics Committees/Institutional Review Boards and if data support the continued development of RG6042.

The HD Natural History Study will run at up to 17 sites in Canada, Germany, the United Kingdom and the United States. For more information about the study/trial sites in Canada visit www.huntingtonsociety.ca/hd-clinical-trials or contact the local Roche Medical Information team: Canada: 1-888-662-6728

What is the Phase III GENERATION HD1 study?

The GENERATION HD1 study will evaluate the efficacy and safety of RG6042 treatment for manifest HD. The study will run over a period of 25 months (approx. two years). GENERATION HD1 is designed to determine whether RG6042 is safe and effective, and therefore includes a comparison to placebo. Participants will be randomised to one of three treatment study arms: RG6042 monthly, RG6042 once every two months (bi-monthly) or placebo monthly. This means for every two participants randomised to RG6042, one will receive placebo. The study is “double-blinded,” meaning neither the participant nor his/her investigator or site staff will know which study arm the participant is assigned.

For all patients who complete the GENERATION HD1 study, an open-label extension study with the option of receiving RG6042 (no placebo control) is planned, pending eligibility, approval by Authorities and Ethics Committees/Institutional Review Boards and if data support the continued development of RG6042.

The GENERATION HD1 study will enroll up to 660 patients with manifest HD at 80-90 sites in approximately 15 countries around the world. Planned sites have been announced for Canada and the United States of America.

For more information about the study/trial sites visit www.huntingtonsociety.ca/hd-clinical-trials or contact our Clinical Trial Information Support Line 1-888-662-6728. Information about additional countries/sites involved in the study will be announced, as those details are finalised.

How are the clinical study sites selected?

A variety of factors influence site selection, including assessments on experience with HD studies, clinic infrastructure capacity to run the study as well as usual site activities, ability to operationalise the study as quickly and completely as possible, patient population, and geographic location.

What if there is not a study site near where I live? Can I relocate to participate in a study?

Clinical studies are subject to international, national and local laws and regulations. Additionally, factors such as institutional site policies, health insurance and travel burden may impact your ability to relocate and be accepted into one of the study sites. Eligibility and enrolment are decided by the study investigator at each site, who takes into account all these factors and may also wish to speak to you or your local HD specialist for more information. Whether your HD centre is selected for participation or not, this is no reflection on the quality of the many outstanding HD clinics and dedicated care providers around the world. The need in HD is greater than the capacity of our development programme. We have designed the programme to provide the required data to health authorities so that the benefits and risks of RG6042 can be determined as quickly as possible. Our ultimate goal is that this investigational medicine can be approved by health authorities, and made accessible to the broader HD community.

Can I access RG6042 outside of clinical studies?

Currently, access to RG6042 is only through clinical study participation because the benefits and risks of RG6042 are not yet fully understood. This means that we are not able to grant pre-approval, compassionate use or “right-to-try” requests at this time. As our understanding of the benefits and risks of RG6042 grows, we will regularly evaluate this position.

Your clinical studies are in early manifest and manifest HD. Will you study RG6042 in other patient populations (e.g., juvenile onset HD or prodromal HD)?

We recognise the critical medical need for a treatment for HD, especially for people living with severe forms like juvenile onset HD. Once there is sufficient scientific and safety rationale, our team will consult with HD community experts and explore the potential use of RG6042 in populations beyond manifest HD.


For the full history of updates on this trial, click here.


We support those facing huntington disease
 Latest Blog
Erin’s Story

Spring 2019 Horizon Article Family Planning with HD: Adoption By Tiffany Spencer When Erin and her husband were 31 years
Read more

 Questions

Questions

Let Us Know How We Can Help
Enter your name, email address and question(s) below for assistance.

 Connect
 Newsletter

Newsletter

Stay in Touch!
Enter your email address below to receive occasional updates.

Newsletter Sign-up