The Trial

Thursday, July 21st, 2016

I received the news from my dad today that I will be going to Nemours hospital on Monday to do the consultation for the treatment we have been trying to get in since the beginning of this year. I couldn't believe that I actually was going to take part of a trial, we've been-or at least I have been-dreaming of this day since I understood that I was different. I have had this disability since birth, so I know nothing else. But when I was younger, I had much more mobility and strength than I do now. I am a sixteen year old girl that has always had a dream of getting stronger, not weaker as I have been. Who wants to get weaker? But today was the day that I knew things were going to change, though I am not quite sure if it is going to be for the better or worse. This is a trial treatment, not a full blown we-know-this-will-work-enjoy-your-new-life treatment, so I'm testing out what side affects there are. This is an excerpt of what is said on one of the forms for my parents to sign.

"CK-2127107 is being investigated as a potential new therapy for improving skeletal muscle function, such as muscular weakness and muscle fatigue, in patients with SMA. The main purpose of this research study is to measure how the drug works in the body and the relationship between the amount of drug taken and its effect on the body. This research study will also look at the safety and tolerability of CK-2127107. By safety and tolerability, we mean we will collect information about whether there are any side effects from taking CK-2127107 that may make it difficult for patients to take it. Additionally, this research study will look at how much CK-2127107 gets into the blood stream and how long it takes the body to get rid of it. This study will also see if the drug changes how much force your child's muscles exert, how well he/she breathes, and how well he/she does a variety of activities such as rolling over and using his/her arms and hands. For those who walk, this study will also investigate if the speed or distance he/she walks is impacted by CK-2127107.
This is a randomized, double-blind, placebo-controlled, multiple dose research study where participants are selected at random (like by a flip of a coin) to either take CK-2127107 or placebo. "Randomized" means that participants will be assigned by chance (like by a flip of a coin) to get either CK-2127107 or placebo. Your child will have a 2 to 1 chance of receiving CK-2127107 over placebo, meaning it is twice as likely that participants will be assigned to active investigational drug as to placebo. "Double-blind" means that neither you, your child, the study doctor, nor any of the study staff will know if your child is receiving CK-2127107 or placebo, but this information will be available in case of an emergency.
If your child is eligible to participate in this research study, the study drug (CK-2127107 or placebo) will be given twice a day for eight weeks."

Sounds like fun, right? They also have to take blood from me, so this means I can't eat three hours prior to the visit. All I can have is water because it makes it easier to take blood from me.
To be completely honest, I'm kind of scared to do this trial. I could get better, I could get horrible side affects... Or I could get the placebo. There are so many possibilities that could happen, but I feel like I owe it to myself and my family to do this. No one is making me do this study, other than me, myself and I.

Well, it's around eleven thirty here and I have to get up early to go with my grandmother, Nanny, to an appointment for her. It'll be fun though, we get to have Panera for lunch.

~Rebel W.