Validity of a condition specific outcome measure for fragile X syndrome: the Aberrant Behaviour Checklist-utility index

2016 ◽  
Vol 60 (9) ◽  
pp. 844-855 ◽  
Author(s):  
M. Raspa ◽  
P. Sacco ◽  
S.D. Candrilli ◽  
E. Bishop ◽  
J. Petrillo
Keyword(s):  
Fragile X Syndrome ◽  
Outcome Measure ◽  
Fragile X ◽  
Aberrant Behaviour ◽  
Utility Index ◽  
Specific Outcome

scholarly journals Combining Lovastatin and Minocycline for the Treatment of Fragile X Syndrome: Results From the LovaMiX Clinical Trial

2022 ◽  
Vol 12 ◽  
Author(s):  
Camille Champigny ◽  
Florence Morin-Parent ◽  
Laurence Bellehumeur-Lefebvre ◽  
Artuela Çaku ◽  
Jean-François Lepage ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Fragile X Syndrome ◽  
Outcome Measures ◽  
Primary Outcome ◽  
Outcome Measure ◽  
Fragile X ◽  
Combined Therapy ◽  
Primary Outcome Measure ◽  
Secondary Outcome ◽  
Global Score

Background: Limited success of previous clinical trials for Fragile X syndrome (FXS) has led researchers to consider combining different drugs to correct the pleiotropic consequences caused by the absence of the Fragile X mental retardation protein (FMRP). Here, we report the results of the LovaMiX clinical trial, the first trial for FXS combining two disease-modifying drugs, lovastatin, and minocycline, which have both shown positive effects when used independently.Aim: The main goals of the study were to assess the safety and efficacy of a treatment combining lovastatin and minocycline for patients with FXS.Design: Pilot Phase II open-label clinical trial. Patients with a molecular diagnostic of FXS were first randomized to receive, in two-step titration either lovastatin or minocycline for 8 weeks, followed by dual treatment with lovastatin 40 mg and minocycline 100 mg for 2 weeks. Clinical assessments were performed at the beginning, after 8 weeks of monotherapy, and at week 20 (12 weeks of combined therapy).Outcome Measures: The primary outcome measure was the Aberrant Behavior Checklist-Community (ABC-C) global score. Secondary outcome measures included subscales of the FXS specific ABC-C (ABC-CFX), the Anxiety, Depression, and Mood Scale (ADAMS), the Social Responsiveness Scale (SRS), the Behavior Rating Inventory of Executive Functions (BRIEF), and the Vineland Adaptive Behavior Scale second edition (VABS-II).Results: Twenty-one individuals out of 22 completed the trial. There were no serious adverse events related to the use of either drugs alone or in combination, suggesting good tolerability and safety profile of the combined therapy. Significant improvement was noted on the primary outcome measure with a 40% decrease on ABC-C global score with the combined therapy. Several outcome measures also showed significance.Conclusion: The combination of lovastatin and minocycline is safe in patients for FXS individuals and appears to improve several elements of the behavior. These results set the stage for a larger, placebo-controlled double-blind clinical trial to confirm the beneficial effects of the combined therapy.

scholarly journals Caregiver Burden in Fragile X Syndrome as a Function of a Health State Utility Index in the United States

2013 ◽  
Vol 16 (7) ◽  
pp. A625
Author(s):  
M. Raspa ◽  
E. Bishop ◽  
S.D. Candrilli ◽  
D. Mitra ◽  
P. Sacco ◽  
...  
Keyword(s):  
United States ◽  
Fragile X Syndrome ◽  
Caregiver Burden ◽  
Fragile X ◽  
The United States ◽  
Health State Utility ◽  
Health State ◽  
Utility Index

scholarly journals Developing a utility index for the Aberrant Behavior Checklist (ABC-C) for fragile X syndrome

2014 ◽  
Vol 24 (2) ◽  
pp. 305-314 ◽  
Author(s):  
Cicely Kerr ◽  
Katie Breheny ◽  
Andrew Lloyd ◽  
John Brazier ◽  
Donald B. Bailey ◽  
...  
Keyword(s):  
Fragile X Syndrome ◽  
Fragile X ◽  
Aberrant Behavior ◽  
Aberrant Behavior Checklist ◽  
Utility Index

scholarly journals Occupations as an Outcome Measure in a Clinical Trial: Fragile X Syndrome and Sertraline

2017 ◽  
Author(s):  
◽  
Michelle Beckwith ◽  
Bina Nguyen ◽  
Jennifer Sik ◽  
Kenneth Yu
Keyword(s):  
Clinical Trial ◽  
Fragile X Syndrome ◽  
Outcome Measure ◽  
Fragile X

scholarly journals Social communication in fragile X syndrome: pilot examination of the Brief Observation of Social Communication Change (BOSCC)

2022 ◽  
Vol 14 (1) ◽  
Author(s):  
Rebecca Shaffer ◽  
Angela John Thurman ◽  
Lucienne Ronco ◽  
Diego Cadavid ◽  
Shane Raines ◽  
...  
Keyword(s):  
Fragile X Syndrome ◽  
Social Communication ◽  
Outcome Measure ◽  
Fragile X ◽  
Outcome Measurement ◽  
Autism Spectrum ◽  
Divergent Validity ◽  
Minimally Verbal ◽  
Retest Reliability ◽  
Test Retest Reliability

Abstract Background Social communication is a key area of difficulty in fragile X syndrome (FXS) and there are not yet adequate outcome measurement tools. Appropriate outcome measures for FXS have been identified as a key area of research interest in order to evaluate future therapeutic trials. The Brief Observation of Social Communication Change-Minimally Verbal (BOSCC-MV), an outcome measure with strong psychometrics developed for autism spectrum disorder, has promise as an outcome measure to assess social communication change with FXS participants. Methods We examined the BOSCC-MV via central coders in this multi-site-trial to assess its appropriateness for FXS. Eighteen minimally verbal males ages 3–12 years were enrolled and assessed on two consecutive days and 7 participants completed a third visit 6 months later. We examined test-retest reliability, inter-rater reliability, and both convergent and divergent validity with standard clinical measures including the Autism Diagnostic and Observation Schedule-2, Vineland 3, Social Responsiveness Scale, and the Aberrant Behavior Checklist. Results The BOSCC-MV in FXS demonstrated strong inter-rater and test-retest reliability, comparable to previous trials in idiopathic ASD. Strong convergent validity was found with Autism Diagnostic Observation Schedule-2 and Vineland-3. Divergent validity was demonstrated between BOSCC-MV and unrelated measures. Conclusions The BOSCC-MV shows promise as a FXS social communication outcome measure, warranting further large-scale evaluation.

Living with Fragile X Syndrome

2017 ◽  
Author(s):  
◽  
Rebecca Schira ◽  
Samantha Alexander ◽  
Noelani Brisbane ◽  
Kaitlyn Williams
Keyword(s):  
Fragile X Syndrome ◽  
Fragile X

Fragile X Syndrome and Family Occupations

2019 ◽  
Author(s):  
Decerie Mendoza ◽  
Tracy Ye ◽  
Martina Dualan ◽  
Elena Javier
Keyword(s):  
Fragile X Syndrome ◽  
Fragile X
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