A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease

NCT00081497 | Completed Phase 4 Results

• 1 other identifier: AGAL02503
• Study Type: interventional
• Target: 67
• Locations: 6 countries
• Sites: 25

Brief Summary

People with Fabry Disease have an alteration in their genetic material (DNA) which causes a deficiency of the alpha-galactosidase A enzyme. Fabrazyme (agalsidase beta) is a drug that helps to break down and removes certain types of fatty substances called "glycolipids". These glycolipids are normally present within the body in most cells. In 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 (globatriaosylceramide or GL-3) levels in these tissues in particular is thought to cause the clinical symptoms that are common to Fabry disease. This study analyzed the safety and efficacy of Fabrazyme in the treatment of patients with Fabry disease that previously participated in the AGAL-008-00 (NCT0074984) study.

Conditions

Fabry Disease

Keywords

alpha-galactosidase A; a-GAL; r-haGAL; Fabry; GL-3; Fabrazyme

Outcome Measures

Primary Outcomes (1)

• Difference in Inverse Serum Creatinine Within Patients' Slopes Between the Placebo AGAL-008-00 (NCT00074984) and Fabrazyme AGAL02503 (NCT00081497) Periods

  The primary efficacy analysis was the summary of change in slope of inverse serum creatinine for Placebo/Fabrazyme patients in the Intent to Treat (ITT) Population. It compared the placebo period slope with the Fabrazyme period slope.

  Placebo period AGAL-008-00 (up to 35 months) through Fabrazyme period AGAL02503 (18 months)

Secondary Outcomes (4)

• Serum Creatinine at Pre-Fabrazyme and 6, 12, and 18 Months

  Pre-Fabrazyme, 6, 12, and 18 months

• Estimated Glomerular Filtration Rate (eGFR) at Pre-Fabrazyme and 6, 12, and 18 Months

  Pre-Fabrazyme, 6, 12, and 18 months

• Plasma Globotriaosylceramide (GL-3) (Normal Plasma GL-3 Level is ≤ 7.03 µg/mL) at Pre-Fabrazyme and 6, 12, and 18 Months

  Pre-Fabrazyme and 6, 12, and 18 months

• Proteinuria at Pre-Fabrazyme and 6, 12, and 18 Months

  Pre-Fabrazyme and 6, 12, and 18 months

Study Arms (1)

Fabrazyme 1.0 mg/kg every 2 weeks

EXPERIMENTAL

This is an open-label extension study to AGAL-008-00 (NCT00074984) and all patients received Fabrazyme treatment.

Biological: agalsidase beta

Interventions

agalsidase beta BIOLOGICAL

1.0 mg/kg every 2 weeks

Also known as: Fabrazyme, r-hαGAL

Fabrazyme 1.0 mg/kg every 2 weeks

Eligibility Criteria

• Age: 16 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients must have successfully completed the previous double-blind study AGAL-008-00 (NCT00074984)
• Patients must provide written informed consent prior to study participation
• Female patients of childbearing potential must have a negative pregnancy test prior to each dosing and all female patients must use a medically accepted form of contraception throughout the study

You may not qualify if:

• The patient was unable to complete AGAL-008-00 (NCT00074984)
• The patient has undergone kidney transplantation or is currently on dialysis
• The patient has diabetes mellitus or presence of confounding renal disease
• The patient has a clinically significant organic disease or an unstable condition that precludes participation
• The patient is unwilling to comply with the protocol requirements

Sponsors & Collaborators

• Genzyme, a Sanofi Company: lead

Study Sites (25)

University of Alabama at Birmingham

Birmingham, Alabama, 35294-0006, United States

Location

Cedars-Sinai Medical Center

Los Angeles, California, 90048, United States

Location

University of San Francisco

San Francisco, California, 94143, United States

Location

University of Connecticut Health Partners

West Hartford, Connecticut, 06119, United States

Location

Oncology Hematology Association

Coral Springs, Florida, 33065, United States

Location

Emory University School of Medicine

Atlanta, Georgia, 30322, United States

Location

Children's Memorial Hospital

Chicago, Illinois, 60614, United States

Location

University of Kansas Medical Center

Kansas City, Kansas, 66160-7233, United States

Location

Gene Therapy Center - Department of Pediatrics and Institute of Human Genetics

Minneapolis, Minnesota, 55455, United States

Location

Children's Hospital

Buffalo, New York, 14209, United States

Location

Mount Sinai School of Medicine

New York, New York, 10029, United States

Location

University of Rochester School of Medicine

Rochester, New York, 14642, United States

Location

Duke University Medical Center

Durham, North Carolina, 27710, United States

Location

Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

University of Pittsburgh

Pittsburgh, Pennsylvania, 15261, United States

Location

Baylor College of Medicine

Houston, Texas, 77030, United States

Location

University of Washington School of Medicine

Seattle, Washington, 98195, United States

Location

Queen Elizabeth II Health Center

Halifax, Nova Scotia, B3H 1V8, Canada

Location

North York General Hospital

Toronto, Ontario, M2K 1E1, Canada

Location

Hopital du Sacre-Coeur de Montreal

Montreal, Quebec, H4J 1C5, Canada

Location

University Hospital

Prague, Czechia

Location

Sopron Megyei Jogu Varos Erzsebet Korhaz

Sopron, 9400, Hungary

Location

Klinika Chorob Metabolicznych Instytut

Warsaw, 04-730, Poland

Location

Hope Hospital

Manchester, M6 8HD, United Kingdom

Location

MeSH Terms

Conditions

Fabry Disease

Interventions

agalsidase beta

Condition Hierarchy (Ancestors)

Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Cerebral Small Vessel Diseases; Cerebrovascular Disorders; Vascular Diseases; Cardiovascular Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolism, Inborn Errors; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Limitations and Caveats

No valid conclusions can be made from the predefined primary efficacy analysis. The sponsor believes that it is more statistically appropriate to compare placebo patients with Fabrazyme patients as they were randomized in the original Phase 4 trial.

Results Point of Contact

• Title: Genzyme Medical Information
• Organization: Genzyme Corporation

800-745-4447

Study Officials

• Medical Monitor

  Genzyme, a Sanofi Company

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY

Study Record Dates

• First Submitted: April 14, 2004
• First Posted: April 16, 2004
• Study Start: January 1, 2004
• Primary Completion: September 1, 2005
• Study Completion: September 1, 2005
• Last Updated: April 2, 2015
• Results First Posted: August 18, 2010

Record last verified: 2015-03

Locations

PL (1); GB (1); CZ (1); CA (3); US (18); HU (1)