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Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) 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: NCT04049760
Recruitment Status : Active, not recruiting
First Posted : August 8, 2019
Last Update Posted : July 20, 2022
Sponsor:
Information provided by (Responsible Party):
Amicus Therapeutics

Study Description
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Brief Summary:
This is a long-term, Open-label Study to Evaluate the Safety, Pharmacodynamics, and Efficacy of Migalastat in Subjects > 12 Years of Age With Fabry Disease and Amenable GLA Variants

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

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 15 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Long-term, Open-label Study to Evaluate the Safety, Pharmacodynamics, and Efficacy of Migalastat in Subjects > 12 Years of Age With Fabry Disease and Amenable GLA Variants
Actual Study Start Date : October 14, 2019
Estimated Primary Completion Date : December 1, 2025
Estimated Study Completion Date : December 1, 2025

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: migalastat HCl 150 mg
One migalastat 123 mg capsule equivalent to 150 mg migalastat HCl will be administered every other day (QOD) during the treatment period.
 Drug: migalastat HCl 150 mg
migalastat HCl 150 mg capsule
Other Names:
  • migalastat
  • AT1001


Outcome Measures
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Primary Outcome Measures :
  1. incidence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), and AEs leading to discontinuation of study drug [ Time Frame: Month 60 ]
  2. change in body weight in kilograms [ Time Frame: baseline over time; Up to 5 years ]
  3. change in height in centimeters [ Time Frame: baseline over time; Up to 5 years ]
  4. changes in Electrocardiogram (ECG) results [ Time Frame: baseline over time; Up to 5 years ]
    A 12-lead ECG will be obtained.

  5. incidence of changes in echocardiogram results [ Time Frame: baseline over time; Up to 5 years ]
    Systolic and diastolic heart function and structure is assessed by ultrasound of the heart. Echocardiogram parameters include left ventricular mass index (LVMi), ejection fraction, fractional shortening, left ventricular internal diameter end diastole and end systole, midwall fractional shortening, and wall thickness.

  6. change in Tanner stage [ Time Frame: Every 6 Months; Up to 5 years ]
    Tanner Staging of Sexual Development will be used to assess sexual development (i.e. breast development (B1 to B5) and pubic hair development (Ph-1 to Ph-5) in females and pubic hair and genetical development (G1-G5) in males.

  7. incidence of concomitant medications use [ Time Frame: Every 1 Month; Up to 5 years ]

Secondary Outcome Measures :
  1. change in plasma levels of lyso-Gb3 [ Time Frame: Every 6 Months; Up to 5 years ]
  2. change in eGFR [ Time Frame: Every 6 Months; Up to 5 years ]
  3. change in urine protein [ Time Frame: Every 6 Months; Up to 5 years ]
  4. change in albumin levels [ Time Frame: Every 6 Months; Up to 5 years ]
  5. change in Left Ventricular Mass Index (LVMi) [ Time Frame: Every Year; Up to 5 years ]
  6. change in Fabry-Specific Pediatric Health and Pain Questionnaire (FPHPQ)scores [ Time Frame: Every 3 months; Up to 5 years ]
    The FPHPQ includes questions about Fabry disease-specific symptoms (eg, sweating, pain, dizziness and tiredness, heat and cold intolerance, swollen eyelids, gastrointestinal symptoms, feeling thirsty, difficulty hearing, ringing or buzzing noise in the ears, and ability and enjoyment to participate in sports). The frequency of these symptoms will be rated using a 5-point Likert scale (always (worse), often, sometimes, seldom, never (better)). Pain intensity is measured on a 10-point scale, numeric responses are given for onset of pain and school days missed, and yes/no questions are posed about difficulty hearing and other problems not specifically mentioned. There are 2 age-specific self-report versions for children 8 to 12 years and 13 to 18 years, respectively.

  7. change in Pediatric and Quality of Life Inventory™ (PedsQL™) scores [ Time Frame: Every 3 months; Up to 5 years ]
    The PedsQL™ is a modular approach to measuring health-related quality of life in healthy children and adolescents and those with acute and chronic health conditions. It consists of 23 items and includes questions about physical functioning, emotional functioning, social functioning, and school functioning relative to the prior 7 days, using a 5-point scale (never (better), almost never, sometimes, often and almost always (worse)). Both parents or legally-authorized representatives and subjects complete the appropriate version of the PedsQL independently of one another. Parents or legally-authorized representatives and subjects may self-administer the questions after introductory instructions are given by study site personnel.


Eligibility Criteria
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Ages Eligible for Study:   12 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male or female subjects diagnosed with Fabry disease > 12 years of age who completed Study AT1001-020
  • Subject's parent or legally-authorized representative is willing and able to provide written informed consent and authorization for use and disclosure of personal health information or research-related health information, and subject provides assent, if applicable
  • Subject's parent or legally-authorized representative is willing and able to provide written informed consent and authorization for use and disclosure of personal health information or research-related health information, and subject provides assent, if applicable

Exclusion Criteria:

  • Has moderate or severe renal impairment (eGFR <60 ml/min/1.73 m2 at screening)
  • Has advanced kidney disease requiring dialysis or kidney transplantation
  • History of allergy or sensitivity to study medication (including excipients) or other iminosugars (eg, miglustat, miglitol)
  • Has received any gene therapy at any time or anticipates starting gene therapy during the study period
  • Requires treatment with Glyset (miglitol), Zavesca (miglustat) within 6 months before screening or throughout the study
  • Requires treatment with Replagal (agalsidase alfa), or Fabrazyme (agalsidase beta) within 14 days before screening or throughout the study
  • Subject is treated or has been treated with any investigational/experimental drug, biologic or device within 30 days before screening
  • Any intercurrent illness or condition or concomitant medication use considered to be a contraindication at screening or baseline or that may preclude the subject from fulfilling the protocol requirements or suggests to the investigator that the potential subject may have an unacceptable risk by participating in this study
  • Pregnant or breast-feeding
  • Otherwise unsuitable for the study in the opinion of the investigator
Contacts and Locations
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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): NCT04049760


Locations
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United States, Florida
University of South Florida
Tampa, Florida, United States, 33606
United States, Georgia
The Emory Clinic
Atlanta, Georgia, United States, 30322
United States, Minnesota
University of Minnesota Masonic Children's Hospital and Clinics
Minneapolis, Minnesota, United States, 55454
United States, Ohio
Cincinnati Children's Hospital
Cincinnati, Ohio, United States, 45229
United States, Virginia
Lysosomal & Rare Disorders Research & Treatment Center
Fairfax, Virginia, United States, 22030
United Kingdom
Royal Free London NHS Foundation Trust
London, United Kingdom
Sponsors and Collaborators
Amicus Therapeutics
More Information
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Responsible Party: Amicus Therapeutics
ClinicalTrials.gov Identifier: NCT04049760    
Other Study ID Numbers: AT1001-036
2019-000222-21 ( EudraCT Number )
First Posted: August 8, 2019    Key Record Dates
Last Update Posted: July 20, 2022
Last Verified: March 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Amicus Therapeutics:
Lysosomal storage disease
migalastat
Additional relevant MeSH terms:
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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