About our Clinical Research Program
Welcome to Marin Endocrine Research. We are actively engaged in new and practical research in many areas of endocrinology and metabolism with the purpose of incorporating cutting edge pharmaceutical advances into our community based practice.
To date we have conducted over 75 studies. Our goal is to continue advance medical knowledge in the areas of diabetes, endocrinology and metabolism and to provide opportunities for informed patients to participate in carefully chosen clinical trials.
Since the earliest studies in 1975, our research program has steadily expanded and is now nationally recognized. With a current active staff of ten members, several major publications and active working relationships with nearly every major endocrine pharmaceutical company, we now have research experience that includes clinical studies in diabetes, hormone replacement therapy, hypercholesterolemia, hyperlipidemias, obesity and osteoporosis.
Our mission in directing the research department at Marin Endocrine Care & Research is to evaluate promising new medications in our field of endocrinology and metabolism, to expand knowledge about new treatments, to gain further understanding of new advances in pharmaceuticals and to make these available to the Marin community while contributing scientifically to the community at large.
Our research watchwords are: Safety, Efficacy and Utility
Safety is our primary requirement for considering our participation in a clinical trial. All of the available safety data are reviewed before considering each new study. The ways in which a medication is metabolized and excreted along with its side effects are reviewed. Prior studies in humans are carefully scrutinized, are as animal studies. If a study-drug has shown good safety data in preliminary investigations we consider the efficacy.
Efficacy is reviewed carefully to be certain that further research is likely to lead to medications that will be effective and will have a significant impact on controlling or curing a disease. We are especially interested in drugs which work by novel mechanisms to control a disease process or its complications.
Utility is our third important criteria for new study evaluations. Our assessments include a determination as to whether a new medication is likely to be of practical use in the treatment of a disease. We assess patient acceptability, ease of administration, dosing, side effects and drug-drug interactions.
Our area of expertise is in phase 2, 3 and 4 studies. (see below) which are usually the final hurdles before new medications are given FDA approval. We have a long track record in conducting phase 3 studies.
We are currently engaged in studies for the treatment of diabetes with inhaled insulin, insulin sensitizers and stimulators. We have new and ongoing studies of medications that may reduce both cholesterol and triglycerides. Research protocols for obesity, osteoporosis, menopause therapies and hypertension have all been part of our past and present experience.
We are very proud of our research history. We have succeeded in assisting in the development of many new and exciting advances in drug therapy of endocrine disease. Our staff and our ethics are conscientious.
What are the phases of new medication development?
After a new compound is studied and tested in tissue culture and animal models, a series of phased human trials are begun.
Phase 1 Trials: Proof of Concept
These are the earliest studies in the life of a new drug or treatment. They are usually small trials, recruiting up to 30 patients (often a lot less). The trial may be open to people without disease who serve as normal controls as well as people with the disease in question. Phase 1 testing explores whether a new medication might help treat the condition under study. Phase 1 trials are done to find out:
- The safe dose range
- The side effects
- How the body copes with the drug
- If the treatment is effective
The initial group of patients to take part are given a very small dose of the drug. If all goes well, the next group will get a slightly higher dose. With each group taking part, the dose is gradually increased and the effects monitored. Side effects are recorded and assessed. In a phase 1 trial, a large number of blood tests are performed, as the researchers evalute the effects of the medication including it’s metabolism and disposal.
Phase 1 trials are important because they are the first step in finding new treatments for the future.
Phase 2 Trials: Extending the testing in the disease state
About (70%) new treatments tested at phase 1 make it to phase 2 trials. These trials may be done on people who all have same medical condition.
Phase 2 trials are done to determine:
- More about side effects and how to manage them
- The most effective dose to use
- If the new treatment works well enough to test in phase 3
Although these treatments have been tested at phase 1, patients may still encounter rare side effects that are unknown. Drugs can affect people in different ways.
Therefore Phase 2 trials are often larger than phase 1. There may be up to 50 people taking part. If the results of phase 2 trials show that a new treatment may be as good as existing treatment, or better, it then moves to phase 3.
Phase 3 Trials
These trials compare new treatments with the best currently available treatment (the standard treatment). They may compare a completely new treatment with the standard treatment, or Different doses or ways of giving a standard treatment.
Phase 3 trials are usually much larger than phase 1 or 2. This is because differences in success rates may be small. As such, some studies need many patients in the trial to prove that the medication is effective. They are generally the final studies needed for FDA approval.
Sometimes phase 3 trials involve thousands of patients in many different research centers often in different countries, to extend the observations of safety, efficacy and utility to a broad range of patient populations.
Phase 3 trials are usually randomized. This means the researchers put the people taking part into 2 groups at random. One group gets the new treatment and the other the standard treatment. To avoid biases, patients and investigators do not know who is on active drugs or placebo (“double blind”) study.
Trial overviews are studies that combine all the results from phase 3 trials of a new treatment. They are sometimes called meta-analyses. The idea is to get a broader picture of how well a treatment works. The more data (information) you have, the more accurate the results are likely to be.
Phase 4 trials are done after a drug has been shown to work and has been granted a license. The goal of phase 4 studies is to explore additional properties of the study drug either to demonstrate long term effectiveness or to explore the use of the medication in the treatment of other diseases. The main reasons pharmaceutical companies run phase 4 trials are to find out:
- More about the side effects and safety of the drug
- What the long term risks and benefits are
- How well the drug works when it’s used more widely than in clinical trials