Status:
WITHDRAWN
Study to Assess PXL065 in Subjects With Adrenomyeloneuropathy (AMN) Form of X-linked Adrenoleukodystrophy (X-ALD or ALD)
Lead Sponsor:
Poxel SA
Conditions:
ALD (Adrenoleukodystrophy)
Eligibility:
MALE
18-65 years
Phase:
PHASE2
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 wil...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
September 1 2023
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
September 1 2024
Estimated Enrollment :
Patients enrolled
Trial Details
Trial ID
NCT05200104
Start Date
September 1 2023
End Date
September 1 2024
Last Update
December 16 2024
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