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

Poxel SA

Overview

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).

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 Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Conditions

Adrenomyeloneuropathy

Interventions

PXL770DRUG

Tablet

Eligibility

Sex: MALEMin age: 18 YearsMax age: 65 Years

Medical Language ↔ Plain English

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 V2. 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.

Outcomes

Primary Outcomes

Pharmacokinetic (PK) parameters

Peak plasma concentration (Cmax) for 500mg QD

Time frame: 4 week

Pk parameters

Area under the plasma concentration versus time curve (AUC)0-24 for 500mg QD

Time frame: 4 week

PK parameters

Cmax for 250mg BID

Time frame: 4 week

PK parameters

AUC0-8 for 250mg BID

Time frame: 4 week

Data from ClinicalTrials.gov

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