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| The European trial of ACR16 yields good results |
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The Lighthouse has been following ACR16 since 2005. We provided the first Internet coverage of this promising drug
and have been following its progress ever since. The news from Neurosearch that its European study resulted in significant
improvement in the voluntary and involuntary movement is exciting. Before considering approval of the drug, the FDA
will want to see the results of the U.S. trial which is still enrolling but the European results are a major step on the path
to a new treatment for Huntington's Disease.
ACR16 is a small molecule belonging to a pharmacological class called dopamine stabilizers. It can enhance or inhibit activity
depending on the initial level. In other words, if dopamine activity levels are too high, ACR-16 can decrease them, but if
activity is too low, ACR-16 can increase it. This contrasts with neuroleptics where a reduction in activity of this neurotransmitter
occurs, regardless of initial level.
Dopamine is an important neurotransmitter which plays a role in cognition, mood, attention, learning, motor activity, leep,
and behavior, so targeting it needs to be done carefully. Dopamine appears to play a role in Huntington’s Disease. Dopamine
receptors are progressively reduced with the progression of the disease. In addition, there is some evidence that there is
an abnormal sensitivity to dopamine in the medium spiny neurons affected in HD.
ACR-16 appears to work by strengthening cortical control of the basal ganglia. The striatum, which is part of the basal
ganglia, is the brain's autopilot. Once we have learned a behavior, we need not give it our full attention. The striatum will
control the activity while our cortex is thinking about something else. Once the striatum begins degenerating in HD, the cerebral
cortex loses control of the basal ganglia. This is why HD patients have trouble with multitasking.
The trial tested ACR16 for its ability to improve motor symptoms rather than as a disease modifying treatment. That
will have to be determined later. We at the Lighthouse approve of Neurosearch's strategy of testing ACR16 as a treatment
for symptoms since 1) treatment of symptoms is important for quality of life and 2) the shorter time needed for a trial like
this will get treatments to the HD community sooner.
The good news about ACR16 is cause for celebration. Personally, I am ecstatic.
It's not too late to sign up for the U.S. trial. More information about the trial can be found here: Clinicaltrials.gov
A list of participating sites can be found here: Participating sites for HART
-- Marsha L. Miller, Ph.D.
02-03-2010
NEW CLINICAL TRIALS SET TO BEGIN!
Dear HDTrials Member
We are writing
to inform you of the opportunity to register for the CREST-E Trial. (Creatine Safety, Tolerability & Efficacy in
Huntington’s Disease)
The purpose of the Study is to Purpose: To determine whether high dose creatine can slow
the worsening of Huntington¹s disease by testing its effects on clinical symptoms and quality of life over a 3-year time period.
The effects of creatine on radiologic, blood and urine biomarkers will also be studied and its safety for long-term use will
be assessed.
At the end of this email you will find a list of all of the current Trial Sites. Please contact your local
site to learn if you are eligible to participate in CREST-E and notify members of your family about sites in their area.
Participants needed: 650 individuals 18 years of age or older.
- The participant must have the diagnosis of Huntington’s disease in early or
moderate stages and be able to ambulate (walk without assistance) and not need nursing care. Women must not be pregnant
or become pregnant during the trial.
- It is encouraged – but not required – that each participant have
a family member or partner who can assist in trial participation. You will specifically be asked to provide information
on a person who may be contacted if there is difficulty reaching you.
- If there is a partner or family member who assists you, that person will be asked
to sign a consent form allowing for their completion of a questionnaire regarding their feelings and experiences in caring
for another person.
- The participant must be competent (fully understand) to consent (agree) to be in
the trial. Each participant will be asked to designate a “research proxy” who will help decide whether it
is a good idea for the participant to continue in the trial if the ability to consent is lost during the trial.
- Must be free of unstable psychiatric or medical illness.
- May be taking psychotropic medications including antidepressants and neuroleptics
(or antipsychotics) or drugs for anxiety if dosages have been stable for at least a month before starting the trial. Any changes
to medications that are needed during the trial will be recorded, but the participant may remain in the trial.
- More information on medication requirements can be obtained by a phone call to the
investigator at your study location.
- At designated MRI centers you may be asked to take MRI pictures of your brain.
Trial duration: 3 years
Number of visits and phone contact: There will be sixteen (16) study
visits and eighteen (18) telephone contacts over three years.
Visits and procedures for the participant:
• The first
or screening visit (2-3 hours) includes a medical history and complete physical and neurological exam, blood, urine and electrocardiogram
(EKG) testing. You must be in good health except for Huntington’s, and have subsequent normal lab test results
be able to participate in the trial. If you are accepted into the trial you will be randomized (by a computer “flip
of a coin”) to receive either creatine or placebo powder and scheduled for your first trial visit within the next month.
•
The baseline visit (2-3 hours) includes a brief medical exam. You will be asked about your emotional state and function, and
you will undergo motor (movement) and cognitive (thinking) tests. You will receive your first dose of study drug (either creatine
or placebo) and be watched for any side effects for an hour during this visit. Your will receive packets of study drug (creatine
or placebo powder) at the end of this visit to be taken over the next month. During the month following this visit you
will increase the number of packets of study drug from 3 to 5 packets (half of dose taken twice a day).
• The
next four (4) follow-up visits (1-2 hours) will occur at monthly intervals. This is the “titration” part of the
trial when the dosage of study drug is gradually increased (or titrated) up to a maximum of 8 packets (4 twice a day) or a
number that can be taken without side effects. Each of these visits will include blood, urine and EKG testing.
Blood work during the first follow-up visit will include HD gene testing. Study drug will be given out after each visit
that will last till the next visit. You may be asked to return for blood testing in-between monthly visits.
•
The next 9 follow-up visits occur after reaching stable dosing of study drug. During the remainder of the first year
visits occur at 3-month intervals, and during the final 2 years visits occur every 4 months. About half of these visits will
take between 1–2 hours to complete; the others will take 2-3 hours. All visits require blood, urine and EKG testing.
The longer visits include tests to assess your movement, emotional, mental and cognitive (thinking) functioning.
•
For those who are at MRI centers: Four MRI evaluations will be performed. One at baseline, then at yearly intervals
thereafter.
• Between visits there will be eighteen (18) scheduled phone calls lasting about 5-10 minutes to
check on how well you are doing and if there are side effects.
• A final visit one month after completing participation
in the trial will take 1-2 hours. This visit includes blood, urine and EKG testing.
If a caregiver is taking part
in the study: That person will be asked to complete a questionnaire 4 times during the study to assess their feelings about
taking care of another person. This will occur during the baseline visit and at 3 yearly intervals.
CREST-E Trial Sites:
Please call the nearest site to learn if you are qualified to participate in CREST-E. Please tell them you hard about the
study through HDTrials.org
CA, Irvine
University of California-Irvine
Shari Niswonger
949-824-8116
CA, Sacramento
University of California-Davis
Nicole
Mans
916-734-6697
CO, Littleton
Colorado Neurological Institute
Diane Erickson
303
762-6674
GA, Augusta
Medical College of Georgia
Paula Jackson
706-721-4152
GA,
Atlanta
Emory University School of Medicine
Elaine Sperin
404-728-4786
IA, Iowa City
University of Iowa
Nancy Hale
319-353-4537
IL, Chicago
Rush University Medical Center
Jeana Jaglin
312-942-5003
KS, Kansas City
University of Kansas Medical Center
Carolyn
Gray
913-588-6983
MD, Baltimore University of Maryland
Connie Nickerson
410-328-4349
MA,
Charlestown
Massachusetts General Hospital
Erin Chung
617-724-2227
MO, St. Louise
Washington Univ. School of Medicine
Pat Deppen
314-362-8548
MN, Golden Valley
Struthers Parkinson’s Center
Sarah Lenarz
952-993-5903
MD, Baltimore
University of Maryland School of
Medicine
Michelle Cines
410-328-0157
NC, Durham
Duke University Medical Ctr
Peggy Perry-Trice
919-684-0865
NC, Winston-Salem
Wake
Forest University School of Medicine
Christine O’Neill
336-716-8611
NY, Albany
Albany
Medical College
Mary Eglow
518-262-6611
NY, Manhasset
North Shore-LIJ Health System
Barbara
Shannon
516-562-2905
NY, New York
Columbia University Medical Center
Carol Moskowitz
212-305-5779
NY,
Rochester
University of Rochester
Nancy Pearson
585-341-7500
OH, Cincinnati
University
of Cincinnati
Maureen Gartner
513-558-0018
OH, Columbus
Ohio State University
Allison
Seward
614-688-8672
PA, Philadelphia
University of Pennsylvania
Mary Lloyd
215-829-2365
PA,
Pittsburgh
University of Pittsburgh
Nancy Lucarelli
412-692-4659
TN, Memphis
University of Tennessee Health
Science Center
Nathan Jones
901-448-6180
TX, Houston
Baylor College of Medicine
Alicia Palao
713-798-3974
Ongoing Studies
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