Clinical trial NCT00447460: Treatment of Refractory (Acute or Chronic) Graft-Versus-Host Disease by the Infusion of Expanded in-Vitro Allogenic Mesenchymal Stem Cell
Official title: A Phase I/II Trial in Treating Patients With Graft-Versus-Host Disease by the Infusion of Expanded in-Vitro Allogenic Mesenchymal Stem Cell
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE1
PHASE2
Conditions: Graft-vs-Host Disease (GVHD)
Keywords: Graft-vs-host disease (GVHD)
Mesenchymal stem cell (MSC)
Allogeneic hematopoietic stem cell transplant
Allotransplant
Allogenic
Refractory
CSM/EICH2005
Lead sponsor: University of Salamanca
Sponsor class: OTHER
Collaborators: name: Haematology Service,University Hospital of Salamanca, MªConsuelo del Cañizo Fernández-Roldán
First posted: 2007-03-14
Enrollment: 15
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: Clinical trial based on the use of a new therapeutic strategy (MSC infusion) for the treatment of patients who have developed a GVHD refractory to the usual therapeutic measures after undergoing an allogenic hematopoietic stem cell transplant.

Detailed description: This is a multicenter, single dose study of allogenic mesenchymal stem cell (1-2 x 10\^6 MSC/Kg recipient´s bodyweight).

MSC will be infused, by a central venous catheter, to patients diagnosed with GVHD refractory to first-line or subsequent treatment.

All patients will receive the same treatment. MSC suspension will be obtained from the bone marrow aspiration of a family donor and expanded in-vitro in a specific culture medium with autologous donor´s serum and with no animal-derived products.

Eligibility criteria: Inclusion Criteria:

* Patients with hematological malignancies who had been undergone an allogenic hematopoietic stem cell transplant and diagnosed with GVHD refractory to a usual treatment.
* Adequate cardiac function with no evidence of uncontrolled high blood pressure,congestive heart failure, angina pectoris, acute myocardial infarction within 6 months prior to the process.
* Adequate pulmonary function with no evidence of chronic obstructive or severe restrictive pulmonary disease.
* Patients with ages between 18 and 65 years.
* Signature of informed consent form is required to be done by patient and donor.

Exclusion Criteria:

* Patients whose hematopathology has not been controlled by the transplant or is in progress.
* Patients with bacterial, viral or fungal infection not being controlled with the adequate treatment.
* Patients with an inadequate cardiac or pulmonary function.
* Patients who, in the investigator´s point of view, are not in situation to tolerate the treatment.
* Patients who do not have the required donor (HLA-identical sibling donor and not HLA-identical sibling donor).
* Pregnant females or childbearing potential who are not on adequate contraceptive measures.
* Patients \<18 or \>65 years.
* Patients who do not sign the informed consent.

Primary outcomes:
measure: Determine the safety/efficacy of expanded in-vitro allogenic mesenchymal stem cell infusion in patients developing a GVHD refractory to the usual therapeutic measures after undergoing an allogeneic hematopoietic stem cell transplant.
measure: Efficacy will be evaluated in terms of GVHD response.
measure: Safety will be evaluated in terms of incidence of adverse events and toxicities related to the administration of MSC.
Secondary outcomes:
measure: Evaluation of infectious complications after MSC infusion.
measure: Analyse the influence of MSC infusion on the posttransplant relapse risk of the base disease.
measure: Study the influence of MSC infusion on DFS and OS.
measure: Determine MSC grafted into the bone marrow (or in other organs).
Other outcomes:
Locations:
city: Navarra
country: Spain
facility: University Clinic of Navarra
state: Pamplona
city: Barcelona
country: Spain
facility: Santa Creu i Sant Pau Hospital
city: Barcelona
country: Spain
facility: Clinic i Provincial Hospital
city: Salamanca
country: Spain
facility: University Hospital of Salamanca
Study officials:
affiliation: Haematology Service, University Hospital of Salamanca
name: Consuelo del Cañizo, MD
role: STUDY_CHAIR
affiliation: Haematology Service, University Hospital of Salamanca
name: José A Pérez-Simón, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Haematology Service, Santa Creu i Sant Pau Hospital, Barcelona
name: David Varcárcel Ferrerías, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Haematology Service, Clinic i Provincial Hospital, Barcelona
name: Carmen Martínez Muñoz, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Haematology Service, University Clinic of Navarra
name: José Rifón Roca, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Perez-Simon JA, Lopez-Villar O, Andreu EJ, Rifon J, Muntion S, Diez Campelo M, Sanchez-Guijo FM, Martinez C, Valcarcel D, Canizo CD. Mesenchymal stem cells expanded in vitro with human serum for the treatment of acute and chronic graft-versus-host disease: results of a phase I/II clinical trial. Haematologica. 2011 Jul;96(7):1072-6. doi: 10.3324/haematol.2010.038356. Epub 2011 Mar 10.