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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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6 January 2025 |
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Main ID: |
NCT05200104 |
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Date of registration:
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06/01/2022 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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Study to Assess PXL065 in Subjects With Adrenomyeloneuropathy (AMN) Form of X-linked Adrenoleukodystrophy (X-ALD or ALD)
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Scientific title:
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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) |
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Date of first enrolment:
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September 2023 |
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Target sample size:
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0 |
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Recruitment status: |
Withdrawn |
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URL:
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https://clinicaltrials.gov/ct2/show/NCT05200104 |
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Study type:
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Interventional |
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Study design:
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Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 2
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Key inclusion & exclusion criteria
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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
Age minimum:
18 Years
Age maximum:
65 Years
Gender:
Male
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Health Condition(s) or Problem(s) studied
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ALD (Adrenoleukodystrophy)
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Intervention(s)
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Drug: PXL065
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Primary Outcome(s)
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Pharmacokinetic
[Time Frame: Week 4]
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Secondary ID(s)
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PXL065-005
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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