A Phase 1 interventional study of Betrixaban in VTE Prophylaxis, sponsored by Alexion Pharmaceuticals, Inc.. Terminated at 20 sites in 4 countries. Open to participants aged 2 Years to 17 Years. Per ClinicalTrials.gov, last updated 2024-01-12.
Sponsored by Alexion Pharmaceuticals, Inc. (part of AstraZeneca) · Phase 1, Interventional, and Treatment
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).
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.
Alexion Pharmaceuticals, Inc. is the lead sponsor of 255 studies on the registry; 23 are open to participants now.
Of its 101 completed or terminated interventional studies of FDA-regulated products, 75 (74%) have results posted.
First submitted before Alexion Pharmaceuticals, Inc. became part of AstraZeneca.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Pediatric participant who was assessed to be at risk for VTE but did not require immediate anticoagulant therapy, for example:
Exclusion Criteria:
Participants who meet any one of the following exclusion criteria were excluded from the study:
Participant had hepatic disease associated with one or more of the following:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Blood tests of how long blood takes to clot. The INR is the prothrombin time (PT) put on a common scale; about 1 is usual for someone not taking a blood thinner.
Criteria often make an exception for people on a blood thinner such as warfarin, whose INR is raised on purpose.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
INR, PT and aPTT (clotting tests)
Blood tests of how long blood takes to clot. The INR is the prothrombin time (PT) put on a common scale; about 1 is usual for someone not taking a blood thinner.
Criteria often make an exception for people on a blood thinner such as warfarin, whose INR is raised on purpose.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
QT and QTc
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECG (electrocardiogram)
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GFR and eGFR (kidney filtering)
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Participants received a single, oral dose of betrixaban at 40 mg in a fed state, and had 10 PK blood sampling time points.
Drug: Betrixaban
Participants received a single, oral dose of betrixaban at 80 mg in a fed state, and had 5 PK sampling time points.
Drug: Betrixaban
Factor Xa inhibitor.
Also known as: PRT054021
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.
Time frame: 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.
Time frame: Up to 6 days post dose
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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: Up to 7 days post dose
| Milestone | Cohort 1: Betrixaban 40 mg | Cohort 2: Betrixaban 80 mg |
|---|---|---|
| Started | 3 | 18 |
| Received at least 1 dose of study drug | 3 | 18 |
| Evaluated through day 7 follow-up | 3 | 18 |
| Completed | 3 | 18 |
| Not completed | 0 | 0 |
Following the Sponsor's decision to cease developing betrixaban, data for AUC(0-inf) were not collected.
No measurements were reported for this outcome.
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.
| nanograms (ng)/milliliters (mL) | Cohort 1: Betrixaban 40 mg | Cohort 2: Betrixaban 80 mg |
|---|---|---|
| Maximum Observed Plasma Concentration (Cmax) Of Betrixaban | NA (0.200 to 2.57) | NA (0.200 to 48.5) |
Following the Sponsor's decision to cease developing betrixaban, data for AUC(0-last) were not collected.
No measurements were reported for this outcome.
Following the Sponsor's decision to cease developing betrixaban, data for t½ were not collected.
No measurements were reported for this outcome.
The Tmax that the highest (maximum) Cmax of betrixaban was observed per group up to Day 6 (120 hours) post dosing is reported.
| hours | Cohort 1: Betrixaban 40 mg | Cohort 2: Betrixaban 80 mg |
|---|---|---|
| Time To Maximum Observed Plasma Concentration (Tmax) Of Betrixaban | 2 (1 to 6) | 2 (1 to 12) |
Following the Sponsor's decision to cease developing betrixaban, data for CL were not collected.
No measurements were reported for this outcome.
Following the Sponsor's decision to cease developing betrixaban, data for Vd were not collected.
No measurements were reported for this outcome.
Following the Sponsor's decision to cease developing betrixaban, data for thrombin levels were not collected.
No measurements were reported for this outcome.
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.
| Participants | Cohort 1: Betrixaban 40 mg | Cohort 2: Betrixaban 80 mg |
|---|---|---|
| Count Of Participants With Treatment-related Adverse Events | 0 | 1 |
Collected over Up to 7 days post dose.. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Cohort 1: Betrixaban 40 mg | 0/3 (0%) | 0/3 (0%) | 0/3 (0%) |
| Cohort 2: Betrixaban 80 mg | 0/18 (0%) | 0/18 (0%) | 5/18 (27.8%) |
| Event | Cohort 1: Betrixaban 40 mg | Cohort 2: Betrixaban 80 mg |
|---|---|---|
| HypertensionVascular disorders | 0/3 | 1/18 |
| HypotensionVascular disorders | 0/3 | 1/18 |
| BradycardiaCardiac disorders | 0/3 | 1/18 |
| HeadacheNervous system disorders | 0/3 | 1/18 |
| Abdominal discomfortGastrointestinal disorders | 0/3 | 1/18 |
| Abdominal pain upperGastrointestinal disorders | 0/3 | 1/18 |
| SinusitisInfections and infestations | 0/3 | 1/18 |
Enrolled population: all participants enrolled in Part 1 of the study.
| Age, Customized(Participants) | Cohort 1: Betrixaban 40 mg | Cohort 2: Betrixaban 80 mg | Total |
|---|---|---|---|
| Age Categorical — In utero | 0 | 0 | 0 |
| Age Categorical — Preterm newborn - gestational age < 37 wk | 0 | 0 | 0 |
| Age Categorical — Newborns (0-27 days) | 0 | 0 | 0 |
| Age Categorical — Children (2-11 years) | 0 | 0 | 0 |
| Age Categorical — Adolescents (12-17 years) | 3 | 18 | 21 |
| Age Categorical — Adults (18-64 years) | 0 | 0 | 0 |
| Age Categorical — From 65 to 84 years | 0 | 0 | 0 |
| Age Categorical — 85 years and over | 0 | 0 | 0 |
| Sex: Female, Male(Participants) | Cohort 1: Betrixaban 40 mg | Cohort 2: Betrixaban 80 mg | Total |
|---|---|---|---|
| Female | 2 | 9 | 11 |
| Male | 1 | 9 | 10 |
| Ethnicity (NIH/OMB)(Participants) | Cohort 1: Betrixaban 40 mg | Cohort 2: Betrixaban 80 mg | Total |
|---|---|---|---|
| Hispanic or Latino | 0 | 0 | 0 |
| Not Hispanic or Latino | 3 | 17 | 20 |
| Unknown or Not Reported | 0 | 1 | 1 |
| Race (NIH/OMB)(Participants) | Cohort 1: Betrixaban 40 mg | Cohort 2: Betrixaban 80 mg | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 |
| Asian | 0 | 0 | 0 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 0 | 2 | 2 |
| White | 3 | 15 | 18 |
| More than one race | 0 | 0 | 0 |
| Unknown or Not Reported | 0 | 1 | 1 |
| Region of Enrollment(participants) | Cohort 1: Betrixaban 40 mg | Cohort 2: Betrixaban 80 mg | Total |
|---|---|---|---|
| United Kingdom | 0 | 1 | 1 |
| Ukraine | 0 | 2 | 2 |
| Russia | 3 | 11 | 14 |
| United States | 0 | 4 | 4 |
PDFs hosted by the registry, ClinicalTrials.gov.
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Studies of the drug tested here: Betrixaban
Alexion Pharmaceuticals, Inc. →