A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants
ConditionFabry Disease
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age2-11
SponsorAmicus Therapeutics
About this trial
An open-label study to evaluate the safety, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of migalastat treatment in pediatric subjects 2 to \< 12 years of age with Fabry disease and with amenable GLA variants.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Male or female subjects, diagnosed with Fabry disease who are between ages 2 and < 12 years at randomization (subjects aged 11 years must have birthdays > 30 days after randomization)
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 has a GLA variant documented in his/her medical record that is amenable to migalastat prior to Visit 2.
Subject has not received ERT (eg, Replagal® [agalsidase alfa] or Fabrazyme® [agalsidase beta]) for at least 14 days prior to Baseline visit.
Disqualifiers
Has moderate or severe renal impairment (eGFR < 60 mL/min/1.73 m2 at Visit 1 [screening]).
Has advanced kidney disease requiring dialysis or kidney transplantation.
History of allergy or sensitivity to migalastat (including excipients) or other iminosugars (eg, miglustat, miglitol).
Has received any investigational/experimental drug, biologic, or device within 30 days or 5 half-lives of the investigational product (whichever is longer) before Visit 1 (screening).
Trial design
Design model
Single group
Treatments tested in this trial
Migalastat HCl 20 mg
DrugMigalastat will be supplied as 20-mg dispersible tablets. Migalastat 20-mg dispersible tablets contain 16 mg migalastat free base.
Treatment groups
8 Participants
are divided into 1 treatment groupGroup A: Migalastat HCl 20 mg Dispersible TabletsExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Safety: Incidence of TEAEs, SAEs, and AEs leading to discontinuation of study drug
Time frame
Day 1 (after dosing) through Month 12 and follow-up (30 days after last dose)2
Pharmacokinetics (PK): Maximum Observed Plasma Concentration (Cmax) of Migalastat
Time frame
0 to 12 hours postdose during the first month of study and trough samples at Months 6 and 123
Pharmacokinetics (PK): Minimum Observed Plasma Concentration (Cmin) of Migalastat
Time frame
0 to 12 hours postdose during the first month of study and trough samples at Months 6 and 124
Pharmacokinetics (PK): Area Under The Plasma Concentration-time Curve Over The Dosing Interval (AUCtau) of Migalastat
Time frame
0 to 12 hours postdose during the first month of study and trough samples at Months 6 and 12Secondary outcomes
1
Pharmacodynamic: Change in plasma levels of lyso-Gb3 and its analogs from baseline
Time frame
Baseline to Months 3, 6, and 12/ET2
Efficacy: Change in eGFR from baseline
eGFR is calculated using the modified Schwartz formula for creatinine clearance. eGFR will also be calculated separately using the CKD-EPI40 equation.
Time frame
Baseline to Months 1, 3, 6, and 12/ET3
Efficacy: Change in urine protein and albumin/microalbumin levels from baseline
Time frame
Baseline to Months 3, 6, and 12/ET4
Efficacy: Change in Left Ventricular Mass Index (LVMi) from baseline
Time frame
Baseline to Month 12/ETSponsors and contacts
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Source ClinicalTrials.gov