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

NCT ID: NCT05710367

Last Updated: 2023-02-02

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: PHASE2
• Total Enrollment: 46 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2023-08-31
• Study Completion Date: 2024-08-31

Brief 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.

Conditions

Fabry Disease

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Dapagliflozin 10mg Tab

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

Group Type: EXPERIMENTAL

Dapagliflozin 10mg Tab

Intervention Type: DRUG

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

Placebo

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

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

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

Interventions

Dapagliflozin 10mg Tab

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

Intervention Type: DRUG

Placebo

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

Intervention Type: DRUG

Eligibility Criteria

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

• 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.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 70 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Albina Nowak, MD

OTHER

Sponsor Role: lead

Responsible Party

Albina Nowak, MD

Principal Investigator, Senior Physician of Endocrinology, Diabetes and Clinical Nutrition

Responsibility Role: SPONSOR_INVESTIGATOR

Central Contacts

Albina Nowak, MD

Role: CONTACT

albina.nowak@usz.ch

+41 (0)43 253 8872

Israa Abdullah, MD-PhD

Role: CONTACT

israa.abdullah@usz.ch

+41762710188

Other Identifiers

SGLT2Is46

Identifier Type: -

Identifier Source: org_study_id