Phase 2
Completed N=17
This Study is Designed to Evaluate PD/PK and Safety of Replagal Manufactured by Two Different Processes.
Source: ClinicalTrials.gov NCT01304277 ↗Enrolled (actual)
17
Serious AEs
11.8%
Results posted
Apr 2014
Primary outcomePrimary: Change From Baseline to Week 16 (EOS) in Urine Gb3 Levels — -28.68 (nmol/g creatinine)
Summary
This study is designed to evaluate safety and PK/PD in Canadian Fabry patients.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline to Week 16 (EOS) in Urine Gb3 Levels |
-28.68 | — |
| SECONDARY Change From Baseline to Week 16 (EOS) in Plasma Gb3 Levels |
0.170 | — |
| SECONDARY Dose-normalized Area Under the Concentration-time Curve From Time 0 to the Time of the Last Quantifiable Sample (AUClast/Dose) |
0.65; 1.05 | — |
| SECONDARY Dose-normalized AUC Extrapolated to Infinity (AUC∞/Dose) |
0.67; 1.07 | — |
| SECONDARY Dose-normalized Maximum Serum Concentration (Cmax/Dose) |
0.81; 1.05 | — |
| SECONDARY To Assess Safety and Tolerability by Anti-agalsidase Alfa Antibody Status (in Serum) at End of Study |
13; 2; 2 | — |
| SECONDARY Overall Summary of TEAEs by Treatment (Replagal RB and Replagal AF) |
9; 5; 5; 8; 12; 12 | — |
Eligibility Criteria
Inclusion Criteria
- The patient must be diagnosed with Fabry disease using the following criteria: The patient is a hemizygous male with Fabry disease as confirmed by a deficiency of α-galactosidase A activity measured in serum, leukocytes, or fibroblasts or has a confirmed mutation of the α-galactosidase A gene.
- Patient is male and between 18 and 65 years of age, inclusive.
- Patient must be willing to remain in the clinic as required by the study and comply with the procedures and evaluations of the study.
- At the time of confirmation of study eligibility visit, patients must have received at least 26 weeks of treatment with RB Replagal at a dose of 0.2 mg/kg administered IV EOW.
- Patient provides informed consent.
Patients who are naive to ERT:
- Treatment naive patients must have a pretreatment plasma Gb3 level above the normal range (if value is available).
Exclusion Criteria
- Patient is unable to be venipunctured and/or tolerate venous access.
- Patient has tested positive for anti-agalsidase alfa antibodies either at screening or confirmation of eligibility visit.
- Patient had pre-ERT plasma Gb3 levels within the normal range (if value is available).
- Patient is participating in any other Shire HGT investigational study.
- Patient is currently on dialysis, is expected to begin dialysis during the study, has received a kidney transplant, or is on the renal transplant waiting list.
- Patient is unable to comply with the protocol (eg, clinical relevant medical condition making implementation of the protocol difficult, unstable social situation, or otherwise unlikely to complete the study) or is, in the opinion of the Investigator, otherwise unsuited for the study.
- The patient is enrolled in another clinical study that involves clinical investigations or use of any investigational product (drug or device), except for the Canadian Fabry Disease Initiative, within 6 months prior to receiving the first dose of AF Replagal in this study or at any time during the study.
- The patient has previously received AF Replagal prior to study entry.
Data sourced from ClinicalTrials.gov (NCT01304277). 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.