Clinical Trials

Step 10: Clinical trials and how to find them

A clinical trial is a research study of new treatment that involves patients. Each study is designed to find better ways to prevent, detect, diagnose, or treat cancer and to answer scientific questions. Clinical trials may offer patients access to promising treatments that have not yet been approved, but have been studied in a laboratory setting and may have already been studied in other myeloma patients. The experimental drugs used in clinical trials are free-of-charge to the patient. 

The IMF’s Myeloma Matrix is a helpful up-to-date online resource that lists myeloma drugs under development and tracks them from discovery to FDA-approved.

View the Myeloma Matrix

Common terms used in clinical trials:

  • Control group – The arm of a randomized clinical trial that gets the standard treatment or placebo (no treatment).
  • Experimental group – The arm of a randomized trial that gets the new treatment.
  • Randomized clinical trial – A research study in which subjects are randomly assigned to receive a particular treatment or not.
  • Arm – One of the treatment groups of a randomized trial. The majority of randomized trials have two, but some have more. 
  • End point – The goal of the trial; what a clinical trial is trying to measure or find out. Typical end points include measurements of toxicity, response rate, and survival.
  • Double blind – Aspect of a randomized trial in which neither the participant nor the investigator knows the arm of the trial to which the patient is assigned. The purpose is to eliminate any bias in the reporting of results. 
  • Phase I trial – A trial designed to determine the maximum-tolerated dose (MTD) of a new drug or a new combination of drugs. It is usually the first human testing of a new treatment, although in phase I trials of combination therapies, the individual elements may already have been well tested. Patients in phase I trials generally have advanced cancer that is refractory to all standard treatment. In a typical phase I trial, successive groups (“cohorts”) of 3 to 6 patients are given the treatment. All patients in a cohort get the same dose. The first cohort typically gets a very low dose, and the dose is raised in each subsequent cohort until a set number of patients experience dose-limiting toxicity (DLT). The dose level used for the previous cohort is then taken to be the MTD. This dose is then used in a phase II trial.
  • Phase II trial – A trial designed to determine the response rate of a new therapy that has already been tested in phase I trials. Typically, 14 to 50 patients with one type of cancer are treated to see how many have a response. Patients are usually required to have advanced cancer that is refractory to any standard treatment, and in addition, they must have measurable disease. If results from a phase II trial are promising enough, the treatment may then be tested in a phase III trial. If the results are obviously much better than the standard treatment, then it may not be necessary to do a phase III trial, and the treatment may become standard-based on phase II trial results.
  • Phase III trial – A trial designed to compare two or more treatments for a given type and stage of cancer. The end point of a phase III trial is usually survival or disease-free survival. Phase III trials are usually randomized, so patients don’t choose which treatment they receive. A typical phase III trial has 50 to thousands of patients. Some phase III trials compare a new treatment that has had good results in phase II trials with an older, well known, standard treatment. Other phase III trials compare treatments that are already in common use. Some treatments in phase III trials may be available outside the clinical trial setting.
  • Phase IV trial – Even after a drug has been approved by the United States Food and Drug Administration (FDA) for use in a particular indication, there may be need for additional studies. Phase IV clinical trials may be required by regulatory authorities or may be undertaken by the sponsoring company for a variety of reasons. For example, safety surveillance is designed to detect any rare or long-term side effects over a larger patient popula­tion and longer time period than was pos­sible during the phase I-III clinical trials.