Before any new drug can be marketed to the public, it has to go through a complicated and lengthy testing and approval process through the Food and Drug Administration (FDA). This process entails successful completion of various stages, including clinical trials. At any point along the way, testing may be stalled, halted, or lengthened due to FDA requests for additional information or further clinical trials. Is it any wonder that it takes so long for drugs – even the ones with the greatest promise or hope – to come to market? When it comes to drugs for the prevention, treatment, or potential cure for addiction, the entire process can be frustrating and disconcerting. Still, clinical trials offer the best hope for tomorrow.

What Are Clinical Trials?

The general public may have a vague understanding of what a clinical trial is – it’s a test to see if a drug will be okay to put on the market – but the more precise definition comes from ClnicalTrials.gov. According to the site, clinical trials “are generally considered to be biomedical or health-related research studies in human beings that follow a pre-defined protocol. ClinicalTrials.gov includes both interventional and observational types of studies [trials]. Interventional studies are those in which the research subjects are assigned by the investigator to a treatment or other intervention, and their outcomes are measured. Observational studies are those in which individuals are observed and their outcomes are measured by the investigators.”

Sounds pretty clear, right? But there’s still a lot of mystery and misinformation surrounding clinical trials, who can participate in them, how long they last, and what’s involved. Let’s take a brief look before we go further into the benefits of clinical trials and some promising drugs that may be coming for the prevention, treatment or – dare we say, cure – for addictions.

About Clinical Trial Participants

First of all, who would want to participate in a clinical trial? After all, there are no guarantees, and participants might not even receive the drug being tested. They may be given a placebo, thinking that they were getting the drug. And there are risks involved in testing new drugs. Who would be so foolish as to subject themselves to such potential risk?

The answer is that there are many reasons why people participate in clinical trials.

• Some individuals desire to play a more active role in their own health care and believe, after careful investigation and talking with their doctors, that participating in a particular clinical trial may give them access to medication that may improve their health or lengthen their life span. The brutal truth is that some people have exhausted all hope with currently available drugs and will do anything, take any new drug or try any new treatment, simply to prolong their lives. These individuals may not be healthy enough to participate in a controlled clinical trial, but may be able to participate in an expanded access trial of a new investigational drug (IND).

• Others want to gain access to new medical treatments and drugs before they are widely available to the public – which can mean years ahead of public release. These are the experimenters, people who believe that they can benefit from trying a new drug.

• There’s also the wish to provide valuable information to the medical field. These individuals may be considered altruistic. For some individuals, the reasons for participating in clinical trials include all three.

Just because someone wants to participate in a clinical trial, however, doesn’t mean that they’ll be able to do so. Each clinical trial has inclusion and exclusion criteria. This is an important principle of medical research that helps to produce reliable testing results. Inclusion criteria are factors that allow individuals to participate in a certain clinical trial, while exclusion criteria are those which prohibit individuals from participating.

These factors are not discriminatory or personal. They are based on parameters researchers deem most relevant to producing a viable sample of participants for the particular drug/condition being tested, and to keep participants safe during the clinical trial. These criteria are based on factors such as age, gender, type and stage of a disease, previous treatment history, and other medical factors. In addition, before participating in a clinical trial, the individual must qualify for the trial in question. For some clinical trials, researchers are looking for individuals with a particular disease or condition that will be studies during the trial, while other trials require healthy individuals.

Benefits and Risks of Clinical Trials

The benefits to participating in clinical trials are very similar to the reasons why people decide to participate in them in the first place. The best clinical trials are those that are well-designed and well-executed – and are the best approach for eligible participants to:

• Play an active role in their own health care

• Be able to gain access to new treatments and drugs before they are widely available to the public

• Receive expert medical care during the clinical trial at leading health care facilities

• Help others by contributing to medical research

But there are also risks to participating in clinical trials – some of them potentially severe:

• Clinical trials may involve experimental treatment that could involve unpleasant, serious, or even life-threatening side effects. Some of the possible side effects include headache, nausea, hair loss, skin irritation, or other physical problems. Experimental treatments must be evaluated for both immediate and long-term side effects and/or adverse reactions.

• The experimental treatment may not be effective for the participant in the clinical trial.

• The clinical trial protocol may involve considerable time that the participant would not have been subjected to if he/she were not in the trial – such as trips to and from the testing facility or study site, more treatments, hospital stays, or complex dosage requirements.

Things to Consider Before Participating in a Clinical Trial

Anyone who is contemplating participation in a clinical trial – or the loved ones or friends of someone thinking about doing so – should satisfy themselves that they feel comfortable with the parameters, testing, dosage, length, potential risks and other factors of clinical trial. Potential clinical trial participants should ask plenty of questions of the health care team conducting the trial, to include:

• What is the purpose of the study?
• Who will be in the study?
• What is the basis for researchers believing the treatment to be tested will be effective? Has it been tested before?
• What are the kinds of tests and/or treatment that will be involved?
• Tell me about the potential risks, side effects, and benefits – and how do they compare with my current treatment?
• How will my daily life be impacted by this clinical trial?
• How long will the trial last?
• Will I need to be hospitalized for any part of the clinical trial?
• Who pays for the experimental treatment?
• What about reimbursement for any of my other expenses?
• Is any type of follow-up care part of this study? If so, what type is it?
• How will I know if the experimental treatment is working? Will I be given the results of the clinical trial I am participating in?
• Tell me who will be in charge of my care.

Different Types of Treatment Trials

With the vast variety of medical diseases and conditions, there are clinical trials that must be conducted before any new type of treatment or drug can be made available. There are various types of treatment trials to accommodate testing needs. These include:

Treatment Trials – These test experimental treatments, new combinations of drugs, or new approaches to radiation therapy or surgery.

Prevention Trials – These trials search for better ways to prevent disease in people who have never had the disease or to prevent the disease from recurring. Such approaches may include vaccines, medicines, and changes in lifestyle, and use of vitamins and minerals.

Diagnostic Trials – As the name implies, diagnostic trials are conducted to find better procedures or ways to diagnose a particular disease or condition.

Screening Trials – In screening trials, the purpose is to test the best way to detect certain diseases or health conditions.

Quality of Life Trials – Also called supportive care trials, quality of life trials explore ways to improve the comfort and quality of life for persons with a chronic illness.

Examples of Current or Concluded Clinical Trials

With respect to the prevention, treatment or potential vaccine or cure for various types of addiction, there are some clinical trials that are either currently in process or have completed testing of one stage or another. The following are examples gleaned from the ClinicalTrials.gov site. They show just a sampling of different types of clinical trials and studies for addictions and are by no means all-inclusive.

• Tobacco Tactics Website for Operating Engineers – Sponsored by the University of Michigan, this trial is currently recruiting participants. This is a research study to test tobacco tactics intervention among operating engineers (heavy equipment operators). The intervention group will be directed to the Tobacco Tactics website (http://bcbsm-operatingengineers.nursing.umich.edu/), while the control group will be directed to the state-supported 1-800- quit now hotline during annual safety training that all operating engineers are required to attend. Web-based quitting intervention includes provision of over-the-counter (OTC) nicotine replacement therapy, followed by nurse email and/or telephone counseling. It is anticipated that operating engineers randomized to the Tobacco Tactics website intervention will have at least equal or greater decreases in smoking at 30-day and 6-month follow-up compared to those persons randomized to the 1-800 quit line. Researchers also expect that participants randomized to the Tobacco Tactics website intervention will a) be able to accomplish tasks, b) be able to accomplish goals with skill and speed, c) be able to operate the system, d) be satisfied with the website, e) access the site repeatedly, and f) spend time on the site during the intervention period.

• Eating Disorders Prevention: An Effectiveness Trial for At-Risk College Students – Sponsored by the Oregon Research Institute, with collaborators at University of Texas at Austin and Drexel University, this 3-site effectiveness trial, which is currently recruiting participants, will test whether a brief dissonance-based eating disorder prevention program produces intervention effects when college counselors, psychologists, and nurses are responsible for participant recruitment, screening, and intervention delivery under ecologically-valid conditions. The condition is eating disorders-obesity. Threshold and subthreshold eating disorders affect over 10 percent of young women and are associated with functional impairment, distress, psychiatric comorbidity, medical complications, mortality, and risk for onset of obesity. It is a pressing public health priority to develop effective prevention programs for eating pathology. This proposed product will be the first effectiveness trial to test whether an eating disorder prevention program with strong empirical support from efficacy trials produces effects under ecologically-valid conditions among high-risk college students. This is a vital step toward widespread dissemination of programs developed with funding from the National Institutes of Health (NIH). Inclusion criteria: 1) registered student at participating school 2) self-reports of body image concerns. Exclusion criteria: meets DSM-IV criteria for anorexia nervosa, bulimia nervosa, or binge eating disorder.

• Developing an Intervention to Address Suicide Risk During Substance Abuse Disorder – Currently recruiting, this 2-year study is sponsored by the University of Michigan, with collaboration by the National Institute on Drug Abuse (NIDA). The study is designed to 1) adapt and refine an existing cognitive-behavioral treatment (CBT) intervention to decrease suicidal thoughts and behaviors for use in Substance Use Disorder (SUD) treatment settings, and 2) conduct a pilot, randomized control trial with 50 patients in treatment for SUDs comparing the CBT intervention to an enhanced control condition. Investigators hope to determine the feasibility of implementing these procedures in residential SUD treatment, as well as determining whether the intervention decreases the level of suicide ideation, suicide attempts, hopelessness, and frequency of substance use relative to the controlled condition. Research indicates that problematic alcohol and drug use are closely linked with fatal and non-fatal suicide attempts. But there are no data on the effectiveness of specific interventions designed to decrease suicidal thoughts and behaviors during an episode of SUD treatment in individuals at high risk for suicide. From a public health perspective, SUD treatment programs contain large numbers of patients at high risk for future suicidal behaviors. Therefore, they have the potential to play a central role in efforts to decrease suicide. Inclusion criteria: over 18 years of age, report a past lifetime suicide attempt, report a current suicidal ideation, resided in treatment less than 4 weeks, in treatment less than 6 months. Exclusion criteria: mental incompetence (for example, unable to provide informed consent), diagnosis of schizophrenia or currently psychotic.

• Efficacy Trial of Warrior Check-Up – This trial is not yet open for recruitment, but the expected study dates are July 2010 through January 2014. Sponsored by the University of Washington, with collaboration by the U.S. Army Research and Medical Materiel Command, the study will help develop and test a brief telephone-delivered motivational enhancement intervention for substance-abusing military personnel who are not currently in treatment. The hypotheses being tested in this Phase II study are that this intervention will prompt a willingness to participate voluntarily in a self-appraisal of substance abuse behavior and consequences, self-initiated change or enrollment in a treatment or self-help program, and cessation of abuse of alcohol or other drugs. Rates of heavy drinking are higher among military personnel than in the general population and are even higher among recently-deployed personnel. While counseling can be effective, substance abusers do not tend to voluntarily seek treatment. Adapting the motivational enhancement intervention (MET),, also called “check-up,” is warranted for 3 key reasons:1) it has the potential to overcome barriers to treatment seeking, 2) it has the potential of attracting voluntary participation, and 3) protocols for disseminating this low-cost intervention for use with deployed military personnel can readily be developed and implemented. Inclusion criteria: current abuse or dependence on one or more substances, not currently enrolled in a counseling program focused on substance abuse, currently serving in the Army or other branch of the military. Exclusion criteria: non-fluency in English, evidence of psychosis.

• Treatment Study: Reducing Cocaine/Heroin Abuse with SR-Amphetamine and Buprenorphine (ARC) – Currently still recruiting, this treatment research study, sponsored by Wayne State University in collaboration with the National Institute on Drug Abuse (NIDA), is for individuals who currently have cocaine use or dependence and may also have heroin dependence. The purpose of the study, which takes 11 weeks, is to test whether oral sustained release d-amphetamine (SR-AMP) is safe and more effective than placebo for preventing relapse for cocaine use for individuals who abuse or are dependent on cocaine. Researchers are also interested whether, for individuals dependent on cocaine and heroin, SR-AMP is safe and effective for preventing cocaine relapse in combination with buprenorphine. Many medications have been tested, and failed, for treating cocaine dependence alone or in cocaine abusers who also use heroin. Extensive inclusion criteria and precise exclusion criteria.

• Study of Medical Treatment for Methamphetamine Addiction – This Phase II study, sponsored by NIDA, with collaboration by the University of California, Los Angeles (UCLA), is to determine if buprenorphine can help people reduce or stop their methamphetamine use and to investigate whether genetic variations influence whether people respond to buprenorphine treatment for methamphetamine addiction. Currently there are no medications approved for the treatment of methamphetamine addiction. Buprenorphine has been approved by the FDA for the treatment of depression and for cessation of cigarette smoking. Study start date was January 2009. Expected study completion date is April 2012. Detailed inclusion and exclusion criteria.

• Topiramate in the Treatment of Pathological Gambling – This Phase IV study is active, but no longer recruiting participants. Sponsor is Mount Sinai School of Medicine, in collaboration with Ortho-McNeil, Inc. The study is to assess the efficacy of topiramate (Topomax) in the treatment of pathological gambling. Pathological gambling is a debilitating disorder, generally leading to severe personal, familial, financial, social, and occupational impairments. Patients experience a progressive inability to resist impulses to gamble, and gambling significantly disrupts the patient’s functioning in personal, familial, and/or vocational spheres. Topiramate has shown preliminary efficacy in some impulse control disorders. There are extensive inclusion and exclusion criteria for this 14-week outpatient study.

Research Continues to Identify New Treatments and Drugs for Addiction

What’s important to take away from this information is that research continues in the field of addiction prevention and treatment. Researchers, funded by NIDA, and in collaboration with leading universities, pharmaceuticals, and private concerns, are expanding their efforts to identify new medications, treatments, vaccines, and potential cures for the various types of addictions, polysubstance abuse, and co-occurring substance abuse and mental health disorders. While it can be frustrating to think about how long it takes to develop, test, and gain final approval of such treatment or medications, the good news is that the field of addiction research is gaining in prominence. In the end, what this means for the millions of Americans suffering from an addiction, or whose family members or friends are addicted is that there is hope for tomorrow. Clinical trials are an important, and critical step, in the realization of those hopes.