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WithdrawnNCT05146284Updated Dec 16, 2024

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

A Phase 2 interventional study of PXL770 in Adrenomyeloneuropathy, sponsored by Poxel SA. Withdrawn. Open to male participants aged 18 Years to 65 Years. Per ClinicalTrials.gov, last updated 2024-12-16.

Sponsored by Poxel SA · Phase 2, Interventional, and Treatment

Why this study was withdrawn
Lack of funding to initiate the study
Phase
Phase 2
Study type
Interventional
Enrollment
0
Allocation
Randomized
Ages
18 Years to 65 Years
Sex
Male
01

Study 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).

Read the 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.

02

Conditions studied

  • Adrenomyeloneuropathy

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03

In context

Adrenoleukodystrophy

68 studies on the registry are indexed under Adrenoleukodystrophy; 19 are open to participants now.

Browse Adrenoleukodystrophy studies →

Lead sponsor

Poxel SA is the lead sponsor of 14 studies on the registry; none are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years to 65 Years
Sexes eligible
Male
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

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 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

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 hematopoietic stem cell transplant or gene therapy.
Terms in these criteria, in plain language(2)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Informed consent

Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.

You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.

Allogeneic and autologous transplant

Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.

05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
None (open label)
Enrollment
0 participants (actual)

Study arms

  • Experimental
    PXL770 500 mg QD

    Drug: PXL770

  • Experimental
    PXL770 250 mg BID

    Drug: PXL770

Interventions

06

What researchers measure

Primary outcomes

  1. Pharmacokinetic (PK) parameters

    Peak plasma concentration (Cmax) for 500mg QD

    Time frame: 4 week

  2. Pk parameters

    Area under the plasma concentration versus time curve (AUC)0-24 for 500mg QD

    Time frame: 4 week

  3. PK parameters

    Cmax for 250mg BID

    Time frame: 4 week

  4. PK parameters

    AUC0-8 for 250mg BID

    Time frame: 4 week

07

Study locations

No study locations are listed for this record.

08

References and documents

Individual participant data

Plan to share: No — There is no plan to share IPD with other researchers

No publications or documents are linked to this record.

09

Timeline

Tracking since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reached

Start is estimated for Sep 2023; primary completion for Sep 2024, 12 months later.

How these figures are worked out, and which studies are left out of them.

Registered
Nov 16, 2021
First posted
Dec 6, 2021
StartEst.
Sep 2023
Primary completionEst.
Sep 2024
CompletionEst.
Sep 2024
Last update
Dec 16, 2024
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on Dec 16, 2024, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT05146284
Lead sponsor
Poxel SA
Responsible party
Sponsor
First posted
Dec 6, 2021
Start date
Sep 2023 (estimated)
Primary completion
Sep 2024 (estimated)
Completion
Sep 2024 (estimated)
Last update
Dec 16, 2024

Oversight

Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is withdrawn, as verified in Dec 2024. You cannot join it, but the record below documents what was studied.

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Discussion

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Studies of the drug tested here: Pxl-770

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