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Status unknownNCT05710367Updated Feb 2, 2023

Effects Of Sodium Glucose Cotranspoter 2 Inhibitors On Heart And Kidneys In Fabry Disease Patients

A Phase 2 interventional study of Dapagliflozin 10mg Tab and Placebo in Fabry Disease, sponsored by Albina Nowak, MD. Status unknown. Open to participants aged 18 Years to 70 Years. Per ClinicalTrials.gov, last updated 2023-02-02.

Sponsored by Albina Nowak, MD · Phase 2, Interventional, and Treatment

The sponsor has not verified this record recently (last verified Jan 2023), so the status shown — last known as Not yet recruiting — may be out of date.
Phase
Phase 2
Study type
Interventional
Enrollment
46
Allocation
Randomized
Ages
18 Years to 70 Years
Sex
All
01

Study 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.

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.

02

Conditions studied

  • Fabry Disease

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03

Who can participate

Ages eligible
18 Years to 70 Years
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  • 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
  • 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

Exclusion criteria

Exclusion Criteria:

  • 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.

  • 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:

    1. History of active inflammatory bowel disease within the last six months;
    2. Major gastrointestinal tract surgery such as gastrectomy, gastroenterostomy, or bowel resection;
    3. Gastro-intestinal ulcers and/or gastrointestinal or rectal bleeding within last six months;
    4. Pancreatic injury or pancreatitis within the last six months;
    5. 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.
04

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
None (open label)
Enrollment
46 participants (estimated)

Study arms

  • Experimental
    Dapagliflozin 10mg Tab

    SGLT2is- Dapagliflozin (Forxiga): 10 mg/d, oral drug

    Drug: Dapagliflozin 10mg Tab

  • Placebo comparator
    Placebo

    Placebo tablet will have the same color, taste, smell and package as the verum tablet

    Drug: Placebo

Interventions

  • DrugDapagliflozin 10mg Tab

    Forxiga® as an add-on treatment in patients with renal and/or cardiac association FD in an exploratory framework.

  • DrugPlacebo

    matched oral drug. Placebo tablet will have the same color, taste, smell and package as the verum tablet

05

What researchers measure

Primary outcomes

  1. 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.

    Time frame: Baseline, 6 months and 12 months

  2. 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.

    Time frame: Baseline, 6 months and 12 months

  3. 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.

    Time frame: Baseline, 6 months and 12 months

Secondary outcomes

  1. 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.

    Time frame: Baseline, 6 months and 12 months

  2. 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.

    Time frame: Baseline, 6 months and 12 months

  3. 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

    Time frame: Baseline, 6 months and 12 months

  4. 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

    Time frame: Baseline, 6 months and 12 months

Other outcomes

  1. Chlosterol level will be assessed at baseline and after 6 and 12 months of treatment with study drug and placebo

    Chlosterol level are measured using a minimal of 2.5 ml heparin-plasma sample with Enzymatic color test (CHOD-POD method)

    Time frame: Baseline, 6 months and 12 months

06

Study locations

No study locations are listed for this record.

07

References and documents

Individual participant data

Plan to share: Undecided

No publications or documents are linked to this record.

08

Registry details

Key details

Study ID
NCT05710367
Lead sponsor
Albina Nowak, MD
Responsible party
Albina Nowak, MD (Principal Investigator, Senior Physician of Endocrinology, Diabetes and Clinical Nutrition, University of Zurich) — Sponsor-investigator
First posted
Feb 2, 2023
Start date
Aug 2023 (estimated)
Primary completion
Aug 2024 (estimated)
Completion
Aug 2024 (estimated)
Last update
Feb 2, 2023

Study contacts

Albina Nowak, MD
Contact
albina.nowak@usz.ch
+41 (0)43 253 8872
Israa Abdullah, MD-PhD
Contact
israa.abdullah@usz.ch
+41762710188

Oversight

FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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