An Open-Label, Study to Evaluate safety and efficacy of Edoxaban tosylate in children with heart disease at risk of a blood clot.

Phase 1

• Conditions: thromboembolism; MedDRA version: 20.0Level: LLTClassification code 10043566Term: ThromboembolismSystem Organ Class: 100000004866; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2017-000475-90-HU
• Lead Sponsor: Daiichi Sankyo Inc

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2017-11-23
• Last Update Posted: 2 March 2022

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 150

Inclusion Criteria

Subjects must satisfy all of the following criteria to be eligible for the study:

1.Children with cardiac diseases who are at risk for thromboembolic complications and require at least 3 months antithrombotic anticoagulant prophylaxis. Either one of the following:
a.Children with cardiac disease who have a history of cardiac shunt occlusion/thrombosis, with shunt still in place (secondary prevention).
OR
b.Children with cardiac disease who require (including those already taking, and those not yet taking) anticoagulation for primary prevention of TE.
Cardiac conditions known to significantly increase the risk of thrombosis (hence, indications for primary TE prevention) are defined in Antithrombotic Therapy and Prevention of Thrombosis.1 Some examples of cardiac conditions at risk of thrombosis are Fontan surgery, heart failure, and Kawasaki disease, and Blalock-Taussig and Glenn surgery.

2.Male or female children between 1 and <18 years of age. Children between 38 weeks gestational age and 1 year of age will be included in the study, however, only after the safety and efficacy data of 50 subjects between 1 and <18 years of age in the edoxaban arm have been evaluated at the end of the 3-month treatment period.

3.Subject and/or parent(s)/legal guardian(s) or legally acceptable representative is informed and provides signed consent for the child to participate in the study with edoxaban treatment. Pediatric subjects with appropriate intellectual maturity will be required to sign an assent form in addition to the signed informed consent from the parent(s)/legal guardian(s) or any legally acceptable representative.

4.Female subjects of childbearing potential must test negative for pregnancy at Screening and must consent to avoid becoming pregnant by using a locally approved contraception method throughout the study. For locally approved contraceptive methods.
Are the trial subjects under 18? yes
Number of subjects for this age range: 150
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Subjects who meet any of the following criteria will be disqualified from entering the study:

1.Subjects with evidence of symptomatic venous or arterial thrombosis and/or asymptomatic intracardiac thrombosis confirmed by a transthoracic echocardiogram during study screening period.Note: Valid echocardiograms are images taken within 5 weeks prior to Randomization Visit.

2.Subjects with mechanical heart valves.

3.Subjects with active bleeding or high risk of bleeding contraindicating treatment with anticoagulant.

4.Co-administration of antithrombotic therapy is contraindicated in edoxaban arm and SOC arm except for low dose aspirin defined as 1 to 5 mg/kg/day with maximum of 100 mg/day.

5.Subjects with hepatic disease associated with coagulopathy leading to a clinically relevant bleeding risk (aPTT >50 seconds or international normalized ratio [INR] >2.0 not related to anticoagulation therapy) or ALT >5 × the upper limit of normal (ULN) or total bilirubin (TBL) >2 × ULN with direct bilirubin >20% of the total at Screening.

6.Subjects with estimated glomerular filtration rate (eGFR) <30% of normal for age and size.

7.Subjects with stage 2 hypertension defined as blood pressure (BP) systolic and/or diastolic confirmed >99th percentile plus 5 mmHg.

8.Subjects with thrombocytopenia (thrombocytes <50 × 109/L).

9.Subjects with Fontan procedure with a history of or signs/symptoms suggestive of protein-losing enteropathy.

10.Subjects with a life expectancy less than the expected study duration (3 months).

11.Subjects who are known to be pregnant or breastfeeding.

12.Subjects with any condition that, as judged by the Investigator, would place the subject at increased risk of harm if he/she participated in the study.

13.Subjects with a contraindication to the use of heparin and/or VKA (UFH or LMWH) and/or VKA

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified