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
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|Condition or disease||Intervention/treatment||Phase|
|Adrenomyeloneuropathy||Drug: PXL770||Phase 2|
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.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||24 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||None (Open Label)|
|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|
|Experimental: PXL770 500 mg QD||
|Experimental: PXL770 250 mg BID||
- Pharmacokinetic (PK) parameters [ Time Frame: 4 week ]Peak plasma concentration (Cmax) for 500mg QD
- Pk parameters [ Time Frame: 4 week ]Area under the plasma concentration versus time curve (AUC)0-24 for 500mg QD
- PK parameters [ Time Frame: 4 week ]Cmax for 250mg BID
- PK parameters [ Time Frame: 4 week ]AUC0-8 for 250mg BID
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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|
- 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.
- 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.
|Responsible Party:||Poxel SA|
|Other Study ID Numbers:||
|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|
|Studies a U.S. FDA-regulated Drug Product:||Yes|
|Studies a U.S. FDA-regulated Device Product:||No|
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Central Nervous System Diseases
Nervous System Diseases
Hereditary Central Nervous System Demyelinating Diseases
Mental Retardation, X-Linked
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Metabolism, Inborn Errors
Adrenal Gland Diseases
Endocrine System Diseases