HD Insights
A H u n t i n g t o n d i s e a s e r e s e a r c h
Issue N
o
8 -- Fall 2014
p e r i o d i c a l
TM
Copyright © Huntington Study Group 2014. All rights reserved.
The largest randomized, controlled trial ever conducted in HD has been halted for futility. On August 9, 2014, the Huntington
Study Group (HSG) reported that the ongoing 2CARE study, a phase 3 trial of Coenzyme Q10 (CoQ
10
) in HD (see HD Insights,
Vol. 6), was stopped by the National Institutes of Neurological Disorders and Stroke on July 14, 2014, after an interim analysis
conducted by the Data and Safety Monitoring Board found that it would be "very unlikely" to see a significant effect of 2400 mg
CoQ
10
compared to placebo by the scheduled end of the trial.
1
The results, while disappointing, are consistent with prior trials
of CoQ
10
for neurological disorders such as Parkinson disease, in which the QE3
phase III trial of CoQ
10
was also halted prematurely for futility.
2
The 2CARE study was a randomized, double-blind, placebo-controlled study
conducted by the HSG under the direction of Merit Cudkowicz, MD, MSc, from
Massachusetts General Hospital, Michael McDermott, PhD, from the Biostatistics
department at the University of Rochester, and Karl Kieburtz, MD, MPH, Director
of the University of Rochester's Clinical and Translational Sciences Institute.
Beginning in March 2008, the trial enrolled 609 individuals with HD in 48 sites in
Australia and North America. Data collection was scheduled to end in August
2017.
Despite the failure of CoQ
10
, the study's leaders report that the data collected in
the study will certainly prove useful in future research. Dr. Merit Cudkowicz commented that, "We will make this dataset
available to all HD researchers… to mine for new ideas about HD. [This] is the longest follow up dataset from any clinical trial
and should lead to new insights and directions." Dr. Karl Kieburtz agreed, saying, "While CoQ
[10]
does not appear useful in HD,
the work itself is not 'futile' since we expect to have learned a great deal about HD progression."
Largest HD Clinical Trial Halted
Meet the Investigator
Dr. Hoa Nguyen shares his work
on the BACHD rat model and
with predictive testing for HD
in Germany.
Pages 4-6
Animal Models of HD
Dr. Mahmoud Pouladi
discusses ongoing
research on animal
models of HD.
Page 2
MDS 2014 HD Highlights
The June 2014 MDS
Congress in Stockholm,
Sweden provided a glimpse
into the promising future of
HD research.
Page 11
Meet the Compound:
Olesoxime
Developed for use in
neurodegenerative
illnesses, Trophos's lead
compound shows promise
for HD.
Page 6
Repair-HD
A group of European
investigators is taking on
translation of stem cell
therapy for HD.
Page 9-10
1
huntington-study-group.org/LinkClick.aspx?fileticket=LurnlLVT3rw%3d&tabid=95
2
ninds.nih.gov/disorders/clinical_trials/CoQ10-Trial-Update.htm
Coenzyme Q
10
"very unlikely" to show significant benefit in HD in 2CARE study
"We will make this
dataset available to
all HD researchers…
to mine for new ideas
about HD."
HD Insights, Vol. 8 1