A Safety and Efficacy Study of Fabrazyme® Replacement Therapy in Patients With Cardiac Fabry Disease

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT00140621
• Date of registration: 30/08/2005
• Prospective Registration: No
• Primary sponsor: Genzyme, a Sanofi Company
• Public title: A Safety and Efficacy Study of Fabrazyme® Replacement Therapy in Patients With Cardiac Fabry Disease
• Scientific title: A Multicenter Open-label Study of the Safety and Efficacy of a-galactosidase A (R-h a-GAL) Replacement Therapy in Patients With Cardiac Fabry Disease
• Date of first enrolment: July 2005
• Target sample size: 6
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00140621
• Study type: Interventional
• Phase: Phase 4

Countries of recruitment

Japan

Contacts

• Name: Medical Monitor
• Affiliation: Genzyme, a Sanofi Company

Key inclusion & exclusion 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 (a-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 a-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



Age minimum: 20 Years
Age maximum: 64 Years
Gender: All

Health Condition(s) or Problem(s) studied

Fabry Disease

Intervention(s)

Drug: Agalsidase beta

Primary Outcome(s)

Change From Baseline in Interventricular Septum and Left Ventricular Posterior Wall Thickness at Week 156 [Time Frame: Baseline to Week 156]

Percent Change From Baseline in Interventricular Septum and Left Ventricular Posterior Wall Thickness at Week 156 [Time Frame: Baseline to Week 156]

Percent Change From Baseline in Left Ventricular Mass (LVM) at Week 156 [Time Frame: Baseline to Week 156]

Change From Baseline in LVM at Week 156 [Time Frame: Baseline to Week 156]

Secondary Outcome(s)

Change From Baseline in Short Form (36) Health Survey (SF-36) Scores at Week 156 [Time Frame: Baseline to Week 156]

Number of Participants in Overall Cardiac Function Assessment and Clinical Symptoms at Week 156: Change From Baseline in Cardiac Function Test [Time Frame: Baseline to Week 156]

Percent Change From Baseline in GL-3 Plasma Levels at Week 156 [Time Frame: Baseline to Week 156]

Secondary ID(s)

AGAL03204

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 15/04/2015
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00140621