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O estudo clínico NCT04973657 para Adrenomyeloneuropathy Form (AMN) of X-linked Adrenoleukodystrophy (X-ALD) está concluído. Consulte a visualização em cartões do Radar de Estudos Clínicos e as ferramentas de descoberta de IA para ver todos os detalhes. Ou pergunte qualquer coisa aqui. | ||
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Viking TherapeuticsA Study to Assess the Pharmacodynamics of VK0214 in Male Subjects With AMN Fase I 24 Randomizado Escalonamento de dose
Os detalhes do estudo clínico estão disponíveis principalmente em inglês. No entanto, a IA Trial Radar pode ajudar! Basta clicar em 'Explicar o estudo' para visualizar e discutir as informações do estudo no idioma selecionado.
O estudo clínico NCT04973657 avaliou outros para Adrenomyeloneuropathy Form (AMN) of X-linked Adrenoleukodystrophy (X-ALD). Foi um estudo intervencionista de Fase I. Seu status atual é: concluído. O estudo iniciou em 22 de junho de 2021 e incluiu 24 participantes. Coordenado por Viking Therapeutics e foi concluído em 8 de novembro de 2024. Essas informações foram atualizadas no ClinicalTrials.gov em 17 de fevereiro de 2025.
Resumo
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.
Descrição detalhada
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 ...
Mostrar maisTítulo oficial
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)
Condições médicas
Adrenomyeloneuropathy Form (AMN) of X-linked Adrenoleukodystrophy (X-ALD)Outros IDs do estudo
- VK0214-102
Número NCT
Data de início (real)
2021-06-22
Última atualização postada
2025-02-17
Data de conclusão (estimada)
2024-11-08
Inscrição (estimada)
24
Tipo de estudo
Intervencionista
FASE
Fase I
Status
Concluído
Palavras-chave
AMN, XALD
Propósito principal
Outro
Alocação do design
Randomizado
Modelo de intervenção
Sequencial
Cegamento (Mascaramento)
Quádruplo-cego
Braços / Intervenções
| Grupo de participantes/Braço | Intervenção/Tratamento |
|---|---|
ExperimentalVK0214 Active 20mg 20mg QD | VK0214 API in capsule |
ExperimentalVK0214 Active 40mg 40mg QD | VK0214 API in capsule |
Comparador placeboPlacebo Placebo QD | PLACEBO API in capsule |
Desfecho primário
Desfecho secundário
| Medida de desfecho | Descrição da medida | Prazo |
|---|---|---|
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 |
| Medida de desfecho | Descrição da medida | Prazo |
|---|---|---|
Evaluate the Pharmacokinetics of VK0214 | Pharmacokinetic profile of VK0214 by measuring Peak Plasma Concentration (Cmax) | 28 days |
Critérios de elegibilidade
Idades elegíveis
Adulto, Idoso
Idade mínima
18 Years
Sexos elegíveis
Todos
- 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 TherapeuticsSem dados de contato.
9 Locais do estudo em 5 países
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