VTE ProphylaxisPhase 1NCT03346083
Study Evaluating Betrixaban in Pediatric Participants
A Phase 1 study testing Betrixaban in people with VTE Prophylaxis, run by Alexion Pharmaceuticals, Inc. at 20 sites in 4 countries.
Trial summary
This trial was a Phase 1, open-label, multicenter study of the pharmacokinetics (PK), pharmacodynamics (PD), and safety of a single dose of betrixaban in pediatric participants at risk of venous thromboembolism (VTE). Read more about this study
- Who is it for?
- Ages 2 to 17 years
- Conditions the study is for
- VTE Prophylaxis
- What is being tested?
- Betrixaban
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 21 participants
- Where
- 20 sitesin 4 countries
- Study dates
- 13 Jul 2018to 8 Oct 2019
- Phase
- Phase 1
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Pediatric participants in the following age categories: 12 to < 18 years of age and 2 to < 12 years of age. Part 1 of the study enrolled only adolescent participants 12 to < 18 years of age.
- Pediatric participant who was assessed to be at risk for VTE but did not require immediate anticoagulant therapy, for example:
- Had previous thrombosis and completed a course of anti-coagulant therapy, and is considered to have a risk for recurrence of VTE, or
- Had any stable disease with a risk for arterial or venous thromboembolism, or
- Had any functional central venous access device in the upper or lower venous system.
- Participant had normalized coagulation parameters (international normalized ratio or partial thromboplastin time, as appropriate) within 7 days of study drug administration.
Who cannot join
- Participants who meet any one of the following exclusion criteria were excluded from the study:
- Participant received any dose of anti-coagulant therapy within 7 days of Day 1.
- Participant had active bleeding or had a comorbid disorder that placed the participant at high risk for bleeding.
- Participant had a comorbid disorder that placed the participant at risk of death within 90 days of enrollment.
- Participant had abnormal coagulation tests at baseline.
- Participant had recent or planned invasive procedures, including lumbar puncture and removal of non-peripherally placed central lines during study.
Show all 25Show fewer
- Participant had hepatic disease associated with one or more of the following:
- Transaminase levels ≥ 2.5 × upper limit of normal (ULN) or bilirubin ≥ 1.5 × ULN at baseline.
- Coagulopathy leading to a clinically relevant bleeding risk, or hepatic transaminase level of > 2 × ULN or total bilirubin > 2 × ULN with direct bilirubin > 20% of the total.
- Platelet count < 75 × 10^9/liter or hemoglobin < 10.0 mg/deciliter.
- Hypertension.
- Participant had known congenital or acquired bleeding diathesis.
- Participant required concomitant therapy with a strong P-glycoprotein inhibitor.
- Participant had previous history of any non-traumatic bleeding event that was life threatening or required medical attention.
- Participant had been administered thrombolytic therapy, or had undergone thrombectomy, or insertion of a caval filter to treat prior VTE.
- Participant had known inherited or acquired bleeding diathesis or coagulopathy.
- Participant had abnormal QTcF interval on baseline electrocardiogram.
- Participant received a dose of any antiplatelet medication (including aspirin) within 14 days before study drug dosing.
- Participant had malabsorption disorders (for example, cystic fibrosis or short bowel syndrome).
- Participant had an estimated glomerular filtration rate < 30 milliliters/minute.
- Participant was unable or reluctant to cooperate with the study procedures.
- Participant had hypersensitivity to other Factor Xa inhibitors, or the components of the dosage form.
- Participant had participated in a study with an investigational drug or medical device within 30 days prior to administration of betrixaban.
- Participant was female and of childbearing potential and was either pregnant or breastfeeding a child.
- Participant was sexually active and was not using medically accepted contraceptive method (if applicable).
Trial locations and contacts
20 sites in 4 countries.
20 sites in 4 countries
Russia6 sites
Children's Hospital of Tatarstan Republic
Kazan', 420138
Federal State Institution
Kemerovo, 650002
Children's City Clinical Hospital
Moscow, 119049
Pirogov Russian National Research Medical University
Moscow, 125412
State Budgetary Institution
Nizhny Novgorod, 603136
Saint Petersburg State Pediatric Medical University
Saint Petersburg, 194100
United States5 sites
Children's Healthcare of Atlanta
Atlanta, Georgia, 30322
Rainbow Babies & Children's Hospital
Cleveland, Ohio, 44106
Children's Hospital Los Angeles
Los Angeles, California, 90027
ACTCA, Axis Clinical Trials
Los Angeles, California, 90036
Tulane Medical Center
New Orleans, Louisiana, 70001
United Kingdom5 sites
Birmingham Women's and Children's NHS Foundation Trust
Birmingham, B4 6NH
Addenbrooke's Hospital
Cambridge, CB2 0QQ
Children's Hospital for Wales
Cardiff, CF14 4XW
Glenfield Hospital
Leicester, LE3 9QP
Evelina London Children's Hospital
London, SE1 7EH
Ukraine4 sites
Ivano-Frankivsk Regional Children Clinical Hospital
Ivano-Frankivsk, 76000
Odessa Regional Children Clinical Hospital
Odesa
Sumy Regional Children's Hospital
Sumy
Vinnytsia Regional Children's Clinical Hospital, Department of Anesthesiology and Intensive Care
Vinnitsa
About this study
Official title: A Phase 1, Open-Label, Single-Dose, Non-Randomized Study to Evaluate Pharmacokinetics, Pharmacodynamics, and Safety of Betrixaban in Pediatric Patients
This trial was a Phase 1, open-label, multicenter study of the pharmacokinetics (PK), pharmacodynamics (PD), and safety of a single dose of betrixaban in pediatric participants at risk of venous thromboembolism (VTE).
Read the detailed description
This study was to be conducted in 2 parts: Part 1 and Part 2. Part 1 (the initial opening of the study) was conducted in 21 adolescent participants (12 to < 18 years of age) who were assessed to be at risk for VTE. Participants in Part 1 received either 40 or 80 milligrams (mg) of study drug. The PK and PD data from Part 1 was to be used for dose determination for the next youngest age group using population PK and physiological-based PK modeling and simulation. Following analysis of Part 1 data, Part 2 of the study was to commence and enroll 12 participants 2 to < 12 years of age. However, after completion of Part 1 and prior to initiating Part 2, the Sponsor decided to cease developing betrixaban, prompting early study closure.
- Conditions
- VTE Prophylaxis
- Being tested
- Betrixaban
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BetrixabanDrug
Also called PRT054021
Factor Xa inhibitor.
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1: Betrixaban 40 mg | Experimental | Participants received a single, oral dose of betrixaban at 40 mg in a fed state, and had 10 PK blood sampling time points. Receives: Drug: Betrixaban |
| Cohort 2: Betrixaban 80 mg | Experimental | Participants received a single, oral dose of betrixaban at 80 mg in a fed state, and had 5 PK sampling time points. Receives: Drug: Betrixaban |
What is being measured
The primary outcome is the main question the study is designed to answer.
Area Under The Plasma Concentration-Time Curve From 0 To Infinity (AUC(0-inf)) Of Betrixaban
Following the Sponsor's decision to cease developing betrixaban, data for AUC(0-inf) were not collected.
Measured: Up to 6 days post dose
Maximum Observed Plasma Concentration (Cmax) Of Betrixaban
Data reported as "0.200" indicates that the data are below the lower limit of quantification. Note that the Measure of Central Tendency could not be determined for Cohort 1 or Cohort 2 due to the values that are below the lower limit of quantification.
Measured: Up to 6 days post dose
Secondary outcomes (7)
AUC To The Last Measurable Concentration Above The Quantitation Limit (AUC(0-last)) Of Betrixaban
Following the Sponsor's decision to cease developing betrixaban, data for AUC(0-last) were not collected.
Measured: Up to 6 days post dose
Terminal Plasma Half-life (t½) Of Betrixaban
Following the Sponsor's decision to cease developing betrixaban, data for t½ were not collected.
Measured: Up to 6 days post dose
Time To Maximum Observed Plasma Concentration (Tmax) Of Betrixaban
The Tmax that the highest (maximum) Cmax of betrixaban was observed per group up to Day 6 (120 hours) post dosing is reported.
Measured: Up to 6 days post dose
Apparent Total Body Clearance Of Betrixaban From Plasma (CL)
Following the Sponsor's decision to cease developing betrixaban, data for CL were not collected.
Measured: Up to 6 days post dose
Apparent Volume Of Distribution (Vd) Of Betrixaban
Following the Sponsor's decision to cease developing betrixaban, data for Vd were not collected.
Measured: Up to 6 days post dose
Percent Change From Baseline In Thrombin Level At Day 6
Following the Sponsor's decision to cease developing betrixaban, data for thrombin levels were not collected.
Measured: Baseline, Day 6
Count Of Participants With Treatment-related Adverse Events
A treatment-related adverse event was any undesirable event or any untoward medical occurrence that occurs to a participant during the course of a study, or the protocol-defined time after study termination. An Investigator qualified in medicine made the determination of relationship to the investigational product for each adverse event (Unrelated, Unlikely Related, Possibly Related, or Probably Related). If the relationship between the adverse event and the investigational product was determined to be "possible" or "probable", the event was considered to be related to the investigational product for the purposes of expedited regulatory reporting. One participant experienced a mild study-drug-related headache that resolved in less than 2 hours. A summary of serious and all other non-serious adverse events, regardless of causality, is located in the Reported Adverse Events module.
Measured: Up to 7 days post dose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Nov 2017
- Study start13 Jul 2018
- Primary completion8 Oct 2019
- Study completion8 Oct 2019
Why it stopped: After completion of Part 1 and prior to initiating Part 2, the Sponsor decided to cease developing betrixaban, prompting early study closure.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for VTE ProphylaxisStudies of the same condition, open and under way.
- More trials run by Alexion Pharmaceuticals, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 21 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Portola Pharmaceuticals, LLC (a wholly owned subsidiary of Alexion Pharmaceuticals)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03346083
- Sponsor's protocol ID
- 16-021
- Other IDs
- 2018-002562-40, 2018
- Status last verified
- Apr 2023
- First submitted
- 7 Nov 2017
- Last updated
- 12 Jan 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Jan 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1, Open-Label, Single-Dose, Non-Randomized Study to Evaluate Pharmacokinetics, Pharmacodynamics, and Safety of Betrixaban in Pediatric Patients
- ClinicalTrials.gov ID
- NCT03346083
- Acronym / sponsor ID
- 16-021
- Conditions
- VTE Prophylaxis
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: After completion of Part 1 and prior to initiating Part 2, the Sponsor decided to cease developing betrixaban, prompting early study closure.
- Enrolment
- 21 participants (actual)
- Lead sponsor
- Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Portola Pharmaceuticals, LLC (a wholly owned subsidiary of Alexion Pharmaceuticals)
- Study start
- 13 Jul 2018 (actual)
- Primary completion
- 8 Oct 2019 (actual)
- Study completion
- 8 Oct 2019 (actual)
- First posted
- 17 Nov 2017 (actual)
- Results
- Posted 12 Jan 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This trial was a Phase 1, open-label, multicenter study of the pharmacokinetics (PK), pharmacodynamics (PD), and safety of a single dose of betrixaban in pediatric participants at risk of venous thromboembolism (VTE).
Detailed description
This study was to be conducted in 2 parts: Part 1 and Part 2. Part 1 (the initial opening of the study) was conducted in 21 adolescent participants (12 to < 18 years of age) who were assessed to be at risk for VTE. Participants in Part 1 received either 40 or 80 milligrams (mg) of study drug. The PK and PD data from Part 1 was to be used for dose determination for the next youngest age group using population PK and physiological-based PK modeling and simulation. Following analysis of Part 1 data, Part 2 of the study was to commence and enroll 12 participants 2 to < 12 years of age. However, after completion of Part 1 and prior to initiating Part 2, the Sponsor decided to cease developing betrixaban, prompting early study closure.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cohort 1: Betrixaban 40 mg | Experimental | Drug: BetrixabanParticipants received a single, oral dose of betrixaban at 40 mg in a fed state, and had 10 PK blood sampling time points. |
| Cohort 2: Betrixaban 80 mg | Experimental | Drug: BetrixabanParticipants received a single, oral dose of betrixaban at 80 mg in a fed state, and had 5 PK sampling time points. |
Who may qualify
- Ages
- 2 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- Pediatric participants in the following age categories: 12 to < 18 years of age and 2 to < 12 years of age. Part 1 of the study enrolled only adolescent participants 12 to < 18 years of age.
- Pediatric participant who was assessed to be at risk for VTE but did not require immediate anticoagulant therapy, for example:
- Participant had normalized coagulation parameters (international normalized ratio or partial thromboplastin time, as appropriate) within 7 days of study drug administration.
Key exclusion criteria (25 listed)
- Participants who meet any one of the following exclusion criteria were excluded from the study:
- Participant received any dose of anti-coagulant therapy within 7 days of Day 1.
- Participant had active bleeding or had a comorbid disorder that placed the participant at high risk for bleeding.
- Participant had a comorbid disorder that placed the participant at risk of death within 90 days of enrollment.
- Participant had abnormal coagulation tests at baseline.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BetrixabanAlso: PRT054021Factor Xa inhibitor. | Drug | Cohort 1: Betrixaban 40 mg, Cohort 2: Betrixaban 80 mg |
Primary outcome
Area Under The Plasma Concentration-Time Curve From 0 To Infinity (AUC(0-inf)) Of Betrixaban
Time frame: Up to 6 days post dose
Following the Sponsor's decision to cease developing betrixaban, data for AUC(0-inf) were not collected.
Maximum Observed Plasma Concentration (Cmax) Of Betrixaban
Time frame: Up to 6 days post dose
Data reported as "0.200" indicates that the data are below the lower limit of quantification. Note that the Measure of Central Tendency could not be determined for Cohort 1 or Cohort 2 due to the values that are below the lower limit of quantification.
7 secondary outcomes
AUC To The Last Measurable Concentration Above The Quantitation Limit (AUC(0-last)) Of Betrixaban
Time frame: Up to 6 days post dose
Terminal Plasma Half-life (t½) Of Betrixaban
Time frame: Up to 6 days post dose
Time To Maximum Observed Plasma Concentration (Tmax) Of Betrixaban
Time frame: Up to 6 days post dose
Apparent Total Body Clearance Of Betrixaban From Plasma (CL)
Time frame: Up to 6 days post dose
Apparent Volume Of Distribution (Vd) Of Betrixaban
Time frame: Up to 6 days post dose
Percent Change From Baseline In Thrombin Level At Day 6
Time frame: Baseline, Day 6
Count Of Participants With Treatment-related Adverse Events
Time frame: Up to 7 days post dose
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Jan 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
20 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Betrixaban in people with VTE Prophylaxis, run by Alexion Pharmaceuticals, Inc. at 20 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 25 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 12 Jan 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 20 sites in Russia, United States, United Kingdom, Ukraine. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 12 Jan 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Area Under The Plasma Concentration-Time Curve From 0 To Infinity (AUC(0-inf)) Of Betrixaban (time frame: Up to 6 days post dose). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Jan 2024.