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Tominersen for Huntington's Disease

Phase 2

Recruiting

Research Sponsored by Hoffmann-La Roche

Eligibility Criteria Checklist

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

Must have

Total body weight > 40 kg and a body mass index within the range of 18-32 kg/m2

Huntington's disease (HD) gene expansion mutation carrier status with a CAP score of 400-500 inclusive

Timeline

Screening 3 weeks

Treatment Varies

Follow Up from baseline, months 4, 8, 12, 16 and up to approximately month 24

Awards & highlights

Study Summary

This trial will test if a new drug is safe and effective for people with Huntington's Disease.

Who is the study for?

This trial is for adults over 40 kg with a BMI of 18-32 who have Huntington's disease and a specific genetic mutation score. It's open to those in the early stages or just before symptoms start, but not to anyone who has used gene therapy, brain surgery, certain medications recently, or women who are pregnant or planning pregnancy.Check my eligibility

What is being tested?

The study tests Tominersen (at two different doses: 60 mg and 100 mg) against a placebo to check its safety and effectiveness in slowing down Huntington's Disease progression. Participants will be randomly assigned to receive either the drug or placebo.See study design

What are the potential side effects?

While the side effects aren't specified here, similar drugs can cause reactions at injection sites, flu-like symptoms, potential liver issues, nervous system problems like dizziness or seizures, and could affect blood clotting.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I weigh more than 40 kg and my BMI is between 18-32.

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I carry the Huntington's disease gene with a CAP score between 400-500.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ from baseline, months 4, 8, 12, 16 and up to approximately month 24

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~from baseline, months 4, 8, 12, 16 and up to approximately month 24

This trial's timeline: 3 weeks for screening, Varies for treatment, and from baseline, months 4, 8, 12, 16 and up to approximately month 24 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Change from baseline in Total Functional Capacity (TFC) Scores (U.S. sites) at 16 months

Change from baseline in clinical laboratory results - Cerebrospinal fluid (CSF) White Blood Cell (WBC) (1/uL)

Change from baseline in clinical laboratory results Cerebrospinal fluid (CSF) protein (g/L)

+4 more

Secondary outcome measures

Change from Baseline at 16 Months for the Assessments of Stroop Word Reading (SWR) Scores

Change from baseline at 16 months for the assessments of Symbol Digit Modalities Test (SDMT) Scores

Change from baseline at 16 months for the assessments of TFC (non-U.S. sites) Scores

+7 more

Trial Design

3Treatment groups

Experimental Treatment

Placebo Group

Group I: Tominersen 60 mgExperimental Treatment1 Intervention

Group II: Tominersen 100 mgExperimental Treatment1 Intervention

Group III: PlaceboPlacebo Group1 Intervention

0 / 2Eligibility criteria met

Find a Location

Who is running the clinical trial?

Hoffmann-La RocheLead Sponsor

2,432 Previous Clinical Trials

1,089,861 Total Patients Enrolled

Media Library

Placebo Clinical Trial Eligibility Overview. Trial Name: NCT05686551 — Phase 2

Huntington's Disease Research Study Groups: Tominersen 60 mg, Tominersen 100 mg, Placebo

Huntington's Disease Clinical Trial 2023: Placebo Highlights & Side Effects. Trial Name: NCT05686551 — Phase 2

Placebo 2023 Treatment Timeline for Medical Study. Trial Name: NCT05686551 — Phase 2

Frequently Asked Questions

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

Is the age limitation for this research restricted to persons aged 85 or younger?

"The age range for potential participants of this trial is from 25 to 50 years old. Alternatively, there are 1 clinical trials available specifically for minors and 28 different options for seniors."

Answered by AI

Has Tominersen 60 mg been granted the necessary federal authorization for public use?

"As this is a Phase 2 trial, there are some data sources substantiating the safety of Tominersen 60 mg; thus, it received an approximate score of 2."

Answered by AI

What is the ceiling for patient enrollment in this clinical trial?

"Indeed, according to information hosted on clinicaltrials.gov, this study is currently recruiting volunteers. It was initially posted on 30th January 2023 and last updated 6th January 2023; it seeks 360 participants from a single site."

Answered by AI

Is this experiment open to recruitment at the present time?

"Affirmative, the clinicaltrials.gov database suggests that this research study is in progress and actively recruiting patients. It was initially shared to the public on January 30th 2023 with its last edit occurring one week prior. 360 volunteers are needed for this trial at a single location."

Answered by AI

Do I meet the qualifications to participate in this experiment?

"This is a research initiative aiming to enrol 360 individuals who have Huntington's disease and range in age from 25 - 50. The main eligibility criteria involve having an expanded HD gene mutation, possessing a CAP score between 400-500 (calculated using Age x (CAG repeat length − 33.66), being at the prodromal stage of HD with DCL 2–3 and TMS >6 or IS = 100, or demonstrating early manifest HD symptoms such as DCL 4, TMS > 6, 100 ≥ IS ≥ 70 and TFC≥8)."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

GENERATION HD2. A Study to Evaluate the Safety, Biomarkers, and Efficacy of Tominersen Compared With Placebo in Participants With Prodromal and Early Manifest Huntington's Disease.

2023. "GENERATION HD2. A Study to Evaluate the Safety, Biomarkers, and Efficacy of Tominersen Compared With Placebo in Participants With Prodromal and Early Manifest Huntington's Disease.". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05686551.

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