Fabry DiseasePhase 3NCT02194985
Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease
A Phase 3 study testing migalastat HCl 150 mg in people with Fabry Disease, run by Amicus Therapeutics at 28 sites in 15 countries.
Trial summary
This is an open-label extension study intended to provide continued treatment with migalastat hydrochloride (HCl) for participants with Fabry disease who completed treatment of a previous migalastat HCl study. The study assessed the long-term safety and effectiveness of migalastat HCl. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Fabry Disease
- What is being tested?
- migalastat HCl 150 mg
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 84 participants
- Where
- 28 sitesin 15 countries
- Study dates
- 14 Mar 2015to 23 Oct 2019
- Phase
- Phase 3
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Participant had completed treatment in a previous study of migalastat HCl given as a monotherapy
- Male and female participant agreed to use protocol-identified acceptable contraception
- Participant was willing to provide written informed consent and authorization for use and disclosure of Personal Health Information (PHI)
Who cannot join
- Participant's last available estimated glomerular filtration rate (eGFR) in the previous study was <30 milliliter (mL)/minute (min)/1.73 meters squared (m^2); unless there was measured GFR available within 3 months of Baseline Visit, which was >30 mL/min/1.73 m^2
- Participant had undergone, or was scheduled to undergo kidney transplantation or was currently on dialysis
- Participant had a documented transient ischemic attack, stroke, unstable angina, or myocardial infarction within the 3 months before Baseline Visit
- Participant had clinically significant unstable cardiac disease in the opinion of the investigator (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)
- Participant had a history of allergy or sensitivity to AT1001 (including excipients) or other iminosugars (for example, miglustat, miglitol)
- Participant required treatment with Glyset® (miglitol) or Zavesca® (miglustat)
- Participants with severe or unsuitable concomitant medical condition
- Participants with clinically significant abnormal laboratory value(s) and/or clinically significant electrocardiogram (ECG) findings
Trial locations and contacts
28 sites in 15 countries.
28 sites in 15 countries
United States7 sites
Clinical Study Site
Atlanta, Georgia, 30322
Clinical Study Site
Dallas, Texas, 75226
Clinical Study Site
Fairfax, Virginia, 22030
Clinical Study Site
Grand Rapids, Michigan, 49525
Clinical Study Site
Kansas City, Kansas, 66160
Clinical Study Site
New York, New York, 10016
Clinical Study Site
Portland, Oregon, 97239
Japan4 sites
Clinical Study Site
Niigata, 951-8520
Clinical Study Site
Osaka, 545-8586
Clinical Study Site
Suita, Osaka, 565-0871
Clinical Study Site
Tokyo, 105-8471
Australia2 sites
Clinical Study Site
Adelaide, 5000
Clinical Study Site
Parkville, 3050
France2 sites
Clinical Study Site
Garches, 92380
Clinical Study Site
Lille, 59037
United Kingdom2 sites
Clinical Study Site
London, NW3 2QG
Clinical Study Site
London, WC1N3BG
About this study
Official title: An Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Migalastat Hydrochloride Monotherapy in Subjects With Fabry Disease
This is an open-label extension study intended to provide continued treatment with migalastat hydrochloride (HCl) for participants with Fabry disease who completed treatment of a previous migalastat HCl study. The study assessed the long-term safety and effectiveness of migalastat HCl.
- Conditions
- Fabry Disease
- Being tested
- migalastat HCl 150 mg
- Keywords
- Amicus Therapeutics, Galafold, Migalastat, AT1001
- 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 HCl 150 mgDrug
Also called AT1001, Galafold
Migalastat HCl 150 mg (equivalent to 123 mg migalastat) was provided as capsules in blister packs. One capsule was taken orally every other day.
| Study group | Role | What they receive |
|---|---|---|
| Migalastat HCl 150 mg | Experimental | Migalastat HCl 150 milligram (mg). Receives: Drug: migalastat HCl 150 mg |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number Of Participants Experiencing Adverse Events (AEs)
An AE was defined as any untoward medical occurrence in a participant administered migalastat that did not necessarily have a causal relationship with the treatment. Each AE was recorded at time of reporting; visits typically occurred every 6 months. Serious AEs were life threatening or resulted in death, resulted in disability/incapacity, hospitalization or prolonged hospitalization, or a congenital anomaly. The criteria for AE severity were: Mild: awareness of sign or symptom, does not interfere with normal everyday activities; Moderate: discomforting, interferes with normal everyday activities, but able to function; Severe: incapacitating, prevents normal everyday activities or significantly affects clinical status and requires medical intervention. A summary of serious and all other non-serious AEs regardless of causality is located in the Reported Adverse Events module.
Measured: Day 1 after first dose to approximately 30 days after last treatment, median duration of 3.1 years
Secondary outcomes (8)
Annualized Rate Of Change In The Estimated Glomerular Filtration Rate (eGFR)
The annualized rate of change in the eGFR was assessed per participant by the slope of the simple linear regression between the observed values and the assessment times. It was calculated by using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation (eGFR [CKD-EPI]) and the Modification of Diet in Renal Disease (MDRD) equation (eGFR [MDRD]). The equations are as follows:
eGFR [MDRD] = 175 × (1/Serum Creatinine in mg/deciliter^1.154) × (1/Age in years^0.203) × 0.742 [if female] × 1.212 [if black] × 0.808 [if Japanese];
eGFR [CKD-EPI] = 141 × min(Serum creatinine [Scr]/k, 1)α × max(Scr/k, 1) - 1.209 × 0.993Age × 1.018 [if female] × 1.159 [if black],
where Scr is serum creatinine, k is 0.7 for females and 0.9 for males, α is -0.329 for females and -0.411 for males, min indicates the minimum of Scr/k or 1, and max indicates the maximum of Scr/k or 1.
Participants with at least a Baseline and a post-Baseline value are presented.
Measured: Baseline to approximately 30 days after last treatment, median duration of 3.1 years
Change From Baseline In eGFR At End Of Study
The change from baseline in eGFR was calculated using eGFR[CKD-EPI] and eGFR[MDRD] equations. Baseline was defined as the data collected at Month 0, if not available, it was the last visit of the previous (feeder) study if done within 6 months. End of Study was the last recorded observation for each participant (approximately 30 days after last treatment). Only participants with both a Baseline value and an End of Study value were included in the change from baseline analysis.
Measured: Baseline to approximately 30 days after last treatment, median duration of 3.1 years
Change From Baseline In Plasma Globotriaosylsphingosine (Lyso-Gb3) To End Of Study
Concentrations of lyso-Gb3 were measured in plasma using a qualified assay. Baseline was defined as the data collected at Month 0, if not available, it was the last visit of the previous (feeder) study if done within 6 months. End of study was the last recorded observation for each participant (approximately 30 days after last treatment). Only participants with both a Baseline value and an End of Study value were included in the change from baseline analysis.
Measured: Baseline to approximately 30 days after last treatment, median duration of 3.1 years
Change From Baseline In White Blood Cell α-Gal A Activity To End Of Study
The activity of the α-galactosidase A (α-Gal A) enzyme was measured in leukocyte lysate by a validated fluorometric assay method, using 4-methylumbelliferone as a reference. The activity values obtained were normalized to protein (measured using a colorimetric assay). Baseline was defined as the data collected at Month 0, if not available, it was the last visit of the previous (feeder) study if done within 6 months. End of Study was the last recorded observation for each participant (approximately 30 days after last treatment). Results for male participants are reported. Only participants with both a Baseline value and an End of Study value were included in the change from baseline analysis.
Measured: Baseline to approximately 30 days after last treatment, median duration of 3.1 years
Change From Baseline In 24-hour Urine Protein To End Of Study
A 24-hour urine sample was collected to measure 24-hour urine protein. Baseline was defined as the data collected at Month 0, if not available, it was the last visit of the previous (feeder) study if done within 6 months. End of Study was the last recorded observation for each participant (approximately 30 days after last treatment). Only participants with both a Baseline value and an End of Study value were included in the change from baseline analysis.
Measured: Baseline to approximately 30 days after last treatment, median duration of 3.1 years
Change From Baseline In Left Ventricular Mass (LVM) To End Of Study
LVM was measured by echocardiography. Baseline was defined as the data collected at Month 0, if not available, it was the last visit of the previous (feeder) study if done within 6 months. End of Study was the last recorded observation for each participant (approximately 30 days after last treatment). Only participants with both a Baseline value and an End of Study value were included in the change from baseline analysis.
Measured: Baseline to approximately 30 days after last treatment, median duration of 3.1 years
Change From Baseline In Left Ventricular Mass Index (LVMi) To End Of Study
LVMi was measured by echocardiography. Baseline was defined as the data collected at Month 0, if not available, it was the last visit of the previous (feeder) study if done within 6 months. End of Study was the last recorded observation for each participant (approximately 30 days after last treatment). Only participants with both a Baseline value and an End of Study value were included in the change from baseline analysis.
Measured: Baseline to approximately 30 days after last treatment, median duration of 3.1 years
Change From Baseline In Patient Reported Quality Of Life To End Of Study, As Assessed By The Short Form-36 (SF-36) Questionnaire
The SF-36 is a participant self-rated questionnaire that is a general measure of perceived health status comprising 36 questions, which yields an 8-scale health profile (Physical Functioning, Role Physical, Bodily Pain, General Health, Vitality, Social Functioning, Role Emotional, and Mental Health). Scores on each item are summed and averaged (range: 0=worst to 100=best). Scores were normed to the US population. Higher score indicates less disability. A positive change from baseline indicates improvement. Baseline was defined as the data collected in the last visit of the previous (feeder) study. Only participants with both a Baseline value and an End of Study value were included in the change from baseline analysis.
Measured: Baseline to approximately 30 days after last treatment, median duration of 3.1 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Jul 2014 (estimated)
- Study start14 Mar 2015
- Primary completion23 Oct 2019
- Study completion23 Oct 2019
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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 84 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
- NCT02194985
- Sponsor's protocol ID
- AT1001-042
- Other IDs
- 2014-002701-38, 2014
- Status last verified
- Nov 2020
- First submitted
- 17 Jul 2014
- Last updated
- 21 Dec 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Expanded access
- Available
Publications
Narita I, Ohashi T, Sakai N, Hamazaki T, Skuban N, Castelli JP, Lagast H, Barth JA. Efficacy and safety of migalastat in a Japanese population: a subgroup analysis of the ATTRACT study. Clin Exp Nephrol. 2020 Feb;24(2):157-166. doi: 10.1007/s10157-019-01810-w. Epub 2019 Dec 30.
PubMed 31889231
Study at a glance
From the registry record, last updated by the sponsor on 21 Dec 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Migalastat Hydrochloride Monotherapy in Subjects With Fabry Disease
- ClinicalTrials.gov ID
- NCT02194985
- Acronym / sponsor ID
- AT1001-042
- Conditions
- Fabry Disease
- Keywords
- Amicus Therapeutics, Galafold, Migalastat, AT1001
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 84 participants (actual)
- Lead sponsor
- Amicus TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 14 Mar 2015 (actual)
- Primary completion
- 23 Oct 2019 (actual)
- Study completion
- 23 Oct 2019 (actual)
- First posted
- 21 Jul 2014 (estimated)
- Results
- Posted 21 Dec 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is an open-label extension study intended to provide continued treatment with migalastat hydrochloride (HCl) for participants with Fabry disease who completed treatment of a previous migalastat HCl study. The study assessed the long-term safety and effectiveness of migalastat HCl.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Migalastat HCl 150 mg | Experimental | Drug: migalastat HCl 150 mgMigalastat HCl 150 milligram (mg). |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Participant had completed treatment in a previous study of migalastat HCl given as a monotherapy
- Male and female participant agreed to use protocol-identified acceptable contraception
- Participant was willing to provide written informed consent and authorization for use and disclosure of Personal Health Information (PHI)
Key exclusion criteria (8 listed)
- Participant's last available estimated glomerular filtration rate (eGFR) in the previous study was <30 milliliter (mL)/minute (min)/1.73 meters squared (m^2); unless there was measured GFR available within 3 months of Baseline Visit, which was >30 mL/min/1.73 m^2
- Participant had undergone, or was scheduled to undergo kidney transplantation or was currently on dialysis
- Participant had a documented transient ischemic attack, stroke, unstable angina, or myocardial infarction within the 3 months before Baseline Visit
- Participant had clinically significant unstable cardiac disease in the opinion of the investigator (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)
- Participant had a history of allergy or sensitivity to AT1001 (including excipients) or other iminosugars (for example, miglustat, miglitol)
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 HCl 150 mgAlso: AT1001, GalafoldMigalastat HCl 150 mg (equivalent to 123 mg migalastat) was provided as capsules in blister packs. One capsule was taken orally every other day. | Drug | Migalastat HCl 150 mg |
Primary outcome
Number Of Participants Experiencing Adverse Events (AEs)
Time frame: Day 1 after first dose to approximately 30 days after last treatment, median duration of 3.1 years
An AE was defined as any untoward medical occurrence in a participant administered migalastat that did not necessarily have a causal relationship with the treatment. Each AE was recorded at time of reporting; visits typically occurred every 6 months. Serious AEs were life threatening or resulted in death, resulted in disability/incapacity, hospitalization or prolonged hospitalization, or a congenital anomaly. The criteria for AE severity were: Mild: awareness of sign or symptom, does not interfere with normal everyday activities; Moderate: discomforting, interferes with normal everyday activities, but able to function; Severe: incapacitating, prevents normal everyday activities or significantly affects clinical status and requires medical intervention. A summary of serious and all other non-serious AEs regardless of causality is located in the Reported Adverse Events module.
8 secondary outcomes
Annualized Rate Of Change In The Estimated Glomerular Filtration Rate (eGFR)
Time frame: Baseline to approximately 30 days after last treatment, median duration of 3.1 years
Change From Baseline In eGFR At End Of Study
Time frame: Baseline to approximately 30 days after last treatment, median duration of 3.1 years
Change From Baseline In Plasma Globotriaosylsphingosine (Lyso-Gb3) To End Of Study
Time frame: Baseline to approximately 30 days after last treatment, median duration of 3.1 years
Change From Baseline In White Blood Cell α-Gal A Activity To End Of Study
Time frame: Baseline to approximately 30 days after last treatment, median duration of 3.1 years
Change From Baseline In 24-hour Urine Protein To End Of Study
Time frame: Baseline to approximately 30 days after last treatment, median duration of 3.1 years
Change From Baseline In Left Ventricular Mass (LVM) To End Of Study
Time frame: Baseline to approximately 30 days after last treatment, median duration of 3.1 years
Change From Baseline In Left Ventricular Mass Index (LVMi) To End Of Study
Time frame: Baseline to approximately 30 days after last treatment, median duration of 3.1 years
Change From Baseline In Patient Reported Quality Of Life To End Of Study, As Assessed By The Short Form-36 (SF-36) Questionnaire
Time frame: Baseline to approximately 30 days after last treatment, median duration of 3.1 years
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Dec 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 31889231Narita I, Ohashi T, Sakai N, Hamazaki T, Skuban N, Castelli JP, Lagast H, Barth JA. Efficacy and safety of migalastat in a Japanese population: a subgroup analysis of the ATTRACT study. Clin Exp Nephrol. 2020 Feb;24(2):157-166. doi: 10.1007/s10157-019-01810-w. Epub 2019 Dec 30.
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
28 sites in 15 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing migalastat HCl 150 mg in people with Fabry Disease, run by Amicus Therapeutics at 28 sites in 15 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 3 inclusion and 8 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 21 Dec 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 28 sites in United States, Japan, Australia, France, Italy and 10 more countries. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 21 Dec 2020). 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?
Number Of Participants Experiencing Adverse Events (AEs) (time frame: Day 1 after first dose to approximately 30 days after last treatment, median duration of 3.1 years). 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 NCT02194985, 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 21 Dec 2020.