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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT02194985
Registration number
NCT02194985
Ethics application status
Date submitted
17/07/2014
Date registered
21/07/2014
Date last updated
21/12/2020
Titles & IDs
Public title
Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease
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Scientific title
An Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Migalastat Hydrochloride Monotherapy in Subjects With Fabry Disease
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Secondary ID [1]
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2014-002701-38
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Secondary ID [2]
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AT1001-042
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Fabry Disease
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Condition category
Condition code
Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Metabolic and Endocrine
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Metabolic disorders
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Metabolic and Endocrine
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Other metabolic disorders
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Intervention/exposure
Study type
Interventional(has expanded access)
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Description of intervention(s) / exposure
Treatment: Drugs - migalastat HCl 150 mg
Experimental: Migalastat HCl 150 mg - Migalastat HCl 150 milligram (mg).
Treatment: Drugs: migalastat HCl 150 mg
Migalastat HCl 150 mg (equivalent to 123 mg migalastat) was provided as capsules in blister packs. One capsule was taken orally every other day.
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Number Of Participants Experiencing Adverse Events (AEs)
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Assessment method [1]
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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.
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Timepoint [1]
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Day 1 after first dose to approximately 30 days after last treatment, median duration of 3.1 years
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Secondary outcome [1]
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Annualized Rate Of Change In The Estimated Glomerular Filtration Rate (eGFR)
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Assessment method [1]
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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)a × 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, a 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.
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Timepoint [1]
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Baseline to approximately 30 days after last treatment, median duration of 3.1 years
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Secondary outcome [2]
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Change From Baseline In eGFR At End Of Study
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Assessment method [2]
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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.
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Timepoint [2]
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Baseline to approximately 30 days after last treatment, median duration of 3.1 years
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Secondary outcome [3]
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Change From Baseline In Plasma Globotriaosylsphingosine (Lyso-Gb3) To End Of Study
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Assessment method [3]
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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.
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Timepoint [3]
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Baseline to approximately 30 days after last treatment, median duration of 3.1 years
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Secondary outcome [4]
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Change From Baseline In White Blood Cell a-Gal A Activity To End Of Study
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Assessment method [4]
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The activity of the a-galactosidase A (a-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.
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Timepoint [4]
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Baseline to approximately 30 days after last treatment, median duration of 3.1 years
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Secondary outcome [5]
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Change From Baseline In 24-hour Urine Protein To End Of Study
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Assessment method [5]
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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.
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Timepoint [5]
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Baseline to approximately 30 days after last treatment, median duration of 3.1 years
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Secondary outcome [6]
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Change From Baseline In Left Ventricular Mass (LVM) To End Of Study
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Assessment method [6]
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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.
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Timepoint [6]
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Baseline to approximately 30 days after last treatment, median duration of 3.1 years
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Secondary outcome [7]
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Change From Baseline In Left Ventricular Mass Index (LVMi) To End Of Study
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Assessment method [7]
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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.
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Timepoint [7]
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Baseline to approximately 30 days after last treatment, median duration of 3.1 years
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Secondary outcome [8]
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Change From Baseline In Patient Reported Quality Of Life To End Of Study, As Assessed By The Short Form-36 (SF-36) Questionnaire
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Assessment method [8]
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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.
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Timepoint [8]
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Baseline to approximately 30 days after last treatment, median duration of 3.1 years
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Eligibility
Key inclusion criteria
- 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)
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Minimum age
18
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
- 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
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Study design
Purpose of the study
Treatment
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Allocation to intervention
N/A
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
14/03/2015
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
23/10/2019
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Sample size
Target
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Accrual to date
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Final
84
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Recruitment in Australia
Recruitment state(s)
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Recruitment hospital [1]
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Clinical Study Site - Adelaide
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Recruitment hospital [2]
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Clinical Study Site - Parkville
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Recruitment postcode(s) [1]
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5000 - Adelaide
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Recruitment postcode(s) [2]
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3050 - Parkville
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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Georgia
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Country [2]
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United States of America
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State/province [2]
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Kansas
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Country [3]
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United States of America
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State/province [3]
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Michigan
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Country [4]
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United States of America
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State/province [4]
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New York
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Country [5]
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United States of America
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State/province [5]
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Oregon
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Country [6]
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United States of America
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State/province [6]
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Texas
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Country [7]
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United States of America
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State/province [7]
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Virginia
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Country [8]
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Argentina
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State/province [8]
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Pilar
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Country [9]
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Austria
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State/province [9]
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Wien
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Country [10]
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Belgium
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State/province [10]
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Edegem
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Country [11]
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Brazil
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State/province [11]
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Porto Alegre
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Canada
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State/province [12]
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Quebec
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Country [13]
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Denmark
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State/province [13]
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Copenhagen
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Country [14]
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Egypt
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State/province [14]
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Cairo
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Country [15]
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France
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State/province [15]
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Garches
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Country [16]
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France
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State/province [16]
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Lille
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Country [17]
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Italy
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State/province [17]
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Firenze
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Country [18]
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Italy
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State/province [18]
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Roma
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Country [19]
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Japan
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State/province [19]
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Osaka
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Country [20]
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Japan
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State/province [20]
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Niigata
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Country [21]
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Japan
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State/province [21]
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Osaka-shi
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Country [22]
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Japan
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State/province [22]
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Tokyo
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Country [23]
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Spain
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State/province [23]
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Barcelona
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Country [24]
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Turkey
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State/province [24]
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Ankara
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United Kingdom
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State/province [25]
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London
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Amicus Therapeutics
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
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.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT02194985
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Medical Monitor Clinical Research
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Address
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Amicus Therapeutics
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Address
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Country
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Phone
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Fax
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Email
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT02194985
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