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. If you’d like to learn more, read this interview with a parent about her experience.
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.
Fragile X Studies Currently Accepting Participants
Please visit clinicaltrials.gov for a complete listing of Fragile X syndrome clinical trials.
Trials Awaiting Results
- 2015 – Neuren’s trofinetide showed some success in small Phase 2 clinical trials in Fragile X syndrome and in Rett Syndrome
- a pilot lithium trial — Elizabeth Berry-Kravis, MD, PhD, and funded by FRAXA. Positive results published
- pilot trial of CX516 — Elizabeth Berry-Kravis, and funded by FRAXA.
- Phase III trials of arbaclofen – Seaside Therapeutics. Results were disappointing in Fragile X and autism.