Fabry DiseasePhase 2NCT01196871

Drug-Drug Interaction Study Between AT1001 (Migalastat Hydrochloride) and Agalsidase in Participants With Fabry Disease

A Phase 2 study testing Migalastat HCl, Agalsidase Beta and Agalsidase Alfa in people with Fabry Disease, run by Amicus Therapeutics at 10 sites in 5 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

Search all trials

Trial 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. Read more about this study

Who is it for?: Ages 18 to 65 years, men only
Conditions the study is for: Fabry Disease
What is being tested?: Migalastat HCl, Agalsidase Beta and Agalsidase Alfa
Type of treatment: Pill or oral medicine, Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 20 participants
Where: 10 sitesin 5 countries
Study dates: 2 Feb 2011to 9 Oct 2012
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 to 65 years
Men only
No healthy volunteers
Adult, Older adult

Who may be able to join

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

Who cannot join

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

Trial locations and contacts

10 sites in 5 countries.

Country

City, postcode or hospital

10 sites in 5 countries

United States5 sites

Study site
Birmingham, Alabama, 35294
Study site
Decatur, Georgia, 30033
Study site
Iowa City, Iowa, 52242
Study site
Kansas City, Kansas, 66160
Study site
Springfield, Virginia, 22152

Other trials in United States

Australia2 sites

Study site
Nedlands
Study site
Parkville

Other trials in Australia

Belgium1 site

Study site
Edegem

Other trials in Belgium

Canada1 site

Study site
Montreal

Other trials in Canada

Netherlands1 site

Study site
Amsterdam

Other trials in Netherlands

About this study

Official title: An Open-label Phase 2A Study to Investigate Drug-Drug Interactions Between AT1001 (Migalastat Hydrochloride) and Agalsidase in Subjects With Fabry Disease

Read the detailed description

This open-label study was conducted in 2 stages (Stage 1, Stage 2). Stage 1 included migalastat 150 mg; Stage 2 included migalastat 450 mg. Each dose of migalastat was selected to evaluate interaction with each of 3 doses of recombinant agalsidase: 0.5 mg/kilogram (kg) agalsidase beta; 1.0 mg/kg agalsidase beta; 0.2 mg/kg agalsidase alfa.

Migalastat was administered orally. Agalsidase alfa was administered as a 40-minute intravenous (IV) infusion and agalsidase beta was administered as a 2-hour (hr) IV infusion.

Stage 1 consisted of 3 treatment periods with 14 days intervening between each period.

Period 1, Day 1: agalsidase was administered alone.

Period 2, Day 1: migalastat was administered, followed 2 hrs later by agalsidase.

Period 3, Day 7: migalastat was administered alone.

Stage 2 consisted of two 14-day treatment periods in which the plasma exposure of migalastat was characterized when migalastat was administered with agalsidase solely to confirm the attainment of adequate migalastat plasma concentrations.

Period 1, Day 1: agalsidase was administered as an IV infusion using a calibrated infusion pump.

Period 2, Day 1: migalastat was administered, followed 2 hrs later by agalsidase.

Conditions: Fabry Disease
Being tested: Migalastat HCl, Agalsidase Beta, Agalsidase Alfa
Keywords: Amicus Therapeutics, AT1001, Galafold, Migalastat, Pharmacokinetics
Medical subject headings: Fabry Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Migalastat HClDrug
Also called AT1001, Galafold, migalastat
Oral capsules, single dose
Agalsidase BetaBiological
Also called Fabrazyme
IV infusion, single dose
Agalsidase AlfaBiological
Also called Replagal
IV infusion, single dose

Study groups in NCT01196871
Study group	Role	What they receive
Agalsidase Beta (0.5 mg/kg)-Migalastat (150 mg)	Experimental	Not described Receives: Drug: Migalastat HCl; Biological: Agalsidase Beta
Agalsidase Beta (1.0 mg/kg)-Migalastat (150 mg)	Experimental	Not described Receives: Drug: Migalastat HCl; Biological: Agalsidase Beta
Agalsidase Alfa (0.2 mg/kg)-Migalastat (150 mg)	Experimental	Not described Receives: Drug: Migalastat HCl; Biological: Agalsidase Alfa
Agalsidase Beta (0.5 mg/kg)-Migalastat (450 mg)	Experimental	Not described Receives: Drug: Migalastat HCl; Biological: Agalsidase Beta
Agalsidase Beta (1.0 mg/kg)-Migalastat (450 mg)	Experimental	Not described Receives: Drug: Migalastat HCl; Biological: Agalsidase Beta
Agalsidase Alfa (0.2 mg/kg)-Migalastat (450 mg)	Experimental	Not described Receives: Drug: Migalastat HCl; Biological: Agalsidase Alfa

What is being measured

The primary outcome is the main question the study is designed to answer.

Change In Area Under The Plasma Concentration Versus Time Curve (AUC) For Active α-Galactosidase A (α-Gal A) Levels After Administration Of Migalastat
This measure characterized the effects of migalastat on the plasma PK of agalsidase by measurement of the active α-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.
Measured: 0 hr, 2 hr, 2 days, 7 days, 14 days post dose
Change In Maximum Observed Plasma Concentration (Cmax) For Active α-Gal A Levels After Administration Of Migalastat
This measure characterized the effects of migalastat on the plasma PK of agalsidase by measurement of the active α-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.
Measured: 0 hr, 2 hr, 2 days, 7 days, 14 days post dose
Change In Time To Maximum Observed Plasma Concentration (Tmax) And Terminal Elimination Half-life (T1/2) For Active α-Gal A Levels After Administration Of Migalastat
This measure characterized the effects of migalastat on the plasma PK of agalsidase by measurement of the active α-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.
Measured: 0 hr, 2 hr, 2 days, 7 days, 14 days post dose
Change In AUC For Total α-Gal A Protein Levels After Administration Of Migalastat
This measure characterized the effects of migalastat on the plasma PK of agalsidase by measurement of the α-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.
Measured: 0 hr, 2 hr, 2 days, 7 days, 14 days post dose
Change In Percentage Of AUCinfinity Extrapolated From The Last Time Point At Which Concentration Is Quantified To Infinity (AUCextrapolated %) For Total α-Gal A Protein Levels After Administration Of Migalastat
This measure characterized the effects of migalastat on the plasma PK of agalsidase by measurement of the α-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 α-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.
Measured: 0 hr, 2 hr, 2 days, 7 days, 14 days post dose
Change In Cmax For Total α-Gal A Protein Levels After Administration Of Migalastat
This measure characterized the effects of migalastat on the plasma PK of agalsidase by measurement of the α-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.
Measured: 0 hr, 2 hr, 2 days, 7 days, 14 days post dose
Change In Tmax And T1/2 For Total α-Gal A Protein Levels After Administration Of Migalastat
This measure characterized the effects of migalastat on the plasma PK of agalsidase by measurement of the total α-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.
Measured: 0 hr, 2 hr, 2 days, 7 days, 14 days post dose
Change In AUC For Migalastat After Administration Of Agalsidase
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.
Measured: 0 hr, 1 day post dose
Change In Cmax For Migalastat After Administration Of Agalsidase
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.
Measured: 0 hr, 1 day post dose
Change In Tmax And T1/2 For Migalastat After Administration Of Agalsidase
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.
Measured: 0 hr, 1 day post dose

Secondary outcomes (1)

Change From Baseline To Day 7 In Active α-Gal A In Skin Following Treatment With Agalsidase Alone And Co-administration With Migalastat
This measure characterized the effects of agalsidase and migalastat on α-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. α-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.
Measured: Baseline, Day 7

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

2 Feb 20119 Oct 2012

First posted9 Sep 2010 (estimated)
Study start2 Feb 2011
Primary completion9 Oct 2012
Study completion9 Oct 2012

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (1)Papers the sponsor has linked to this study.
Other trials for Fabry DiseaseStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Sequential
Model description: A 2-stage design was used to study the effects of 2 dose levels of migalastat, while a 2- or 3-period design within each stage enabled study of the effects of one drug on the PK, pharmacodynamics, and safety of the other drug.
Masking: None (open label)
Enrolment: 20 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01196871
Sponsor's protocol ID: AT1001-013
Status last verified: Nov 2018
First submitted: 7 Sep 2010
Last updated: 19 Dec 2018
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes

Publications

Warnock DG, Bichet DG, Holida M, Goker-Alpan O, Nicholls K, Thomas M, Eyskens F, Shankar S, Adera M, Sitaraman S, Khanna R, Flanagan JJ, Wustman BA, Barth J, Barlow C, Valenzano KJ, Lockhart DJ, Boudes P, Johnson FK. Oral Migalastat HCl Leads to Greater Systemic Exposure and Tissue Levels of Active alpha-Galactosidase A in Fabry Patients when Co-Administered with Infused Agalsidase. PLoS One. 2015 Aug 7;10(8):e0134341. doi: 10.1371/journal.pone.0134341. eCollection 2015.
PubMed 26252393

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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All Fabry Disease trials

Study at a glance

From the registry record, last updated by the sponsor on 19 Dec 2018. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: An Open-label Phase 2A Study to Investigate Drug-Drug Interactions Between AT1001 (Migalastat Hydrochloride) and Agalsidase in Subjects With Fabry Disease
ClinicalTrials.gov ID: NCT01196871
Acronym / sponsor ID: AT1001-013
Conditions: Fabry Disease
Keywords: Amicus Therapeutics, AT1001, Galafold, Migalastat, Pharmacokinetics
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 20 participants (actual)
Lead sponsor: Amicus TherapeuticsIndustry (pharma, biotech, devices)
Study start: 2 Feb 2011 (actual)
Primary completion: 9 Oct 2012 (actual)
Study completion: 9 Oct 2012 (actual)
First posted: 9 Sep 2010 (estimated)
Results: Posted 1 Nov 2018

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Migalastat was administered orally. Agalsidase alfa was administered as a 40-minute intravenous (IV) infusion and agalsidase beta was administered as a 2-hour (hr) IV infusion.

Stage 1 consisted of 3 treatment periods with 14 days intervening between each period.

Period 1, Day 1: agalsidase was administered alone.

Period 2, Day 1: migalastat was administered, followed 2 hrs later by agalsidase.

Period 3, Day 7: migalastat was administered alone.

Period 1, Day 1: agalsidase was administered as an IV infusion using a calibrated infusion pump.

Period 2, Day 1: migalastat was administered, followed 2 hrs later by agalsidase.

Study design

Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Sequential
Masking: None (open label)
Number of arms: 6
Model description: A 2-stage design was used to study the effects of 2 dose levels of migalastat, while a 2- or 3-period design within each stage enabled study of the effects of one drug on the PK, pharmacodynamics, and safety of the other drug.

Arm	Type	Receives
Agalsidase Beta (0.5 mg/kg)-Migalastat (150 mg)	Experimental	Drug: Migalastat HCl, Biological: Agalsidase Beta
Agalsidase Beta (1.0 mg/kg)-Migalastat (150 mg)	Experimental	Drug: Migalastat HCl, Biological: Agalsidase Beta
Agalsidase Alfa (0.2 mg/kg)-Migalastat (150 mg)	Experimental	Drug: Migalastat HCl, Biological: Agalsidase Alfa
Agalsidase Beta (0.5 mg/kg)-Migalastat (450 mg)	Experimental	Drug: Migalastat HCl, Biological: Agalsidase Beta
Agalsidase Beta (1.0 mg/kg)-Migalastat (450 mg)	Experimental	Drug: Migalastat HCl, Biological: Agalsidase Beta
Agalsidase Alfa (0.2 mg/kg)-Migalastat (450 mg)	Experimental	Drug: Migalastat HCl, Biological: Agalsidase Alfa

Who may qualify

Ages: 18 to 65 years
Sex: Male only
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (7 listed)

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

Key exclusion criteria (6 listed)

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

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
Migalastat HClAlso: AT1001, Galafold, migalastatOral capsules, single dose	Drug	Agalsidase Alfa (0.2 mg/kg)-Migalastat (150 mg), Agalsidase Alfa (0.2 mg/kg)-Migalastat (450 mg), Agalsidase Beta (0.5 mg/kg)-Migalastat (150 mg), Agalsidase Beta (0.5 mg/kg)-Migalastat (450 mg), Agalsidase Beta (1.0 mg/kg)-Migalastat (150 mg), Agalsidase Beta (1.0 mg/kg)-Migalastat (450 mg)
Agalsidase BetaAlso: FabrazymeIV infusion, single dose	Biological	Agalsidase Beta (0.5 mg/kg)-Migalastat (150 mg), Agalsidase Beta (0.5 mg/kg)-Migalastat (450 mg), Agalsidase Beta (1.0 mg/kg)-Migalastat (150 mg), Agalsidase Beta (1.0 mg/kg)-Migalastat (450 mg)
Agalsidase AlfaAlso: ReplagalIV infusion, single dose	Biological	Agalsidase Alfa (0.2 mg/kg)-Migalastat (150 mg), Agalsidase Alfa (0.2 mg/kg)-Migalastat (450 mg)

Primary outcome

Change In Area Under The Plasma Concentration Versus Time Curve (AUC) For Active α-Galactosidase A (α-Gal A) Levels After Administration Of Migalastat
Time frame: 0 hr, 2 hr, 2 days, 7 days, 14 days post dose
This measure characterized the effects of migalastat on the plasma PK of agalsidase by measurement of the active α-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.
Change In Maximum Observed Plasma Concentration (Cmax) For Active α-Gal A Levels After Administration Of Migalastat
Time frame: 0 hr, 2 hr, 2 days, 7 days, 14 days post dose
This measure characterized the effects of migalastat on the plasma PK of agalsidase by measurement of the active α-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.
Change In Time To Maximum Observed Plasma Concentration (Tmax) And Terminal Elimination Half-life (T1/2) For Active α-Gal A Levels After Administration Of Migalastat
Time frame: 0 hr, 2 hr, 2 days, 7 days, 14 days post dose
This measure characterized the effects of migalastat on the plasma PK of agalsidase by measurement of the active α-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.
Change In AUC For Total α-Gal A Protein Levels After Administration Of Migalastat
Time frame: 0 hr, 2 hr, 2 days, 7 days, 14 days post dose
This measure characterized the effects of migalastat on the plasma PK of agalsidase by measurement of the α-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.
Change In Percentage Of AUCinfinity Extrapolated From The Last Time Point At Which Concentration Is Quantified To Infinity (AUCextrapolated %) For Total α-Gal A Protein Levels After Administration Of Migalastat
Time frame: 0 hr, 2 hr, 2 days, 7 days, 14 days post dose
This measure characterized the effects of migalastat on the plasma PK of agalsidase by measurement of the α-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 α-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.
Change In Cmax For Total α-Gal A Protein Levels After Administration Of Migalastat
Time frame: 0 hr, 2 hr, 2 days, 7 days, 14 days post dose
This measure characterized the effects of migalastat on the plasma PK of agalsidase by measurement of the α-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.
Change In Tmax And T1/2 For Total α-Gal A Protein Levels After Administration Of Migalastat
Time frame: 0 hr, 2 hr, 2 days, 7 days, 14 days post dose
This measure characterized the effects of migalastat on the plasma PK of agalsidase by measurement of the total α-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.
Change In AUC For Migalastat After Administration Of Agalsidase
Time frame: 0 hr, 1 day post dose
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.
Change In Cmax For Migalastat After Administration Of Agalsidase
Time frame: 0 hr, 1 day post dose
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.
Change In Tmax And T1/2 For Migalastat After Administration Of Agalsidase
Time frame: 0 hr, 1 day post dose
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.

1 secondary outcome

Change From Baseline To Day 7 In Active α-Gal A In Skin Following Treatment With Agalsidase Alone And Co-administration With Migalastat
Time frame: Baseline, Day 7

Results and safety

Results posted to the registry on 1 Nov 2018, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Agalsidase Beta (0.5 mg/kg)-Migalastat (150 mg)	5	5
Agalsidase Beta (1.0 mg/kg)-Migalastat (150 mg)	3	3
Agalsidase Alfa (0.2 mg/kg)-Migalastat (150 mg)	4	4
Agalsidase Beta (0.5 mg/kg)-Migalastat (450 mg)	0	0
Agalsidase Beta (1.0 mg/kg)-Migalastat (450 mg)	0	0
Agalsidase Alfa (0.2 mg/kg)-Migalastat (450 mg)	0	0

Primary outcome: Change In Area Under The Plasma Concentration Versus Time Curve (AUC) For Active α-Galactosidase A (α-Gal A) Levels After Administration Of Migalastat

Time frame: 0 hr, 2 hr, 2 days, 7 days, 14 days post dose. Mean (Standard Deviation). Unit: hr*[nmol/hr/mL]

Measure	Agalsidase Beta (0.5 mg/kg)-Migalastat (150 mg) n = 5	Agalsidase Beta (1.0 mg/kg)-Migalastat (150 mg) n = 3	Agalsidase Alfa (0.2 mg/kg)-Migalastat (150 mg) n = 4	Agalsidase Beta (0.5 mg/kg)-Migalastat (450 mg) n = 1	Agalsidase Beta (1.0 mg/kg)-Migalastat (450 mg) n = 6	Agalsidase Alfa (0.2 mg/kg)-Migalastat (450 mg) n = 4
Period 1: AUC0-t	1132.72 (223.215)	4730.47 (1045.068)	384.20 (86.904)	2409.02 (NA)	5670.76 (3926.295)	787.23 (509.967)
Period 2: AUC0-t	3255.09 (904.716)	9541.21 (2813.139)	1602.58 (425.798)	6127.75 (NA)	9908.56 (3097.746)	2199.98 (875.140)
Period 1: AUCinfinity	1145.73 (217.894)	4871.66 (1242.965)	388.10 (83.770)	2523.87 (NA)	5851.62 (4345.407)	800.38 (509.308)
Period 2: AUCinfinity	3287.09 (907.073)	9765.04 (3118.875)	1626.67 (407.595)	6197.71 (NA)	10280.79 (3399.342)	2253.95 (859.586)

Agalsidase Beta (0.5 mg/kg)-Migalastat (150 mg) vs Agalsidase Beta (1.0 mg/kg)-Migalastat (150 mg) vs Agalsidase Alfa (0.2 mg/kg)-Migalastat (150 mg): AUC0-t Ratio: 2.942 (90% CI 2.439 to 3.548)
Agalsidase Beta (0.5 mg/kg)-Migalastat (150 mg) vs Agalsidase Beta (1.0 mg/kg)-Migalastat (150 mg) vs Agalsidase Alfa (0.2 mg/kg)-Migalastat (150 mg): AUCinfinity Ratio: 2.942 (90% CI 2.431 to 3.560)
Agalsidase Beta (0.5 mg/kg)-Migalastat (450 mg) vs Agalsidase Beta (1.0 mg/kg)-Migalastat (450 mg) vs Agalsidase Alfa (0.2 mg/kg)-Migalastat (450 mg): AUC0-t Ratio: 2.354 (90% CI 1.826 to 3.035)

Population analysed: PK Population: all participants with evaluable PK parameter data who had successfully completed at least Period 1 and Period 2 in any stage. All PK analyses were performed using the PK population.

Primary outcome: Change In Maximum Observed Plasma Concentration (Cmax) For Active α-Gal A Levels After Administration Of Migalastat

Time frame: 0 hr, 2 hr, 2 days, 7 days, 14 days post dose. Mean (Standard Deviation). Unit: nmol/hr/mL

Measure	Agalsidase Beta (0.5 mg/kg)-Migalastat (150 mg) n = 5	Agalsidase Beta (1.0 mg/kg)-Migalastat (150 mg) n = 3	Agalsidase Alfa (0.2 mg/kg)-Migalastat (150 mg) n = 4	Agalsidase Beta (0.5 mg/kg)-Migalastat (450 mg) n = 1	Agalsidase Beta (1.0 mg/kg)-Migalastat (450 mg) n = 6	Agalsidase Alfa (0.2 mg/kg)-Migalastat (450 mg) n = 4
Period 1:Cmax	514.18 (87.458)	1682.13 (407.862)	309.02 (91.568)	684.36 (NA)	1775.12 (684.734)	370.91 (113.362)
Period 2: Cmax	899.93 (235.701)	2400.86 (928.257)	515.34 (77.207)	1351.18 (NA)	2413.31 (798.090)	619.00 (141.411)

Agalsidase Beta (0.5 mg/kg)-Migalastat (150 mg) vs Agalsidase Beta (1.0 mg/kg)-Migalastat (150 mg) vs Agalsidase Alfa (0.2 mg/kg)-Migalastat (150 mg): Cmax Ratio: 1.629 (90% CI 1.440 to 1.843)
Agalsidase Beta (0.5 mg/kg)-Migalastat (450 mg) vs Agalsidase Beta (1.0 mg/kg)-Migalastat (450 mg) vs Agalsidase Alfa (0.2 mg/kg)-Migalastat (450 mg): Cmax Ratio: 1.546 (90% CI 1.300 to 1.838)

Primary outcome: Change In Time To Maximum Observed Plasma Concentration (Tmax) And Terminal Elimination Half-life (T1/2) For Active α-Gal A Levels After Administration Of Migalastat

Time frame: 0 hr, 2 hr, 2 days, 7 days, 14 days post dose. Mean (Standard Deviation). Unit: hr

Measure	Agalsidase Beta (0.5 mg/kg)-Migalastat (150 mg) n = 5	Agalsidase Beta (1.0 mg/kg)-Migalastat (150 mg) n = 3	Agalsidase Alfa (0.2 mg/kg)-Migalastat (150 mg) n = 4	Agalsidase Beta (0.5 mg/kg)-Migalastat (450 mg) n = 1	Agalsidase Beta (1.0 mg/kg)-Migalastat (450 mg) n = 6	Agalsidase Alfa (0.2 mg/kg)-Migalastat (450 mg) n = 4
Period 1: tmax	2.07 (0.149)	2.16 (0.778)	0.75 (0.170)	3.00 (NA)	2.58 (0.801)	0.75 (0.170)
Period 2: tmax	2.30 (0.447)	2.41 (0.526)	0.75 (0.170)	3.00 (NA)	2.58 (0.801)	0.75 (0.170)
Period 1: t1/2	3.91 (2.054)	5.33 (4.082)	4.48 (3.132)	6.50 (NA)	3.11 (1.875)	5.15 (3.645)
Period 2: t1/2	3.51 (1.304)	4.30 (1.712)	4.27 (1.536)	3.49 (NA)	4.96 (1.532)	5.31 (2.462)

Primary outcome: Change In AUC For Total α-Gal A Protein Levels After Administration Of Migalastat

Time frame: 0 hr, 2 hr, 2 days, 7 days, 14 days post dose. Mean (Standard Deviation). Unit: hr*[ng/hr/mL]

Measure	Agalsidase Beta (0.5 mg/kg)-Migalastat (150 mg) n = 5	Agalsidase Beta (1.0 mg/kg)-Migalastat (150 mg) n = 3	Agalsidase Alfa (0.2 mg/kg)-Migalastat (150 mg) n = 4	Agalsidase Beta (0.5 mg/kg)-Migalastat (450 mg) n = 1	Agalsidase Beta (1.0 mg/kg)-Migalastat (450 mg) n = 6	Agalsidase Alfa (0.2 mg/kg)-Migalastat (450 mg) n = 4
Period 1: AUC0-t	57187.48 (47381.96)	29905.87 (20470.28)	42256.74 (13868.00)	16229.13 (NA)	17200.63 (16906.97)	28770.37 (10941.44)
Period 2: AUC0-t	56895.40 (45753.77)	34298.23 (17022.13)	42528.55 (14652.93)	16272.03 (NA)	22031.50 (16754.04)	29816.41 (8956.789)

Agalsidase Beta (0.5 mg/kg)-Migalastat (150 mg) vs Agalsidase Beta (1.0 mg/kg)-Migalastat (150 mg) vs Agalsidase Alfa (0.2 mg/kg)-Migalastat (150 mg): AUC0-t Ratio: 1.025 (90% CI 0.921 to 1.140)
Agalsidase Beta (0.5 mg/kg)-Migalastat (450 mg) vs Agalsidase Beta (1.0 mg/kg)-Migalastat (450 mg) vs Agalsidase Alfa (0.2 mg/kg)-Migalastat (450 mg): AUC0-t Ratio: 1.256 (90% CI 1.026 to 1.538)

1 secondary outcome is reported in the full results.

Adverse events

Time frame: Adverse events (AEs) were monitored up to 5.5 months after the first dose of study drug. For Period (Pd) 1 and 2 of Stages 1 and 2, AEs were reported starting 1 day prior (Day -1) to the first dose of study drug (Agalsidase Beta [Agal-B] or Agalsidase Alfa [Agal-A]) to 1 month after the last dose of study drug. For Pd 3 of Stage 1, AEs were reported starting on the day of the first dose of study drug (Day 1) to 1 month after the last dose of study drug (Migalastat [Mig]).. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Stage 1 Pd 1: 0.5 mg/kg Agal-B (Arm 1)	0 of 5 (0%)	4 of 5 (80%)	Not reported
Stage 1 Pd 2: 150 mg Mig Before 0.5 mg/kg Agal-B (Arm 1)	0 of 5 (0%)	0 of 5 (0%)	Not reported
Stage 1 Pd 3: 150 mg Mig (Arm 1)	0 of 5 (0%)	3 of 5 (60%)	Not reported
Stage 1 Pd 1: 1.0 mg/kg Agal-B (Arm 2)	0 of 3 (0%)	3 of 3 (100%)	Not reported
Stage 1 Pd 2: 150 mg Mig Before 1.0 mg/kg Agal-B (Arm 2)	0 of 3 (0%)	2 of 3 (66.7%)	Not reported
Stage 1 Pd 3: 150 mg Mig (Arm 2)	0 of 3 (0%)	0 of 3 (0%)	Not reported
Stage 1 Pd 1: 0.2 mg/kg Agal-A (Arm 3)	0 of 4 (0%)	1 of 4 (25%)	Not reported
Stage 1 Pd 2: 150 mg Mig Before 0.2 mg/kg Agal-A (Arm 3)	0 of 4 (0%)	0 of 4 (0%)	Not reported
Stage 1 Pd 3: 150 mg Mig (Arm 3)	0 of 4 (0%)	2 of 4 (50%)	Not reported
Stage 2 Pd 1: 0.5 mg/kg Agal-B (Arm 4)	1 of 2 (50%)	1 of 2 (50%)	Not reported
Stage 2 Pd 2: 450 mg Mig Before 0.5 mg/kg Agal-B (Arm 4)	0 of 1 (0%)	0 of 1 (0%)	Not reported
Stage 2 Pd 1: 1.0 mg/kg Agal-B (Arm 5)	0 of 6 (0%)	1 of 6 (16.7%)	Not reported
Stage 2 Pd 2: 450 Mig Before 1.0 mg/kg Agal-B (Arm 5)	0 of 6 (0%)	1 of 6 (16.7%)	Not reported
Stage 2 Pd 1: 0.2 mg/kg Agal-A (Arm 6)	0 of 4 (0%)	3 of 4 (75%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 19 Dec 2018.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Results publication: PubMed 26252393Warnock DG, Bichet DG, Holida M, Goker-Alpan O, Nicholls K, Thomas M, Eyskens F, Shankar S, Adera M, Sitaraman S, Khanna R, Flanagan JJ, Wustman BA, Barth J, Barlow C, Valenzano KJ, Lockhart DJ, Boudes P, Johnson FK. Oral Migalastat HCl Leads to Greater Systemic Exposure and Tissue Levels of Active alpha-Galactosidase A in Fabry Patients when Co-Administered with Infused Agalsidase. PLoS One. 2015 Aug 7;10(8):e0134341. doi: 10.1371/journal.pone.0134341. eCollection 2015.

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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Medical Monitor Clinical Research study director

Amicus Therapeutics

10 sites in 5 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Migalastat HCl, Agalsidase Beta and Agalsidase Alfa in people with Fabry Disease, run by Amicus Therapeutics at 10 sites in 5 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 19 Dec 2018. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 10 sites in United States, Australia, Belgium, Canada, Netherlands. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 1 Nov 2018). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change In Area Under The Plasma Concentration Versus Time Curve (AUC) For Active α-Galactosidase A (α-Gal A) Levels After Administration Of Migalastat (time frame: 0 hr, 2 hr, 2 days, 7 days, 14 days post dose). The study lists 10 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01196871, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Dec 2018.