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

Albina Nowak, MD

Status and phase: Not yet enrolling; Phase 2

Conditions: Fabry Disease

Treatments: Drug: Dapagliflozin 10mg Tab; Drug: Placebo

Study type: Interventional

Funder types: Other

Identifiers

NCT05710367

SGLT2Is46

Details and patient eligibility

About

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.

Enrollment: 46 estimated patients

Sex: All

Ages: 18 to 70 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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46 participants in 2 patient groups, including a placebo group

Dapagliflozin 10mg Tab

Experimental group

Description:

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

Treatment:

Drug: Dapagliflozin 10mg Tab

Placebo

Placebo Comparator group

Description:

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

Treatment:

Drug: Placebo