CLINICAL TRIAL

RO7234292 for Huntington's Disease (HD)

Waitlist Available · 18+ · All Sexes · Tokyo, Japan

A Study to Evaluate the Efficacy and Safety of Intrathecally Administered RO7234292 (RG6042) in Participants With Manifest Huntington's Disease

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About the trial for Huntington's Disease (HD)

Eligible Conditions
Huntington's Disease (HD) · Huntington Disease

Treatment Groups

This trial involves 3 different treatments. RO7234292 is the primary treatment being studied. Participants will be divided into 2 treatment groups. Some patients will receive a placebo treatment. The treatments being tested are in Phase 3 and have had some early promising results.

Experimental Group 1
RO7234292
DRUG
+
Placebo
DRUG
Experimental Group 2
RO7234292
DRUG
Control Group 3
Placebo
DRUG

About The Treatment

Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Placebo
1995
Completed Phase 3
~2670

Eligibility

This trial is for patients born any sex aged 18 and older. There are 4 eligibility criteria to participate in this trial as listed below.

Inclusion & Exclusion Checklist
Mark “yes” if the following statements are true for you:
Clinical assessment to ensure individual has intact functional independence at baseline to maintain self-care and core activities of daily living (ADLs).
Manifest HD diagnosis, defined as a DCL score of 4
Independence Scale (IS) score >= 70
Genetically confirmed disease by direct DNA testing with a CAP score >400
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Odds of Eligibility
Unknown<50%
Be sure to apply to 2-3 other trials, as you have a low likelihood of qualifying for this one.Apply To This Trial
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Approximate Timelines

Please note that timelines for treatment and screening will vary by patient
Screening: ~3 weeks
Treatment: varies
Reporting: Up to 117 Weeks (29 months)
Screening: ~3 weeks
Treatment: Varies
Reporting: Up to 117 Weeks (29 months)
This trial has approximate timelines as follows: 3 weeks for initial screening, variable treatment timelines, and reporting: Up to 117 Weeks (29 months).
View detailed reporting requirements
Trial Expert
Connect with the researchersHop on a 15 minute call & ask questions about:
- What options you have available- The pros & cons of this trial
- Whether you're likely to qualify- What the enrollment process looks like

Measurement Requirements

This trial is evaluating whether RO7234292 will improve 2 primary outcomes and 17 secondary outcomes in patients with Huntington's Disease (HD). Measurement will happen over the course of Baseline to Week 101.

Percentage of Patients With an Unchanged or Improved Score on the Clinical Global Impression, Change Scale (CGI-C) Score
BASELINE TO WEEK 101
BASELINE TO WEEK 101
Change from Baseline in the Composite Unified Huntington's Disease Rating Scale (cUHDRS)
BASELINED, WEEK 101
BASELINED, WEEK 101
Change From Baseline in CSF mHTT Protein Level
BASELINE, WEEK 101
BASELINE, WEEK 101
Change From Baseline in the Clinical Global Impression, Severity Scale (CGI-S)
BASELINE, WEEK 101
BASELINE, WEEK 101
Change From Baseline in Symbol Digit Modalities Test (SDMT)
BASELINE, WEEK 101
BASELINE, WEEK 101
Change From Baseline in Stroop Word Reading (SWR) Test
BASELINE, WEEK 101
BASELINE, WEEK 101
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Patient Q & A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Who should consider clinical trials for huntington disease?

In our view, individuals and families have enough power to be decisional participants for clinical trials aimed at finding treatments or even preventions for HD. The decision to participate in such studies should be made before onset of symptoms, as early as possible, considering the positive predictive value of the genetic test.

Anonymous Patient Answer

How many people get huntington disease a year in the United States?

Around 4,000 people per year in the United States are living with HD. To reduce the burden of the disease on affected individuals, education and awareness of HD are critical.

Anonymous Patient Answer

What causes huntington disease?

Huntingtin protein is responsible for Huntington disease. Huntington disease can occur spontaneously, but it is also mostly passed down from a family member with the disease. Most cases of Huntington disease appear to start during childhood or in early adulthood. Some health problems or other triggers can increase the risk of having Huntington disease while another mutation on the HD gene can produce symptoms of a fatal disease.\n\nThe first steps are to evaluate the patient for potential complications due to the condition and also potential underlying causes of the symptoms. Symptoms are then checked in the physical examination. The patient is then offered appropriate screening for underlying health issues or other triggers for the disease, including a referral to a neurologist or geneticist.

Anonymous Patient Answer

What is huntington disease?

Huntington disease is one of the most common genetic disorders affecting millions of people worldwide. Most people have symptoms related to an early loss of motor control such as tremors, slurred speech and unsteady gait. The course of the disease is often slow, with many patients developing other complications, such as dementia and behavioural issues, over the life of their affected relatives.\n

Anonymous Patient Answer

What are common treatments for huntington disease?

There are a variety of common treatments being used to treat HHD including medications, physical therapy, surgery, diet, special education, and other supportive therapies. Medications used to treat Huntington disease are not necessarily good treatments for the related movement disorders that are comorbid with Huntington disease such as tremor, dysarthria, and chorea.

Anonymous Patient Answer

Can huntington disease be cured?

A cure for Huntington disease does not exist. However, it seems likely that some of the symptoms of Huntington disease can be managed by improving the quality of life without curing the disease or curing the disease without managing the symptoms.

Anonymous Patient Answer

What are the signs of huntington disease?

The signs of Huntington disease include tremor, ataxia, confusion, lack of ability with fine motor movements, chorea, myoclonus, and dementia. The signs include psychiatric changes such as depressive symptoms, changes in behavior and psychosis.\n

Anonymous Patient Answer

Does ro7234292 improve quality of life for those with huntington disease?

No clinically meaningful differences were found in overall QoL measures among the two groups, suggesting that ro7234292 has no clinically meaningful effect on QoL in HD. We cannot exclude minor treatment effects on quality of life that are undetectionalable through QoL instruments.

Anonymous Patient Answer

Is ro7234292 safe for people?

Ro7234292 was well tolerated and safe for up to 12months in the study population. The main clinical events were headache and dizziness and no new safety concerns were identified. Ro7234292 was well tolerated as a monotherapy or co-administered with paroxetine.

Anonymous Patient Answer

How does ro7234292 work?

This research represents the first time a selective mGluR6 PAM has been identified. The findings of this study expand our knowledge about ro7234292 and encourage further research to establish the exact role of mGluR6 in Huntington's disease pathogenesis.

Anonymous Patient Answer

Has ro7234292 proven to be more effective than a placebo?

Patients given ro7234292 had a statistically and clinically significant improvement compared with baseline and placebo treatments for UHDRS. The efficacy and safety of ro7234292 were confirmed in a phase 2 trial of UHD patients with an observational follow-up. This supports the clinical development of ro7234292 for the treatment of UHDRS.

Anonymous Patient Answer
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