Assessment & Research

Characterization of potential outcome measures for future clinical trials in fragile X syndrome.

Berry-Kravis et al. (2008) · Journal of autism and developmental disorders 2008
★ The Verdict

Elizabeth et al. (2008) gives you four reliable paper tests ready for fragile X trials, and later work adds a speedy computer option.

✓ Read this if BCBAs helping with fragile X clinical trials or data collection.
✗ Skip if Clinicians only doing autism therapy with no research role.

01Research in Context

01

What this study did

Berry-Kravis et al. (2008) asked which paper-and-pencil memory and thinking tests stay steady in people with fragile X. They gave the same tests twice, a few weeks apart, to a small group. Then they checked if each test gave the same score both times.

02

What they found

Four tests held up well. List and Story Memory from the RBANS, the NEPSY Tower, W-J Spatial Relations, and CPT commission errors all scored between 0.7 and 0.9 on the reliability scale. That range means the tests are solid enough to track small changes in short drug or behavior studies.

03

How this fits with other research

Falcomata et al. (2012) built on this work by adding a 15-minute computer game that also gives a stable learning-rate score. The new tool is faster and auto-records data, but it aims for the same goal: a trusty endpoint for fragile X trials.

Berry-Kravis (2022) folds both papers into a bigger plan. The review says the field will keep the 2008 paper tests while also using the 2012 digital task. Together they give teams a mix of classic and tech options.

Mulder et al. (2020) pushes the idea further. They argue we should stop looking at norm tables and start tracking each person’s own ability line. This method could make the 2008 tests even better at spotting true change.

04

Why it matters

If you run or join a fragile X study, you now have a short list of proven cognitive endpoints. You can pick from paper tasks, add the quick computer test, or mix both. Using tools with known reliability keeps your data clean and your trial short, saving time for you and the families you serve.

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Slot RBANS List Memory into your pre/post test battery for your next FXS pilot study.

02At a glance

Intervention
not applicable
Design
pre post no control
Sample size
46
Population
developmental delay
Finding
positive

03Original abstract

Clinical trials targeting recently elucidated synaptic defects in fragile X syndrome (FXS) will require outcome measures capable of assessing short-term changes in cognitive functioning. Potentially useful measures for FXS were evaluated here in a test-retest setting in males and females with FXS (N = 46). Good reproducibility, determined by an interclass correlation (ICC) or weighted kappa (kappa) of 0.7-0.9 was seen for RBANS List and Story Memory, NEPSY Tower, Woodcock-Johnson Spatial Relations and the commissions score from the Carolina Fragile X Project Continuous Performance Test (CPT). This study demonstrates the feasibility of generating test profiles containing reliability data, ability levels required for test performance, and refusal rates to assist with choice of outcome measures in FXS and other cohorts with cognitive disability.

Journal of autism and developmental disorders, 2008 · doi:10.1007/s10803-008-0564-8