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A Phase I, Randomized, Single-Blind, Four-Period Cross-Over, Placebo-Controlled, Dose-Escalation Study to Evaluate the Safety and Pharmacokinetics of Single Oral Doses of GR181413A/AT1001 in Healthy Japanese Subjects

17 december 2013 uppdaterad av: Amicus Therapeutics
GR181413A/AT1001 (migalastat hydrochloride) is a low molecular weight iminosugar, an analog of the terminal galactose group that is cleaved from the substrate GL-3. This compound was researched and developed as a drug for treatment of Fabry disease. This study, MGM115806, will be the first administration of GR181413A/AT1001 to Japanese subjects to investigate the safety, tolerability and pharmacokinetics of single oral doses in healthy Japanese adult subjects. Approximately 12 subjects will receive three treatments of 50, 150 and 450 mg GR181413A/AT1001 under fasted conditions plus placebo in a dose ascending crossover design. Serial pharmacokinetic samples will be collected and safety assessments will be performed following each dose. The pharmacokinetics and dose proportionality of GR181413A/AT1001 after single oral doses of GR181413A/AT1001 at the dose levels of 50, 150 and 450 mg under fasted conditions will be assessed.

Studieöversikt

Studietyp

Interventionell

Inskrivning (Faktisk)

14

Fas

  • Fas 1

Kontakter och platser

Det här avsnittet innehåller kontaktuppgifter för dem som genomför studien och information om var denna studie genomförs.

Studieorter

    • New South Wales
      • Randwick, New South Wales, Australien, 2031
        • GSK Investigational Site

Deltagandekriterier

Forskare letar efter personer som passar en viss beskrivning, så kallade behörighetskriterier. Några exempel på dessa kriterier är en persons allmänna hälsotillstånd eller tidigare behandlingar.

Urvalskriterier

Åldrar som är berättigade till studier

20 år till 55 år (Vuxen)

Tar emot friska volontärer

Nej

Kön som är behöriga för studier

Allt

Beskrivning

Inclusion Criteria:

  1. Healthy as determined by a responsible and experienced physician, based on a medical evaluation including medical history, physical examination, laboratory tests and cardiac monitoring. A subject with a clinical abnormality or laboratory parameters outside the reference range for the population being studied may be included only if the Investigator and the GSK Medical Monitor agree that the finding is unlikely to introduce additional risk factors and will not interfere with the study procedures.
  2. Male or female between 20 and 55 years of age inclusive, at the time of signing the informed consent.
  3. A female subject is eligible to participate if she is of: Non-childbearing potential defined as pre-menopausal female.
  4. Male subjects with female partners of child-bearing potential must agree to use one of the contraception methods. This criterion must be followed from the time of the first dose of study medication until 5 terminal half-lives post-last dose.
  5. Body weight >=50 kg and BMI within the range 18.5 - 29.0 kg/m2 (inclusive).
  6. Capable of giving written informed consent, which includes compliance with the requirements and restrictions listed in the consent form.
  7. AST, ALT, alkaline phosphatase and bilirubin > 1.5xULN (isolated bilirubin >1.5xULN is acceptable if bilirubin is fractionated and direct bilirubin <35%).
  8. Single QTcB or QTcF < 450 msec; or QTc < 480 msec in subjects with Bundle Branch Block.
  9. Japanese defined being born in Japan, having four ethnic Japanese grandparents, holding a Japanese passport or identity papers and being able to speak Japanese. Japanese subjects should be also have lived outside Japan for less than 10 years.

Exclusion Criteria:

  1. A positive pre-study Hepatitis B surface antigen or positive Hepatitis C antibody result within 3 months of screening.
  2. A positive pre-study drug/alcohol screen.
  3. A positive test for HIV antibody.
  4. History of regular alcohol consumption within 6 months of the study
  5. The subject has participated in a clinical trial and has received an investigational product within the following time period prior to the first dosing day in the current study: 30 days, 5 half-lives or twice the duration of the biological effect of the investigational product (whichever is longest).
  6. Exposure to more than four new chemical entities within 12 months prior to the first dosing day.
  7. Use of prescription or non-prescription drugs, including vitamins, herbal and dietary supplements (including St Johns Wort) within 7 days (or 14 days if the drug is a potential enzyme inducer) or 5 half-lives (whichever is longer) prior to the first dose of study medication, unless in the opinion of the Investigator and GSK Medical Monitor the medication will not interfere with the study procedures or compromise subject safety.
  8. History of sensitivity to any of the study medications, or components thereof or a history of drug or other allergy that, in the opinion of the investigator or GSK Medical Monitor, contraindicates their participation.
  9. Where participation in the study would result in donation of blood or blood products in excess of 500 mL within a 56 day period.
  10. History or regular use of tobacco- or nicotine-containing products within 6 months prior to screening.
  11. Pregnant females as determined by positive serum or urine hCG test at screening or prior to dosing.
  12. Lactating females.
  13. Unwillingness or inability to follow the procedures outlined in the protocol.
  14. Subject is mentally or legally incapacitated.

Studieplan

Det här avsnittet ger detaljer om studieplanen, inklusive hur studien är utformad och vad studien mäter.

Hur är studien utformad?

Designdetaljer

  • Tilldelning: Randomiserad
  • Interventionsmodell: Crossover tilldelning
  • Maskning: Enda

Vapen och interventioner

Deltagargrupp / Arm
Intervention / Behandling
Placebo-jämförare: Placebo
placebo
Powder for reconstitution
Matched, Size 2, hard gelatin capsule, white opaque/blue opaque
Solution matched
Size 2, hard gelatin capsule, white opaque / blue opaque
Experimentell: 50 mg
GR181413A/AT1001
Powder for reconstitution
Matched, Size 2, hard gelatin capsule, white opaque/blue opaque
Solution matched
Experimentell: 150 mg
GR181413A/AT1001
Size 2, hard gelatin capsule, white opaque / blue opaque
Experimentell: 450 mg
GR181413A/AT1001
Size 2, hard gelatin capsule, white opaque / blue opaque

Vad mäter studien?

Primära resultatmått

Resultatmått
Åtgärdsbeskrivning
Tidsram
Profile of plasma pharmacokinetics
Tidsram: 0, 0.5, 1, 1.5, 2, 3, 3.5, 4, 5, 6, 8, 10, 12h post dose
AUC, Cmax, tmax, Tlast , t1/2, CL/F, Vz/F
0, 0.5, 1, 1.5, 2, 3, 3.5, 4, 5, 6, 8, 10, 12h post dose
Number of Participants with adverse events
Tidsram: up to 24 hr
up to 24 hr
Change from baseline in clinical laboratory tests (hematology, chemistry and urinalysis)
Tidsram: 0, 24h post dose
0, 24h post dose
Change from baseline in vital signs (blood pressure and heart rate)
Tidsram: 0 ,1 , 2, 3, 4, 5, 6, 24h post dose
0 ,1 , 2, 3, 4, 5, 6, 24h post dose
Change from baseline in 12-lead ECG
Tidsram: 0, i, 2, 3, 6, 24h post dose
0, i, 2, 3, 6, 24h post dose
Profile of urine pharmacokinetics
Tidsram: 0-4, 4-10, 10-12, 12-24h post dose
Ae, CLr, %Fx
0-4, 4-10, 10-12, 12-24h post dose

Samarbetspartners och utredare

Det är här du hittar personer och organisationer som är involverade i denna studie.

Publikationer och användbara länkar

Den som ansvarar för att lägga in information om studien tillhandahåller frivilligt dessa publikationer. Dessa kan handla om allt som har med studien att göra.

Studieavstämningsdatum

Dessa datum spårar framstegen för inlämningar av studieposter och sammanfattande resultat till ClinicalTrials.gov. Studieposter och rapporterade resultat granskas av National Library of Medicine (NLM) för att säkerställa att de uppfyller specifika kvalitetskontrollstandarder innan de publiceras på den offentliga webbplatsen.

Studera stora datum

Studiestart

1 september 2011

Primärt slutförande (Faktisk)

1 december 2011

Avslutad studie (Faktisk)

1 december 2011

Studieregistreringsdatum

Först inskickad

10 november 2011

Först inskickad som uppfyllde QC-kriterierna

9 maj 2013

Första postat (Uppskatta)

15 maj 2013

Uppdateringar av studier

Senaste uppdatering publicerad (Uppskatta)

18 december 2013

Senaste inskickade uppdateringen som uppfyllde QC-kriterierna

17 december 2013

Senast verifierad

1 december 2013

Mer information

Denna information hämtades direkt från webbplatsen clinicaltrials.gov utan några ändringar. Om du har några önskemål om att ändra, ta bort eller uppdatera dina studieuppgifter, vänligen kontakta register@clinicaltrials.gov. Så snart en ändring har implementerats på clinicaltrials.gov, kommer denna att uppdateras automatiskt även på vår webbplats .

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