European Huntington's Disease Network – Advancing Research, Conducting Trials, Improving Care

European Huntington's Disease Network – Advancing Research, Conducting Trials, Improving Care
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Welcome to the
European Huntington's Disease Network
Our Mission
EHDN is an independent nonprofit network dedicated to advancing research, conducting clinical trials and improving care for people impacted by Huntington’s disease. The network is comprised of clinicians, researchers and people affected by HD, working together to accomplish our mission.
LEARN MORE ABOUT EHDN
EHDN Newsletter: Issue 57
Click here to read our current newsletter: March 2026
The next Newsletter will be published in
July 2026-
watch this space!
Featured Article:
“From Strasbourg to
Kraków: An Interview with Nayana Lahiri and Hoa Nguyen”
(Issue 57, March 2026)
Newsletter Archive
We welcome feedback and suggestions:
newsletter@ehdn.org
To receive the EHDN News via email, sign up for the EHDN newsletter at the bottom of this page↓
EHDN Elections 2026
Nominations
for the Executive Committee and Scientific and Bioethics Advisory Committee are now open (1–30 April 2026).
Find out
more
or submit your
nomination
today.
MORE!
The
Behavioral Phenotype Working Group
is conducting an
expert opinion survey
on treatment strategies for neuropsychiatric symptoms in advanced Huntington’s Disease. Closing date
01 June 2026
. Click
here
for more information.
Latest Updates on EHDN-Endorsed Study: AMT-130 (uniQure)
Following a
Type A meeting
held in January 2026, uniQure
announced
on 2 March 2026 that the FDA ‘cannot agree that data from the phase 1/2 studies, compared to an external control, provide sufficient primary evidence of effectiveness to support a marketing application for AMT-130. The FDA strongly recommended that uniQure conduct a prospective, randomised, double-blind, sham surgery-controlled study.’
Further discussions between uniQure and the FDA are anticipated regarding phase 3 development considerations. uniQure plans to request a Type B meeting in the second quarter of 2026 to further discuss potential study design approaches.
Click
here
for uniQure’s 2025 financial results and company updates released on 2 March 2026. All uniQure press releases are available
here
about Enroll-HD.
The seventh release of the Enroll-HD periodic dataset, PDS7, has been fully curated and is now available.
PDS7 contains data from
30,511 Enroll-HD study participants
encompassing
112,992 study visits
, making it one of the largest cohort datasets available to researchers. Read more
here
– – –
ave the
ate – – –
EHDN Clinical Research Congress 2026
in
Kraków, October 22-24
Registration
will be open from
1 June to 31 July 2026
Abstract submission
will be open from
1 to 30 June 2026
Novartis Pharmaceuticals INVEST-HD study
most recently endorsed* by EHDN
Study aims and goal:
Phase III study to investigate the efficacy, safety and tolerability of Votoplam in participants with Huntington’s Disease.
No. of study participants and sites:
It is planned to enrol 770 participants globally. The number of sites and countries is still to be determined.
For further information
see the
clinical trial registration
*EHDN endorsement:
The EHDN endorses protocols it finds to be of a high scientific and ethical quality. The endorsement decision is made on the basis of protocol review and discussion by the EHDN Executive Committee following recommendations from the independent EHDN Scientific and Bioethical Advisory Committee. Trial protocol endorsement should not be viewed as sponsor promotion or recruitment incitement.
[EHDN endorsed studies overview]
Hoffmann-La Roche POINT-HD study endorsed* by EHDN
Study aims and goal:
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RG6496 in Huntington’s Disease.
No. of study participants and sites:
The study plans to enrol participants from 2 sites in Australia and New Zealand.
For further information
see the
clinical trial registration
*EHDN endorsement:
The EHDN endorses protocols it finds to be of a high scientific and ethical quality. The endorsement decision is made on the basis of protocol review and discussion by the EHDN Executive Committee following recommendations from the independent EHDN Scientific and Bioethical Advisory Committee. Trial protocol endorsement should not be viewed as sponsor promotion or recruitment incitement.
[EHDN endorsed studies overview]
Vico Therapeutics B. V. study endorsed* by EHDN
Study aims and goal:
The goal of this first-in-human clinical trial is to assess the safety and tolerability of four doses of a new study drug called VO659 in people with genetic disorders called spinocerebellar ataxia type 1, type 3 or Huntington’s disease.
No. of study participants and sites:
The study plans to enrol participants from 14 sites in Denmark, France, Germany, Israel, Netherlands and United Kingdom.
For further information
see the
clinical trial registration
*EHDN endorsement:
The EHDN endorses protocols it finds to be of a high scientific and ethical quality. The endorsement decision is made on the basis of protocol review and discussion by the EHDN Executive Committee following recommendations from the independent EHDN Scientific and Bioethical Advisory Committee. Trial protocol endorsement should not be viewed as sponsor promotion or recruitment incitement.
[EHDN endorsed studies overview]
Alnylam Pharmaceuticals Global study endorsed* by EHDN
Study aims and goal:
ALN-HTT02 is a novel C16-siRNA conjugate for huntingtin-lowering. This Phase Ib study aims to evaluate ALN-HTT02 in adults with Huntington’s Disease.
No. of study participants and sites:
The study plans to enrol participants from 2 sites in Australia and New Zealand.
For further information
see the
clinical trial registration
*EHDN endorsement:
The EHDN endorses protocols it finds to be of a high scientific and ethical quality. The endorsement decision is made on the basis of protocol review and discussion by the EHDN Executive Committee following recommendations from the independent EHDN Scientific and Bioethical Advisory Committee. Trial protocol endorsement should not be viewed as sponsor promotion or recruitment incitement.
[EHDN endorsed studies overview]
Active EHDN endorsed* studies
*EHDN endorsement:
The EHDN endorses protocols it finds to be of a high scientific and ethical quality. ...
• Alnylam Pharmaceuticals – ALN-HTT02-001
• Hoffmann-La Roche – POINT-HD
• Hoffmann-La Roche – GENERATION-HD2
• Novartis Pharmaceuticals – HTT227
• UniQure Biopharma B.V. – HD GeneTRX2
• Vico Therapeutics – VO659-CT01
↪MORE
HD PATIENTS & FAMILIES
Researchers, clinicians, and HD families
work together within the EHDN to fulfil our mission, and anyone who has an interest in HD may become an
EHDN member
. You can also find out more
about HD
clinical sites
and research participation opportunities near you, as well as ways to get in touch with further resources for you and your family.
HD CLINICIANS & RESEARCHERS
The EHDN advances research
by providing access to
large longitudinal clinical datasets and biosamples
from the
Enroll-HD
and
REGISTRY
studies as well as through the
working groups
fellowship exchange programmes
seed funds
grant manager support
scientific input
and
operational support
for clinical trials, development of
clinical assessment tools
and
guidelines for best practice in HD care
, and arranging biennial scientific
plenary meetings
CLINICAL TRIAL SPONSORS
The EHDN is keen to work with sponsors
of HD clinical trials
– from the pharmaceutical industry or academia – to help facilitate the conduct of their clinical trials. We offer
a range of services
to expedite clinical trial set-up and recruitment as well as provide scientific input via
endorsement of protocols
feasibility assessment
operational support
recruitment support
and
clinical rater training
Full List of Services
Participate
Our Locations
Research Projects
The EHDN supports HD research and clinical development at all stages, from basic and applied science through to the development of new treatment strategies, clinical assessment tools, and best clinical practice guidelines, to site feasibility assessment, site selection and patient recruitment for clinical trials.
See all research
Comparison of Huntington’s Disease in Europe and North America
Validation of a prognostic index for Huntington’s disease
Identification of genetic variants associated with Huntington’s disease progression: a genome-wide association study
Age of onset in Huntington’s disease is influenced by CAG repeat variations in other polyglutamine disease-associated genes