Relapsed Refractory Multiple Myeloma (RRMM) Glossary

Chronic: Persisting over a long period of time.

Clinical trial: 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.

• Accrual – The process of enrolling patients in a clinical trial, or the number of patients already enrolled or anticipated to be enrolled in a clinical trial.

• Arm – A treatment group in a randomized study, in which there are two or more arms.

• Cohort – A group of patients in the same study receiving the same treatment or placebo (no treatment).

• Control group – The arm of a randomized clinical trial that receives the standard treatment or placebo.

• Endpoint – The goal of the study. A clinical trial endpoint may aim to measure toxicity, response rate, or survival.

• Experimental group – The arm of a randomized trial that gets the new treatment.

• Randomized clinical trial – A study in which patients are randomly assigned to receive a particular treatment.

• Double blind – When neither the patient nor the investigator knows the arm of the trial to which the patient is randomized. The purpose is to eliminate any bias in the reporting of results.

• Phase I clinical trial – A study to determine the maximumtolerated dose (MTD) and safety profile of a new drug or a new combination of drugs. It may be the first testing of a new treatment in humans. Please note that in combination therapies, the individual elements may already have been well tested in humans.

• Phase II clinical trial – A study designed to determine the efficacy and safety of a new therapy that has been tested in a phase I trial. Patients are usually required to have measurable disease that is refractory to any standard treatment. If the results of a phase II study are clearly much better than the standard treatment, then the treatment may be approved without being tested in a phase III study. If results from a phase II study are promising, the treatment may then be tested in a phase III study.

• Phase III clinical trial – A study that compares two or more treatments. The endpoint of a phase III study may be survival or progression-free survival (PFS). Phase III studies are usually randomized, so patients don’t choose which treatment they receive. Some phase III trials compare a new treatment that had good results in phase II study with a standard -of-care treatment; other phase III studies compare treatments that are already in common use.

• Phase IV clinical trial – Even after a drug has been approved by the U.S. Food and Drug Administration (FDA) for use in a particular indication, there may be need for additional studies. For example, safety surveillance is designed t o 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.