Pro-ADM vs PCT in Patients With Complications Post Hematopoietic Stem Cell Transplantation

Prospective Non-Interventional Trial to Evaluate Kinetics, Diagnostic and Prognostic Value of Pro-ADM as Compared to PCT in Patients Presenting With Infections or Other Complications Post Hematopoietic Stem Cell Transplantation (HSCT)

Prospective, multicenter, spontaneous, non-interventional, non pharmacological. Study aimed at evaluate kinetics, diagnostic and prognostic value of pro-ADM (proadrenomedullin) as compared to PCT (procalcitonin) in patients presenting with infections or other complications post hematopoietic stem cell transplantation (HSCT)

Study Overview

• Status: Recruiting
• Conditions: Stem Cell Transplant Complications
• Intervention / Treatment: Other: observation

Detailed Description

Evaluation of diagnostic performance of pro-ADM as compared to palliative care therapy (PCT) in paediatric and young adult patients with any type of complications after hematopoietic stem cell transplantation or with transplant related complications.

• Study Type: Observational
• Enrollment (Estimated): 200

Contacts and Locations

• Study Contact: Name: Celeste Cagnazzo, PhD; Phone Number: 00390113135171; Email: celeste.cagnazzo@unito.it

Study Locations

• Italy
  • Turin, Italy, 10126
    • Recruiting
    • AOU Città della Salute e della Scienza di Torino - Presidio Infantile Regina Margherita
    • Contact: Franca Fagioli, MD; Phone Number: 5997 0039011313; Email: franca.fagioli@unito.it

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 4 weeks to 39 years (Child, Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Probability Sample

Study Population

Pediatric and young adult patients affected by malignant or non-malignant disorders referred for HCST

Description

Inclusion Criteria:

• Written informed consent of the parents/legal guardians and patient's assent/consent according to national regulations. For subjects who turn 18 years of age during participation in this study, participation in the future use portion of the study after their 18th birthday requires that they sign the informed consent statement following turning 18 years of age.
• Age at time of registration from 28 days to less than 39 years of age.
• Malignant disease (i.e. Acute lymphocytic leukemia (ALL), Acute myeloid leukemia (AML), Myelodysplastic syndromes (MDS)) or nonmalignant disease (e.g. immunodeficiency or inherited disorders of metabolism or hemoglobinopathies etc.) indicated for allogeneic HSCT.

Exclusion Criteria:

• Age less than 28 days
• Obese patients with body mass index: > 30 kg/m^2.
• Impaired cardiac function: severe cardiac insufficiency indicated by left ventricle ejection fraction (LVEF) ≤ 35%.
• Impaired liver function indicated by Bilirubin >3 times the upper limit of normal (ULN), or aspartate aminotransferase/alanine aminotransferase (AST/ALT) >10 times ULN
• Impaired renal function indicated by estimated glomerular filtration rate (GFR), according to the Schwartz formula for ages 1-17 and according to CDKEPI creatinine equation for ages above 17 years old) < 60 mL/min/1,73m^2.
• Concurrent severe active infection at the start of conditioning regimen

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Paediatric patients receiving HCTS with infectious complications post HCTS; Paediatric patients receiving allogeneic hematopoietic stem cell transplantation (HCTS) and who present with infectious complications post HSCT or with transplant related complications (acute graft-versus-host disease- GvHD-, sinusoidal obstruction syndrome -SOS-, engraftment-ES- and pre-engraftment syndrome- pre-ES-, graft failure, thrombotic microangiopathy associated with HSCT- TA-TMA or those without complications post HSCT).	Other: observation; observation and prospective data collection
Paediatric patients receiving HCTS without infectious complications post HCTS	Other: observation; observation and prospective data collection

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Evaluation of diagnostic performance of pro-ADM as compared to PCT in patients receiving HSCT	In order to achieve the objective, it will be considered paediatric and young adults patients affected by malignant/non-malignant disorders who have been subjected to HSCT and present with infectious complications post HSCT or with transplant related complications (acute graft-versus-host disease (GvHD), sinusoidal obstruction syndrome (SOS), engraftment (ES) and pre-engraftment syndrome (pre-E), graft failure, thrombotic microangiopathy associated with HSCT (TA-TMA) or those without complications post HSCT).	180 days after HSCT

Collaborators and Investigators

• Sponsor: Azienda Ospedaliera Ospedale Infantile Regina Margherita Sant'Anna
• Investigators: Principal Investigator: Franca Fagioli, MD, AOU Città della Salute e della Scienza di Torino - OIRM; Study Chair: Manuela Spadea, MD, AOU Città della Salute e della Scienza di Torino - OIRM

Study record dates

Study Major Dates

• Study Start (Actual): October 20, 2022
• Primary Completion (Estimated): September 1, 2024
• Study Completion (Estimated): March 1, 2025

Study Registration Dates

• First Submitted: August 29, 2022
• First Submitted That Met QC Criteria: August 29, 2022
• First Posted (Actual): August 31, 2022

Study Record Updates

• Last Update Posted (Actual): May 25, 2023
• Last Update Submitted That Met QC Criteria: May 23, 2023
• Last Verified: May 1, 2023

More Information

Terms related to this study

• Other Study ID Numbers: Trap-ADM

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No