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Evaluation of GeranylGeranylAcetone in Heart Failure With Preserved Ejection Fraction

Phase 2
Completed
Conditions
Heart Failure With Preserved Ejection Fraction
Interventions
Diagnostic Test: Iohexol measurement
Diagnostic Test: Echocardiography
Diagnostic Test: 6-minute walking distance test
Diagnostic Test: EndoPAT
Diagnostic Test: Para-amino Hippuric Acid test
Diagnostic Test: Electrocardiogram
Registration Number
NCT05672134
Lead Sponsor
Amsterdam UMC, location VUmc
Brief Summary

The goal of this double-blind randomized, placebo-controlled cross-over trial is to evaluate the effectiveness of GerenylGeranylAcetone (GGA) in patients with Heart Failure with a preserved ejection fraction.

The main questions it aims to answer are:

* What is the effect of GGA on diastolic function?

* What is the effect of GGA on endothelial function?

Main study tasks:

* Participants will be treated with either GGA or placebo for 13 weeks. After this they will have a break (wash-out) period for 6 weeks and then cross over to the other study arm.

* Cardiac function will be measured using echocardiogram in all participants

* Renal measurements and endothelial measurements will be performed on the participants.

* Participants will perform a 5 minute walking distance test for functional capacity.

* Participants will fill out questionnaires to score signs \& symptoms.

Researchers will compare the patients to themselves to see if the drug improves diastolic- and endothelial function.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
43
Inclusion Criteria
  1. Age≥ 50 years

  2. Patients with a diagnosis of symptomatic chronic heart failure (New York Heart Association class II or III) AND preserved systolic LV function (LV ejection fraction or LVEF ≥ 50%) documented within the last 6 months AND evidence of diastolic LV dysfunction with at least 1 out of the following 4 criteria:

    • HFA-PEFF score ≥5
    • H2FPEF score ≥6
    • HFpEF according to the 2021 ESC HF Guidelines (NT-proBNP>125 pg/ml AND either LV mass indexed or LVMI >95 g/m2 for women and >115 g/m2 for men OR left atrial volume indexed or LAVI >34 ml/m2 OR mean e; septal/lateral < 9 cm/s) OR E/e' >13 OR TR velocity at rest >2,8m/s.
    • Pulmonary capillary wedge pressure (PCWP) >15 mmHg and/or >25 mmHg during exercise.
Exclusion Criteria
  1. Current acute decompensated heart failure, requiring hospitalization or augmented therapy with intravenous diuretics, vasodilators, and/or inotropic drugs
  2. Acute coronary syndrome, transient ischemic attack/cerebrovascular accident, major surgery within the previous 3 months
  3. Hemoglobin <9 g/dl at screening
  4. LVEF <40% measured at any time point in the history of the patient
  5. History of mitral valve repair or replacement
  6. Presence of significant valvular disease defined as mitral valve regurgitation defined as grade ≥ 3+ MR; tricuspid valve regurgitation defined as grade ≥ 2+ TR; aortic valve disease defined as ≥ 2+ AR or > moderate AS
  7. Acute myocarditis within 3 months prior to randomization
  8. Infiltrative cardiomyopathy
  9. Genetic cardiomyopathy
  10. Severe pulmonary disease requiring home oxygen or chronic oral steroid therapy
  11. Precapillary pulmonary hypertension
  12. BMI >40 kg/m2
  13. Estimated glomerular filtration rate (GFR) <20 ml/min or >90 ml/min
  14. History of solid organ transplantation including kidney transplantation
  15. Atrial fibrillation or atrial flutter with resting ventricular rate >110 bpm
  16. Not able to undergo the complete study protocol
  17. Doubt about compliance
  18. Pre-menopausal women who are nursing, pregnant, or of child-bearing potential and not practicing an acceptable method of birth control
  19. Chronic absorption problems
  20. Proven allergy for lactose products or cow-milk.
  21. Proven allergy for Iodide-containing contrast, Iohexol or PAH.
  22. Any documented or suspected malignancy or history of malignancy within 1 year prior to screening, except appropriately treated basal cell carcinoma of the skin or in situ carcinoma of the cervix
  23. Currently enrolled in another investigational device or drug trial
  24. Estimated life expectancy <1 year

Study & Design

Study Type
INTERVENTIONAL
Study Design
CROSSOVER
Arm && Interventions
GroupInterventionDescription
Experimental armGeranylgeranylacetone (GGA)Patients will receive 13 weeks of experimental treatment, followed by a 7 week wash-out period, continuing with 13 weeks of placebo as per crossover design.
Experimental armEndoPATPatients will receive 13 weeks of experimental treatment, followed by a 7 week wash-out period, continuing with 13 weeks of placebo as per crossover design.
Experimental armElectrocardiogramPatients will receive 13 weeks of experimental treatment, followed by a 7 week wash-out period, continuing with 13 weeks of placebo as per crossover design.
Experimental armIohexol measurementPatients will receive 13 weeks of experimental treatment, followed by a 7 week wash-out period, continuing with 13 weeks of placebo as per crossover design.
Experimental arm6-minute walking distance testPatients will receive 13 weeks of experimental treatment, followed by a 7 week wash-out period, continuing with 13 weeks of placebo as per crossover design.
Experimental armPara-amino Hippuric Acid testPatients will receive 13 weeks of experimental treatment, followed by a 7 week wash-out period, continuing with 13 weeks of placebo as per crossover design.
Placebo armEndoPATPatients will receive 13 weeks of placebo treatment, followed by a 7 week wash-out period, continuing with 13 weeks of experimental treatment as per crossover design.
Placebo armPara-amino Hippuric Acid testPatients will receive 13 weeks of placebo treatment, followed by a 7 week wash-out period, continuing with 13 weeks of experimental treatment as per crossover design.
Experimental armEchocardiographyPatients will receive 13 weeks of experimental treatment, followed by a 7 week wash-out period, continuing with 13 weeks of placebo as per crossover design.
Placebo armIohexol measurementPatients will receive 13 weeks of placebo treatment, followed by a 7 week wash-out period, continuing with 13 weeks of experimental treatment as per crossover design.
Placebo arm6-minute walking distance testPatients will receive 13 weeks of placebo treatment, followed by a 7 week wash-out period, continuing with 13 weeks of experimental treatment as per crossover design.
Placebo armElectrocardiogramPatients will receive 13 weeks of placebo treatment, followed by a 7 week wash-out period, continuing with 13 weeks of experimental treatment as per crossover design.
Placebo armGeranylgeranylacetone (GGA)Patients will receive 13 weeks of placebo treatment, followed by a 7 week wash-out period, continuing with 13 weeks of experimental treatment as per crossover design.
Placebo armEchocardiographyPatients will receive 13 weeks of placebo treatment, followed by a 7 week wash-out period, continuing with 13 weeks of experimental treatment as per crossover design.
Primary Outcome Measures
NameTimeMethod
Filling pressuresafter 13 weeks of treatment

Changes in echocardiography determined filling pressures (E/e')?

Endothelial functionafter 13 weeks of treatment

Changes in EndoPAT®-derived reactive hyperemia index (RHI)

Secondary Outcome Measures
NameTimeMethod
Pulmonary Artery PressureAfter 13 weeks of treatment

Change in echocardiographically determined PAP

Left atrial global strainAfter 13 weeks of treatment

Changes in echogardiographic measured LA global strain (LAGS)

Nitrate (microvascular marker)After 13 weeks of treatment

Changes in nitrate concentration in serum

Left Ventricular global longitudinal strainAter 13 weeks of treatment

Changes in echocardiographically determinded LV global longitudinal strain (LGS)

Measured Glomerular Filtration Rate (mGFR)After 13 weeks of treatment

Changes in mGFR using Iohexol measurements in urine

Patient reported symptomsAfter 13 weeks of treatment

Evaluation of symptoms using New York Heart Association class (NYHA)

Left atrial volumesAfter 13 weeks of treatment

Changes in echogardiographic measured left atrial volume index (LAVI)

Left atrial emptying fractionsAfter 13 weeks of treatment.

Changes in echogardiographic LA emptying fractions. Formula: (LA maximum volume-LA minimum volume)/LA maximum volume × 100%

CRP (inflammatory biomarker)After 13 weeks of treatment

Changes in CRP concentration in serum

H2S (microvascular marker)After 13 weeks of treatment

Changes in H2S concentration in serum

Urine Albumine Creatinin RatioAfter 13 weeks of treatment

Changes in UACR concentration measured in urine.

Right Ventricular systolic functionAfter 13 weeks of treatment

Change in echocardiographically determined RV TAPSE

Left Ventricular Myocardial relaxationAter 13 weeks of treatment

Change in echocardiographically determined myocardial relaxation (e')

Left Ventricular distensibilityAfter 13 weeks of treatment

Change in echocardiographically determined LV distensibility, measured by E.

Quality of life assessmentAfter 13 weeks of treatment.

Evaluation of quality of life using the Kansas City Cariomyopathy Questionnaire

Functional capacityAfter 13 weeks of treatment.

Evaluation of Functional Capacity using 6-minute walking distance test (6MWD)

Nitrosated hemoglobin (microvascular marker)After 13 weeks of treatment

Changes in Nitrosated hemoglobin (Hb(NO)4) concentration in serum

Endothelin-1 (microvascular marker)After 13 weeks of treatment

Changes in Endothelin-1 concentration in serum

Neutrophil gelatinase associated lipocalin (NGAL)After 13 weeks of treatment

Changes in NGAL concentration measured in urine and serum

Effective Renal Plasma Flow (ERPF)After 13 weeks of treatment

Changes in ERPF using PAH-measurements in urine

Renal vascular resistance (RVR)After 13 weeks of treatment

Changes in intrakidney hemodynamic function (Systemic Blood pressure / Renal Blood Flow)

Kidney Injury marker 1 (KIM-1)After 13 weeks of treatment

Changes in KIM-1 concentration measured in urine and serum

Trial Locations

Locations (1)

Amsterdam UMC, loc VUmc

🇳🇱

Amsterdam, Noord-Holland, Netherlands

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