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

NCT05146284 | Withdrawn Phase 2 FDA Drug

• 1 other identifier: PXL770-011
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

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).

Conditions

Adrenomyeloneuropathy

Outcome Measures

Primary Outcomes (4)

• Pharmacokinetic (PK) parameters

  Peak plasma concentration (Cmax) for 500mg QD

  4 week

• Pk parameters

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

  4 week

• PK parameters

  Cmax for 250mg BID

  4 week

• PK parameters

  AUC0-8 for 250mg BID

  4 week

Study Arms (2)

PXL770 500 mg QD

EXPERIMENTAL

Drug: PXL770

PXL770 250 mg BID

EXPERIMENTAL

Drug: PXL770

Interventions

PXL770 DRUG

Tablet

PXL770 250 mg BID; PXL770 500 mg QD

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Poxel SA: lead

MeSH Terms

Conditions

Adrenoleukodystrophy

Interventions

2-chloro-3-(1-hydroxy-5,6,7,8-tetrahydronaphthalen-2-yl)-6-oxo-5-phenyl-7H-thieno)(2,3-b)pyridin-4-olate potassium

Condition Hierarchy (Ancestors)

Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Hereditary Central Nervous System Demyelinating Diseases; Leukoencephalopathies; Demyelinating Diseases; X-Linked Intellectual Disability; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Heredodegenerative Disorders, Nervous System; Metabolism, Inborn Errors; Peroxisomal Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Adrenal Insufficiency; Adrenal Gland Diseases; Endocrine System Diseases

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 16, 2021
• First Posted: December 6, 2021
• Study Start: September 1, 2023
• Primary Completion: September 1, 2024
• Study Completion: September 1, 2024
• Last Updated: December 16, 2024

Record last verified: 2024-12

Data Sharing

• IPD Sharing: Will not share