Menu

The Zafgen Clinical Trial Partial Hold

I would like to assure our PWS members that both PWSA (USA) and FPWR have been involved with discussions with Zafgen and the FDA regarding safety issues on the Zafgen clinical trial. A “partial hold” means that the study can continue with additional safety monitoring. We are in full support of the additional safety monitoring and continuation of the trial. The additional monitoring will provide additional protection for patients enrolled in the trial.

We think it’s important to note three things about clinical trials in general:

1) Safety issues can emerge in clinical studies. The FDA, patient organizations, and the sponsoring company work diligently to protect all participants in a clinical trial, but clinical trials by definition are uncertain. Deaths in a clinical trial are highly unusual but serious adverse events can happen.

2) Serious adverse events can happen to individuals with PWS whether or not they are in a clinical trial. Just because an individual in a trial has an adverse event does not necessarily mean the drug caused it. Looking at recent sudden deaths in PWS reported to us at PWSA (USA), we are quite aware that there are several reasons for sudden deaths of people not on any clinical trials.

3) Without individuals willing to participate in clinical trials, we will never identify treatments that can help our community of loved ones. It’s important as a community that we continue to participate in clinical trials.

I want to especially thank Dr. Jim Loker for the extensive work he has done on the cardiac and vascular issues with PWS and the study of deaths that will be reported at the PWSA (USA) national conference in November. This information has been very valuable while we all search for an understanding of potential risk factors in PWS. The three of us (Jim Loker, Theresa Strong, and me) have been involved with this process and do understand the concerns of our families because we are PWS parents ourselves. My own son has enrolled in this clinical trial for the past 6 months and at this point he is definitely taking the drug. With the additional safety monitoring, I am comfortable in keeping him on beloranib.

 

Janalee Heinemann, MSW

PWSA (USA) Coordinator of Research & International Affairs

Share this Post!

About the Author : pwsa


0 Comment

Related post

  TOP