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

16. července 2019 aktualizováno: 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.

Přehled studie

Detailní popis

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.

Typ studie

Intervenční

Zápis (Aktuální)

48

Fáze

  • Fáze 1

Kontakty a umístění

Tato část poskytuje kontaktní údaje pro ty, kteří studii provádějí, a informace o tom, kde se tato studie provádí.

Studijní místa

      • Cape Town, Jižní Afrika
        • Cinical Pharmacology, University of Cape Town

Kritéria účasti

Výzkumníci hledají lidi, kteří odpovídají určitému popisu, kterému se říká kritéria způsobilosti. Některé příklady těchto kritérií jsou celkový zdravotní stav osoby nebo předchozí léčba.

Kritéria způsobilosti

Věk způsobilý ke studiu

18 let až 55 let (Dospělý)

Přijímá zdravé dobrovolníky

Ne

Pohlaví způsobilá ke studiu

Všechno

Popis

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

Studijní plán

Tato část poskytuje podrobnosti o studijním plánu, včetně toho, jak je studie navržena a co studie měří.

Jak je studie koncipována?

Detaily designu

  • Primární účel: Léčba
  • Přidělení: Randomizované
  • Intervenční model: Přiřazení jedné skupiny
  • Maskování: Čtyřnásobek

Zbraně a zásahy

Skupina účastníků / Arm
Intervence / Léčba
Experimentální: 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
Ostatní jména:
  • MMV390048
Supplied as "powder in bottle" formulation for reconstitution pre-dose
Ostatní jména:
  • Placebo
Experimentální: 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
Ostatní jména:
  • Placebo
Supplied as "powder in bottle" formulation for reconstitution pre-dose.
Ostatní jména:
  • MMV390048
Experimentální: 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
Ostatní jména:
  • Placebo
Supplied as "powder in bottle" formulation for reconstitution pre-dose
Ostatní jména:
  • MMV390048
Experimentální: 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
Ostatní jména:
  • Placebo
Supplied as "powder in bottle" formulation for reconstitution pre-dose
Ostatní jména:
  • MMV390048
Experimentální: 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
Ostatní jména:
  • Placebo
Supplied as "powder in bottle" formulation for reconstitution pre-dose
Ostatní jména:
  • MMV390048
Experimentální: 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
Ostatní jména:
  • Placebo
Supplied as "powder in bottle" formulation for reconstitution pre-dose
Ostatní jména:
  • MMV390048

Co je měření studie?

Primární výstupní opatření

Měření výsledku
Popis opatření
Časové okno
Number of Participants With Adverse Events
Časové okno: 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
Časové okno: 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
Časové okno: 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

Sekundární výstupní opatření

Měření výsledku
Popis opatření
Časové okno
Determine ex Vivo Efficacy (IC50)
Časové okno: 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

Spolupracovníci a vyšetřovatelé

Zde najdete lidi a organizace zapojené do této studie.

Spolupracovníci

Vyšetřovatelé

  • Vrchní vyšetřovatel: Karen Barnes, Prof, University of Cape Town

Publikace a užitečné odkazy

Osoba odpovědná za zadávání informací o studiu tyto publikace poskytuje dobrovolně. Mohou se týkat čehokoli, co souvisí se studiem.

Termíny studijních záznamů

Tato data sledují průběh záznamů studie a předkládání souhrnných výsledků na ClinicalTrials.gov. Záznamy ze studií a hlášené výsledky jsou před zveřejněním na veřejné webové stránce přezkoumány Národní lékařskou knihovnou (NLM), aby se ujistily, že splňují specifické standardy kontroly kvality.

Hlavní termíny studia

Začátek studia

1. května 2014

Primární dokončení (Aktuální)

1. února 2015

Dokončení studie (Aktuální)

1. února 2015

Termíny zápisu do studia

První předloženo

29. srpna 2014

První předloženo, které splnilo kritéria kontroly kvality

29. srpna 2014

První zveřejněno (Odhad)

3. září 2014

Aktualizace studijních záznamů

Poslední zveřejněná aktualizace (Aktuální)

17. července 2019

Odeslaná poslední aktualizace, která splnila kritéria kontroly kvality

16. července 2019

Naposledy ověřeno

1. února 2018

Více informací

Termíny související s touto studií

Další identifikační čísla studie

  • MMV_MMV390048_14_01

Plán pro data jednotlivých účastníků (IPD)

Plánujete sdílet data jednotlivých účastníků (IPD)?

Ne

Informace o lécích a zařízeních, studijní dokumenty

Studuje lékový produkt regulovaný americkým FDA

Ne

Studuje produkt zařízení regulovaný americkým úřadem FDA

Ne

produkt vyrobený a vyvážený z USA

Ne

Tyto informace byly beze změn načteny přímo z webu clinicaltrials.gov. Máte-li jakékoli požadavky na změnu, odstranění nebo aktualizaci podrobností studie, kontaktujte prosím register@clinicaltrials.gov. Jakmile bude změna implementována na clinicaltrials.gov, bude automaticky aktualizována i na našem webu .

Klinické studie na MMV390048 5mg

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