Phase 3
Completed N=267
Open Label Study Comparing Efficacy and Safety of Dabigatran Etexilate to Standard of Care in Paediatric Patients With Venous Thromboembolism (VTE)
Source: ClinicalTrials.gov NCT01895777 ↗Enrolled (actual)
267
Serious AEs
16.2%
Results posted
Jul 2020
Primary outcomePrimary: Composite Primary Endpoint — 81; 38; 170; 83 Participants — p== 0.0001
Summary
The main objectives of this large phase IIb/III paediatric study are to assess the efficacy and safety of dabigatran etexilate relative to standard of care and to document the appropriateness of the proposed dabigatran etexilate dosing algorithm for use in patients from birth to less than 18 years of age.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Composite Primary Endpoint |
81; 38; 170; 83; 177; 89 | = 0.0001 sig |
| SECONDARY Freedom From Major Bleeding Events (MBEs) |
0.977; 0.977 | — |
| SECONDARY Steady State Plasma Concentrations of Total Dabigatran at Visit 3 |
79.8 | — |
| SECONDARY Steady State Plasma Concentrations After at Least 3 Days Following Any Dabigatran Etexilate Dose Adjustment |
81.7 | — |
| SECONDARY Frequency of Dose Adjustment During the Treatment Phase |
63; 56; 25; 6; 175; 82 | — |
| SECONDARY Frequency of Patients Switching the Type of Anti-coagulation Therapy Including Dabigatran Etexilate to Standard of Care Treatment and Switching From One Standard of Care Treatment to Another |
22; 2 | — |
| SECONDARY Freedom From Thrombus Progression at End of Therapy Compared With Baseline |
148; 73 | — |
| SECONDARY All Bleeding Events |
38; 22; 4; 2; 2; 1 | — |
| SECONDARY All-cause Mortality |
0; 1 | 0.9976 |
| SECONDARY All Components of the Primary Efficacy Endpoint |
81; 38; 7; 7; 0; 1 | — |
| SECONDARY Assessment of the Acceptability of an Age-appropriate Formulation at End of Therapy (Capsules) |
1.0; 1.0; 1.6 | — |
| SECONDARY Assessment of the Acceptability of an Age-appropriate Formulation at End of Therapy (Pellets) |
1.2; 1.2 | — |
| SECONDARY Assessment of the Acceptability of an Age-appropriate Formulation at End of Therapy (Oral Liquid Formulation - OLF) |
1.6; 1.2; 1.4; 1.8 | — |
Eligibility Criteria
Inclusion criteria
- Male or female subjects 0 to less than 18 years of age at the time of informed consent / assent
- Documented diagnosis of clinically stable VTE (e.g. DVT, PE, central line thrombosis, sinus vein thrombosis) per investigator judgment, initially treated (minimum of 5 to 7 days, but not longer than 21 days) with parenteral anticoagulation therapy, such as unfractionated heparin (UFH) or a low molecular weight heparin (LMWH).
- Clinical indication for at least 3 month of treatment with anticoagulants for the VTE episode defined under the above inclusion criterion.
- Written informed consent provided by the patient's parent or legal guardian and assent provided by the patient (if applicable) at the time of informed consent form (ICF) signature according to local regulations.
Exclusion criteria
- Conditions associated with an increased risk of bleeding
- Renal dysfunction (eGFR 3 × upper limit of normal (ULN) within 3 months of screening
- Pregnant or breast feeding females. Females who have reached menarche and are not using a medically accepted contraceptive method per local guidelines. Acceptable methods of birth control must be used in a correct and consistent manner
- Patients in stratum 3 (0 to < 2 years) with gestational age at birth < 37 weeks or with body weight lower than the 3rd percentile
- Anemia (hemoglobin < 80g/L) or thrombocytopenia (platelet count < 80 x 109/L) at screening. Transfusions during the screening period are allowed, provided that a satisfactory hemoglobin or platelet level is attained prior to visit 2
- Patients who have taken prohibited or restricted medication within one week of the first dose of study medication other than medication for prior VTE treatment and P-glycoprotein inhibitors..
- Patients who have received an investigational drug in the past 30 days prior to screening
- Patients who are allergic/sensitive to any component of the study medication including solvent
- Patients or parents/legal guardians considered unreliable to participate in the trial per investigator judgment or any condition which would present a safety hazard to the patient based on investigator judgment
- Patients or parents/legal guardians who are unwilling or unable to undergo or permit repeat of the baseline imaging tests required to confirm thrombus resolution at study day 84 (or eEOT, whichever comes first) or in whom repeating such imaging tests at these pre-specified time points may not be medically in the patient's best interest. Examples may include unwarranted radiation exposure as a result of a repeat CT scan at day 84 for a patient with an isolated case of pulmonary embolism evaluated at baseline solely by a CT scan. In such cases, the baseline radiological assessment (e.g. CT) may be supplemented with an acceptable non-radiological assessment at baseline (e.g. MRI) which could then be repeated at day 84 hence alleviating any potential unwarranted radiation exposure.
- Further exclusion criteria apply
Data sourced from ClinicalTrials.gov (NCT01895777). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.