NAFLD/NASH Clinical Trials Overview
Delve into the world of NAFLD/NASH treatment research, where promising drugs are undergoing rigorous clinical trials. Understand the phases of testing mandated by the FDA, and learn how participation in trials can offer access to innovative therapies while contributing to medical progress.
The first NASH/MASH drug (Rezdiffra from Madrigal Pharmaceuticals) was recently approved. That drug is designated for people with advanced fibrosis, but not yet advanced to cirrhosis. There are a number of other drugs in testing.
A drug cannot be brought to market in the United States without prior FDA approval. Clinical trials are conducted in order to get FDA approval. This is usually a lengthy and expensive process that requires the completion of three phases described by the U.S. Department of Health and Human Services as follows:
• A Phase I trial tests an experimental treatment on a small group of often healthy people (20 to 80) to judge its safety and side effects and to find the correct drug dosage.
• A Phase II trial uses more people (100 to 300). While the emphasis in Phase I is on safety, the emphasis in Phase II is on effectiveness. This phase aims to obtain preliminary data on whether the drug works in people who have a certain disease or condition. These trials also continue to study safety, including short-term side effects. This phase can last several years.
• A Phase III trial gathers more information about safety and effectiveness, studying different populations and different dosages, using the drug in combination with other drugs. The number of subjects usually ranges from several hundred to about 3,000 people. If the FDA agrees that the trial results are positive, it will approve the experimental drug or device.
Results of each phase are reported and evaluated upon completion before moving onto the next phase. A Phase VI trial is conducted after commercial use is provided to assure the drug’s effectiveness and safety.
If you are interested in participating in a clinical trial, we recommend searching for suitable trials using a search tool such as the one facilitated by Antidote (antidote.me). See the link below. This search tool uses your personal information to match you with an appropriate trial.
Why would you want to participate in a clinical trial? People generally participate for one or more of the following reasons:
• Involvement with innovative therapies for their condition
• Contact with medical experts for the duration of the trial
• Participating for the common good through contributing to medical research
While there may be compensation or expense reimbursement involved with trial participation, it is typically not enough that people participate for financial reasons. Also, be aware that, for those involved with NAFLD or NASH trials, liver biopsies will be part of the trial. Biopsies are required by the FDA as a measurement tool in assessing the effectiveness of the new drug.