Clinical Trials

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 marketed 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 with 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.
The results of each phase are reported and evaluated upon completion before proceeding to the next phase. After commercial use is provided, a Phase VI trial is conducted 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 by 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. The FDA requires biopsies as a measurement tool in assessing the effectiveness of the new drug.

The US Department of Health and Human Services has published a list of questions to consider when considering trial participation. Here is the link: https://www.hhs.gov/ohrp/education-and-outreach/about-research-participation/questions-to-ask/index.html