When a Clinical Trial Fails

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Published on June 29, 2021 | On June 1, the STAGED-PKD study was halted due to an inability to meet futility criteria. The study was looking at an investigational drug called venglustat to treat ADPKD. Over 500 patients signed up to participate. What does it mean to stop a clinical trial for futility? What does that mean for the patients who were taking the investigational drug?


The Stages of a Clinical Trial

The FDA is charged with oversight of all clinical trials going on in the U.S. at any time. A new drug must move through three stages before it can be reviewed for approval by the FDA.

Phase I

The new drug is tested for safety and side effects in a small number of healthy volunteers, and some cases, volunteers with PKD

Phase II

Using a small number of volunteers with PKD, the new drug is tested for safety, dose ranging, and preliminary effectiveness.

Phase III

The new drug is tested in a large number of volunteers with PKD to establish effectiveness, monitor side effects, and compare results with current treatments. The data collected during the clinical trial is analyzed and then submitted to the FDA for regulatory review. This can take one to three years. If there’s an intent to apply for an NDA (new drug application), it’s communicated to the FDA and shapes the design of the study. Once it’s approved, the new drug can be prescribed by physicians to treat PKD.

Phase IV

These studies are done after the drug has been approved by the FDA and it’s in use (considered a post-marketing study). Additional information about risks, benefits, and optimal use is collected and analyzed.


Ending a Clinical Trial for Futility

“Futility” means a clinical trial is very unlikely to provide evidence that an investigational therapy will impact disease progression. Although the therapy may be safe based on earlier phase trials, data from the current study is sometimes analyzed before the study ends. This is to make sure that there’s a reasonable chance that a treatment benefit may be observed before continuing with the often years-long trial. If in the case of venglustat in ADPKD, little difference is seen between the kidney function, size, and/or disease progression of those taking the study drug compared to those on placebo (a sugar pill), the study can be halted. This decision was not made due to safety reasons.​​

“Although the STAGED-PKD study was carefully designed, the interim analysis of the available data did not demonstrate the meaningful effect of venglustat on annualized total kidney volume change. This news disappointed participants, coordinators, and organizers who were committed to this study. Hopefully, the knowledge gained during this clinical trial will inform future studies that will improve the lives of people with ADPKD.”

—Michal Mrug, M.D., Study Investigator


The Benefits of a Clinical Trial Ending

This is obviously disappointing for the entire PKD community who hoped the clinical trial would lead to a new FDA-approved treatment option for patients. But it can also have a few benefits:

  • Participating in a clinical trial can be a heavy burden on patients. Even more so if they have to take time off work or travel far to the study site. This decision helps to ensure that ADPKD patients will be able to participate in clinical studies with the best potential to be safe and effective
  • Participants may be eligible for other clinical trials that show more potential benefit
  • Resources can now be redistributed to other, more promising research

“It was very disappointing to hear from my study doctor that the trial was ending early. I’m near stage 4 now and most treatments are too late for me. But I want to advance treatments for my two children who have PKD. I hope there will be more available for them than there was for me and my mother.”

—STAGED-PKD Clinical Trial Participant


How to Increase the Odds of Finding a Treatment

Although it’s exciting to see multiple therapies in the research pipeline for PKD, we can expect that some won’t make it into clinical practice. Some studies end early, like STAGED-PKD; some continue to the end but are rejected by the FDA because they show little benefit to the patient. Then there are a small few that are reviewed by the FDA and approved for certain patients, like tolvaptan. What’s the best way to keep the PKD research pipeline full of clinical trials and increase our chances of getting a therapy through research obstacles? Sign up to participate and help provide the data to tell investigators whether a drug might impact the disease. We can help connect you to ongoing clinical trials in your area through our Act Alert program.

To those patients who signed up to participate in STAGED-PKD, thank you! There are a few other studies you may qualify for—check them out here.


Interested in learning more about clinical trials? Read more FAQs here.



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