--- title: A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease nct_id: NCT00081497 overall_status: COMPLETED phase: PHASE4 sponsor: Genzyme, a Sanofi Company study_type: INTERVENTIONAL primary_condition: Fabry Disease countries: United States, Canada, Czechia, Hungary, Poland, United Kingdom canonical_url: "https://parkinsonspathways.com/agent/trials/NCT00081497.md" clinicaltrials_gov: "https://clinicaltrials.gov/study/NCT00081497" ct_last_update_post_date: 2015-04-02 last_seen_at: "2026-05-12T06:54:01.885Z" source: ClinicalTrials.gov (mirrored, no enrichment) --- # A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease **Official Title:** Multi-Center, Open-Label Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease That Previously Participated in the AGAL-008-00 Study **NCT ID:** [NCT00081497](https://clinicaltrials.gov/study/NCT00081497) ## Key Facts - **Status:** COMPLETED - **Phase:** PHASE4 - **Study Type:** INTERVENTIONAL - **Target Enrollment:** 67 - **Lead Sponsor:** Genzyme, a Sanofi Company - **Conditions:** Fabry Disease - **Start Date:** 2004-01 - **Completion Date:** 2005-09 - **CT.gov Last Update:** 2015-04-02 ## 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. ## Eligibility - **Minimum age:** 16 Years - **Sex:** ALL - **Healthy Volunteers:** No ``` Inclusion Criteria: * 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 Exclusion Criteria: * 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 ``` ## Arms - **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. ## Interventions - **agalsidase beta** (BIOLOGICAL) — 1.0 mg/kg every 2 weeks ## Primary Outcomes - **Difference in Inverse Serum Creatinine Within Patients' Slopes Between the Placebo AGAL-008-00 (NCT00074984) and Fabrazyme AGAL02503 (NCT00081497) Periods** _(time frame: Placebo period AGAL-008-00 (up to 35 months) through Fabrazyme period AGAL02503 (18 months))_ — 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. ## Secondary Outcomes - **Serum Creatinine at Pre-Fabrazyme and 6, 12, and 18 Months** _(time frame: Pre-Fabrazyme, 6, 12, and 18 months)_ - **Estimated Glomerular Filtration Rate (eGFR) at Pre-Fabrazyme and 6, 12, and 18 Months** _(time frame: 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** _(time frame: Pre-Fabrazyme and 6, 12, and 18 months)_ - **Proteinuria at Pre-Fabrazyme and 6, 12, and 18 Months** _(time frame: Pre-Fabrazyme and 6, 12, and 18 months)_ ## Locations (25) - University of Alabama at Birmingham, Birmingham, Alabama, United States - Cedars-Sinai Medical Center, Los Angeles, California, United States - University of San Francisco, San Francisco, California, United States - University of Connecticut Health Partners, West Hartford, Connecticut, United States - Oncology Hematology Association, Coral Springs, Florida, United States - Emory University School of Medicine, Atlanta, Georgia, United States - Children's Memorial Hospital, Chicago, Illinois, United States - University of Kansas Medical Center, Kansas City, Kansas, United States - Gene Therapy Center - Department of Pediatrics and Institute of Human Genetics, Minneapolis, Minnesota, United States - Children's Hospital, Buffalo, New York, United States - Mount Sinai School of Medicine, New York, New York, United States - University of Rochester School of Medicine, Rochester, New York, United States - Duke University Medical Center, Durham, North Carolina, United States - Children's Hospital Medical Center, Cincinnati, Ohio, United States - Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States - University of Pittsburgh, Pittsburgh, Pennsylvania, United States - Baylor College of Medicine, Houston, Texas, United States - University of Washington School of Medicine, Seattle, Washington, United States - Queen Elizabeth II Health Center, Halifax, Nova Scotia, Canada - North York General Hospital, Toronto, Ontario, Canada - Hopital du Sacre-Coeur de Montreal, Montreal, Quebec, Canada - University Hospital, Prague, Czechia - Sopron Megyei Jogu Varos Erzsebet Korhaz, Sopron, Hungary - Klinika Chorob Metabolicznych Instytut, Warsaw, Poland - Hope Hospital, Manchester, United Kingdom ## Recent Field Changes (last 30 days) - `status.overallStatus` — added _(2026-05-12)_ - `status.primaryCompletionDate` — added _(2026-05-12)_ - `status.completionDate` — added _(2026-05-12)_ - `status.lastUpdatePostDate` — added _(2026-05-12)_ - `design.phases` — added _(2026-05-12)_ - `design.enrollmentCount` — added _(2026-05-12)_ - `eligibility.criteria` — added _(2026-05-12)_ - `eligibility.minAge` — added _(2026-05-12)_ - `eligibility.sex` — added _(2026-05-12)_ - `outcomes.primary` — added _(2026-05-12)_ - `outcomes.secondary` — added _(2026-05-12)_ - `armsInterventions.arms` — added _(2026-05-12)_ - `armsInterventions.interventions` — added _(2026-05-12)_ - `sponsor.lead` — added _(2026-05-12)_ - `results.hasResults` — added _(2026-05-12)_ - `locations.university of alabama at birmingham|birmingham|alabama|united states` — added _(2026-05-12)_ - `locations.cedars-sinai medical center|los angeles|california|united states` — added _(2026-05-12)_ - `locations.university of san francisco|san francisco|california|united states` — added _(2026-05-12)_ - `locations.university of connecticut health partners|west hartford|connecticut|united states` — added _(2026-05-12)_ - `locations.oncology hematology association|coral springs|florida|united states` — added _(2026-05-12)_ - `locations.emory university school of medicine|atlanta|georgia|united states` — added _(2026-05-12)_ - `locations.children's memorial hospital|chicago|illinois|united states` — added _(2026-05-12)_ - `locations.university of kansas medical center|kansas city|kansas|united states` — added _(2026-05-12)_ - `locations.gene therapy center - department of pediatrics and institute of human genetics|minneapolis|minnesota|united states` — added _(2026-05-12)_ - `locations.children's hospital|buffalo|new york|united states` — added _(2026-05-12)_ - `locations.mount sinai school of medicine|new york|new york|united states` — added _(2026-05-12)_ - `locations.university of rochester school of medicine|rochester|new york|united states` — added _(2026-05-12)_ - `locations.duke university medical center|durham|north carolina|united states` — added _(2026-05-12)_ - `locations.children's hospital medical center|cincinnati|ohio|united states` — added _(2026-05-12)_ - `locations.children's hospital of philadelphia|philadelphia|pennsylvania|united states` — added _(2026-05-12)_ - `locations.university of pittsburgh|pittsburgh|pennsylvania|united states` — added _(2026-05-12)_ - `locations.baylor college of medicine|houston|texas|united states` — added _(2026-05-12)_ - `locations.university of washington school of medicine|seattle|washington|united states` — added _(2026-05-12)_ - `locations.queen elizabeth ii health center|halifax|nova scotia|canada` — added _(2026-05-12)_ - `locations.north york general hospital|toronto|ontario|canada` — added _(2026-05-12)_ - `locations.hopital du sacre-coeur de montreal|montreal|quebec|canada` — added _(2026-05-12)_ - `locations.university hospital|prague||czechia` — added _(2026-05-12)_ - `locations.sopron megyei jogu varos erzsebet korhaz|sopron||hungary` — added _(2026-05-12)_ - `locations.klinika chorob metabolicznych instytut|warsaw||poland` — added _(2026-05-12)_ - `locations.hope hospital|manchester||united kingdom` — added _(2026-05-12)_ --- *Canonical: https://parkinsonspathways.com/agent/trials/NCT00081497.md* *Source data (authoritative): https://clinicaltrials.gov/study/NCT00081497* *This page is a raw mirror with no AI summary, no editorial enrichment, and no Parkinson's-specific filtering.*