A major milestone in the journey to our gene therapy clinical trial has arrived – the official registration of the natural history study with the clinical trial.gov website. Even more exciting? This natural history study covers two types of Batten disease – CLN7 and CLN5.
If you are like me when I first heard of a clinical trial study, you have a few questions like, “what is a natural history study?” or “why can’t we just get to trial already????”
Both good questions. Maybe I can offer some insight from one newbie rare parent to another. Here are a few frequently asked questions about natural history studies, and the CLN7 and CLN5 trial study in particular!
Frequently Asked Questions about the Batten disease type CLN7 and CLN5 Clinical Trial Study:
1. What is a clinical study? What is a natural history study? Why is this needed??
A clinical study is a study designed to evaluate efficacy of an unproven treatment. A natural history study is a study of patients with a specific disease like Batten CLN7, to understand and document the progression of a disease for the purpose of evaluating the success of a future experimental treatment. Pretty much we need to complete this study so we can have data to show the treatment in the clinical trial works and it is a must have before we get to actual gene therapy trial. We are so grateful that the study is almost here!
2. When is the natural history study for CLN7 and CLN5?
The CLN7 and CLN5 clinical trial natural history study will initiate in Feb 2019 and run for several years. Participants will be asked to meet with study doctors in person initially.
3. Who benefits from the CLN7 natural history study?
Observational data from the study will be used to complete the proposal for and evaluate the success of the gene therapy clinical trials for CLN7 and CLN5. This study creates a foundation of knowledge used in the trial, as well as for future study. My hope? All families confronted with these diseases in the future will ultimately benefit from this work today.
4. What will be done in the CLN7 and CLN5 natural history study?
Participants will be evaluated over the course of approximately one week to generate a baseline of abilities including motor skills and cognitive levels. Different tests and evaluations will be used to compare our kids progression to a standard scale of regression established for Batten disease.
5. Will my child qualify for the clinical trial if we participate?
The natural history study is not intended to be used as a means of deciding trial participants, but to establish a baseline of the disease progression. A separate criterion will be used to determine participants. Inclusion criteria will always prioritize patient safety for participation in clinical trial.
6. Do I have to pay to participate?
The CLN7 and CLN5 participant evaluations will be sponsored by the trial sponsor, not by individuals. A call center has been set up to help you in the path to participation. If you would like to talk about support to participate please contact the call center at 1-877-237-5020
7. What if I want my child to participate but cannot travel?
You can still make a huge difference without traveling!! Records of MRIs, EEGs, genetic test results, and parent observations of disease progression are all helpful to the success of the CLN7 and CLN5 natural history study. Information can be submitted remotely, please contact the call center at 1-877-237-5020
8. I have more questions – who can I contact?
To get the latest information on the natural history study, please contact 1-877-237-5020. To review the posting for the CLN5 and CLN7 natural history study, check out clinicaltrial.gov