Clinical Trials Logo

Clinical Trial Summary

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.


Clinical Trial Description

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. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT02642653
Study type Interventional
Source University of California, Davis
Contact
Status Completed
Phase Phase 4
Start date January 2016
Completion date July 2018

See also
  Status Clinical Trial Phase
Recruiting NCT05418049 - Evaluating the Neurophysiologic and Clinical Effects of Single Dose Drug Challenge Phase 2
Enrolling by invitation NCT01364818 - Brain Connectivity in Neurodevelopmental Disorders in Response to Treatment N/A
Completed NCT01120626 - Randomized Controlled Study of Donepezil in Fragile X Syndrome Phase 2
Completed NCT00965432 - A Single-Dose Study in Normal Volunteers to Assess the Safety, Tolerability and Pharmacokinetics of STX107 Phase 1
Completed NCT01204151 - Teaching Math Skills to Individuals With Fragile X Syndrome N/A
Active, not recruiting NCT00334971 - Aromatase Activity and Ovarian Growth Factors in African-American Versus Caucasian Women N/A
Enrolling by invitation NCT06139172 - Promoting Prosocial Behavior in Syndromic Intellectual and Developmental Disabilities N/A
Recruiting NCT04977986 - Clinical Study of Cannabidiol in Children, Adolescents, and Young Adults With Fragile X Syndrome Phase 3
Active, not recruiting NCT04698551 - NIPD on cffDNA for Triplet Repeat Diseases
Completed NCT03722290 - Metformin in Children and Adults With Fragile X Syndrome Phase 2
Completed NCT05030129 - Single Blind Study of Ergoloid Mesylates, 5-HTP and the Combination in Adult Males With Fragile X Syndrome Phase 2
Recruiting NCT05957549 - Tracking Early Emergence of Sound Perception Impairments in FXS With Multimodal fNIRS/EEG N/A
Recruiting NCT04141163 - Metformin in Patients With Fragile X Phase 1/Phase 2
Not yet recruiting NCT06081348 - Sertraline vs. Placebo in the Treatment of Anxiety in Children and AdoLescents With NeurodevelopMental Disorders Phase 2
Completed NCT00858689 - Add-on Pilot Trial of Minocycline to Treat Fragile X Syndrome N/A
Enrolling by invitation NCT03655223 - Early Check: Expanded Screening in Newborns
Enrolling by invitation NCT03802799 - Open Label Extension to Assess the Long-Term Safety and Tolerability of ZYN002 in Children and Adolescents With FXS Phase 2/Phase 3
Recruiting NCT05295277 - Validation of Optical Genome Mapping for the Identification of Constitutional Genomic Variants in a Postnatal Cohort
Enrolling by invitation NCT03836300 - Parent and Infant Inter(X)Action Intervention (PIXI) N/A
Completed NCT01725152 - Ganaxolone Treatment in Children With Fragile X Syndrome Phase 2