Clinical trial NCT02385955: Phase II Study of Myeloablative Double Unit Umbilical Cord Blood Transplantation in Adult Patients
Official title: Prospective Multicenter Phase II Study of Myeloablative Double Unit Umbilical Cord Blood Transplantation in Adult Patients With Hematologic Malignancies
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
Conditions: Hematologic Malignancy
Lead sponsor: Seoul National University Hospital
Sponsor class: OTHER
Collaborators: name: The Korean Society of Blood and Marrow Transplantation
First posted: 2015-03-11
Enrollment: 39
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This study aimed to evaluate the efficacy and safety of myeloablative double unit umbilical cord blood transplantation (dUCBT) in adult patients with hematologic malignancies. Two myeloablative conditioning regimens will be used in this study: (1) total body irradation (TBI), cyclophosphamide, and cytarabine, or (2) thiotepa, busulfan, and fludarabine.


Eligibility criteria: Inclusion Criteria:

* Patients with hematologic malignancies requiring hematopoietic stem cell transplantation: (1) high risk acute myeloid leukemia in remission, (2) high risk acute lymphoblastic leukemia in remission, or (3) IPSS intermediate-2, high, or transfusion-dependent myelodysplastic syndrome
* Patients who have suitable cord blood units for dUCBT
* Patients with ECOG performance status 0-1
* Patients with adequate organ function: lung, kidney, liver, heart, etc.
* Informed consent

Exclusion Criteria:

* Patients who can receive hematopoietic stem cell transplantation from HLA-matched sibling or unrelated donors
* Patients who have anti-HLA antibodies
* Patients with any evidence of central nervous system (CNS) involvement of disease
* Patients with uncontrolled diabetes
* Patients with uncontrolled hypertension
* Patients with any evidence of active infection
* Positive for human immunodeficiency virus (HIV)
* Previous history of other malignancy within 5 years (Basal cell carcinoma of skin, cervical carcinoma in situ, and differentiated thyroid carcinoma with curative resection are permitted.)
* Women who are pregnant or breastfeeding
* Patients with previous history of allogeneic stem cell transplantation
* Patients with major psychotic disorder or drug/alcohol abuser
* Inappropriate patients according to the investigators' opinion

Primary outcomes:
measure: Overall survival
timeFrame: 2 years
Secondary outcomes:
measure: Progression-free survival
timeFrame: 2 years
measure: Time to relapse or progression
timeFrame: 2 years
measure: Time to engraftment
timeFrame: 2 years
measure: Acute graft-versus-host disease
timeFrame: 2 years
measure: Chronic graft-versus-host disease
timeFrame: 2 years
measure: Non-relapse mortality
timeFrame: 2 years
measure: Other transplant-related serious adverse events
timeFrame: 2 years
Other outcomes:
Locations:
Study officials:
References:
citation: Takahashi S, Ooi J, Tomonari A, Konuma T, Tsukada N, Oiwa-Monna M, Fukuno K, Uchiyama M, Takasugi K, Iseki T, Tojo A, Yamaguchi T, Asano S. Comparative single-institute analysis of cord blood transplantation from unrelated donors with bone marrow or peripheral blood stem-cell transplants from related donors in adult patients with hematologic malignancies after myeloablative conditioning regimen. Blood. 2007 Feb 1;109(3):1322-30. doi: 10.1182/blood-2006-04-020172. Epub 2006 Oct 12.
citation: Mori T, Tanaka M, Kobayashi T, Ohashi K, Fujisawa S, Yokota A, Fujita H, Nakaseko C, Sakura T, Nannya Y, Takahashi S, Kanamori H, Kanda Y, Sakamaki H, Okamoto S; Kanto Study Group for Cell Therapy. Prospective multicenter study of single-unit cord blood transplantation with myeloablative conditioning for adult patients with high-risk hematologic malignancies. Biol Blood Marrow Transplant. 2013 Mar;19(3):486-91. doi: 10.1016/j.bbmt.2012.12.007. Epub 2012 Dec 16.
citation: Sanz J, Boluda JC, Martin C, Gonzalez M, Ferra C, Serrano D, de Heredia CD, Barrenetxea C, Martinez AM, Solano C, Sanz MA, Sanz GF; Grupo Espanol de Trasplante Hematopoyetico y Terapia Celular (GETH). Single-unit umbilical cord blood transplantation from unrelated donors in patients with hematological malignancy using busulfan, thiotepa, fludarabine and ATG as myeloablative conditioning regimen. Bone Marrow Transplant. 2012 Oct;47(10):1287-93. doi: 10.1038/bmt.2012.13. Epub 2012 Feb 13.
citation: Ruggeri A, Sanz G, Bittencourt H, Sanz J, Rambaldi A, Volt F, Yakoub-Agha I, Ribera JM, Mannone L, Sierra J, Mohty M, Solano C, Nabhan S, Arcese W, Gluckman E, Labopin M, Rocha V; Eurocord and Acute Leukemia Working Party of European Blood and Marrow Transplant Group. Comparison of outcomes after single or double cord blood transplantation in adults with acute leukemia using different types of myeloablative conditioning regimen, a retrospective study on behalf of Eurocord and the Acute Leukemia Working Party of EBMT. Leukemia. 2014 Apr;28(4):779-86. doi: 10.1038/leu.2013.259. Epub 2013 Sep 5.