Fabry DiseaseNCT02090608
Paricalcitol in Fabry Disease
A study testing Paricalcitol in people with Fabry Disease and Proteinuria, run by Federico II University at 1 site in Italy.
Trial summary
Proteinuria is the predominant risk factor for renal disease progression in Fabry disease (FD). When urine protein excretion is controlled to <0.50 g/24 hr, the rate loss of glomerular filtration rate (GFR) is not significantly different from 0. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Fabry Disease, Proteinuria
- What is being tested?
- Paricalcitol
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 14 participants
- Where
- 1 siteItaly
- Study dates
- Mar 2012to Dec 2013
- Phase
- Interventional
- 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 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- genetically proven FD
- stable dose of ERT for at least 12 months
- stable dose of ACEi or ARB titrated to maximum tolerated dosage for at least 6 months
- persistent proteinuria >0.50 g/24 h despite the use of ERT and ACEi/ARBs in 2 consecutive samples within 12 weeks
Who cannot join
- steroid/immunosuppressive treatment or glomerular filtration rate change >30% in the past 3 months
- PTH levels <20 pg/mL
- serum phosphorus >5.0 mg/dL
- serum calcium (adjusted for albumin) >10.0 mg/dL
- active malignancy.
Trial locations and contacts
1 site in 1 country.
Italy1 site
federico II university, department of nephrology
Naples, Naples, 80129
About this study
Proteinuria is the predominant risk factor for renal disease progression in Fabry disease (FD). When urine protein excretion is controlled to <0.50 g/24 hr, the rate loss of glomerular filtration rate (GFR) is not significantly different from 0. However, enzyme replacement therapy (ERT) alone does not decrease proteinuria and it has been recommended that patients receiving ERT also receive anti Renin-Angiotensin-System (RAS) therapy. Emerging evidences show that paricalcitol (PCT) reduces proteinuria in presence of intensified inhibition of RAS; however, there is no evidence in FD. The aim of this study is to evaluate the antiproteinuric effect of PCT in FD patients with proteinuria >0.50 g/24 hr persisting despite the ERT and anti-RAS therapy titrated to maximum tolerated dosage.
- Conditions
- Fabry Disease, Proteinuria
- Being tested
- Paricalcitol
- Medical subject headings
- Fabry Disease, Proteinuria
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ParicalcitolDrug
Paricalcitol was administered at the dose of 1 mcg/die
| Study group | Role | What they receive |
|---|---|---|
| Paricalcitol | Experimental | In patients identified by the inclusion criteria, data will be collected at baseline , during administration of oral Paricalcitol (PCT) (after 1, 3 and 6 months), and three months after PCT withdrawal. PCT will administered at dosage of 1 mcg/day; this dosage was chosen as it is not associated with excessive decline of parathyroid hormone (PTH) levels in most patients Receives: Drug: Paricalcitol |
What is being measured
The primary outcome is the main question the study is designed to answer.
Effect of paricalcitol on proteinuria reduction
Fourteen Fabry patients will be selected and studied in the first six months of add-on oral PCT (1 mcg/day) and, in order to verify the dependence of proteinuria reduction on PCT, three months after drug withdrawal.
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Mar 2014 (estimated)
- Study startMar 2012
- Primary completionDec 2013
- Study completionDec 2013
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.
- More trials run by Federico II UniversityThe sponsor's other registered studies.
- 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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 14 participants (actual)
Sponsor and investigators
- Lead sponsor
- Federico II University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Eleonora Riccio, Federico II University
- Investigators
- eleonora riccio, md, Principal investigator, Federico II University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02090608
- Sponsor's protocol ID
- PCT-FD
- Status last verified
- Jan 2014
- First submitted
- 14 Mar 2014
- Last updated
- 18 Mar 2014
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Mar 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Paricalcitol in Fabry Disease
- ClinicalTrials.gov ID
- NCT02090608
- Acronym / sponsor ID
- PCT-FD
- Conditions
- Fabry Disease, Proteinuria
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 14 participants (actual)
- Lead sponsor
- Federico II UniversityUniversity, hospital or other
- Study start
- Mar 2012
- Primary completion
- Dec 2013 (actual)
- Study completion
- Dec 2013 (actual)
- First posted
- 18 Mar 2014 (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.
Proteinuria is the predominant risk factor for renal disease progression in Fabry disease (FD). When urine protein excretion is controlled to <0.50 g/24 hr, the rate loss of glomerular filtration rate (GFR) is not significantly different from 0. However, enzyme replacement therapy (ERT) alone does not decrease proteinuria and it has been recommended that patients receiving ERT also receive anti Renin-Angiotensin-System (RAS) therapy. Emerging evidences show that paricalcitol (PCT) reduces proteinuria in presence of intensified inhibition of RAS; however, there is no evidence in FD. The aim of this study is to evaluate the antiproteinuric effect of PCT in FD patients with proteinuria >0.50 g/24 hr persisting despite the ERT and anti-RAS therapy titrated to maximum tolerated dosage.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Paricalcitol | Experimental | Drug: ParicalcitolIn patients identified by the inclusion criteria, data will be collected at baseline , during administration of oral Paricalcitol (PCT) (after 1, 3 and 6 months), and three months after PCT withdrawal. PCT will administered at dosage of 1 mcg/day; this dosage was chosen as it is not associated with excessive decline of parathyroid hormone (PTH) levels in most patients |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- genetically proven FD
- stable dose of ERT for at least 12 months
- stable dose of ACEi or ARB titrated to maximum tolerated dosage for at least 6 months
- persistent proteinuria >0.50 g/24 h despite the use of ERT and ACEi/ARBs in 2 consecutive samples within 12 weeks
Key exclusion criteria (5 listed)
- steroid/immunosuppressive treatment or glomerular filtration rate change >30% in the past 3 months
- PTH levels <20 pg/mL
- serum phosphorus >5.0 mg/dL
- serum calcium (adjusted for albumin) >10.0 mg/dL
- active malignancy.
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 |
|---|---|---|
| ParicalcitolParicalcitol was administered at the dose of 1 mcg/die | Drug | Paricalcitol |
Primary outcome
Effect of paricalcitol on proteinuria reduction
Time frame: 6 months
Fourteen Fabry patients will be selected and studied in the first six months of add-on oral PCT (1 mcg/day) and, in order to verify the dependence of proteinuria reduction on PCT, three months after drug withdrawal.
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 18 Mar 2014.
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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.
eleonora riccio, md principal investigator
Federico II University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Paricalcitol in people with Fabry Disease and Proteinuria, run by Federico II University at 1 site in Italy.
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 4 inclusion and 5 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 18 Mar 2014. 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 Italy. 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?
Effect of paricalcitol on proteinuria reduction (time frame: 6 months). 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 18 Mar 2014; AzTrials last refreshed its copy on 3 Nov 2026.