RO7234292 for Huntington's Disease

No longer recruiting at 45 trial locations

Overseen By Reference Study ID Number: BN40955 https://forpatients.roche.com/

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Hoffmann-La Roche

Must not be taking: Antiplatelets, Anticoagulants

Disqualifiers: Bleeding diathesis, Coagulopathy, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 8

Duration: Up to approximately 3 years

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to assess the long-term safety and tolerability of a drug called RO7234292 (RG6042) for individuals with Huntington's disease who participated in earlier studies. Participants will receive the drug in various ways, based on their previous treatment experiences. Those who completed prior Huntington's studies with Roche or Genentech and have a confirmed diagnosis may qualify for this trial. As a Phase 3 trial, this study represents the final step before FDA approval, providing participants an opportunity to contribute to a potentially groundbreaking treatment.

Will I have to stop taking my current medications?

The trial requires that you stop taking antiplatelet or anticoagulant medications (like aspirin or warfarin) at least 14 days before joining and during the study. For other medications, the protocol does not specify, so it's best to discuss with the study team.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Studies have shown that RG6042, the treatment under study for Huntington's disease, is generally well-tolerated in humans. Past research reported no serious side effects in those who received this treatment. Most unwanted effects were mild and not directly linked to the drug. This suggests that RG6042 is likely safe for participants. However, discussing any concerns with a healthcare provider before joining a trial is important. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Researchers are excited about RO7234292 (RG6042) because it offers a novel approach to treating Huntington's disease. Unlike existing treatments that mainly address symptoms, RO7234292 targets the root cause by reducing the production of the mutant huntingtin protein, which is responsible for the disease's progression. This treatment is an antisense oligonucleotide, a unique class of drugs designed to bind to the genetic material and prevent the creation of harmful proteins. By directly targeting the genetic cause, RO7234292 has the potential to slow down or even halt the progression of Huntington's disease, offering hope for more effective long-term management.

What evidence suggests that RO7234292 (RG6042) could be an effective treatment for Huntington's disease?

Research has shown that RO7234292 (also known as RG6042) is a promising treatment for Huntington's disease because it targets the disease's root cause. Earlier studies demonstrated that this treatment reduces levels of the harmful mutant huntingtin (mHTT) protein, which plays a key role in Huntington's disease. Lowering mHTT has been linked to improvements in symptoms. In this trial, participants will receive RO7234292 either every 8 weeks (Q8W) or every 16 weeks (Q16W). These findings suggest that RO7234292 can effectively slow the disease by addressing its underlying causes. Although long-term data collection continues, the initial results are encouraging. ^{1 3 6 7 8}

Who Is on the Research Team?

Clinical Trials

Principal Investigator

Hoffmann-La Roche

Are You a Good Fit for This Trial?

Inclusion Criteria

The text suggests that the individual should have functional independence in order to maintain self-care and core activities of daily living.

during the course of the trial and for three months after the last dose of the study medication Ladies who could potentially become pregnant must agree to not have sex or use contraception during the trial and for three months after the last dose of the study drug.

will have the opportunity to enroll in an open-label extension study Patients who were screened and eligible for the Phase III study BN40423 but could not be randomized prior to the close of enrollment due to challenges relating to the COVID-19 pandemic will have the opportunity to enroll in an open-label extension study.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive RO7234292 (RG6042) every 8 weeks or every 16 weeks by bolus intrathecal injection

Up to approximately 3 years

Visits every 8 or 16 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

5 months

Open-label extension

Participants continue to receive RO7234292 (RG6042) to evaluate long-term safety and tolerability

Long-term

What Are the Treatments Tested in This Trial?

Interventions

• RO7234292 (RG6042)

How Is the Trial Designed?

Group I: RO7234292 (RG6042) Q8W Experimental Treatment 1 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) Q16W Experimental Treatment 1 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.

Who Is Running the Clinical Trial?

Hoffmann-La Roche

Lead Sponsor

Trials

2,482

Recruited

1,107,000+

Headquarters

Basel, Switzerland

Known For

Precision medicine

Top Products

Avastin, Herceptin, Rituxan, Accu-Chek

Dr. Levi Garraway

Hoffmann-La Roche

Chief Medical Officer since 2019

MD from the University of Basel

Dr. Thomas Schinecker

Hoffmann-La Roche

Chief Executive Officer since 2023

PhD in Molecular Biology from New York University

Citations

1. ir.ionis.com ir.ionis.com/news-releases/news-release-details/ionis-htt-rx-rg6042-top-line-data-demonstrate-significant

IONIS-HTT Rx (RG6042) Top-Line Data Demonstrate ... The data demonstrate that IONIS-HTT Rx (RG6042) is the first drug in development to lower the disease-causing protein in people with HD.

2. clinicaltrials.gov clinicaltrials.gov/study/NCT03761849

NCT03761849 | A Study to Evaluate the Efficacy and ... Completed. A Study to Evaluate the Efficacy and Safety of Intrathecally Administered RO7234292 (RG6042) in Participants With Manifest Huntington's Disease.

3. ir.ionis.com ir.ionis.com/news-releases/news-release-details/new-data-ionis-htt-rx-phase-12-study-demonstrates-correlation

New Data from IONIS-HTT Rx Phase 1/2 Study ... The study demonstrated correlations between reductions in mutant huntingtin (mHTT), the disease-causing protein, and improvements in clinical measures of ...

4. clinicaltrials.gov clinicaltrials.gov/study/NCT03842969

Study Details | NCT03842969 | An Open-Label Extension ... This study will evaluate the long-term safety and tolerability of RO7234292 (RG6042) in participants who have completed other F. Hoffmann-La Roche, ...

5. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10674993/

Huntington's Disease Drug Development: A Phase 3 ... The primary outcome was a change in the UHDRS TMC score from baseline to the maintenance period (an average of weeks 10 and 12 scores). A ...

6. en.hdbuzz.net en.hdbuzz.net/265/

HDSA FAQ on the Roche/Genentech RG6042 program There were no serious side effects in people who received RG6042. The safety and tolerability data from the study provided the necessary ...

7. trial.medpath.com trial.medpath.com/clinical-trial/08b1ec83ef8874d2/jrct2080224757-efficacy-safety-ro7234292-huntington-disease

A Study to Evaluate the Efficacy and Safety of Intrathecally ... What are the potential adverse events associated with RO7234292 (RG6042) in neurodegenerative disorders? Are there any combination therapies or competitor ...

8. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC6839470/

Huntington's Disease Clinical Trials Corner: June 2019 A Study to Evaluate the Efficacy and Safety of Intrathecally Administered RO7234292 (RG6042) in Patients With Manifest Huntington's Disease.