Hematologic NeoplasmsPhase 3PATHNCT04448184
Platelet Transfusions in Hematopoietic Stem Cell Transplantation (The PATH III Trial)
A Phase 3 study testing Tranexamic Acid in people with Hematologic Neoplasms, run by Ottawa Hospital Research Institute at 12 sites in Canada.
Trial summary
It is hypothesized that a strategy using prophylactic oral and intravenous Tranexamic Acid (TXA) with therapeutic platelet transfusions (if required) is safe and more effective than prophylactic platelet transfusions in patients undergoing an autologous hematopoietic stem cell transplantation (ASCT). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hematologic Neoplasms
- What is being tested?
- Tranexamic Acid
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 662 participants
- Where
- 12 sitesCanada
- Study dates
- 16 Feb 2022to Feb 2027
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Adults 18 years or older undergoing ASCT for a hematologic malignancy
- Patients providing written informed consent prior to starting transplantation
Who cannot join
- A previous WHO grade 2, 3 or 4 bleeding event within the past year
- A previous or current unprovoked thrombotic event defined as a pulmonary embolism, deep vein thrombosis, cerebral thrombosis
- A current provoked thrombotic event (e.g. catheter-related thrombosis) within last month and/or still requiring anticoagulant treatment.
- A requirement for therapeutic anticoagulant or anti-platelet drugs during ASCT
- Active angina (chest pain of presumed cardiac origin either at rest or with activity)
- Current or previous (within 2 weeks) urinary tract bleeding
Show all 14Show fewer
- An inherited hemostatic or thrombotic disorder
- Coagulopathy defined as a prothrombin time '/International Normalization Ratio (INR) or activated partial thromboplastin time more than 1.5 times the upper limit of normal or fibrinogen less than 2 g/L
- Previously documented history of refractoriness to platelet transfusion secondary to HLA antibodies (Refractoriness is defined as 2 consecutive ABO matched platelet transfusions with platelet increment of < 7.5 and the presence of anti-HLA antibodies)
- Significant renal impairment (creatinine more than 1.5 times the upper limit of normal or a eGFR less than 0.5 mL/min/1.78m2)
- Pregnant or breast-feeding
- Unwilling or unable to provide informed consent
- Participant has acquired disturbances to his/her colour vision (does not apply to congenital colour blindness)
- Participant has known sensitivity or allergy to Tranexamic Acid or any of its ingredients
Trial locations and contacts
12 sites in 1 country. Contact the study team or a site near you to ask about taking part.
12 sites in 1 country
Canada12 sites, 11 recruiting
- Recruiting
Tom Baker Cancer Centre
Calgary, Alberta, T2N4N2
Jason Tay, MD, 403-944-1880, Jason.Tay@ahs.ca
Jason Tay, MD
- Recruiting
Cross Cancer Institute
Edmonton, Alberta
Irwindeep Sandhu, MD
irwindeep.sandhu2@albertahealthservices.ca
- Not yet recruiting
Dalhousie University
Halifax, Nova Scotia
Mahmood El-Sawy, MD
- Recruiting
Hamilton Health Sciences - Juravinski Hospital and Cancer Centre
Hamilton, Ontario, L8V 1C3
Kylie Lepic, MD, 905 521-2100 ext. 62491
Kylie Lepic, MD
- Recruiting
London Health Sciences Centre
London, Ontario, N6A5W9
Anargyros Xenocostas, MD, 519-685-8500 ext. 58631, Anargyros.Xenocostas@lhsc.on.ca
Anargyros Xenocostas, MD
- Not yet recruiting
Hopital Maisonneuve-Rosemont
Montreal, Quebec
Silvy Lachance, MD
- Recruiting
The Ottawa Hospital
Ottawa, Ontario, K1H8L6
Alan Tinmouth, MD, 613-737-8899 ext. 73914, atinmouth@ohri.ca
Alan Tinmouth, MD MSc RCPSC
- Recruiting
Saskatchewan Cancer Agency
Saskatoon, Saskatchewan
Mohamed Elementary, MD, mohamed.elemary@saskcancer.ca
- Recruiting
Niagara Health System
St. Catharines, Ontario, L2S 0A9
Ramsha Faisal, 905-378-4647 ext. 44266, Ramsha.faisal@niagarahealth.on.ca
Mohammad Refaei, MD
- Recruiting
Eastern Regional Health Authority
St. John's, Newfoundland and Labrador, A1B 3V6
David Jones, MD, Davidm.jones@easternhealth.ca
- Not yet recruiting
Memorial University
St. John's, Newfoundland and Labrador
Kirsty Tompkins, MD
- Withdrawn
Princess Margaret Cancer Centre
Toronto, Ontario, M5G 2M9
About this study
Official title: Platelet Transfusions in Hematopoietic Stem Cell Transplantation - The PATH Phase III Trial
It is hypothesized that a strategy using prophylactic oral and intravenous Tranexamic Acid (TXA) with therapeutic platelet transfusions (if required) is safe and more effective than prophylactic platelet transfusions in patients undergoing an autologous hematopoietic stem cell transplantation (ASCT).
Read the detailed description
In Canada, over 1,500 autologous hematopoietic stem cell transplantations (ASCT) are performed annually for hematologic malignancies. It is currently standard practice to provide a prophylactic transfusion of platelets to prevent bleeding when the daily measured platelet count is less than 10 x 109/L. A patient may require up to six adult platelet doses during the post-transplant period. However, the true benefit of prophylactic platelet transfusions in the ASCT setting is unclear and has been called into question by several recent studies.
Prophylactic platelet transfusions may not only be unnecessary, they may be detrimental to the patient. Among blood products, platelet transfusions are associated with the highest risk of both infectious and non-infectious complications: this would include bacterial infections and allergic /febrile reactions. Moreover, the potential overuse of platelet products places a significant burden on a scarce health care resource that is provided through volunteer donations.
An alternative strategy to prevent bleeding and reduce the need for platelet transfusions involves administering Tranexamic Acid, an antifibrinolytic agent to stabilize blood clots and reduce bleeding. Tranexamic Acid is safe and effective in many clinical scenarios, and may be a reasonable alternative for prophylactic platelet transfusions. In the setting of ASCT, Tranexamic Acid may reduce bleeding and further enhance a strategy of therapeutic platelet transfusions where platelets are administered only in the event of active bleeding symptoms.
The effect of prophylactic platelet transfusions and Tranexamic Acid on clinical, quality of life and economic outcomes in patients receiving ASCT is unknown. The primary aim of this research program is to perform a randomized controlled trial to determine whether a strategy of prophylactic Tranexamic Acid (with therapeutic platelet transfusions) is safe and effective compared to prophylactic platelet transfusions in patients undergoing ASCT.
A pilot trial demonstrated feasibility by successfully recruiting 100 patients and these patients will be rolled over into the phase III study. The treatment assignment and bleeding outcomes for these patients remain blinded.
- Conditions
- Hematologic Neoplasms
- Being tested
- Tranexamic Acid
- Medical subject headings
- Hematologic Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Tranexamic AcidDrug
Also called SteriMax Inc., Cyclokapron®
Patients allocated to the prophylactic Tranexamic Acid group will receive a standardized routine oral or intravenous dose of Tranexamic Acid 1 gram three times daily.
Tranexamic Acid will start when Platelet count is less than 50 x 109/L and continue until platelet engraftment. Patients in this group will not receive routine prophylactic platelet transfusions. Subjects unable to swallow oral Tranexamic Acid pills may have the tablets crushed, administered via nasogastric (NG) tube or the medication will be administered intravenously.
| Study group | Role | What they receive |
|---|---|---|
| Prophylactic Platelet Transfusion | No intervention | Patients allocated to the prophylactic platelet transfusion group will receive a platelet transfusion when the measured platelet count is less than 10 x 109/L. |
| Prophylactic Tranexamic Acid | Experimental | Patients allocated to the prophylactic Tranexamic Acid group will receive a standardized routine oral or intravenous dose of Tranexamic Acid 1 gram three times daily. Receives: Drug: Tranexamic Acid |
What is being measured
The primary outcome is the main question the study is designed to answer.
WHO (World Health Organization) bleeding events of Grade 2 or higher
Measured: Daily, up to 30 days
Secondary outcomes (19)
WHO bleeding events of Grade 3 or 4
Measured: Daily, up to 30 days
Time from randomization to bleeding of WHO events Grade 2 or higher
Measured: Daily, up to 30 days
Number of days with bleeding of WHO bleeding events Grade 2 or higher
Measured: Daily, up to 30 days
Bleeding Severity Measurement Scale (BSMS) for bleeding events Grade 2 or higher
The BSMS scale measures bleeding grade and classification from 0-2. 0 indicates no bleeding. Grade 1 bleeding consists of trace bleeding and mild bleeding and is not clinically significant. Grade 2 bleeding consists of serious bleeding, serious bleeding causing significant morbidity, and fatal bleeding. Grade 2 bleeding is clinically significant.
Measured: Daily, up to 30 days
Number of platelet and/or red blood cell transfusions
Measured: Daily, up to 30 days
Adverse reactions related to tranexamic acid
Number and type of reactions will be recorded.
Measured: Daily, up to 30 days.
Venous thromboembolism grade 2 or higher
Measured: Daily, up to 30 days.
Adverse reactions related to platelet transfusion
Number and type of reactions will be recorded.
Measured: Daily, up to 30 days.
Time to platelet count recovery
Measured: Daily, up to 30 days.
Number of days with a platelet count < 10 x 109/L
Measured: Daily, up to 30 days.
LOS (Length of hospital stay)
LOS = admission date - discharge date
Measured: LOS will be measured as the number of days elapsed between hospital admission and hospital discharge date up to 30 days.
Transplant related outcome: Bearman Scoring System for Organ Toxicity following HSCT
This is a validated scoring system to assess toxicity during HSCT. In this system, grade I toxicity is reversible without treatment and grade 2 is not life threatening, but requires treatment. Grade 3 requires life-support intervention and grade 4 is fatal. Regimen-related toxicity in each organ system was scored as the highest grade achieved in that organ system through day 28, except that deaths occurring after day 28 as a result of regimen-related toxicity occurring before day 28 are also scored as grade 4. Adverse events that could be attributed to infection (culture-documented), bleeding or other medications are not scored as regimen-related toxicity. The maximum toxicity is the highest grade recorded in any individual organ system and the cumulative toxicity score is the sum of the highest grades recorded for all eight organ systems.
Measured: Day 30
Transplant related outcome: Incidence of infections at Day 30 following ASCT
Measured: Day 30
Transplant related outcome: Mortality at Day 30 and 180
Measured: Day 30, Day 180
Economic Analyses
Incremental cost effectiveness ratios
Measured: 5 years
Quality of Life Measure: FACT-Thrombocytopenia 18
The FACT consists of 5 subscales that measure physical well-being, functional well-being, social/family well-being and emotional well-being. The BMT subscale of the FACT includes additional items specifically designed to test quality of life and symptoms specific to transplant patients.
Measured: Weekly, up to 30 days
Quality of Life Measure: FACT- BMT
The FACT-BMT scale is valid and sensitive to clinical change in transplant recipients. It is the most consistently used scale amongst the Canadian Bone Marrow Transplant Group (CBMTG). It is the preferred scale in several Canadian multicentre trials in stem cell transplantation. FACT- Thrombocytopenia 18 is valid measure to elicit quality of life due to thrombocytopenia, and will complement the FACT-BMT scale.
Measured: Day 30, Day 90, Day 180
Quality of Life Measure: GAD-7
GAD-7 is a short validated scale that assesses symptoms of generalized anxiety and is commonly used in medical settings. There is no specific validated scale to assess anxiety of patients who are at risk for bleeding.
Measured: Weekly, up to 30 days
Quality of Life Measure: EQ-5D
EQ-5D is a standardized measure of health status to provide a simple, generic measure of health for clinical and economic appraisal. It is applicable to a wide range of health conditions and treatments; it provides a simple descriptive profile and a single index value for health status that can be used in the clinical and economic evaluation of health care. It is cognitively undemanding, taking only a few minutes to complete.
Measured: Weekly, up to 30 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Jun 2020
- Study start16 Feb 2022
- Primary completionFeb 2027 (estimated)
- Study completionFeb 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hematologic NeoplasmsStudies of the same condition, open and under way.
- More trials run by Ottawa Hospital Research InstituteThe 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 3
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 662 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Ottawa Hospital Research Institute
- Sponsor type
- University, hospital or other
- Collaborators
- Alberta Cancer Foundation
- Responsible party
- Sponsor
- Investigators
- Alan Tinmouth, MD, Principal investigator, The Ottawa Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04448184
- Sponsor's protocol ID
- 2068
- Acronym
- PATH
- Status last verified
- Jan 2026
- First submitted
- 24 Apr 2020
- Last updated
- 8 Jan 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Platelet Transfusions in Hematopoietic Stem Cell Transplantation - The PATH Phase III Trial
- ClinicalTrials.gov ID
- NCT04448184
- Acronym / sponsor ID
- PATH / 2068
- Conditions
- Hematologic Neoplasms
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 662 participants (estimated)
- Lead sponsor
- Ottawa Hospital Research InstituteUniversity, hospital or other
- Collaborators
- Alberta Cancer Foundation
- Study start
- 16 Feb 2022 (actual)
- Primary completion
- Feb 2027 (estimated)
- Study completion
- Feb 2027 (estimated)
- First posted
- 25 Jun 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
It is hypothesized that a strategy using prophylactic oral and intravenous Tranexamic Acid (TXA) with therapeutic platelet transfusions (if required) is safe and more effective than prophylactic platelet transfusions in patients undergoing an autologous hematopoietic stem cell transplantation (ASCT).
Detailed description
In Canada, over 1,500 autologous hematopoietic stem cell transplantations (ASCT) are performed annually for hematologic malignancies. It is currently standard practice to provide a prophylactic transfusion of platelets to prevent bleeding when the daily measured platelet count is less than 10 x 109/L. A patient may require up to six adult platelet doses during the post-transplant period. However, the true benefit of prophylactic platelet transfusions in the ASCT setting is unclear and has been called into question by several recent studies.
Prophylactic platelet transfusions may not only be unnecessary, they may be detrimental to the patient. Among blood products, platelet transfusions are associated with the highest risk of both infectious and non-infectious complications: this would include bacterial infections and allergic /febrile reactions. Moreover, the potential overuse of platelet products places a significant burden on a scarce health care resource that is provided through volunteer donations.
An alternative strategy to prevent bleeding and reduce the need for platelet transfusions involves administering Tranexamic Acid, an antifibrinolytic agent to stabilize blood clots and reduce bleeding. Tranexamic Acid is safe and effective in many clinical scenarios, and may be a reasonable alternative for prophylactic platelet transfusions. In the setting of ASCT, Tranexamic Acid may reduce bleeding and further enhance a strategy of therapeutic platelet transfusions where platelets are administered only in the event of active bleeding symptoms.
The effect of prophylactic platelet transfusions and Tranexamic Acid on clinical, quality of life and economic outcomes in patients receiving ASCT is unknown. The primary aim of this research program is to perform a randomized controlled trial to determine whether a strategy of prophylactic Tranexamic Acid (with therapeutic platelet transfusions) is safe and effective compared to prophylactic platelet transfusions in patients undergoing ASCT.
A pilot trial demonstrated feasibility by successfully recruiting 100 patients and these patients will be rolled over into the phase III study. The treatment assignment and bleeding outcomes for these patients remain blinded.
Study design
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Prophylactic Platelet Transfusion | No intervention | –Patients allocated to the prophylactic platelet transfusion group will receive a platelet transfusion when the measured platelet count is less than 10 x 109/L. |
| Prophylactic Tranexamic Acid | Experimental | Drug: Tranexamic AcidPatients allocated to the prophylactic Tranexamic Acid group will receive a standardized routine oral or intravenous dose of Tranexamic Acid 1 gram three times daily. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Adults 18 years or older undergoing ASCT for a hematologic malignancy
- Patients providing written informed consent prior to starting transplantation
Key exclusion criteria (14 listed)
- A previous WHO grade 2, 3 or 4 bleeding event within the past year
- A previous or current unprovoked thrombotic event defined as a pulmonary embolism, deep vein thrombosis, cerebral thrombosis
- A current provoked thrombotic event (e.g. catheter-related thrombosis) within last month and/or still requiring anticoagulant treatment.
- A requirement for therapeutic anticoagulant or anti-platelet drugs during ASCT
- Active angina (chest pain of presumed cardiac origin either at rest or with activity)
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 |
|---|---|---|
| Tranexamic AcidAlso: SteriMax Inc., Cyclokapron®Patients allocated to the prophylactic Tranexamic Acid group will receive a standardized routine oral or intravenous dose of Tranexamic Acid 1 gram three times daily. Tranexamic Acid will start when Platelet count is less than 50 x 109/L and continue until platelet engraftment. Patients in this group will not receive routine prophylactic platelet transfusions. Subjects unable to swallow oral Tranexamic Acid pills may have the tablets crushed, administered via nasogastric (NG) tube or the medication will be administered intravenously. | Drug | Prophylactic Tranexamic Acid |
Primary outcome
WHO (World Health Organization) bleeding events of Grade 2 or higher
Time frame: Daily, up to 30 days
19 secondary outcomes
WHO bleeding events of Grade 3 or 4
Time frame: Daily, up to 30 days
Time from randomization to bleeding of WHO events Grade 2 or higher
Time frame: Daily, up to 30 days
Number of days with bleeding of WHO bleeding events Grade 2 or higher
Time frame: Daily, up to 30 days
Bleeding Severity Measurement Scale (BSMS) for bleeding events Grade 2 or higher
Time frame: Daily, up to 30 days
Number of platelet and/or red blood cell transfusions
Time frame: Daily, up to 30 days
Adverse reactions related to tranexamic acid
Time frame: Daily, up to 30 days.
Venous thromboembolism grade 2 or higher
Time frame: Daily, up to 30 days.
Adverse reactions related to platelet transfusion
Time frame: Daily, up to 30 days.
Time to platelet count recovery
Time frame: Daily, up to 30 days.
Number of days with a platelet count < 10 x 109/L
Time frame: Daily, up to 30 days.
LOS (Length of hospital stay)
Time frame: LOS will be measured as the number of days elapsed between hospital admission and hospital discharge date up to 30 days.
Transplant related outcome: Bearman Scoring System for Organ Toxicity following HSCT
Time frame: Day 30
Transplant related outcome: Incidence of infections at Day 30 following ASCT
Time frame: Day 30
Transplant related outcome: Mortality at Day 30 and 180
Time frame: Day 30, Day 180
Economic Analyses
Time frame: 5 years
Quality of Life Measure: FACT-Thrombocytopenia 18
Time frame: Weekly, up to 30 days
Quality of Life Measure: FACT- BMT
Time frame: Day 30, Day 90, Day 180
Quality of Life Measure: GAD-7
Time frame: Weekly, up to 30 days
Quality of Life Measure: EQ-5D
Time frame: Weekly, up to 30 days
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Jan 2026.
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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.
Alan Tinmouth, MD principal investigator
The Ottawa Hospital
12 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Tranexamic Acid in people with Hematologic Neoplasms, run by Ottawa Hospital Research Institute at 12 sites in Canada.
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 2 inclusion and 14 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: looking for participants now, as of the sponsor's last update on 8 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in Canada. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Sohail Robert, RN (613-737-8899 ext. 71892, sorobert@ohri.ca), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
WHO (World Health Organization) bleeding events of Grade 2 or higher (time frame: Daily, up to 30 days). 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 8 Jan 2026.