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. 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 University of Kentucky.

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 and Dr. Giles Watts are other investigators in the study.

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. By doing this study, we hope to learn enough about the fundamental causes of IBMPFD to devise treatments for it.

WHERE IS THE STUDY GOING TO TAKE PLACE AND HOW LONG WILL IT LAST? 

The research procedures will be conducted at the General Clinical Research Center, 5th Floor, University of Kentucky Medical Center, once during the study.This facility is called the GCRC for short. The visit will take a full day. We will follow up with email, standard mail or phone contact with you yearly for the indefinite future.

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. You will also have an echocardiogram which records heart activity using an ultrasound process. The technician will apply a colorless gel to your chest and use a handheld piece of equipment to take pictures of your heart. This process will last approximately 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 some time 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

Echocardiogram

30 minutes

Physical Examination

45 minutes

Total

4 hours & 15 minutes

ARE THERE REASONS WHY YOU 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 Presymptomatic FTD, IRB # 02-0237-F1V) to be eligible. You must be over 20 years 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 POSSIBLE RISKS 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(EKG)

Skin allergy to electrode paste

Occasional

Mild

Echocardiogram

Skin allergy to gel or paste

Rare

Mild

Physical Examination

Fatigue

Occasional

Mild

In addition to the risks listed above, you may experience a previously unknown risk or side effect.


WILL YOU BENEFIT FROM TAKING 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 TAKE PART 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 DONíT WANT TO TAKE 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 YOU TO 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 University of Kentucky may not be allowed to bill your insurance company, Medicare or Medicaid for the medical procedures done strictly for research.

Cost for your travel, meals, taxi transportation and parking, and hotel stay will be paid by the imaging grant ("Imaging Brain Structure/Function in Presymptomatic 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 ("General Clinical Research Center"; M01 RR02602; Jay Perman, MD principal investigator) and by an NIH grant ("Genetic basis of myopathy with Paget's disease of bone"; R01AR050236, Virginia Kimonis MD principal investigator). This includes meals and snacks provided to you at the GCRC.

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 Lexington for the study.

Medicare, or Medicaid will pay medically necessary costs ( if you have any questions regarding Medicare/Medicaid coverage you should contact Medicare by calling 1-800-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 THE INFORMATION 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 Food and Drug Administration, the National Institutes of Health, or the University of Kentucky may need to examine your records according to regulations and applicable law.

CAN YOUR TAKING PART IN 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 YOU GET 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 University of Kentucky does not have funds set aside to pay for the cost of any care or treatment that might be necessary because you get hurt or sick while taking part in this study. Also, the University of Kentucky will not pay for any wages you may lose if you are harmed by this study.

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 insurerís 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 calling 1-800-Medicare (1-800-633-4227) or Medicaid 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).

WILL YOU RECEIVE ANY REWARDS FOR TAKING PART IN THIS STUDY?

You will not receive any rewards or payment for taking part in the study.

WHAT IF YOU HAVE QUESTIONS, 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 at 859-323-1113.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 University of Kentucky at 859-257-9428 or toll free at 1-866-400-9428. We will give you a signed copy of this consent form to take with you. The GCRC also has a designated subject advocate for you, who can be reached at 859-323-5049, ext. 230.

WHAT ELSE DO YOU NEED TO KNOW?

The study is funded by the National Institutes of Health under three grants: (1) "Imaging Brain Structure/Function in Presymptomatic 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.

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.

 

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Signature of person agreeing to take part in the study†††††††††††††††††† ††††††††† Date

 

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Printed name of person agreeing to take part in the study

 

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Name of [authorized] person obtaining informed consent††††††††††††††† ††††††††† Date

 

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Signature of Investigator†††††††††††††