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Information on our clinical trials

 

Natural History Study

At a glance
Active, not recruiting
Observational study
Involves cognitive tests
Involves blood tests
Involves Magnetic Resonance Imaging
Involves repeated lumbar punctures

About the study

The HD Natural History Study monitors 100 patients with early Huntington’s disease over a period of 15 months with clinical assessments, lumbar punctures and magnetic resonance of the brain. 

The outcomes for this study include the change from baseline in clinical measures, biomarkers of neuronal injury and in brain atrophy.  The change in CSF mutant Huntingtin levels from baseline will also be measured. 

What is involved? 

The study lasts for 15 months and includes an initial screening visit to determine eligibility. For eligible participants  further four clinic visits take place with lumbar punctures, MRI brain scans, blood tests, physical, neurological examinations and vital sign measurements. Participants are also required to complete daily assessments using a smart phone at home and wearing a smart watch. 

After study participation, eligible participants will be offered to join a Roche-sponsored study of an experimental drug called RG6042 which is being tested for the treatment of HD.

Who can take part?
Patients between 25 and 65 years of age with stage I or stage II Huntington’s disease.


https://clinicaltrials.gov/ct2/show/NCT03664804?term=Roche&cond=Huntington+Disease

GEN-HD1

At a glance
Discontinued  
Drug trial 
Involves cognitive tests
Involves blood tests
Involves magnetic resonance imaging
Involves repeated lumbar punctures

About the study
A Roche sponsored phase III clinical trial evaluating the efficacy and safety of Tominersen (RG6042), an investigational drug that lowers mutant Huntingtin in the cerebrospinal fluid (CSF) of Huntington’s disease patients. 

This trial recruited 791 ambulatory Huntington’s disease patients between 25 and 65 years of age with CAP score greater than 400 across 97 study sites. Patients were randomized into three groups. One group received Tominersen every two months, another group received alternating doses of the drug or placebo every two months and a last group received only placebo every two months. Participants were regularly assessed with lumbar punctures, MRI brain scans, blood tests, physical, neurological examinations and vital sign reviews. Participants were also required to complete daily assessments using a smart phone and wear a smart watch. 

The trial was discontinued on 22 March 2021 following the independent Data Monitoring Committee’s recommendation that all dosing be stopped. Patients continue to be followed for safety and efficacy outcomes as per protocol. No new or emerging safety signals were identified in the review of the data from this study.

https://clinicaltrials.gov/ct2/show/NCT03761849 
 

GEN EXTEND

Dosing Suspended, not recruiting 

A Roche sponsored study aiming to evaluate the long-term safety and tolerability of Tominersen (RG6042) an investigational drug that lowers Huntingtin in the cerebrospinal fluid (CSF) of Huntington's disease patients in a dose-dependent manner.
Only participants who have completed other Roche-sponsored and/or Genentech-sponsored studies in Huntington's disease (HD) in the development programme for Tominersen have been recruited in this study.


The drug was administered intrathecally into the spinal fluid every 8 or every 16 weeks. Patients attend the study site every 8 weeks in order to perform complete neurological and physical exams, cognitive tests, blood and urine analyses and lumbar punctures. 


Dosing was paused on 22 March 2021 following recommendations by the Independent Data Monitoring Committee. Participants continue to be monitored for safety and efficacy outcomes as per study protocol. No new or emerging safety signals were identified in the review of the data from this study.


https://clinicaltrials.gov/ct2/show/NCT03842969 
 

 

DIGITAL HD

Studying digital biomarkers in Huntington's disease

Now recruiting

Digital-HD is a longitudinal observational study, in collaboration with Roche, which aims to test a new way to assess the clinical feature and progression of Huntington's disease (HD) using digital electronic sensors in a smartphone, smartwatch and Microsoft Kinect system.

The use of wearable sensors allows the collection of deatiled and precise information about HD in participants' everyday lives. We hope this will give us a better understanding of the disease.