A Study of Fabrazyme in Pediatric Patients With Fabry Disease

NCT ID: NCT00074958

Last Updated: 2015-04-02

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 16 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2002-10-31
• Study Completion Date: 2005-07-31

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.

Conditions

Fabry Disease

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Fabrazyme

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

Group Type: EXPERIMENTAL

Fabrazyme (agalsidase beta)

Intervention Type: BIOLOGICAL

1 mg/kg every 2 weeks

Interventions

Fabrazyme (agalsidase beta)

1 mg/kg every 2 weeks

Intervention Type: BIOLOGICAL

Other Intervention Names

r-hαGAL

Eligibility Criteria

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

• Minimum Eligible Age: 7 Years
• Maximum Eligible Age: 15 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Genzyme, a Sanofi Company

INDUSTRY

Sponsor Role: lead

Responsible Party

Genzyme Corporation

Principal Investigators

Medical Monitor

Role: STUDY_DIRECTOR

Genzyme, a Sanofi Company

Locations

University of Arizona

Tucson, Arizona, United States

Hopital Edouard Herriot

Lyon, , France

Hopital de la Timone Enfants

Marseille, , France

Hopital Europeen Georges Pompidou

Paris, , France

Instytut Pomnik Centrum Zdrowia Dziecka

Warsaw, , Poland

Royal Manchester Children's Hospital

Pendlebury, Manchester, United Kingdom

Great Ormond Street Hospital for Sick Children

London, , United Kingdom

Countries

United States; France; Poland; United Kingdom

References

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. doi: 10.1016/j.jpeds.2007.09.007. Epub 2007 Dec 3.

Reference Type: DERIVED

PMID: 18346516 (View on PubMed)

Other Identifiers

AGAL-016-01

Identifier Type: -

Identifier Source: org_study_id