• PDtrials - Parkinson's Disease Clinical Trials
• /Clinical Research 101
• /Educational Webinar
• /Educational Webinar - January 2009

We were not able to accommodate every question that was asked of Ken Getz during the hour-long webinar on January 14th.  Those questions that did not receive a response were reviewed and categorized, and the answers are provided below.

Should you have further questions, please email us at info@pdtrials.org. 

Getting Involved and Getting More Information


Q:  What is the best resource for finding out about Parkinson’s studies that are enrolling, besides asking my doctor?

A:  The www.PDtrials.org website provides a listing of clinical  ClinicalDealing with or based on observation and treatment of people, as opposed to basic science carried out in the laboratory or in animals. studies for Parkinson’s disease (PD) that are currently taking place throughout the US and are seeking participants.

You may also sign up for the monthly PDtrials email newsletter, containing the latest PD trials that are seeking participants as well as the month’s current PD clinical research  Clinical ResearchPatient-oriented research conducted with human participants (or on material of human origin such as tissues and specimens) for which an investigator directly interacts with human participants. This area of research includes: mechanisms of human disease, therapeutic interventions, clinical trials, or development of new technologies. news.  PDtrials also produces a print version of the list of studies featured on the website.  You may sign up to receive these updates here.   

Q:  Is PDtrials the only resource for Parkinson’s clinical trial  Clinical Trial (Clinical Study)A scientific study involving human participants to determine the safety and efficacy of new therapies or new ways of using known treatments. information?

A:  Though www.PDtrials.org is the most up-to-date, comprehensive and patient-friendly website that contains Parkinson’s trial listings and news, there are other resources that you should explore.

For instance, the federal government sponsors websites that provide information on clinical studies and educational resources of all kinds, the most popular being www.clinicaltrials.gov. Other websites that are not disease-specific, such as www.centerwatch.com, are very informative and contain some PD clinical trial listings.

You may also wish to look into online clinical trial resources and listings provided by major Parkinson’s research centers based in hospitals, universities and clinical research institutes. And, investigate other general clinical trial listing services that have sections dedicated to PD.

Q:  I subscribe to PDtrials and have looked for trials that I might participate in, and I haven’t yet seen one that I am right for.  Why is that?

A:  Each trial has its own set of eligibility criteria,  Eligibility CriteriaGuidelines for determining who can qualify to participate in a specific clinical trial. Requiring that all participants share certain characteristics (e.g. age, gender, medical history) ensures that trial results will be due to what is under study and not other factors, helping researchers achieve accurate and meaningful results. which vary greatly from trial to trial.  If you do not meet the eligibility criteria, which often includes age limits, current medications or pre-existing conditions, you would not be an appropriate candidate for that trial.

It’s important to remember that, if you don’t qualify for one trial today, another one may come along tomorrow for which you do qualify.  Try not to get discouraged and to keep looking.  Your participation will be valuable when you find the right trial for you.

Eligibility for Clinical Studies


Q: I have Parkinson’s, but my medication seems to be working and I feel like I’m doing well these days.  So, is now really the right time to participate in a trial?

A:  It’s worth exploring clinical trial participation at any time, in any stage of the disease. There are many studies that you might participate in simply to gain a better understanding of PD, to learn from leading neurologists in the field, and to advance scientific understanding of Parkinson’s. Many studies don’t test experimental treatments at all, but test new exercise programs or study the genetic  GeneticReferring to a condition that is caused by a person's genes (heredity). background of a family with high incidence of PD.

Q:  I’ve heard that trials for Parkinson’s usually want newly diagnosed participants, because they are a "clean slate."  Is that true?

A:  While it’s true that some trials call for participants who are "de novo"  De NovoLiterally Latin meaning “from the beginning”, generally refers to Parkinson’s patients who have not yet begun medication for their disease. or rather have not yet taken medication for their Parkinson’s, not every trial does.  In fact, the vast majority of studies listed on www.PDtrials.org do not call for de novo participants.  You must check the eligibility criteria for each study to understand the requirements of the participants for that study.  Some studies are looking for advanced Parkinson’s patients, and some are looking for participants who do not even have PD. Clinical studies are looking for all kinds of participants; there is never one requirement that applies to all trials.

Q:  Is there a list on which I can register my qualifications?

A:  There is not one central place in the US where you might register your information in order to be contacted to participate in certain studies  for Parkinson’s.  But there are registries at individual institutions in specific locations, such as:

• A Memory Disorders Registry at Cedars- Sinai Medical Center
• Washington Parkinson’s Disease Registry for Washington State’s people with Parkinson’s:  Contact 1-800-329-8387
• Parkinson’s Disease NeuroGenebank at Scripps Clinic Registry (collecting blood samples for genetic research)
  

Clinical Trials:  General Concerns about Participation


Q:  Would being in a clinical trial affect my medical insurance?

A:  No. It is customary that the study sponsor  SponsorOrganizations, medical institutions, foundations, pharmaceutical companies, or federal agencies (e.g. National Institutes of Health, Department of Defense, and Department of Veteran's Affairs) that fund clinical trials. reimburses the study site(s) and physicians for the work they do in the study, and the participant’s insurance is not billed. 

Q:  Are travel expenses always covered by the study?  If they are not, are there any other sources of financial assistance?

A:  Parking and mileage costs are usually covered.  However, if study visits require a participant to be at a study site for a prolonged visit, there are usually reimbursements for "lost time or travel."  Customarily participants are not enrolled in studies if they would need to travel long distances to the site.  There is typically no other financial assistance available to help with study transportation outside of what the sponsor provides.

Q:  Does my doctor have to recommend me for study participation?

A:  You do not need a recommendation from your doctor to enroll in a clinical study.  However, it is a good idea to talk with your physician before enrolling in a study, particularly if the study involves interventions that will require you to alter or discontinue your treatment regimen. 

PDtrials encourages communication between the study participant and his or her doctor.  If you are looking into study participation by doing research on your own, one of the steps should be to discuss the trials you come across with your family physician or neurologist.  NeurologistA physician skilled in the diagnosis and treatment of diseases of the nervous system.

Q:  What is the average length of time that a clinical trial lasts?

A:  Clinical studies can vary greatly in length, from one site visit to several years of participation.  When you inquire about study participation, ask about the length of time that will be required of you, from study enrollment to completion, including any follow-up requirements (post-completion visits or check-ins). 
For other questions you should ask when considering trial participation, click here.

Specific Research Topics


Q:  Are there studies for PD that enroll people who don’t have Parkinson’s?

A:  Yes, there are many studies enrolling people without PD.  If you search by location or other search criteria on PDtrials.org, you will see that some studies are labeled with this symbol  .  This indicates that this study is also, or perhaps only, enrolling people who don’t have Parkinson’s. Often called “healthy participants” these friends and family members of people with Parkinson’s are often needed for genetic studies, or to test the safety of certain interventions on unaffected individuals. See the PD & You section of the PDtrials.org site for more information.

Q:  Are there any clinical trials that study Parkinson’s caregivers?

A:  There are occasional studies that examine the effect that caring for a person with PD has on the caregiver.  Caregivers are also always welcome to seek participation in clinical studies as a "healthy participant" (see the answer to the above question). And often caregivers are very useful in aiding people with PD to look into the possibility of participating in a clinical study and in helping to research their options.

Q:  How close are we to stem cell research for Parkinson’s?

A:  The science of stem cells  Stem CellsImmature cells that go on to develop specialized functions. Scientists work primarily with two types of stem cells. Adult stem cells are immature cells found in specialized tissues, such as the brain or muscles. They might be able to develop into any type of cell found in the tissue from which they originated. Embryonic stem cells are immature cells found in embryos with the potential to develop into a wide variety of specialized cell types. offers great promise for the development of future treatments of disease, including Parkinson’s.   Scientists are still working to understand how stem cells work, and how they can be coaxed into developing into other cells in the body that can be applied to treatments for specific diseases.   So far, most of the clinical applications have been in areas such as bone marrow and blood, but the promise of stem cells  – especially those that are derived from human embryos, and those derived through a new technique known as induced pluripotent stem-cells (IPS) – is much wider. For a layperson’s guide to the science of stem cells, check out a recent article in PDF News and Review.  

Q:  What is involved in clinical studies for Deep Brain Stimulation  Deep Brain Stimulation (DBS)Procedure in which a small, surgically implanted, battery-operated medical device delivers electrical stimulation, and "turns-off" brain regions that produce Parkinson’s symptoms. (DBS)?

A:  All studies, whether they involve an experimental treatment, surgery or other therapy  TherapyAnother word for “treatment”. are conducted in a similar manner. Prospective participants come to a screening  ScreeningPeriod of selection of clinical trial participants based on ELIGIBILITY CRITERIA. visit to determine eligibility to participate, a baseline  BaselinBeginning measurements against which a participant’s progress can be quantified at the end of a study. visit where study specific evaluations are performed prior to actually receiving the study treatment (medication or surgery) and then follow up visits are conducted during the length of the study to collect information and monitor the participants health and safety.

Trust, Accountability and Patient Rights


Q:  How can we address the problem of pharmaceuticals companies suddenly halting trials while the participants are still on the study treatment?  What are the repercussions the companies face when that happens?  And how do they handle the repercussions for the participants?

A:  There is always the possibility that a clinical trial on an experimental treatment will be delayed or terminated.  This is the nature of studying treatments for which there is limited knowledge about safety and effectiveness.  EffectivenessAbility of a drug or treatment to produce a result.   There are many repercussions including how to notify participants quickly; how to safely transition participants off the study treatment and, if necessary, onto another treatment; how to inform participants’ health care providers to ensure that they are engaged in support for the participant; how to close down the trial adequately, comply with regulations and archive study data.

Most pharmaceutical company sponsors have developed procedures to ensure that they notify site personnel and participants as soon as a decision has been made.  And, many sponsors have developed policies for handling clinical trial terminations particularly for protocols studying treatments for very serious illnesses.  Still, there are cases every year where study terminations have not been well-managed and staff and participants are left having to wait for additional information from the sponsor.

Participants can protect themselves from clinical trial termination risk to some extent by asking at the outset of the study what steps the site will take for its participants in the event that a study has to be terminated; and periodically speaking with the investigator and study coordinator during the study to solicit updates on what the sponsor has learned about the experimental treatment.

Q:  When I see trials and research studies advertised in the media, how do I know which ones to trust?

A:  Studies are promoted in different ways, and a study that advertises in the media for participants isn’t necessarily less legitimate than one that doesn’t.  Clinical studies, particularly larger ones for experimental treatments which often enroll hundreds of participants, continually face challenges in obtaining the number of participants needed.  Advertising is just one way for them to get the word out to people outside of the study site’s immediate network. 

You should always carefully research every study you are considering and ask all the necessary questions before enrolling.  See this PDtrials resource for a comprehensive overview of the Questions to Ask.

Q:  Can I stop my involvement in a trial at any time?

A:  Yes.  You do not ever have to stay in a study if you have a change of heart after it has begun. Part of the informed consent  Informed ConsentThe process of providing information to potential study participants to help them decide whether or not to enroll in a specific clinical trial. agreement should contain a clause that allows you to leave at any time without any repercussions.  However, you should always alert the study coordinator as soon as you decide to withdraw from a study.

For complete information on your rights as a participant, visit the Rights and Responsibilities section of the PDtrials website.

Q:  If an adverse event happens to a participant during a trial, are all the other participants informed of this event?

A:  Events that occur during a study fall into two categories: adverse events and serious adverse events. All events which may or may not be related to the study treatment are recorded and followed by the study personnel until resolved. Adverse events would include those events that occur  in the course of our lives such as colds, flu, arthritis, infection,  etc. Only serious adverse events are reported immediately (within 24 hours) to the sponsor and the institutional review board  Institutional Review BoardA committee of physicians, statisticians, researchers, community advocates, and others chosen by an institution to initially approve and periodically review research projects involving human participants. Federal law requires this to ensure protection of participants’ safety, rights and welfare. (IRB)  IRBA committee of physicians, statisticians, researchers, community advocates, and others chosen by an institution to initially approve and periodically review research projects involving human participants. Federal law requires this to ensure protection of participants’ safety, rights and welfare. . Serious adverse events include death, an event considered life threatening, requires hospitalization, events which result in persistent or significant disability or incapacitation or is a congenital anomaly/birth defect.  Any new information that evolves during the study is reported to participants.

Q:  In a blinded study, are the participants ever informed, even after the trial, whether they received the investigational treatment or a placebo?  PlaceboAn inactive substance or procedure (often a pill, liquid, or powder) that has no biological effect. In clinical trials, experimental treatments are often compared with placebos to assess the treatment's effectiveness. In some studies, the participants will be assigned to a control group and will receive a placebo instead of an active drug or treatment.

A:  Yes, they are informed. After the "database is locked" (when the data has been cleaned for errors or mistakes) the study data is analyzed and reported. At that time the investigator is informed of the study randomization  RandomizationA method based on chance by which study participants are assigned to a treatment group (arm). Randomization minimizes the differences among groups by equally distributing people with particular characteristics among all the trial arms. The researchers do not know which treatment is better. (which patient was assigned to which treatment or placebo) the investigator informs the participants of this information.