Inhaled Tranexamic Acid for Bleeding in Cancer Patients

Overseen By Nisha Rathi, MD

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: M.D. Anderson Cancer Center

Must not be taking: Anti-epileptics, Anti-fibrinolytics

Disqualifiers: DNR, 100% FIO2, COVID-19, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 5 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 3-5 days

60 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you cannot participate if you are on anti-fibrinolytic therapy (medications that prevent blood clots from breaking down).

What data supports the effectiveness of the drug Tranexamic Acid for bleeding in cancer patients?

Research suggests that inhaled tranexamic acid can quickly control bleeding in patients with non-massive hemoptysis (coughing up blood), which is a common issue in cancer patients with respiratory involvement. Additionally, tranexamic acid is known to reduce blood loss in surgical settings, indicating its potential to manage bleeding effectively. ^{1 2 3 4 5}

Is inhaled tranexamic acid safe for cancer patients?

Tranexamic acid (TXA) has been studied for safety in various conditions, including cancer patients undergoing chemotherapy or surgery. In a study with cancer patients, TXA did not significantly increase serious bleeding or thrombotic events (blood clots), and common side effects were similar to those in the placebo group, including diarrhea, fatigue, and nausea. ^{1 2 3 6 7}

How is the drug tranexamic acid unique for treating bleeding in cancer patients?

Tranexamic acid (TXA) is unique because it can be inhaled, offering a noninvasive option for cancer patients with bleeding in the respiratory system, unlike other treatments that may require invasive procedures. ^{1 2 3 8 9}

What is the purpose of this trial?

This is a drug study that will examine if inhaled tranexamic acid can improve mortality in patients with cancer-related pulmonary hemorrhage and respiratory failure as compared to usual care.

Research Team

Nisha Rathi, MD

Principal Investigator

M.D. Anderson Cancer Center

Eligibility Criteria

This trial is for adults over 18 with blood cancers who are on mechanical ventilation due to lung bleeding. They must be expected to live more than 48 hours, not have a severe brain bleed, no DNR orders, and not be pregnant or have COVID-19. People with recent blood clots, known allergies to tranexamic acid, color vision issues, seizures under treatment, or prior use of inhaled tranexamic acid can't join.

Inclusion Criteria

Evidence of diffuse alveolar hemorrhage by bronchoscopic examination

Persistently bloody secretions upon endotracheal tube suctioning

I have signed the consent form, or my legal representative has.

Exclusion Criteria

You have a nose or mouth that is bleeding and causing bloody secretions.

I am currently taking medication for epilepsy.

I am currently infected with COVID-19.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

1-2 weeks

Treatment

Participants receive inhaled tranexamic acid or usual care for pulmonary hemorrhage

3-5 days

Daily administration

Follow-up

Participants are monitored for safety and effectiveness after treatment

28 days

Extended Follow-up

Monitoring of secondary outcomes such as ICU and hospital mortality rate, ventilator days, and length of stay

100 days

Treatment Details

Interventions

• Tranexamic Acid
• Usual Care

Trial Overview The study tests if breathing in the drug tranexamic acid helps reduce death rates in cancer patients with lung bleeding compared to standard treatments. Patients will either receive the usual care or the added inhalation therapy and their survival outcomes will be compared.

Participant Groups

Group I: Group B: Usual Care Experimental Treatment 1 Intervention

usual care

Group II: Group A: Inhaled tranexamic acid Experimental Treatment 1 Intervention

will receive 500 mg/5ml nebulized tranexamic acid every 8 hours for at least 3 days, and up to 5 days

Tranexamic Acid is already approved in United States, European Union, Canada, Japan for the following indications:

Approved in United States as Tranexamic Acid for:

• Heavy menstrual bleeding
• Prevention of excessive bleeding during surgeries

Approved in European Union as Tranexamic Acid for:

• Heavy menstrual bleeding
• Prevention of excessive bleeding during surgeries
• Hereditary angioedema

Approved in Canada as Tranexamic Acid for:

• Heavy menstrual bleeding
• Prevention of excessive bleeding during surgeries

Approved in Japan as Tranexamic Acid for:

• Heavy menstrual bleeding
• Prevention of excessive bleeding during surgeries

Who Is Running the Clinical Trial?

M.D. Anderson Cancer Center

Lead Sponsor

Trials

3,107

Recruited

1,813,000+

Findings from Research

A case study demonstrated that nebulized tranexamic acid (TXA) effectively resolved significant hemoptysis in a patient with an invasive airway malignancy within 15 minutes, indicating its rapid action.

The use of nebulized TXA appears to be a safe and noninvasive option for managing hemoptysis, suggesting its potential as a palliative treatment for patients experiencing chronic or acute bleeding.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Nebulized Tranexamic Acid as a Noninvasive Therapy for Cancer-Related Hemoptysis. Hankerson, MJ., Raffetto, B., Mallon, WK., et al.[2015]

In a review of five studies involving 20,047 patients, tranexamic acid (TXA) significantly reduced short-term mortality in patients with hemoptysis, with a risk ratio of 0.78, indicating a 22% lower risk of death compared to controls.

TXA also led to shorter bleeding times and hospital stays, and reduced the need for further medical interventions, all without increasing the risk of major or minor adverse effects.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Does tranexamic acid reduce risk of mortality on patients with hemoptysis?: A protocol for systematic review and meta-analysis. Chen, LF., Wang, TC., Lin, TY., et al.[2023]

In a study of 282 cancer patients undergoing hip or knee arthroplasty, the use of tranexamic acid (TXA) did not increase the risk of complications such as venous thromboembolism within 90 days or 1 year, suggesting it is safe for this population.

TXA was associated with a trend towards lower risk of complications, indicating that it may be beneficial for reducing blood loss in cancer patients undergoing surgery without compromising their safety.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Tranexamic acid in patients with current or former cancer undergoing hip and knee arthroplasty. Varady, NH., Chen, AF., Drayer, NJ., et al.[2021]

Inhaled tranexamic acid (TXA) is effective in quickly controlling bleeding in patients with non-massive haemoptysis, based on the best available evidence from a review of 34 papers.

The review highlights that nebulized TXA leads to a fast resolution of bleeding, suggesting it could be a beneficial treatment option for this condition.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

BET 1: Does inhaled tranexamic acid reduce morbidity in adults with haemoptysis? Mervau, J., Jason, S., Jones, JS.[2020]

In a study of 83 patients undergoing spinal tumor surgery, the use of tranexamic acid (TXA) did not significantly reduce overall, intraoperative, or postoperative blood loss, indicating its moderate efficacy in this context.

However, the TXA group experienced a significant reduction in the volume of blood transfusions needed, without any increase in postoperative complications, suggesting that TXA may help minimize the need for transfusions during surgery.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Blood loss and perioperative transfusions related to surgery for spinal tumors. Relevance of tranexamic acid. Damade, C., Tesson, G., Gilard, V., et al.[2020]

In a clinical trial involving 356 patients with hematologic malignancies, prophylactic use of tranexamic acid (TXA) did not significantly reduce the incidence of WHO grade ≥2 bleeding compared to a placebo, with bleeding observed in 50.3% of the TXA group and 54.2% of the placebo group.

There were no significant differences in platelet transfusions, days alive without bleeding, or serious adverse events between the TXA and placebo groups, indicating that TXA may not be effective for preventing bleeding in this patient population.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Prophylactic tranexamic acid in patients with hematologic malignancy: a placebo-controlled, randomized clinical trial. Gernsheimer, TB., Brown, SP., Triulzi, DJ., et al.[2023]

In a study of 164 patients undergoing total hip replacement, a single injection of 30 mg/kg tranexamic acid (TXA) was found to be as effective as a continuous infusion in reducing blood loss, with no significant difference in mean blood loss between the two methods.

Both treatment methods were safe, with no reported deaths or major thromboembolic events within 90 days post-surgery, suggesting that the single injection is a practical and effective option for routine use in these patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Single tranexamic acid dose to reduce perioperative morbidity in primary total hip replacement: a randomised clinical trial. Hourlier, H., Fennema, P.[2023]

Locally injected tranexamic acid (TXA) at a concentration of 1 mg/mL significantly improved the clarity of the surgical field during subcutaneous tumor excisions, especially in patients on antithrombotic drugs, leading to better surgical outcomes.

The use of TXA also reduced the incidence of minor postoperative complications, such as hematomas and infections, and shortened hospital stays by allowing for quicker drain removal.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy of local infiltration of tranexamic acid in subcutaneous surgery: A protocol from a single medical center. Chang, YJ., Lin, YH., Wang, PL., et al.[2023]

A systematic review of 57 studies involving 5,049 patients found that the antifibrinolytic drug tranexamic acid (TXA) has a low frequency of thrombotic events, with less than 1% incidence of limb ischaemia and myocardial infarction.

For patients treated with TXA or epsilonaminocaproic acid (EACA) after spontaneous bleeding, the occurrence of deep vein thrombosis or pulmonary embolism was 1.9% and 3.0%, respectively, indicating that these drugs are generally safe, especially outside of subarachnoid haemorrhage cases.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The frequency of thrombotic events among adults given antifibrinolytic drugs for spontaneous bleeding: systematic review and meta-analysis of observational studies and randomized trials. Ross, J., Al-Shahi Salman, R.[2022]

References

1. United States pubmed.ncbi.nlm.nih.gov

Nebulized Tranexamic Acid as a Noninvasive Therapy for Cancer-Related Hemoptysis. [2015]

2. United States pubmed.ncbi.nlm.nih.gov

Does tranexamic acid reduce risk of mortality on patients with hemoptysis?: A protocol for systematic review and meta-analysis. [2023]

3. United States pubmed.ncbi.nlm.nih.gov

Tranexamic acid in patients with current or former cancer undergoing hip and knee arthroplasty. [2021]

4. England pubmed.ncbi.nlm.nih.gov

BET 1: Does inhaled tranexamic acid reduce morbidity in adults with haemoptysis? [2020]

5. France pubmed.ncbi.nlm.nih.gov

Blood loss and perioperative transfusions related to surgery for spinal tumors. Relevance of tranexamic acid. [2020]

6. United States pubmed.ncbi.nlm.nih.gov

Prophylactic tranexamic acid in patients with hematologic malignancy: a placebo-controlled, randomized clinical trial. [2023]

7. United States pubmed.ncbi.nlm.nih.gov

Single tranexamic acid dose to reduce perioperative morbidity in primary total hip replacement: a randomised clinical trial. [2023]

8. United States pubmed.ncbi.nlm.nih.gov

Efficacy of local infiltration of tranexamic acid in subcutaneous surgery: A protocol from a single medical center. [2023]

9. United Arab Emirates pubmed.ncbi.nlm.nih.gov

The frequency of thrombotic events among adults given antifibrinolytic drugs for spontaneous bleeding: systematic review and meta-analysis of observational studies and randomized trials. [2022]