Potential markers for sample size estimations in hereditary spastic paraplegia type 5.
Qianqian LinYing LiuZhixian YeJianping HuWenjie CaiQiang WengWan-Jin ChenNing WangDairong CaoYi LinYing FuPublished in: Orphanet journal of rare diseases (2021)
SPG5 patients showed distinct MRI features of spinal cord atrophy without significant brain alterations. Our finding supports the measurements of spinal cord on T9 level as potential endpoint for SPG5 clinical trials. Trial registration ClinicalTrials.gov, NCT04006418. Registered 05 July 2019, https://clinicaltrials.gov/ct2/show/NCT04006418?term=NCT04006418&draw=2&rank=1 .
Keyphrases
- spinal cord
- clinical trial
- end stage renal disease
- contrast enhanced
- spinal cord injury
- neuropathic pain
- newly diagnosed
- ejection fraction
- chronic kidney disease
- computed tomography
- magnetic resonance imaging
- preterm infants
- phase ii
- human health
- magnetic resonance
- patient reported outcomes
- randomized controlled trial
- white matter
- brain injury
- image quality
- botulinum toxin
- cerebral palsy
- subarachnoid hemorrhage