Back to results

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

Primary Purpose

Adrenomyeloneuropathy

Status

Not yet recruiting

Phase

Phase 2

Locations

Study Type

Interventional

Intervention

PXL770

About this trial

This is an interventional treatment trial for Adrenomyeloneuropathy

Eligibility Criteria

18 Years - 65 Years (Adult, Older Adult)MaleDoes not accept healthy volunteers

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.

Sites / Locations

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Arm Label

PXL770 500 mg QD

PXL770 250 mg BID

Arm Description

Outcomes

Primary Outcome Measures

Pharmacokinetic (PK) parameters

Peak plasma concentration (Cmax) for 500mg QD

Pk parameters

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

PK parameters

Cmax for 250mg BID

PK parameters

AUC0-8 for 250mg BID

Secondary Outcome Measures

Full Information

NCT ID

NCT05146284

First Posted

November 16, 2021

Last Updated

April 3, 2023

Sponsor

Poxel SA

1. Study Identification

Unique Protocol Identification Number

NCT05146284

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)

Study Type

Interventional

2. Study Status

Record Verification Date

April 2023

Overall Recruitment Status

Not yet recruiting

Study Start Date

September 2023 (Anticipated)

Primary Completion Date

September 2024 (Anticipated)

Study Completion Date

September 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Poxel SA

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Adrenomyeloneuropathy

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Parallel Assignment

Masking

None (Open Label)

Allocation

Randomized

Enrollment

24 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

PXL770 500 mg QD

Arm Type

Experimental

Arm Title

PXL770 250 mg BID

Arm Type

Experimental

Intervention Type

Drug

Intervention Name(s)

PXL770

Intervention Description

Tablet

Primary Outcome Measure Information:

Title

Pharmacokinetic (PK) parameters

Description

Peak plasma concentration (Cmax) for 500mg QD

Time Frame

4 week

Title

Pk parameters

Description

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

Time Frame

4 week

Title

PK parameters

Description

Cmax for 250mg BID

Time Frame

4 week

Title

PK parameters

Description

AUC0-8 for 250mg BID

Time Frame

4 week

10. Eligibility

Sex

Male

Minimum Age & Unit of Time

18 Years

Maximum Age & Unit of Time

65 Years

Accepts Healthy Volunteers

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.

12. IPD Sharing Statement

Plan to Share IPD

IPD Sharing Plan Description

There is no plan to share IPD with other researchers

Learn more about this trial

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

We'll reach out to this number within 24 hrs