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
About this trial
This is an interventional treatment trial for ALD (Adrenoleukodystrophy)
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
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
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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