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Antisense Oligonucleotide

An Open-Label Extension Study to Evaluate Long-Term Safety and Tolerability of RO7234292 (RG6042) in Huntington's Disease Participants Who Participated in Prior Roche and Genentech Sponsored Studies

Phase 3

Waitlist Available

Research Sponsored by Hoffmann-La Roche

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to approximately 3 years

Awards & highlights

No Placebo-Only Group

Pivotal Trial

Summary

This trial will test the long-term safety of a drug called RO7234292 (RG6042) in people who have already participated in earlier studies for Huntington's disease. The drug is given through an injection into the spinal fluid periodically. The goal is to see if the drug remains safe and tolerable over a longer period. RO7234292 (RG6042) is being tested for its ability to target and reduce the production of the huntingtin protein, which is implicated in Huntington's disease.

Eligible Conditions

Huntington's Disease

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to approximately 3 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to approximately 3 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to approximately 3 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Change From Baseline in Cognition, Using Montreal Cognitive Assessment (MoCA)

Number of Participants With Suicidal Ideation, Suicidal Behavior, and Self-Injurious Behavior Without Suicidal Intent Based on the Columbia Suicide Severity Rating Scale (C-SSRS)

Percentage of Participants With Treatment Emergent Adverse Events

Side effects data

From 2022 Phase 3 trial • 236 Patients • NCT03842969

50%

Fall

14%

Contusion

14%

Procedural pain

14%

Back pain

14%

Anxiety

14%

Irritability

14%

Haematoma

Sinus congestion

Pigmentation disorder

Euthanasia

Injection site infection

Cerebral ventricle dilatation

Onychomycosis

Psoriatic arthropathy

Coordination abnormal

Tinnitus

Vision blurred

Diarrhoea

Gastrooesophageal reflux disease

Fatigue

Oedema peripheral

Tooth infection

Urinary tract infection

Clavicle fracture

Eye contusion

Foreign body aspiration

Periorbital haematoma

Skin abrasion

Skin laceration

Spinal compression fracture

Blood alkaline phosphatase increased

CSF protein increased

Vitamin D decreased

Bursitis

Enthesopathy

Exostosis

Fracture pain

Limb mass

Musculoskeletal stiffness

Spinal osteoarthritis

Vertebral foraminal stenosis

Vertebral osteophyte

Amnesia

Aphasia

Ataxia

Balance disorder

Cerebellar atrophy

Dysarthria

Extensor plantar response

Hypotonia

Memory impairment

Motor dysfunction

Parkinsonism

Pleocytosis

Post-traumatic headache

Restless legs syndrome

Affect lability

Depressed mood

Depression

Insomnia

Rhinitis allergic

Lymphoedema

100%

80%

60%

40%

20%

Study treatment Arm

Tominersen 120mg Q4W

Tominersen 120 mg Q8W

Tominersen 120 mg Q16W

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups

Experimental Treatment

Group I: RO7234292 (RG6042) Q8WExperimental Treatment1 Intervention

Participants who received open-label RO7234292 Q4W in a preceding study or who received RO7234292 Q4W in this study may be randomly allocated to receive RO7234292 Q8W. Participants who previously received open-label of RO7234292 Q8W in a preceding study or are currently receiving RO7234292 Q8W in this study will receive RO7234292 Q8W. Participants who previously received placebo, or did not previously receive treatment with RO7234292 or received short-term treatment with a treatment-free follow-up period may be randomly allocated to receive RO7234292 Q8W. Participants who previously received blinded placebo Q8W may receive RO7234292 Q8W. Participants who received blinded RO7234292 Q8W will receive open-label RO7234929 Q8W. Participants who received blinded placebo Q8W may receive RO7234292 Q8W. Participants who received blinded RO7234292 Q4W or blinded placebo Q4W may be randomly allocated to receive open-label of RO7234292 Q8W.

Group II: RO7234292 (RG6042) Q16WExperimental Treatment1 Intervention

Participants who previously received open-label RO7234292 Q4W in a preceding study or who received RO7234292 Q4W in this study may be randomly allocated to receive RO7234292 Q16W. Participants who previously received placebo in a preceding study or did not previously receive treatment with RO7234292 or received short-term treatment with a treatment-free follow-up period may be randomly allocated to receive RO7234292 Q16W. Participants who previously received blinded placebo Q8W will receive RO7234292 Q8W. Participants who previously received blinded RO7234292 Q16W will receive open-label RO7234292 Q16W. Participants who received blinded RO7234292 Q4W or blinded placebo Q4W may be randomly allocated to receive open-label of RO7234292 Q16W. Participants who previously received open-label RO7234292 Q16W will receive open-label RO7234292 Q16W.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

RO7234292 (RG6042)

2019

Completed Phase 3

~300

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