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NCT05146284 | Not yet recruiting | Adrenomyeloneuropathy

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

Pohel SA

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).

Condition or disease

Adrenomyeloneuropathy

Intervention/treatment

PXL770

Phase

Phase 2

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.}}

Study Type : Interventional
Estimated Enrollment : 24 participants
Masking : None (Open Label)
Primary Purpose : Treatment
Official Title : A Randomized Open-label Phase 2a Study to Assess PXL770 After 12 Weeks of Treatment in Male Subjects With Adrenomyeloneuropathy (AMN) Form of X-linked Adrenoleukodystrophy (X-ALD or ALD)
Actual Study Start Date : September 2023
Estimated Primary Completion Date : September 2024
Estimated Study Completion Date : September 2024
Arm Intervention/treatment

Experimental: PXL770 500 mg QD

Drug: PXL770

Experimental: PXL770 250 mg BID

Drug: PXL770
Ages Eligible for Study: 18 Years to 65 Years
Sexes Eligible for Study: Male
Accepts Healthy Volunteers: No
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.
Study to Assess PXL770 in Subjects With Adrenomyeloneuropathy (AMN) Form of X-linked Adrenoleukodystrophy (X-ALD or ALD)

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

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