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Phase I Study of Ascending Doses of MMV390048 in Healthy Adult Volunteers

16 de julho de 2019 atualizado por: Medicines for Malaria Venture

A Single Centre, Two-part, Double-blind, Randomized, Placebo-controlled Phase I Study to Investigate the Safety, Tolerability, and Pharmacokinetic Profile of Ascending Doses of MMV390048 in Healthy Adult Volunteers

This is a first-in-human study of MMV390048. The study will evaluate the safety, tolerability and pharmacokinetic properties of escalating single and multiple doses of MMV390048 when administered to healthy male volunteers and female volunteers of non-childbearing potential.

In addition, the effect of food on the pharmacokinetics and tolerability of MMV390048 will be investigated.

Visão geral do estudo

Descrição detalhada

The study is a single centre, double-blind, randomised, placebo-controlled, ascending dose study in healthy male and female volunteers (of non-childbearing potential) aged 18 to 55 years.

The study will be divided into two parts. The first part will comprise up to seven fasted cohorts (8 to 10 volunteers in each) that will receive a single, ascending dose (SAD) of MMV390048 to assess its safety, tolerability and pharmacokinetic profile. The starting dose administered to the first cohort will be 5 mg. An additional cohort (cohort 8, re-using volunteers from one of the previous cohorts) will receive a single dose of MMV390048 in a fed state to evaluate the effect of food on the pharmacokinetics and tolerability of the compound.

The data obtained from each cohort during the SAD part of the study will undergo a formal review by the Safety Review Team (SRT). Should the safety profile of the compound be deemed acceptable, and the pharmacokinetic parameters indicate that acceptable levels of the drug to elicit a pharmacodynamic response can be achieved in human plasma, the study will then proceed to the second part.

During the second part of the study volunteers will receive multiple, ascending doses (MAD) of MMV390048 to assess the pharmacokinetics, safety and tolerability following multiple oral doses. Up to three cohorts of eight volunteers each will be enrolled into this part of the study. Each volunteer will receive three consecutive daily doses of MMV390048.

Tipo de estudo

Intervencional

Inscrição (Real)

48

Estágio

  • Fase 1

Contactos e Locais

Esta seção fornece os detalhes de contato para aqueles que conduzem o estudo e informações sobre onde este estudo está sendo realizado.

Locais de estudo

      • Cape Town, África do Sul
        • Cinical Pharmacology, University of Cape Town

Critérios de participação

Os pesquisadores procuram pessoas que se encaixem em uma determinada descrição, chamada de critérios de elegibilidade. Alguns exemplos desses critérios são a condição geral de saúde de uma pessoa ou tratamentos anteriores.

Critérios de elegibilidade

Idades elegíveis para estudo

18 anos a 55 anos (Adulto)

Aceita Voluntários Saudáveis

Não

Gêneros Elegíveis para o Estudo

Tudo

Descrição

Inclusion Criteria:

  • written informed consent
  • Male and female (of non-childbearing potential); age 18 to 55 years, in good health as determined by past medical history, physical examination, vital signs, electrocardiogram, and laboratory tests at screening
  • Hematology, clinical chemistry and urinalysis results at screening that are within the local laboratory reference range or, if outside the range, not clinically significant. AST, ALT, lactate dehydrogenase, total bilirubin, haptoglobin and hemoglobin must be within the normal reference ranges
  • Body weight at least 50kg and body mass index within 18 to 32kg/m2
  • Good peripheral venous access
  • Able to communicate well with the investigator, to understand and comply with the requirements of the study
  • Agree to stay in contact with the study site for the duration of the study, provide updated contact information as necessary, and have no current plans to move away from the study area for the duration of the study

Exclusion Criteria:

  • Any acute illness upon admission to the unit on Day -1 or prior to dosing on Day 1
  • Use of any other investigational drug within 30 days or five half-lives (whichever is longer) prior to the first dose of MMV390048
  • history of hypersensitivity to any drugs
  • history of anaphylaxis or severe allergic reaction
  • Resting vital signs at either screening or baseline outside the defined ranges
  • Orthostatic changes in blood pressure and heart rate measurements greater than: 20 mmHg drop in systolic blood pressure; 10 mmHg drop in diastolic blood pressure; 20 beats per minute increase in heart rate
  • history of clinically significant ECG abnormalities, or any of the defined ECG abnormalities at either screening or baseline
  • History of malignancy of any organ system (other than localized basal cell carcinoma of the skin), treated or untreated, within the past five years, regardless of whether there is evidence of local recurrence or metastases
  • Pregnant or nursing (lactating) women
  • Women of child-bearing potential
  • males physiologically capable of conceiving offspring UNLESS the volunteer agrees to use condoms and ensure that his partner(s) is either not of child-bearing potential or uses a highly effective method of contraception for the entire duration of the study and for twelve weeks following the last study drug administration
  • Smokers (use of tobacco products in the previous three months)
  • Use of any prescription drugs, herbal supplements, over--the--counter medication or dietary supplements (vitamins included) within four weeks prior to initial dosing
  • Intake of grapefruit, grapefruit juice or other products containing grapefruit within 28 days of the first drug administration of the study drug
  • Excessive intake of caffeine drinks or energy drinks within 48 hours before admission defined as more than three 250 ml cups of coffee a day
  • Donation or loss of 400 ml or more of blood within eight weeks prior to screening or initial dosing
  • Plasma donation (>100 ml) within 60 days prior to first dosing
  • Hemoglobin levels below 12.5 g/dl (males) or 11.5 g/dl (females) at screening
  • Haptoglobin levels outside the reference range
  • Positive direct anti-globulin test
  • Liver enzymes other than ALT, AST and lactate dehydrogenase elevated ≥1.5 x ULN within two weeks prior to initial dosing
  • history of autonomic dysfunction within 3 years and/or recurrent history
  • History of immunodeficiency diseases, including a confirmed positive HIV test result
  • Positive Hepatitis B surface antigen or Hepatitis C antibody test result
  • History of recurrent infection
  • history of endocrine disease, in particular adrenal disorders such as Cushing's syndrome or Addison's disease, or diabetes mellitus
  • history of Gilbert's Syndrome
  • history of photosensitivity
  • history of any food allergy
  • Any surgical or medical condition which might significantly alter the absorption, distribution, metabolism, or excretion of drugs, or which may jeopardise the safety of the volunteer or the objectives of the study
  • History or presence of impaired renal function as indicated by clinically significantly abnormal creatinine or urea values, or abnormal urinary constituents
  • History of drug or alcohol abuse within the 12 months prior to dosing, or evidence of such abuse as indicated by the tests and laboratory assays at screening and/or baseline
  • Any clinically significant mental disorder that could limit the validity of informed consent or the volunteer's ability to comply with protocol requirements

Plano de estudo

Esta seção fornece detalhes do plano de estudo, incluindo como o estudo é projetado e o que o estudo está medindo.

Como o estudo é projetado?

Detalhes do projeto

  • Finalidade Principal: Tratamento
  • Alocação: Randomizado
  • Modelo Intervencional: Atribuição de grupo único
  • Mascaramento: Quadruplicar

Armas e Intervenções

Grupo de Participantes / Braço
Intervenção / Tratamento
Experimental: Cohort SAD1 Fasted
Five fasted cohorts will receive a single, ascending dose of MMV390048. The starting dose will be 5mg. Cohort SAD6 will receive a single dose in a fed state
Supplied as "powder in bottle" formulation for reconstitution pre-dose
Outros nomes:
  • MMV390048
Supplied as "powder in bottle" formulation for reconstitution pre-dose
Outros nomes:
  • Placebo
Experimental: Cohort SAD2 Fasted
Five fasted cohorts will receive a single, ascending dose of MMV390048. The starting dose will be 5mg. Cohort SAD6 will receive a single dose in a fed state
Supplied as "powder in bottle" formulation for reconstitution pre-dose
Outros nomes:
  • Placebo
Supplied as "powder in bottle" formulation for reconstitution pre-dose.
Outros nomes:
  • MMV390048
Experimental: Cohort SAD3 Fasted
Five fasted cohorts will receive a single, ascending dose of MMV390048. The starting dose will be 5mg. Cohort SAD6 will receive a single dose in a fed state
Supplied as "powder in bottle" formulation for reconstitution pre-dose
Outros nomes:
  • Placebo
Supplied as "powder in bottle" formulation for reconstitution pre-dose
Outros nomes:
  • MMV390048
Experimental: Cohort SAD4 Fasted
Five fasted cohorts will receive a single, ascending dose of MMV390048. The starting dose will be 5mg. Cohort SAD6 will receive a single dose in a fed state
Supplied as "powder in bottle" formulation for reconstitution pre-dose
Outros nomes:
  • Placebo
Supplied as "powder in bottle" formulation for reconstitution pre-dose
Outros nomes:
  • MMV390048
Experimental: Cohort SAD5 Fasted
Five fasted cohorts will receive a single, ascending dose of MMV390048. The starting dose will be 5mg. Cohort SAD6 will receive a single dose in a fed state
Supplied as "powder in bottle" formulation for reconstitution pre-dose
Outros nomes:
  • Placebo
Supplied as "powder in bottle" formulation for reconstitution pre-dose
Outros nomes:
  • MMV390048
Experimental: Cohort SAD6 Fed
Cohort SAD6, reusing volunteers from one of the previous cohorts, will receive a single dose in a fed state to evaluate the effect of food on the pharmacokinetics and tolerability
Supplied as "powder in bottle" formulation for reconstitution pre-dose
Outros nomes:
  • Placebo
Supplied as "powder in bottle" formulation for reconstitution pre-dose
Outros nomes:
  • MMV390048

O que o estudo está medindo?

Medidas de resultados primários

Medida de resultado
Descrição da medida
Prazo
Number of Participants With Adverse Events
Prazo: up to D29 or longer according to half life
Subject will be in-house up to D3, and then have a follow up visit at the site on D5, 7, 10, 14, 19, 26, 29 or longer according to half life
up to D29 or longer according to half life
Area Under the Plasma Concentration Versus Time Curve (AUC) of MMV390048
Prazo: 0.5, 1, 2, 3, 4, 6, 8, 12, 24, 48, 96, 144, 216, 312, 432, 600, 672 hours post-dose
Pk blood collection - additional PK point may be planned final visit depending on emerging PK data, unnecessary PK points could be eliminated for the latter cohorts Investigate the effect of food on the pharmacokinetic and tolerability of the investigational drug in cohort 4 and 8
0.5, 1, 2, 3, 4, 6, 8, 12, 24, 48, 96, 144, 216, 312, 432, 600, 672 hours post-dose
Half-life of MMV390048
Prazo: 0.5, 1, 2, 3, 4, 6, 8, 12, 24, 48, 96, 144, 216, 312, 432, 600, 672 hours post-dose
Pk blood collection Investigate the effect of food on the pharmacokinetic and tolerability of the investigational drug in cohort 4 and 8
0.5, 1, 2, 3, 4, 6, 8, 12, 24, 48, 96, 144, 216, 312, 432, 600, 672 hours post-dose

Medidas de resultados secundários

Medida de resultado
Descrição da medida
Prazo
Determine ex Vivo Efficacy (IC50)
Prazo: up to 144 hr post dose
Blood collection to determine efficacy of investigational drug against parasites using an ex vivo malaria assay - this was done only for cohort 3 The experimentally obtained bioassay IC50 values were determined and compared to IC50 obtained with reference serum sample spiked with a known amount of MMV390048 titrated into the P. falciparum assay.
up to 144 hr post dose

Colaboradores e Investigadores

É aqui que você encontrará pessoas e organizações envolvidas com este estudo.

Colaboradores

Investigadores

  • Investigador principal: Karen Barnes, Prof, University of Cape Town

Publicações e links úteis

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Datas de registro do estudo

Essas datas acompanham o progresso do registro do estudo e os envios de resumo dos resultados para ClinicalTrials.gov. Os registros do estudo e os resultados relatados são revisados ​​pela National Library of Medicine (NLM) para garantir que atendam aos padrões específicos de controle de qualidade antes de serem publicados no site público.

Datas Principais do Estudo

Início do estudo

1 de maio de 2014

Conclusão Primária (Real)

1 de fevereiro de 2015

Conclusão do estudo (Real)

1 de fevereiro de 2015

Datas de inscrição no estudo

Enviado pela primeira vez

29 de agosto de 2014

Enviado pela primeira vez que atendeu aos critérios de CQ

29 de agosto de 2014

Primeira postagem (Estimativa)

3 de setembro de 2014

Atualizações de registro de estudo

Última Atualização Postada (Real)

17 de julho de 2019

Última atualização enviada que atendeu aos critérios de controle de qualidade

16 de julho de 2019

Última verificação

1 de fevereiro de 2018

Mais Informações

Termos relacionados a este estudo

Outros números de identificação do estudo

  • MMV_MMV390048_14_01

Plano para dados de participantes individuais (IPD)

Planeja compartilhar dados de participantes individuais (IPD)?

Não

Informações sobre medicamentos e dispositivos, documentos de estudo

Estuda um medicamento regulamentado pela FDA dos EUA

Não

Estuda um produto de dispositivo regulamentado pela FDA dos EUA

Não

produto fabricado e exportado dos EUA

Não

Essas informações foram obtidas diretamente do site clinicaltrials.gov sem nenhuma alteração. Se você tiver alguma solicitação para alterar, remover ou atualizar os detalhes do seu estudo, entre em contato com register@clinicaltrials.gov. Assim que uma alteração for implementada em clinicaltrials.gov, ela também será atualizada automaticamente em nosso site .

Ensaios clínicos em MMV390048 5mg

3
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