Research

Understanding Clinical Trials

Choosing to participate in a clinical trial is an important personal decision. The frequently asked questions (FAQs) below and linked pages provide detailed information about clinical trials. In addition, it is often helpful to talk to a physician, family members, or friends before deciding to join a trial. For answers to your specific questions, contact the study research staff or one of our neurosurgeons.

What Are Clinical Trials?

A clinical trial is a research study in human volunteers to answer specific health questions. Carefully conducted clinical trials are the safest and fastest way to the discovery of effective treatments and therefore new ways of improving health.

Clinical trials are conducted according to a plan called a protocol. The protocol describes what types of patients may enter the study, the schedules of tests and procedures, the drugs and dosages, and the length of study, as well as the outcomes that will be measured. Each person participating in the study must agree to the rules set out by the protocol.

The Food and Drug Administration’s job is to make sure medical treatments are safe and effective for people to use. The FDA, however, does not develop new therapies or conduct the clinical trials to demonstrate safety and effectiveness. FDA staff members meet with researchers and perform inspections of clinical trial study sites to protect the rights of participants and to verify the quality and integrity of the data.

For more information about clinical trials and how to participate in one of our trials, click on the links below:

  • Types of Clinical Trials
  • Phases of Clinical Trials
  • Protocols and IRBs
  • Participating in a Clinical Trial
  • Participating in a Clinical Trial
Types of Clinical Trials

There are many different kinds of clinical trials, including:

  • Treatment trials study new drugs, combinations of drugs, or new approaches to surgery or radiation therapy. This could include, for example, pain medicines, steroid injections, radiation treatment, or spinal instrumentation.
  • Prevention trials look for better ways to prevent disease in people who have never had the disease or to prevent a disease from returning. These approaches may include medicines, vitamins, vaccines, minerals, or lifestyle changes.
  • Diagnostic trials are conducted to find better tests or procedures for diagnosing a particular disease or condition.
  • Screening trials test the best way to find certain diseases or health conditions through screening tests such as magnetic resonance imaging, for example.
  • Quality of Life trials study ways to improve comfort and the quality of life for individuals with a chronic illness.

Usually, clinical trials compare a new product or therapy to something else to see if it works as well or better to treat or prevent a disease or condition. In a blinded study, a participant may be randomly assigned to receive the test product or an existing, approved therapy. In some studies, participants may be assigned to receive a placebo (a product with no therapeutic action that looks like the test product). Comparison with a placebo can be the fastest and surest way to demonstrate therapeutic effectiveness of new products. Placebos are not used, however, where a patient would be put at risk, particularly in the study of treatments for serious illnesses. Most studies of this kind compare new products to an approved therapy. Potential participants are told before they enter a trial whether placebos are going to be used in the study.

Phases of Clinical Trials

The four phases of clinical trials are:

  • Phase I: These trials are the first stage of testing in human subjects. Normally, a small (20–100) group of healthy volunteers is selected. This phase includes trials designed to assess the safety of a drug, procedure, or device (the treatment). In Phase I trials, little is known about the treatment’s effectiveness in humans.
  • Phase II: Once the initial safety of the study treatment has been confirmed in Phase I trials, Phase II trials are performed on larger groups (20–300) to test the effectiveness (efficacy) of the treatment. When the development process for a new treatment fails, this usually occurs during Phase II trials when the treatment is discovered to not work as planned. When the benefits of treatment outweigh side effects and/or risks, the next phase of testing can begin.
  • Phase III: These studies are randomized, controlled, multi-center trials on large patient groups (300–3,000). The trials in this phase are aimed at being the definitive assessment of how effective the treatment is in comparison with the current standard proven treatment.
  • Phase IV: These trials are also known as Post Marketing Surveillance Trials. Phase IV trials involve the safety surveillance and ongoing technical support of a FDA-approved drug, device, or procedure. Phase IV studies may be required by the FDA or may be undertaken by the sponsoring company (manufacturer). An example of this type of study is the study of aspirin, which was initially approved as a pain reliever, but was later found to be useful as a blood thinner.
Protocols and IRBs

A clinical trial requires a well-designed protocol, collaboration among various medical specialties, and timely and accurate reporting of the findings. It is conducted under the direction of a principal investigator (PI) and approved by an Institutional Review Board.

What Is a Protocol?

A protocol is the study plan on which all clinical trials are based. The plan is carefully designed to safeguard the health of the participants as well as answer specific research questions. A protocol describes what types of people may participate in the trial; the schedule of tests, procedures, medications, and dosages; and the length of the study. While in a clinical trial, participants following a protocol are seen regularly by the research staff to monitor their health and to determine the safety and effectiveness of their treatment. Every doctor/researcher and research facility involved in the clinical trial are required to follow the protocol.

Role of the Institutional Review Board

An institutional review board (IRB) is a committee that has been formally designated to approve, monitor, and review biomedical and behavioral research involving humans with the aim of protecting the rights and welfare of the research subjects. The purpose of an IRB review is to assure, both in advance and by periodic review, that appropriate steps are taken to protect those individuals participating as subjects in a research study. To accomplish this purpose, IRBs review research protocols and related materials (e.g., informed consent documents and investigator brochures) on an ongoing basis throughout the duration of the clinical trial.

Participating in a Clinical Trial

Considering Participation in a Clinical Trial?

Participation in a clinical trial is strictly voluntary. You may decide to participate for access to a medical treatment that is not yet available; however, remember that treatment will not benefit all patients. If you decide to participate in a clinical trial, you will be helping doctors learn more about specific conditions and treatment options which may, in the future, help others with the same disease.

Benefits of Participating

By participating in a clinical trial, you will get to:

  • Play an active role in your health care.
  • Gain access to new research treatments before they are widely available.
  • Obtain expert medical care during the trial from physician investigators who are specialists in disease being studied.
  • Help others by contributing to medical research.

Risks of Participating

Before participating in a clinical trial, you need to be aware of the following risks:

  • There may be unpleasant, serious, or even life-threatening side effects to experimental treatment.
  • The experimental treatment may not be effective for you, and/or it may not be better than the standard treatment.
  • In a randomized study, participants may not choose which treatment they will receive.
  • The protocol may require more of your time and attention than would a non-protocol treatment, including trips to the study site, more treatments, hospital stays, or complex dosage requirements.

For listing of current national clinical trials and additional resources, you may visit these websites:

www.clinicaltrials.gov

www.centerwatch.com

clinicalresearch.nih.gov

www.ciscrp.org

Are Clinical Trials Safe?

The FDA works to protect participants in clinical trials and to ensure that people have reliable information as they decide whether to join a clinical trial. The federal government has regulations and guidelines for clinical research to protect participants from unreasonable risks. Although efforts are made to control the risks to clinical trial participants, some risks may be unavoidable because of the uncertainty inherent in medical research studies involving new medical treatments. The government requires researchers to give prospective participants complete and accurate information about what will happen during the trial.

Current Clinical Trials

ANRD

Title: “PulseRider Aneurysm Neck Reconstruction Device (ANRD)”

Study Sponsor: Cerenovus

Principal Investigator: Matthew Lawson, MD

Study Synopsis: The PulseRider is authorized by the Federal Law for use with embolic coils for the treatment of un-ruptured, wide-neck, intracranial aneurysms with neck widths≥ 4 mm or dome to neck ration < 2 originating on or near a vessel bifurcation of the basilar tip or carotid terminus with at least a portion of the aneurysm neck overlapping the lumen of the parent artery.

The PulseRider have been approved by the Food and Drug Administration (FDA) with a Humanitarian Use Device (HUD) designation. This designation is recognition that the device has a limited patient population of fewer than 8,000 individuals in the United States per year.

Study Status: Implanting

For more information, contact Lutheria Hollis, CCRC at (850) 201-2350

ASTROH

Title: “Aneurysmal Subarachnoid Hemorrhage Trial Randomizing Heparin: ASTROH”

Study Sponsor: University of Louisville School of Medicine

Principal Investigator: Narlin Beaty, MD

Study Synopsis: To investigate the safety and the clinical effect of a continuous low-dose intravenous unfractionated heparin (LDIVH) infusion for the prevention of aneurysmal subarachnoid hemorrhage (aSAH) induced neurocognitive dysfunction.

The study will randomize patients in a 1:1 open label arm. Study patients randomized to LDIVH arm will receive a continuous low dose infusion of UFH for up to 14 days and the Control group will receive the normal standard of care (SOC) which is 5000 units twice a day.

Study Status: Open for enrollment.

For more information, contact Lutheria Hollis, CCRC, at (850) 201-2350.

Codman Enterprise Vascular Reconstruction Device and Delivery System

Title: “Humanitarian Use Device for wide neck intracranial (brain) saccular/fusiform (like a balloon) aneurysms either ruptured or unruptured.”

Study Sponsor: Johnson & Johnson, Inc.

Principal Investigator: Matthew Lawson, MD

Study Synopsis: The use of the Codman Enterprise Stent under the approval of the Humanitarian Device Exemption (HDE) H060001in treatment of wide neck aneurysms via an endovascular approach.

The CODMAN ENTERPRISE Vascular Reconstruction Device and Delivery System is a new device that has been approved by the Food and Drug Administration (FDA) as a Humanitarian Use Device (HUD) for use as an alternative treatment. A HUD device used to diagnose or treat a disease or condition affecting fewer than 4,000 individuals in the United States per year. The probable benefit to health outweighs the risks of injury or illness.

Study Status: Only for patients with wide neck intracranial aneurysm(s), either ruptured or unruptured.

For more information, contact Lutheria Hollis, CCRC, at (850) 877-5115.

Florida Center for Brain Tumor Research

Title: “Florida Center for Brain Tumor Research”

Study Sponsor: University of Florida

Principal Investigator: Matthew Lawson, MD

Study Synopsis: To find cures and develop treatment modalities for brain tumor through the coordinated effort among the state’s public and private universities, hospitals and biomedical industry.

All patients with brain tumors who sign informed consent to participate are eligible.

Study Status: Open for enrollment.

For more information, contact Lutheria Hollis, CCRC, at (850) 201-2350.

Neuroform Microdelivery Stent System

Title: “Humanitarian Use Device for wide neck intracranial (brain) saccular/fusiform aneurysms either ruptured or unruptured.”

Study Sponsor: Boston Scientific

Principal Investigator: Matthew Lawson, MD

Study Synopsis: The Neuroform Microdelivery Stent System is authorized by the Federal Law for use with embolic coils for the treatment of wide neck, intracranial, saccular aneurysms arising from a parent vessel with a diameter of ≥ 2mm and ≤4.5mm that are not amenable to treatment with surgical clipping.

The Neuroform™ Microdelivery Stent System or Neuroform EZ is a device that has been approved by the Food and Drug Administration (FDA) as a Humanitarian Use Device (HUD) for use as an alternative treatment. A HUD is a device used to diagnose or treat a disease or condition affecting fewer than 4,000 individuals in the United States per year. The probable benefit to health outweighs the risks of injury or illness.

Study Status: Only for patients with wide neck intracranial aneurysm(s), either ruptured or unruptured.

For more information, contact Lutheria Hollis, CCRC, at (850) 877-5115.

RELIEF- A Global Registry to Evaluate Long Term Effectiveness of Neurostimulation Therapy for Pain and NAVITAS Sub Study

Title: “RELIEF- A Global Registry to Evaluate Long Term Effectiveness of Neurostimulation Therapy for Pain and NAVITAS Sub Study – A study to Characterize the Relationship between Select Objective Metrics and Clinical Outcomes in Chronic Pain Patients treated with Boston Scientific Neurostimulation Systems”

Study Sponsor: Boston Scientific
Principal Investigator: Albert Lee, MD

Study Synopsis: To better understand the long term results of chronic pain patients treated with BSC Neurostimulation systems and describe the relationship between certain measurements and clinical outcomes in chronic pain for patients treated with Boston Scientific commercially approved neurostimulation systems.

Patients who qualify will be asked to completed surveys about their pain, they will wear chest and wrist biosensors, collect data on eDiary data application including Pain log and complete Fitness assessments.

Study Status: Open for enrollment.

For more information, contact Lutheria Hollis, CCRC, at (850) 201-2350.

Wingspan Stent System

Title: “Wingspan Stent System with Gateway PTA Balloon Catheter”

Study Sponsor: Stryker

Principal Investigator: Matthew Lawson, MD

Study Synopsis: Humanitarian Use Device: For use in improving cerebral artery lumen diameter in patients with intracranial atherosclerotic disease, refractory to medical therapy in intracranial vessels with≥ 50% stenosis that are accessible to the system.

The Wingspan Stent System with Gateway PTA Balloon Catheter is a new device that has been approved by the Food and Drug Administration (FDA) as a Humanitarian Use Device (HUD) for use as an alternative treatment. A HUD device used to diagnose or treat a disease or condition affecting fewer than 4,000 individuals in the United States per year. The probable benefit to health outweighs the risks of injury or illness.

Study Status: Only for patients with intracranial atherosclerotic disease with more than 50% stenosis.

For more information, contact Lutheria Hollis, CCRC, at (850) 877-5115.

For Our Research Colleagues

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The Neurosurgeons of Tallahassee Neurological Clinic initiated the Neurosurgery Clinical Research Division in 2007. Our physician investigators are specialists in treating nervous system disorders, neurotrauma, neurological tumors, disorders of the spine, peripheral nerve disorders, and in interventional Pain Management.

Our therapeutic areas of expertise are:

  • Brain and Spinal Cord Tumors
  • Chiari I Malformation
  • Hydrocephalus
  • Degenerative Disc Disease/Disc Herniations
  • Peripheral Nerve Entrapment Syndromes
  • Peripheral Nerve/Artery/Muscle Biopsies
  • Traumatic or Spontaneous Compression Fractures
  • Traumatic Brain Injury
  • Spinal Cord Injury
  • Epidural Steroid Injections
  • Spinal Cord Stimulator Therapy
  • Intrathecal Drug Therapy

IRB

For trials that require an inpatient component or use of hospital facilities, we are required to use the Institutional Review Board at Tallahassee Memorial HealthCare (TMH). Trials that do not require inpatient services or the use of hospital facilities may be approved through a central IRB or our local IRB.

Contact

For more information, please contact Lutheria Hollis, CCRC at (850) 877-5115.

Our Patient Demographics

Our comprehensive patient base consists of our neurology and neurosurgery outpatient clinic, Pain Management clinic, and Marianna, Florida, satellite office. Our exclusive neurosurgery contract with Tallahassee Memorial Healthcare also gives us an expanded patient base from which to draw research participants. Our patient population includes males and females of all age groups. Located in Leon County, Tallahassee is home to three major universities and a large population of retirees, young people, and families. The residents of Leon County are highly educated, with nearly 50% of county residents holding a bachelor’s, master’s, or doctorate degree.

Our Research Facilities

Only 15 minutes from Tallahassee Regional Airport, Tallahassee Neurological Clinic is located in the Professional Office Building which is conveniently attached to Tallahassee Memorial HealthCare (TMH). Through our affiliation with TMH, our clinical trials division has the ability to perform both inpatient and outpatient clinical trials.

Our neurosurgery outpatient clinic has:

  • 12 fully-equipped private exam rooms with high-speed internet connections for data upload
  • Attached covered parking garage
  • Office connected to the hospital and clinical lab
  • Locked storage for study supplies
  • Temperature-controlled storage for investigational agent
  • Complete standard operating procedures and training
  • Electronic medical records
  • 24/7 call coverage for MD/ARNP or Physician Assistant access
  • Daily FedEx, UPS pick-up

Inpatient Facility

  • Our affiliate hospital, TMH, is a 770-bed regional medical center
  • Accredited Level II Trauma Center
  • Certified Stroke Center
  • On-site 24-hour radiology including MRI
  • 10-bed state of the art neurological intensive care unit
  • 32-bed neurology/neurosurgery inpatient unit equipped with telemetry and continuous pulse-oximetry capabilities
  • 53-bed Emergency Department
  • Full rehabilitation services including acute rehabilitation center within walking distance of the clinic and hospital
  • 17 operative suites with microscopes, fluoroscopy, and STEALTH navigation
  • Inpatient investigational pharmacy
  • Full service CLIA- and CAP-approved clinical laboratories
  • Locked -40⁰ freezer
  • Ambient centrifuge
  • Daily dry ice drops

Glossary

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Blinding: A procedure in which one or more parties to the clinical trial (i.e., the subject/patient, investigator, and/or sponsor medical personnel) are kept unaware of the treatment assignment(s) of the clinical trial participants. Used to remove potential for study bias.

Certified Clinical Research Coordinator (CCRC): A research professional with greater than two years clinical research experience and with certification earned by passing required program and exam.

Clinical Trial: A research study designed to test the safety and/or effectiveness of drugs, devices, treatments, or preventive measures in humans.

Control Group: Study participants assigned to receive standard treatment.

Double Blind: Neither the subject nor the investigator is aware of which treatment the subject is receiving. A double-blind design is generally considered to provide the most reliable data from a clinical trial. This type of clinical trial, however, is usually more complicated to initiate and conduct than single-blind or open-label trails.

Efficacy: The ability to produce a desired favorable effect in treating or preventing the studied disease or condition. Phase II clinical trials gauge efficacy and Phase III trials confirm it. For a description of all the phases of a clinical trial, click here.

Exclusion Criteria: The characteristics that prevent a subject from participating in a clinical trial, as outlined in the study protocol. These criteria might include things such as age, gender, the type and stage of a disease, previous treatment history, and other medical conditions. Every clinical trial has specific guidelines regarding participation.

Experimental Group: Study participants assigned to the experimental group receive the drug, device, treatment, or intervention under investigation. In some studies, all participants are in the experimental group where in others, participants are assigned to either an experimental group or to a control group.

FDA (Food and Drug Administration): The FDA is the U.S. government agency that enforces laws on the manufacturing, testing, and use of drugs and medical devices. The FDA must approve drugs and medical devices for marketing before they are made commercially available.

Inclusion Criteria: A list of criteria (such as disease type, age, gender, and previous treatment) that must be met by all study subjects. Every clinical trial has specific guidelines regarding participation.

Informed Consent: A participant’s agreement to be in a study after being fully informed about what participating will involve. Informed consent begins with a discussion between the researchers and the prospective participants. The discussion includes important information about the research study such as:

  • The purpose of the study
  • The procedures involved
  • The risks and benefits of participating
  • How long the study will last
  • How the participant’s confidentiality will be protected
  • The key contacts for the study
  • What will happen if the study causes harm to the participants
  • That participation is voluntary and participants are free to withdraw from the study at any time

Based on this discussion with the researcher, participants are asked to sign a consent form that includes this same important information in writing. Participants are encouraged to talk with their physician, family, and friends and to ask questions whenever they need more information before signing it. Once the form is signed, participants are given a copy of the signed consent form so that they can review it at any time. Informed consent is an ongoing process, continuing during and after the study.

Institutional Review Board (IRB): A committee that has been formally designated to approve, monitor, and review biomedical and behavioral research involving humans with the aim to protect the rights and welfare of the research subjects. In the United States, regulations from the Food and Drug Administration (FDA) and the Department of Health and Human Services (specifically, the Office for Human Research Protections) have empowered IRBs to approve, require modifications in planned research prior to approval, or disapprove research. An IRB performs critical oversight functions for research conducted on human subjects that are scientific, ethical, and regulatory.

Investigational or Experimental Device: A medical device (such as an artificial heart valve or a screw used to hold bones together) that has not yet received approval from the U.S. Food and Drug Administration (FDA) for marketing.

Investigational or Experimental Drug: A drug that is not yet approved for marketing—so it is not commercially available.

Investigator: A medical professional, usually a physician (but may also be a nurse, pharmacist or other health care professional), under whose direction an investigational drug is administered or dispensed. A principal investigator is responsible for the overall conduct of the clinical trial at his/her site.

Monitor: A person employed by the sponsor or CRO who reviews study records to determine that a study is being conducted in accordance with the protocol. A monitor’s duties may include, but are not limited to, helping to plan and initiate a study, and assessing the conduct of studies. Monitors work with the clinical research coordinator to check all data and documentation from the study.

Multi-Center Clinical Trials: A clinical trial conducted according to a single study protocol and identical methods at more than one investigative site, and therefore, carried out by more than one investigator.

Office for Human Research Protection (OHRP): A federal government agency that issues Assurances and overseas compliance of regulatory guidelines by research institutions.

Open-Label Study: A study in which all parties (patient, physician, and study coordinator) are informed of the drug and dose being administered. In an open-label study, none of the participants are given placebos. These are usually conducted with Phase I & II studies.

Phase I Study: The first of four phases of clinical trials, Phase I studies are designed to establish the effects of a new drug in humans. These studies are usually conducted on small populations of healthy humans to specifically determine a drug’s toxicity, absorption, distribution, and metabolism.

Phase II Study: After the successful completion of Phase I trials, a drug is then tested for safety and efficacy in a slightly larger population of individuals who are afflicted with the disease or condition for which the drug was developed.

Phase IIa Study: Pilot clinical trials to evaluate efficacy (and safety) in selected populations of subjects with the disease or condition to be treated, diagnosed, or prevented. Objectives may focus on dose-response, type of subject, frequency of dosing, or numerous other characteristics of safety and efficacy.

Phase IIb Study: Well-controlled trials to evaluate efficacy (and safety) in subjects with the disease or condition to be treated, diagnosed, or prevented. These clinical trials usually represent the most rigorous demonstration of a medicine’s efficacy.

Phase III Study: The third and last pre-approval round of testing of a drug is conducted on large populations of afflicted patients. Phase III studies usually test the new drug in comparison with the standard therapy currently being used for the disease in question. The results of these trials usually provide the information that is included in the package insert and labeling.

Phase IIIa Study: Trials conducted after efficacy of the medicine is demonstrated, but prior to regulatory submission of a New Drug/product Application (NDA) or other dossier. These clinical trials are conducted in subjects’ populations for which the medicine is eventually intended. Phase IIIa clinical trials generate additional data on both safety and efficacy in relatively large numbers of subjects in both controlled and uncontrolled trials. Clinical trials are also conducted in special groups of subjects (e.g. renal failure subjects) or under special conditions dictated by the nature of the medicine and disease. These trials often provide much of the information needed for the package insert and labeling of the medicine.

Phase IIIb Study: Clinical trials conducted after regulatory submission of an NDA or other dossier, but prior to the medicine’s approval and launch. These trials may supplement earlier trials, complete earlier trials, or may be directed towards new types of trials (e.g., quality of life, marketing) or Phase IV evaluations. This is the period between submission and approval of a regulatory dossier for marketing authorization.

Phase IV Study: After a drug has been approved by the FDA, Phase IV studies are conducted to compare the drug to a competitor, explore additional patient populations, or to further study any adverse events.

Placebo: An inactive substance that may look like medicine, but contains no medicine (i.e., a “sugar pill” with no treatment value). In some studies, the participants in a control group may be given a placebo.

Protocol: The formal design or action plan of a research study. A protocol is carefully designed to safeguard the health of the participants as well as answer specific research questions. The protocol explains what will be done, when, how, to whom, and why. A protocol describes what types of people may participate in the trial; the schedule of tests, procedures, medications, and dosages; and the length of the study. While in a clinical trial, participants following a protocol are seen regularly by the research staff to monitor their health and to determine the safety and effectiveness of their treatment. Every doctor/researcher and research facility involved in the clinical trial must follow the same protocol so that at the end of the study, information from all of the researchers can be combined and compared.

Randomization: The process of assigning trial participants to treatment or comparator groups based on chance in order to reduce potential bias. Typically, one group receives the experimental treatment, and the other group receives either the current standard treatment or a placebo. Participants should understand that they may or may not receive the new treatment. Randomized trials may be “blinded” or “double-blinded.” In a blinded trial, the participants do not know which treatment they will receive. In a double-blind trial, both the participants and the investigator/research staff do not know to which group participants have been assigned.

Single-Blind Study: A study in which one party, either the investigator or participant, is unaware of what medication the participant is taking; also called single-masked study.

Sponsor: An individual, company, institution, or organization that takes responsibility for the initiation, management, and/or financing of a clinical trial.

Standard Treatment: A treatment currently in wide use and approved by the FDA, considered to be effective in the treatment of a specific disease or condition.