Cord Blood Transplantation With Myeloablative Conditioning and Post-transplant Cyclophosphamide (COmPACt Study)

Recruiting

• Conditions: Hematological Malignancies; Cord Blood Transplantation

• Registration Number: NCT03802773
• Lead Sponsor: Fondazione Policlinico Universitario Agostino Gemelli IRCCS

Brief Summary

Despite anti-thymocyte globulin has a mainstay role in preventing GvHD (and non-relapse mortality) in CB transplantation, it also induces delayed immune recovery, increased risk of cytomegalovirus and Epstein-Barr virus reactivation, post-transplant lymphoproliferative diseases, overall accounting for increased transplant-related mortality and/or increased relapse incidence. All these findings support the use of alternative approaches for in vivo T cell depletion in the setting of CB transplantation.

Detailed Description

Study objectives. The primary objective is to evaluate neutrophil and platelet engraftment and day +100 CD4+ cell count in patients receiving matched CB unit transplant with a myeloablative conditioning regimen and a GVHD prophylaxis including post-transplant cyclophosphamide. Secondary objective is to estimate the incidence and severity of acute and chronic GVHD. Study endpoints. Primary endpoints are hematopoietic engraftment and day +100 CD4+ cell count. Secondary endpoints are acute and chronic GVHD

Study Timeline

• Study First Submitted: 2019-01-10
• Study First Submitted QC: 2019-01-11
• Study First Posted: 2019-01-14
• Study Start: 2019-03-14
• Status Verified: 2024-12
• Last Update Submitted: 2025-04-07
• Last Update Posted: 2025-04-09
• Primary Completion: 2025-11-26
• Study Completion: 2025-12-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 10

Inclusion Criteria

• Age: ≥ 18 ≤ 75 years old
• CB unit transplantation (TNC> 2,0 x10^7/kg and > 4/6 loci HLA matched)
• Myeloablative conditioning regimen consisting of either Thiotepa/ Busulfan/ Fludarabine or TBI/ Fludarabine
• GVHD prophylaxis including PT-Cy (30 mg/kg or more on days + 3and +5) CSA and MMF
• Diagnosis of 1 of the following hematological malignancies: Acute Myeloid Leukemia (AML), Myelodysplastic Syndrome, high risk Acute Lymphoblastic Leukemia (ALL), Bi phenotypic/undifferentiated leukemia, Chronic Myeloid Leukemia resistant to TK inhibitors, Ph-neg Myeloproliferative Neoplasms, resistant/relapsing Non-Hodgkin's lymphoma ineligible for an autologous transplant.

Exclusion Criteria

• Positive serologic markers for human immunodeficiency virus (HIV)
• Acute hepatitis B virus (HBV) or acute hepatitis C virus (HCV) infection

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
hematopoietic reconstitution	first post-transplant 1 day with neutrophil > 0.5x10^9/L and platelet > 20x10^9/L	number of days for neutrophil (\> 0.5x10\^9/L) and platelet (\> 20x10\^9/L) engraftment

Secondary Outcome Measures

Name	Time	Method
immune reconstitution	day 100 from transplant	number of CD4+cells at day 100

Trial Locations

Locations (1)

Fondazione Policlinico Universitario A. Gemelli IRCCS; 🇮🇹 Roma, RM, Italy