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De klinische studie NCT04973657 voor Adrenomyeloneuropathy Form (AMN) of X-linked Adrenoleukodystrophy (X-ALD) is afgerond. Bekijk de kaartweergave van de Klinische Studies Radar en de AI-ontdekkingstools voor alle details. Of stel hier een vraag.
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A Study to Assess the Pharmacodynamics of VK0214 in Male Subjects With AMN Fase 1 24 Gerandomiseerd Dosis-escalatie

Afgerond
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De klinische studie NCT04973657 onderzocht overige bij Adrenomyeloneuropathy Form (AMN) of X-linked Adrenoleukodystrophy (X-ALD). Deze Fase 1 interventioneel studie is nu afgerond. Er werd gestreefd naar inclusie van 24 deelnemers vanaf 22 juni 2021. De studie werd geleid door Viking Therapeutics en was gepland te worden voltooid op 8 november 2024. Laatste update op ClinicalTrials.gov: 17 februari 2025.
Beknopte samenvatting
Subjects will be administered multiple doses of VK0214 in a cohort dose escalation process. A total of up to 36 subjects will be enrolled in the study, into one of three dose cohorts. For each cohort, up to 12 subjects will be randomized to receive VK0214 or placebo in a 3:1 ratio so that there will be a total of up to 9 subjects for each of the active doses and up to 3 subjects dosed with placebo in each cohort.
Uitgebreide beschrijving
The first part of VK0214-102 will include the first 2 dose cohorts and placebo, which will be dosed in parallel (up to N=24). The first 2 doses used in these cohorts will be 20 mg QD and 40 mg QD for 28 days. A Dose Level Review Team (DLRT) meeting will be held at the end of the first 2 parallel doses.

The DLRT will review safety, tolerability, and preliminary efficacy data from cohort 1 and 2. If the data analyzed ...

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Officiële titel

Phase 1b, Multi-center, Randomized, Double-Blind, Placebo-Controlled, Study to Evaluate the Safety, Tolerability, PD and PK of VK0214, in Subjects With the Adrenomyeloneuropathy Form (AMN) of X-linked Adrenoleukodystrophy (X-ALD)

Aandoeningen
Adrenomyeloneuropathy Form (AMN) of X-linked Adrenoleukodystrophy (X-ALD)
Andere studie-ID's
  • VK0214-102
NCT-ID
NCT04973657
Startdatum (Werkelijk)
2021-06-22
Laatste update geplaatst
2025-02-17
Verwachte einddatum
2024-11-08
Inschrijving (Geschat)
24
Studietype
Interventioneel
FASE
Fase 1
Status
Afgerond
Trefwoorden
AMN, XALD
Primaire doel
Overige
Toewijzing
Gerandomiseerd
Interventiemodel
Sequentieel
Blindering
Viervoudig blind
Armen / Interventies
Deelnemersgroep/StudiearmInterventie/Behandeling
ExperimenteelVK0214 Active 20mg
20mg QD
VK0214
API in capsule
ExperimenteelVK0214 Active 40mg
40mg QD
VK0214
API in capsule
Placebo-comparatorPlacebo
Placebo QD
PLACEBO
API in capsule
Primaire uitkomst
UitkomstmaatBeschrijving van de uitkomstmaatTijdsbestek
Incidence of treatment-emergent adverse events (TEAEs) and treatment-emergent serious AEs (TESAEs)
Evaluate the safety and tolerability of multiple oral doses of VK0214 in subjects with AMN
28 days
Secundaire uitkomst
UitkomstmaatBeschrijving van de uitkomstmaatTijdsbestek
Evaluate the Pharmacokinetics of VK0214
Pharmacokinetic profile of VK0214 by measuring Peak Plasma Concentration (Cmax)
28 days
Geschiktheidscriteria

Leeftijd van deelnemers
Volwassene, Oudere volwassene
Minimumleeftijd
18 Years
Geslachten die in aanmerking komen voor de studie
Allen
  • Provide a personally signed and dated informed consent document indicating that the subject has been informed of all pertinent aspects of the study and is willing and able to participate.
  • Have diagnosis of X-linked adrenoleukodystrophy (X-ALD) by genetic testing and elevated VLCFAs.
  • Present clinical features of AMN, or adrenal insufficiency.
  • Subjects must be 18 years of age and older.

  • Have the cerebral form of X-ALD, based on medical records, or based on MRI performed within the last 12 months or MRI at screening
  • Have been treated with any drug targeting TRβ or with any drug being tested as therapy for X-ALD AMN form within the past 30 days before screening
  • History or presence of clinically significant acute or unstable hepatic, renal, gastrointestinal, pulmonary, immunological, endocrine, diabetes, hematological or oncological that in the opinion of the investigator would pose a significant risk for the subject
  • Subject has untreated primary adrenal insufficiency based on morning plasma cortisol and ACTH at screening
  • Lorenzo's Oil. Currently taking Lorenzo's Oil or erucic acid containing product, or other lipid lowering agent known to effect VLCFA levels
Viking Therapeutics, Inc. logoViking Therapeutics
Geen contactgegevens beschikbaar
9 Studielocaties in 5 landen

California

Viking Clinical Site 203, Palo Alto, California, 94303, United States

Florida

Viking Clinical Site 207, Gainesville, Florida, 32610, United States

Maryland

Viking Clinical Site 201, Baltimore, Maryland, 21205, United States

Washington

Viking Clinical Site 205, Seattle, Washington, 98195, United States
Viking Clinical Site 214, Bordeaux, 33076, France
Viking Clinical Site 215, Montpellier, 34295, France
Viking Clinical Site 209, Leipzig, 04103, Germany
Viking Clinical Site 210, Milan, 20133, Italy
Viking Clinical Site 212, London, WC1N3BG, United Kingdom