Understanding Gene Therapy Clinical Trials

What is the primary purpose of preclinical studies in gene therapy?

• To finalize the dosage for human trials

• To assess the treatment's effectiveness and safety in test tubes and animals

• To determine the market potential of a treatment

• To test the treatment on humans

What is the role of the FDA in the clinical trial process?

• To provide funding for the trials

• To market the new treatment

• To review the investigational new drug application and ensure volunteer safety

• To conduct the clinical trials

During which phase of clinical trials is the treatment tested on a larger group to confirm its effectiveness?

• Phase 1

• Phase 2

• Phase 3

• Preclinical phase

What happens if a clinical trial phase is not deemed safe?

• The trial is immediately approved

• The trial is conducted on a smaller group

• The trial moves to the next phase

• The trial is halted and does not proceed to the next phase

How long can the entire process from clinical trials to FDA approval typically take?

• Eight years or more

• Two years

• Six months

• One year