Trial Outcomes & Findings for Tranexamic Acid Use to Reduce Blood Transfusion in Pediatric Cancer Patients Undergoing Limb Salvage Procedure (NCT NCT04410042)
NCT ID: NCT04410042
Last Updated: 2024-12-20
Results Overview
The intra-or post-operative volumes of transfused blood for both the TXA treated group and placebo group will be estimated with a two-sided 95% confidence interval. The blood volumes transfused per kilogram of body weight of the two groups (TXA vs. Placebo) will be evaluated using a two-sided student's t-test after log(1+x) transformation.
Recruitment status
TERMINATED
Study phase
PHASE3
Target enrollment
15 participants
Primary outcome timeframe
intra- or post-operatively transfused blood volume (mL/kg), 6 months
Results posted on
2024-12-20
Participant Flow
As of the data freeze date (12/14/2023), 15 patients are enrolled and 13 of them are eligible. The ineligible reason for one patient is ANC did not meet eligible levels prior to surgery. The second ineligible patient was found to be on oral estrogen contraception after receiving study drug and undergoing surgery. One patient was consented, then consent was withdrawn prior to any study procedures. This patient was not randomized.
Participant milestones
| Measure |
Arm A-Tranexamic Acid
At initiation of surgical preparation, participants randomized to the active treatment arm will receive tranexamic acid 10 mg/kg (max 1 g), given via syringe pump programmed to infuse over 15 minutes. If no unacceptable toxicities occur, a second dose of tranexamic acid IV push over 5 to 15 minutes will be given 6 hours (with a window of +/- 30 minutes) after the first dose (either intra- or post-operatively).
Tranexamic Acid: Given IV
|
Arm B-Placebo
At initiation of surgical preparation, participants randomized to the placebo treatment arm will receive 0.9% sodium chloride (salt water). It will be matched in appearance, volume, and administration to the active treatment arm with tranexamic acid. If no unacceptable toxicities occur, a second dose of placebo IV push over 5 to 15 minutes will be given 6 hours (with a window of +/- 30 minutes) after the first dose (either intra- or post-operatively).
0.9% sodium chloride: Given IV
|
|---|---|---|
|
Overall Study
STARTED
|
7
|
7
|
|
Overall Study
COMPLETED
|
6
|
6
|
|
Overall Study
NOT COMPLETED
|
1
|
1
|
Reasons for withdrawal
| Measure |
Arm A-Tranexamic Acid
At initiation of surgical preparation, participants randomized to the active treatment arm will receive tranexamic acid 10 mg/kg (max 1 g), given via syringe pump programmed to infuse over 15 minutes. If no unacceptable toxicities occur, a second dose of tranexamic acid IV push over 5 to 15 minutes will be given 6 hours (with a window of +/- 30 minutes) after the first dose (either intra- or post-operatively).
Tranexamic Acid: Given IV
|
Arm B-Placebo
At initiation of surgical preparation, participants randomized to the placebo treatment arm will receive 0.9% sodium chloride (salt water). It will be matched in appearance, volume, and administration to the active treatment arm with tranexamic acid. If no unacceptable toxicities occur, a second dose of placebo IV push over 5 to 15 minutes will be given 6 hours (with a window of +/- 30 minutes) after the first dose (either intra- or post-operatively).
0.9% sodium chloride: Given IV
|
|---|---|---|
|
Overall Study
Not Eligible
|
1
|
1
|
Baseline Characteristics
Tranexamic Acid Use to Reduce Blood Transfusion in Pediatric Cancer Patients Undergoing Limb Salvage Procedure
Baseline characteristics by cohort
| Measure |
Arm A-Tranexamic Acid
n=6 Participants
At initiation of surgical preparation, participants randomized to the active treatment arm will receive tranexamic acid 10 mg/kg (max 1 g), given via syringe pump programmed to infuse over 15 minutes. If no unacceptable toxicities occur, a second dose of tranexamic acid IV push over 5 to 15 minutes will be given 6 hours (with a window of +/- 30 minutes) after the first dose (either intra- or post-operatively).
Tranexamic Acid: Given IV
|
Arm B-Placebo
n=6 Participants
At initiation of surgical preparation, participants randomized to the placebo treatment arm will receive 0.9% sodium chloride (salt water). It will be matched in appearance, volume, and administration to the active treatment arm with tranexamic acid. If no unacceptable toxicities occur, a second dose of placebo IV push over 5 to 15 minutes will be given 6 hours (with a window of +/- 30 minutes) after the first dose (either intra- or post-operatively).
0.9% sodium chloride: Given IV
|
Total
n=12 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
16.40 years
STANDARD_DEVIATION 2.47 • n=5 Participants
|
13.77 years
STANDARD_DEVIATION 3.25 • n=7 Participants
|
15.08 years
STANDARD_DEVIATION 3.07 • n=5 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
7 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
White
|
5 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
10 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Asian
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: intra- or post-operatively transfused blood volume (mL/kg), 6 monthsThe intra-or post-operative volumes of transfused blood for both the TXA treated group and placebo group will be estimated with a two-sided 95% confidence interval. The blood volumes transfused per kilogram of body weight of the two groups (TXA vs. Placebo) will be evaluated using a two-sided student's t-test after log(1+x) transformation.
Outcome measures
| Measure |
Arm A-Tranexamic Acid
n=6 Participants
At initiation of surgical preparation, participants randomized to the active treatment arm will receive tranexamic acid 10 mg/kg (max 1 g), given via syringe pump programmed to infuse over 15 minutes. If no unacceptable toxicities occur, a second dose of tranexamic acid IV push over 5 to 15 minutes will be given 6 hours (with a window of +/- 30 minutes) after the first dose (either intra- or post-operatively).
Tranexamic Acid: Given IV
|
Arm B-Placebo
n=6 Participants
At initiation of surgical preparation, participants randomized to the placebo treatment arm will receive 0.9% sodium chloride (salt water). It will be matched in appearance, volume, and administration to the active treatment arm with tranexamic acid. If no unacceptable toxicities occur, a second dose of placebo IV push over 5 to 15 minutes will be given 6 hours (with a window of +/- 30 minutes) after the first dose (either intra- or post-operatively).
0.9% sodium chloride: Given IV
|
TXAKIDS
n=12 Participants
Eligible participants undergoing limb salvage procedures will be randomized to receive either TXA or placebo peri-operatively - one dose at initiation of surgical preparation and a second dose 6 hours after the first.
|
|---|---|---|---|
|
To Evaluate the Difference in intra-or Post-operatively Transfused Blood Volume (mL/kg) for Patients Undergoing Limb Salvage Procedures of the Distal Femur or Proximal Tibia Who Are Randomized to Receive Perioperative Tranexamic Acid (TXA) Versus Placebo.
Transfused blood volume, intra-operative (intra-op)
|
5.04 mL/kg
Standard Deviation 2.34
|
5.15 mL/kg
Standard Deviation 2.85
|
5.12 mL/kg
Standard Deviation 2.51
|
|
To Evaluate the Difference in intra-or Post-operatively Transfused Blood Volume (mL/kg) for Patients Undergoing Limb Salvage Procedures of the Distal Femur or Proximal Tibia Who Are Randomized to Receive Perioperative Tranexamic Acid (TXA) Versus Placebo.
Transfused blood volume, post-operative (post-op)
|
5.74 mL/kg
Standard Deviation 1.79
|
5.64 mL/kg
Standard Deviation 2.06
|
5.71 mL/kg
Standard Deviation 1.85
|
SECONDARY outcome
Timeframe: changes in platelets from pre-op to post-op level, 6 monthsSummary statistics will be provided for the changes in platelet level for both the TXA and placebo group. Two sample t-test or Wilcoxon rank sum test will be used to compare the differences between the two groups. Multiple comparison correction might be used for p-values to address the multiple testing issues due to measurements at multiple time points.
Outcome measures
| Measure |
Arm A-Tranexamic Acid
n=6 Participants
At initiation of surgical preparation, participants randomized to the active treatment arm will receive tranexamic acid 10 mg/kg (max 1 g), given via syringe pump programmed to infuse over 15 minutes. If no unacceptable toxicities occur, a second dose of tranexamic acid IV push over 5 to 15 minutes will be given 6 hours (with a window of +/- 30 minutes) after the first dose (either intra- or post-operatively).
Tranexamic Acid: Given IV
|
Arm B-Placebo
n=6 Participants
At initiation of surgical preparation, participants randomized to the placebo treatment arm will receive 0.9% sodium chloride (salt water). It will be matched in appearance, volume, and administration to the active treatment arm with tranexamic acid. If no unacceptable toxicities occur, a second dose of placebo IV push over 5 to 15 minutes will be given 6 hours (with a window of +/- 30 minutes) after the first dose (either intra- or post-operatively).
0.9% sodium chloride: Given IV
|
TXAKIDS
n=12 Participants
Eligible participants undergoing limb salvage procedures will be randomized to receive either TXA or placebo peri-operatively - one dose at initiation of surgical preparation and a second dose 6 hours after the first.
|
|---|---|---|---|
|
To Evaluate Changes in Platelets From Pre-op to Post-op Level for Patients Randomized to Receive Perioperative TXA Versus Placebo.
Platelets (intra-op)
|
227.00 10e3 platelets/mm^3
Standard Deviation 98.04
|
239.84 10e3 platelets/mm^3
Standard Deviation 75.99
|
233.42 10e3 platelets/mm^3
Standard Deviation 83.90
|
|
To Evaluate Changes in Platelets From Pre-op to Post-op Level for Patients Randomized to Receive Perioperative TXA Versus Placebo.
Platelets (post-op)
|
162.33 10e3 platelets/mm^3
Standard Deviation 25.74
|
168.83 10e3 platelets/mm^3
Standard Deviation 73.20
|
165.58 10e3 platelets/mm^3
Standard Deviation 52.42
|
SECONDARY outcome
Timeframe: changes in hemoglobin from pre-op to post-op level, 6 monthsSummary statistics will be provided for the decline in hemoglobin from pre-op to post-op level, for both the TXA and placebo group. Two sample t-test or Wilcoxon rank sum test will be used to compare the differences between the two groups. Multiple comparison correction might be used for p-values to address the multiple testing issues due to measurements at multiple time points.
Outcome measures
| Measure |
Arm A-Tranexamic Acid
n=6 Participants
At initiation of surgical preparation, participants randomized to the active treatment arm will receive tranexamic acid 10 mg/kg (max 1 g), given via syringe pump programmed to infuse over 15 minutes. If no unacceptable toxicities occur, a second dose of tranexamic acid IV push over 5 to 15 minutes will be given 6 hours (with a window of +/- 30 minutes) after the first dose (either intra- or post-operatively).
Tranexamic Acid: Given IV
|
Arm B-Placebo
n=6 Participants
At initiation of surgical preparation, participants randomized to the placebo treatment arm will receive 0.9% sodium chloride (salt water). It will be matched in appearance, volume, and administration to the active treatment arm with tranexamic acid. If no unacceptable toxicities occur, a second dose of placebo IV push over 5 to 15 minutes will be given 6 hours (with a window of +/- 30 minutes) after the first dose (either intra- or post-operatively).
0.9% sodium chloride: Given IV
|
TXAKIDS
n=12 Participants
Eligible participants undergoing limb salvage procedures will be randomized to receive either TXA or placebo peri-operatively - one dose at initiation of surgical preparation and a second dose 6 hours after the first.
|
|---|---|---|---|
|
To Evaluate Changes in Hemoglobin From Pre-op to Post-op Level for Patients Randomized to Receive Perioperative TXA Versus Placebo.
Hemoglobin (intra-op)
|
11.70 g/dL
Standard Deviation 2.13
|
11.00 g/dL
Standard Deviation 1.64
|
11.35 g/dL
Standard Deviation 1.85
|
|
To Evaluate Changes in Hemoglobin From Pre-op to Post-op Level for Patients Randomized to Receive Perioperative TXA Versus Placebo.
Hemoglobin (post-op)
|
8.23 g/dL
Standard Deviation 1.30
|
7.77 g/dL
Standard Deviation 0.70
|
8.00 g/dL
Standard Deviation 1.02
|
SECONDARY outcome
Timeframe: Conclusion of surgery to time of drain removal (prior to discharge from inpatient)Summary statistics will be provided for postoperative daily surgical drain output (in milliliters per 24 hour period for the duration of the drain) for each group. The group difference will be compared using two-sample t-test or Wilcoxon rank sum test depending on the distribution of the observed data. Multiple comparison correction might be used for p-values to address the multiple testing issues due to measurements at multiple time points.
Outcome measures
| Measure |
Arm A-Tranexamic Acid
n=6 Participants
At initiation of surgical preparation, participants randomized to the active treatment arm will receive tranexamic acid 10 mg/kg (max 1 g), given via syringe pump programmed to infuse over 15 minutes. If no unacceptable toxicities occur, a second dose of tranexamic acid IV push over 5 to 15 minutes will be given 6 hours (with a window of +/- 30 minutes) after the first dose (either intra- or post-operatively).
Tranexamic Acid: Given IV
|
Arm B-Placebo
n=6 Participants
At initiation of surgical preparation, participants randomized to the placebo treatment arm will receive 0.9% sodium chloride (salt water). It will be matched in appearance, volume, and administration to the active treatment arm with tranexamic acid. If no unacceptable toxicities occur, a second dose of placebo IV push over 5 to 15 minutes will be given 6 hours (with a window of +/- 30 minutes) after the first dose (either intra- or post-operatively).
0.9% sodium chloride: Given IV
|
TXAKIDS
n=12 Participants
Eligible participants undergoing limb salvage procedures will be randomized to receive either TXA or placebo peri-operatively - one dose at initiation of surgical preparation and a second dose 6 hours after the first.
|
|---|---|---|---|
|
To Evaluate Differences in Post-operative Daily Surgical Drain Output for Patients Randomized to Receive Perioperative TXA Versus Placebo.
|
246.00 ml
Standard Deviation 146.81
|
83.20 ml
Standard Deviation 79.43
|
144.25 ml
Standard Deviation 129.86
|
SECONDARY outcome
Timeframe: At conclusion of surgeryThe EBL for pre-op to post-op level, for both the TXA treated group and placebo group will be estimated with a two-sided 95% confidence interval. The EBL of the two groups (TXA vs. Placebo) will be evaluated using a two-sample t-test or Wilcoxon rank sum test depending on the distribution of the observed data.
Outcome measures
| Measure |
Arm A-Tranexamic Acid
n=6 Participants
At initiation of surgical preparation, participants randomized to the active treatment arm will receive tranexamic acid 10 mg/kg (max 1 g), given via syringe pump programmed to infuse over 15 minutes. If no unacceptable toxicities occur, a second dose of tranexamic acid IV push over 5 to 15 minutes will be given 6 hours (with a window of +/- 30 minutes) after the first dose (either intra- or post-operatively).
Tranexamic Acid: Given IV
|
Arm B-Placebo
n=6 Participants
At initiation of surgical preparation, participants randomized to the placebo treatment arm will receive 0.9% sodium chloride (salt water). It will be matched in appearance, volume, and administration to the active treatment arm with tranexamic acid. If no unacceptable toxicities occur, a second dose of placebo IV push over 5 to 15 minutes will be given 6 hours (with a window of +/- 30 minutes) after the first dose (either intra- or post-operatively).
0.9% sodium chloride: Given IV
|
TXAKIDS
n=12 Participants
Eligible participants undergoing limb salvage procedures will be randomized to receive either TXA or placebo peri-operatively - one dose at initiation of surgical preparation and a second dose 6 hours after the first.
|
|---|---|---|---|
|
To Evaluate Changes in Estimated Blood Loss (EBL) for Patients Randomized to Receive Perioperative TXA Versus Placebo.
|
669.67 ml
Standard Deviation 551.15
|
808.67 ml
Standard Deviation 607.53
|
739.17 ml
Standard Deviation 557.78
|
SECONDARY outcome
Timeframe: At conclusion of surgeryMean and Standard Deviation of log Transfused Blood Volume for both the TXA treated group and placebo group will be estimated.
Outcome measures
| Measure |
Arm A-Tranexamic Acid
n=6 Participants
At initiation of surgical preparation, participants randomized to the active treatment arm will receive tranexamic acid 10 mg/kg (max 1 g), given via syringe pump programmed to infuse over 15 minutes. If no unacceptable toxicities occur, a second dose of tranexamic acid IV push over 5 to 15 minutes will be given 6 hours (with a window of +/- 30 minutes) after the first dose (either intra- or post-operatively).
Tranexamic Acid: Given IV
|
Arm B-Placebo
n=6 Participants
At initiation of surgical preparation, participants randomized to the placebo treatment arm will receive 0.9% sodium chloride (salt water). It will be matched in appearance, volume, and administration to the active treatment arm with tranexamic acid. If no unacceptable toxicities occur, a second dose of placebo IV push over 5 to 15 minutes will be given 6 hours (with a window of +/- 30 minutes) after the first dose (either intra- or post-operatively).
0.9% sodium chloride: Given IV
|
TXAKIDS
n=12 Participants
Eligible participants undergoing limb salvage procedures will be randomized to receive either TXA or placebo peri-operatively - one dose at initiation of surgical preparation and a second dose 6 hours after the first.
|
|---|---|---|---|
|
To Evaluate Log Transfused Blood Volume Between the intra-or Post-operatively for Patients Randomized to Receive Perioperative TXA and Placebo, Respectively.
|
1.68 log(mL/kg)
Standard Deviation 0.32
|
1.67 log(mL/kg)
Standard Deviation 0.38
|
1.68 log(mL/kg)
Standard Deviation 0.34
|
SECONDARY outcome
Timeframe: At conclusion of surgeryMean and Standard Deviation of log Estimated Blood Loss for both the TXA treated group and placebo group will be estimated.
Outcome measures
| Measure |
Arm A-Tranexamic Acid
n=6 Participants
At initiation of surgical preparation, participants randomized to the active treatment arm will receive tranexamic acid 10 mg/kg (max 1 g), given via syringe pump programmed to infuse over 15 minutes. If no unacceptable toxicities occur, a second dose of tranexamic acid IV push over 5 to 15 minutes will be given 6 hours (with a window of +/- 30 minutes) after the first dose (either intra- or post-operatively).
Tranexamic Acid: Given IV
|
Arm B-Placebo
n=6 Participants
At initiation of surgical preparation, participants randomized to the placebo treatment arm will receive 0.9% sodium chloride (salt water). It will be matched in appearance, volume, and administration to the active treatment arm with tranexamic acid. If no unacceptable toxicities occur, a second dose of placebo IV push over 5 to 15 minutes will be given 6 hours (with a window of +/- 30 minutes) after the first dose (either intra- or post-operatively).
0.9% sodium chloride: Given IV
|
TXAKIDS
n=12 Participants
Eligible participants undergoing limb salvage procedures will be randomized to receive either TXA or placebo peri-operatively - one dose at initiation of surgical preparation and a second dose 6 hours after the first.
|
|---|---|---|---|
|
To Evaluate Log Estimated Blood Loss (EBL) Between the intra-or Post-operatively for Patients Randomized to Receive Perioperative TXA and Placebo, Respectively.
|
6.11 log(mL)
Standard Deviation 0.67
|
6.77 log(mL)
Standard Deviation 0.56
|
6.38 log(mL)
Standard Deviation 0.70
|
SECONDARY outcome
Timeframe: At conclusion of surgeryRegression model will be used to access the correlation between the log transformed intra-or post-operatively transfused blood volume and EBL.
Outcome measures
| Measure |
Arm A-Tranexamic Acid
n=6 Participants
At initiation of surgical preparation, participants randomized to the active treatment arm will receive tranexamic acid 10 mg/kg (max 1 g), given via syringe pump programmed to infuse over 15 minutes. If no unacceptable toxicities occur, a second dose of tranexamic acid IV push over 5 to 15 minutes will be given 6 hours (with a window of +/- 30 minutes) after the first dose (either intra- or post-operatively).
Tranexamic Acid: Given IV
|
Arm B-Placebo
n=6 Participants
At initiation of surgical preparation, participants randomized to the placebo treatment arm will receive 0.9% sodium chloride (salt water). It will be matched in appearance, volume, and administration to the active treatment arm with tranexamic acid. If no unacceptable toxicities occur, a second dose of placebo IV push over 5 to 15 minutes will be given 6 hours (with a window of +/- 30 minutes) after the first dose (either intra- or post-operatively).
0.9% sodium chloride: Given IV
|
TXAKIDS
n=12 Participants
Eligible participants undergoing limb salvage procedures will be randomized to receive either TXA or placebo peri-operatively - one dose at initiation of surgical preparation and a second dose 6 hours after the first.
|
|---|---|---|---|
|
To Evaluate the Association Between the intra-or Post-operatively Transfused Blood Volume and Estimated Blood Loss (EBL) for Patients Randomized to Receive Perioperative TXA and Placebo, Respectively.
|
0.36 correlation coefficient
|
-0.42 correlation coefficient
|
0.03 correlation coefficient
|
Adverse Events
Arm A-Tranexamic Acid
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
Arm B-Placebo
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Arm A-Tranexamic Acid
n=6 participants at risk
At initiation of surgical preparation, participants randomized to the active treatment arm will receive tranexamic acid 10 mg/kg (max 1 g), given via syringe pump programmed to infuse over 15 minutes. If no unacceptable toxicities occur, a second dose of tranexamic acid IV push over 5 to 15 minutes will be given 6 hours (with a window of +/- 30 minutes) after the first dose (either intra- or post-operatively).
Tranexamic Acid: Given IV
|
Arm B-Placebo
n=6 participants at risk
At initiation of surgical preparation, participants randomized to the placebo treatment arm will receive 0.9% sodium chloride (salt water). It will be matched in appearance, volume, and administration to the active treatment arm with tranexamic acid. If no unacceptable toxicities occur, a second dose of placebo IV push over 5 to 15 minutes will be given 6 hours (with a window of +/- 30 minutes) after the first dose (either intra- or post-operatively).
0.9% sodium chloride: Given IV
|
|---|---|---|
|
Injury, poisoning and procedural complications
Injury, poisoning and procedural complications - Other
|
16.7%
1/6 • Adverse events were collected from Post-Anesthesia Care Unit (PACU) to 6 months.
|
0.00%
0/6 • Adverse events were collected from Post-Anesthesia Care Unit (PACU) to 6 months.
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
0.00%
0/6 • Adverse events were collected from Post-Anesthesia Care Unit (PACU) to 6 months.
|
16.7%
1/6 • Adverse events were collected from Post-Anesthesia Care Unit (PACU) to 6 months.
|
Additional Information
Michael D. Neel, MD
St. Jude Children's Research Hospital
Phone: 901-259-1600
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place