- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01853852
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
December 17, 2013 updated by: 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.
Study Overview
Status
Completed
Conditions
Intervention / Treatment
Study Type
Interventional
Enrollment (Actual)
14
Phase
- Phase 1
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
-
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New South Wales
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Randwick, New South Wales, Australia, 2031
- GSK Investigational Site
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-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
20 years to 55 years (Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
Inclusion Criteria:
- 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.
- Male or female between 20 and 55 years of age inclusive, at the time of signing the informed consent.
- A female subject is eligible to participate if she is of: Non-childbearing potential defined as pre-menopausal female.
- 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.
- Body weight >=50 kg and BMI within the range 18.5 - 29.0 kg/m2 (inclusive).
- Capable of giving written informed consent, which includes compliance with the requirements and restrictions listed in the consent form.
- AST, ALT, alkaline phosphatase and bilirubin > 1.5xULN (isolated bilirubin >1.5xULN is acceptable if bilirubin is fractionated and direct bilirubin <35%).
- Single QTcB or QTcF < 450 msec; or QTc < 480 msec in subjects with Bundle Branch Block.
- 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:
- A positive pre-study Hepatitis B surface antigen or positive Hepatitis C antibody result within 3 months of screening.
- A positive pre-study drug/alcohol screen.
- A positive test for HIV antibody.
- History of regular alcohol consumption within 6 months of the study
- 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).
- Exposure to more than four new chemical entities within 12 months prior to the first dosing day.
- 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.
- 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.
- Where participation in the study would result in donation of blood or blood products in excess of 500 mL within a 56 day period.
- History or regular use of tobacco- or nicotine-containing products within 6 months prior to screening.
- Pregnant females as determined by positive serum or urine hCG test at screening or prior to dosing.
- Lactating females.
- Unwillingness or inability to follow the procedures outlined in the protocol.
- Subject is mentally or legally incapacitated.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Allocation: Randomized
- Interventional Model: Crossover Assignment
- Masking: Single
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Placebo Comparator: 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
|
Experimental: 50 mg
GR181413A/AT1001
|
Powder for reconstitution
Matched, Size 2, hard gelatin capsule, white opaque/blue opaque
Solution matched
|
Experimental: 150 mg
GR181413A/AT1001
|
Size 2, hard gelatin capsule, white opaque / blue opaque
|
Experimental: 450 mg
GR181413A/AT1001
|
Size 2, hard gelatin capsule, white opaque / blue opaque
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Profile of plasma pharmacokinetics
Time Frame: 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
Time Frame: up to 24 hr
|
up to 24 hr
|
|
Change from baseline in clinical laboratory tests (hematology, chemistry and urinalysis)
Time Frame: 0, 24h post dose
|
0, 24h post dose
|
|
Change from baseline in vital signs (blood pressure and heart rate)
Time Frame: 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
Time Frame: 0, i, 2, 3, 6, 24h post dose
|
0, i, 2, 3, 6, 24h post dose
|
|
Profile of urine pharmacokinetics
Time Frame: 0-4, 4-10, 10-12, 12-24h post dose
|
Ae, CLr, %Fx
|
0-4, 4-10, 10-12, 12-24h post dose
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start
September 1, 2011
Primary Completion (Actual)
December 1, 2011
Study Completion (Actual)
December 1, 2011
Study Registration Dates
First Submitted
November 10, 2011
First Submitted That Met QC Criteria
May 9, 2013
First Posted (Estimate)
May 15, 2013
Study Record Updates
Last Update Posted (Estimate)
December 18, 2013
Last Update Submitted That Met QC Criteria
December 17, 2013
Last Verified
December 1, 2013
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Cardiovascular Diseases
- Vascular Diseases
- Metabolic Diseases
- Cerebrovascular Disorders
- Brain Diseases
- Central Nervous System Diseases
- Nervous System Diseases
- Genetic Diseases, Inborn
- Genetic Diseases, X-Linked
- Metabolism, Inborn Errors
- Lysosomal Storage Diseases
- Lipid Metabolism Disorders
- Brain Diseases, Metabolic
- Brain Diseases, Metabolic, Inborn
- Sphingolipidoses
- Lysosomal Storage Diseases, Nervous System
- Cerebral Small Vessel Diseases
- Lipidoses
- Lipid Metabolism, Inborn Errors
- Fabry Disease
- Molecular Mechanisms of Pharmacological Action
- Anti-Infective Agents
- Antiviral Agents
- Enzyme Inhibitors
- 1-Deoxynojirimycin
Other Study ID Numbers
- 115806
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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