NCT05146284

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

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)

• Status: Withdrawn
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 0 (actual)
• Sponsor: Poxel SA · Industry
• Sex: Male
• Age: 18 Years – 65 Years
• Healthy volunteers: Not accepted

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).

Detailed 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.

Conditions

• Adrenomyeloneuropathy

Interventions

Type	Name	Description
DRUG	PXL770	Tablet

Timeline

• Start date: 2023-09-01
• Primary completion: 2024-09-01
• Completion: 2024-09-01
• First posted: 2021-12-06
• Last updated: 2024-12-16

Regulatory

• FDA-regulated drug study