Huntington's Disease - Healing Genes

Huntington’s Disease

Share This Post

Share on facebook
Share on linkedin
Share on twitter
Share on email

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

A Randomized, Multicenter, Double-Blind, Placebo-Controlled, Phase III Clinical Study to Evaluate the Efficacy and Safety of Intrathecally Administered RO7234292 (RG6042) in Patients With Manifest Huntington’s Disease

Phase 3


Researchers at Roche are conducting a large, multi-center Phase 3 trial of a drug designed to reduce symptoms of Huntington’s Disease (HD). The drug binds to the molecules that instruct cells to make huntingtin protein, which causes the neurological symptoms of the disease. By binding to huntingtin, the drug reduces the symptoms.

The clinical trial seeks a large number of patients with active HD who will be assigned to either placebo treatment or the actual drug. Both groups of patients receive a spinal injection of the treatment every 4 weeks or every 8 weeks, with follow up for ~2 years every ~4 months.


  • Be 25 to 65 years of age
  • Have a genetically confirmed diagnosis of Huntington’s Disease
  • Clinical assessment to ensure individual has intact functional independence at baseline to maintain self-care and core activities of daily living (ADLs).


  1. Prescreening tests to confirm eligibility and safety of the patient to participate.
  2. Weekly intrathecal (into the spine) injections every 4 or 8 weeks for many weeks
  3. Follow up every 4 months for at least 2 years


The study site is at 19 different locations nationwide.


Roche Worldwide at 888-662-6728 | [email protected]

Reference Study ID Number BN40423



Hoffmann-La Roche



Or go online:

Subscribe To Our Newsletter

Get updates and learn from the best

More To Explore

Vanquishing the Virus: 160+ COVID-19 Drug and Vaccine Candidates in Development

While President Trump continues to promote antimalarial drugs such as hydroxychloroquine sulfate and azithromycin to treat COVID-19—“What do you have to lose?” he exclaimed during the April 4 Coronavirus Task Force Briefing—the global community of drug discovery researchers and biopharmas is developing a much broader