Study to Assess PXL770 in Subjects With Adrenomyeloneurop... | NCT05146284

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

NCT05146284

Sponsor

Poxel SA

Status

Withdrawn

Last Update Posted

Dec 16, 2024Actual

Enrollment

0Actual

Phase

Phase 2

Conditions

Adrenomyeloneuropathy

Interventions

PXL770

Countries

Not provided

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Dec 2024

Overall Recruitment Status or Expanded Access Status

Withdrawn

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Lack of funding to initiate the study

Expanded Access Info

Start Date

Sep 2023Estimated

Primary Completion Date

Sep 2024Estimated

Completion Date

Sep 2024Estimated

First Submitted Date

Nov 16, 2021

First Submission Date that Met QC Criteria

Dec 3, 2021

First Posted Date

Dec 6, 2021Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Dec 10, 2024

Last Update Posted Date

Dec 16, 2024Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Yes

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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.

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 2

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

0Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
PXL770 500 mg QD	Experimental		Drug: PXL770
PXL770 250 mg BID	Experimental		Drug: PXL770

Interventions

Name	Type	Description	Arm Group Labels	Other Names
PXL770	Drug	Tablet	PXL770 250 mg BID PXL770 500 mg QD

Outcomes Module

Primary Outcomes

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

Secondary Outcomes

Not provided

Other Outcomes

Not provided

Eligibility Module

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 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.

Accepts Healthy Volunteers

Sex

Male

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

65 Years

Standard Ages

AdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

IPD Sharing Statement Module

Plan to Share IPD

Description

There is no plan to share IPD with other researchers

Types

Not provided

Time Frame

Not provided

Access Criteria

Not provided

URL

Not provided

Condition Browse Module

MeSH Terms

ID	Term
D000326	Adrenoleukodystrophy

Ancestor Terms

ID	Term
D020739	Brain Diseases, Metabolic, Inborn
D001928	Brain Diseases, Metabolic
D001927	Brain Diseases
D002493	Central Nervous System Diseases

Browse Leaves

Not provided

Browse Branches

Not provided

Intervention Browse Module

MeSH Terms

ID	Term
C000719997	2-chloro-3-(1-hydroxy-5,6,7,8-tetrahydronaphthalen-2-yl)-6-oxo-5-phenyl-7H-thieno)(2,3-b)pyridin-4-olate potassium

Ancestor Terms

Not provided

Browse Leaves

Not provided

Browse Branches

Not provided