Trial reportJournal of patient-reported outcomes2026
Assessing signs and impacts of achondroplasia: psychometric evaluation of the Achondroplasia Child Experience Measures.
Trial report in Journal of patient-reported outcomes, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT04085523 (ACcomplisH), which is not on this map. Not yet cited in PubMed.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
ACcomplisH: A Phase 2, Multicenter, Double-blind, Randomized, Placebo-controlled, Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of Subcutaneous Doses of TransCon CNP Administered Once Weekly for 52 Weeks in Prepubertal Children With Achondroplasia Followed by an Open-Label Extension Period
Who cites it
0 citing papers in PubMed.
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Authors and funding
3 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundAchondroplasia, although rare, is the most common genetic form of skeletal dysplasia and is associated with a wide range of clinical manifestations and complications throughout the body. This study evaluated the psychometric measurement properties of the Achondroplasia Child Experience Measures (ACEMs), which were previously conceptually developed as condition-specific, observer-reported outcome measures to assess the signs (ACEM-OSM) and impacts (ACEM-Impact) of achondroplasia in children aged 2 to < 12 years. METHODOLOGY: Data for the psychometric evaluation were from a non-interventional, observational study (N = 200 (impacts) and N = 148 (signs)) and a phase 2, multicenter, double-blind, randomized, placebo-controlled, dose escalation interventional trial (N = 57) of navepegritide (TransCon CNP) administered once weekly. The larger observational study sample size allowed for evaluation of distributional properties, test-retest reliability, and construct validity. Trial data was analyzed to confirm reliability and validity findings and evaluate responsiveness.
resultsFactor analysis and qualitative results identified six domains for the ACEM-Impact and one domain for the ACEM-OSM. The psychometric properties of both measures were found to be acceptable for reliability and validity for the intended use. An 8-item ACEM-OSM and a 25-item ACEM-Impact were finalized.
conclusionsThe ACEMs are reliable and valid measures of the signs and impacts of achondroplasia on children. Incorporation of these measures in clinical and research settings can aid in assessing new treatments and improve our understanding of the experience of those living with achondroplasia.
trial registrationNational Institutes of Health Clinicaltrials.gov (ID NCT04085523) and European Union Drug Regulating Authorities Clinical Trials database (EudraCT 2019-002754-22).
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