- About Fragile X
- About FRAXA
- Toward a Cure
- Get Involved
- Ways to Give
To those of you who have family members with Fragile X, thank you for being interested in clinical trials! This is a challenging phase for all of us, and as exciting as it is, we know many of you have questions about what it means to be in a clinical trial.
Each study is supervised by a doctor that specializes in Fragile X. These trials are free, you do not have to tell your insurance company, and you can leave any time you want. Travel costs are usually covered.
The FDA requires two successful large-scale clinical trials before it will consider approving a new treatment. If studies are successful and the FDA approves new drugs, others with Fragile X can also have access to these medicines.
Informational webinar by Dr. Alex Kolevzon, 10/15/2014
NNZ-2566 has a novel mechanism of action compared with other drugs that have been tested for Fragile X syndrome. It is a modified version of a biologically active part of Insulin-like Growth Factor (IGF-1). IGF-1 is a protein that helps cells grow and respond to stress or damage. In the brain, IGF-1 contributes to the growth of brain cells and the connections between them (synapses).
The biologically active part of IGF-1 is called glypromate or GPE and supports the function of brain cells in a different way than IGF-1. GPE occurs naturally in the brain. NNZ-2566 is a version of GPE modified to make it available to take as an oral medication. NNZ-2566 may help improve brain function by enhancing functions normally performed by GPE and by increasing IGF-1 levels in the brain. NNZ-2566 has been shown in animal models of Fragile X to improve performance in tests of behavior and cognition.
FDA has granted two special designations for Neuren’s development of NNZ-2566 in Fragile X: Fast Track Designation and Orphan Drug Designation.
This trial will include 60 participants between the ages of 14-40. Taking part in this study involves 8 visits to the doctor’s office over 10 weeks. The study medication and all study-related evaluations will be provided at no cost. Some support for travel is available.
UC Davis MIND Institute
Principal Investigator: Randi Hagerman, MD
Contact: Lindsey Partington 916-703-0427 email@example.com
Emory University – Recruiting
Principal Investigator: Jeannie Visootsak, MD
Contact: Krista Charen 404-778-8479 firstname.lastname@example.org<
Rush University Medical Center
Principal Investigator: Elizabeth M Berry-Kravis, MD
Contact: Jamie A Chin (312) 942-0025 Jamie_A_Chin@rush.edu
University of Massachusetts Medical School
Principal Investigator: Jean Frazier, MD
Contact: Ann Foley 774-455-4103 email@example.com
|NYC, New York (click for flyer)|
Mount Sinai School of Medicine
Principal Investigator: Alexander Kolevzon, MD
Contact: Hillary Rieger 212-241-2826 firstname.lastname@example.org
Suburban Research Associates
Principal Investigator: Shivkumar Hatti, MD
Contact: Nathan Blubaugh 610-891-9024 ext 103 email@example.com
or Contact: Nikki Thomas 610-891-7200 Nthomas@suburbanresearch.com
Children’s Hospital Colorado
Principal Investigator: Nicole Tartaglia, MD
Contact: Nanastasia Welnick 720-777-8608 Nanastasia.Welnick@childrenscolorado.org
Alcobra’s metadoxine trial has completed recruiting. Learn more about it at Clinicaltrials.gov at http://clinicaltrials.gov/ct2/show/NCT02126995
For all Fragile X medication trials visit http://www.clinicaltrials.gov/ct2/results?term=fragile+x
To learn how trials work, visit http://clinicaltrials.gov/ct2/info/understand