Fabry DiseasePhase 1NCT02082327
A Phase 1 Study To Evaluate the Safety of Migalastat Hydrochloride Given Intravenously to Healthy Volunteers
A Phase 1 study testing IV migalastat HCl and oral migalastat HCl in people with Fabry Disease, run by Amicus Therapeutics at 1 site in Netherlands.
Trial summary
The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics of IV migalastat HCl alone and to determine absolute bioavailability for oral migalastat HCl as compared to IV administered migalastat HCl in healthy volunteers. Read more about this study
- Who is it for?
- Ages 18 to 45 yearsHealthy volunteers can take part
- Conditions the study is for
- Fabry Disease
- What is being tested?
- IV migalastat HCl and oral migalastat HCl
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 31 participants
- Where
- 1 siteNetherlands
- Study dates
- Mar 2014to Jun 2014
- 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 45 years
- All sexes
- Healthy volunteers accepted
- Adult
Who may be able to join
- Males and females between 18 and 45 years of age.
- Body weight range ≥ 50 kg ≤ 100 kg and BMI within the range 18.5 - 29.9 kg/m2.
- Healthy as determined by a responsible and experienced physician, based on a medial evaluation.
- Male and female subjects of childbearing potential agree to adhere to the contraception requirements.
- Capable of giving written informed consent.
Who cannot join
- History of sensitivity to migalastat HCl or related iminosugars (eg, miglitol, miglustat), or other significant drug allergy.
- Past medical history, or physical examination findings, of clinically significant abnormalities that may put the subject at risk or interfere with outcome variables.
- Positive pre-study drug/alcohol screen.
- Pregnant or lactating females.
- The subject has participated in a clinical trial and has received an investigational product within 60 days prior to the first dosing day in the current study.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase 1, Randomized, Double-Blind, Placebo-Controlled, Single Dose Escalation Trial to Evaluate the Safety, Tolerability and Pharmacokinetics of Migalastat Hydrochloride Given Intravenously to Healthy Volunteers With an Open-Label, Randomized, Two-Way Crossover Arm
The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics of IV migalastat HCl alone and to determine absolute bioavailability for oral migalastat HCl as compared to IV administered migalastat HCl in healthy volunteers. The data from this study will help us understand how migalastat works in the body and will help us determine what would be an effective dose in future studies with migalastat hydrochloride.
- Conditions
- Fabry Disease
- Being tested
- IV migalastat HCl, IV placebo, oral migalastat HCl
- Keywords
- Fabry Disease, Fabry, Lysosomal storage disorders, LSD, Amicus, migalastat HCl, migalastat, AT1001
- 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.
IV migalastat HClDrug
Also called AT1001
IV placeboDrug
Also called Sodium Chloride 0.9%
oral migalastat HClDrug
Also called AT1001
| Study group | Role | What they receive |
|---|---|---|
| 0.3 mg/kg | Experimental | IV infusion of migalastat HCl or placebo Receives: Drug: IV migalastat HCl; Drug: IV placebo |
| 1 mg/kg | Experimental | IV infusion of migalastat HCl or placebo Receives: Drug: IV migalastat HCl; Drug: IV placebo |
| 10 mg/kg | Experimental | IV infusion of migalastat HCl or placebo Receives: Drug: IV migalastat HCl; Drug: IV placebo |
| 150 mg IV | Experimental | 150 mg single IV infusion Receives: Drug: IV migalastat HCl; Drug: oral migalastat HCl |
| 150 mg oral | Experimental | 150 mg single oral dose Receives: Drug: IV migalastat HCl; Drug: oral migalastat HCl |
What is being measured
The primary outcome is the main question the study is designed to answer.
Plasma pharmacokinetics of migalastat
To investigate the effect on the body of migalastat following a single 2 hour IV infusion in healthy subjects.
Measured: Pre-dose, 0.25, 0.5, 1, 1.5, 2.0, 2.5, 3, 4, 6, 8, 10, 12, 16, 24, 48
Safety and tolerability of migalastat
Adverse events, clinical laboratory test values, vital signs, ECG, physical examinations
Measured: 48 hours
Secondary outcomes (2)
Plasma pharmacokinetics of migalastat
To assess the dose proportionality of migalastat following a single 2 hour IV infusion
Measured: Pre-dose, 0.25, 0.5, 1, 1.5, 2.0, 2.5, 3, 4, 6, 8, 10, 12, 16, 24, 48
Urinary pharmacokinetics
To estimate the urinary excretion of unchanged migalastat following a single 2 hour IV infusion in healthy subjects
Measured: Pre dose, between 0-6, 6-12 and 12-24 hours after start of infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Mar 2014 (estimated)
- Study startMar 2014
- Primary completionJun 2014
- Study completionJun 2014
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
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 31 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, Amicus Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02082327
- Sponsor's protocol ID
- AT1001-018
- Other IDs
- 2013-005553-75
- Status last verified
- Apr 2014
- First submitted
- 6 Mar 2014
- Last updated
- 18 Jun 2014
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Jun 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1, Randomized, Double-Blind, Placebo-Controlled, Single Dose Escalation Trial to Evaluate the Safety, Tolerability and Pharmacokinetics of Migalastat Hydrochloride Given Intravenously to Healthy Volunteers With an Open-Label, Randomized, Two-Way Crossover Arm
- ClinicalTrials.gov ID
- NCT02082327
- Acronym / sponsor ID
- AT1001-018
- Conditions
- Fabry Disease
- Keywords
- Fabry Disease, Fabry, Lysosomal storage disorders, LSD, Amicus, migalastat HCl, migalastat, AT1001
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 31 participants (actual)
- Lead sponsor
- Amicus TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- Mar 2014
- Primary completion
- Jun 2014 (actual)
- Study completion
- Jun 2014 (actual)
- First posted
- 10 Mar 2014 (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.
The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics of IV migalastat HCl alone and to determine absolute bioavailability for oral migalastat HCl as compared to IV administered migalastat HCl in healthy volunteers. The data from this study will help us understand how migalastat works in the body and will help us determine what would be an effective dose in future studies with migalastat hydrochloride.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| 0.3 mg/kg | Experimental | Drug: IV migalastat HCl, Drug: IV placeboIV infusion of migalastat HCl or placebo |
| 1 mg/kg | Experimental | Drug: IV migalastat HCl, Drug: IV placeboIV infusion of migalastat HCl or placebo |
| 10 mg/kg | Experimental | Drug: IV migalastat HCl, Drug: IV placeboIV infusion of migalastat HCl or placebo |
| 150 mg IV | Experimental | Drug: IV migalastat HCl, Drug: oral migalastat HCl150 mg single IV infusion |
| 150 mg oral | Experimental | Drug: IV migalastat HCl, Drug: oral migalastat HCl150 mg single oral dose |
Who may qualify
- Ages
- 18 to 45 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (5 listed)
- Males and females between 18 and 45 years of age.
- Body weight range ≥ 50 kg ≤ 100 kg and BMI within the range 18.5 - 29.9 kg/m2.
- Healthy as determined by a responsible and experienced physician, based on a medial evaluation.
- Male and female subjects of childbearing potential agree to adhere to the contraception requirements.
- Capable of giving written informed consent.
Key exclusion criteria (5 listed)
- History of sensitivity to migalastat HCl or related iminosugars (eg, miglitol, miglustat), or other significant drug allergy.
- Past medical history, or physical examination findings, of clinically significant abnormalities that may put the subject at risk or interfere with outcome variables.
- Positive pre-study drug/alcohol screen.
- Pregnant or lactating females.
- The subject has participated in a clinical trial and has received an investigational product within 60 days prior to the first dosing day in the current study.
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 |
|---|---|---|
| IV migalastat HClAlso: AT1001 | Drug | 0.3 mg/kg, 1 mg/kg, 10 mg/kg, 150 mg IV, 150 mg oral |
| IV placeboAlso: Sodium Chloride 0.9% | Drug | 0.3 mg/kg, 1 mg/kg, 10 mg/kg |
| oral migalastat HClAlso: AT1001 | Drug | 150 mg IV, 150 mg oral |
Primary outcome
Plasma pharmacokinetics of migalastat
Time frame: Pre-dose, 0.25, 0.5, 1, 1.5, 2.0, 2.5, 3, 4, 6, 8, 10, 12, 16, 24, 48
To investigate the effect on the body of migalastat following a single 2 hour IV infusion in healthy subjects.
Safety and tolerability of migalastat
Time frame: 48 hours
Adverse events, clinical laboratory test values, vital signs, ECG, physical examinations
2 secondary outcomes
Plasma pharmacokinetics of migalastat
Time frame: Pre-dose, 0.25, 0.5, 1, 1.5, 2.0, 2.5, 3, 4, 6, 8, 10, 12, 16, 24, 48
Urinary pharmacokinetics
Time frame: Pre dose, between 0-6, 6-12 and 12-24 hours after start of infusion
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 Jun 2014.
Related trials for Fabry Disease
RecruitingPhase 3
A Study to Learn How Well Lucerastat Works and How Safe it is in Untreated Adult Male Participants With Fabry Disease
Fabry Disease
RecruitingObservational study
A Study of Patients With Fabry Disease (US Specific)
Fabry Disease
RecruitingObservational study
A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding
Fabry Disease
Active, not recruiting
Evaluation of the Effect of a Low-FODMAP Diet on Gastrointestinal Symptoms and Inflammatory Markers in Fabry Patients
Fabry Disease
RecruitingPhase 3
A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants
Fabry Disease
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
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 IV migalastat HCl and oral migalastat HCl in people with Fabry Disease, run by Amicus Therapeutics at 1 site in Netherlands.
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 5 inclusion and 5 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 Jun 2014. 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 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 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?
Plasma pharmacokinetics of migalastat (time frame: Pre-dose, 0.25, 0.5, 1, 1.5, 2.0, 2.5, 3, 4, 6, 8, 10, 12, 16, 24, 48). The study lists 2 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 Jun 2014.