Arbaclofen in fragile X syndrome: results of phase 3 trials

  title={Arbaclofen in fragile X syndrome: results of phase 3 trials},
  author={Elizabeth M. Berry-Kravis and Randi Hagerman and Jeannie Visootsak and Dejan B. Budimirovic and Walter E. Kaufmann and Maryann Cherubini and Peter Zarevics and Karen L Walton-Bowen and Paul P. Wang and Mark F. Bear and Randall L. Carpenter},
  booktitle={Journal of Neurodevelopmental Disorders},
Arbaclofen improved multiple abnormal phenotypes in animal models of fragile X syndrome (FXS) and showed promising results in a phase 2 clinical study. The objective of the study is to determine safety and efficacy of arbaclofen for social avoidance in FXS. Two phase 3 placebo-controlled trials were conducted, a flexible dose trial in subjects age 12–50 (209FX301, adolescent/adult study) and a fixed dose trial in subjects age 5–11 (209FX302, child study). The primary endpoint for both trials… CONTINUE READING
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Publications referenced by this paper.
Showing 1-10 of 44 references

Factor analysis of the Parenting Stress Index-Short Form with parents of young children with autism spectrum disorders.

Autism research : official journal of the International Society for Autism Research • 2011
View 10 Excerpts
Highly Influenced

Risperidone in children with autism and serious behavioral problems.

The New England journal of medicine • 2002
View 14 Excerpts
Highly Influenced

Sensitivity of an eye tracking paradigm as an outcome measure in a fragile X syndrome targeted treatment clinical trial

SM Sansone, E Berry-Kravis, +3 authors D. Hessl
Annual Gatlinburg conference on research and theory in intellectual and developmental disabilities; San Diego • 2016
View 1 Excerpt

The treatment of fragile X syndrome with Trofinetide (NNZ-2566)

E Berry-Kravis, CA Erickson, +6 authors H Needelman
In Proceedings of the 15th international fragile X conference; San Antonio • 2016
View 1 Excerpt

Analysis of arbaclofen responses in fragile X syndrome

A De Sonia, J Visootsak, +3 authors N Welnik
In: 14th International Fragile X Conference; • 2014
View 1 Excerpt

Development of the fragile X syndrome rating scale

M Snape, J Horrigan, +6 authors N. Jones
IMFAR • 2014
View 1 Excerpt

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