Phase 4
N=6
A Safety and Efficacy Study of Fabrazyme® Replacement Therapy in Patients With Cardiac Fabry Disease
Fabry Disease
Bottom Line
View on ClinicalTrials.gov: NCT00140621 ↗Enrolled (actual)
6
Serious AEs
16.7%
Results posted
Apr 2015
Primary outcome: Primary: Percent Change From Baseline in Interventricular Septum and Left Ventricular Posterior Wall Thickness at Week 156 — -3.31; -6.34 percent change
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 4
- Interventions
- Agalsidase beta (Drug)
- Age
- Adult · 20+ yrs
- Sex
- All
- Sponsor
- Genzyme, a Sanofi Company
- Primary completion
- Aug 2012
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percent Change From Baseline in Interventricular Septum and Left Ventricular Posterior Wall Thickness at Week 156 |
-3.31; -6.34 | — |
| PRIMARY Change From Baseline in Interventricular Septum and Left Ventricular Posterior Wall Thickness at Week 156 |
0.37; -0.33 | — |
| PRIMARY Percent Change From Baseline in Left Ventricular Mass (LVM) at Week 156 |
-4.14 | — |
| PRIMARY Change From Baseline in LVM at Week 156 |
22.70 | — |
| SECONDARY Number of Participants in Overall Cardiac Function Assessment and Clinical Symptoms at Week 156: Change From Baseline in Cardiac Function Test |
5; 0; 1 | — |
| SECONDARY Percent Change From Baseline in GL-3 Plasma Levels at Week 156 |
-18.91 | — |
| SECONDARY Change From Baseline in Short Form (36) Health Survey (SF-36) Scores at Week 156 |
6.868; 4.380 | — |
Summary
This is a multi-center, open label, phase IV study conducted to evaluate the efficacy and safety of agalsidase beta (Fabrazyme [recombinant form]) administered by intravenous drip infusion in participants with cardiac Fabry disease.
Participants participated for 4 weeks or less in the baseline period and 156 weeks for the treatment period.
Eligibility Criteria
Inclusion Criteria
- Participants definitively diagnosed with cardiac Fabry disease (who fulfill all of the following criteria)
- In the case of male participants, documented plasma or leukocyte alpha-galactosidase A (α-GAL) activity was no more than 20 percent (%) of normal value (except for heterozygous female participants)
- Left ventricular hypertrophy was noted.
- Accumulation of globotriaosylceramide (GL-3) in the myocardium or a genetic deficiency associated with α-GAL was confirmed
- Or in the case of heterozygous female participants, when the family (father or son) was diagnosed with Fabry disease. (Father or son was related by birth.)
- Without symptoms or signs of Fabry, such as acroparesthesia, angiokeratomas, abnormal sweating, pain of distal extremities, chronic abdominal pain/diarrhea and corneal opacities were observed, except for proteinuria sign.
- Participants with interventricular and posterior wall thickness of at least 13 millimeter (mm) on echocardiography within 3 months before signed date to informed consent
- Participants in whom cardiac function was rated as Class I or II according to the New York Heart Association (NYHA) classification when giving informed consent.
- Participants classification: inpatients and outpatients
- Participants who had given written informed consent before the study-related baseline tests.
Exclusion Criteria
- Participants with severe hypertension (for example, blood pressure more than or equal to 180 millimeter of mercury [mmHg] and/or blood pressure more than or equal to 110 mmHg in spite of adequate medication)
- Participants whose serum creatinine level was higher than the upper normal limit within 3 months (12 weeks) prior to giving informed consent.
- Participants who had undergone kidney transplantation or were currently on dialysis.
- Participants with any serious hepatic disorder. Participants who had abnormal hepatic function test values within 3 months (12 weeks) prior to giving informed consent (when either alanine aminotransferase [ALT] or aspartate aminotransferase [AST] level exceeded the value five times as high as the upper normal limit).
- Permanent pacemaker or defibrillator implanted participants
- Pregnant or lactating women
- Participants who had taken this drug for 6 months (26 weeks) or more before giving informed consent.
- Participants who had participated in a clinical study employing any other investigational product within 3 months prior to giving informed consent.
- Enzyme replacement therapy history, except for agalsidase beta
- Participants who were unwilling to comply with the requirements of the protocol.
- Others judged by the investigator or sub-investigator to be ineligible for the study
Data sourced from ClinicalTrials.gov (NCT00140621). 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.