Parkinson’s Disease (PD) has a monogenic cause in about 5% of patients. 1-2% of PD patients have a mutation in the LRRK2 gene, thought to result in LRRK2 overactivity. The ability of LRRK2 inhibitors to slow disease progression in such patients is currently being investigated. The penetrance of LRRK2 mutations is thought to be 30-40%.

GBA mutations occur in about 10% of PD patients and confer a significant risk of PD development. Several different strategies are being investigated to slow disease progression in GBA mutation patients.

Rocky Mountain Movement Disorders Center is currently conducting interventional and observational clinical trials for patients with LRRK2 or GBA mutations. There is currently no proven therapy to slow disease progression in any subgroup of PD patients, but we are most likely to be successful in those with a known genetic mutation for whom we can prove target engagement with a gene-targeted specific therapy.

Statistically, less than 1% of the US population participates in clinical trials, yet 72% stated they would participate if recommended by their doctors. Together, we can leverage this large gap by adopting a more inclusive approach to clinical research as a care option (CRAACO).

If you have patients that might be interested in participating in clinical research who have LRRK2 or GBA mutations, please contact our research coordinator: Dominick DeMarsico

Call Us: (303) 357-5447
Email Us: [email protected]

Please note that we do not wish to infringe upon your autonomy regarding what is best for your patient. Patients will of course be returned to their physicians for ongoing care once the have completed their participation in any clinical trials. 

About the Principal Investigator (PI)
Rajeev Kumar, MD

Dr. Kumar has extensive experience and expertise in treating and researching Parkinson’s disease, Huntington’s disease, Dystonia, and many other debilitating movement disorders. Dr. Kumar is trained in neurology at the Mayo Clinic and in movement disorders with Dr. Anthony Lang at the University of Toronto. He currently serves at the Medical Director of Rocky Mountain Movement Disorders Center, CenExel Rocky Mountain Clinical Research and the Huntington’s Society of America Center of Excellence associated with the Movement Disorders Foundation.

Rocky Mountain Clinical Research (RMCR) is one of the busiest movement disorders clinical trial research centers in the world, conducting Phases 1-3 trials in Parkinson’s disease, atypical parkinsonian disorders, Huntington’s disease, Essential tremor, Dystonia, and other movement disorders.

Rocky Mountain Movement Disorders Center has been caring for patients from throughout the United States, with hypokinetic and hyperkinetic movement disorders for more than 15 years. RMMDC offers comprehensive and customized treatment plans for our patients to have the highest quality of life and works collaboratively with CenExel RMCR.

To find treatments and ultimately a cure for Parkinson’s disease, patients need to volunteer for clinical trials. Many trials fail, and most are delayed because of limited patient participation. All clinical trials need volunteers.

30% of clinical trials fail before they begin because they do not have enough volunteers.
85% of clinical trials are delayed because it takes a long time to find volunteers.
100% of clinical trials need volunteers to help find treatments and a cure for PD.

Clinical Trials FAQS

  • Overview: Clinical trials are done with human volunteers to add to medical knowledge to try and find new ways to treat, prevent, or detect diseases. They are a way to test if new investigational medications are safe and effective in treating the disease they are intended for. There are two types of clinical trials-interventional and observational.
  • Types of studies: Interventional studies are what people typically think of when thinking of research. Individuals are given a certain intervention (medication, surgery, etc.) to see if it is helpful. Observational studies gather important information about diseases and often look at cause and effect relationships. Patients in observational studies are not offered any new treatment options.

The idea for a clinical trial usually starts in a laboratory. Researchers first test their new product with animals to see if it does what they hope. They look to see if it is safe and helpful. Once this is done, the drug can be tested in humans to see if we get the same results.

  • Sponsor: Every study is funded by an organization such as a pharmaceutical company, federally funded agencies (such as NIH), or an individual or organization. They are referred to as the sponsor of the study. The sponsor makes a lot of decisions about the study and chooses sites (or locations) where the study will be performed.
  • Site: There are many locations where the study can be performed. At each site, there is a principal investigator (PI), who is usually a doctor, and study coordinators conduct the study. While in a study, participants will generally see the same study coordinator and provider throughout the study. Most of the study tasks will be done with the study coordinator, and they will be the primary contact throughout the study.
  • Protocol: Each study has a written out plan of what everyone conducting the study needs to do; this is called the protocol. The protocol is written to protect the participants’ health and answer a particular research question. The medication and dosages are reviewed in the protocol as well as who is eligible for the study. The protocol also outlines what needs to be done at each visit and what period they need to be done in.
  • IRB review: Studies are reviewed by an independent committee called the Institutional Review Board (IRB). The IRB reviews the protocol and study materials to confirm that, in their eyes, the risk is worth the potential benefit of the study. The primary goal of the IRB is to ensure the rights of the participants are maintained and that the study is conducted ethically.
  • FDA: The Food and Drug Administration (FDA) also provides oversight to interventional studies to ensure that volunteers’ rights and welfare are maintained and that the data is accurate. The FDA guides on what should be done during a study. Once a study is completed, the sponsor can submit their data to the FDA to be reviewed. The FDA decides if there is enough evidence that the investigational medication is safe and effective to be approved and prescribed.
  • Phase 1: This phase of studies is done with a small number of patients, typically 20-100 people, either healthy volunteers or people with the disease. Phase 1 studies are used to evaluate the safety of the drug, determine a safe dose, and look at side effects. This phase does not look at the effectiveness of the drug at all. The FDA states that approximately 70% of drugs move on from this phase.
  • Phase 2: Phase 2 studies are done to determine the effectiveness and further look at the safety of the investigational product. This phase of the study is done in up to several hundred patients with the disease. The FDA states that approximately 33% of drugs move to the next phase.
  • Phase 3: In phase 3 studies, 300-3000 patients with the disease participate to further look at the safety and efficacy of the investigational product. The FDA states that approximately 25-30% of drugs move to the next phase.
  • Phase 4: These studies with several thousands of patients and are done after the drug has been approved by the FDA to provide further long-term data including risks, benefits, and optimal use.

Not everyone can, or has the desire, to participate in clinical trials, but finding volunteers is very important. A drug may look promising in animal models, but it needs to be tested in humans to ensure that it is safe and effective. New treatment options cannot be approved without clinical trials. One of the biggest obstacles in getting new medications approved is recruitment. Sponsors consistently have challenges getting enough patients into their studies in the time that they had predicted. This is costly, delays therapy, and can discourage further clinical research.

  • Hope: many patients find that working towards new treatment options gives them hope; not only for themselves but also for future generations.
  • Medical attention: In clinical research, patient safety is the primary concern. Safety is constantly being monitored, and particpants will be made aware of any concerns that may arise. If, during a trial, we find that we cannot treat a patient adequately, or their safety is jeopardized, they will be removed from the study. Additionally, the participant will have more frequent visits with doctors who are monitoring their condition while participating in the study.
  • New options: When participating in a clinical trial, patients have the chance of receiving new, cutting edge therapies that are not currently available. Since this is an investigational product, it cannot be promised that this will help the condition. Additionally, in Phases 2 and 3 studies some patients do not receive study medication during the study. Some participants receive placebo (a sugar pill) so that they can be compared to the participants receiving the investigational product to see if it does make a difference. Many studies will give patients who complete the study the opportunity to definitely receive the investigation product without a chance of placebo. This is called an open-label.

Inclusion/Exclusion: Each study has criteria that the participants must meet called inclusion/exclusion. These include things such as age, gender, disease stage, medical history, and medications that they are currently taking. Inclusion/exclusion criteria are not created to exclude certain patients from research studies, but to ensure that participants are safe and that the data is precise.

  • Time: Each clinical trial requires the volunteers time, but the requirement for each study is different. Before enrolling into a clinical trial, it is important to know how much time will need to be committed to the study.
  • Participants should personally weigh the risks and benefits involved in the study and make sure that they feel comfortable.
  • Each study has different risks associated and should be referenced from the Informed Consent Form for known potential side effects.
  • Since these are investigational treatments and medications there may be unknown side effects.
  • It is important to consider other treatment options available because while in the trial participants maybe limited to other treatment options. However, if another treatment is clearly better at any time during the trial, the doctor will take them out of the trial to provide them with the best possible care.
  • Many clinical trials do not help the patients involved.

Informed consent: The informed consent document is created to ensure that each patient completely understands the study and makes an informed decision about their participation. The consent form contains information about the risks and benefits, the purpose of the study, the duration, and the procedures.

If interested in a study, each participant will be given a consent form to review. It is encouraged that patients take time to read this document fully and ask any questions that they have during their review. Once a qualified participant reviews the consent and decides that they would like to enroll in a particular clinical trial, they will be brought to the site.

The first visit will begin by reviewing the informed consent form with the research staff. If the volunteer decides to participate, the informed consent document will be signed at this time.  If a patient is cognitively unable to sign for themselves, they can verbally agree to the study and their legally authorized representative can sign the consent form on their behalf. The informed consent is not a contract; patients are free to withdraw from the study at any time.  Informed consent is an ongoing process and the research staff should ensure that one wants to continue to participate throughout the study.

  • Confidentiality: Confidentiality is important to patients and researchers during clinical trials. When participating in a clinical trial, volunteers are given a random number so that their name is not associated with the data contributed throughout the study. As with a regular doctor, researchers are required to be HIPAA compliant.
  • Drug vs placebo: Most phase 2 and 3 studies are randomized. This means that each patient is randomly assigned to get one of the dosages of the investigational product or placebo. Generally, neither the subject nor the doctor will know which was randomized to during the trial. Randomization creates groups that are as alike as possible. This is important in clinical trials because it allows the data from the control group to be compared to the group on the study medication. This comparison provides the best way to prove the effectiveness of a new medication.
  • Visits: Each study differs in how many visits there are, the lengths of the visits, and what is done at each visit. During the study, partcipants will be required to come to the research site and perform the procedures outlined in the informed consent document; this generally includes safety measures, like blood draws and vitals, and questionnaires to help determine if the investigational product is working.
  • Communication: While participating in a clinical trial, participants must be open and communicate with the research coordinator. The research coordinator needs to be made aware of any health changes; even if the volunteers do not think it is related to the study medication. It is also important that  any medication changes are communicated while receiving an investigational product. This is for participant safety to ensure that none of the other medications interact.

The Common Rule is a federal policy regarding Human Subjects Protection that applies to 17 Federal agencies and offices. The main elements of the Common Rule include: Requirements for assuring compliance by research institutions, requirements for researchers’ obtaining and documenting informed consent, and requirements for Institutional Review Board (IRB) membership, function, operations, review of research, and record keeping. The Common Rule also includes additional protections for certain vulnerable research subjects: Subpart B provides additional protections for pregnant women, in vitro fertilization, and fetuses, Subpart C contains additional protections for prisoners, and Subpart D does the same for children.

FDA Good Clinical Practice regulations are a standard for the design, conduct, performance, monitoring, auditing, recording, analysis, and reporting of clinical trials in a way that provides assurance that the data and reported results are credible and accurate and that the rights, safety, and well-being of trial subjects are protected.