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 Participants
Chicago, IL
2-Period Crossover Study of BPN14770 in Adults Males With Fragile X Syndrome – Rush University Medical Center
Subjects will receive both active treatment with BPN14770 capsules and matching placebo capsules in the course of the study. One treatment will be administered during each of the 12-week study periods. Please contact the study coordinator Melissa R Baer, MS, CCRC, at Melissa_R_Baer@rush.edu or (312) 563-6636. Principal Investigator: Elizabeth Berry-Kravis, MD, PhD.
Details at Clinicaltrials.gov.
Sacramento, CA
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. Please contact the study coordinator Erika Bickel at esbickel@ucdavis.edu or (916) 703-0281.
Cincinnati, OH
Trial of acamprosate
Completed Trials
Nashville, TN
Phase I Trial of Gaboxadol
This preliminary trial for children ages 13-17 with Fragile X Syndrome or Angelman Syndrome was at Vanderbilt Children’s Hospital and sponsored by Ovid Therapeutics.
Quebec, Canada
Combining Lovastatin and Minocycline
This pilot randomized Phase II trial is exploring the tolerability and the synergistic effect of a combined therapy using minocycline, which is often used to treat acne, and lovastatin, which is used to lower cholesterol. Both drugs target specific alterations in the brain of Fragile X patients that would potentially have a combined powerful effect on their behavior. https://clinicaltrials.gov/ct2/show/NCT02680379
Sacramento, CA
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. Please contact the study coordinator Erika Bickel at esbickel@ucdavis.edu or (916) 703-0281.
Cincinnati, OH
Trial of acamprosate
- 2012-15: Epigallocatechin gallate (mega green tea extract) – Mara Dierssen – Barcelona, Spain. Trial results are being analyzed.
- 2/2016 – Double Blind Crossover Trial of Ganaxolone – Investigators: Frank Kooy and Anke Van Dijck- Antwerp, Belgium, sponsored by FRAXA Research Foundation. Dr. Randi Hagerman conducted a part of this trial in Davis, CA, sponsored by Marinus Pharmaceuticals.
“Consistent with the mechanism of action and preclinical studies, anxious FXS patients treated with ganaxolone showed clinically relevant reductions in anxiety across several scales and subscales sensitive to measuring changes in anxious behavior. These results are encouraging and suggest that ganaxolone may improve anxiety in children with FXS, which could potentially allow them, as well as their families, to experience a more stabilized lifestyle.” – Frank Kooy, PhD.
- 12/2015 – Neuren’s trofinetide showed some success in small Phase 2 clinical trials in Fragile X syndrome and in Rett Syndrome
- 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
