Enrolling in a Clinical Research Trial

Is enrolling in a clinical research trial right for you? This depends on a number of factors, including your medical condition.

• Find out about clinical research trials that are available for HAE.
• Carefully research the locations, treatments, and protocols for these medications.
• Contact a clinical research trial site coordinator to get answers to specific questions you may have about clinical research trials.
• Does the study allow for additional treatment dosage if the first dose does not bring you rapid relief?
• Does the placebo group receive treatment after a specified duration of time? Or receive “rescue” medication or pain medication during an attack?
• What should happen if you have a life-threatening attack?
• Has the investigational medication been used in other countries? If so, for how long? And with what success rate?
• What assistance will the sponsor company provide in terms of transportation or hotel accommodations?
• Is the medication identical to what’s been used overseas? Or is it similar?
• What is known about the medication’s effectiveness and safety in other countries? What side effects, if any, can you expect? How long do these side effects last?
• If you are currently taking an HAE medication, will you need to discontinue it? If so, what are the risks or possible benefits?
• How long has the sponsor company existed?
• What is the likelihood of the company having the resources to get FDA approval and provide an adequate supply of the medication once it is approved?
• What medical tests, office visits, and treatment costs will be covered in the clinical research trial?
• What other medications has the company studied and/or brought to market? What is its track record?

Only you can decide whether enrolling in a clinical research trial is the right decision for you and your family. Weigh the short- and long-term benefits against personal costs. Be sure that you have all the facts and that all your questions have been answered to your satisfaction.