A Phase IIb, Monocentric, Non-Profit, Open-label Trial for the Intrathecal Administration of AAV9/AP4M1 for Hereditary Spastic Paraplegia Type 50 (SPG50)

Hereditary Spastic Paraplegia Type 50 (SPG50)

Change in total percent score of the eight Major Motor Milestones derived from the Gross Motor Function Measure (GMFM)-88 from baseline at W156.

Developmental Milestones – Bayley Scale of Infant and Toddler Develop-ment 3rd Edition (Bayley-3) Cognitive Domain – Change in Total Raw Score from Baseline at W156., Gross and Fine Motor Function (GMFM-88 full scale) – Change in Total Score from Baseline at W156., Disease Severity (Spastic Paraplegia Rating Scale [SPRS]) – Change in Total Score from Baseline at W156., Disease Severity (Clinical Global Impression [CGI-I]) – Change in Physician-assessed CGI-I from Baseline at W156., Differential Ability Scales, 2nd Edition (DAS-II) - Change in Total Score from Baseline at W156., Developmental Milestones (Developmental Quotients [DQs]) – Change in DQs for Gross Motor, Adaptive behavior and Cognitive Scores from Base-line., Axonal Damage (Plasma neurofilament light chain levels [NfL]) – Change in Plasma NfL levels from Baseline., Health Related Quality of Life (The Caregiver Priorities and Child Health In-dex of Life with Disabilities [CPCHILD]) – Change in Total Score from Base-line., Disease Severity (Parent-rated Global Impression [PGI-I]) – Change in Glob-al Impression as assessed by the primary caregiver from Baseline., Axonal Damage (Cerebrospinal fluid [CSF] neurofilament light chain levels [NfL]) – Change in CSF NfL levels from Baseline., Serum and/or Plasma and CSF biomarkers – Change in Biomarker Levels from Baseline., Developmental Milestones (Vineland Adaptive Behavior Scale 3rd Edition [Vineland-3]) – Change in Total Score from Baseline.

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