Clinical trial NCT02194985: Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease
Official title: An Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Migalastat Hydrochloride Monotherapy in Subjects With Fabry Disease
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Fabry Disease
Keywords: Amicus Therapeutics
Galafold
Migalastat
AT1001
Lead sponsor: Amicus Therapeutics
Sponsor class: INDUSTRY
First posted: 2014-07-21
Enrollment: 84
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: 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)

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

Primary outcomes:
measure: Number Of Participants Experiencing Adverse Events (AEs)
timeFrame: Day 1 after first dose to approximately 30 days after last treatment, median duration of 3.1 years
Secondary outcomes:
measure: Annualized Rate Of Change In The Estimated Glomerular Filtration Rate (eGFR)
timeFrame: Baseline to approximately 30 days after last treatment, median duration of 3.1 years
measure: Change From Baseline In eGFR At End Of Study
timeFrame: Baseline to approximately 30 days after last treatment, median duration of 3.1 years
measure: Change From Baseline In Plasma Globotriaosylsphingosine (Lyso-Gb3) To End Of Study
timeFrame: Baseline to approximately 30 days after last treatment, median duration of 3.1 years
measure: Change From Baseline In White Blood Cell α-Gal A Activity To End Of Study
timeFrame: Baseline to approximately 30 days after last treatment, median duration of 3.1 years
measure: Change From Baseline In 24-hour Urine Protein To End Of Study
timeFrame: Baseline to approximately 30 days after last treatment, median duration of 3.1 years
measure: Change From Baseline In Left Ventricular Mass (LVM) To End Of Study
timeFrame: Baseline to approximately 30 days after last treatment, median duration of 3.1 years
measure: Change From Baseline In Left Ventricular Mass Index (LVMi) To End Of Study
timeFrame: Baseline to approximately 30 days after last treatment, median duration of 3.1 years
measure: Change From Baseline In Patient Reported Quality Of Life To End Of Study, As Assessed By The Short Form-36 (SF-36) Questionnaire
timeFrame: Baseline to approximately 30 days after last treatment, median duration of 3.1 years
Other outcomes:
Locations:
city: Atlanta
country: United States
facility: Clinical Study Site
state: Georgia
city: Kansas City
country: United States
facility: Clinical Study Site
state: Kansas
city: Grand Rapids
country: United States
facility: Clinical Study Site
state: Michigan
city: New York
country: United States
facility: Clinical Study Site
state: New York
city: Portland
country: United States
facility: Clinical Study Site
state: Oregon
city: Dallas
country: United States
facility: Clinical Study Site
state: Texas
city: Fairfax
country: United States
facility: Clinical Study Site
state: Virginia
city: Pilar
country: Argentina
facility: Clinical Study Site
city: Adelaide
country: Australia
facility: Clinical Study Site
city: Parkville
country: Australia
facility: Clinical Study Site
city: Wien
country: Austria
facility: Clinical Study Site
city: Edegem
country: Belgium
facility: Clinical Study Site
city: Porto Alegre
country: Brazil
facility: Clinical Study Site
city: Montreal
country: Canada
facility: Clinical Study Site
state: Quebec
city: Copenhagen
country: Denmark
facility: Clinical Study Site
city: Cairo
country: Egypt
facility: Clinical Study Site
city: Garches
country: France
facility: Clinical Study Site
city: Lille
country: France
facility: Clinical Study Site
city: Firenze
country: Italy
facility: Clinical Study Site
city: Roma
country: Italy
facility: Clinical Study Site
city: Suita
country: Japan
facility: Clinical Study Site
state: Osaka
city: Niigata
country: Japan
facility: Clinical Study Site
city: Osaka-shi
country: Japan
facility: Clinical Study Site
city: Tokyo
country: Japan
facility: Clinical Study Site
city: Barcelona
country: Spain
facility: Clinical Study Site
city: Ankara
country: Turkey
facility: Clinical Study Site
city: London
country: United Kingdom
facility: Clinical Study Site
city: London
country: United Kingdom
facility: Clinical Study Site
Study officials:
affiliation: Amicus Therapeutics
name: Medical Monitor Clinical Research
role: STUDY_DIRECTOR
References:
citation: 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.