First global patient recruited to new Huntington's Disease trial

The first participant in the world to take part in a new Huntington's Disease (HD) trial has been recruited in London.

The patient was recruited at University College London Hospitals NHS Foundation Trust (UCLH).

The study, supported by the NIHR Research Delivery Network, is investigating the safety of a medication called ALN-HTT02. It will seek to understand the effects in the body and whether it can reduce levels of the huntingtin (HTT) protein in the brain. A faulty version of the HTT gene causes HD by damaging nerve cells in the brain.

The study medication is designed to use RNA interference (RNAi) to silence the faulty HTT gene. The aim is to temporarily stop the body’s production of HTT protein. ALN-HTT02 is administered through an injection into the fluid around the spinal cord.

HD is an inherited brain condition. It is a progressive disease and gradually reduces a person's ability to walk, talk, think, and communicate. Around 7,000 people are living with HD in the UK.

Treatments and support are available to help reduce some of the symptoms associated with the disease. These include physiotherapy to help with movement and balance, and speech and language therapy for feeding and communication problems.

There are currently no treatments to prevent the disease from progressing.

Professor Sarah Tabrizi, Director of the Huntington’s Disease Centre, UCL Queen Square Institute of Neurology, and consultant at UCLH, is leading the study within the UK. She said: “We are very excited about this trial as the drug, ALN-HTT02, targets a specific region of the HTT gene called exon 1, which we now think is a key toxicity driver of the damage seen in Huntington’s Disease.

"I am so pleased we have begun this important and innovative trial, which we hope will bring us closer to an effective treatment for HD and make a meaningful difference for patients and families affected by this devastating condition. I’m looking forward to the smooth running of the study and, hopefully, seeing ALN-HTT02 progress to further clinical development.”

The sponsor of this first-in-human trial, Alnylam Pharmaceuticals, has co-developed the investigational medication under its collaboration with Regeneron Pharmaceuticals.

“Professor Tabrizi and UCLH are widely recognised for their expertise in HD and their patient-centric approach to clinical research,” said Kevin Sloan, Vice President, Development Programs at Alnylam who leads the ALN-HTT02 program. “We’re thrilled to be collaborating with established leaders in the space to advance an investigational RNAi therapeutic that we believe has the potential to alter the course of this devastating disease.”

The goal is to enroll up to 54 participants in the study. Participants initially enter the ‘double-blind period’ of the study for the first 6 months. During this phase they are randomly assigned to receive a single injection of either ALN-HTT02 or placebo. The placebo injection arm of the study contains no active ingredients. Following the double-blind period, participants who receive the placebo can then choose to receive ALN-HTT02.

Multiple other sites are expected to participate in the study across the UK, Canada, United States and Germany.

This study was one of the first early-phase studies to be part of the National Contract Value Review process (NCVR). Early phase clinical trials, also known as phase I and phase II trials, are the first steps in testing new drugs or treatments in humans.

The NCVR is the UK’s standardised, national approach to costing and contracting for commercial contract research. It is mandatory for commercial companies delivering clinical trials in NHS trusts.

The NHS is made up of many separate organisations in the UK. NCVR is a system-wide process that enables those organisations to work as a single organisation. This increases the speed of costing and contracting activities for commercial sponsors setting up studies. It achieves this by using model agreements and a standardised pricing structure.

NCVR, which has now been rolled out nationally, has replaced the time-consuming process which required commercial sponsors to negotiate independently with each hospital or GP surgery to agree contract terms and prices.

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