Immune Response in Subjects With Fabry Disease Who Are Switching From Agalsidase Alfa to Agalsidase Beta

NCT01745185 | Completed

• 1 other identifier: 12-CFCT-03
• Study Type: observational
• Target: 30
• Locations: 1 country
• Sites: 1

Brief Summary

This study is a prospective active comparator study to assess the immune response elicited by human recombinant agalsidase therapy in subjects who are switching from agalsidase alfa to agalsidase beta with Fabry disease. Fabry disease is an X-linked lysosomal storage disorder, due to deficient alpha-galactosidase A activity. The progressive accumulation of globotriaosylceramide (GL-3) in the lysosomes of the vascular endothelial cells of multiple organ systems like the kidneys, heart, skin, and brain, leads to a microvascular disease. In Fabry disease, nephropathy dominates and renal function impairment occurs as a result of accumulation of GL-3 in renal cells

Conditions

Fabry Disease

Keywords

Fabry Disease; immunity; antibody

Outcome Measures

Primary Outcomes (1)

• Monitoring antibody formation against agalsidase alfa and beta

  Blood samples will be collected prior to infusion (screening \& month 12). At baseline, antibodies against agalsidase alfa and beta measured, and at 12 months, antibodies against agalsidase beta will be measured by ELISA technique and will be isotyped immunoglobulins (IgG, IgA, IgM, or IgE). Positive samples will subsequently tested for enzyme neutralizing activity using an in vitro assay. Antibody measurements will be done by Shire Human Genetics Therapies, INC.

  12 months

Secondary Outcomes (1)

• Measurement of plasma/urine Gb3 and plasma lyso-Gb3

  12 months

Study Arms (2)

Fabry disease switch group

Subjects will include individuals with Fabry disease who are switching from agalsidase alfa to agalsidase beta

Control Group

Controls will include individuals with Fabry disease who have only received agalsidase beta as treatment in their lifetime.

Eligibility Criteria

• Age: 7 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

30 subjects of (7 and above) who meet eligibility criteria.

You may qualify if:

• Confirmed diagnosis of Fabry disease
• Have been treated with ERT using recombinant human agalsidase A.

You may not qualify if:

• If the diagnosis of Fabry disease is not confirmed
• If the subject or guardian is not able to provide consent
• Any chronic immunosuppressive state or therapy such as patients on dialysis or post-transplantation immunosuppressive therapy.

Sponsors & Collaborators

• O & O Alpan LLC: lead

Study Sites (1)

O&O Alpan

Fairfax, Virginia, 22030, United States

Location

Related Publications (1)

• Benichou B, Goyal S, Sung C, Norfleet AM, O'Brien F. A retrospective analysis of the potential impact of IgG antibodies to agalsidase beta on efficacy during enzyme replacement therapy for Fabry disease. Mol Genet Metab. 2009 Jan;96(1):4-12. doi: 10.1016/j.ymgme.2008.10.004. Epub 2008 Nov 20.

  PMID: 19022694 BACKGROUND

Related Links

• O\&O Alpan LLC

Biospecimen

Retention: SAMPLES WITH DNA

Blood, Urine

MeSH Terms

Conditions

Fabry Disease

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

Study Officials

• Ozlem Goker-Alpan, MD

  O & O Alpan LLC

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 5, 2012
• First Posted: December 10, 2012
• Study Start: June 1, 2012
• Primary Completion: April 7, 2015
• Study Completion: August 7, 2016
• Last Updated: April 4, 2017

Record last verified: 2017-04

Locations

US (1)