Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 1 of 1 for:    NCT02194985
Previous Study | Return to List | Next Study

Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02194985
Recruitment Status : Completed
First Posted : July 21, 2014
Results First Posted : December 21, 2020
Last Update Posted : December 21, 2020
Sponsor:
Information provided by (Responsible Party):
Amicus Therapeutics

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.

Condition or disease Intervention/treatment Phase
Fabry Disease Drug: migalastat HCl 150 mg Phase 3

Expanded Access : An investigational treatment associated with this study has been approved for sale to the public.   More info ...

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 84 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Migalastat Hydrochloride Monotherapy in Subjects With Fabry Disease
Actual Study Start Date : March 14, 2015
Actual Primary Completion Date : October 23, 2019
Actual Study Completion Date : October 23, 2019


Arm Intervention/treatment
Experimental: Migalastat HCl 150 mg
Migalastat HCl 150 milligram (mg).
Drug: 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.
Other Names:
  • AT1001
  • Galafold




Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. 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 ]

    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.


  2. 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 ]
    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.

  3. 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 ]
    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.

  4. 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 ]
    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.

  5. 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 ]
    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.

  6. 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 ]
    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.

  7. 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 ]
    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.

  8. 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 ]
    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.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02194985


Locations
Layout table for location information
United States, Georgia
Clinical Study Site
Atlanta, Georgia, United States, 30322
United States, Kansas
Clinical Study Site
Kansas City, Kansas, United States, 66160
United States, Michigan
Clinical Study Site
Grand Rapids, Michigan, United States, 49525
United States, New York
Clinical Study Site
New York, New York, United States, 10016
United States, Oregon
Clinical Study Site
Portland, Oregon, United States, 97239
United States, Texas
Clinical Study Site
Dallas, Texas, United States, 75226
United States, Virginia
Clinical Study Site
Fairfax, Virginia, United States, 22030
Argentina
Clinical Study Site
Pilar, Argentina, B1629ODT
Australia
Clinical Study Site
Adelaide, Australia, 5000
Clinical Study Site
Parkville, Australia, 3050
Austria
Clinical Study Site
Wien, Austria, 1090
Belgium
Clinical Study Site
Edegem, Belgium, 2650
Brazil
Clinical Study Site
Porto Alegre, Brazil, 90035-903
Canada, Quebec
Clinical Study Site
Montreal, Quebec, Canada, H4J 1C5
Denmark
Clinical Study Site
Copenhagen, Denmark, 2100
Egypt
Clinical Study Site
Cairo, Egypt, 11451
France
Clinical Study Site
Garches, France, 92380
Clinical Study Site
Lille, France, 59037
Italy
Clinical Study Site
Firenze, Italy, 50134
Clinical Study Site
Roma, Italy, 00168
Japan
Clinical Study Site
Suita, Osaka, Japan, 565-0871
Clinical Study Site
Niigata, Japan, 951-8520
Clinical Study Site
Osaka-shi, Japan, 545-8586
Clinical Study Site
Tokyo, Japan, 105-8471
Spain
Clinical Study Site
Barcelona, Spain, 08025
Turkey
Clinical Study Site
Ankara, Turkey, 06500
United Kingdom
Clinical Study Site
London, United Kingdom, NW3 2QG
Clinical Study Site
London, United Kingdom, WC1N3BG
Sponsors and Collaborators
Amicus Therapeutics
Investigators
Layout table for investigator information
Study Director: Medical Monitor Clinical Research Amicus Therapeutics
  Study Documents (Full-Text)

Documents provided by Amicus Therapeutics:
Study Protocol  [PDF] October 21, 2015
Statistical Analysis Plan  [PDF] August 21, 2019

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Layout table for additonal information
Responsible Party: Amicus Therapeutics
ClinicalTrials.gov Identifier: NCT02194985    
Other Study ID Numbers: AT1001-042
2014-002701-38 ( EudraCT Number )
First Posted: July 21, 2014    Key Record Dates
Results First Posted: December 21, 2020
Last Update Posted: December 21, 2020
Last Verified: November 2020
Keywords provided by Amicus Therapeutics:
Amicus Therapeutics
Galafold
Migalastat
AT1001
Additional relevant MeSH terms:
Layout table for MeSH terms
Fabry Disease
Sphingolipidoses
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Cerebral Small Vessel Diseases
Cerebrovascular Disorders
Vascular Diseases
Cardiovascular Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders