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Study to Assess PXL065 in Subjects With Adrenomyeloneuropathy (AMN) Form of X-linked Adrenoleukodystrophy (X-ALD or ALD)

Primary Purpose

ALD (Adrenoleukodystrophy)

Status
Not yet recruiting
Phase
Phase 2
Locations
Study Type
Interventional
Intervention
PXL065
Sponsored by
Poxel SA
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for ALD (Adrenoleukodystrophy)

Eligibility Criteria

18 Years - 65 Years (Adult, Older Adult)MaleDoes not accept 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

Sites / Locations

    Arms of the Study

    Arm 1

    Arm Type

    Experimental

    Arm Label

    PXL065

    Arm Description

    PXL065 22.5 mg QD

    Outcomes

    Primary Outcome Measures

    Pharmacokinetic
    Deuterated and protonated forms of (R)-pioglitazone and (S)-pioglitazone: Cmax
    PharmacoKinetic
    Deuterated and protonated forms of (R)-pioglitazone and (S)-pioglitazone: AUC0-24 (AUCtau)

    Secondary Outcome Measures

    Full Information

    First Posted
    January 6, 2022
    Last Updated
    April 3, 2023
    Sponsor
    Poxel SA
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    1. Study Identification

    Unique Protocol Identification Number
    NCT05200104
    Brief Title
    Study to Assess PXL065 in Subjects With Adrenomyeloneuropathy (AMN) Form of X-linked Adrenoleukodystrophy (X-ALD or ALD)
    Official Title
    An Open-label Phase 2a Study to Assess the Pharmacokinetics and Pharmacodynamic of PXL065 Parameters After 12 Weeks of Treatment in Male Subjects With Adrenomyeloneuropathy (AMN) Form of X-linked Adrenoleukodystrophy (X-ALD or ALD)
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    April 2023
    Overall Recruitment Status
    Not yet recruiting
    Study Start Date
    September 2023 (Anticipated)
    Primary Completion Date
    September 2024 (Anticipated)
    Study Completion Date
    September 2024 (Anticipated)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    Poxel SA

    4. Oversight

    Studies a U.S. FDA-regulated Drug Product
    Yes
    Studies a U.S. FDA-regulated Device Product
    No
    Data Monitoring Committee
    No

    5. Study Description

    Brief Summary
    A randomized open-label Phase 2a study to assess the pharmacokinetics and pharmacodynamic parameters of PXL065 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 PXL065 after 12 weeks of treatment in male subjects with adrenomyeloneuropathy (AMN). There will be a total of 3 study periods. Screening period of a maximum of 4 weeks prior to the open-label Treatment Period (Baseline Visit - V2). This period can be exceptionally extended for 2 weeks Open-label Treatment Period of 12 weeks Follow-up Period of 2 weeks after the last intake of the treatment (V5-End of Treatment Visit (EoT)). During the treatment period, very long chain fatty acids (VLCFA) will be assessed every 4 weeks, to evaluate the kinetics of the PXL065 effect. Neurofilament light (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 continued monitoring the subjects' safety and evaluation of the kinetics of the 2 main biomarkers (VLCFA and NfL) at 2 weeks after the last intake of the treatment.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    ALD (Adrenoleukodystrophy)

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 2
    Interventional Study Model
    Single Group Assignment
    Masking
    None (Open Label)
    Allocation
    N/A
    Enrollment
    12 (Anticipated)

    8. Arms, Groups, and Interventions

    Arm Title
    PXL065
    Arm Type
    Experimental
    Arm Description
    PXL065 22.5 mg QD
    Intervention Type
    Drug
    Intervention Name(s)
    PXL065
    Intervention Description
    PXL065 22.5 mg QD
    Primary Outcome Measure Information:
    Title
    Pharmacokinetic
    Description
    Deuterated and protonated forms of (R)-pioglitazone and (S)-pioglitazone: Cmax
    Time Frame
    Week 4
    Title
    PharmacoKinetic
    Description
    Deuterated and protonated forms of (R)-pioglitazone and (S)-pioglitazone: AUC0-24 (AUCtau)
    Time Frame
    Week 4

    10. Eligibility

    Sex
    Male
    Minimum Age & Unit of Time
    18 Years
    Maximum Age & Unit of Time
    65 Years
    Accepts Healthy Volunteers
    No
    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

    12. IPD Sharing Statement

    Learn more about this trial

    Study to Assess PXL065 in Subjects With Adrenomyeloneuropathy (AMN) Form of X-linked Adrenoleukodystrophy (X-ALD or ALD)

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