Fabry: Renal Function During Long-term ERT by 51Cr-EDTA Clearance

NCT02969200 | Completed DMC

• 1 other identifier: FAB-KIDNEY
• Study Type: observational
• Target: 52
• Locations: 1 country
• Sites: 1

Brief Summary

The aim of this study was to investigate renal function decline by measured glomerular filtration rate (mGFR) in patients with FD during enzyme replacement therapy, and to explore the influence of age on renal function in FD.

Conditions

Fabry Disease

Keywords

Fabry Disease; Nephropathy; Measured GFR; Enzyme replacement therapy

Outcome Measures

Primary Outcomes (1)

• measured glomerular filtration rate

  GFR was measured at least once a year by the one sample 51Cr-ethylenendiaminetetra acetic acid (EDTA) clearance technique using two (for duplicate determination) plasma samples 200 min after the injection of 4 (3.8-4.2) MBq 51Cr-EDTA. In children (\< 15y) the injected 51Cr-activity was 3 MBq, and the blood-samples were collected 120 min after radiotracer injection. (\< 5y: 2 MBq).

  Assessed every 6-12 months; from baseline and up to 15 years

Secondary Outcomes (1)

• urinary protein excretion

  Assessed every 6-12 months; from baseline and up to 15 years

Interventions

Enzyme replacement therapy DRUG

All patients included in the study have received enzyme replacement therapy with either agalsidase alfa and/or agalsidase beta

Also known as: Fabrazyme (agalsidase beta), Replagal (agalsidase alfa)

Eligibility Criteria

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

Study Population

Patients with Fabry disease

You may qualify if:

• Genetically and/or enzymatically verified Fabry disease

You may not qualify if:

• End-stage renal disease prior to baseline (GFR \<15 ml/min/1.73m2, dialysis or renal transplant)
• Patient has not received enzyme replacement therapy during follow-up
• Patient has had less than 3 measurement of GFR during follow-up

Sponsors & Collaborators

• Ulla Feldt-Rasmussen: lead

Study Sites (1)

National University Hospital, Department of Medical Endocrinology

Copenhagen, DK-2100, Denmark

Location

MeSH Terms

Conditions

Fabry Disease; Kidney Diseases

Interventions

Enzyme Replacement Therapy; agalsidase beta; agalsidase alfa

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; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases

Intervention Hierarchy (Ancestors)

Enzyme Therapy; Drug Therapy; Therapeutics

Study Officials

• Ulla V Feldt-Rasmussen, MD, DMSc

  Department of Medical Endocrinology, Copenhagen University Hospital, Rigshospitalet

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Professor, Chief physician

Study Record Dates

• First Submitted: November 2, 2016
• First Posted: November 21, 2016
• Study Start: April 1, 2015
• Primary Completion: September 1, 2016
• Study Completion: December 9, 2016
• Last Updated: October 16, 2018

Record last verified: 2018-10

Data Sharing

• IPD Sharing: Will not share

Locations

DK (1)