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

16 de julio de 2019 actualizado 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.

Descripción general del estudio

Descripción detallada

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 estudio

Intervencionista

Inscripción (Actual)

48

Fase

  • Fase 1

Contactos y Ubicaciones

Esta sección proporciona los datos de contacto de quienes realizan el estudio e información sobre dónde se lleva a cabo este estudio.

Ubicaciones de estudio

      • Cape Town, Sudáfrica
        • Cinical Pharmacology, University of Cape Town

Criterios de participación

Los investigadores buscan personas que se ajusten a una determinada descripción, denominada criterio de elegibilidad. Algunos ejemplos de estos criterios son el estado de salud general de una persona o tratamientos previos.

Criterio de elegibilidad

Edades elegibles para estudiar

18 años a 55 años (Adulto)

Acepta Voluntarios Saludables

No

Géneros elegibles para el estudio

Todos

Descripción

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

Plan de estudios

Esta sección proporciona detalles del plan de estudio, incluido cómo está diseñado el estudio y qué mide el estudio.

¿Cómo está diseñado el estudio?

Detalles de diseño

  • Propósito principal: Tratamiento
  • Asignación: Aleatorizado
  • Modelo Intervencionista: Asignación de un solo grupo
  • Enmascaramiento: Cuadruplicar

Armas e Intervenciones

Grupo de participantes/brazo
Intervención / Tratamiento
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
Otros nombres:
  • MMV390048
Supplied as "powder in bottle" formulation for reconstitution pre-dose
Otros nombres:
  • 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
Otros nombres:
  • Placebo
Supplied as "powder in bottle" formulation for reconstitution pre-dose.
Otros nombres:
  • 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
Otros nombres:
  • Placebo
Supplied as "powder in bottle" formulation for reconstitution pre-dose
Otros nombres:
  • 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
Otros nombres:
  • Placebo
Supplied as "powder in bottle" formulation for reconstitution pre-dose
Otros nombres:
  • 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
Otros nombres:
  • Placebo
Supplied as "powder in bottle" formulation for reconstitution pre-dose
Otros nombres:
  • 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
Otros nombres:
  • Placebo
Supplied as "powder in bottle" formulation for reconstitution pre-dose
Otros nombres:
  • MMV390048

¿Qué mide el estudio?

Medidas de resultado primarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Number of Participants With Adverse Events
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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 resultado secundarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Determine ex Vivo Efficacy (IC50)
Periodo de tiempo: 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

Aquí es donde encontrará personas y organizaciones involucradas en este estudio.

Colaboradores

Investigadores

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

Publicaciones y enlaces útiles

La persona responsable de ingresar información sobre el estudio proporciona voluntariamente estas publicaciones. Estos pueden ser sobre cualquier cosa relacionada con el estudio.

Fechas de registro del estudio

Estas fechas rastrean el progreso del registro del estudio y los envíos de resultados resumidos a ClinicalTrials.gov. Los registros del estudio y los resultados informados son revisados ​​por la Biblioteca Nacional de Medicina (NLM) para asegurarse de que cumplan con los estándares de control de calidad específicos antes de publicarlos en el sitio web público.

Fechas importantes del estudio

Inicio del estudio

1 de mayo de 2014

Finalización primaria (Actual)

1 de febrero de 2015

Finalización del estudio (Actual)

1 de febrero de 2015

Fechas de registro del estudio

Enviado por primera vez

29 de agosto de 2014

Primero enviado que cumplió con los criterios de control de calidad

29 de agosto de 2014

Publicado por primera vez (Estimar)

3 de septiembre de 2014

Actualizaciones de registros de estudio

Última actualización publicada (Actual)

17 de julio de 2019

Última actualización enviada que cumplió con los criterios de control de calidad

16 de julio de 2019

Última verificación

1 de febrero de 2018

Más información

Términos relacionados con este estudio

Otros números de identificación del estudio

  • MMV_MMV390048_14_01

Plan de datos de participantes individuales (IPD)

¿Planea compartir datos de participantes individuales (IPD)?

No

Información sobre medicamentos y dispositivos, documentos del estudio

Estudia un producto farmacéutico regulado por la FDA de EE. UU.

No

Estudia un producto de dispositivo regulado por la FDA de EE. UU.

No

producto fabricado y exportado desde los EE. UU.

No

Esta información se obtuvo directamente del sitio web clinicaltrials.gov sin cambios. Si tiene alguna solicitud para cambiar, eliminar o actualizar los detalles de su estudio, comuníquese con register@clinicaltrials.gov. Tan pronto como se implemente un cambio en clinicaltrials.gov, también se actualizará automáticamente en nuestro sitio web. .

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