Fragile X Syndrome Clinical Trial
Official title:
Combining Lovastatin and a Parent-Implemented Language Intervention in a Multimodal Treatment for Fragile X Syndrome
The purpose of the study is to test the efficacy of a 20 week multi-modal treatment comprised of lovastatin or placebo, and the Parent-implemented Language Intervention (PILI) in children with fragile X syndrome (FXS). Children will be randomized to drug or placebo in a double-blind design with all participating in the PILI. The primary endpoint will be to measure improvements in spoken language and behavior among lovastatin-treated than placebo treated participants.
This is the first multi-modal treatment to combine a targeted treatment for FXS, lovastatin,
with an innovative parent-implemented intervention (PILI) targeting language and challenging
behavior delivered through telehealth technology.The hypothesis is that targeted treatments
will be more effective when applied in combination with PILI. Examination of whether changes
in the activity of key pathways/proteins influenced by fragile X mental retardation protein
(FMRP) (the mitogen-activated protein kinase (ERK) and MMP-9) are biomarkers of treatment
responsiveness. Because lovastatin is also an anti-inflammatory, characterization of MEK/ERK
signaling in peripheral immune cells both pre- and post- treatment will be carried out to
determine whether levels of these signaling molecules are predictive biomarkers of treatment
response. It is hypothesized that those individuals with elevated inflammatory cytokine
profiles will be most responsive to lovastatin treatment. Once modeled in FXS, results from
these studies can then be applied to other neurodevelopmental disorders including
RASopathies.
The behavioral component of the proposed multi-modal treatment will be a Parent-implemented
Intervention (PILI) that targets improvements in spoken language and challenging behavior for
10- to 17-year-olds with FXS by increasing parental verbal responsiveness (PVR) within
picture-book based story-telling episodes. Parents will be encouraged to use the targeted
strategies in other everyday interactions with their child. The intervention will be
delivered to parents in their homes by way of video teleconferencing (VTC). Participants will
be randomly assigned to receive the behavioral intervention alone or in combination with
Lovastatin.
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