Fabry DiseasePhase 2NCT05710367
Effects Of Sodium Glucose Cotranspoter 2 Inhibitors On Heart And Kidneys In Fabry Disease Patients
A Phase 2 study testing Dapagliflozin 10mg Tab in people with Fabry Disease, run by Albina Nowak, MD.
Trial summary
The goal of this clinical trial is to test dapagliflizone in Fabry patients. The main questions it aims to answer are: Has 10 mg/d of dapagliflozin a positive effect on kidney functions of Fabry patients. Has 10 mg/d of dapagliflozin a positive effect on heart functions in Fabry patients. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Fabry Disease
- What is being tested?
- Dapagliflozin 10mg Tab
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 46 participants
- Where
- No sites listed yet
- Study dates
- Aug 2023to Aug 2024
- Phase
- Phase 2
- 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 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age: 18-70 years
- Patients with genetically confirmed Fabry disease.
- On treatment with Enzyme Replacement Therapy (ERT).
- ERT or chaperone therapy at stable dose for at least 3 last months
- Albuminuria >35 mg/day and/or proteinuria >150 mg/day
- eGFR ≥25 mL/min/1.73 m2
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- On a stable dose of an ACEi, ARB or renin receptors blockers for at least 4 weeks prior to randomization
- Sufficient command of German language.
- Signed and dated informed consent.
- Known cardiac association of FD
Who cannot join
- Known hypersensitivity, allergy or contraindications to dapagliflozin.
- Diagnosis of type 1 or type 2 diabetes mellitus
- Patients with any disease (other than Fabry disease) affecting the heart and the kidnys.
- History of kidney transplantation.
- Active malignancy.
- Use of the co-interventional treatments (Aldosterone antagonists, Continuous use of NSAIDs or systemic steroids) within 6 weeks of screening will not be allowed.
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- Any medication, surgical or medical condition which might significantly alter the absorption, distribution, metabolism, or excretion of medications including, but not limited to any of the following:
- History of active inflammatory bowel disease within the last six months;
- Major gastrointestinal tract surgery such as gastrectomy, gastroenterostomy, or bowel resection;
- Gastro-intestinal ulcers and/or gastrointestinal or rectal bleeding within last six months;
- Pancreatic injury or pancreatitis within the last six months;
- Evidence of hepatic disease as determined by any one of the following: ALT or AST values exceeding 3x ULN at the screening visit, a history of hepatic encephalopathy, a history of esophageal varices, or a history of portocaval shunt;
- Subject who, in the assessment of the investigator, may be at risk for dehydration or volume depletion that may affect the interpretation of efficacy or safety data.
- Donation or loss of 400 mL or more of blood within 8 weeks prior to initial dosing.
- Any surgical or medical condition, which in the opinion of the investigator, may place the patient at higher risk from his/her participation in the study, or is likely to prevent the patient from complying with the requirements of the study or completing the study.
- Women who are pregnant or breast feeding; intention to become pregnant during the course of the study, lack of safe contraception.
- Patients with known or suspected non-compliance, drug or alcohol abuse, including Marijuana cigarettes.
- Participation in another study with investigational drugs within the 30 days preceding and during the present study.
- Enrolment of the investigator, his/her family members, employees and other dependent persons.
- Inability to follow the procedures of the study, e.g. due to language problems, psychological disorders, dementia, etc. of the participant.
Trial locations and contacts
No sites are listed yet.
About this study
Official title: Effects Of Sodium Glucose Cotranspoter 2 Inhibitors On Heart And Kidneys In Fabry Disease Patients; A Prospective, Randomized, Double-Blind, Placebo- Controlled Study.
The goal of this clinical trial is to test dapagliflizone in Fabry patients. The main questions it aims to answer are:
- Has 10 mg/d of dapagliflozin a positive effect on kidney functions of Fabry patients.
- Has 10 mg/d of dapagliflozin a positive effect on heart functions in Fabry patients.
Participants will be asked to
- Sign an informed consent
- Give a blood and urine samples
- Be subjected to Echocardiography investigation
- Take 10 mg/day Dapagliflizone
Researchers will compare treatment to placebo groups to see if kidneys and heart functions will be improved in the treatment group better more than the placebo group.
- Conditions
- Fabry Disease
- Being tested
- Dapagliflozin 10mg Tab, Placebo
- Medical subject headings
- Fabry Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Dapagliflozin 10mg TabDrug
Forxiga® as an add-on treatment in patients with renal and/or cardiac association FD in an exploratory framework.
PlaceboDrug
matched oral drug. Placebo tablet will have the same color, taste, smell and package as the verum tablet
| Study group | Role | What they receive |
|---|---|---|
| Dapagliflozin 10mg Tab | Experimental | SGLT2is- Dapagliflozin (Forxiga): 10 mg/d, oral drug Receives: Drug: Dapagliflozin 10mg Tab |
| Placebo | Placebo comparator | Placebo tablet will have the same color, taste, smell and package as the verum tablet Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Assess the change of eGFR in treatment months 6, 12 and at baseline
The CKD-EPI (Chronic Kidney Disease Epidemiology Collaboration) 2009 formula are used to evaluate the calculated GFR.
eGFR in month 6 and 12 are compared to baseline eGFR.
Measured: Baseline, 6 months and 12 months
Assess the change of Protein /creatinine ratio in urine
Total protein is measured in the morning sample of urine, minimal volume of 10 ml are collected and analyzed using Immune nephelometry method. Protein concentration are reported in relation to creatinine.
Measured: Baseline, 6 months and 12 months
Assess the change of Albumin/creatinine ratio in urine
Albumin is measured in the morning sample of urine, minimal volume of 10 ml are collected and analyzed using Immune nephelometry method. Albumin concentration are reported in relation to creatinine.
Measured: Baseline, 6 months and 12 months
Secondary outcomes (4)
NT-pro BNP level will be assessed at baseline and after 6 and 12 months of treatment with study drug and placebo
NT-pro BNP are assessed using a minimal of 2.5 ml heparin-plasma sample with Electro- Chemiluminescent Immunoassay technique.
Measured: Baseline, 6 months and 12 months
Troponin I levels will be assessed at baseline and after 6 and 12 months of treatment with
Troponin I are measured in heparin whole blood (min. 2.5 ml) using Chemiluminescent Microparticle Immunoassay.
Measured: Baseline, 6 months and 12 months
LVMMI parameter will be assessed at baseline and after 6 and 12 months of treatment with study drug and placebo
LVMMI parameter are assessed using M-mode echocardiography in Cardiology clinic
Measured: Baseline, 6 months and 12 months
Septal thickness parameter will be assessed at baseline and after 6 and 12 months of treatment with study drug and placebo
Septal thickness parameter are assessed using M-mode echocardiography in Cardiology clinic
Measured: Baseline, 6 months and 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Feb 2023
- Study startAug 2023 (estimated)
- Primary completionAug 2024 (estimated)
- Study completionAug 2024 (estimated)
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
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 46 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Albina Nowak, MD
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Albina Nowak, MD, University of Zurich
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05710367
- Sponsor's protocol ID
- SGLT2Is46
- Status last verified
- Jan 2023
- First submitted
- 10 Jan 2023
- Last updated
- 2 Feb 2023
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 2 Feb 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effects Of Sodium Glucose Cotranspoter 2 Inhibitors On Heart And Kidneys In Fabry Disease Patients; A Prospective, Randomized, Double-Blind, Placebo- Controlled Study.
- ClinicalTrials.gov ID
- NCT05710367
- Acronym / sponsor ID
- SGLT2Is46
- Conditions
- Fabry Disease
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 46 participants (estimated)
- Lead sponsor
- Albina Nowak, MDUniversity, hospital or other
- Study start
- Aug 2023 (estimated)
- Primary completion
- Aug 2024 (estimated)
- Study completion
- Aug 2024 (estimated)
- First posted
- 2 Feb 2023 (actual)
- 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 goal of this clinical trial is to test dapagliflizone in Fabry patients. The main questions it aims to answer are:
- Has 10 mg/d of dapagliflozin a positive effect on kidney functions of Fabry patients.
- Has 10 mg/d of dapagliflozin a positive effect on heart functions in Fabry patients.
Participants will be asked to
- Sign an informed consent
- Give a blood and urine samples
- Be subjected to Echocardiography investigation
- Take 10 mg/day Dapagliflizone
Researchers will compare treatment to placebo groups to see if kidneys and heart functions will be improved in the treatment group better more than the placebo group.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Dapagliflozin 10mg Tab | Experimental | Drug: Dapagliflozin 10mg TabSGLT2is- Dapagliflozin (Forxiga): 10 mg/d, oral drug |
| Placebo | Placebo comparator | Drug: PlaceboPlacebo tablet will have the same color, taste, smell and package as the verum tablet |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Age: 18-70 years
- Patients with genetically confirmed Fabry disease.
- On treatment with Enzyme Replacement Therapy (ERT).
- ERT or chaperone therapy at stable dose for at least 3 last months
- Albuminuria >35 mg/day and/or proteinuria >150 mg/day
Key exclusion criteria (20 listed)
- Known hypersensitivity, allergy or contraindications to dapagliflozin.
- Diagnosis of type 1 or type 2 diabetes mellitus
- Patients with any disease (other than Fabry disease) affecting the heart and the kidnys.
- History of kidney transplantation.
- 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 |
|---|---|---|
| Dapagliflozin 10mg TabForxiga® as an add-on treatment in patients with renal and/or cardiac association FD in an exploratory framework. | Drug | Dapagliflozin 10mg Tab |
| Placebomatched oral drug. Placebo tablet will have the same color, taste, smell and package as the verum tablet | Drug | Placebo |
Primary outcome
Assess the change of eGFR in treatment months 6, 12 and at baseline
Time frame: Baseline, 6 months and 12 months
The CKD-EPI (Chronic Kidney Disease Epidemiology Collaboration) 2009 formula are used to evaluate the calculated GFR. eGFR in month 6 and 12 are compared to baseline eGFR.
Assess the change of Protein /creatinine ratio in urine
Time frame: Baseline, 6 months and 12 months
Total protein is measured in the morning sample of urine, minimal volume of 10 ml are collected and analyzed using Immune nephelometry method. Protein concentration are reported in relation to creatinine.
Assess the change of Albumin/creatinine ratio in urine
Time frame: Baseline, 6 months and 12 months
Albumin is measured in the morning sample of urine, minimal volume of 10 ml are collected and analyzed using Immune nephelometry method. Albumin concentration are reported in relation to creatinine.
4 secondary outcomes
NT-pro BNP level will be assessed at baseline and after 6 and 12 months of treatment with study drug and placebo
Time frame: Baseline, 6 months and 12 months
Troponin I levels will be assessed at baseline and after 6 and 12 months of treatment with
Time frame: Baseline, 6 months and 12 months
LVMMI parameter will be assessed at baseline and after 6 and 12 months of treatment with study drug and placebo
Time frame: Baseline, 6 months and 12 months
Septal thickness parameter will be assessed at baseline and after 6 and 12 months of treatment with study drug and placebo
Time frame: Baseline, 6 months and 12 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Feb 2023.
- Individual participant dataSharing planned: Undecided
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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.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Dapagliflozin 10mg Tab in people with Fabry Disease, run by Albina Nowak, MD.
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 10 inclusion and 20 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: status not confirmed, as of the sponsor's last update on 2 Feb 2023. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Albina Nowak, MD (+41 (0)43 253 8872, albina.nowak@usz.ch), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Assess the change of eGFR in treatment months 6, 12 and at baseline (time frame: Baseline, 6 months and 12 months). The study lists 3 primary outcomes in all. 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 2 Feb 2023.