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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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ClinicalTrials.gov Identifier: NCT05146284
Recruitment Status : Not yet recruiting
First Posted : December 6, 2021
Last Update Posted : April 4, 2023
Information provided by (Responsible Party):
Poxel 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 Intervention/treatment Phase
Adrenomyeloneuropathy Drug: PXL770 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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 24 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
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)
Estimated 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

Primary Outcome Measures :
  1. Pharmacokinetic (PK) parameters [ Time Frame: 4 week ]
    Peak plasma concentration (Cmax) for 500mg QD

  2. Pk parameters [ Time Frame: 4 week ]
    Area under the plasma concentration versus time curve (AUC)0-24 for 500mg QD

  3. PK parameters [ Time Frame: 4 week ]
    Cmax for 250mg BID

  4. PK parameters [ Time Frame: 4 week ]
    AUC0-8 for 250mg BID

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No

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.
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Responsible Party: Poxel SA
ClinicalTrials.gov Identifier: NCT05146284    
Other Study ID Numbers: PXL770-011
First Posted: December 6, 2021    Key Record Dates
Last Update Posted: April 4, 2023
Last Verified: April 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Plan Description: There is no plan to share IPD with other researchers

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Hereditary Central Nervous System Demyelinating Diseases
Demyelinating Diseases
Mental Retardation, X-Linked
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Metabolism, Inborn Errors
Peroxisomal Disorders
Metabolic Diseases
Adrenal Insufficiency
Adrenal Gland Diseases
Endocrine System Diseases