Hannah’s Hope Fund (HHF) is funding a Natural History Study (NHS) at Columbia University Medical Center, in NYC. Dr. Douglas Sproule is the Principle Investigator (PI) of this study. The purpose of this study is to track the untreated course of the disease, with the goal of arriving at clinical outcome measures. For any clinical trial, you must tell the FDA how you plan to measure efficacy of an experimental treatment. In order to do this, you must obtain pretreatment data sets, to compare to post-treatment data sets to see if the experimental treatment has slowed progression, stopped progression, sped progression or had no impact on disease progression. Because so few GAN patients exist, it’s important parents seriously consider having their child participate in the NHS. All travel and medical expenses are paid for by HHF. The study consists of 5 visits to Columbia Univ., spread 6 months apart. If you are not able to travel, or do not wish to participate in the NHS, your child’s medical record, past MRIs on CD, nerve conduction velocity results etc. are all valuable to the natural history study.
When at Columbia for the Natural History Study, you will also be asked (both parents and affected child) to consent to a skin punch biopsy. This precious tissue is critically important to GAN research toward an eventual cure. Please reference GAN Cell and Tissue Bank tab for more information this.
Please collect all medical record information and forward it to Jonathan Marra, the clinical resource coordinator for the NHS. To learn more about the NHS study or to send medical record information, please contact Jonathan at: firstname.lastname@example.org.
Click here for the link to the recruitment advertisement for this study.