Clinical Trials & Studies for Mitochondrial Disease

Clinical trials are a key component of research. Scientists use medical knowledge to ask and answer scientific or medical questions in areas such as medication, medical device, teaching concepts, or behavioral change. Researchers first test new therapies in labs and/or animals. Treatments that are safe are moved into clinical trials to move the drug or therapy toward approval by the FDA.

Commonly Used Terms

This list was developed to provide easy-to-understand definitions of commonly used terms in clinical trials. Commonly Used Clinical Terms

Types of Clinical Trials

Treatment

Test new treatments or devices, new combinations of drugs, or new approaches to surgery or radiation therapy. Treatment trials are often categorized by phases I through III.

Natural History

Provide information about how health and disease progress over time.

Prevention

Evaluate the effectiveness of ways to reduce the risk of developing a disease or preventing a disease from returning.

Diagnositic

Develop better tests or procedures to identify/diagnose a particular disease or condition.

Screening

Assess new ways of detecting disease earlier in healthy people.

Quality of Life (or Supportive Care)

Evaluate measures to improve comfort of and quality of life for people with chronic illnesses through better therapies or psychosocial interventions.

Clinical Trial Phases

Researchers test an experimental drug or treatment in a small group of people (20–80) for the first time, often using healthy volunteers to test drug metabolism, safety, and dose ranges.

The purpose is to evaluate its safety and identify side effects.

The experimental drug or treatment is administered to a larger group of people (100–300) to determine its effectiveness, optimal therapeutic dose, and to further evaluate its safety. This phase may be blinded (unknown if patient receives the study drug) or open label studies (known if patient receives study drug) in the target population.

The target population encompasses the group for which the drug is ultimately intended. Any drug metabolism, safety, or dosing difference between the Phase I healthy individuals and the Phase II target population is also evaluated.

Hints of efficacy may be obtained as well.

Researchers test an experimental drug or treatment in a small group of people (20–80) for the first time, often using healthy volunteers to test drug metabolism, safety, and dose ranges.

The purpose is to evaluate its safety and identify side effects.

Researchers test an experimental drug or treatment in a small group of people (20–80) for the first time, often using healthy volunteers to test drug metabolism, safety, and dose ranges.

The purpose is to evaluate its safety and identify side effects.