Trial on patients with adrenomyeloneuropathy to assess PXL770's behaviour, a new potential medicine, in the body

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)

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2021-006223-18-NL

Enrollment

Registered

2022-02-17

Start date

2022-07-22

Completion date

Unknown

Last updated

2024-12-16

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Adrenomyeloneuropathy (AMN)

Interventions

Product Name: PXL770 Product Code: PXL770 Pharmaceutical Form: Film-coated tablet INN or Proposed INN: PXL770 CAS Number: 1523493-78-8 Current Sponsor code: PXL770 Other descriptive name: PXL770 Conce

Eligibility

Sex/Gender

Male

Inclusion criteria

Inclusion criteria: 1. Male subjects with either a confirmed diagnosis of AMN by genetic testing or a family history of ALD together with an elevation in VLCFA obtained from overnight fasting plasma sample at V1. 2. Age: = 18 to = 65 years at informed consent signature. 3. Normal brain MRI or brain MRI showing non-specific abnormalities that can be observed in AMN subjects without signs of C-ALD Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 22 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 2

Exclusion criteria

Exclusion criteria: Target disease exclusions 1. Any progressive neurological disease other than AMN. 2. Arrested or progressing C-ALD as defined by cerebral lesions. 3. Prior receipt of an allogeneic hematopoietic stem cell transplant or gene therapy.

Design outcomes

Primary

Measure	Time frame
Main Objective: Efficacy : To assess the PK of PXL770 in AMN subjects at the daily dose of 500mg OD and 250mg BID after 4 weeks of treatment ;Secondary Objective: 1. To assess the safety and tolerability of PXL770 in AMN subjects at the daily dose of 500mg OD and 250mg BID after 12 weeks of treatment 2.To assess the effect of PXL770 at the daily dose of 500mg OD and 250mg BID on PD biomarkers: - VLCFA in plasma - NfL in plasma - Lipid and glycemic parameters in AMN subjects up to 12 weeks of treatment 3.To compare the 2 doses regimens of PXL770 (BID versus OD) on PK, safety and PD parameters in AMN subjects up to 12 weeks of treatment Exploratory Objectives : To assess the effect of PXL770 at the daily dose of 500mg OD and 250mg BID on other parameters: - VLCFA in PBMC - Biomarkers of inflammation - Biomarkers of PXL770 mechanism of action in AMN subjects up to 12 weeks of treatment;Primary end point(s): Primary plasma PK parameters of PXL770: Cmax and AUC0-24 at V3 for 500mg OD treatment group and Cmax and AUC0-8 at Week 4 (V3) for 250mg BID treatment group.;Timepoint(s) of evaluation of this end point: Week 4 (V3)	—

Measure

Time frame

Main Objective: Efficacy : To assess the PK of PXL770 in AMN subjects at the daily dose of 500mg OD and 250mg BID after 4 weeks of treatment ;Secondary Objective: 1. To assess the safety and tolerability of PXL770 in AMN subjects at the daily dose of 500mg OD and 250mg BID after 12 weeks of treatment 2.To assess the effect of PXL770 at the daily dose of 500mg OD and 250mg BID on PD biomarkers: - VLCFA in plasma - NfL in plasma - Lipid and glycemic parameters in AMN subjects up to 12 weeks of treatment 3.To compare the 2 doses regimens of PXL770 (BID versus OD) on PK, safety and PD parameters in AMN subjects up to 12 weeks of treatment Exploratory Objectives : To assess the effect of PXL770 at the daily dose of 500mg OD and 250mg BID on other parameters: - VLCFA in PBMC - Biomarkers of inflammation - Biomarkers of PXL770 mechanism of action in AMN subjects up to 12 weeks of treatment;Primary end point(s): Primary plasma PK parameters of PXL770: Cmax and AUC0-24 at V3 for 500mg OD treatment group and Cmax and AUC0-8 at Week 4 (V3) for 250mg BID treatment group.;Timepoint(s) of evaluation of this end point: Week 4 (V3)

—

Secondary

Measure	Time frame
Secondary end point(s): Secondary plasma PK parameters of PXL770: • Ctrough at Week 4, week 8 and week 12, • Cavg, CLss/F, AUC0-8 (for 500mg OD treatment group) tmax and AUClast at week 8.;Timepoint(s) of evaluation of this end point: Week 4, week 8 and week 12	—

Countries

Germany, Netherlands

Contacts

Public ContactCarole THANG

Poxel S.A.

carole.thang@poxelpharma.com+33437372010

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026