Information in this section is adapted from the National Cancer Institute website and other sources believed to be reliable.

Clinical trials are research studies conducted with the hopes of determining safer and more effective means to treat serious illnesses. Before the U.S. Food and Drug Administration (FDA) can approve a new treatment for human use, it must undergo extensive testing to determine its safety and efficacy in treating a specific disease. Human clinical trials conducted with patient volunteers typically follow years of preclinical research and development in laboratories and animal studies.

Preclinical studies are conducted to determine potential new treatments for specific disease indications. During a new drug's preclinical development, the sponsor's primary goal is to determine if the product is safe for initial use in humans and if the compound exhibits pharmacological activity that justifies commercial development. Animal models are frequently used to help determine a treatment's safety prior to introducing it to humans. When a product is identified as a viable candidate for further development, the sponsor will prepare and submit an Investigational New Drug (IND) application to the FDA.

An IND must contain information in three broad areas:

• Animal Pharmacology and Toxicology Studies – Preclinical data to permit an assessment as to whether the product is reasonably safe for initial testing in humans. 
• Manufacturing Information – Information pertaining to the composition, manufacturer, stability, and controls used for manufacturing the drug substance and the drug product. This information is assessed to ensure that the company can adequately produce and supply consistent batches of the drug.
• Clinical Protocols and Investigator Information – Detailed protocols for proposed clinical studies to assess whether the initial-phase trials will expose subjects to unnecessary risks

Phases of Clinical Study

Phase 1 Trial: Clinical trial conducted to evaluate the safety of a drug or therapy. How a drug should be administered (e.g. oral, injection) and dose levels are also evaluated. Phase 1 trials typically involve a small group, depending on the disease indication.

Phase 2 Trial: Clinical trial conducted to further evaluate the safety of a drug or therapy and to evaluate its efficacy. In addition, researchers focus on determining optimal dose levels. Phase 2 trials typically involve a larger group than Phase 1 trials, depending on the disease indication.

Phase 3 Trial: Clinical trial conducted to confirm the efficacy of a new drug or therapy. Trial participants are usually included in one of two study groups: one which will receive the new drug being evaluated, and one that receives the already approved, current standard of treatment. Phase 3 trials can enroll anywhere from 100 to thousands of patients.

During Phase 3 trials, study sponsors will begin to assemble an application, either a Biologics License Application (BLA) or a New Drug Application (NDA), for submission to the FDA demonstrating the investigational drug or therapy's safety and efficacy based on the data gathered from the clinical trials. The FDA will evaluate the submission and make a determination as to the approvability of the application. Drugs and therapies that are approved by the FDA can then be commercially marketed throughout the United States.

People interested in taking part in a clinical trial should talk with their doctor. Information about clinical trials is available from the Cancer Information Service of the National Cancer Institute (NCI) in a booklet Taking Part in Clinical Trials: What Cancer Patients Need to Know. This booklet describes how research studies are carried out and explains their possible benefits and risks. This booklet is available online at:
http://www.cancer.gov/clinicaltrials/Taking-Part-in-Cancer-Treatment-Research-Studies. In addition, the NCI’s Web site, http://www.cancer.gov/clinicaltrials, provides information about clinical trials. It also offers detailed information about specific ongoing studies by linking to PDQ, a cancer information database developed by NCI.

Helpful Links to Online Resources:

NCI Head and Neck Cancer Home Page:
http://www.cancer.gov/cancertopics/types/head-and-neck/

NCI Fact Sheet: Head and Neck Cancer: Questions and Answers
http://www.cancer.gov/cancertopics/factsheet/sites-types/head-and-neck/

NCI - Support and Resources
http://www.cancer.gov/cancertopics/support

NCI National Service (Support Group) Organizations Database
https://cissecure.nci.nih.gov/factsheet/FactsheetSearch.aspx?FSType=8.1

Medline Plus: (a service of the National Library of Medicine and the National Institutes of Health):
http://www.nlm.nih.gov/medlineplus/headandneckcancer.html

The American Cancer Society:
http://www.cancer.org

The American Academy of Otolaryngology: http://www.entnet.org/HealthInformation/headNeckCancer.cfm

American Head and Neck Society:
http://www.headandneckcancer.org/patienteducation/index.php

There is additional information on the Internet regarding clinical trials being conducted in multiple disease indications including at:
http://www.clinicaltrials.gov

Note: IRX Therapeutics does not endorse the information included in the above websites.