This WAS a phase 3 trial where HD participants were injected in the spine with a drug called Tominersen, an ASO. The drug was believed to lower the amount of mHHT in brain cells. The delivery of the drug was stopped after it was advised by an independent committee. The participants were asked to remain and continue with data collection for the remaining period of the trial. 85% chose to continue.
Estimated Study Completion Date: March 17, 2022
Ages Eligible for Study
25 Years to 65 Years (Adult, Older Adult)
Sexes Eligible for Study:
Accepts Healthy Volunteers:
- 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
- Clinical assessment to ensure individual has intact functional independence at baseline to maintain self-care and core activities of daily living (ADLs).
- Any serious medical condition or clinically significant laboratory, or vital sign abnormality or claustrophobia at screening that, in the investigator’s judgment, precludes the patient’s safe participation in and completion of the study
- Pregnant or breastfeeding, or intending to become pregnant during the study or within 5 months after the final dose of study drug
Roche provides update on tominersen programme in manifest Huntington’s disease
Dosing to stop in Phase III clinical study of tominersen following recommendation from an Independent Data Monitoring Committee (iDMC)