ClinicaliQ Trial Snapshot
- A Study of Migalastat in Pediatric Subjects (2 to — Recruiting • Phase III • Cardiology / Cardiovascular • NCT06904261.
- What is being tested: Migalastat, a pharmacological chaperone, is being evaluated for safety, pharmacokinetics, pharmacodynamics, and efficacy in treating paediatric Fabry disease patients aged 2 to <12 years who carry amenable GLA gene variants.
- Patient eligibility overview: The study includes children aged 2 to under 12 years with confirmed Fabry disease and specifically those with GLA variants known to respond to migalastat treatment (amenable variants), representing an understudied paediatric population.
Use This Page For
- Quick orientation before opening the registry record.
- Checking recruitment status, phase and sponsor at a glance.
- Connecting this trial to nearby guidelines, Drug Science and education.
What This Trial Is Studying
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 Snapshot
- * 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. * Subject has at least 1 documented complication (ie, historical or current laboratory abnormality or sign/symptom) of Fabry disease * If of reproductive potential, both male and female subjects agree 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.
Use the source registry for the full inclusion and exclusion criteria before discussing referral or enrolment.