Patient Services

Clinical Trials

What Are Clinical Trials ?

Clinical trials are the rigorous, scientific studies of new drugs in development. They are administered in a carefully controlled process by which patients’ rights and well-being are always paramount. Every new drug has to go through the clinical trial process before it is FDA approved, including all the “miracle drugs” of the last decade (such as Gleevec®, Rituxan®, and Herceptin®.

Clinical Trial Phases

PHASE I

Phase one clinical trials are the first study of a new agent in humans. The drug has gone through exhaustive testing in cell lines and animals, and appear promising for humans.

PHASE II

Phase two clinical trials utilize the information gained from Phase one testing to find the safest and most effective dose and its schedule. The new agent is then offered to appropriate groups of patients.

PHASE III

Phase three clinical trials determine if a new therapy is an improvement in comparison to standard of care.

More Information about Clinical Trials

Genetic Diseases

TCRF also has programs for two genetic disorders. Gaucher disease is a rare genetic disorder caused by a defective gene, which prevents the body from producing an important enzyme called glucocerebrosidase. Dr. Barry E. Rosenbloom directs The Comprehensive Gaucher Treatment Center at Tower Hematology Oncology Medical Group.

For more information, please visit the Gaucher West website.

Pompe disease is a rare, inherited genetic disease that disables the heart and muscles that results in a deficiency of the enzyme alpha-glucosidase (GAA).

For more information regarding our clinical trial or genetic disease programs please contact:

Marie Fuerst, RN, MS, Director of Clinical Research Services
(310) 285-7242