Clinical Trials
Clinical trials are critical in developing new and better therapies
The Foundation for Peripheral Neuropathy is honored to be able to partner with some of the greatest scientists to help facilitate their clinical research trials on peripheral neuropathy, often able to serve as a platform to connect researchers and industry leaders with a population of peripheral neuropathy patients for clinical trials. Under-enrollment in trials is one of the greatest challenges clinical researchers face as it slows research progress and deters potential funders from investing in research. We all pay the price in terms of higher costs and longer time horizons to get to therapeutic breakthroughs. Across all diseases, 85 percent of clinical trials finish late due to difficulties enrolling participants and nearly one-third of trials fail to recruit a single subject and cannot ever begin.
To take action and find a clinical trial near you, visit ClinicalTrials.gov, a registry of publicly and privately supported clinical studies of human participants conducted around the world.
We have also partnered with Antidote to help you find and take part in a clinical trial that is right for you. Simply answer a few questions and Antidote’s smart search engine will match you to a trial, quickly and easily.
FPN can help facilitate clinical trials
Clinical trials pave the way for improved treatments for peripheral neuropathy, as well as give hope to patients suffering from this condition. FPN is pleased to be able to be the ‘connector’ in these important initiatives.
FPN communicates with a wide network of peripheral neuropathy patients through its various education and communication platforms.
These patients are eager to learn about the most recent research on peripheral neuropathy, as well as participate in clinical trials that are appropriate for them. FPN has been able to announce clinical trials to its constituents to the benefit of researchers and patients. Please contact Lindsay Colbert at [email protected] to find out more.
Clinical trial phases
Clinical trials are conducted in a series of steps, called phases – each phase is designed to answer a separate research question.
Phase 1 Clinical Trials
In Phase 1 trials the candidate drug is tested in people for the first time. These studies are usually conducted with a small number of healthy volunteers, generally 100 or less. The main goal of a Phase 1 clinical trial is to assess the safety of the medicine when used in humans. Doctors/researchers look at the pharmacokinetics of a drug: How is it absorbed? How is it metabolized and eliminated from the body? They also study the drug’s pharmacodynamics: Does it cause side effects? These closely monitored trials are designed to help researchers determine what the safe dosing range is and if the candidate medicine should move on to the next stage of development.
Phase 2 Clinical Trials
In Phase 2 clinical trials, doctors/researchers evaluate the candidate drug’s effectiveness in 100 to 500 patient volunteers. Many Phase II trials study patients receiving the drug compared with patients receiving a different treatment, either an inactive substance (placebo), or a different drug that is usually considered the standard of care for the disease.
Doctors/researchers also analyze optimal dose strength and schedules for using the drug and examine the possible short-term side effects (adverse events) and risks associated with the drug. If the drug continues to show promise, they prepare for the much larger Phase 3 trials.
Phase 3 Clinical Trials
Phase 3 clinical trials generate statistically significant data about the safety, efficacy and the overall benefit-risk relationship of the investigational medicine. Phase 3 trials may enroll 1,000 to 5,000 patients or more across numerous clinical trials sites around the world. This phase of research is essential in determining whether the drug is safe and effective.
Each patient enrolled in a Phase 3 clinical trial has a chance of being in one of the following groups:
- Control group – the group that gets the standard treatment
- Study group – the group that gets the new treatment being tested
Doctors/researchers do not know if the new treatment is better than the standard treatment, but they believe it is as good and may be better.
After the Phase 3 trial, the FDA reviews the clinical trial results to make sure the treatment is safe and effective for people to use. The FDA decides whether to approve the treatment so that it is available for all patients.
How are patients put into groups?
A computer decides which patients are in the control group and which patients are in the study group. Patients have a chance of being in either group. The patient and doctor do not decide. It is random and due to chance alone. This helps to avoid bias in the clinical trial. (Bias happens when human choices affect a study’s results.)
Would my doctor know which group I am in?
In single blind studies, patients do not know whether they are in the control or study group, but the doctor does. In double blind studies, neither the patients nor the doctors know which patients are in each group. (In case of an emergency, doctors can find this information in the study file.)
Would I be given a placebo?
A placebo is something that looks like medicine, but is not. If a placebo is used, it is given together with the best standard treatment. This allows doctors/researchers to compare standard treatment alone to standard treatment with a new drug.
Phase 4 Clinical Trials
In Phase 4 trials, doctors/researchers study treatments that the FDA has already approved. The goal of Phase 4 clinical trials is to continue studying side effects of a new treatment.
From the NINDS- for researchers
NINDS (National Institute on Neurological Disorders and Stroke) established the Early Phase Pain Investigation Clinical Network (EPPIC-Net) as a part of the NIH HEAL (Helping to End Addiction Long-term) Initiative. EPPIC-Net’s mission is to accelerate and enhance clinical testing of novel, non-addictive pharmacologic and non-pharmacologic therapeutic “assets,” including new and repurposed small molecules, biologics, natural products, and devices, targeted to pain conditions of high unmet need.