Phase 3
Completed N=22
Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to <18 Years)
Source: ClinicalTrials.gov NCT03500094 ↗Enrolled (actual)
22
Serious AEs
4.8%
Results posted
Nov 2021
Primary outcomePrimary: Number Of Participants Who Experienced Treatment-related Treatment-emergent Adverse Events (TEAEs) — 5 Participants
◆ Published Evidence
No publication linked
No peer-reviewed publication reporting this trial's results has been linked yet. This can indicate results are unpublished — a known publication-bias signal. We re-check periodically.
Summary
This was an open-label study to evaluate the safety, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of migalastat treatment in pediatric participants 12 to <18 years of age with Fabry disease and amenable gene encoding α-galactosidase A (GLA) variants.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number Of Participants Who Experienced Treatment-related Treatment-emergent Adverse Events (TEAEs) |
5 | — |
| PRIMARY Pharmacokinetics (PK): Area Under The Plasma Concentration-time Curve Over The Dosing Interval (AUCtau) Of Migalastat |
8920; 8430; 8740 | — |
| PRIMARY PK: Maximum Observed Plasma Concentration (Cmax) Of Migalastat |
1220; 1160; 1200 | — |
| SECONDARY Change In eGFR From Baseline To Month 12 |
-1.6; -1.6 | — |
| SECONDARY Annualized Rate Of Change From Baseline |
-1.5 | — |
| SECONDARY Change From Baseline In Total Urine Protein And Urine Albumin Levels At Month 12 |
36.0; 36.2; 16.2; 15.6 | — |
| SECONDARY Change From Baseline In Left Ventricular Mass Index (LVMi) |
-3.9; 4.9; -4.4; 4.3 | — |
| SECONDARY Change In Plasma Levels Of Globotriaosylsphingosine (Lyso-Gb3) |
-14.0; 12.5; -14.0; 11.3 | — |
| SECONDARY FABPRO-GI And Pain Scores |
0.9; 0.4; 1.0; 0.4; 0.6; 1.2 | — |
| SECONDARY Patient's Global Impression Of Change (PGI-C) Scores |
12; 7; 0; 10; 8; 1 | — |
| SECONDARY Number Of Participants Who Experienced Sudden Onset Of Pain As Assessed Using The Fabry-Specific Pediatric Health And Pain Questionnaire (FPHPQ) |
4; 5; 4; 3 | — |
| SECONDARY Change From Baseline In FPHPQ Score For Pain Intensity |
0.4; 0.4 | — |
| SECONDARY Change From Baseline In Pediatric Quality Of Life (PedsQL) At Month 12 |
2.2; 3.1 | — |
Eligibility Criteria
Key Inclusion Criteria
- Willing and able to provide written consent or assent (participant and parent/legal guardian, as applicable)
- Male or female between 12 and <18 years of age diagnosed with Fabry disease
- Confirmed, amenable GLA variant
- Participant weighed at least 45 kg (99 pounds) at screening
- Participant had never been treated with ERT or had not received ERT for 14 days prior to screening
- Participant had at least 1 complication (such as, laboratory abnormality and/or sign/symptom) of Fabry disease
- Participant was able to swallow study medication whole
Key Exclusion Criteria
- Had moderate or severe renal impairment (estimated glomerular filtration rate (eGFR) <60 milliliter/minute/1.73 meter squared (m^2) at screening)
- Had advanced kidney disease requiring dialysis or kidney transplantation
- History of allergy or sensitivity to study medication (including excipients) or other iminosugars (for example, miglustat, miglitol)
- Had received any gene therapy at any time or anticipated starting gene therapy during the study period
- Required treatment with Glyset (miglitol) and/or Zavesca (miglustat) within 6 months before screening or throughout the study
- Required treatment with Replagal (agalsidase alfa), or Fabrazyme (agalsidase beta) within 14 days before screening or throughout the study
- Participant was treated or had 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 have precluded the participant from fulfilling the protocol requirements or suggested to the investigator that the potential participant may have had an unacceptable risk by participating in this study
- Pregnant or breast-feeding or planned to become pregnant during the study period
- Otherwise unsuitable for the study in the opinion of the investigator
Data sourced from ClinicalTrials.gov (NCT03500094). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.