Clinical trial NCT01129739: Safety and Efficacy Study of Umbilical Cord/Placenta-Derived Mesenchymal Stem Cells to Treat Myelodysplastic Syndromes
Official title: Phase II Study of Umbilical Cord/Placenta-Derived Mesenchymal Stem Cells to Treat RA and RARS of MDS
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
Conditions: Myelodysplastic Syndromes
Keywords: Bone Marrow Disease
MDS-RA
MDS-RARS
Umbilical Cord/placenta-Derived MSC
Transplant
Lead sponsor: Shandong University
Sponsor class: OTHER
Collaborators: name: National Natural Science Foundation of China
First posted: 2010-05-25
Enrollment: 30
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to evaluate the safety and efficacy of mesenchymal stem cells (MSCs) derived from human umbilical cord/placenta at a dose of 1.0E+6 MSC/kg on the subjects for refractory anemia (RA) and refractory anemia with ring sideroblast (RARS) of myelodysplastic syndromes (MDS).

Detailed description: Myelodysplastic syndromes are bone marrow stem cell disorders resulting in disorderly and ineffective hematopoiesis. MDS is characterized by variable degrees of cytopenias (anemia, neutropenia, and thrombocytopenia ) and risk of transformation to leukemia.

To date treatment of MDS is unsatisfactory: chemotherapy has a limited role in the management of leukemic progression; autologous stem cell transplantation does not prolong relapse-free survival and stem cell transplantation is poorly tolerated in older individuals. Some MDS patients have been shown to respond to a wide variety of immunosuppressive agents ranging from corticosteroids to CsA and antithymocyte globulin (ATG). However, the overall response rate is less than 30%. In fact, few treatments appear to change the natural history of MDS.

The management of MDS patients therefore remains to be improved. Human MSCs isolated from Wharton's jelly of the umbilical cord/placenta have been shown to have immunosuppressive, stimulating hematopoiesis and tissue repairing properties. This study will evaluate the safety and effectiveness of MSC transplant in the MDS patients.

This study will last about 3 years. Participants will be randomly assigned to receive either MSC transplant (Group 1) or CsA therapy alone (Group 2). Patients will undergo MSC transplant at the start of the study (defined as Day 0). After 3 months, patients will receive the second MSC transplantation when one responds well to the treatment. After 3, 6 and 12 months from the first transplantation, patients will be evaluated.

Eligibility criteria: Inclusion Criteria:

1. Patient age 18\~80 years old with plan to infuse MSCs.
2. Histologically documented or cytologically confirmed diagnosis of MDS with WHO classification of MDS-RA and MDS-RARS.
3. Patients must have an ECOG 0\~2.
4. No moderate or sever organ dysfunction: Ejection fraction\>45%; Creatinine \<176 mmol/L.
5. No active severe viral or fungus infection.
6. Each patient must sign written informed consent.

Exclusion Criteria:

1. Psychiatric condition that would limit informed consent.
2. HIV positive
3. Positive Pregnancy Test
4. Patient has enrolled another clinical trial study within last 4 weeks.

Primary outcomes:
measure: MDS clinical symptoms (mainly anemia symptoms)
timeFrame: 1 year
measure: A routine blood test
timeFrame: 1 year
measure: Bone borrow cytomorphologic examination
timeFrame: 1 year
Secondary outcomes:
measure: Percentage of T regulatory cell population in peripheral blood
timeFrame: 1 year
Other outcomes:
Locations:
city: Jinan
country: China
facility: Department of Hematology of the 2nd Hospital of Shandong University
state: Shandong
Study officials:
affiliation: Department of Hematology of The 2nd Hospital of Shandong University
name: cheng yun zheng, PhD
role: PRINCIPAL_INVESTIGATOR
References: