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Study to Assess PXL770 in Subjects With Adrenomyeloneuropathy (AMN) Form of X-linked Adrenoleukodystrophy (X-ALD or ALD)

Adrenomyeloneuropathy Clinical Trial

Official title:
A Randomized Open-label Phase 2a Study to Assess PXL770 After 12 Weeks of Treatment in Male Subjects With Adrenomyeloneuropathy (AMN) Form of X-linked Adrenoleukodystrophy (X-ALD or ALD)

Clinical Trial Summary

A randomized open-label Phase 2a study to assess the pharmacokinetics and pharmacodynamic parameters of PXL770 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 PXL770 after 12 weeks of treatment in male subjects with adrenomyeloneuropathy (AMN). There are 3 study periods. - Screening Visit: within a maximum of 4 weeks prior to the open-label Treatment Period - Open-label Treatment Period: 12 weeks - Follow-up Period: 2 weeks after the last intake of the treatment During the treatment period, VLCFA will be assessed every 4 weeks, to evaluate the kinetics of the effect. 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 monitoring the subjects' safety as well as the duration of the effect on the 2 main biomarkers (VLCFA and NfL) at 2 and 4 weeks after the drug withdrawal. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT05146284
Study type Interventional
Source Poxel SA
Contact
Status Not yet recruiting
Phase Phase 2
Start date September 2023
Completion date September 2024
View Details
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