Graft Vs Host DiseaseNCT06080490
Tacrolimus Blood Concentration and Transplant-related Outcomes in Pediatric HSCT Recipients
An observational study of Graft Vs Host Disease and Transplant Failure, run by University of Pisa at 1 site in Italy.
Trial summary
The goal of this retrospective observational study is to investigate any possible association among tacrolimus (TAC) blood concentrations, clinical efficacy and tolerability. Therefore, the main questions it aims to answer are: 1. Read more about this study
- Who is it for?
- Ages 1 month to 18 years
- Conditions the study is for
- Graft Vs Host Disease, Transplant Failure, Stem Cell Transplant Complications
- What is being tested?
- TAC C/D ratio
- How many people
- 125 participants
- Where
- 1 siteItaly
- Study dates
- 29 Sep 2023to 31 Dec 2024
- Phase
- Observational
- 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 month to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Patients who underwent allo-HSCT procedure between January 2011and December 2022 at IRCC Burlo Garofolo
Who may be able to join
- Age of the patients between 0 and 18 years (pediatric)
- Patients undergoing allogeneic bone marrow transplantation
- Patients' GVHD prophylaxis with tacrolimus
- Signed informed consent
Who cannot join
- Patients undergoing autologous bone marrow transplantation
- Patients not undergoing GVHD prophylaxis
- Patients undergoing cyclosporin prophylaxis
- Incomplete pharmacokinetic/pharmacodynamic data
- Lack of any informed consent
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Factors Affecting the Tacrolimus Blood Concentration and Its Impact on Transplant-related Outcomes in Pediatric HSCT Recipients: a Single-center Retrospective Study
The goal of this retrospective observational study is to investigate any possible association among tacrolimus (TAC) blood concentrations, clinical efficacy and tolerability.
Therefore, the main questions it aims to answer are:
- to clarify which variables, how and to what extent influence daily TAC blood concentrations in pediatric allo-hematopoietic stem cell transplantation (HSCT) recipients;
- to investigate the incidence of graft-versus-host disease (GVHD) and graft failure according to TAC exposure.
Pediatric patients administered TAC to prevent GVHD after an allogeneic bone marrow transplantation.
Read the detailed description
Patients undergoing allo-HSCT will be recruited at IRCC Burlo Garofolo, collecting demographic and clinical characteristics from electronic medical records in a retrospective manner: age, sex, primary disease, donor type and stem cell source, body weight (BW), white blood cell (WBC) count, platelet count, red blood cell (RBC) counts, hemoglobin (HGB) and hematocrit (HCT) levels, red cell concentrate (RCC) transfusions. Other clinical laboratory data (i.e., liver and kidney function) will be included, as well as concomitant drugs administered to patients after allo-HSCT (i.e, mycophenolate mofetil, steroids, pantoprazole, voriconazole, methotrexate). Data will be collected, validated, and presented according to Good Clinical Practice (GCP) principles to support the statistical analyses and subsequent reporting. Once selected according to predefined inclusion requirements, data will be collected anonymously; an identification code will be assigned to each case. As per GCP, all collected information will be recorded, handled, and stored in a way that allows its accurate reporting, interpretation, and verification with user access restriction, and data will be retrievable by designated personnel only.
Because of the retrospective nature of the present research protocol, all eligible patients will be included in the study population only if their medical and laboratory records will be complete.
Statistical analyses (for descriptive and comparative aims) will be performed stratifying patients by body weight, age, gender, and source of stem cells. Univariate and multivariate tests will be used, together with Kaplan-Meyer tes, Cox proportional hazard model and ROC curve analysis.
- Being tested
- TAC C/D ratio
- Keywords
- HSCT, Tacrolimus, GVHD, Pediatric patients
- Medical subject headings
- Graft vs Host Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TAC C/D ratioDrug
Also called Tacrolimus
Pharmacokinetics of TAC according to possible influencing factors
What is being measured
The primary outcome is the main question the study is designed to answer.
TAC pharmacokinetics
Evaluation of factors affecting TAC blood concentration/dose (C/D) ratio
Measured: 0-30 days
Secondary outcomes (2)
GVHD
Evaluation of GVHD in patients according to TAC C/D
Measured: 0-180 days
Graft failure
Evaluation of graft failure in patients according to TAC C/D
Measured: 0-180 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Oct 2023
- Study start29 Sep 2023
- Primary completion29 Sep 2024
- Study completion31 Dec 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Graft Vs Host DiseaseStudies 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
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 125 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Pisa
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Antonello Di Paolo, M.D., Ph.D., University of Pisa
- Investigators
- Natalia Maximova, MD, Principal investigator, IRCCS Burlo Garofolo - Trieste - ITALY
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06080490
- Sponsor's protocol ID
- GEN/INT 0001973
- Status last verified
- Jun 2025
- First submitted
- 6 Oct 2023
- Last updated
- 5 Jun 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided: IPD sharing plan is under evaluation by local IRB
Publications
Braidotti S, Curci D, Maestro A, Zanon D, Maximova N, Di Paolo A. Effect of early post-hematopoietic stem cell transplant tacrolimus concentration on transplant outcomes in pediatric recipients: One facility's ten-year experience of immunosuppression with tacrolimus. Int Immunopharmacol. 2024 Sep 10;138:112636. doi: 10.1016/j.intimp.2024.112636. Epub 2024 Jul 10.
PubMed 38991629
Study at a glance
From the registry record, last updated by the sponsor on 5 Jun 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Factors Affecting the Tacrolimus Blood Concentration and Its Impact on Transplant-related Outcomes in Pediatric HSCT Recipients: a Single-center Retrospective Study
- ClinicalTrials.gov ID
- NCT06080490
- Acronym / sponsor ID
- GEN/INT 0001973
- Keywords
- HSCT, Tacrolimus, GVHD, Pediatric patients
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 125 participants (actual)
- Lead sponsor
- University of PisaUniversity, hospital or other
- Study start
- 29 Sep 2023 (actual)
- Primary completion
- 29 Sep 2024 (actual)
- Study completion
- 31 Dec 2024 (actual)
- First posted
- 12 Oct 2023 (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 goal of this retrospective observational study is to investigate any possible association among tacrolimus (TAC) blood concentrations, clinical efficacy and tolerability.
Therefore, the main questions it aims to answer are:
- to clarify which variables, how and to what extent influence daily TAC blood concentrations in pediatric allo-hematopoietic stem cell transplantation (HSCT) recipients;
- to investigate the incidence of graft-versus-host disease (GVHD) and graft failure according to TAC exposure.
Pediatric patients administered TAC to prevent GVHD after an allogeneic bone marrow transplantation.
Detailed description
Patients undergoing allo-HSCT will be recruited at IRCC Burlo Garofolo, collecting demographic and clinical characteristics from electronic medical records in a retrospective manner: age, sex, primary disease, donor type and stem cell source, body weight (BW), white blood cell (WBC) count, platelet count, red blood cell (RBC) counts, hemoglobin (HGB) and hematocrit (HCT) levels, red cell concentrate (RCC) transfusions. Other clinical laboratory data (i.e., liver and kidney function) will be included, as well as concomitant drugs administered to patients after allo-HSCT (i.e, mycophenolate mofetil, steroids, pantoprazole, voriconazole, methotrexate). Data will be collected, validated, and presented according to Good Clinical Practice (GCP) principles to support the statistical analyses and subsequent reporting. Once selected according to predefined inclusion requirements, data will be collected anonymously; an identification code will be assigned to each case. As per GCP, all collected information will be recorded, handled, and stored in a way that allows its accurate reporting, interpretation, and verification with user access restriction, and data will be retrievable by designated personnel only.
Because of the retrospective nature of the present research protocol, all eligible patients will be included in the study population only if their medical and laboratory records will be complete.
Statistical analyses (for descriptive and comparative aims) will be performed stratifying patients by body weight, age, gender, and source of stem cells. Univariate and multivariate tests will be used, together with Kaplan-Meyer tes, Cox proportional hazard model and ROC curve analysis.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
Who may qualify
- Ages
- 1 month to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Patients who underwent allo-HSCT procedure between January 2011and December 2022 at IRCC Burlo Garofolo
Key inclusion criteria (4 listed)
- Age of the patients between 0 and 18 years (pediatric)
- Patients undergoing allogeneic bone marrow transplantation
- Patients' GVHD prophylaxis with tacrolimus
- Signed informed consent
Key exclusion criteria (5 listed)
- Patients undergoing autologous bone marrow transplantation
- Patients not undergoing GVHD prophylaxis
- Patients undergoing cyclosporin prophylaxis
- Incomplete pharmacokinetic/pharmacodynamic data
- Lack of any informed consent
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 |
|---|---|---|
| TAC C/D ratioAlso: TacrolimusPharmacokinetics of TAC according to possible influencing factors | Drug | – |
Primary outcome
TAC pharmacokinetics
Time frame: 0-30 days
Evaluation of factors affecting TAC blood concentration/dose (C/D) ratio
2 secondary outcomes
GVHD
Time frame: 0-180 days
Graft failure
Time frame: 0-180 days
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Jun 2025.
- Results publication: PubMed 38991629Braidotti S, Curci D, Maestro A, Zanon D, Maximova N, Di Paolo A. Effect of early post-hematopoietic stem cell transplant tacrolimus concentration on transplant outcomes in pediatric recipients: One facility's ten-year experience of immunosuppression with tacrolimus. Int Immunopharmacol. 2024 Sep 10;138:112636. doi: 10.1016/j.intimp.2024.112636. Epub 2024 Jul 10.
- Individual participant dataSharing planned: Undecided. IPD sharing plan is under evaluation by local IRB
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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.
Natalia Maximova, MD principal investigator
IRCCS Burlo Garofolo - Trieste - ITALY
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Graft Vs Host Disease and Transplant Failure, run by University of Pisa at 1 site in Italy.
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 4 inclusion and 5 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: finished, as of the sponsor's last update on 5 Jun 2025. 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 Italy. 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.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
TAC pharmacokinetics (time frame: 0-30 days). 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 NCT06080490, which also lists 1 linked publication.
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 5 Jun 2025.