NCT04410042

Tranexamic Acid Use to Reduce Blood Transfusion in Pediatric Cancer Patients Undergoing Limb Salvage Procedure

Terminated Phase 3 Results posted Last updated 20 December 2024

What this trial tests

Phase 3 trial testing Tranexamic Acid in Cancer of the Bone in 15 participants. Terminated before completion.

Timeline

29 January 2021

Primary endpoint
16 January 2024

16 January 2024

Quick facts

Lead sponsor	St. Jude Children's Research Hospital
Phase	Phase 3
Status	Terminated
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	triple
Primary purpose	supportive care
Enrollment	15
Start date	29 January 2021
Primary completion	16 January 2024
Estimated completion	16 January 2024
Sites	1 location across United States

Drugs / interventions tested

• Tranexamic Acid (Tranexamic Acid) — full drug profile →
• 0.9% sodium chloride

Conditions studied

• Cancer of the Bone — all drugs for Cancer of the Bone →
• Limb Salvage — all drugs for Limb Salvage →

Sponsor

St. Jude Children's Research Hospital

Who can join

Under 24, any sex, with Cancer of the Bone or Limb Salvage. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

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. Primary · intra- or post-operatively transfused blood volume (mL/kg), 6 months

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.

Transfused blood volume, intra-operative (intra-op)

Group	Value	95% CI
Arm A-Tranexamic Acid	5.04	± 2.34
Arm B-Placebo	5.15	± 2.85
TXAKIDS	5.12	± 2.51

Transfused blood volume, post-operative (post-op)

Group	Value	95% CI
Arm A-Tranexamic Acid	5.74	± 1.79
Arm B-Placebo	5.64	± 2.06
TXAKIDS	5.71	± 1.85

To Evaluate Changes in Platelets From Pre-op to Post-op Level for Patients Randomized to Receive Perioperative TXA Versus Placebo. Secondary · changes in platelets from pre-op to post-op level, 6 months

Summary 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.

Platelets (intra-op)

Group	Value	95% CI
Arm A-Tranexamic Acid	227.00	± 98.04
Arm B-Placebo	239.84	± 75.99
TXAKIDS	233.42	± 83.90

Platelets (post-op)

Group	Value	95% CI
Arm A-Tranexamic Acid	162.33	± 25.74
Arm B-Placebo	168.83	± 73.20
TXAKIDS	165.58	± 52.42

To Evaluate Changes in Hemoglobin From Pre-op to Post-op Level for Patients Randomized to Receive Perioperative TXA Versus Placebo. Secondary · changes in hemoglobin from pre-op to post-op level, 6 months

Summary 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.

Hemoglobin (intra-op)

Group	Value	95% CI
Arm A-Tranexamic Acid	11.70	± 2.13
Arm B-Placebo	11.00	± 1.64
TXAKIDS	11.35	± 1.85

Hemoglobin (post-op)

Group	Value	95% CI
Arm A-Tranexamic Acid	8.23	± 1.30
Arm B-Placebo	7.77	± 0.70
TXAKIDS	8.00	± 1.02

To Evaluate Differences in Post-operative Daily Surgical Drain Output for Patients Randomized to Receive Perioperative TXA Versus Placebo. Secondary · 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.

Group	Value	95% CI
Arm A-Tranexamic Acid	246.00	± 146.81
Arm B-Placebo	83.20	± 79.43
TXAKIDS	144.25	± 129.86

To Evaluate Changes in Estimated Blood Loss (EBL) for Patients Randomized to Receive Perioperative TXA Versus Placebo. Secondary · At conclusion of surgery

The 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.

Group	Value	95% CI
Arm A-Tranexamic Acid	669.67	± 551.15
Arm B-Placebo	808.67	± 607.53
TXAKIDS	739.17	± 557.78

To Evaluate Log Transfused Blood Volume Between the intra-or Post-operatively for Patients Randomized to Receive Perioperative TXA and Placebo, Respectively. Secondary · At conclusion of surgery

Mean and Standard Deviation of log Transfused Blood Volume for both the TXA treated group and placebo group will be estimated.

Group	Value	95% CI
Arm A-Tranexamic Acid	1.68	± 0.32
Arm B-Placebo	1.67	± 0.38
TXAKIDS	1.68	± 0.34

To Evaluate Log Estimated Blood Loss (EBL) Between the intra-or Post-operatively for Patients Randomized to Receive Perioperative TXA and Placebo, Respectively. Secondary · At conclusion of surgery

Mean and Standard Deviation of log Estimated Blood Loss for both the TXA treated group and placebo group will be estimated.

Group	Value	95% CI
Arm A-Tranexamic Acid	6.11	± 0.67
Arm B-Placebo	6.77	± 0.56
TXAKIDS	6.38	± 0.70

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. Secondary · At conclusion of surgery

Regression model will be used to access the correlation between the log transformed intra-or post-operatively transfused blood volume and EBL.

Group	Value	95% CI
Arm A-Tranexamic Acid	0.36
Arm B-Placebo	-0.42
TXAKIDS	0.03

Adverse events — posted to ClinicalTrials.gov

Time frame: Adverse events were collected from Post-Anesthesia Care Unit (PACU) to 6 months.. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Arm A-Tranexamic Acid

Serious: 0/6 (0%)

Deaths: 0/6

Arm B-Placebo

Serious: 0/6 (0%)

Deaths: 0/6

Other adverse events (2 terms — click to expand)

Reaction	System	Arm A-Tranexamic Acid	Arm B-Placebo
Injury, poisoning and procedural complications - Other	Injury, poisoning and procedural complications	—	—
Pain in extremity	Musculoskeletal and connective tissue disorders	—	—

Data from ClinicalTrials.gov NCT04410042 adverse events section.

Sponsor's own description

This is a randomized double-blind control trial evaluating the use Tranexamic acid (TXA) to decrease blood loss and transfusion requirements in pediatric and young adult cancer patients undergoing a limb salvage procedure that frequently requires perioperative or post-operative transfusions of blood products.

Primary Objective

* 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.

Secondary Objectives

* To evaluate changes in platelets and in hemoglobin from pre-op to post-op level for patients randomized to receive perioperative TXA versus placebo.
* To evaluate differences in post-operative daily surgical drain output for patients randomized to receive perioperative TXA versus placebo.
* To evaluate changes in estimated blood loss (EBL) for patients randomized to receive perioperative TXA versus placebo.
* 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.

Exploratory Objectives

* To evaluate differences in functional outcomes post-operatively for patients randomized to receive perioperative TXA versus placebo.
* To explore if significant correlations are observed between parameters reported with rotational thromboelastometry (ROTEM®) and EBL and transfusion requirements in pediatric and young adult patients undergoing limb salvage procedure who are randomized to perioperative TXA versus placebo.
* To evaluate differences in the prevalence and management of wound complications such as superficial or periprosthetic infections, wound dehiscence, contact dermatitis, post- operative hematomas, or any other clinically significant wound complication between patients randomized to receive perioperative TXA versus placebo.

Publications & conference data

1 peer-reviewed publication reference this trial (live from Europe PMC):

1. Efficacy and Safety of Tranexamic Acid in Cancer Surgery. An Update of Clinical Findings and Ongoing Research.
   Zec T, Di Napoli R, Fievez L, Ben Aziz M, et al · · 2022 · cited 6× · PMID 35818514 · DOI 10.2147/jmdh.s337250