DU176b-C-U313_Edoxaban Tosylate in Paediatric Cardiac Diseases
Research type
Research Study
Full title
An open-label, randomised, parallel-group, multicentre, observational trial to evaluate safety and efficacy of edoxaban tosylate in children from 38 weeks gestational age to less than 18 years of age with cardiac diseases at risk of thromboembolic events.
IRAS ID
238090
Contact name
Piers Daubeney
Contact email
Sponsor organisation
Daiichi Sankyo Inc
Eudract number
2017-000475-90
Duration of Study in the UK
2 years, 9 months, 28 days
Research summary
Research Summary
This is a Phase 3 open-label, randomised, parallel-group, multicentre, observational trial to learn if and how edoxaban is better than the current available drugs in children with cardiac diseases at risk of thromboembolic complications, how blood tests may change after taking edoxaban and how safe it is to take this drug.
About 150 participants will be recruited globally and randomised in a 2:1 ratio into one of two treatment arms.
1) Edoxaban treatment arm
2) Standard of Care treatment arm (SOC)Of the planned 150 participants, 100 participants will be treated with edoxaban and 50 participants will be given SOC.
The total duration of the study will be approximately 3 years. The minimum duration for each participant will be 4 months (3 months of main treatment period and 30 days follow up period) and the maximum participation will be 13 months (3 months main treatment period, 9 months extension period and 30 days follow-up).USA and the European Union will participate in this study with additional regions included if required.
Summary of Results
Protocol: An Open-label, Randomised, Parallel-group, Multicentre, Observational Trial to Evaluate Safety and Efficacy of Edoxaban Tosylate in Children From 38 Weeks Gestational Age to Less Than 18 Years of Age With Cardiac Diseases at Risk of Thromboembolic Events Study Name: DU17b-C-U313
Sponsor: Daiichi Sankyo, Inc.General Information About the Trial: The purpose of this study was to test how safe and effective edoxaban is in treating children with heart conditions who are at risk of a type of blood clot called a thromboembolism.
Thromboembolisms in children are generally treated using heparins and vitamin K blockers. Heparins work to prevent blood clots while vitamin K blockers reduce blood clots. The drawbacks of these medicines are that they must be given through injections, involve close monitoring, and require dose adjustments. Edoxaban is only required to be taken once a day by mouth and may offer a better treatment option than standard medicines. This Phase 3 study compared the safety and efficacy (or effectiveness) of edoxaban with standard treatment.Research Questions: The main questions researchers wanted to answer during the study were:
1) How many children experienced a major bleeding event or a clinically relevant non-major bleeding event during the study?2) How long after starting treatment did the children experience a major bleeding event or a clinically relevant non-major bleeding event?
Note: A major bleeding event was any bleeding that occurred in a major part of the body, such as the brain, lungs, or stomach. A clinically relevant non-major bleeding event was any bleeding that does not meet the criteria for major bleeding but requires treatment, hospitalization, or reduction in daily activity.Patients Included in the Study: Children who were 38 weeks gestational age up to 18 years old with a heart condition who were at risk of developing blood clots and required treatment were enrolled in this study. The average age of the children was 8 years old.
Medicines Studied: Edoxaban (oral tablet or liquid mixture [suspension]) compared with heparin standard treatment (tablet or injection)
Overall Results: Overall, edoxaban was safe and effective in children with heart conditions.
During the main treatment period, 1 out of 109 (1.0%) children on edoxaban had a clinically relevant non-major bleeding event (nosebleed) and 1 out of 58 (2.0%) children on standard treatment had a clinically relevant non-major bleeding event (bleeding in the digestive tract).
In terms of the onset of bleeding events, 1 child had a clinically relevant non-major bleeding event 10 days after starting edoxaban treatment and 1 child had a clinically relevant non-major bleeding event 58 days (about 2 months) after starting standard treatment.
During the extension period, 1 out of 144 (0.7%) children on edoxaban had a major bleeding event (liver injury) following an accident and a non-major bleeding event (nosebleed).
In terms of the onset of bleeding events, 1 child had a major bleeding event 224 days (more than 7 months) after starting edoxaban treatment and a clinically relevant non major bleeding event 247 days (more than 8 months) after starting treatment.Side Effects: During the main treatment period, 7 out of 109 (6%) children who were on edoxaban reported side effects and 1 out of 58 (2%) children on standard treatment reported side effects. The most common side effect (occurring in at least 2 children) was nosebleed.
During the extension period, 9 out of 144 (6%) children reported side effects. The most common side effects (occurring in at least 2 children) were headache and nosebleed.
Side effects are considered serious if they cause death, are life-threatening, cause lasting problems, or require hospitalization. No child had a serious side effect or died due to a side effect during this study.
Some children stopped study treatment because of side effects. During the main treatment period, 1 child stopped edoxaban treatment due to blood clot in the artery.
During the extension period, 2 children stopped edoxaban treatment due to heart attack and blockage of blood flow to the heart muscle.How Has the Study Helped Patients and Researchers: These study findings allowed researchers to learn about the safety and effects of edoxaban in the prevention of blood vessels being blocked by clots in children with heart conditions.
Are There Any Plans for Further Studies: Other studies for edoxaban are ongoing and the sponsor plans to conduct more studies in the future.
Where Can I Find Additional Information About This Study: https://eur03.safelinks.protection.outlook.com/?url=http%3A%2F%2Fwww.clinicaltrials.gov%2F&data=05%7C01%7Capprovals%40hra.nhs.uk%7C4f6ea1414fd04167a78808da6af263ad%7C8e1f0acad87d4f20939e36243d574267%7C0%7C0%7C637939886529079747%7CUnknown%7CTWFpbGZsb3d8eyJWIjoiMC4wLjAwMDAiLCJQIjoiV2luMzIiLCJBTiI6Ik1haWwiLCJXVCI6Mn0%3D%7C3000%7C%7C%7C&sdata=UFSOctF3Y73E2VbzH8m9bDugWDBpHvpjqZ%2BEExSjusY%3D&reserved=0 (keyword: DU176b-C-U313)
REC name
London - Central Research Ethics Committee
REC reference
18/LO/0034
Date of REC Opinion
27 Mar 2018
REC opinion
Further Information Favourable Opinion