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Patient Perspective: Frankie's Experience Participating in a Clinical Trial

PatientPerspective Frankie1Hello! My name is Frankie Perazzola, 24 years old and diagnosed with FA at age 22. I started STEADFAST/Actimmune/ Interferon Gamma in February 2016 which later came to an unexpected stop in November of 2016. This drug was supposed to increase levels of frataxin which all FA patients lack. Since frataxin is essential for energy production, Actimmune was expected to increase the specific protein which would potentially increase energy levels and possibly increase mitochondrial function. Two simultaneous shots, every other day were required by injection in my arms and legs. The first portion of the study, I did not know if I was injecting the drug or a placebo. However, I was guaranteed the drug for the second portion and even had the option of continuing until approval by the FDA- which I later opted for. 

Another option I went for was the muscle biopsies- which is essentially a mini operation requiring a small incision on the thigh that leaves you a tad sore afterwards. No anesthesia required. I learned through my main neurologist, Dr. Perlman, that muscle biopsies were a better way to measure frataxin levels. By better, I mean a more accurate way to measure the levels compared to just giving blood and cheek swabs, because muscle is considered affected tissue. If I was participating in a trial, I wanted to give everything I could in order for my results to be as precise as possible. 

PatientPerspective Frankie2The reason the trial was stopped was because the drug did not meet certain clinical endpoints or biomarkers that were set at the beginning that would demonstrate evidence for approval. As a patient who was getting injections six times a week, spending tremendous amount of time at the doctor’s office and giving endless amounts of blood and muscle tissue to this trial- it was definitely frustrating to hear it was cancelled. I felt like I had wasted my time until I was able to reflect on the process. Because of this trial, I had access to one of the top neurologist in the US who specifically deals with Friedreich’s Ataxia. I learned copious amounts of knowledge about my body and what to expect with my progression. The fear I had of needles was completely diminished. And most importantly, I helped in contributing to the research, which is the only to determine if a drug is working. This research didn't have the outcome I was hoping for but it is the only way we learn and can move treatments forward to the whole community.

I felt needed and that I was directly impacting the scientific process of curing FA. I felt important. To be honest, I truly enjoyed the experience. I met other FA patients and family in passing during our doctor visits which has inspired me to fight even harder. To anyone debating on whether to participate in a trial- educate yourself and do it. Decide whether or not you agree with the drug and the process for you or your child and dive in. FA is rare as we all know, so the more people we have participating, the faster we can figure out a treatment which will fast track our way to eventually discovering a cure!

To find a list of studies that are looking for volunteers, please visit curefa.org/active-clinical-trials.

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