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Clinical Trial Summary

A randomized open-label Phase 2a study to assess the pharmacokinetics and pharmacodynamic parameters of PXL065 after 12 weeks of treatment in male subjects with adrenomyeloneuropathy (AMN).


Clinical Trial Description

A randomized open-label Phase 2a study to assess the pharmacokinetics and pharmacodynamic parameters of PXL065 after 12 weeks of treatment in male subjects with adrenomyeloneuropathy (AMN). There will be a total of 3 study periods. - Screening period of a maximum of 4 weeks prior to the open-label Treatment Period (Baseline Visit - V2). This period can be exceptionally extended for 2 weeks - Open-label Treatment Period of 12 weeks - Follow-up Period of 2 weeks after the last intake of the treatment (V5-End of Treatment Visit (EoT)). During the treatment period, very long chain fatty acids (VLCFA) will be assessed every 4 weeks, to evaluate the kinetics of the PXL065 effect. Neurofilament light (NfL) will be assessed after 8 and 12 weeks of treatment, and other exploratory biomarkers after 12 weeks of treatment. A follow up period will allow continued monitoring the subjects' safety and evaluation of the kinetics of the 2 main biomarkers (VLCFA and NfL) at 2 weeks after the last intake of the treatment. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT05200104
Study type Interventional
Source Poxel SA
Contact
Status Not yet recruiting
Phase Phase 2
Start date July 2022
Completion date March 2023

See also
  Status Clinical Trial Phase
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