AGLU01602: A Study of the Safety and Efficacy of rhGAA in Patients With Infantile-Onset Pompe Disease

This study has been completed.

Sponsored By:	Genzyme
Information Provided By:	Genzyme
ClinicalTrials.gov Identifier:	NCT00059280

Purpose

Pompe disease (also known as glycogen storage disease type II, “GSD-II”) is caused by a deficiency of a critical enzyme in the body called acid alpha-glucosidase (GAA). Normally, GAA is used by the body's cells to break down glycogen (a stored form of sugar) within specialized structures called lysosomes. In patients with Pompe disease, an excessive amount of glycogen accumulates and is stored in various tissues, especially heart and skeletal muscle, which prevents their normal function. This study is being conducted to evaluate the safety and effectiveness of recombinant human acid alpha-glucosidase (rhGAA) as a potential enzyme replacement therapy for Pompe disease. Patients diagnosed with infantile-onset Pompe disease who are less than or equal to 6 months old will be studied.

Condition	Intervention	Phase
Glycogen Storage Disease Type II	Drug: recombinant human acid alpha-glucosidase (rhGAA)	Phase II, Phase III

Study Type: Interventional

Study Design: Treatment, Randomized, Open Label, Historical Control, Factorial Assignment, Safety/Efficacy Study

Official Title: An Open-Label, Multicenter, Multinational Study of the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of Recombinant Human Acid Alpha-Glucosidase Treatment in Patients Less Than 6 Months Old With Infantile-Onset Pompe Disease

Further Study Details:

Expected Total Enrollment: 16

Study start: April 2003

Study completion: December 2005

Eligibility

Ages Eligible for Study: up to 26 Weeks, Genders Eligible for Study: Both

Criteria

Inclusion Criteria:

The patient or the patient’s legal guardian(s) must provide written informed consent prior to any study-related procedures being performed;

The patient must have clinical symptoms (documented in his or her medical record) of infantile-onset Pompe disease. In addition, the patient must have:

The patient must be no older than 26 weeks and 0 days, when he/she receives the first dose of rhGAA;

The patient and his/her legal guardian(s) must have the ability to comply with the clinical protocol.

Exclusion Criteria:

Symptoms of respiratory insufficiency, including:

Major congenital abnormality;

Clinically significant organic disease (with the exception of symptoms relating to Pompe disease), including clinically significant cardiovascular, hepatic, pulmonary, neurologic, or renal disease, or other medical condition, serious intercurrent illness, or extenuating circumstance that, in the opinion of the Investigator, would preclude participation in the trial or potentially decrease survival;

Use of any investigational product within 30 days prior to study enrollment;

Received enzyme replacement therapy with GAA from any source.

Location Information

Florida
University of Florida College of Medicine, Gainesville, Florida, 32610-00266, United States

North Carolina
Duke University Medical Center, Durham, North Carolina, 27710, United States

Ohio
Children's Hospital Medical Center, Cincinnati, Ohio, 45229, United States

Utah
University of Utah Medical Center, Salt Lake City, Utah, 84132, United States

France
Pediatrique Hopital deBrousse, Lyon, France

Israel
Rambam Medical Center, Haifa, 31096, Israel

Taiwan
National Taiwan University Hospital, Taipei, 100, Taiwan

United Kingdom
Royal Manchester Children's Hospital, Manchester, United Kingdom

More Information

Myozyme® FDA-approved labeling information

Study ID Numbers: AGLU01602
ClinicalTrials.gov Identifier: NCT00059280
Health Authority:United States: Food and Drug Administration