Fabry DiseasePhase 1NCT01489995
Migalastat Food Effect Study
A Phase 1 study testing A (migalastat), B (migalastat) and C (migalastat) in people with Fabry Disease, run by Amicus Therapeutics at 1 site in United States.
Trial summary
A 5-period crossover study to evaluate the effect of meal type and timing on migalastat HCl pharmacokinetics in healthy male and female subjects. Subjects will be randomly assigned to 1 of 5 treatment sequences and will receive each treatment over the course of 5 weekly periods. Read more about this study
- Who is it for?
- Ages 18 to 65 yearsHealthy volunteers can take part
- Conditions the study is for
- Fabry Disease
- What is being tested?
- A (migalastat), B (migalastat) and C (migalastat)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- Oct 2011to Dec 2011
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Male or Female aged 18 to 65 years inclusive
- Healthy, as determined by study physician
- Capable of giving informed consent
Who cannot join
- Positive for HIV or Hepatitis B and/or C viruses
- History of drug or alcohol abuse or addiction within 2 years
- Smoker or consumes tobacco products
- Participation in a clinical trial within 30 days of scheduled first dose
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Randomized, Open-Label, 5-Period Crossover Study to Evaluate the Effect of Meal Type and Timing on the Pharmacokinetics of Migalastat Hydrochloride in Healthy Volunteers.
A 5-period crossover study to evaluate the effect of meal type and timing on migalastat HCl pharmacokinetics in healthy male and female subjects. Subjects will be randomly assigned to 1 of 5 treatment sequences and will receive each treatment over the course of 5 weekly periods.
Read the detailed description
This is a Phase 1, randomized, open-label, 5-period crossover study to evaluate the effect of meal type and timing on migalastat HCl pharmacokinetics in healthy male and female subjects between the ages of 18 and 65 years. A total of 20 subjects will be enrolled such that approximately 14 evaluable subjects complete dosing and critical assessments. Subjects will be randomly assigned to 1 of 5 treatment sequences and will receive each treatment over the course of 5 successive weekly periods including a single dose of migalastat HCl 150 mg in the fasting state as the reference treatment. There will be at least a 7-day washout period between each dose of migalastat HCl and a follow-up visit approximately 7 to 10 days after the last dose in Period 5.
All subjects will be screened within 28 days of admission to the clinical unit. In each period, subjects will check in to the clinical unit the day prior to drug administration and have relevant assessments to ensure continued eligibility for dose administration. On Day 1 of each period, subjects will receive a single dose of migalastat HCl within 1 of the following 5 treatment regimens as follows:
- 150 mg migalastat HCl in the fasting state (reference arm)
- 150 mg migalastat HCl with simultaneous consumption of a glucose drink
- 150 mg migalastat HCl 1 hour before consumption of a high fat meal
- 150 mg migalastat HCl 1 hour before consumption of a light meal
- 150 mg migalastat HCl 1 hour after consumption of a light meal
Subjects will be confined to the clinical unit for 24 hours after dosing with serial blood samples collected for PK analysis. Safety will be assessed throughout the study by monitoring clinical laboratory tests, ECGs, physical examinations, vital signs, and AEs.
- Conditions
- Fabry Disease
- Being tested
- A (migalastat), B (migalastat), C (migalastat), D (migalastat), E (migalastat)
- Keywords
- Food effect, GR181413A, AT1001, migalastat hydrochloride, Lysosomal storage disorders, Pharmacokinetics
- Medical subject headings
- Fabry Disease, Lysosomal Storage Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
A (migalastat)Drug
150 mg migalastat HCl in the fasting state (reference arm)
B (migalastat)Drug
150 mg migalastat HCl with simultaneous consumption of a glucose drink
C (migalastat)Drug
150 mg migalastat HCl 1 hour before consumption of a high fat meal
D (migalastat)Drug
150 mg migalastat HCl 1 hour before consumption of a light meal
E (migalastat)Drug
150 mg migalastat HCl 1 hour after consumption of a light meal
| Study group | Role | What they receive |
|---|---|---|
| Reference | Experimental | Fasted Receives: Drug: A (migalastat) |
| Glucose Drink | Experimental | Fed Receives: Drug: B (migalastat) |
| Before High Fat Meal | Experimental | Fed Receives: Drug: C (migalastat) |
| Before Light Meal | Experimental | Fed Receives: Drug: D (migalastat) |
| After Light Meal | Experimental | Fed Receives: Drug: E (migalastat) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum observed plasma concentration of migalastat HCl after a single dose
Cmax
Measured: 5 weeks (60 PK timepoints)
Time of occurence of maximum observed plasma concentration of migalastat HCl after a single dose
tmax
Measured: 5 weeks (60 PK timepoints)
Area under the plasma concentration-time curve of migalastat HCl after a single dose from time 0 (before dosing) to infinity
AUC (0 to infinity)
Measured: 5 weeks (60 PK timepoints)
Terminal phase half life of migalastat HCl after a single dose
t1/2
Measured: 5 weeks (60 PK timepoints)
Apparent clearance following oral dosing of migalastat HCl after a single dose
CL/F
Measured: 5 weeks (60 PK timepoints)
Area under the plasma concentration-time curve of migalastat HCl after a single dose from time 0 (before dosing) to the time of the last quantifiable concentration
AUC (0 to t)
Measured: 5 weeks (60 PK timepoints)
Secondary outcomes (5)
Adverse Events
Measured: 5 weeks
Clinical Laboratory Tests
Measured: 5 weeks
Vital Signs
Measured: 5 weeks
ECGs
Measured: 5 weeks
Physical Examination
Measured: 5 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Dec 2011 (estimated)
- Study startOct 2011
- Primary completionDec 2011
- Study completionDec 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- Amicus Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Monitor, Clinical Research, Study director, AmicusTherapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01489995
- Sponsor's protocol ID
- 116050
- Status last verified
- Dec 2013
- First submitted
- 20 Oct 2011
- Last updated
- 18 Dec 2013
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Dec 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Open-Label, 5-Period Crossover Study to Evaluate the Effect of Meal Type and Timing on the Pharmacokinetics of Migalastat Hydrochloride in Healthy Volunteers.
- ClinicalTrials.gov ID
- NCT01489995
- Acronym / sponsor ID
- 116050
- Conditions
- Fabry Disease
- Keywords
- Food effect, GR181413A, AT1001, migalastat hydrochloride, Lysosomal storage disorders, Pharmacokinetics
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- Amicus TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- Oct 2011
- Primary completion
- Dec 2011 (actual)
- Study completion
- Dec 2011 (actual)
- First posted
- 12 Dec 2011 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A 5-period crossover study to evaluate the effect of meal type and timing on migalastat HCl pharmacokinetics in healthy male and female subjects. Subjects will be randomly assigned to 1 of 5 treatment sequences and will receive each treatment over the course of 5 weekly periods.
Detailed description
This is a Phase 1, randomized, open-label, 5-period crossover study to evaluate the effect of meal type and timing on migalastat HCl pharmacokinetics in healthy male and female subjects between the ages of 18 and 65 years. A total of 20 subjects will be enrolled such that approximately 14 evaluable subjects complete dosing and critical assessments. Subjects will be randomly assigned to 1 of 5 treatment sequences and will receive each treatment over the course of 5 successive weekly periods including a single dose of migalastat HCl 150 mg in the fasting state as the reference treatment. There will be at least a 7-day washout period between each dose of migalastat HCl and a follow-up visit approximately 7 to 10 days after the last dose in Period 5.
All subjects will be screened within 28 days of admission to the clinical unit. In each period, subjects will check in to the clinical unit the day prior to drug administration and have relevant assessments to ensure continued eligibility for dose administration. On Day 1 of each period, subjects will receive a single dose of migalastat HCl within 1 of the following 5 treatment regimens as follows:
- 150 mg migalastat HCl in the fasting state (reference arm)
- 150 mg migalastat HCl with simultaneous consumption of a glucose drink
- 150 mg migalastat HCl 1 hour before consumption of a high fat meal
- 150 mg migalastat HCl 1 hour before consumption of a light meal
- 150 mg migalastat HCl 1 hour after consumption of a light meal
Subjects will be confined to the clinical unit for 24 hours after dosing with serial blood samples collected for PK analysis. Safety will be assessed throughout the study by monitoring clinical laboratory tests, ECGs, physical examinations, vital signs, and AEs.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| Reference | Experimental | Drug: A (migalastat)Fasted |
| Glucose Drink | Experimental | Drug: B (migalastat)Fed |
| Before High Fat Meal | Experimental | Drug: C (migalastat)Fed |
| Before Light Meal | Experimental | Drug: D (migalastat)Fed |
| After Light Meal | Experimental | Drug: E (migalastat)Fed |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Male or Female aged 18 to 65 years inclusive
- Healthy, as determined by study physician
- Capable of giving informed consent
Key exclusion criteria (4 listed)
- Positive for HIV or Hepatitis B and/or C viruses
- History of drug or alcohol abuse or addiction within 2 years
- Smoker or consumes tobacco products
- Participation in a clinical trial within 30 days of scheduled first dose
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 |
|---|---|---|
| A (migalastat)150 mg migalastat HCl in the fasting state (reference arm) | Drug | Reference |
| B (migalastat)150 mg migalastat HCl with simultaneous consumption of a glucose drink | Drug | Glucose Drink |
| C (migalastat)150 mg migalastat HCl 1 hour before consumption of a high fat meal | Drug | Before High Fat Meal |
| D (migalastat)150 mg migalastat HCl 1 hour before consumption of a light meal | Drug | Before Light Meal |
| E (migalastat)150 mg migalastat HCl 1 hour after consumption of a light meal | Drug | After Light Meal |
Primary outcome
Maximum observed plasma concentration of migalastat HCl after a single dose
Time frame: 5 weeks (60 PK timepoints)
Cmax
Time of occurence of maximum observed plasma concentration of migalastat HCl after a single dose
Time frame: 5 weeks (60 PK timepoints)
tmax
Area under the plasma concentration-time curve of migalastat HCl after a single dose from time 0 (before dosing) to infinity
Time frame: 5 weeks (60 PK timepoints)
AUC (0 to infinity)
Terminal phase half life of migalastat HCl after a single dose
Time frame: 5 weeks (60 PK timepoints)
t1/2
Apparent clearance following oral dosing of migalastat HCl after a single dose
Time frame: 5 weeks (60 PK timepoints)
CL/F
Area under the plasma concentration-time curve of migalastat HCl after a single dose from time 0 (before dosing) to the time of the last quantifiable concentration
Time frame: 5 weeks (60 PK timepoints)
AUC (0 to t)
5 secondary outcomes
Adverse Events
Time frame: 5 weeks
Clinical Laboratory Tests
Time frame: 5 weeks
Vital Signs
Time frame: 5 weeks
ECGs
Time frame: 5 weeks
Physical Examination
Time frame: 5 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Dec 2013.
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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
AmicusTherapeutics
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing A (migalastat), B (migalastat) and C (migalastat) in people with Fabry Disease, run by Amicus Therapeutics at 1 site in United States.
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 3 inclusion and 4 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 18 Dec 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Maximum observed plasma concentration of migalastat HCl after a single dose (time frame: 5 weeks (60 PK timepoints)). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 18 Dec 2013.