Consent to Participate in a Research Study
Characterization of Familial Myopathy, Paget's Disease of
Bone and Dementia
WHY ARE YOU BEING INVITED TO TAKE PART IN
THIS RESEARCH?
You
are being invited to take part in a research study about inclusion body
myopathy, Paget's disease of bone, and frontotemporal dementia (IBMPFD). You are being invited to take part in this
research study because you are a member of a family with someone who suffers
from IBMPFD. You may have symptoms of
IBMPFD, carry the gene for IBMPFD but have no symptoms, or be a member of a
family with IBMPFD but not carry the gene yourself, and still participate. If you volunteer to take part in this study,
you will be one of about 300 people to do so nationally and one of 100 at the
WHO IS DOING THE STUDY?
The
person in charge of this study is Dr. Charles Smith of the department of
Neurology. There may be other people on
the research team assisting at different times during the study. Dr. Virginia Kimonis (
WHAT IS THE PURPOSE OF THIS STUDY?
The purpose of this
study is to collect information and specimens (blood, urine, and samples of
skin and muscle) for research into the causes and possible treatments for
IBMPFD. Samples of blood, skin, and
muscle will be used for genetic research.
By doing this study, we hope to learn enough about the fundamental
causes of IBMPFD to devise treatments for it.
At present, there is no known effective treatment or prevention for
IBMPFD.
WHERE IS THE STUDY GOING TO TAKE PLACEAND
HOW LONG WILL IT LAST?
The research procedures
will be conducted at the
WHAT WILL YOU BE ASKED TO DO?
You will come to the
GCRC in the morning and have some breakfast.
Registered nurses will draw 45cc (three tablespoons) of blood from your
arm and collect a urine specimen, taking about 30 minutes. You will be sent to the Radiology department
for an X-ray survey of your bones taking about an hour. Then you will have an electromyogram (EMG) of
an arm or leg, performed by a physician specialist in muscle disease, for an
hour. During the EMG the doctor will put
a needle into the muscle several times to record electrical activity. You will be asked to relax and tense the
muscle during this recording. This specialist
will also take a punch biopsy of the skin (about the size of the end of an
unsharpened pencil with the depth of a dime) and a biopsy of a muscle. The biopsy of muscle is done by putting a
needle into the muscle and removing a sample about the size of a pencil eraser. Before doing the biopsies, your skin over the
biopsy site will be numbed with a local anesthetic. The biopsies together take about 30
minutes. An electrocardiogram (EKG) will
be done, taking 30 minutes. Electrodes
are pasted on the bare chest before running the EKG, and removed
afterward. You will have a neurologic
examination with testing of muscle strength, reflexes and sensation by a
trained Neurologist; this examination will take 45 minutes. There will be a break for lunch in the middle
of the day. There will be sometime
waiting between tests when you can relax and rest.
All of the tests
(blood-draw, EMG, EKG, X-ray, examination and biopsies) are standard clinical
procedures, but they are being done for research purposes only. None of the procedures are experimental. They are done routinely in clinical practice.
Here is a chart of the
procedures that will be done and how long each is expected to take:
|
Test |
Expected Duration |
|
Blood Drawing & Urine Sample |
30 minutes |
|
Skeletal X-ray Survey |
60 minutes |
|
Electromyogram (EMG) |
60 minutes |
|
Biopsy Skin and Muscle |
30 minutes |
|
Electrocardiogram (EKG) |
30 minutes |
|
Physical Examination |
45 minutes |
|
Total |
4 hours & 15 minutes |
ARE THERE REASONS WHYYOU
SHOULD NOT TAKE PART IN THIS STUDY?
Only members of families
with at least one person suffering from IBMPFD are eligible for this
study. You must also be taking part in
the imaging study (Imaging Brain Structure/Function in Pre-symptomatic FTD, IRB
# 02-0237-F1V) to be eligible. You must
be over 20years of age, and not older than 89 years. If you have a known bleeding disorder such as
hemophilia, or you are taking coumadin or a combination of aspirin and
clopidogrel (Plavix), you cannot participate.
You should not take aspirin within seven days of your visit, because it
increases the risk of bleeding during the EMG or biopsy.
WHAT ARE THE
POSSIBLERISKS AND DISCOMFORTS?
Here is a chart of the
possible risks and discomforts you might experience during your study visit:
|
Test |
Potential Risk or Discomfort |
Likelihood |
Severity |
|
Blood Drawing |
Pain of needle stick |
Usual |
Mild |
|
Blood Drawing |
Bleeding or Bruising |
Occasional |
Mild |
|
Blood Drawing |
Needle site infection |
Rare |
Moderate |
|
Blood Drawing |
Fainting |
Occasional |
Mild |
|
Skeletal Survey |
Lying on hard table |
Occasional |
Mild |
|
Electromyogram (EMG) |
Pain of needle |
Usual |
Mild-Moderate |
|
Electromyogram (EMG |
Bleeding or Bruising |
Occasional |
Mild |
|
Electromyogram (EMG |
Needle site infection |
Rare |
Moderate |
|
Skin Biopsy |
Pain of biopsy |
Occasional |
Very Mild |
|
Skin Biopsy |
Bleeding or Bruising |
Occasional |
Mild |
|
Skin Biopsy |
Biopsy site infection |
Rare |
Moderate |
|
Muscle
Biopsy |
Pain of biopsy |
Usual |
Mild-Moderate |
|
Muscle
Biopsy |
Bleeding or Bruising |
Occasional |
Mild |
|
Muscle
Biopsy |
Biopsy site infection |
Rare |
Moderate |
|
Electrocardiogram |
Skin
allergy to electrode paste |
Occasional |
Mild |
|
Physical
Examination |
Fatigue |
Occasional |
Mild |
There is a risk that
despite our best efforts your identity might become known inadvertently. There is also a risk of finding out your own
genetic status accidentally during this research. Accidental breaches of confidentiality might
impact your insurability, employability, family relationships, immigration
status, future family planning, paternity suits, or cause social
stigmatization. Psychological stress
could come to you or your family members from someone learning your genetic
results by mistake.
In addition to the risks
listed above, you may experience a previously unknown risk or side effect.
WILL YOU BENEFIT
FROMTAKING PART IN THIS STUDY?
There is no guarantee
that you will get any benefit from taking part in this study. We cannot and do not guarantee that you will
receive any personal benefits from taking part in this study. Your willingness to take part, however, may,
in the future, help doctors better understand and/or treat others who have your
condition.
DO YOU HAVE TO TAKEPART
IN THE STUDY?
If you decide to take
part in the study, it should be because you really want to volunteer. You will not lose any benefits or rights you
would normally have if you choose not to volunteer. You can stop at any time during the study and
still keep the benefits and rights you had before volunteering.
IF YOU DO NOT WANT
TOTAKE PART IN THE STUDY, ARE THERE OTHER CHOICES?
If you do not want to be
in the study, there are no other choices except not to take part in the study.
WHAT WILL IT COST YOUTO
PARTICIPATE?
You and/or your
insurance company, Medicare or Medicaid will be responsible for the costs of
all care and treatment you receive during this study that you would normally
receive for your condition. These are
costs that are considered medically reasonable and necessary and will be part
of the care you receive if you do not take part in this study.
In this study we are not
assuming responsibility for your ongoing clinical care and treatment. However, if there is an indication of an
unrecognized or untreated medical disorder, such as Paget's disease of bone, we
would let you know and, with your permission, contact the physician of your
choice to arrange for treatment.
Biphosphonates, for example, can be used to treat active Paget's disease
of bone.
The
Cost for
your travel, meals, taxi transportation and parking, and hotel stay will be
paid by the imaging grant ("Imaging Brain Structure/Function in
Pre-symptomatic FTD"; R01AG-25159, Charles D. Smith MD principal
investigator).
Costs of
all of the procedures described under this consent will be borne by the GCRC
grant ("
Under NIH
rules, you will not be reimbursed for alcohol in any form, such as beer or
wine, even if it is consumed during a meal covered by these protocols. You will also not be reimbursed for side
trips, incidentals, cell phone or shopping expenses resulting from your stay in
Medicare or Medicaid will pay
medically necessary costs (if you have any questions regarding
Medicare/Medicaid coverage you should contact Medicare by calling1-800-Medicare
(1-800-633-4227) or Medicaid at 1-800-635-2570.
A
co-payment/deductible from you may be required by your insurer or
Medicare/Medicaid even if your insurer or Medicare/Medicaid has agreed to pay
the costs.
WHO WILL SEE
THEINFORMATION THAT YOU GIVE?
We will keep private all
research records that identify you to the extent allowed by law.
Your information will be
combined with information from other people taking part in the study. When we write about the study to share it
with other researchers, we will write about the combined information we have
gathered. You will not be identified in
these written materials. We may publish
the results of this study; however, we will keep your name and other
identifying information private.
We will make every
effort to prevent anyone who is not on the research team from knowing that you
gave us information, or what that information is. For example, your name will be kept separate
from the information you give, and these two things will be stored in different
places under lock and key. You should
know, however, that there are some circumstances in which we may have to show
your information to other people. For
example, officials of
the National Institutes of Health or the
DNA extracted from blood
and RNA extracted from muscle will become the property of Dr. Kimonis'
laboratory (vkimonis@uci.edu; (714) 456-2942). She will be able
to link genetic information from these samples to your name and other
identifying information about you. You
have the right to ask Dr, Kimonis to share genetic findings from testing of the
sample. In this event, she may ask to
have genetic results confirmed by a CLIA-approved laboratory test. She will arrange to have the appropriate
genetic counseling available when this information is shared with you.
Cell lines made from
your skin and blood will become the property of the Coriell Institute for
Medical Research. The Coriell Institute
is an independent, not for profit research organization supported by the
National Institutes for Health (http://www.coriell.org/). Genetic material from these cell lines will
be made available to qualified genetic researchers in laboratories around the
world. When this material is shared with
researchers, it is not linked to your name or other identifying information
about you. Researchers will be given
medical information concerning the sample, but your identity cannot be
discovered from them. You will not be
able to find out genetic testing results from research on these samples. If commercially valuable products result from
research on your samples, you will not share in any of the profits. None of the investigators, Drs Smith Kimonis
and
You have the right to
withdraw your consent at any time, and have the cell lines made from your
samples destroyed. You can do this by
contacting Dr. Smith, who will communicate this decision to the Coriell
Institute. However, we would not be able
to recover and destroy material already shared with other researchers by
Coriell, only the existing cell lines established at Coriell Institute.
CAN YOUR TAKING PARTIN
THE STUDY END EARLY?
If you decide to take
part in the study you still have the right to decide at any time that you no
longer want to continue. You will not be
treated differently if you decide to stop taking part in the study.
The individuals conducting the study
may need to withdraw you from the study.
This may occur if you are not able to follow
the directions they give you, if they find that your being in the study is more
risk than benefit to you, or if the agency funding the study decides to stop
the study early for a variety of scientific reasons.
WHAT HAPPENS IF YOUGET
HURT OR SICK DURING THE STUDY?
If you believe you are
hurt or if you get sick because of something that is due to the study, you should call
Dr. Charles Smith at 859-323-1113 immediately.
If you are unable to contact him, call Barbara Martin, the study
coordinator at 859-323-0494. Dr. Smith
will determine what type of treatment, if any, that is best for you at that
time.
It is important for you to
understand that the
Medical costs that
result from research related harm can not be included as regular medical
costs. Therefore, the medical costs
related to your care and treatment because of research related harm will be
your responsibility. Your insurer may agree to pay those
costs (you should ask your insurer if you have any questions regarding your
insurer’s willingness to pay under these circumstances).
Medicare
or Medicaid will pay medically necessary costs (if you have any questions
regarding Medicare/Medicaid coverage you should contact Medicare by
calling1-800-Medicare (1-800-633-4227) or Medicaid 1-800-635-2570.
A co-payment/deductible from you maybe required by your insurer or Medicare/Medicaid even if your insurer or Medicare/Medicaid has agreed to pay the costs).
WILL YOU RECEIVE
ANYREWARDS FOR TAKING PART IN THIS STUDY?
You will not receive any
rewards or payment for taking part in the study.
WHAT IF YOU HAVEQUESTIONS,
SUGGESTIONS, CONCERNS, OR COMPLAINTS?
Before you decide
whether to accept this invitation to take part in the study, please ask any
questions that might come to mind now.
Later, if you have questions, suggestions, concerns, or complaints about
the study, you can contact the investigator, Dr. Charles Smith
at859-323-1113. If you have any
questions about your rights as a volunteer in this research, contact the staff
in the Office of Research Integrity at the
WHAT ELSE DO YOU NEEDTO
KNOW?
The
study is funded by the National Institutes of Health under three grants:
(1)"Imaging Brain Structure/Function in Pre-symptomatic
FTD";R01AG-25159, Charles D. Smith MD principal investigator, (2) the
"General Clinical Research Center"; M01 RR02602; Jay Perman, MD
principal investigator, and (3) "Genetic basis of myopathy with Paget's
disease of bone"; R01AR050236, Virginia Kimonis MD principal
investigator. Dr. Kimonis is the Chief
of the Genetics and Metabolism Division, Department of Pediatrics, University
of California Medical School,
You will be told if any
new information is learned which may affect your condition or influence your
willingness to continue taking part in this study. For this purpose we will keep your contact
information in a secure file on a passworded computer server.
_____________________________________________ ____________
Signature
of person agreeing to take part in the study Date
_____________________________________________
Printed
name of person agreeing to take part in the study
_____________________________________________ ____________
Name
of [authorized] person obtaining informed consent Date
_________________________________________
Signature
of Investigator