Severe Renal Disease Study in Fabry Patients Treated With Fabrazyme

Genzyme

Status and phase

Terminated

Phase 2

Conditions

Fabry Disease

Chronic Kidney Disease, Stage IV (Severe)

Treatments

Biological: Fabrazyme (agalsidase beta)

Study type

Interventional

Funder types

Industry

Other

Identifiers

NCT00837824

AGAL-022-02

Details and patient eligibility

About

This study was designed to determine appropriate treatment with Fabrazyme at a biweekly dose of either 1 mg/kg or 3 mg/kg in a population of patients with severe renal disease burden.

Enrollment

20 patients

Sex

All

Ages

16+ years old

Volunteers

No Healthy Volunteers

Inclusion criteria

provided written informed consent prior to any study-related procedures being performed.
be ≥16 years old.
have a current diagnosis of Fabry disease (defined as abnormal α-galactosidase (α GAL) enzyme levels or Fabry genotype).
have one of the following clinical conditions present at enrollment: serum creatinine level greater than 3.0 mg/dL (an average of two values at least one week apart), or be currently on dialysis, or be status post kidney transplant by greater than 3 months.
have the ability to comply with the requirements of the protocol
have a negative pregnancy test, if a female patient of childbearing potential. In addition, all female patients of childbearing potential must use a medically accepted method of contraception throughout the study.

Exclusion criteria

if they did not meet the specific inclusion criteria.
if they had participated in a study employing an investigational drug within 30 days of the start of their participation in this trial.
had previously received enzyme replacement therapy (ERT) for their Fabry disease.
had diabetic nephropathy.
were pregnant or lactating.
were unwilling to comply with the requirements of the protocol.