Clinical trial NCT03802773: Cord Blood Transplantation With Myeloablative Conditioning and Post-transplant Cyclophosphamide (COmPACt Study)
Official title: Cord Blood Transplantation With Myeloablative Conditioning and Post-transplant Cyclophosphamide in Patients With Hematological Malignancies (The COmPACt Study)
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Cord Blood Transplantation
Hematological Malignancies
Lead sponsor: Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Sponsor class: OTHER
First posted: 2019-01-14
Enrollment: 10
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients with no matched (\<7/8 HLA loci) sibling or unrelated donor, candidate to receive a transplant of a matched CB unit (TNC\> 2,0 x10\^7/kg and \> 4/6 loci HLA)

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

Eligibility criteria: 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

Primary outcomes:
measure: hematopoietic reconstitution
timeFrame: first post-transplant 1 day with neutrophil > 0.5x10^9/L and platelet > 20x10^9/L
Secondary outcomes:
measure: immune reconstitution
timeFrame: day 100 from transplant
Other outcomes:
Locations:
city: Roma
country: Italy
facility: Fondazione Policlinico Universitario A. Gemelli IRCCS
state: RM
Study officials:
affiliation: Fondazione Policlinico Universitario A. Gemelli, IRCCS
name: Patrizia Chiusolo, MD
role: PRINCIPAL_INVESTIGATOR
References: