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 Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT05146284
• Other study ID #: PXL770-011
• Status: Not yet recruiting
• Phase: Phase 2
• Start date: September 2023
• Est. completion date: September 2024

Study information

• Verified date: April 2023
• Source: Poxel SA
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

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).

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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 24
• Est. completion date: September 2024
• Est. primary completion date: September 2024
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 18 Years to 65 Years

Eligibility

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.

Related Conditions & MeSH terms

• Adrenoleukodystrophy
• Adrenomyeloneuropathy

Intervention

Drug:
• PXL770
Tablet

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Poxel SA

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Pharmacokinetic (PK) parameters	Peak plasma concentration (Cmax) for 500mg QD	4 week
Primary	Pk parameters	Area under the plasma concentration versus time curve (AUC)0-24 for 500mg QD	4 week
Primary	PK parameters	Cmax for 250mg BID	4 week
Primary	PK parameters	AUC0-8 for 250mg BID	4 week