AGAL01601: A Study of Fabrazyme in Pediatric Patients with Fabry Disease

This study is complete.

Sponsored By:	Genzyme
Information Provided By:	Genzyme
ClinicalTrials.gov Identifier:	NCT00074958

Purpose

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 will explore the safety and pharmacokinetics of Fabrazyme in pediatric patients aged between 7 and 15 years.

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

Study Type: Interventional

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

Official Title: A Multi-Center, Phase 2, Open-Label Study of Fabrazyme (Recombinant Human a-Galactosidase A) Replacement Therapy in Pediatric Patients with Fabry Disease

Further Study Details:

Primary Outcomes: Safety and efficacy without a placebo control group; Baseline assessments of plasma GL-3; Skin biopsy; ECG, echocardiography and GFR will be evaluated

Secondary Outcomes: Pain; Concomitant pain medication; GI symptoms; Autonomic neuropathy symptoms; Quality of life; Pharmacokinetics (Pk)

Total Enrollment: 16
Study Start: 2002-10; Study Completion: 2005-05

Eligibility

Ages Eligible for Study: 7 Years - 15 Years, Genders Eligible for Study: Both

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 between age 7 and 15

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

The patient must be wheelchair bound (unable to ambulate with the use of assistive devices, such as walker, cane, or crutches).

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

Location Information

Arizona
University of Arizona, Tucson, Arizona, 85724, United States

France
Hopital Europeen Georges Pompidou, Paris, Cedex 15, France
Hopital Edouard Herriot, Lyon, Cedex 03, France
Hopital de la Timone Enfants, Marseille, Cedex 05, France

Poland
Instytut Pomnik Centrum Zdrowia Dziecka, Warsaw, 04-730, Poland

United Kingdom
Great Ormond Street Hospital for Sick Children, London, WC1N 3JH, United Kingdom

More Information

Fabrazyme® FDA-approved labeling information

Study ID Numbers: AGAL01601
ClinicalTrials.gov Identifier: NCT00074958
Health Authority: United States: Food and Drug Administration