Tacrolimus Blood Concentration and Transplant-related Outcomes in Pediatric HSCT Recipients

NCT06080490 | Completed FDA Drug

• 1 other identifier: GEN/INT 0001973
• Study Type: observational
• Target: 125
• Locations: 1 country
• Sites: 1

Brief 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. 1.to clarify which variables, how and to what extent influence daily TAC blood concentrations in pediatric allo-hematopoietic stem cell transplantation (HSCT) recipients;
2. 2.to investigate the incidence of graft-versus-host disease (GVHD) and graft failure according to TAC exposure.

Conditions

Graft Vs Host Disease; Transplant Failure; Stem Cell Transplant Complications

Keywords

HSCT; Tacrolimus; GVHD; Pediatric patients

Outcome Measures

Primary Outcomes (1)

• TAC pharmacokinetics

  Evaluation of factors affecting TAC blood concentration/dose (C/D) ratio

  0-30 days

Secondary Outcomes (2)

• GVHD

  0-180 days

• Graft failure

  0-180 days

Interventions

TAC C/D ratio DRUG

Pharmacokinetics of TAC according to possible influencing factors

Also known as: Tacrolimus

Eligibility Criteria

• Age: 1 Month - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

Patients who underwent allo-HSCT procedure between January 2011and December 2022 at IRCC Burlo Garofolo

You may qualify if:

• Age of the patients between 0 and 18 years (pediatric)
• Patients undergoing allogeneic bone marrow transplantation
• Patients' GVHD prophylaxis with tacrolimus
• Signed informed consent

You may not qualify if:

• Patients undergoing autologous bone marrow transplantation
• Patients not undergoing GVHD prophylaxis
• Patients undergoing cyclosporin prophylaxis
• Incomplete pharmacokinetic/pharmacodynamic data
• Lack of any informed consent

Sponsors & Collaborators

• University of Pisa: lead

Study Sites (1)

IRCCS Burlo Garofolo

Trieste, 34137, Italy

Location

Related Publications (1)

• 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.

  PMID: 38991629 RESULT

MeSH Terms

Conditions

Graft vs Host Disease

Interventions

Tacrolimus

Condition Hierarchy (Ancestors)

Immune System Diseases

Intervention Hierarchy (Ancestors)

Macrolides; Lactones; Organic Chemicals

Study Officials

• Natalia Maximova, MD

  IRCCS Burlo Garofolo - Trieste - ITALY

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor of Pharmacology

Study Record Dates

• First Submitted: October 6, 2023
• First Posted: October 12, 2023
• Study Start: September 29, 2023
• Primary Completion: September 29, 2024
• Study Completion: December 31, 2024
• Last Updated: June 5, 2025

Record last verified: 2025-06

Locations

IT (1)