Fabry DiseaseNCT02969200
Fabry: Renal Function During Long-term ERT by 51Cr-EDTA Clearance
An observational study of Fabry Disease, run by Ulla Feldt-Rasmussen at 1 site in Denmark.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Fabry Disease
- What is being tested?
- Enzyme replacement therapy
- How many people
- 52 participants
- Where
- 1 siteDenmark
- Study dates
- Apr 2015to 9 Dec 2016
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients with Fabry disease
Who may be able to join
- Genetically and/or enzymatically verified Fabry disease
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
Denmark1 site
National University Hospital, Department of Medical Endocrinology
Copenhagen, DK-2100
About this study
Official title: Fabry Disease: Renal Function During Long-term Enzyme Replacement Therapy Evaluated by Gold Standard GFR 51Cr-EDTA Clearance
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.
Read the detailed description
Nephropathy is common in Fabry disease (FD). Renal function decline is often the first sign of major organ involvement, sometimes progressing to end-stage renal failure. Available studies of renal function during enzyme replacement therapy have shown inconsistent results, and are based on different composition of patient materials and follow-up time.
Most investigations have used estimated glomerular filtration rate (eGFR) for evaluating renal function. GFR is an important indicator of renal function. eGFR based on a serum creatinine measurement is most commonly used in FD. However, this method has been shown to be unreliable, as serum creatinine levels are influenced by other factors than renal function such as ethnic group, muscle mass, age, hydration and diet. Performance of eGFR in detecting minor changes in renal function is poor. A 10 year old review on renal function evaluation in patients with FD recommended the use of GFR based on an exogenous marker, e.g. Cr-EDTA. Nevertheless, only few studies have used mGFR for evaluation of renal function and to our knowledge, the present study is the first to describe the rate of renal function decline with consecutive mGFR values in a nationwide population of patients with FD.
Renal function declines with age in renal healthy individuals. To our knowledge, the present study is the first to age-standardize renal function in patients with FD to adjust for age-dependent renal deterioration.
- Conditions
- Fabry Disease
- Being tested
- Enzyme replacement therapy
- Keywords
- Fabry Disease, Nephropathy, Measured GFR, Enzyme replacement therapy
- Medical subject headings
- Fabry Disease, Kidney Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Enzyme replacement therapyDrug
Also called Fabrazyme (agalsidase beta), Replagal (agalsidase alfa)
All patients included in the study have received enzyme replacement therapy with either agalsidase alfa and/or agalsidase beta
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: Assessed every 6-12 months; from baseline and up to 15 years
Secondary outcomes (1)
urinary protein excretion
Repeated twenty-four hour urine samples were collected by patients at home, the last 24 hours before coming to the hospital. These samples were analysed for albumin, creatinine and protein. Furthermore spot urine samples were applied and analysed for albumin, creatinine and protein. Albumin-creatinine-ratio was calculated and abnormal values were defined as > 30 mg/g. Urine protein- and albumin values below detection limit (< 0.04 g/L and < 3 mg/L, respectively) were converted to zero for statistical analyses.
Measured: Assessed every 6-12 months; from baseline and up to 15 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Nov 2016 (estimated)
- Study startApr 2015
- Primary completionSep 2016
- Study completion9 Dec 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Fabry DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 52 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ulla Feldt-Rasmussen
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Ulla Feldt-Rasmussen, Copenhagen University Hospital, Rigshospitalet
- Investigators
- Ulla V Feldt-Rasmussen, MD, DMSc, Principal investigator, Department of Medical Endocrinology, Copenhagen University Hospital, Rigshospitalet
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02969200
- Sponsor's protocol ID
- FAB-KIDNEY
- Status last verified
- Oct 2018
- First submitted
- 2 Nov 2016
- Last updated
- 16 Oct 2018
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Oct 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Fabry Disease: Renal Function During Long-term Enzyme Replacement Therapy Evaluated by Gold Standard GFR 51Cr-EDTA Clearance
- ClinicalTrials.gov ID
- NCT02969200
- Acronym / sponsor ID
- FAB-KIDNEY
- Conditions
- Fabry Disease
- Keywords
- Fabry Disease, Nephropathy, Measured GFR, Enzyme replacement therapy
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 52 participants (actual)
- Lead sponsor
- Ulla Feldt-RasmussenUniversity, hospital or other
- Study start
- Apr 2015
- Primary completion
- Sep 2016 (actual)
- Study completion
- 9 Dec 2016 (actual)
- First posted
- 21 Nov 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Nephropathy is common in Fabry disease (FD). Renal function decline is often the first sign of major organ involvement, sometimes progressing to end-stage renal failure. Available studies of renal function during enzyme replacement therapy have shown inconsistent results, and are based on different composition of patient materials and follow-up time.
Most investigations have used estimated glomerular filtration rate (eGFR) for evaluating renal function. GFR is an important indicator of renal function. eGFR based on a serum creatinine measurement is most commonly used in FD. However, this method has been shown to be unreliable, as serum creatinine levels are influenced by other factors than renal function such as ethnic group, muscle mass, age, hydration and diet. Performance of eGFR in detecting minor changes in renal function is poor. A 10 year old review on renal function evaluation in patients with FD recommended the use of GFR based on an exogenous marker, e.g. Cr-EDTA. Nevertheless, only few studies have used mGFR for evaluation of renal function and to our knowledge, the present study is the first to describe the rate of renal function decline with consecutive mGFR values in a nationwide population of patients with FD.
Renal function declines with age in renal healthy individuals. To our knowledge, the present study is the first to age-standardize renal function in patients with FD to adjust for age-dependent renal deterioration.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients with Fabry disease
Key inclusion criteria (1 listed)
- Genetically and/or enzymatically verified Fabry disease
Key exclusion criteria (3 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Enzyme replacement therapyAlso: Fabrazyme (agalsidase beta), Replagal (agalsidase alfa)All patients included in the study have received enzyme replacement therapy with either agalsidase alfa and/or agalsidase beta | Drug | – |
Primary outcome
measured glomerular filtration rate
Time frame: Assessed every 6-12 months; from baseline and up to 15 years
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).
1 secondary outcome
urinary protein excretion
Time frame: Assessed every 6-12 months; from baseline and up to 15 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Oct 2018.
- Individual participant dataSharing planned: No
Related trials for Fabry Disease
RecruitingObservational study
A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding
Fabry Disease
RecruitingObservational study
A Study of Patients With Fabry Disease (US Specific)
Fabry Disease
Active, not recruiting
Evaluation of the Effect of a Low-FODMAP Diet on Gastrointestinal Symptoms and Inflammatory Markers in Fabry Patients
Fabry Disease
RecruitingPhase 3
A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants
Fabry Disease
RecruitingObservational study
Fabry Disease Registry & Pregnancy Sub-registry
Fabry Disease
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Ulla V Feldt-Rasmussen, MD, DMSc principal investigator
Department of Medical Endocrinology, Copenhagen University Hospital, Rigshospitalet
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Fabry Disease, run by Ulla Feldt-Rasmussen at 1 site in Denmark.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 16 Oct 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Denmark. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
measured glomerular filtration rate (time frame: Assessed every 6-12 months; from baseline and up to 15 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Oct 2018.