10/14/2021
CRITICAL RESEARCH STUDY ANNOUNCED!!
The following was written by Dr. Miller:
“Running for Research is proud to announce our fundraising study topic for the 2022 campaign. This year, the money raised will fund a multi-center trial of Diazoxide Choline Controlled-Release (DCCR) in patients with Prader-Willi Syndrome who are in Nutritional Phases 1b, 2a, or 2b. This study will be conducted at 4 sites across the United States, including: University of Florida, University of Louisville, the University of Minnesota and Nationwide Children's Hospital in Columbus,Ohio.
The goals of this study are to evaluate the effects of DCCR in pre-hyperphagic individuals with PWS on body composition, insulin resistance, appetite progression, and sleep, amongst other metabolic parameters. This will be an open label trial with no placebo being given. Patients who wish to participate but do not want to be on an investigational medication will serve as the control group for the study.
For both groups of participants, there will be 3 in-person visits the first year of the study (screening, baseline, and 1 year) and then annual visits thereafter. Interim visits will be conducted using telemedicine and interim blood work and safety analysis will be performed locally in between the in person site visits by local lab/pediatrician. Dose of DCCR will be titrated up slowly to the maximum dose (4 mg/kg/day) noted to be clinically effective in previous trials.
This study is being run by the investigators at each participating site and will be sponsored exclusively through Running for Research PWS. DCCR is made by Soleno Therapeutics, and they will be supplying the investigational medication for the study, but are not involved in the funding or conduct of the study.“
RFR is absolutely thrilled to be involved in making this study a reality. As you know, DCCR has shown very promising results in the treatment of hyperphagia in persons with PWS/Phase 3. Dr. Miller believes DCCR has the potential to be a preventative medication in those individuals who have yet to develop hyperphagia. If this theory proves true, it will completely change the trajectory of the development of PWS. Imagine the possibilities for our loved ones. This is what our community has been hoping for for so long!
Because this study will be 100% donation financed, we must raise a minimum of $ 150,000 to begin the initial phase. So far, RFR has raised a bit over $52,000 so we have some work to do. But do it we will! NOW we must come together and gather the resources necessary to make it happen.
I want to reassure everyone who has raised money and donated to prior campaigns that this announcement will have NO IMPACT on previously planned and funded study topics( gut microbiome, hypoglycemia and a new medication for skin picking/hair pulling). These studies will move forward as promised.
I also want to make very clear that participation in our fundraiser and/or donating to RFR will NOT guarantee placement in this study or any of the other studies RFR funds. All study recruitment will follow clear and concise guidelines and anyone who fits the recruitment profile will be considered for study placement.
Please help us get this study off of the ground! Visit our website: www.runningforresearchpws.com to register and/or to make a donation.
Thank you!
Running for Research ♥ Prader-Willi Syndrome (RFR) came into being in April of 2018. It was born from a conversation between Kelly Shad Guillou and Dr. Jennifer Miller. Kelly and her daughter Clementine, a child who lives with PWS, were visiting Dr. Miller at the University of Florida for a regula...
