Clinical trial NCT01728389: Intrabone Transplantation of Allogenic Peripheral Blood Stem Cells in Patients With Myeloid and Lymphoid Malignancies.
Official title: Efficacy and Safety of Direct Intrabone Transplantation of Peripheral Blood Haematopoietic Stem Cells Form HLA-matched Sibling Donors in Patients With Myeloid and Lymphoid Malignancies.
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE1
PHASE2
Conditions: Leukemia
Lymphoma
Keywords: allogenic stem cell transplantation
peripheral blood stem cells
intrabone
intra-bone
leukemia
lymphoma
Lead sponsor: Maria Sklodowska-Curie National Research Institute of Oncology
Sponsor class: OTHER
First posted: 2012-11-19
Enrollment: 10
Sex: ALL
Minimum age (years): 18
Maximum age (years): 55
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of the study is to evaluate efficacy and safety of direct intrabone transplantation procedure of peripheral blood haematopoietic stem cells form HLA-matched sibling donors in patients with myeloid and lymphoid malignancies.

Detailed description: Allogenic haematopoietic stem cell transplantation is an established treatment option for haematologic malignancies, especially leukaemias and lymphomas, providing curative potential. The optimal donor is HLA-matched sibling and G-CSF stimulated peripheral blood is nowadays the most common source of stem cells. Routinely used route of stem cell transplantation is intravenous infusion via central venous catheter. Based on the animal studies, only 10-15% of intravenously transplanted stem cells migrate to haematopoietic sites while the rest is lost in other organs. Results of studies of direct intrabone allogenic cord-blood cells transplantation in humans confirm that this route of transplantation is associated with less probability of graft failure and moreover may reduce risk of graft-versus-host disease and malignancy relapse. For those purposes in the current study we investigate intrabone route of allogenic peripheral blood stem cell transplantation. Our intention is to achieve fast engraftment and minimize risk of relapse and graft graft-versus-host disease.

Eligibility criteria: Inclusion Criteria:

* Age 18-55 years
* Diagnosis of leukaemia or lymphoma
* Indication for allogenic stem cell transplantation according to European Group for Blood and Marrow Transplantation guidelines
* HLA-matched sibling donor qualified for peripheral blood stem cell donation
* Performance status WHO 0-1
* Written informed consent

Exclusion Criteria:

* Organ dysfunction: elevated ALT, AST, bilirubin, AF; creatinine \>1.5 upper normal limit; LVEF \<45%
* Active infection
* Unstable diabetes
* Psychiatric diseases
* Obesity or anatomical obstacle for direct intrabone transplantation

Primary outcomes:
measure: Neutrophil engraftment (ANC > 0,5 G/l)
timeFrame: 28 days
Secondary outcomes:
measure: Lineage chimerism of peripheral blood T-cell lymphocytes
timeFrame: 1 year
measure: Platelet engraftment (Plt > 20 G/l)
timeFrame: 28 days
measure: Adverse reactions related with intrabone transplantation procedure
timeFrame: 28 days
Other outcomes:
measure: Relapse incidence
timeFrame: 1 year
measure: Overall survival
timeFrame: 1 year
measure: Disease-free survival
timeFrame: 1 year
measure: Graft-versus-host disease incidence
timeFrame: 1 year
Locations:
city: Gliwice
country: Poland
facility: MSC Memorial CAncer Center and Institute of Oncology
Study officials:
affiliation: Maria Sklodowska-Curie Memorial Cancer Center and Institute of Oncology, Gliwice Branch, Gliwice, Poland
name: Tomasz Czerw, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Frassoni F, Gualandi F, Podesta M, Raiola AM, Ibatici A, Piaggio G, Sessarego M, Sessarego N, Gobbi M, Sacchi N, Labopin M, Bacigalupo A. Direct intrabone transplant of unrelated cord-blood cells in acute leukaemia: a phase I/II study. Lancet Oncol. 2008 Sep;9(9):831-9. doi: 10.1016/S1470-2045(08)70180-3. Epub 2008 Aug 8.
citation: Okada M, Yoshihara S, Taniguchi K, Kaida K, Ikegame K, Kato R, Tamaki H, Inoue T, Soma T, Kai S, Kato S, Ogawa H. Intrabone marrow transplantation of unwashed cord blood using reduced-intensity conditioning treatment: a phase I study. Biol Blood Marrow Transplant. 2012 Apr;18(4):633-9. doi: 10.1016/j.bbmt.2011.08.010. Epub 2011 Aug 23.
citation: Massollo M, Podesta M, Marini C, Morbelli S, Cassanelli C, Pinto V, Ubezio G, Curti G, Uccelli A, Frassoni F, Sambuceti G. Contact with the bone marrow microenvironment readdresses the fate of transplanted hematopoietic stem cells. Exp Hematol. 2010 Oct;38(10):968-77. doi: 10.1016/j.exphem.2010.06.003. Epub 2010 Jun 13.