Highlights from the 2020 CHDI HD Therapeutics Conference are now here! This year’s Postcard covers HTT-lowering therapeutics, including an
HD Clinical Trials In Canada
The Huntington Society of Canada (HSC) plays a key role in bridging the relationship between researchers and individuals by educating Canadians on the importance of the clinical trial process, how to get involved and why participation is so crucial. The urgency lies in not only educating as many people as possible, including efforts in rural and culturally diverse communities, but also ensuring clinicians have sustainable mechanisms in place to support the process.
HSC is emerging as a leader in linking researchers and clinicians in order to continually prepare for future trials. Drugs being tested now have been specifically designed for Huntington disease (HD). This is a significant step forward. Managing HD will take the collective efforts of researchers, scientists, clinicians, the Huntington Society of Canada and HD families all have to work together. It is our best chance for success.
2015 was an important year for HD clinical trials. Participation in Enroll-HD will facilitate your involvement in clinical trials when they are available in your area. Enroll-HD is a platform that allows health-care professionals, scientists, and families affected by HD to work together towards a better understanding of HD and identify effective treatments. Their objective is to create the world’s largest database for clinical research on HD, eventually including information from as many as 20,000 people in 33 countries. Visit www.Enroll-HD.org to learn more and to find out the closest site for you to participate.
Clinical Trials Currently In Progress
- ENROLL-HD is a observational study for HD families that monitors how HD appears and changes in people over time. The study’s overarching goal is to accelerate the discovery and development of new therapeutics through a better understanding of HD. This study is complete.
- HDClarity – an observational Study; Cerebrospinal Fluid Collection Initiative to Facilitate Therapeutic Development for Huntington’s Disease
- PIVOT-HD study offered by PTC Therapeutics, Inc. is investigating PTC518 to evaluate the safety and pharmacodynamics effects compared with placebo in participants with HD.
- DIMENSION study offered by Sage Therapeutics, Inc. is investigating SAGE-718 for the treatment of cognitive symptoms associated with Huntington disease (HD) in adults with premanifest or early manifest HD.
- PURVIEW extension study offered by Sage Therapeutics, Inc. is evaluating the safety and tolerability of SAGE-718 in adults with Huntington disease (HD).
- WVE-003 – Select HD study (Phase 1b/2a) will evaluate the safety, tolerability, PK, and PD of WVE-003 in adult patients with early-manifest HD who carry the targeted single nucleotide polymorphism (SNP) – SNP3.
- Valbenazine (by invitation only) is a Phase 3, open-label study to evaluate the long-term safety and tolerability of valbenazine, and to provide subjects continued access to valbenazine for the treatment of chorea associated with Huntington disease.
STOPPED CLINICAL TRIALS
- VIBRANT-HD is the first study of branaplam in adults with Huntington’s Disease (HD) to determine the correct dose required to lower mutant huntingtin protein (mHTT) levels in the cerebrospinal fluid (CSF) to a degree expected to be efficacious over longer periods of time. VIBRANT-HD was discontinued on December 8, 2022. For more information please click here.
- WVE-HDSNP2-002 is an open-label extension (OLE) study to evaluate the safety, tolerability, PK, PD, and clinical effects of WVE-120102 in adult patients with early manifest HD who carry a targeted single nucleotide polymorphism, rs362331 (SNP2). To participate in the study, patients must have completed the Phase 1b/2a clinical study WVE-HDSNP2-001. WVE-HDSNP2-002 was discontinued on March 29, 2021. For more information please click here.
- Wave Precision-HD is a clinical trial testing a new drug that lowers the mutant Huntington’s disease protein, whilst leaving the normal huntingtin protein untouched. Wave Life Sciences in sponsoring this trial. WAVE PRECISION-HD1 and PRECISION-HD2 was discontinued on March 29, 2021. For more information please click here.
- GEN-EXTEND will evaluate the long-term safety and tolerability of RO7234292 (RG6042) in participants who have completed other F. Hoffmann-La Roche, Ltd.-sponsored and/or Genentech-sponsored studies in the Huntington’s disease (HD) in the development program for RG6042. GEN-EXTEND was paused on March 22, 2021. For more information please click here.
- GENERATION HD1 will evaluate the efficacy and safety of RG6042 treatment (now known as tominersen), given once every two months (bi-monthly) or once every 4 months (16 weeks) over a period of 25 months (approx. two years). GENERATION HD1 was halted on March 22, 2021. For more information please click here.
COMPLETED CLINICAL TRIALS
- FuRST 2.0 is an observational study to identify real or potential comprehension or usage problems with scale items, response options, instructions and disclaimer statement, which are all components of the FuRST 2.0 scale.
- SIGNAL: The purpose of this study, launched by Vaccinex in 2015, was to evaluate the safety, tolerability, PK, and efficacy of Pepinemab (VX15/2503) in subjects with late prodromal and early manifest Huntington’s disease.
- SHIELD HD is a natural history observational study, developed by Triplet Therapeutics. The study will include about 60 HD gene carriers – both pre-manifest and early manifest individuals – and is intended to inform future clinical trials.
- KINECT-HD is a phase III clinical trial being conducted by the Huntington Study Group (HSG). This is a randomized, double-blind, placebo-controlled study to assess the efficacy, safety and tolerability of valbenazine for the treatment of chorea associated with Huntington disease.
- PROOF – HD will evaluate the efficacy and safety of pridopidine 45mg twice daily (BID) in patients with early stage manifest Huntington Disease (HD).
- Non-invasive Brain Stimulation in Huntington’s Disease aims to study the feasibility of non-invasive brain stimulation via Transcranial pulsed current stimulation (tPCS) in patients of Huntington’s disease.
- Observational Natural History Study – an observational study aiming to further understand the role of mHTT in disease progression.
Suggested resources for people with HD and families interested in HD research: