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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04020055




Registration number
NCT04020055
Ethics application status
Date submitted
24/06/2019
Date registered
15/07/2019

Titles & IDs
Public title
A Study to Evaluate Migalastat in Fabry Subjects with Amenable GLA Variant and Renal Disease
Scientific title
An Open-label Study to Evaluate the Safety and Pharmacokinetics of Migalastat HCl in Subjects with Fabry Disease and Amenable GLA Variants and Severe Renal Impairment or End-Stage Renal Disease Treated with Hemodialysis
Secondary ID [1] 0 0
AT1001-025
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Fabry Disease 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Metabolic and Endocrine 0 0 0 0
Metabolic disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders
Renal and Urogenital 0 0 0 0
Kidney disease

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - migalastat HCl 150 mg

Experimental: Cohort 1: Severe Renal Impairment - All subjects will receive migalastat 123 mg, equivalent to 150 mg migalastat HCl (hereafter, migalastat) at a dose regimen based on their eGFRMDRD result at Visit 1. Subjects will take 1 migalastat capsule orally with water either every 4 or 7 days.

Experimental: Cohort 2: End-Stage Renal Disease - All hemodialysis subjects will receive migalastat 123 mg, equivalent to 150 mg migalastat HCl (hereafter, migalastat). Subjects will take 1 migalastat capsule orally with water every other week.


Treatment: Drugs: migalastat HCl 150 mg
migalastat HCl 150 mg capsule
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Maximum observed concentration (Cmax)
Timepoint [1] 0 0
Baseline through Month 12
Primary outcome [2] 0 0
Time to maximum concentration (tmax)
Timepoint [2] 0 0
Baseline through Month 12
Primary outcome [3] 0 0
Apparent terminal elimination half-life (t½)
Timepoint [3] 0 0
Baseline through Month 12
Primary outcome [4] 0 0
Concentration at the end of a dosing interval at steady state (Ctrough)
Timepoint [4] 0 0
Baseline through Month 12
Primary outcome [5] 0 0
Average plasma migalastat concentration over the dosing interval (Cavg)
Timepoint [5] 0 0
Baseline through Month 12
Primary outcome [6] 0 0
Area under the concentration-time curve at steady state during the dosing interval (AUC0-t)
Timepoint [6] 0 0
Baseline through Month 12
Primary outcome [7] 0 0
Area under the concentration-time curve from zero time (pre-dose) extrapolated to infinite time (AUC0-8)
Timepoint [7] 0 0
Baseline through Month 12
Primary outcome [8] 0 0
Apparent plasma clearance (CL/F)
Timepoint [8] 0 0
Baseline through Month 12
Primary outcome [9] 0 0
Apparent terminal phase volume of distribution (Vz/F)
Timepoint [9] 0 0
Baseline through Month 12
Primary outcome [10] 0 0
Dialysis clearance (CLD)
Timepoint [10] 0 0
Baseline through Month 12
Primary outcome [11] 0 0
Volume of dialysate collected during the interval (VD)
Timepoint [11] 0 0
Baseline through Month 12
Primary outcome [12] 0 0
Mean migalastat concentration in dialysate (CD)
Timepoint [12] 0 0
Baseline through Month 12
Primary outcome [13] 0 0
Amount recovered in dialysate (AeD)
Timepoint [13] 0 0
Baseline through Month 12
Primary outcome [14] 0 0
Fraction of the dose recovered in dialysate (FeD)
Timepoint [14] 0 0
Baseline through Month 12
Primary outcome [15] 0 0
Mean migalastat plasma concentration during the dialysis interval (P)
Timepoint [15] 0 0
Baseline through Month 12
Primary outcome [16] 0 0
Mean inlet area under the curve (AUCinlet)
Timepoint [16] 0 0
Baseline through Month 12
Primary outcome [17] 0 0
Mean outlet area under the curve (AUCoutlet)
Timepoint [17] 0 0
Baseline through Month 12
Primary outcome [18] 0 0
Extraction ratio (ED)
Timepoint [18] 0 0
Baseline through Month 12
Primary outcome [19] 0 0
Dialyzer blood flow (QD)
Timepoint [19] 0 0
Baseline through Month 12
Primary outcome [20] 0 0
Cumulative amount excreted over all collection intervals (Ae0-t)
Timepoint [20] 0 0
Baseline through Month 12
Primary outcome [21] 0 0
Fraction of the dose recovered after the last measurable time point postdose (Fe0-t)
Timepoint [21] 0 0
Baseline through Month 12
Primary outcome [22] 0 0
Renal clearance (CLr)
Timepoint [22] 0 0
Baseline through Month 12
Secondary outcome [1] 0 0
Adverse events (AEs)
Timepoint [1] 0 0
Baseline through Month 12
Secondary outcome [2] 0 0
Number of subjects with abnormal clinical chemistry laboratory test results
Timepoint [2] 0 0
Baseline through Month 12
Secondary outcome [3] 0 0
Number of subjects with abnormal hematology laboratory test results
Timepoint [3] 0 0
Baseline through Month 12
Secondary outcome [4] 0 0
Number of subjects with abnormal urinalysis laboratory test results
Timepoint [4] 0 0
Baseline through Month 12
Secondary outcome [5] 0 0
Number of subjects with abnormal electrocardiogram (ECG) findings as a measure of safety and tolerability
Timepoint [5] 0 0
Baseline through Month 12
Secondary outcome [6] 0 0
Change from baseline in estimated glomerular filtration rate (eGFR) based on the Modification of Diet in Renal Disease equation (eGFR MDRD)
Timepoint [6] 0 0
Baseline through Month 12
Secondary outcome [7] 0 0
Change from baseline eGFR based on the Chronic Kidney Disease Epidemiology Collaboration equation (eGFRCKD-EPI)
Timepoint [7] 0 0
Baseline through Month 12
Secondary outcome [8] 0 0
Change from baseline in plasma globotriaosylsphingosine (lyso-Gb3)
Timepoint [8] 0 0
Baseline through Month 12

Eligibility
Key inclusion criteria
1. Male or female subjects aged 18 years or older, diagnosed with Fabry disease.
2. Subject (or legally authorized representative as applicable) is willing and able to provide written informed consent and authorization for use and disclosure of Personal Health Information
3. Subject has a GLA variant that is amenable to migalastat recorded in their medical records
4. Subject has at least 1 documented eGFR value of < 30 mL/min/1.73 m2 within the last 3 months and has an eGFRMDRD value of < 30 mL/min/1.73 m2 at Visit 1
5. Subjects with ESRD have been on a stable 2- or 3-times a week HD (standard or HDF) regimen for at least 2 months prior to the screening visit
6. Subjects with ESRD must commit to completing at least 4 standard HD or HDF sessions during each 2-week dosing interval.
7. Subjects with ESRD must commit to completing the entire prescribed duration for each dialysis session.
8. If of reproductive potential, both male and female patients agree to use a medically accepted method of contraception
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Subject has undergone kidney transplantation
2. Subject is on peritoneal dialysis
3. Subject is treated or has been treated with another investigational drug (except migalastat) within the 30 days
4. Subject has undergone any gene therapy at any time prior to the study or anticipates undergoing gene therapy during the study.
5. Subject has had a documented transient ischemic attack, stroke, unstable angina, or myocardial infarction
6. Subject has clinically significant unstable cardiac disease
7. Subject has any intercurrent illness or condition that may preclude the subject from fulfilling the protocol requirements
8. Subject has a history of allergy or sensitivity to migalastat (including excipients) or other iminosugars (eg, miglustat, miglitol)
9. Subject requires concurrent treatment with Glyset® (miglitol), Replagal® (agalsidase alfa), or Fabrazyme® (agalsidase beta)
10. Subject requires concurrent treatment with Zavesca® (miglustat) or has been treated with Zavesca
11. Female subject is pregnant or breast-feeding
12. Subject is unable to comply with study requirements
13. In France only, protected persons as defined by the Public Health Code

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
31/10/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
31/12/2025
Actual
Sample size
Target
14
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC,Washingto
Recruitment hospital [1] 0 0
Royal Melbourne Hospital - Parkville
Recruitment hospital [2] 0 0
Royal Perth Hospital - Perth
Recruitment postcode(s) [1] 0 0
3050 - Parkville
Recruitment postcode(s) [2] 0 0
6000 - Perth
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Georgia
Country [2] 0 0
United States of America
State/province [2] 0 0
Ohio
Country [3] 0 0
United States of America
State/province [3] 0 0
Texas
Country [4] 0 0
United States of America
State/province [4] 0 0
Virginia
Country [5] 0 0
France
State/province [5] 0 0
Paris
Country [6] 0 0
Japan
State/province [6] 0 0
Osaka
Country [7] 0 0
Portugal
State/province [7] 0 0
Coimbra
Country [8] 0 0
Spain
State/province [8] 0 0
Barcelona
Country [9] 0 0
Spain
State/province [9] 0 0
Córdoba
Country [10] 0 0
Spain
State/province [10] 0 0
Elda
Country [11] 0 0
Spain
State/province [11] 0 0
Madrid

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Amicus Therapeutics
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Clinical Research
Address 0 0
Amicus Therapeutics
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Amicus Therapeutics Patient Advocacy
Address 0 0
Country 0 0
Phone 0 0
609-662-2000
Fax 0 0
Email 0 0
clinicaltrials@amicusrx.com
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT04020055