Severe Renal Disease Study in Fabry Patients Treated With Fabrazyme

NCT00837824 | Terminated Phase 2 Results DMC

• 1 other identifier: AGAL-022-02
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 9

Brief Summary

This study was designed to determine appropriate treatment with Fabrazyme at a biweekly dose of either 1 mg/kg or 3 mg/kg in a population of patients with severe renal disease burden.

Conditions

Fabry Disease; Chronic Kidney Disease, Stage IV (Severe)

Keywords

Fabry Disease patients with Severe Renal Disease

Outcome Measures

Primary Outcomes (1)

• Time to Clinically Significant Progression of Cardiac Disease, Cerebrovascular Disease, and/or Death Among Fabry Patients With Severe Kidney Disease

  The trial was terminated early due to inadequate study design. During the study period of 7 months, only 1 patient had a clinical event, a stroke, in the Fabrazyme 1 mg/kg treatment arm. The time to event was determined from first dose of Fabrazyme to the date of event.

  7 months

Secondary Outcomes (1)

• Plasma Globotriaosylceramide (GL-3)

  Evaluated at Baseline, Month 3, and Final Visit

Study Arms (2)

Fabrazyme 1mg/kg every 2 weeks

EXPERIMENTAL

Fabrazyme 1.0 mg/kg every 2 weeks

Biological: Fabrazyme (agalsidase beta)

Fabrazyme 3mg/kg every 2 weeks

EXPERIMENTAL

Fabrazyme 3.0 mg/kg every 2 weeks

Biological: Fabrazyme (agalsidase beta)

Interventions

Fabrazyme (agalsidase beta) BIOLOGICAL

1.0 mg/kg every 2 weeks

Also known as: r-hαGAL

Fabrazyme 1mg/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:

• provided written informed consent prior to any study-related procedures being performed.
• be ≥16 years old.
• have a current diagnosis of Fabry disease (defined as abnormal α-galactosidase (α GAL) enzyme levels or Fabry genotype).
• have one of the following clinical conditions present at enrollment: serum creatinine level greater than 3.0 mg/dL (an average of two values at least one week apart), or be currently on dialysis, or be status post kidney transplant by greater than 3 months.
• have the ability to comply with the requirements of the protocol
• have a negative pregnancy test, if a female patient of childbearing potential. In addition, all female patients of childbearing potential must use a medically accepted method of contraception throughout the study.

You may not qualify if:

• if they had participated in a study employing an investigational drug within 30 days of the start of their participation in this trial.
• had previously received enzyme replacement therapy (ERT) for their Fabry disease.
• had diabetic nephropathy.
• were pregnant or lactating.
• were unwilling to comply with the requirements of the protocol.

Sponsors & Collaborators

• Genzyme, a Sanofi Company: lead
• CRL/Medinet: collaborator

Study Sites (9)

Unknown Facility

Farmington, Connecticut, United States

Location

Unknown Facility

Coral Springs, Florida, United States

Location

Unknown Facility

Kansas City, Kansas, United States

Location

Unknown Facility

Boston, Massachusetts, United States

Location

Unknown Facility

New York, New York, United States

Location

Unknown Facility

Rochester, New York, United States

Location

Unknown Facility

Pittsburgh, Pennsylvania, United States

Location

Unknown Facility

Houston, Texas, United States

Location

Unknown Facility

Milwaukee, Wisconsin, United States

Location

MeSH Terms

Conditions

Fabry Disease; Renal Insufficiency, Chronic

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; Renal Insufficiency; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Limitations and Caveats

The trial was terminated early due to inadequate study design, leading to small numbers of subjects analyzed.

Results Point of Contact

• Title: Genzyme MedInfo
• Organization: Genzyme Corporation

medinfo@genzyme.com

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 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY

Study Record Dates

• First Submitted: October 23, 2008
• First Posted: February 5, 2009
• Study Start: December 1, 2002
• Primary Completion: August 1, 2003
• Study Completion: August 1, 2003
• Last Updated: April 7, 2015
• Results First Posted: February 5, 2009

Record last verified: 2015-03

Locations

US (9)