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

16 juillet 2019 mis à jour par: 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.

Aperçu de l'étude

Description détaillée

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.

Type d'étude

Interventionnel

Inscription (Réel)

48

Phase

  • La phase 1

Contacts et emplacements

Cette section fournit les coordonnées de ceux qui mènent l'étude et des informations sur le lieu où cette étude est menée.

Lieux d'étude

      • Cape Town, Afrique du Sud
        • Cinical Pharmacology, University of Cape Town

Critères de participation

Les chercheurs recherchent des personnes qui correspondent à une certaine description, appelée critères d'éligibilité. Certains exemples de ces critères sont l'état de santé général d'une personne ou des traitements antérieurs.

Critère d'éligibilité

Âges éligibles pour étudier

18 ans à 55 ans (Adulte)

Accepte les volontaires sains

Non

Sexes éligibles pour l'étude

Tout

La description

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 d'étude

Cette section fournit des détails sur le plan d'étude, y compris la façon dont l'étude est conçue et ce que l'étude mesure.

Comment l'étude est-elle conçue ?

Détails de conception

  • Objectif principal: Traitement
  • Répartition: Randomisé
  • Modèle interventionnel: Affectation à un seul groupe
  • Masquage: Quadruple

Armes et Interventions

Groupe de participants / Bras
Intervention / Traitement
Expérimental: 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
Autres noms:
  • MMV390048
Supplied as "powder in bottle" formulation for reconstitution pre-dose
Autres noms:
  • Placebo
Expérimental: 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
Autres noms:
  • Placebo
Supplied as "powder in bottle" formulation for reconstitution pre-dose.
Autres noms:
  • MMV390048
Expérimental: 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
Autres noms:
  • Placebo
Supplied as "powder in bottle" formulation for reconstitution pre-dose
Autres noms:
  • MMV390048
Expérimental: 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
Autres noms:
  • Placebo
Supplied as "powder in bottle" formulation for reconstitution pre-dose
Autres noms:
  • MMV390048
Expérimental: 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
Autres noms:
  • Placebo
Supplied as "powder in bottle" formulation for reconstitution pre-dose
Autres noms:
  • MMV390048
Expérimental: 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
Autres noms:
  • Placebo
Supplied as "powder in bottle" formulation for reconstitution pre-dose
Autres noms:
  • MMV390048

Que mesure l'étude ?

Principaux critères de jugement

Mesure des résultats
Description de la mesure
Délai
Number of Participants With Adverse Events
Délai: 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
Délai: 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
Délai: 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

Mesures de résultats secondaires

Mesure des résultats
Description de la mesure
Délai
Determine ex Vivo Efficacy (IC50)
Délai: 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

Collaborateurs et enquêteurs

C'est ici que vous trouverez les personnes et les organisations impliquées dans cette étude.

Collaborateurs

Les enquêteurs

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

Publications et liens utiles

La personne responsable de la saisie des informations sur l'étude fournit volontairement ces publications. Il peut s'agir de tout ce qui concerne l'étude.

Dates d'enregistrement des études

Ces dates suivent la progression des dossiers d'étude et des soumissions de résultats sommaires à ClinicalTrials.gov. Les dossiers d'étude et les résultats rapportés sont examinés par la Bibliothèque nationale de médecine (NLM) pour s'assurer qu'ils répondent à des normes de contrôle de qualité spécifiques avant d'être publiés sur le site Web public.

Dates principales de l'étude

Début de l'étude

1 mai 2014

Achèvement primaire (Réel)

1 février 2015

Achèvement de l'étude (Réel)

1 février 2015

Dates d'inscription aux études

Première soumission

29 août 2014

Première soumission répondant aux critères de contrôle qualité

29 août 2014

Première publication (Estimation)

3 septembre 2014

Mises à jour des dossiers d'étude

Dernière mise à jour publiée (Réel)

17 juillet 2019

Dernière mise à jour soumise répondant aux critères de contrôle qualité

16 juillet 2019

Dernière vérification

1 février 2018

Plus d'information

Termes liés à cette étude

Autres numéros d'identification d'étude

  • MMV_MMV390048_14_01

Plan pour les données individuelles des participants (IPD)

Prévoyez-vous de partager les données individuelles des participants (DPI) ?

Non

Informations sur les médicaments et les dispositifs, documents d'étude

Étudie un produit pharmaceutique réglementé par la FDA américaine

Non

Étudie un produit d'appareil réglementé par la FDA américaine

Non

produit fabriqué et exporté des États-Unis.

Non

Ces informations ont été extraites directement du site Web clinicaltrials.gov sans aucune modification. Si vous avez des demandes de modification, de suppression ou de mise à jour des détails de votre étude, veuillez contacter register@clinicaltrials.gov. Dès qu'un changement est mis en œuvre sur clinicaltrials.gov, il sera également mis à jour automatiquement sur notre site Web .

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