Here at Welichem we've been knocking down boundaries, literally, as our office space expands by an extra 800 sq. feet. Office space adjacent to ours became available, and we needed it to house our increasing business development operations. Now that our clinical trials are under way, the corporate development side of Welichem is more important than ever. As you know from our recent press releases and newsletters, we've been increasing our business development personnel and resources, while pushing our scientific efforts ahead at full speed too.
Our Phase I Clinical Trails are continuing as planned in Montréal. All six patients in Cohort I have been treated with WBI-1001 for at least 14 days and none has experienced any adverse side effects. Patients for Cohort II, in which the dosage level will be increased, are now being recruited. We expect to have more preliminary data in our next Newsletter.
Meantime, we've received a number of questions about clinical trails and related topics. Here then are some frequently asked questions and our answers:
What is a clinical trial?
Clinical trials are biomedical or health-related research studies, performed on human volunteers, which follow a pre-defined protocol, for the purpose of answering specific questions about new drug therapies or treatments. Clinical trials are also a necessary stage to determine whether new drugs or treatments are both safe and effective. Carefully conducted clinical trials are the fastest and safest way to find treatments which work on live patients in the real world beyond the laboratory.
What is a protocol?
All clinical trials are based on a set of rules which together are called a protocol. The protocol is a study plan that describes the candidates eligible to participate in the trial, the schedule of tests and procedures, staff oversight and monitoring, dosages, the length of study, and the methods and objects of measurement. The protocol is the game plan which will provide sufficient data to determine the validity of the trials and their applicability, and may define as well future testing or other steps en route to commercialization of the substance or process that is the subject of the trials.
What are the clinical trial phases?
Clinical trials of experimental drugs proceed through four phases. Typically, phase I is for human pharmacology, phase II is for therapeutic exploration, phase III is for therapeutic confirmation, and phase IV is for therapeutic use:
- In Phase I clinical trials, researchers principally focus on safety. They administer a new drug or treatment in a small group of people, estimate the safety and tolerability of the dose ranges, understand the characteristics of the drug's absorption, distribution, metabolism, and excretion, identify side effects, and assess the potential therapeutic objective.
- In Phase II clinical trials, the focus is on efficacy, or whether patients see beneficial results. The drug or treatment is given to a larger group of people, in a range of dosages, specifically to treat and to measure results of treatment in the indicated conditions. The dosage(s) and regimen for Phase III may also be determined.
- In Phase III studies, the drug or treatment is given to large groups of people to confirm the previous evidence that it is safe and effective for use in the intended indication and recipient population, and to provide an adequately broad patient demographic data set for marketing approval.
- Phase IV studies begin after drug approval and distribution. These studies are not necessary for approval, but are often important for optimizing use of a given drug or treatment. Such studies may focus on interactions between drugs, on various dosage responses, or on safety, efficacy, and dosages in particular patient populations, for example in children, or the elderly.
Are clinical trials safe?
The responsible federal agency (such as Health Canada or the FDA) works to protect participants in clinical trials and to ensure that people have reliable and complete information before joining a clinical trial. The respective federal governments have regulations and guidelines for clinical research for the purpose of protecting participants from unreasonable risks. Proposed clinical trials, and their proposed protocols, cannot proceed until the supervisory agencies have reviewed them thoroughly, obtained any modifications required, and finally given their approval to proceed. Although efforts are made to control risk, clinical trial participants may have unanticipated problems due to inherent uncertainty in medical research and practice.
What is "informed consent"?
Each participant must sign and date, before joining a study, an "informed consent" document, by which the participant confirms his or her willingness to participate in a particular trial, after having been informed of details about the study, including its purpose, duration, required procedures, and key contacts, and foreseeable risks and benefits. Notwithstanding giving his or her informed consent, a participant may withdraw from the clinical trial at any time.
What are "blind" or "masked" studies?
In many clinical trials, one group of patients will be given an experimental drug or treatment, while a control group is given either a standard treatment for the illness, or a placebo, or no treatment at all. Blinding or masking is a procedure in which one or more parties to the trial are kept ignorant of the treatment assignment. In a single-blinded or masked study, the participants do not know whether they are getting the drug being tested, or are in the control group. In a double-blinded or masked study, neither the participants, nor the study staff members including the investigators and monitors, are aware during the trial of which participants are receiving which treatment. In a triple-blinded or masked study, even the biostatistician who conducts data analysis after the trial doesn't know which group each participant was assigned to. Such studies are designed so that neither the patients' nor the doctors' expectations about the experimental drug can influence the results.
What is a placebo?
A placebo is an inactive pill, liquid, or powder that looks like a drug, but contains no active ingredients. The placebo thus has no treatment effects or value. In clinical trials, experimental treatments are often compared with placebos to assess the treatment's effectiveness. In many studies, participants in a control group will receive a placebo instead of an active drug or treatment. Placebos are used to control for the anomalous fact that benefits from taking medication are not always due to the drug itself, but may reflect psychological effects, or so-called "placebo effects." A patient's enthusiasm about a new medication may influence the patient's outcome. A successful drug or treatment candidate must demonstrably outperform a placebo. If a study involves the use of a placebo control, each participant’s informed consent form will explain plans to use a placebo, although the patient will not know whether he or she will receive a placebo or the study's subject drug or treatment.
We hope this discussion has been helpful.
Next month we will continue to keep you posted about our developments.
Have a great September.
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