Clinical Trials

Please Participate in HAE Clinical Trials and Fill Out the HAE Association's Clinical Trial Interest Form

Currently there are three companies conducting clinical trials with the goal of getting their HAE treatments. Rare diseases and small patient populations usually do not receive this much attention, so we must take full advantage of this "once in a lifetime opportunity" for approval of several different HAE therapies. However, nothing will happen unless we make sure each of these trials has sufficient participants. We hope each and every HAE friend will help themselves and other family members by participating.

HAE patients interested in clinical trials can either fill out our Clinical Trial Form, or contact The HAE Association's Clinical Trial Programs Director Donna Davis.

Please note: Any information you provide to the HAE Association is carefully safeguarded to eliminate even the slight possibility of unauthorized disclosure.

Once you have completed the form, Donna Davis or one of her Clinical Associates will contact you with information regarding clinical trial sites in your area. Alternatively, if you have frequent attacks and indicate that you are willing to travel and stay in a hotel near a trial site (travel and lodging expenses will be paid by the trial sponsor), you will be contacted with information regarding how this could be arranged. It is important to note that the HAE Association will work with you, your doctor, and the pharmaceutical company to make sure you have medicine available so you can get treated for attacks that occur after your travel is completed.

Below is some information regarding the companies looking to license their medicines in the United States

Jerini AG (Firazyr) conducted a US clinical trial that did not meet the statistical significance requirements for effectiveness required by the FDA. Firazyr is delivered through a subcutaneous injection and works by blocking the peptide that causes HAE-related swelling. The product has been licensed in Europe and Jerini is now working on plans to conduct another clinical trial in the US.
Pharming NV (recombinant C1 inhibitor) announced successful clinical trial results for their product, Rhucin-a recombinant human C1 inhibitor protein that is derived from the milk of genetically altered rabbits. Clinical trials have shown that Rhucin is a safe and effective treatment for acute HAE attacks. The company plans to apply for a US license in the near future.