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To Assess the Efficacy of Midodrine in Prevention of Cirrhosis Related Complications in Children Awaiting Liver Transplantation.

Not Applicable
Completed
Conditions
Cirrhosis, Liver
Interventions
Other: Standard Medical Treatment
Registration Number
NCT05287100
Lead Sponsor
Institute of Liver and Biliary Sciences, India
Brief Summary

Children with cirrhosis awaiting transplantation are more proned to develop various complications. The pathogenesis of cirrhotic complications (ascites, hyponatremia, acute kidney injury) includes release of vasodilatory molecules like nitric oxide, damage associated molecular pathogens (DAMPs) and pattern associated molecular pathogens (PAMPs) secondary to bacterial translocation, which causes splanchnic bed vasodilation resulting in activation of renin-angiotensin and aldosterone axis(RAAS) causing sodium and water retention and renal vasoconstriction \[1\].

The development of complications in these children may result in death or may preclude them from reaching upto liver transplantation \[2\].

Midodrine is an α1 adrenergic receptor agonist, which increases vascular tone causing rise in the blood pressure, thereby improving renal perfusion and causes RAAS deactivation. The effects of midodrine is documented in reduction of refractory ascites, hepatorenal syndrome and hyponatremia\[2-4\].

One group will receive only standard medical therapy and other group will receive midodrine and standard medical therapy for 6 months. Mean arterial pressure will be monitored at every OPD visit. At the end of 12 weeks, and 24 weeks, plasma renin activity, incidence of complications related to cirrhosis like new onset ascites, increase in grade of ascites, hyponatremia, acute kidney injury and spontaneous bacterial peritonitis will be assessed. Also the transplant free survival and need for albumin transfusion will be compared between the two groups.

In case of liver transplantation or death before 6 months, midodrine will be stopped

Detailed Description

Aim: To determine the efficacy of midodrine in preventing development of complications in children with cirrhosis awaiting liver transplantation

Primary Objective:

1) To compare incidence of complications (Acute kidney injury, New onset ascites or increase in grade of ascites, Spontaneous bacterial peritonitis, Hyponatremia, Hepatic encephalopathy) of cirrhosis in patients receiving midodrine (at a dose of 0.25 - 0.5mg/kg/day) and standard medical therapy versus standard medical therapy alone for 6 months

Secondary Objectives:

* Frequency of development of new onset ascites or increase in grade of ascites by 6 months

* Change in serum sodium from baseline to 6 months

* Change in Mean arterial pressure (MAP) at 1 week and then 2 weekly till the end of the study

* Plasma renin activity at baseline, at 12 weeks and 24 weeks

* Frequency of development of SBP over 6 months

* Change in eGFR from baseline to 6 months

* Frequency of developing AKI by 6 months

* Frequency of development of Hepatic encephalopathy by 6 months

* Proportion of patients developing hypertension at 6 months

* Frequency of development of drug related adverse effects by 6 months

* Requirement of albumin infusion in 2 groups

* Transplant free survival

Methodology:

* Study population :Children and Adolescents of age group upto 18 years with cirrhosis and PELD/ MELDNa score more than 14, on waitlist for liver transplantation following up in the Pediatric Hepatology Department

* Study design: Open label RCT (computer based randomization - block randomization with block size of 4)

* Study period: 6 months weeks for each patient; The study will be conducted between January 2022 and July 2023

* Sample size:

* Pilot study - 10 patients in each group

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
35
Inclusion Criteria

Children and Adolescents of age group upto 18 years with cirrhosis and PELD/ MELDNa score more than 14, on waitlist for liver transplantation following up in the Pediatric Hepatology Department, ILBS will be prospectively included in this study after informed consent

Exclusion Criteria

Presence of Portal vein thrombosis Renal or cardiovascular disease or arterial hypertension Presence of HCC

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Standard medical therapyStandard Medical TreatmentStandard medical therapy as per departmental protocol
MidodrineMidodrine Oral TabletMidodrine starting at 0.25mg/kg/day in 2-3 divided doses, increased to 0.5mg/kg/day after 7 days if MAP does not increase by \>10% ; Midodrine dosage will be decreased by 25% in case of arterial hypertension (\>95th centile BP for the age). Also Standard medical therapy as per departmental protocol will be continued
MidodrineStandard Medical TreatmentMidodrine starting at 0.25mg/kg/day in 2-3 divided doses, increased to 0.5mg/kg/day after 7 days if MAP does not increase by \>10% ; Midodrine dosage will be decreased by 25% in case of arterial hypertension (\>95th centile BP for the age). Also Standard medical therapy as per departmental protocol will be continued
Primary Outcome Measures
NameTimeMethod
Complications between the two groups6 months

• To compare incidence of complications (Acute kidney injury, New onset ascites or increase in grade of ascites, Spontaneous bacterial peritonitis, Hyponatremia, Hepatic encephalopathy) of cirrhosis in patients receiving midodrine (at a dose of 0.25 - 0.5mg/kg/day) and standard medical therapy versus standard medical therapy alone for 6 months

Secondary Outcome Measures
NameTimeMethod
• Frequency of development of new onset ascites or increase in grade of ascites by 6 months6 months
Plasma renin activity at baseline, at 12 weeks and 24 weeks6 months
Frequency of developing AKI by 6 months6 months
Proportion of patients developing hypertension at 6 months6 months
Change in Mean arterial pressure (MAP) at 1 week and then 2 weekly till the end of the study6 months
Frequency of development of drug related adverse effects by 6 months6 months
Requirement of albumin infusion in 2 groups6 months
Frequency of development of Hepatic encephalopathy by 6 months6 months
Transplant free survival6 months
Frequency of development of SBP over 6 months6 months
Change in serum sodium from baseline to 6 months6 months
Change in eGFR from baseline to 6 months6 months

Trial Locations

Locations (1)

Institute of liver and biliary sciences

🇮🇳

New Delhi, India

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