Operational Support For Clinical Trials
Under construction
EHDN Clinical Trial Protocol Advice
Sponsors are encouraged to consult EHDN experts during the protocol development process and receive advice and recommendations on early study concepts, protocol synopses and initial protocol draft(s).
EHDN Clinical Trial Protocol Endorsement
EHDN endorsement is a formal statement from an independent group of HD experts that a protocol meets high scientific and ethical standards. It is widely recognised within the HD community and may guide decisions by potential participants about taking part in specific trials and studies.
Some companies have found that the endorsement decision statement can be helpful to accompany regulatory/ethics submissions. Permission from the EHDN Executive Committee (EC) is required to share the endorsement decision.
The study will also be publicised on the EHDN website and newsletter.
The EC is responsible for the final endorsement decision based on the protocol review comments and recommendations from the independent EHDN Scientific Bioethical Advisory Committee (SBAC). The endorsement response will include advisory comments, if applicable.
Clinical studies endorsed by the EC must meet four criteria:
- They should be based on a solid rationale.
- There should be no safety concerns that would imply that study participants would be exposed to substantial (and unacceptable) risks.
- A proposed clinical study plan includes pre-clinical/clinical data and appears to be appropriate, comprehensive, operational, and likely to yield conclusive, definite results.
- The demands of a proposed clinical study on the network, as well as the clinicians active in running the trial, the research participants, and their families, are acceptable to ensure the effective use of these resources.
Application Requirements and Process
A draft protocol (and supporting documents if applicable) is required for endorsement review. The Investigator Brochure is expected; a review of the Investigator Brochure is not conducted, but it should be made available to justify the decisions made in the protocol.
Approximately 6 weeks should be allowed for the review, but this is dependent on the EC meeting schedule where the final decision is taken (typically the first Tuesday of each month). When submissions are received/planned, timelines for the review will be communicated.
Upon completion of the review, a written response will be sent detailing the endorsement decision or outlining any questions that require clarification.
For additional information regarding endorsement please contact Jenny Townhill. To apply for endorsement, contact endorsement@ehdn.org.
To maintain EHDN endorsement, the submission of the final protocol (if a draft was initially submitted) and any subsequent protocol amendments is required.
For a complete list of current and completed EHDN-endorsed clinical studies, click here.
last update 9 December 2025