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Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease

Primary Purpose

Fabry Disease

Status
Active
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
migalastat HCl 150 mg
Sponsored by
Amicus Therapeutics
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Fabry Disease focused on measuring Lysosomal storage disease, migalastat

Eligibility Criteria

12 Years - 17 Years (Child)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • University of South Florida
  • The Emory Clinic
  • University of Minnesota Masonic Children's Hospital and Clinics
  • Cincinnati Children's Hospital
  • Lysosomal & Rare Disorders Research & Treatment Center
  • Royal Free London NHS Foundation Trust

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

migalastat HCl 150 mg

Arm Description

One migalastat 123 mg capsule equivalent to 150 mg migalastat HCl will be administered every other day (QOD) during the treatment period.

Outcomes

Primary Outcome Measures

incidence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), and AEs leading to discontinuation of study drug
change in body weight in kilograms
change in height in centimeters
changes in Electrocardiogram (ECG) results
A 12-lead ECG will be obtained.
incidence of changes in echocardiogram results
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.
change in Tanner stage
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.
incidence of concomitant medications use

Secondary Outcome Measures

change in plasma levels of lyso-Gb3
change in eGFR
change in urine protein
change in albumin levels
change in Left Ventricular Mass Index (LVMi)
change in Fabry-Specific Pediatric Health and Pain Questionnaire (FPHPQ)scores
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.
change in Pediatric and Quality of Life Inventory™ (PedsQL™) scores
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.

Full Information

First Posted
July 30, 2019
Last Updated
October 13, 2023
Sponsor
Amicus Therapeutics
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1. Study Identification

Unique Protocol Identification Number
NCT04049760
Brief Title
Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease
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
Study Type
Interventional

2. Study Status

Record Verification Date
October 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
October 14, 2019 (Actual)
Primary Completion Date
December 1, 2025 (Anticipated)
Study Completion Date
December 1, 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Amicus Therapeutics

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Fabry Disease
Keywords
Lysosomal storage disease, migalastat

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
15 (Actual)

8. Arms, Groups, and Interventions

Arm Title
migalastat HCl 150 mg
Arm Type
Experimental
Arm Description
One migalastat 123 mg capsule equivalent to 150 mg migalastat HCl will be administered every other day (QOD) during the treatment period.
Intervention Type
Drug
Intervention Name(s)
migalastat HCl 150 mg
Other Intervention Name(s)
migalastat, AT1001
Intervention Description
migalastat HCl 150 mg capsule
Primary Outcome Measure Information:
Title
incidence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), and AEs leading to discontinuation of study drug
Time Frame
Month 60
Title
change in body weight in kilograms
Time Frame
baseline over time; Up to 5 years
Title
change in height in centimeters
Time Frame
baseline over time; Up to 5 years
Title
changes in Electrocardiogram (ECG) results
Description
A 12-lead ECG will be obtained.
Time Frame
baseline over time; Up to 5 years
Title
incidence of changes in echocardiogram results
Description
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.
Time Frame
baseline over time; Up to 5 years
Title
change in Tanner stage
Description
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.
Time Frame
Every 6 Months; Up to 5 years
Title
incidence of concomitant medications use
Time Frame
Every 1 Month; Up to 5 years
Secondary Outcome Measure Information:
Title
change in plasma levels of lyso-Gb3
Time Frame
Every 6 Months; Up to 5 years
Title
change in eGFR
Time Frame
Every 6 Months; Up to 5 years
Title
change in urine protein
Time Frame
Every 6 Months; Up to 5 years
Title
change in albumin levels
Time Frame
Every 6 Months; Up to 5 years
Title
change in Left Ventricular Mass Index (LVMi)
Time Frame
Every Year; Up to 5 years
Title
change in Fabry-Specific Pediatric Health and Pain Questionnaire (FPHPQ)scores
Description
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.
Time Frame
Every 3 months; Up to 5 years
Title
change in Pediatric and Quality of Life Inventory™ (PedsQL™) scores
Description
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.
Time Frame
Every 3 months; Up to 5 years

10. Eligibility

Sex
All
Minimum Age & Unit of Time
12 Years
Maximum Age & Unit of Time
17 Years
Accepts Healthy Volunteers
No
Eligibility 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
Facility Information:
Facility Name
University of South Florida
City
Tampa
State/Province
Florida
ZIP/Postal Code
33606
Country
United States
Facility Name
The Emory Clinic
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Facility Name
University of Minnesota Masonic Children's Hospital and Clinics
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55454
Country
United States
Facility Name
Cincinnati Children's Hospital
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Facility Name
Lysosomal & Rare Disorders Research & Treatment Center
City
Fairfax
State/Province
Virginia
ZIP/Postal Code
22030
Country
United States
Facility Name
Royal Free London NHS Foundation Trust
City
London
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
No

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Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease

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