AGAL01701: A Study of the Safety and Efficacy of Fabrazyme in Patients with Fabry Disease

This study is no longer recruiting patients.

Sponsored By:	Genzyme
Information Provided By:	Genzyme
ClinicalTrials.gov Identifier:	NCT00196716

Purpose

People with Fabry disease have an alteration in their genetic material (DNA) which causes a deficiency of the alpha-galactosidase A enzyme. This enzyme helps to break down and remove certain types of fatty substances called “glycolipids.” These glycolipids are normally present within the body in most cells. In people with Fabry disease, glycolipids build up in various tissues such as the liver, kidney, skin, and blood vessels because alpha-galactosidase A is not present, or is present in small quantities. The build up of glycolipid levels (also referred to as “globotriaosylceramide” or “GL-3”) in these tissues is thought to cause the clinical symptoms that are common to Fabry disease. Symptoms commonly appear during childhood with pain in the hands and feet. This trial is designed to evaluate the efficacy of a lower dose of Fabrazyme in patients who initially received 1.0 mg/kg every 2 weeks of Fabrazyme by investigating if the achieved clearance of glycosphingolipid deposits in the vascular endothelium of the kidney can be maintained at a lower dose.

Condition	Intervention	Phase
Fabry Disease	Drug: Fabrazyme (agalsidase beta)	Phase II, Phase III

Study Type: Interventional

Study Design: Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study

Official Title: A Multicenter, Open-Label Study of Low Dose Maintenance Treatment of Fabrazyme (Recombinant Human Alpha-Galactosidase A (r-h Alpha-GAL)) Replacement Therapy in Patients with Fabry Disease

Further Study Details:

Primary Outcomes: Evaluate 1.0 mg/kg of Fabrazyme followed by a maintenance dose of 0.3 mg/kg of Fabrazyme to clear and maintain clearance of globotriaosylceramide from the vascular endothelium of the kidney in patients with Fabry

Secondary Outcomes: Evaluate the efficacy of Fabrazyme to clear and maintain clearance of GL-3 from the skin and from other cell types in the kidney, to decrease and maintain plasma clearance of GL 3, and to evaluate clinical stabilization of the disease and pain.

Expected Total Enrollment: 21

Study start: June 2003; Study completion: August 2006

Last follow-up: April 2006; Data entry closure: August 2006

Eligibility

Ages Eligible for Study: 16 Years and above, Genders Eligible for Study: Male

Criteria

Inclusion Criteria:

Patients have to be male.

16 years of age or older and have clinical manifestations of Fabry disease.

All patients have to have a plasma αGAL activity of < 1.5 nmol/hr/mL or a documented leukocyte αGAL activity of < 4 nmol/hr/mg.

Exclusion Criteria:

A patient will be excluded if:

There is evidence of renal insufficiency, as defined by serum creatinine greater than or equal to 2.2 mg/dL (194.7 μmol/L) AND/OR has an estimated glomerular filtration rate (GFR) of <80 mL/min (using the equation derived from the Modification of Diet in Renal Disease Study (MDRD)

Has undergone kidney transplantation or is currently on dialysis

Has a clinically significant organic disease or an unstable condition (with the exception of symptoms relating to Fabry disease) that in the opinion of the Investigator would preclude participation in the trial

Has participated in a study employing an investigational drug within 30 days of the start of this trial.

Patients who received prior treatment with enzyme replacement therapy for Fabry disease or who are unable to comply with the clinical protocol are also excluded.

Location Information

Czech Republic
II. interní klinika 1. LF UK, Praha-2, 128 02, Czech Republic

Estonia
Tartu University Clinics, Department of Internal Medicine, Tartu, 51014, Estonia

Poland
Klinika Chorob Metabolicznych, Instytut "Pomnik-Centrum Zdrowia Dziecka", Warsaw, 04-736, Poland

Slovakia
Detská fakultná nemocnica Kramáre I. Interná klinika, Bratislava 37, 833 40, Slovakia

More Information

Fabrazyme® FDA-approved labeling information

Study ID Numbers: AGAL01701
Record first received: September 12, 2005
ClinicalTrials.gov Identifier: NCT00196716
Health Authority: Estonia: The State Agency of Medicine