Clinical trial NCT04049760: 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
Overall status: COMPLETED
Phase: PHASE3
Conditions: Fabry Disease
Keywords: Lysosomal disease
migalastat
Lead sponsor: Amicus Therapeutics
Sponsor class: INDUSTRY
First posted: 2019-08-08
Enrollment: 16
Sex: ALL
Minimum age (years): 12
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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


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
* If of reproductive potential, both male and female subjects agreed to use a medically accepted method of contraception throughout the duration of the study and for up to 30 days after their last dose of migalastat

Exclusion Criteria:

* Subject's last available estimated glomerular filtration rate (eGFR) in the previous study was \< 60 mL/min/1.73 m2
* Subject had advanced kidney disease requiring dialysis or kidney transplantation
* Subject received any investigational/experimental drug, biologic, or device within 30 days before baseline, with the exception of migalastat
* Subject anticipated starting gene therapy during the study period
* Subject had any intercurrent illness or condition at Visit 1 that may have precluded the subject from fulfilling the protocol requirements or suggested to the investigator that the potential subject may have an unacceptable risk by participating in this study
* Subject had a history of allergy or sensitivity to migalastat (including excipients) or other iminosugars (eg, miglustat, miglitol)
* Subject required treatment with Replagal® (agalsidase alfa) or Fabrazyme® (agalsidase beta)
* Subject required treatment with Glyset® (miglitol) or Zavesca® (miglustat)
* Female subject was pregnant or breast-feeding, or was planning to become pregnant during the study period
* In the opinion of the investigator, the subject and/or parent or legally-authorized representative was unlikely or unable to comply with the study requirements

Primary outcomes:
measure: Incidence of Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and AEs (Adverse Events) Leading to Discontinuation of Study Drug
timeFrame: Entire study
Secondary outcomes:
measure: Change From Baseline to Month 24 in Estimated Glomerular Filtration Rate (eGFR)
timeFrame: Baseline, Month 24
measure: Change From Baseline to Month 24 in Urine Protein Levels
timeFrame: Baseline, Month 24
measure: Change From Baseline to Month 24 in Urine Albumin
timeFrame: Baseline, Month 24
measure: Change From Baseline to Month 24 in Left Ventricular Mass Index (LVMi)
timeFrame: Baseline, Month 24
measure: Change From Baseline to Month 24 in Pediatric and Quality of Life Inventory™ (PedsQL™) Scores
timeFrame: Baseline, Month 24
measure: Change From Baseline to Month 24 in Fabry-Specific Pediatric Health and Pain Questionnaire (FPHPQ) Score for Pain Intensity
timeFrame: Baseline, Month 24
measure: Number of Subjects Who Experienced Sudden Onset of Pain As Assessed Using the Fabry-Specific Health and Pain Questionnaire (FPHPQ)
timeFrame: Month 24
measure: Change From Baseline to Month 24 in Plasma Levels of Lyso-Gb3
timeFrame: Baseline, Month 24
Other outcomes:
Locations:
city: Tampa
country: United States
facility: University of South Florida
state: Florida
city: Atlanta
country: United States
facility: The Emory Clinic
state: Georgia
city: Minneapolis
country: United States
facility: University of Minnesota Masonic Children's Hospital and Clinics
state: Minnesota
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital
state: Ohio
city: Fairfax
country: United States
facility: Lysosomal & Rare Disorders Research & Treatment Center
state: Virginia
city: London
country: United Kingdom
facility: Royal Free London NHS Foundation Trust
Study officials:
References: