6 Reasons to Participate in Clinical Trials

By Dr. Lynn Webster, PNN Columnist

The COVID-19 pandemic has left many people feeling helpless -- yet you can help defeat the virus. You may not be a medical researcher, but you can still be part of developing a vaccine. There are three COVID-19 late-phase vaccine trials currently underway in the United States, and they will involve more than 100,000 individuals. 

All stages of research  require volunteer subjects. Early-phase trials require healthy volunteers, who may not directly benefit from a vaccine or medication but participate for other reasons. The current COVID-19 vaccine trials, for example, involve people who do not have the infection. Some will receive the new vaccine and others will get a placebo.

Only after volunteers are exposed to the virus can scientists determine if the vaccine is safe and effective when compared to a placebo. If a participant becomes ill because of exposure during the trial, the sponsor of the study generally pays for their medical expenses.

Not all clinical trials involve a placebo, but most do. Later-phase trials may offer benefits to the volunteers who have the disease or condition that the drug targets.

There are various ways to get involved in a trial. You may be invited to join one of these trials. Your physician may be part of network of providers participating in a study. If so, they can offer you an opportunity for enrollment. Or, if you are looking for a specific treatment, you can go online to find a study that may be of interest to you.

Here are six reasons to consider participating in a clinical trial:

1) Clinical trials provide safer and more effective therapies

Without clinical trials, development of new and more effective treatments would not be possible. Medical advances depend upon people who are willing to participate in trials. Every drug, from vaccines to pain treatments, approved by the FDA has been studied in clinical trials.

2) Clinical trials provide you with free access to a new treatment

When I started to treat people in pain, I conducted clinical trials to provide my patients with cutting-edge technology that was not yet publicly available. I wanted my patients to have access to the best and newest therapies in the pipeline.

Of course, there was no guarantee that the experimental therapies would be effective. However, the treatments were free to participants in the trial. If the risks were perceived as acceptable, it could be worth it for patients to take the chance.

3) Clinical trials can save your life

Late-phase clinical trials, such as the three currently underway for COVID-19, may provide immunization that could save the life of the participant. Through other clinical trials, we have provided lifesaving therapies to subjects who otherwise could not have obtained them because they couldn’t afford the treatments, or the treatments simply were not available to consumers.

4) Clinical trials can save a loved one’s life

Clinical trials provide researchers with more knowledge and potential therapeutic options. Participating in vaccine trials can lead to the approval of a treatment. This could save the life of a family member who may one day need a vaccination or drug treatment.

5) Clinical trials are regulated by FDA

Clinical trials are highly regulated and generally regarded as safe. Most clinical trials sponsored by industry are required to have their protocols reviewed and accepted by the U.S. Food and Drug Administration, which assesses the safety of every trial. The agency will not allow a company to begin a trial that has not been fully vetted by several FDA departments.

In addition, all protocols must be submitted to an institutional review board (IRB) for approval. This is an independent body charged by the FDA to evaluate and monitor trials. The IRBs and principal investigators contract with the FDA to follow guidance from the Federal Office for Human Research Protections. Both IRBs and principal investigators can be criminally charged if they fail to adequately monitor and accurately report results of a trial.

When the FDA grants permission to fast-track a particular therapy because of an emergency situation, protocols may be loosened. The potential benefits, in that case, have been determined to outweigh the risks. We have to hope that the accelerated approval process for a COVID-19 vaccine will not cause adverse events.

Prior to enrolling in a trial, participants are provided with an informed consent form that describes the trial in layman's terms and explicitly states all of the potential risks. Participation in clinical trials is always voluntary, and a person can withdraw from a trial at any time.

6) Clinical trials may compensate participants

Some trials compensate participants for their time and travel. This occurs more often in early-phase trials for which there is no clear clinical benefit to the individuals. Most trials requiring participants to stay overnight in a research facility compensate volunteers. Participants' compensation depends on the amount of time the testing takes and the types of tests that are performed.

Compensation must be reasonable, yet not excessive. Compensation ranges from $50 or less for a simple saliva sample to $10,000 or more for studies that require staying in a facility for 30 days or so. 

You can learn more about clinical trials at the National Institutes of Health website or by visiting the Clinical Trials website. The National Institute of Allergy and Infectious Diseases also has a website to connect volunteers to coronavirus vaccine trials. And COVID Dash has a portal where people can volunteer for dozens of coronavirus studies around the world.

One clinical trial may create the breakthrough that helps us get the pandemic under control. But that can only happen if enough people from a variety of demographics are willing to volunteer to be a part of the effort.

Lynn R. Webster, MD, is a vice president of scientific affairs for PRA Health Sciences and consults with the pharmaceutical industry. He is author of the award-winning book The Painful Truth, and co-producer of the documentary It Hurts Until You Die. Opinions expressed here are those of the author alone and do not reflect the views or policy of PRA Health Sciences. You can find Lynn on Twitter: @LynnRWebsterMD. 

CRPS Patients Needed for Clinical Study

By Pat Anson, Editor

About 80,000 Americans each year are diagnosed with Complex Regional Pain Syndrome (CRPS), a poorly understood condition caused by injury or trauma that leads to throbbing and burning pain that never goes away. It often takes years and multiple doctors before a patient is diagnosed with CRPS – and by then the pain has often migrated to other parts of the body and has become chronic.

That’s the dilemma now faced by Axsome Therapeutics (NASDAQ: AXSM), a biopharmaceutical research company that hopes to win FDA approval for an experimental, non-opioid drug that would be the first medication of any kind approved for treating CRPS --- also known as Reflex Sympathetic Dystrophy (RSD).

Axsome is conducting a Phase 3 clinical study of the drug --- called AXS-02 --- in the United States, Canada, the United Kingdom and Australia.

The challenge? Although Axsome only needs about 190 patients for the CREATE-1 study, it’s having trouble finding enough eligible patients. They’re looking for patients who suffered their initial injury in the last year and who were diagnosed with CRPS in the last six months.

“We’re trying to find patients very early in the stages of CRPS,” says Randall Kaye, MD, chief medical officer of Axsome.  

It typically takes a year or more for a patient to get a CRSP diagnosis because its early symptoms are not all that different from acute pain caused by surgery, a broken bone or some other type of trauma. It takes an experienced doctor to recognize the early signs.

“These are patients who continue to have pain that just doesn’t quite follow the routine course. Even after about a week or two, something is different. The pain is too much or the quality of the pain is just different. They describe a burning sensation or there’s exquisite sensitivity to temperature,” says Kaye. “What happens to these patients is that they continue to see a variety of physicians before they’re given that label of CRPS.”

a CRPS PATIENT 6 MONTHS AFTER leg fracture

“I wish it was easy to diagnose Complex Regional Pain Syndrome,” says Barby Ingle, president of the International Pain Foundation (iPain), who was diagnosed with RSD/CRPS two years after a car accident that injured her shoulder. “I went from having RSD in my face and shoulder. It then spread to my right arm and hand, then my entire right side. By the time I was properly diagnosed I had full body including organ involvement.”

“I have personally spoken to thousands of patients who have been diagnosed with RSD/CRPS. Out of all of them, two were diagnosed within the first 3 months, most took over a year. For me, I saw 43 providers before receiving a proper diagnosis. Most pain providers were not educated and although providers are getting better education now, there are still major delays.”

Opioids and other pain medications only dull the pain of CRPS, but Axsome is hoping that AXS-02 can also treat the underlying condition that causes the disorder.

“I hope so,” says Kaye. “Instead of just relieving pain, we’re getting right at the underlying pathophysiology of the condition.”

AXS-02 is an oral formulation of zoledronic acid, an injectable bisphosphonate that inhibits the production of compounds that cause bone pain. Bisphosphonates have long been used to treat osteoporosis and Kaye believes they might also stop the progression of CRPS.

“It’s pretty straightforward. Patients take one tablet once a week for six weeks and they’re done,” Kaye told Pain News Network. “We don’t think there will be a reoccurrence based on the mechanism of action. But we want to be sure.”

Proving that AXS-02 can do more than just relieve symptoms of CRPS will take time. If it can find enough patients, Axsome hopes to finish the CREATE-1 study in mid-2017. Additional studies may then be needed. If the clinical results are positive, the Food and Drug Administration has granted “fast track” and “orphan drug” designation for AXS-02, which will speed up the application and approval process.

CRPS patients interested in applying for the CREATE-1 study should click here.    

Experimental Painkiller Leaves Six Hospitalized

By Pat Anson, Editor

Six men have been hospitalized in France after being sickened during a clinical trial of an experimental painkiller. One victim has been declared brain-dead and four others are in critical condition.

(Update: The man who was brain-dead has died, according to local media reports.)

The Phase I trial of the drug has been suspended and all 90 participants have been urged to contact a hospital.

According to reports, the experimental drug being developed by the Portuguese company Bial inhibits a brain enzyme that degrades endogenous cannabinoidsthat are produced naturally by the brain to relieve pain.

By degrading the enzyme called FAAH, Bial hopes to develop compounds that increase brain levels of cannabinoids for use as anti-depressants or pain relievers.

French health officials say the unidentified drug “does not contain cannabis or any derivative of cannabis.”

"We were informed that five participants showed severe symptoms. Following the best international medical practices, they were immediately transferred by the company responsible for conducting the clinical trial to observation at the University Hospital of Rennes, being currently under permanent medical supervision," Bial said in a statement.

Phase I trials are usually conducted to prove the safety of a drug, while the effectiveness of a drug is tested in Phase II and III clinical studies.

The men who were hospitalized were all given multiple doses of the drug, starting January 7. Three days later they started developing neurological symptoms. Two people who were given a harmless placebo developed no symptoms.

“It is definitely the product that is responsible,” said Gilles Edan, head of neuroscience at Pontchaillou Hospital in Rennes, where the men are being treated. She said there is no anti-dote to the drug and its effects could be “irreversible.”

The trial in Rennes was being conducted by Biotrial, a firm that conducts early clinical trials in France and Newark, New Jersey. In France, volunteers can earn nearly $5,000 for participating.  

According to the Daily Mail, Biotrial is able to fast-track early patient studies by “combining the favourable regulatory environment in Western Europe with fast and efficient patient recruitment in Eastern Europe.” 

 “Our thoughts go out to the volunteers and their families. We are working hand in hand with the Health Authorities to understand the cause of this accident,” the company said in a statement.

“The trial has been conducted in full compliance with the international regulations and Biotrial’s procedures were followed at every stage throughout the trial, in particular the emergency procedures for the transfer of subjects to the hospital.”