A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease

Genzyme, a Sanofi Company67 enrolled

Overview

People with Fabry Disease have an alteration in their genetic material (DNA) which causes a deficiency of the alpha-galactosidase A enzyme. Fabrazyme (agalsidase beta) is a drug that helps to break down and removes certain types of fatty substances called "glycolipids". These glycolipids are normally present within the body in most cells. In Fabry disease, glycolipids build up in various tissues such as the liver, kidney, skin, and blood vessels because a-galactosidase A is not present, or is present in small quantities. The build up of glycolipid (globatriaosylceramide or GL-3) levels in these tissues in particular is thought to cause the clinical symptoms that are common to Fabry disease. This study analyzed the safety and efficacy of Fabrazyme in the treatment of patients with Fabry disease that previously participated in the AGAL-008-00 (NCT0074984) study.

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Fabry Disease

Interventions

Eligibility

Sex: ALLMin age: 16 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Patients must have successfully completed the previous double-blind study AGAL-008-00 (NCT00074984) * Patients must provide written informed consent prior to study participation * Female patients of childbearing potential must have a negative pregnancy test prior to each dosing and all female patients must use a medically accepted form of contraception throughout the study Exclusion Criteria: * The patient was unable to complete AGAL-008-00 (NCT00074984) * The patient has undergone kidney transplantation or is currently on dialysis * The patient has diabetes mellitus or presence of confounding renal disease * The patient has a clinically significant organic disease or an unstable condition that precludes participation * The patient is unwilling to comply with the protocol requirements

Locations (25)

University of Alabama at Birmingham

Birmingham, Alabama, United States

Cedars-Sinai Medical Center

Los Angeles, California, United States

University of San Francisco

San Francisco, California, United States

University of Connecticut Health Partners

West Hartford, Connecticut, United States

Oncology Hematology Association

Coral Springs, Florida, United States

Emory University School of Medicine

Atlanta, Georgia, United States

Children's Memorial Hospital

Chicago, Illinois, United States

University of Kansas Medical Center

Kansas City, Kansas, United States

Gene Therapy Center - Department of Pediatrics and Institute of Human Genetics

Minneapolis, Minnesota, United States

Children's Hospital

Buffalo, New York, United States

...and 15 more locations

Outcomes

Primary Outcomes

Difference in Inverse Serum Creatinine Within Patients' Slopes Between the Placebo AGAL-008-00 (NCT00074984) and Fabrazyme AGAL02503 (NCT00081497) Periods

The primary efficacy analysis was the summary of change in slope of inverse serum creatinine for Placebo/Fabrazyme patients in the Intent to Treat (ITT) Population. It compared the placebo period slope with the Fabrazyme period slope.

Time frame: Placebo period AGAL-008-00 (up to 35 months) through Fabrazyme period AGAL02503 (18 months)

Secondary Outcomes

Serum Creatinine at Pre-Fabrazyme and 6, 12, and 18 Months

Pre-Fabrazyme=baseline visit of AGAL-008-00 (NCT00074984) for Fabrazyme patients; assessment prior to open-label for placebo patients who transitioned to Fabrazyme in AGAL-008-00 (NCT00074984); assessment prior to first Fabrazyme infusion in AGAL02503 (NCT00081497) for placebo patients who did not transition to Fabrazyme in AGAL-008-00 (NCT00074984).

Time frame: Pre-Fabrazyme, 6, 12, and 18 months

Estimated Glomerular Filtration Rate (eGFR) at Pre-Fabrazyme and 6, 12, and 18 Months

Pre-Fabrazyme=baseline visit of AGAL-00-800 (NCT00074984) for Fabrazyme patients; assessment prior to open-label for placebo patients who transitioned to Fabrazyme in AGAL-008-00 (NCT00074984); assessment prior to first Fabrazyme infusion in AGAL02503 (NCT00081497) for placebo patients who did not transition to Fabrazyme in AGAL-008-00 (NCT00074984).

Time frame: Pre-Fabrazyme, 6, 12, and 18 months

Plasma Globotriaosylceramide (GL-3) (Normal Plasma GL-3 Level is ≤ 7.03 µg/mL) at Pre-Fabrazyme and 6, 12, and 18 Months

Pre-Fabrazyme=baseline visit of AGAL00800 for Fabrazyme patients; assessment prior to open-label for placebo patients who transitioned to Fabrazyme in AGAL00800; assessment prior to first Fabrazyme infusion in AGAL02503 for placebo patients who did not transition to Fabrazyme in AGAL00800.

Time frame: Pre-Fabrazyme and 6, 12, and 18 months

Proteinuria at Pre-Fabrazyme and 6, 12, and 18 Months

Time frame: Pre-Fabrazyme and 6, 12, and 18 months