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

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Brief Title

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

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)

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).
    

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 Phase

Phase 2

Study Type

Interventional


Primary Outcome

Pharmacokinetic (PK) parameters


Condition

Adrenomyeloneuropathy

Intervention

PXL770

Study Arms / Comparison Groups

 PXL770 500 mg QD
Description:  

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

24

Start Date

December 2022

Completion Date

March 2023

Primary Completion Date

March 2023

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.
      

Gender

Male

Ages

18 Years - 65 Years

Accepts Healthy Volunteers

No

Contacts

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Administrative Informations


NCT ID

NCT05146284

Organization ID

PXL770-011


Responsible Party

Sponsor

Study Sponsor

Poxel SA


Study Sponsor

, , 


Verification Date

September 2022