Controlled trial of lovastatin combined with an open-label treatment of a parent-implemented language intervention in youth with fragile X syndrome

Abstract

Background: The purpose of this study was to conduct a 20-week controlled trial of lovastatin (10 to 40 mg/day) in youth with fragile X syndrome (FXS) ages 10 to 17 years, combined with an open-label treatment of a parentimplemented language intervention (PILI), delivered via distance video teleconferencing to both treatment groups, lovastatin and placebo. Method: A randomized, double-blind trial was conducted at one site in the Sacramento, California, metropolitan area. Fourteen participants were assigned to the lovastatin group; two participants terminated early from the study. Sixteen participants were assigned to the placebo group. Lovastatin or placebo was administered orally in a capsule form, starting at 10 mg and increasing weekly or as tolerated by 10 mg increments, up to a maximum dose of 40 mg daily. A PILI was delivered to both groups for 12 weeks, with 4 activities per week, through video teleconferencing by an American Speech-Language Association-certified Speech-Language Pathologist, in collaboration with a Board-Certified Behavior Analyst. Parents were taught to use a set of language facilitation strategies while interacting with their children during a shared storytelling activity. The main outcome measures included absolute change from baseline to final visit in the means for youth total number of story-related utterances, youth number of different word roots, and parent total number of story-related utterances.

Expressive language sampling as a source of outcome measures for treatment studies in fragile X syndrome: feasibility, practice effects, test-retest reliability, and construct validity

“…ELS has several potential advantages compared to typical norm-referenced standardized tests [30], such as the Receptive and Expressive subscales of the Mullen Early Scales [37], the Peabody Picture Vocabulary Test-4th edition [38], the Preschool Language Scale-5 [39], the Clinical Evaluation of Language Fundamentals [40], and the Comprehensive Assessment of Spoken Language [41], all of which have been used in clinical trials for FXS [42][43][44][45]. First, ELS interactive contexts are more closely aligned with performance in real-world contexts, and thus, performance is more likely to be generalizable to activities that are functional and meaningful for the individual [46].…”

Expressive language development in adolescents with Down syndrome and fragile X syndrome: change over time and the role of family-related factors

“…In addition, these measures all display minimal practice effects and have strong test-retest reliability for individuals with FXS and DS in the age range interest, which is important in longitudinal studies that, by definition, involved repeated administration of the same measures [56,63]. Finally, intervention studies in individuals with different neurodevelopmental disorders have shown change in various ELS measures in the face of a lack of change in standardized tests [64][65][66]. Thus, we used naturalistic conversation and narration language sampling techniques in a longitudinal design and computed these diverse measures of expressive language performance in verbal individuals with ID associated with DS and with FXS.…”

Telehealth-enabled behavioral treatment for problem behaviors in boys with fragile X syndrome: a randomized controlled trial

“…Future studies will be needed to compare the efficacy between behavioral treatment with FCT and pharmacological treatment in FXS. Ultimately, it is possible that a combination of pharmacological and behavioral treatment may be the most beneficial treatment strategy [ 13 , 72 ].…”