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Withdrawn

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT05146284

Enrollment

Registered

2021-12-06

Start date

2023-09-30

Completion date

2024-09-30

Last updated

2024-12-16

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Adrenomyeloneuropathy

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.

Interventions

DRUGPXL770

Tablet

Study design

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

MALE

Age

18 Years to 65 Years

Healthy volunteers

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.

Design outcomes

Primary

Measure	Time frame	Description
Pharmacokinetic (PK) parameters	4 week	Peak plasma concentration (Cmax) for 500mg QD
Pk parameters	4 week	Area under the plasma concentration versus time curve (AUC)0-24 for 500mg QD
PK parameters	4 week	Cmax for 250mg BID

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026