Clinical Trial Details
— Status: Not yet recruiting
Administrative data
NCT number |
NCT05200104 |
Other study ID # |
PXL065-005 |
Secondary ID |
|
Status |
Not yet recruiting |
Phase |
Phase 2
|
First received |
|
Last updated |
|
Start date |
September 2023 |
Est. completion date |
September 2024 |
Study information
Verified date |
April 2023 |
Source |
Poxel SA |
Contact |
n/a |
Is FDA regulated |
No |
Health authority |
|
Study type |
Interventional
|
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).
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.