AnemiaPhase 3P-OpTTICCANCT03871244
Pilot Optimizing Transfusion Thresholds in Critically-ill Children With Anaemia
A Phase 3 study testing Less red blood cell transfusions and More red blood cell transfusion (standard transfusion strategy in PICU) in people with Anemia, run by St. Justine's Hospital at 1 site in Canada.
Trial summary
The Pilot Optimizing Transfusion Thresholds in Critically-ill Children with Anaemia (P-OpTTICCA) study is a pilot trial for a large pragmatic international parallel open-label non-inferiority randomised controlled trial. The primary outcome of the pilot study is feasibility. Read more about this study
- Who is it for?
- Ages 1 day to 18 years
- Conditions the study is for
- Anemia
- What is being tested?
- Less red blood cell transfusions and More red blood cell transfusion (standard transfusion strategy in PICU)
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 120 participants
- Where
- 1 siteCanada
- Study dates
- 3 Jun 2020to 31 Dec 2022
- Phase
- Phase 3
- Are there results?
- None posted in the registry
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 1 day to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 1. Hemoglobin concentration ≤ 95 g/L, while in PICU
- 2. Not already participating to a randomized controlled trial on hemoglobin threshold to guide red blood cell transfusion practice
Who cannot join
- Post conception age ≤ 36 weeks or > 18 years at PICU entry
- Refusal of consent by patient and/or parent
- Uncorrected cyanotic cardiac disease, univentricular physiology
- Sickle cell disease
- Brain Death
- Extracorporeal membrane oxygenation (ECMO)
Trial locations and contacts
1 site in 1 country.
Canada1 site, 1 recruiting
- Recruiting
CHU Sainte-Justine
Montreal, Quebec, H3T 1C5
Josée Poirier, 514-345-4931 ext. 4053, josee.poirier.hsj@ssss.gouv.qc.ca
About this study
Official title: Pilot Optimizing Transfusion Thresholds in Critically-ill Children With Anaemia (P-OpTTICCA): a Pilot Trial for a Large Pragmatic International Parallel Open-label Non-inferiority Randomised Controlled Trial.
The Pilot Optimizing Transfusion Thresholds in Critically-ill Children with Anaemia (P-OpTTICCA) study is a pilot trial for a large pragmatic international parallel open-label non-inferiority randomised controlled trial. The primary outcome of the pilot study is feasibility.
Read the detailed description
Patients in paediatric intensive care units (PICU) are among the main groups of transfusion recipients. Blood transfusions expose recipients to infectious and non-infectious serious hazards. Despite an increasing number of red blood cell (RBC) transfusion threshold randomised controlled trials (RCT) to date, only one large RCT, the Transfusion Requirement In PICU (TRIPICU) study (Lacroix. N Engl J Med 2007;356:1609-19) informs practice in PICU. This dearth of paediatric data was reiterated by a recent National Heart, Lung and Blood Institutes state of the art symposium. Despite being internally robust, the generalizability of TRIPICU has been questioned given that nearly 90% of critically ill children with haemoglobin (Hb) level ≤ 95 g/L were not enrolled for a range of pre-specified reasons including severity of illness, clinical instability and uncertain physician buy-in, thus threatening broader application of this critical knowledge into practice. Recent data and evidence summarised in systematic reviews support the need to explore the benefits and safety of Hb threshold ≤ 70 g/L for almost all critically ill children, not only those who would have been enrolled in TRIPICU.
We propose an international pilot study that will inform the design of a large pragmatic non-inferiority RCT (termed OpTTICCA) conceived to derive generalizable transfusion guidance for physicians.
Objectives of the pilot-RCT. Specific aims are to:
- Establish the feasibility of enrolling ≥80% of eligible patients.
- Document adherence to study intervention ≥80% (restrictive transfusion policy).
- Assess the incidence rate of the primary outcome measure of the full RCT (new and progressive multiple organ dysfunction syndrome, which includes mortality).
- Establish the feasibility of using routinely collected clinical information from electronic medical data monitoring system (eMDMS) to enhance cost-efficiency.
In the pilot-RCT, we will enrol 120 patients (20 to 40/site) in PICUs equipped with electronic medical data monitoring system (eMDMS). This trial builds on successful prior RCTs that involved international collaboration and funding (Canada, United Kingdom, France): TRIPICU, Age of Blood Evaluation (ABLE) (Lacroix. N Engl J Med 2015) and Age of Blood in Children in PICU (ABC-PICU study: Tucci et al. Trials 2018). The pilot-RCT will be considered as the vanguard phase of the full OpTTICCA RCT if no important design changes are required.
Expected results. This pilot-RCT will focus on feasibility parameters. It will explore whether we can 1) recruit a much broader eligible patient group, 2) assess protocol adherence, 3) assess the primary outcome measure, and 4) abstract most data electronically. A definitive trial will provide transfusion guidance for many important subgroups in whom evidence is presently lacking including children admitted with bone marrow failure, head injury, some cardiac disorders, and sepsis.
- Conditions
- Anemia
- Being tested
- Less red blood cell transfusions, More red blood cell transfusion (standard transfusion strategy in PICU)
- Keywords
- hemoglobin threshold, critically ill children, randomized controlled trial, red blood cell tranfusions
- Medical subject headings
- Anemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Less red blood cell transfusionsProcedure or surgery
Also called Restrictive red blood cell transfusion strategy
The instructions given to caregivers will be to consider prescribing a red blood cell transfusion only if the hemoglobin level is ≤ 70 g per L.
More red blood cell transfusion (standard transfusion strategy in PICU)Procedure or surgery
The instructions given to caregivers will be to prescribe a red blood cell transfusion according to their usual standard practice
| Study group | Role | What they receive |
|---|---|---|
| Restrictive arm (intervention) | Experimental | Less red blood cell transfusions. The protocol will require that no red blood cell transfusion be given unless the hemoglobin level is below or equal at 70 g per L. Receives: Procedure: Less red blood cell transfusions |
| Standard care arm (comparator) | Active comparator | Clinical teams will follow their usual transfusion practices. Receives: Procedure: More red blood cell transfusion (standard transfusion strategy in PICU) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Recruitment: screening
Proportion of patients eligible for consent approached > 90%;
Measured: One year
Recruitment: consent
Proportion of approached patients who decline consent < 20%,
Measured: One year
Recruitment: enrolment
Recruitment: ≥ 2 patients randomized/site/week.
Measured: One year
Secondary outcomes (8)
Representativeness: exclusion
We recruit > 80% of patients who would have been excluded from TRIPICU (Lacroix et al. N Engl Med 2007).
Measured: One year
Representativeness: opt-out
Proportion of dropout and lost to follow-up is expected to be < 2%.
Measured: One year
Compliance
Proportion of RBC transfusions given with hemoglobin > 70 g/L in the restrictive arm (definition of non-compliance) is < 20%.
Measured: One year
Separation
Post randomisation separation of hemoglobin concentration between both arms in the pilot-RCT is ≥ 10 g/L.
Measured: One year
Suspension
Proportion of children wha are suspended is < 20%.
Measured: One year
Data collection
Most data (> 80%) are abstracted directly from electronic medical data monitoring system.
Measured: One year
Data entry
Proportion of erroneous data entry is < 5%.
Measured: One year.
Outcomes
Incidence rate of the primary outcome measure - new and progressive multiple organ dysfunction syndrome (NPMODS) - in the standard care arm of the pilot-trial is ≥ 20%.
Measured: One year.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Mar 2019
- Study start3 Jun 2020
- Primary completion31 Dec 2021 (estimated)
- Study completion31 Dec 2022 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for AnemiaStudies of the same condition, open and under way.
- 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
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- parallel open-label pragmatic non-inferiority randomised controlled trial (RCT)
- Masking
- Double blind
- Who is masked
- Investigator, Outcomes assessor
- Enrolment
- 120 participants (estimated)
Sponsor and investigators
- Lead sponsor
- St. Justine's Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Oxford University Hospitals NHS Trust, Hôpital Jeanne de Flandre, NHS Blood and Transplant
- Responsible party
- Principal investigator, Jacques Lacroix, MD, FRCPC, Professor, St. Justine's Hospital
- Investigators
- Jacques Lacroix, Principal investigator, St-Justine Hospital Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03871244
- Sponsor's protocol ID
- MP-21-2019-2235
- Acronym
- P-OpTTICCA
- Status last verified
- Sep 2021
- First submitted
- 6 Mar 2019
- Last updated
- 28 Sep 2021
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: The data of P-OpTTICCA will be integrated in the large trial if possible. This will not be possible if P-OpTTICCA shows that significant changes must be made to the research proposal of the full trial; then the data of participants of P-OpTTICCA will be merged to the data of the full trial using a meta-analysis strategy.
Publications
Carson JL, Stanworth SJ, Dennis JA, Fergusson DA, Pagano MB, Roubinian NH, Turgeon AF, Valentine S, Trivella M, Doree C, Hebert PC. Transfusion thresholds and other strategies for guiding red blood cell transfusion. Cochrane Database Syst Rev. 2025 Oct 20;10(10):CD002042. doi: 10.1002/14651858.CD002042.pub6.
PubMed 41114449Tucci M, Sarfatti A, Leteurtre S, Du Pont-Thibodeau G, Fontela P, Ray S, Stanworth SJ, Hebert PC, Roumeliotis N, Sauthier M, Spinella PC, Ducruet T, Jouvet P, Masse B, Robitaille N, Trottier H, Lacroix J; Canadian Critical Care Trials Group and the Groupe Francophone de Reanimation et d'Urgences Pediatriques. Pilot-Optimizing Transfusion Therapy in Critically Ill Children With Anemia: A Pilot Multicenter Electronic Health Record-Enabled Randomized Controlled Trial on Red Blood Cell Transfusion in Critically Ill Children. Pediatr Crit Care Med. 2025 Oct 1;26(10):e1218-e1230. doi: 10.1097/PCC.0000000000003822. Epub 2025 Sep 12.
PubMed 40938139Radford M, Estcourt LJ, Sirotich E, Pitre T, Britto J, Watson M, Brunskill SJ, Fergusson DA, Doree C, Arnold DM. Restrictive versus liberal red blood cell transfusion strategies for people with haematological malignancies treated with intensive chemotherapy or radiotherapy, or both, with or without haematopoietic stem cell support. Cochrane Database Syst Rev. 2024 May 23;5(5):CD011305. doi: 10.1002/14651858.CD011305.pub3.
PubMed 38780066Carson JL, Stanworth SJ, Dennis JA, Trivella M, Roubinian N, Fergusson DA, Triulzi D, Doree C, Hebert PC. Transfusion thresholds for guiding red blood cell transfusion. Cochrane Database Syst Rev. 2021 Dec 21;12(12):CD002042. doi: 10.1002/14651858.CD002042.pub5.
PubMed 34932836
Study at a glance
From the registry record, last updated by the sponsor on 28 Sep 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pilot Optimizing Transfusion Thresholds in Critically-ill Children With Anaemia (P-OpTTICCA): a Pilot Trial for a Large Pragmatic International Parallel Open-label Non-inferiority Randomised Controlled Trial.
- ClinicalTrials.gov ID
- NCT03871244
- Acronym / sponsor ID
- P-OpTTICCA / MP-21-2019-2235
- Conditions
- Anemia
- Keywords
- hemoglobin threshold, critically ill children, randomized controlled trial, red blood cell tranfusions
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 120 participants (estimated)
- Lead sponsor
- St. Justine's HospitalUniversity, hospital or other
- Collaborators
- Oxford University Hospitals NHS Trust, Hôpital Jeanne de Flandre, NHS Blood and Transplant
- Study start
- 3 Jun 2020 (actual)
- Primary completion
- 31 Dec 2021 (estimated)
- Study completion
- 31 Dec 2022 (estimated)
- First posted
- 12 Mar 2019 (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.
The Pilot Optimizing Transfusion Thresholds in Critically-ill Children with Anaemia (P-OpTTICCA) study is a pilot trial for a large pragmatic international parallel open-label non-inferiority randomised controlled trial. The primary outcome of the pilot study is feasibility.
Detailed description
Patients in paediatric intensive care units (PICU) are among the main groups of transfusion recipients. Blood transfusions expose recipients to infectious and non-infectious serious hazards. Despite an increasing number of red blood cell (RBC) transfusion threshold randomised controlled trials (RCT) to date, only one large RCT, the Transfusion Requirement In PICU (TRIPICU) study (Lacroix. N Engl J Med 2007;356:1609-19) informs practice in PICU. This dearth of paediatric data was reiterated by a recent National Heart, Lung and Blood Institutes state of the art symposium. Despite being internally robust, the generalizability of TRIPICU has been questioned given that nearly 90% of critically ill children with haemoglobin (Hb) level ≤ 95 g/L were not enrolled for a range of pre-specified reasons including severity of illness, clinical instability and uncertain physician buy-in, thus threatening broader application of this critical knowledge into practice. Recent data and evidence summarised in systematic reviews support the need to explore the benefits and safety of Hb threshold ≤ 70 g/L for almost all critically ill children, not only those who would have been enrolled in TRIPICU.
We propose an international pilot study that will inform the design of a large pragmatic non-inferiority RCT (termed OpTTICCA) conceived to derive generalizable transfusion guidance for physicians.
Objectives of the pilot-RCT. Specific aims are to:
- Establish the feasibility of enrolling ≥80% of eligible patients.
- Document adherence to study intervention ≥80% (restrictive transfusion policy).
- Assess the incidence rate of the primary outcome measure of the full RCT (new and progressive multiple organ dysfunction syndrome, which includes mortality).
- Establish the feasibility of using routinely collected clinical information from electronic medical data monitoring system (eMDMS) to enhance cost-efficiency.
In the pilot-RCT, we will enrol 120 patients (20 to 40/site) in PICUs equipped with electronic medical data monitoring system (eMDMS). This trial builds on successful prior RCTs that involved international collaboration and funding (Canada, United Kingdom, France): TRIPICU, Age of Blood Evaluation (ABLE) (Lacroix. N Engl J Med 2015) and Age of Blood in Children in PICU (ABC-PICU study: Tucci et al. Trials 2018). The pilot-RCT will be considered as the vanguard phase of the full OpTTICCA RCT if no important design changes are required.
Expected results. This pilot-RCT will focus on feasibility parameters. It will explore whether we can 1) recruit a much broader eligible patient group, 2) assess protocol adherence, 3) assess the primary outcome measure, and 4) abstract most data electronically. A definitive trial will provide transfusion guidance for many important subgroups in whom evidence is presently lacking including children admitted with bone marrow failure, head injury, some cardiac disorders, and sepsis.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- parallel open-label pragmatic non-inferiority randomised controlled trial (RCT)
| Arm | Type | Receives |
|---|---|---|
| Restrictive arm (intervention) | Experimental | Procedure: Less red blood cell transfusionsLess red blood cell transfusions. The protocol will require that no red blood cell transfusion be given unless the hemoglobin level is below or equal at 70 g per L. |
| Standard care arm (comparator) | Active comparator | Procedure: More red blood cell transfusion (standard transfusion strategy in PICU)Clinical teams will follow their usual transfusion practices. |
Who may qualify
- Ages
- 1 day to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (2 listed)
- 1. Hemoglobin concentration ≤ 95 g/L, while in PICU
- 2. Not already participating to a randomized controlled trial on hemoglobin threshold to guide red blood cell transfusion practice
Key exclusion criteria (6 listed)
- Post conception age ≤ 36 weeks or > 18 years at PICU entry
- Refusal of consent by patient and/or parent
- Uncorrected cyanotic cardiac disease, univentricular physiology
- Sickle cell disease
- Brain Death
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 |
|---|---|---|
| Less red blood cell transfusionsAlso: Restrictive red blood cell transfusion strategyThe instructions given to caregivers will be to consider prescribing a red blood cell transfusion only if the hemoglobin level is ≤ 70 g per L. | Procedure or surgery | Restrictive arm (intervention) |
| More red blood cell transfusion (standard transfusion strategy in PICU)The instructions given to caregivers will be to prescribe a red blood cell transfusion according to their usual standard practice | Procedure or surgery | Standard care arm (comparator) |
Primary outcome
Recruitment: screening
Time frame: One year
Proportion of patients eligible for consent approached > 90%;
Recruitment: consent
Time frame: One year
Proportion of approached patients who decline consent < 20%,
Recruitment: enrolment
Time frame: One year
Recruitment: ≥ 2 patients randomized/site/week.
8 secondary outcomes
Representativeness: exclusion
Time frame: One year
Representativeness: opt-out
Time frame: One year
Compliance
Time frame: One year
Separation
Time frame: One year
Suspension
Time frame: One year
Data collection
Time frame: One year
Data entry
Time frame: One year.
Outcomes
Time frame: One year.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Sep 2021.
- Derived from this study: PubMed 41114449Carson JL, Stanworth SJ, Dennis JA, Fergusson DA, Pagano MB, Roubinian NH, Turgeon AF, Valentine S, Trivella M, Doree C, Hebert PC. Transfusion thresholds and other strategies for guiding red blood cell transfusion. Cochrane Database Syst Rev. 2025 Oct 20;10(10):CD002042. doi: 10.1002/14651858.CD002042.pub6.
- Derived from this study: PubMed 40938139Tucci M, Sarfatti A, Leteurtre S, Du Pont-Thibodeau G, Fontela P, Ray S, Stanworth SJ, Hebert PC, Roumeliotis N, Sauthier M, Spinella PC, Ducruet T, Jouvet P, Masse B, Robitaille N, Trottier H, Lacroix J; Canadian Critical Care Trials Group and the Groupe Francophone de Reanimation et d'Urgences Pediatriques. Pilot-Optimizing Transfusion Therapy in Critically Ill Children With Anemia: A Pilot Multicenter Electronic Health Record-Enabled Randomized Controlled Trial on Red Blood Cell Transfusion in Critically Ill Children. Pediatr Crit Care Med. 2025 Oct 1;26(10):e1218-e1230. doi: 10.1097/PCC.0000000000003822. Epub 2025 Sep 12.
- Derived from this study: PubMed 38780066Radford M, Estcourt LJ, Sirotich E, Pitre T, Britto J, Watson M, Brunskill SJ, Fergusson DA, Doree C, Arnold DM. Restrictive versus liberal red blood cell transfusion strategies for people with haematological malignancies treated with intensive chemotherapy or radiotherapy, or both, with or without haematopoietic stem cell support. Cochrane Database Syst Rev. 2024 May 23;5(5):CD011305. doi: 10.1002/14651858.CD011305.pub3.
- Derived from this study: PubMed 34932836Carson JL, Stanworth SJ, Dennis JA, Trivella M, Roubinian N, Fergusson DA, Triulzi D, Doree C, Hebert PC. Transfusion thresholds for guiding red blood cell transfusion. Cochrane Database Syst Rev. 2021 Dec 21;12(12):CD002042. doi: 10.1002/14651858.CD002042.pub5.
- Individual participant dataSharing planned: No. The data of P-OpTTICCA will be integrated in the large trial if possible. This will not be possible if P-OpTTICCA shows that significant changes must be made to the research proposal of the full trial; then the data of participants of P-OpTTICCA will be merged to the data of the full trial using a meta-analysis strategy.
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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. This study is not recruiting at the moment.
Jacques Lacroix principal investigator
St-Justine Hospital Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Less red blood cell transfusions and More red blood cell transfusion (standard transfusion strategy in PICU) in people with Anemia, run by St. Justine's Hospital at 1 site 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 6 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: status not confirmed, as of the sponsor's last update on 28 Sep 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Jacques Lacroix (514-345-4931 ext. 5556, jacques.lacroix.med@ssss.gouv.qc.ca), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Recruitment: screening (time frame: One year). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03871244, which also lists 4 linked publications.
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 28 Sep 2021.