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)

Poxel SA0 sites24 target enrollmentSeptember 2023

ConditionsAdrenomyeloneuropathy

InterventionsPXL770

DrugsPXL770

Overview

Phase: Phase 2
Intervention: PXL770
Conditions: Adrenomyeloneuropathy
Sponsor: Poxel SA
Enrollment: 24
Primary Endpoint: Pharmacokinetic (PK) parameters
Status: Withdrawn
Last Updated: last year

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Similar Trials

Overview

Brief 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.

Registry

clinicaltrials.gov

Start Date

September 2023

End Date

September 2024

Last Updated

last year

Study Type

Interventional

Study Design

Parallel

Sex

Male

Investigators

Sponsor

Poxel SA

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•Male subjects with either a confirmed diagnosis of AMN by genetic testing (mutation in the ATP binding cassette subfamily D (ABCD1 gene)) or a family history of X-linked adrenoleukodystrophy (ALD) together with an elevation in VLCFA obtained from overnight fasting plasma sample at Screening Visit (V1).
•Age: ≥ 18 to ≤ 65 years at informed consent signature.
•Normal brain magnetic resonance imaging (MRI) or brain MRI showing non-specific abnormalities that can be observed in AMN subjects without signs of cerebral form of ALD (C-ALD). MRI must be performed within 6 months prior to V
•If there is no available brain MRI within this period, a brain MRI must be performed before V2.

Exclusion Criteria

•Any progressive neurological disease other than AMN.
•Arrested or progressing C-ALD as defined by cerebral lesions (except for non-specific abnormalities that can be observed in AMN subjects).
•Prior receipt of an allogeneic hematopoietic stem cell transplant or gene therapy.