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 : February 17, 2021
|Condition or disease||Intervention/treatment||Phase|
|Fabry Disease||Drug: migalastat HCl 150 mg||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||15 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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 :||June 1, 2022|
|Estimated Study Completion Date :||June 1, 2022|
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
- incidence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), and AEs leading to discontinuation of study drug [ Time Frame: Month 60 ]
- change in body weight in kilograms [ Time Frame: baseline over time; Up to 5 years ]
- change in height in centimeters [ Time Frame: baseline over time; Up to 5 years ]
- changes in Electrocardiogram (ECG) results [ Time Frame: baseline over time; Up to 5 years ]A 12-lead ECG will be obtained.
- 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.
- 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.
- incidence of concomitant medications use [ Time Frame: Every 1 Month; Up to 5 years ]
- change in plasma levels of lyso-Gb3 [ Time Frame: Every 6 Months; Up to 5 years ]
- change in eGFR [ Time Frame: Every 6 Months; Up to 5 years ]
- change in urine protein [ Time Frame: Every 6 Months; Up to 5 years ]
- change in albumin levels [ Time Frame: Every 6 Months; Up to 5 years ]
- change in Left Ventricular Mass Index (LVMi) [ Time Frame: Every Year; Up to 5 years ]
- 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.
- 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.
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
|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|
|Royal Free London NHS Foundation Trust|
|London, United Kingdom|