A Parent’s Perspective on Informed Consent Philip Moss We Consent: A Parent’s Perspective on Informed Consent Philip Moss

Clinical Medical Trial Not a medical expert in any way….Just a dad. Not “eager” to participate. Scary Many unknowns Side effects Short term –fatigue, nausea, headaches, Long term (not known as children have only taken drug for 1 year). Can a minor “consent” Google is our friend And social media connections of families with the same condition. Other patients help us know what to ask and what to expect.

New Terms for us… Protocol

More “new” terms. Chemotherapy

But Wait…how did we get here? Early 2011. We notice swollen lymph glands and Philip took 2 rounds of antibiotic to shrink it. September 2011- outpatient surgery to remove swollen gland. Mid-September pathology of tumor came back as Neurofibromatosis Neurofibromatosis Type 1 Genetic condition that can be found in 1 in 3,000. Tumors can grow on nerve cells anywhere in the body. No “cure” for condition but some treatment of symptoms. MRI to determine growth rate of tumor. 9 MRI’s between January 2012 and April 2015

MRI of my sons neck View from above looking at top of head Side view of neck…showing it does not extend to brain.

1st Chemo Experience August 2014 began a chemo through UAB. Chemo failed to shrink the tumor. Monthly blood draws to monitor various “levels”. After another MRI it was determined drug was not effective. April 2015 removed from drug Told to go home and wait on a clinical trial that our son could possible qualify to participate…

We are “IN” August 2015 we learned we were accepted to participate in a Phase II study at the NIH in Bethesda, MD. We traveled there for 6 days of qualifying procedures and baseline tests. Got to go to a Nationals game, meet Stephen Strasburg, and walk onto the field!

Assent As a minor (10) can he agree to participate? Clinical trial nurse and medical team did a great job of talking with him and making sure that he understood the possible side effects. In our case, both parents and minor agreed to participate.

Qualifying for participation in trial Echocardiogram Eye Exam

More protocol Per terms of the study, we traveled to NIH 6 times during the first year. 2 trips were simply blood work. 4 trips involved extensive medical check-ups. 4 trips included MRIs including the most recent in early August.

New MRI In April, after 12 sedated MRIs in his lifetime, Philip was able to stay still long enough for a non-sedated MRI. In fact, he was so relaxed in August he fell asleep in the MRI machine!

History of tumor growth Tumor responded to treatment on clinical trial medication. Steady growth rate from 2012 until enrolling in the clinical trial. Steady decline in volumetric analysis. Now down 36%! Increase dosage and will continue to visit NIH 3-4 times per year.

Proof that clinical trial has been successful

Participation in Social Media

Further Assent We, along with Philip, agreed to allow a film crew capture our medical exams and interview us after many of them. Film crew followed up for qualifying and 4 of the next visits! We were asked not to disclose details of this show as producers may not use our footage.

Questions?