2CARE

2CARE (Coenzyme Q10 in Huntington’s Disease) Study Update

September, 2012

Enrollment for the 2CARE study was closed on 25 July 2012, at which time the 609th participant joined the study. The study team has met the challenge of completing enrollment for the largest and longest clinical trial in Huntington disease (HD) to date. Forty-eight sites in North America and Australia contributed to the enrollment of these participants. The study will continue until the last participant completes the study, projected in mid-2017.

The 2CARE study involves collaboration of individuals with HD, caregivers or ‘study buddies’, along with the investigators, coordinators and research staff at the participating sites.

We have come a long way since the first enrollment in April of 2008, and none of our success would have been possible without the commitment and participation of those enrolled in 2CARE. We realize that not everyone can find time to be involved in clinical trials, but thanks to the willingness of those who can, we will be able to learn whether a high dosage of coenzyme Q10 (CoQ) is effective in treating HD.

A very important requirement of being able to learn whether CoQ is effective in slowing the progression of functional decline is that we have enough people remaining in the study through the entire five years. We encourage all enrolled participants to stay committed to the study as much as possible. Remember, HD is a slowly progressive disease, so it will take time to know if there are effects of the study drug.

Beginning in the spring of 2013, we will begin to see some of the earlier enrolled participants complete their five years of participation. We deeply thank all the patients and families who are part of the study, as well as those who have made it possible in so many ways– the National Institute of Neurological Disorders and Stroke, patient advocacy organizations, site investigators, coordinators, and members of the project team.

2CARE Principal Investigators and Study Team of the Huntington Study Group

Announcement of 2CARE Early Study Closure

August 2014

 The National Institute of Neurological Disorders and Stroke (NINDS) stopped its study of coenzyme Q10 for the treatment of Huntington’s disease (HD) on July 14, 2014. The study (2CARE), conducted by the Huntington Study Group (HSG), was stopped for futility. The NINDS and the HSG acted on the recommendation of the study’s independent Data and Safety Monitoring Board (DSMB). Following the most recent DSMB review of the study data, an interim analysis was conducted that showed that, given the current data, it would be very unlikely (less than a 5 in 100 chance) to see a statistically significant benefit of active treatment (coenzyme Q10, 2400 mg/day) over placebo at the scheduled end of the trial. The DSMB also noted a higher number of deaths in the coenzyme Q10 group (7%) in comparison to the placebo group (4%); most of these deaths appeared to be related to HD, which is a severe, progressive neurological disorder. Although this number may have been due to chance, and was not statistically significant, the DSMB noted it in their decision. Site investigators and coordinators have informed participants of the study closure and have encouraged each participant to schedule a final visit to the clinic.

The 2CARE study enrolled 609 research participants with early Huntington’s disease from 48 sites throughout North America and Australia. Participants were randomized to receive either 2400 mg per day of active coenzyme Q10 or matching placebo. The Principal Investigators and the Huntington Study Group are committed to conducting a detailed analysis of the complete data set from the 2CARE study and to disseminating the results through the scientific review process. Even though coenzyme Q10 was not found to be helpful, the study amassed a large amount of longitudinal clinical data which will provide useful information on HD for clinical studies. The study also demonstrated the feasibility of conducting such a large controlled clinical trial in Huntington’s disease. The research team is indebted to the participants for their time and dedication to this study.

S:\RSRB\Watermarking\August 11, 2014\RSRB 13052.Public release early study closure 8 August 2014 FINAL.doc