International SBMA Project (KDA)

Condition(s) studied

Study summary

This project aims at: connecting the existing registries and databases across different countries by comparing the information collected, the administered scales, the biochemical investigations performed; performing a common analysis of fully anonymized data collected in the existing registries and databases by researchers adhering to the study to obtain cross-sectional and longitudinal data. Centres from the following countries have accepted the invitation to join Italy in this project: UK, US, Japan, France, Germany, Korea, Finland, Greece, Canada, Turkey.

Eligibility

Primary outcome measure(s)

• Spinal and Bulbar Muscular Atrophy Functional Rating Scale (SBMAFRS) — 3 years
  Change in total score of the Spinal and Bulbar Muscular Atrophy Functional Rating Scale (SBMAFRS), a disease-specific functional scale assessing bulbar, spinal, trunk, and respiratory function. The SBMAFRS total score ranges from 0 to 56 points. Higher scores indicate better functional status.
• Six-Minute Walk Test (6MWT) Distance — 3 years
  Change in distance walked during the Six-Minute Walk Test (6MWT). The 6MWT measures the total distance (in meters) that a participant is able to walk on a flat surface in six minutes. Greater distance indicates better ambulatory and endurance capacity.
• Age at Onset of First SBMA Symptoms — assessed once
  Age (in years) at onset of first reported SBMA-related symptom (hand tremor/cramps/muscular weakness, dysarthria, dysphagia, handrail requirement, use of support/wheelchair, development of pneumonia)

Trial sites (1)

More Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta trials in Italy