Below is a simple explanation of the different stages in the development of new treatments:
- Observational study | This early phase allows researchers to better understand the natural progression of the disease they are studying. While experimental drugs are not typically provided during observational studies, participants are often able to continue taking existing medications prescribed by their own physicians.
- Phase 1 trial | Before new medications are approved for human patients, researchers give the experimental drugs to a small group of healthy volunteers. Researchers determine appropriate dosage and test for safety and side effects.
- Phase 2 trial | Once an experimental drug is deemed safe for patients, it is administered to a small group of patients to see if it improves health outcomes for the condition being studied and to continue to evaluate safety.
- Phase 3 trial | Once data demonstrates an experimental treatment is both safe and effective, a larger group of patients are given the drug. Researchers continue to evaluate the safety and determine if the new drug is more or less effective than current treatments.
- Phase 4 trial | After the new treatment is marketed to patients, researchers continue to assess the drug’s safety and efficacy.
Alport syndrome Patients, family members, and physicians are invited to join the patient registry. When a new trial is announced, the patient registry will contact any registry participants who meet the study protocol requirements. To learn more about and/or join the U.S. patient registry, contact ASF Staff.