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.
Clinical Trials and Studies recruiting patients
Combining Lovastatin and a Parent-Implemented Language Intervention in a Multimodal Treatment for Fragile X Syndrome - UC Davis MIND Institute
The study is testing the efficacy of a multi-modal treatment comprised of the medication lovastatin or placebo, and a Parent-Implemented Language Intervention (PILI) in children and adolescents with FXS between the ages of 10 and 17.
The study involves 3 visits to the MIND Institute over 20 weeks as well as weekly video conferencing sessions in the home to learn strategies to help the child with language. Study visits include behavioral and language assessments, a medical history, physical exams, parent questionnaires, and blood and urine sampling.
Please contact the study coordinator Erika Bickel at email@example.com or (916) 703-0281.
MRI Study in Children with Fragile X Syndrome or Down Syndrome - U Mass Medical Center
PET and MRI Fragile X Study - National Institute of Mental Health
Recent Trials and Studies Awaiting Results
- 3/2016 - Analysis of Developmental Brain Dysfunction (DBD) in Families with Fragile X Syndrome - sponsored by FRAXA Research Foundation
Principal Investigators: Margaret King and Brenda Finucane, Geisinger Health System.
This study’s goal is to help design future Fragile X clinical trials more effectively and to help predict which potential treatments would be most helpful to individuals.
- 2/2016 - Double Blind Crossover Trial of Ganaxolone - sponsored by FRAXA Research Foundation
Investigators: Frank Kooy and Anke Van Dijck- Antwerp, Belgium. This trial has completed recruitment and results are being analyzed.
- 2/2016 - Clinical trial of Ganaxolone in children with Fragile X - sponsored by Marinus Pharmaceuticals at the MIND Institute, UC Davis. This trial is still running but recruitment is complete.
- 2012-15: Epigallocatechin gallate (mega green tea extract) – Mara Dierssen – Barcelona, Spain. Trial results are being analyzed.
Completed Clinical Trials With Results
- 12/2015 - Neuren’s trofinetide showed some success in small Phase 2 clinical trials in Fragile X syndrome and in Rett Syndrome
Trofinetide 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. IGF-1 contributes to the growth of brain cells and the connections between them (synapses).
FDA has granted two special designations for trofinetide in Fragile X: Fast Track Designation and Orphan Drug Designation. Trofinetide has a different mechanism of action from other drugs that have been tested for Fragile X syndrome.
- Alcobra's metadoxine trial has completed. Learn more at Clinicaltrials.gov
- 2012-13: Open-Label Pilot Trial of Lovastatin - Francois Corbin, MD, PhD — Sherbrooke, Canada
- 2008: Minocycline trial - Carlo Paribello, MD - Toronto, Canada. Positive results were published
- a pilot lithium trial — Elizabeth Berry-Kravis, MD, PhD, and funded by FRAXA. Positive results were published
- pilot trial of CX516 — Elizabeth Berry-Kravis, and funded by FRAXA.
- Phase III trials of arbaclofen were completed by Seaside Therapeutics but results were disappointing in both Fragile X and autism. However, baclofen is an available drug.
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