FDA Clinical Trials

For a treatment or drug to be made available on the U.S. market it must first obtain approval by the FDA (Food and Drug Administration). Before a treatment or drug can be approved, it must first go through several phases of clinical trials in order to ensure safety and efficacy.

Pre-clinical Studies

Initial studies are done to collect data from laboratory and animal tests in order to determine preliminary safety and efficacy information on a drug or treatment. This step allows a company to determine if it is worthwhile moving forward with the proposed treatment.

Investigational New Drug Application

If a company decides to move forward with a proposed treatment following promising pre-clinical study results, it must file an Investigational New Drug Application (IND) with the FDA. Included in this application are animal pharmacology and toxicology studies, chemical and manufacturing information, proposed clinical protocols, and information on clinical investigators that will be conducting the trials.

Phase I Trials

Phase I trials are the first studies in humans and typically enroll only a small number of subjects. Trials are conducted in order to determine safety in humans, safe dosage range, and any possible side effects. If the treatment is safe and tolerated well by the subjects, it can move onto Phase II trials.

Phase II Trials

Phase II trials include many more volunteers and are conducted to determine if treatment is effective and to further evaluate its safety. If treatment is safe and effective it moves onto Phase III trials.

Phase III Trials

Phase III trials are done to determine how effective the new treatment is compared to the existing standard treatment and also to monitor side effects. These trials involve large groups of patients and are typically have a long duration.

Phase IV Trials

This phase, also called Post Marketing Surveillance, takes place after FDA approval and the treatment is available on the market for use. This phase provides for continuing evaluation of the treatment’s side effect on larger populations and after long-term use.

New Drug Application

Once efficacy of a drug or treatment has been demonstrated through clinical trials, the company files a New Drug Application (NDA) with the FDA for permission to market and sell the treatment in the U.S. All the data collected from animal studies and human clinical trials becomes part of the NDA.

California Stem Cell Progress

CSC is currently in the pre-clinical development phase of cell transplant therapies for ALS and SMA Type I and it may be some time before we are FDA-approved to start human clinical trials.