A Study Evaluating Glycosphingolipid Clearance in Patients Treated With Agalsidase Alfa Who Switch to Agalsidase Beta

NCT01650779 | Completed Phase 4 Results

• 1 other identifier: AGAL19412
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 6

Brief Summary

This is an exploratory study to evaluate changes in glycosphingolipid levels and other (exploratory) Fabry disease parameters in male Fabry disease participants who were previously treated with agalsidase alfa (Replagal®) 0.2 milligram per kilogram (mg/kg) every two weeks (q2w) and who are being switched to agalsidase beta (Fabrazyme®) 1.0 mg/kg q2w.

Conditions

Fabry Disease

Keywords

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

Outcome Measures

Primary Outcomes (1)

• Percent Change From Baseline in Plasma Deacylated Globotriaosylceramide (Lyso-GL-3) at Month 2, 4 and 6

  Percent change from baseline = (\[post-baseline value minus baseline value\] divided by \[baseline value\]) multiplied by 100. For levels reported as below quantitative limit (BQL), the lower limit of quantitation (LLOQ) value was divided by 2 and used in the calculation to estimate values in samples that were BQL. The LLOQ for plasma lyso-GL-3 was 5.0 nanogram per milliliter (ng/mL). This study is exploratory because little is known about the dose-response of these biomarkers to enzyme replacement therapy (ERT) or about the clinical significance of the biomarkers.

  Baseline, Month 2, 4, 6

Secondary Outcomes (3)

• Percent Change From Baseline in Plasma Globotriaosylceramide (GL-3) at Month 2, 4 and 6

  Baseline, Month 2, 4, 6

• Percent Change From Baseline in Urine GL-3 at Month 2, 4, and 6

  Baseline, Month 2, 4, 6

• Percent Change From Baseline in Gastrointestinal (GI) Symptoms (Abdominal Pain, Abdominal Distention, and Bowel Irregularities) at Month 2, 4, and 6

  Baseline, Month 2, 4, 6

Study Arms (1)

Agalsidase beta

EXPERIMENTAL

Biological: Agalsidase beta

Interventions

Agalsidase beta BIOLOGICAL

Commercially available agalsidase beta 1.0 mg/kg administered as an intravenous infusion q2w up to Month 6.

Also known as: Fabrazyme®

Agalsidase beta

Eligibility Criteria

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

You may qualify if:

• The participant and/or his parent/legal guardian is willing and able to provide signed informed consent, and the participant, if less than (\<) 18 years of age, is willing to provide assent if deemed able to do so
• Participant is male and has been treated with agalsidase alfa at 0.2 mg/kg q2w for the 12 months prior to switching to agalsidase beta
• The participant has a confirmed diagnosis of Fabry disease by alfa-galactosidase A (alfa-GAL) activity and/or genotyping per local standards
• The participant when switched to agalsidase beta receives the labeled dose, that is, 0.9 to 1.1 mg/kg (1 mg/kg) q2w, and must be willing to maintain the labeled dose for the duration of the study

You may not qualify if:

• The participant is on dialysis or is post renal transplantation
• The participant is in end-stage cardiac failure
• The participant and/or his parent or legal guardian, in the opinion of the investigator, is unable to adhere to the requirements of the study
• The participant has been switched from agalsidase alfa to agalsidase beta and does not have historical blood and urine samples

Sponsors & Collaborators

• Genzyme, a Sanofi Company: lead

Study Sites (6)

Unknown Facility

Coral Springs, Florida, United States

Location

Unknown Facility

Decatur, Georgia, United States

Location

Unknown Facility

Baltimore, Maryland, United States

Location

Unknown Facility

Grand Rapids, Michigan, United States

Location

Unknown Facility

Hellertown, Pennsylvania, United States

Location

Unknown Facility

Fairfax, Virginia, United States

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

This is considered to be an exploratory study for the following reasons: it was based on a small number of participants and has been designed as an open-label, single-arm study as opposed to a two-arm crossover design.

Results Point of Contact

• Title: Trial Transparency Team
• Organization: Sanofi

Contact-us@sanofi.com

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: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 24, 2012
• First Posted: July 26, 2012
• Study Start: April 1, 2012
• Primary Completion: March 1, 2013
• Study Completion: March 1, 2013
• Last Updated: July 10, 2014
• Results First Posted: July 10, 2014

Record last verified: 2014-06

Locations

US (6)