A Study of Fabrazyme in Pediatric Patients With Fabry Disease - Tabular View

Tracking Information

First Received Date ^ICMJE

December 24, 2003

Last Updated Date

August 11, 2009

Start Date ^ICMJE

October 2002

Primary Completion Date

May 2005 (final data collection date for primary outcome measure)

Current Primary Outcome Measures ^ICMJE

Globotriaosylceramide (GL-3) Clearance in Capillary Endothelium in the Skin [ Time Frame: Baseline, Week 24 and Week 48 ] [ Designated as safety issue: No ]

Original Primary Outcome Measures ^ICMJE

Skin biopsy
Safety and efficacy without a placebo control group
Baseline assessments of plasma GL-3
ECG and GFR

Change History

Complete list of historical versions of study NCT00074958 on ClinicalTrials.gov Archive Site

Current Secondary Outcome Measures ^ICMJE

Plasma GL-3 [ Time Frame: Baseline, Week 24 and Week 48 ] [ Designated as safety issue: No ]

Original Secondary Outcome Measures ^ICMJE

Pain
Concomitant pain medication
GI symptoms
Autonomic neuropathy symptoms
Quality of life
Pharmacokinetics (Pk)

Descriptive Information

Brief Title ^ICMJE

A Study of Fabrazyme in Pediatric Patients With Fabry Disease

Official Title ^ICMJE

A Multi-center, Phase 2, Open-Label Study of Fabrazyme (Recombinant Human a-Galactosidase A) Replacement Therapy in Pediatric Patients With Fabry Disease

Brief Summary

People with Fabry disease have an alteration in their genetic material (DNA) which causes a deficiency of the a-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 a-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 study explored the safety, efficacy and pharmacokinetics of Fabrazyme in pediatric patients aged between 7 and 15 years.

Detailed Description

Study Phase

Phase II

Study Type ^ICMJE

Interventional

Study Design ^ICMJE

Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety Study

Condition ^ICMJE

Fabry Disease

Intervention ^ICMJE

Biological: Fabrazyme (agalsidase beta)

Study Arms / Comparison Groups

Experimental: 1.0 mg/kg of Fabrazyme given to the patients every 2 weeks

Publications *

Wraith JE, Tylki-Szymanska A, Guffon N, Lien YH, Tsimaratos M, Vellodi A, Germain DP. Safety and efficacy of enzyme replacement therapy with agalsidase beta: an international, open-label study in pediatric patients with Fabry disease. J Pediatr. 2008 Apr;152(4):563-70, 570.e1. Epub 2007 Dec 3.

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status ^ICMJE

Completed

Enrollment ^ICMJE

Completion Date

July 2005

Primary Completion Date

May 2005 (final data collection date for primary outcome measure)

Eligibility Criteria ^ICMJE

Inclusion criteria:

Patient or legal guardian must provide written informed consent
Patients must have a clinical diagnosis of Fabry disease and active Fabry disease (clinical signs and symptoms)
Patients must be at least 7 years of age but no older than 15 years of age at time of enrollment
Patients must be Tanner Stage ≤ III
Female patients must have a negative pregnancy test prior to each infusion and use a medically accepted form of contraception throughout the study

Exclusion Criteria:

Patient has a clinically significant organic disease (with the exception of symptoms relating to Fabry disease) that in the opinion of the investigator would preclude participation in the trial
Patient has participated in a study employing investigational drug within 30 days of the start of this study
Patient has received prior treatment with enzyme replacement therapy
Patient is unable to comply with the clinical protocol

Gender

Both

Ages

7 Years to 15 Years

Accepts Healthy Volunteers

Contacts ^ICMJE

Contact information is only displayed when the study is recruiting subjects

Location Countries ^ICMJE

United States, France, Poland, United Kingdom

Administrative Information

NCT ID ^ICMJE

NCT00074958

Responsible Party

Medical Monitor, Genzyme Corporation

Study ID Numbers ^ICMJE

AGAL-016-01

Study Sponsor ^ICMJE

Genzyme

Collaborators ^ICMJE

Investigators ^ICMJE

Study Director:

Medical Monitor

Genzyme

Information Provided By

Genzyme

Verification Date

April 2009

^ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP