How to get involved in HD research

Current HD studies open for recruitment include: 

Cohort study - Enroll-HD

Enroll-HD is a prospective observational multi centre multi-national cohort study that we are involved in that is being conducted in multiple native languages. Study procedures include annual assessments conducted during study visits that may coincide with regularly scheduled clinic visits. The study does not include intervention procedures outside of normal clinical care, nor does it include experimental therapies. Enroll-HD is an open-ended, prospective study. Participants will be asked to participate in as many annual study visits as possible. Enroll-HD is funded by CHDI.

The study visit involves memory tests, questionnaires and optional samples, the visits happen once a year for as many years as the participant is happy to continue and takes about 1 hour. For more information, please see the Enroll-HD website here.

Clinical Trials 

Alnylam Trial ALN-HTT02: A Randomized, Double-blind, Placebo-controlled Study, to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered Single Ascending Doses of ALN-HTT02 in Adult Patients with Huntington’s Disease

ALN-HTT02 works by interfering with the messages from a faulty gene causing Huntington’s disease and reducing the amount of HTT protein being made. This research study is to test the safety of a new study drug, ALN-HTT02, and to find out what effects, if any, it has on people with Huntington’s disease (HD). This study represents the first time that ALN-HTT02 has been given to humans. 

The study drug or placebo will be administered through an injection into the fluid around your spinal cord (intrathecally) by a procedure called a lumbar puncture (also known as spinal tap) by the study doctor or by a clinician designated by the study doctor.  The participation in this study is expected to last for at least 14 months, with the option to extend by a further 12 months. The study involves a taking medical history, questionnaires, MRI scans, blood samples, urine samples, ECGs, lumbar punctures and the study drug administration. 

Novartis Trial CHTT227A12301: A randomized, placebo-controlled, double-blind Phase 3 study to evaluate the efficacy, safety and tolerability of votoplam in participants with Huntington’s Disease

Votoplam is designed to lower the amount of a protein called huntingtin (HTT), which plays a role in Huntington’s disease. Votoplam works by changing how the body makes the HTT protein, so that less of it is produced in the brain and other parts of the body. 

The random assignment to either votoplam or placebo will be in a ratio of 3:2, both the placebo or drug is taken orally. The participation in this study is expected to last for at least 24-36 months. The study involves a taking medical history, questionnaires, MRI scans, blood samples, urine samples, ECGs, lumbar punctures and the study drug administration. 

Future Projects

DORA-HD

Zanna Voysey is continuing her work in sleep in Huntington’s, looking at whether improving associated sleep problems might help mitigate symptoms, such as problems with attention, memory and mood, or even slow disease progression. The study will be investigating the use of Daridorexant in Huntington's Disease. 

If you are interested in participating or would like more information on any of these projects, please reach out to Amy or Katie!

Amy Evans (Clinical Trial Assistant) - ahe28@cam.ac.uk 

Katie Andresen (Research Manager & Trial Coordinator) - kera2@cam.ac.uk