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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 décembre 2013 mis à jour par: 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.

Aperçu de l'étude

Type d'étude

Interventionnel

Inscription (Réel)

14

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

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

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

20 ans à 55 ans (Adulte)

Accepte les volontaires sains

Non

Sexes éligibles pour l'étude

Tout

La description

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.

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

  • Répartition: Randomisé
  • Modèle interventionnel: Affectation croisée
  • Masquage: Seul

Armes et Interventions

Groupe de participants / Bras
Intervention / Traitement
Comparateur placebo: 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
Expérimental: 50 mg
GR181413A/AT1001
Powder for reconstitution
Matched, Size 2, hard gelatin capsule, white opaque/blue opaque
Solution matched
Expérimental: 150 mg
GR181413A/AT1001
Size 2, hard gelatin capsule, white opaque / blue opaque
Expérimental: 450 mg
GR181413A/AT1001
Size 2, hard gelatin capsule, white opaque / blue opaque

Que mesure l'étude ?

Principaux critères de jugement

Mesure des résultats
Description de la mesure
Délai
Profile of plasma pharmacokinetics
Délai: 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
Délai: up to 24 hr
up to 24 hr
Change from baseline in clinical laboratory tests (hematology, chemistry and urinalysis)
Délai: 0, 24h post dose
0, 24h post dose
Change from baseline in vital signs (blood pressure and heart rate)
Délai: 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
Délai: 0, i, 2, 3, 6, 24h post dose
0, i, 2, 3, 6, 24h post dose
Profile of urine pharmacokinetics
Délai: 0-4, 4-10, 10-12, 12-24h post dose
Ae, CLr, %Fx
0-4, 4-10, 10-12, 12-24h post dose

Collaborateurs et enquêteurs

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

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 septembre 2011

Achèvement primaire (Réel)

1 décembre 2011

Achèvement de l'étude (Réel)

1 décembre 2011

Dates d'inscription aux études

Première soumission

10 novembre 2011

Première soumission répondant aux critères de contrôle qualité

9 mai 2013

Première publication (Estimation)

15 mai 2013

Mises à jour des dossiers d'étude

Dernière mise à jour publiée (Estimation)

18 décembre 2013

Dernière mise à jour soumise répondant aux critères de contrôle qualité

17 décembre 2013

Dernière vérification

1 décembre 2013

Plus d'information

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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