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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT01196871
Registration number
NCT01196871
Ethics application status
Date submitted
7/09/2010
Date registered
9/09/2010
Date last updated
19/12/2018
Titles & IDs
Public title
Drug-Drug Interaction Study Between AT1001 (Migalastat Hydrochloride) and Agalsidase in Participants With Fabry Disease
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Scientific title
An Open-label Phase 2A Study to Investigate Drug-Drug Interactions Between AT1001 (Migalastat Hydrochloride) and Agalsidase in Subjects With Fabry Disease
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Secondary ID [1]
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AT1001-013
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Fabry Disease
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Condition category
Condition code
Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Metabolic and Endocrine
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Metabolic disorders
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Metabolic and Endocrine
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Other metabolic disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Migalastat HCl
Other interventions - Agalsidase Beta
Other interventions - Agalsidase Alfa
Experimental: Agalsidase Beta (0.5 mg/kg)-Migalastat (150 mg) -
Experimental: Agalsidase Beta (1.0 mg/kg)-Migalastat (150 mg) -
Experimental: Agalsidase Alfa (0.2 mg/kg)-Migalastat (150 mg) -
Experimental: Agalsidase Beta (0.5 mg/kg)-Migalastat (450 mg) -
Experimental: Agalsidase Beta (1.0 mg/kg)-Migalastat (450 mg) -
Experimental: Agalsidase Alfa (0.2 mg/kg)-Migalastat (450 mg) -
Treatment: Drugs: Migalastat HCl
Oral capsules, single dose
Other interventions: Agalsidase Beta
IV infusion, single dose
Other interventions: Agalsidase Alfa
IV infusion, single dose
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Intervention code [1]
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Treatment: Drugs
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Intervention code [2]
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Other interventions
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Change In Area Under The Plasma Concentration Versus Time Curve (AUC) For Active a-Galactosidase A (a-Gal A) Levels After Administration Of Migalastat
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Assessment method [1]
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This measure characterized the effects of migalastat on the plasma PK of agalsidase by measurement of the active a-Gal A enzyme level in plasma using a qualified assay that measured the rate of enzyme activity using an artificial, fluorescent substrate. The agalsidase plasma PK parameter values for AUC extrapolated from time 0 to infinity (AUCinfinity) and AUC to the last time point at which concentration is quantified (AUC0-t) are reported in hr*[nanomoles/hr/milliliter] (hr*[nmol/hr/mL]). In Period 1 of Stages 1 and 2, blood samples were collected: immediately before the agalsidase infusion and over a 24-hr period after infusion; on Days 2, 7, and 14. In Period 2 of Stages 1 and 2, blood samples were collected: prior to dosing with migalastat (2 hr prior to the agalsidase infusion) and 1 hr after migalastat dosing; immediately before initiation of the agalsidase infusion and over a 24-hr period after initiation of the agalsidase infusion; on Days 2, 7, and 14.
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Timepoint [1]
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0 hr, 2 hr, 2 days, 7 days, 14 days post dose
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Primary outcome [2]
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Change In Maximum Observed Plasma Concentration (Cmax) For Active a-Gal A Levels After Administration Of Migalastat
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Assessment method [2]
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This measure characterized the effects of migalastat on the plasma PK of agalsidase by measurement of the active a-Gal A enzyme level in plasma using a qualified assay that measured the rate of enzyme activity using an artificial, fluorescent substrate. The agalsidase plasma PK parameter value for Cmax is reported in nmol/hr/mL. In Period 1 of Stages 1 and 2, blood samples were collected: immediately before the agalsidase infusion and over a 24-hr period after infusion; on Days 2, 7, and 14. In Period 2 of Stages 1 and 2, blood samples were collected: prior to dosing with migalastat (2 hr prior to the agalsidase infusion) and 1 hr after migalastat dosing; immediately before initiation of the agalsidase infusion and over a 24-hr period after initiation of the agalsidase infusion; on Days 2, 7, and 14.
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Timepoint [2]
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0 hr, 2 hr, 2 days, 7 days, 14 days post dose
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Primary outcome [3]
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Change In Time To Maximum Observed Plasma Concentration (Tmax) And Terminal Elimination Half-life (T1/2) For Active a-Gal A Levels After Administration Of Migalastat
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Assessment method [3]
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This measure characterized the effects of migalastat on the plasma PK of agalsidase by measurement of the active a-Gal A enzyme level in plasma using a qualified assay that measured the rate of enzyme activity using an artificial, fluorescent substrate. The agalsidase plasma PK parameter values for tmax and t1/2 are reported in hr. In Period 1 of Stages 1 and 2, blood samples were collected: immediately before the agalsidase infusion and over a 24-hr period after infusion; on Days 2, 7, and 14. In Period 2 of Stages 1 and 2, blood samples were collected: prior to dosing with migalastat (2 hr prior to the agalsidase infusion) and 1 hr after migalastat dosing; immediately before initiation of the agalsidase infusion and over a 24-hr period after initiation of the agalsidase infusion; on Days 2, 7, and 14.
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Timepoint [3]
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0 hr, 2 hr, 2 days, 7 days, 14 days post dose
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Primary outcome [4]
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Change In AUC For Total a-Gal A Protein Levels After Administration Of Migalastat
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Assessment method [4]
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This measure characterized the effects of migalastat on the plasma PK of agalsidase by measurement of the a-Gal A protein level in plasma by Western blot using anti-human Gal A antibody. The agalsidase plasma PK parameter value for AUC0-t is reported in hr*[nanogram (ng)/hr/mL]. In Period 1 of Stages 1 and 2, blood samples were collected: immediately before the agalsidase infusion and over a 24-hr period after infusion; on Days 2, 7, and 14. In Period 2 of Stages 1 and 2, blood samples were collected: prior to dosing with migalastat (2 hr prior to the agalsidase infusion) and 1 hr after migalastat dosing; immediately before initiation of the agalsidase infusion and over a 24-hr period after initiation of the agalsidase infusion; on Days 2, 7, and 14.
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Timepoint [4]
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0 hr, 2 hr, 2 days, 7 days, 14 days post dose
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Primary outcome [5]
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Change In Percentage Of AUCinfinity Extrapolated From The Last Time Point At Which Concentration Is Quantified To Infinity (AUCextrapolated %) For Total a-Gal A Protein Levels After Administration Of Migalastat
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Assessment method [5]
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This measure characterized the effects of migalastat on the plasma PK of agalsidase by measurement of the a-Gal A protein level by Western blot using anti-human Gal A antibody. The agalsidase plasma PK parameter values for AUCextrapolated % are reported. AUCextrapolated % is reported instead of AUCinfinity because small but quantifiable concentrations of a-Gal A protein past 24 hr post-dose extrapolated to infinity comprised >50% of total AUC in most participants and were unevaluable. In Period 1 of Stages 1 and 2, blood samples were collected: immediately before the agalsidase infusion and over a 24-hr period after infusion; on Days 2, 7, and 14. In Period 2 of Stages 1 and 2, blood samples were collected: prior to dosing with migalastat (2 hr prior to the agalsidase infusion) and 1 hour after migalastat dosing; immediately before initiation of the agalsidase infusion and over a 24-hr period after initiation of the agalsidase infusion; on Days 2, 7, and 14.
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Timepoint [5]
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0 hr, 2 hr, 2 days, 7 days, 14 days post dose
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Primary outcome [6]
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Change In Cmax For Total a-Gal A Protein Levels After Administration Of Migalastat
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Assessment method [6]
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This measure characterized the effects of migalastat on the plasma PK of agalsidase by measurement of the a-Gal A protein level in plasma by Western blot using anti-human Gal A antibody. The agalsidase plasma PK parameter values for Cmax is reported in nmol/hr/mL. In Period 1 of Stages 1 and 2, blood samples were collected: immediately before the agalsidase infusion and over a 24-hr period after infusion; on Days 2, 7, and 14. In Period 2 of Stages 1 and 2, blood samples were collected: prior to dosing with migalastat (2 hr prior to the agalsidase infusion) and 1 hr after migalastat dosing; immediately before initiation of the agalsidase infusion and over a 24-hr period after initiation of the agalsidase infusion; on Days 2, 7, and 14.
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Timepoint [6]
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0 hr, 2 hr, 2 days, 7 days, 14 days post dose
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Primary outcome [7]
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Change In Tmax And T1/2 For Total a-Gal A Protein Levels After Administration Of Migalastat
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Assessment method [7]
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This measure characterized the effects of migalastat on the plasma PK of agalsidase by measurement of the total a-Gal A protein level in plasma by Western blot using anti-human Gal A antibody. The agalsidase plasma PK parameter values for tmax and t1/2 are reported in hr. In Period 1 of Stages 1 and 2, blood samples were collected: immediately before the agalsidase infusion and over a 24-hr period after infusion; on Days 2, 7, and 14. In Period 2 of Stages 1 and 2, blood samples were collected: prior to dosing with migalastat (2 hr prior to the agalsidase infusion) and 1 hr after migalastat dosing; immediately before initiation of the agalsidase infusion and over a 24-hr period after initiation of the agalsidase infusion; on Days 2, 7, and 14.
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Timepoint [7]
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0 hr, 2 hr, 2 days, 7 days, 14 days post dose
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Primary outcome [8]
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Change In AUC For Migalastat After Administration Of Agalsidase
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Assessment method [8]
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This measure characterized the effects of agalsidase on the plasma PK of migalastat using a validated liquid chromatography-tandem mass spectrometry (LC-MS) assay. The migalastat plasma PK parameter values for AUCinfinity and AUC0-t are reported in hr*[ng/hr/mL]. In Period 2 of Stages 1 and 2, blood samples were collected: just before dosing with migalastat (2 hr prior to the agalsidase infusion) and at 1 hr after migalastat dosing; immediately before the agalsidase infusion and over a 24-hr period after infusion. In Period 3 (Stage 1 only), blood samples were collected before dosing and over the 24-hr period after administration of migalastat.
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Timepoint [8]
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0 hr, 1 day post dose
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Primary outcome [9]
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Change In Cmax For Migalastat After Administration Of Agalsidase
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Assessment method [9]
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This measure characterized the effects of agalsidase on the plasma PK of migalastat using a validated liquid LC-MS assay. The migalastat plasma PK parameter values for Cmax are reported in nmol/hr/mL. In Period 2 of Stages 1 and 2, blood samples were collected: just before dosing with migalastat (2 hr prior to the agalsidase infusion) and at 1 hr after migalastat dosing; immediately before the agalsidase infusion and over a 24-hr period after infusion. In Period 3 (Stage 1 only), blood samples were collected before dosing and over the 24-hr period after administration of migalastat.
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Timepoint [9]
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0 hr, 1 day post dose
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Primary outcome [10]
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Change In Tmax And T1/2 For Migalastat After Administration Of Agalsidase
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Assessment method [10]
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This measure characterized the effects of agalsidase on the plasma PK of migalastat using a validated LC-MS assay. The migalastat plasma PK parameter values for tmax and t1/2 are reported in hr. In Period 2 of Stages 1 and 2, blood samples were collected: just before dosing with migalastat (2 hr prior to the agalsidase infusion) and at 1 hr after migalastat dosing; immediately before the agalsidase infusion and over a 24-hr period after infusion. In Period 3 (Stage 1 only), blood samples were collected before dosing and over the 24-hr period after administration of migalastat.
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Timepoint [10]
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0 hr, 1 day post dose
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Secondary outcome [1]
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Change From Baseline To Day 7 In Active a-Gal A In Skin Following Treatment With Agalsidase Alone And Co-administration With Migalastat
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Assessment method [1]
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This measure characterized the effects of agalsidase and migalastat on a-Gal A activity in the skin using a qualified assay that measured the rate of enzyme activity using an artificial, fluorescent substrate. Baseline was defined as Day 1/Period 1 pre-infusion level. a-Gal A activity is reported in picomoles/mg/hr (pmol/mg/hr). Biopsy samples were obtained: on Day -1/Period 1; 24 hr after initiation of the infusion during Period 1 and Period 2; on Day 7 of Period 1 and Period 2.
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Timepoint [1]
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Baseline, Day 7
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Eligibility
Key inclusion criteria
- Male diagnosed with Fabry disease and between 18 and 65 years of age, inclusive
- Body mass index between 18-35 kg per meter squared
- Had initiated treatment with agalsidase at least 1 month prior to screening, and had
received at least 2 infusions before screening
- Had stable dose level, dosing regimen, and form of agalsidase for at least 1 month
before screening
- Had an estimated creatinine clearance greater than or equal to 50 milliliters
(mL)/minute at screening
- Agreed to use medically accepted methods of contraception during the study and for 30
days after study completion
- Were willing and able to provide written informed consent
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Minimum age
18
Years
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Maximum age
65
Years
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Sex
Males
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Can healthy volunteers participate?
No
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Key exclusion criteria
- Had a documented transient ischemic attack, ischemic stroke, unstable angina, or
myocardial infarction within the 3 months before screening
- Had clinically significant unstable cardiac disease (for example, cardiac disease
requiring active management, such as symptomatic arrhythmia, unstable angina, or New
York Heart Association class III or IV congestive heart failure)
- History of allergy or sensitivity to study drug (including excipients) or other
iminosugars (such as miglustat, miglitol)
- Required a concomitant medication prohibited by the protocol: Glyset® (miglitol), or
Zavesca® (miglustat)
- Any investigational/experimental drug or device within 30 days of screening, except
for use of investigational enzyme replacement therapy for Fabry disease
- Had any intercurrent illness or condition that might have precluded the participant
from fulfilling the protocol requirements or suggested to the investigator that the
potential participant might have had an unacceptable risk by participating in this
study
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Non-randomised trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Other
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Other design features
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Phase
Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
2/02/2011
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
9/10/2012
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Sample size
Target
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Accrual to date
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Final
20
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Recruitment in Australia
Recruitment state(s)
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Recruitment hospital [1]
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- Nedlands
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Recruitment hospital [2]
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- Parkville
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Recruitment postcode(s) [1]
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- Nedlands
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Recruitment postcode(s) [2]
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- Parkville
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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Alabama
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Country [2]
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United States of America
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State/province [2]
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Georgia
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Country [3]
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United States of America
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State/province [3]
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Iowa
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Country [4]
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United States of America
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State/province [4]
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Kansas
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Country [5]
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United States of America
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State/province [5]
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Virginia
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Country [6]
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Belgium
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State/province [6]
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Edegem
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Country [7]
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Canada
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State/province [7]
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Montreal
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Country [8]
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Netherlands
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State/province [8]
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Amsterdam
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Amicus Therapeutics
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
The objective was to determine the effects of a single dose of migalastat hydrochloride (HCl)
(migalastat) 150 and 450 milligrams (mg) on the safety and plasma pharmacokinetics (PK) of
agalsidase and the effects of agalsidase on the safety and PK of migalastat 150 mg.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT01196871
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Medical Monitor Clinical Research
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Address
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Amicus Therapeutics
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Address
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Country
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Phone
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Fax
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Email
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT01196871
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