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Phase 1 Pediatric Pharmacokinetics/Pharmacodynamics (PK/PD) Study

Phase 1
Conditions
Health Condition 1: D65-D69- Coagulation defects, purpura and other hemorrhagic conditions
Registration Number
CTRI/2018/09/015649
Lead Sponsor
DAIICHI SANKYO INC
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
Open to Recruitment
Sex
Not specified
Target Recruitment
0
Inclusion Criteria

Patients who are at risk of thromboembolic events (e.g., patients with

thrombophilia, congenital heart disease, presence of a central venous

catheter);

- Patients who are completing their standard-of-care anticoagulatory therapy.Note that minimum time from the last dose of the standard of careanticoagulant medication and the dose of the study drug should be at least6 hours for unfractionated heparin; 24 hours for vitamin K antagonists(e.g., warfarin) and any other anticoagulants. Note: Before edoxabantreatment, all patients who receive vitamin K antagonists must have aninternational normalization ratio (INR) value < 2.0 within 48 hours prior tothe treatment. If the patientâ??s INR is > 2.0, the patientâ??s INR should be monitored until it is < 2.0;

-Patients with cardiac conditions who may require anticoagulation therapy; or

-Patients with sickle cell disease who may require anticoagulation therapy.

1. Able to provide written informed assents (patients, when applicable) and ICFs (signed by parent/legal guardian) prior to participating in the study

2. Male or female patients 0 to <18 years of age on the day of dosing

3. Patients 2 to < 18 years of age must have a body mass index (BMI) between the 5th and 95th percentile based on the 2000 CDC Growth Charts (the maximum number of patients in each dose group that have a BMI between 85th and 95th percentile should not be more than 2 patients). Patients < 2 years of age must have a body weight between the 5th and 90th percentile based on the 2000 CDC Growth Charts

4. Female patients who have had menarche must test negative for pregnancy, as per local practice, at screening and check-in

5. Female patients who have had menarche and are sexually active must agree to use an effective contraception method, per local practice, for at least 30 days prior to edoxaban dose

6. Patients/Legal guardian(s) must agree to food and drug restrictions during the study

7. Patients must agree to abstain from the use of nonsteroidal anti-inflammatory drugs (such as ibuprofen), and antiplatelet and anticoagulant agents (except for low-dose aspirin) from 24 hours prior to edoxaban dose until after the last PK sample is collected

8. Patients on low-dose aspirin treatment (1 to 5 mg/kg/day, maximum of 100 mg/day) with an interruption of aspirin 24 hours prior to edoxaban dose and resuming 24 hours after edoxaban dose are permitted to participate in the study

per the Investigatorâ??s judgment that this does not place the patients at risk

9. Patients must agree to abstain from CYP3A4 inhibitors/inducers and P-gp inhibitors/inducers for 14 days prior to the edoxaban dose to until after the last PK sample is collected

10. Patients must agree to abstain from and/or legal guardians must agree not to give the patient cola, tea, coffee, chocolate, and other caffeinated drinks and food from 48 hours before dose administration through the end of the study

11. Other than signs and symptoms characteristic to their disease state, patients are to be in good health as determined by the absence of clinically significant deviations from normal, with respect to medical and surgical history, physical examination, vital signs, and laboratory reports, as deemed by the Investigator and the Sponsor, prior to enrollment

12. Patients must agree to abstain from and/or legal guardians must agree not to give the p

Exclusion Criteria

1. History (within the last 6 months) of abnormal coagulation tests during screening, as defined by local laboratory reference ranges, which are not explained by anticoagulation therapy or temporary concomitant affections

2. Stroke where anticoagulant therapy is contraindicated

3. Patients with stage 2 hypertension defined as blood pressure confirmed > 99th percentile + 5 mmHg

4. Patients with renal function less than 50% of normal for age and size as determined by the National Kidney Disease Education Program version of the Schwartz formula

5. Actively bleeding or has a high risk of bleeding

5. Actively bleeding or has a high risk of bleeding

6. Has a currently active gastrointestinal ulceration or a known history of peptic ulcer or gastrointestinal bleeding (including hematemesis, melena, or rectal bleeding including bleeding from hemorrhoids) within the previous 6 months

7. Has known diabetic retinopathy

8. Has thrombocytopenia at screening ( < 20 Ã? 109/L)

9. Has had other unrelated clinically significant illness within 4 weeks prior to

Day 1, predose

10. Planned invasive procedures during or within 24 hours of study drug administration

11. Patient is receiving high-dose aspirin concurrently or within 2 weeks prior to dosing

12. Use of P-gp or CYP 3A4 inhibitors or inducers within 14 days prior to the edoxaban dose and expected to continue through the study duration

13. Patients with history of major bleeding and/or neurosurgery within 6 months of dosing

14. Patients with history of major trauma or surgery within the last month

15. Patients with known malabsorption disorders (e.g., cystic fibrosis or short bowel syndrome)

Study & Design

Study Type
Interventional
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Pharmacokinetic parameters such as apparent systemic <br/ ><br>clearance (CL/F), apparent volume of distribution (V/F), <br/ ><br>and area under the concentration-time curve (AUC) for <br/ ><br>edoxaban and metabolites, and metabolite/parent ratios <br/ ><br>for AUCTimepoint: within 36 hours post dose
Secondary Outcome Measures
NameTimeMethod
Observed, change-from-baseline, and <br/ ><br>percent-change-from-baseline PT, aPTT, and anti-FXa.Timepoint: within 36 hours post dose;Other: <br/ ><br>Palatability of the liquid formulation will be assessed <br/ ><br>using visual analog scale (VAS) scoresTimepoint: within 30 minutes of dosing;Safety: <br/ ><br>Safety assessments: adverse events, physical <br/ ><br>examination findings, vital signs, clinical laboratory <br/ ><br>assessments, and urinalysis.Timepoint: Predose to Day 10
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