Bone Marrow Failure Disorders
Conditions
Brief summary
The multicenter collaborative clinical study conducted a systematic clinical observation in the treatment of bone marrow failure diseases via UCB&UC-MSCs , in order to observe its clinical efficacy and safety.
Interventions
DRUGCyclosporine A
3-5 mg per kilogram per day.
BIOLOGICALUmbilical cord blood & Umbilical cord derived mesenchymal stem cells
Intravenous infusion of umbilical cord blood and umbilical cord derived mesenchymal stem cells (5-8×10\^7) at 1 week interval.
Sponsors
Shandong Province Third hospital
Dezhou People's Hospital
Children's Hospital Affiliated to Shandong University
The Second Affiliated Hospital of Shandong First Medical University
Tai'an Central Hospital
Linyi People's Hospital
Rizhao People's Hospital
Lanling People's Hospital
Jining First People's Hospital
Jining Medical University
Zibo Municipal Hospital
Binzhou People's Hospital
The Affiliated Hospital of Binzhou Medical College
Shengli Oilfield Hospital
Weihai Municipal Hospital
Weihai Central Hospital
Shandong University of Traditional Chinese Medicine
Gansu Provincial Hospital of Traditional Chinese Medicine (TCM)
Wuwei People's Hospital
Gansu Wuwei Tumor Hospital
The Second Affiliated Hospital of Harbin Medical University
Yuncheng Institute of Hematology
Kaifeng Central Hospital
Zhengzhou Central Hospital
Air Force Hospital of Western War Zone
The First People' s Hospital of Yunnan Province
Zigong No.1 Peoples Hospital
The First People's Hospital of Jingzhou
Jiangxi Province Children's Hospital
Second Affiliated Hospital of Zhengzhou University
Yantai Ludong Hospital (Shandong Provincial Hospital Group)
Shandong Qilu Stem Cells Engineering Co., Ltd.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
1. Patients with bone marrow failure disorders, including aplastic anemia, pure red cell aplastic anemia, immune-related pancytopenia, paroxysmal nocturnal hemoglobinuria, myelodysplastic syndrome, acute arrest of hemopoiesis, and cytopenia of unknown significance; 2. Patients with no severe impairment of liver and kidney function (total bilirubin (TBIL): ≤ 1.5×ULN; ALT or AST: ≤ 2.5×ULN; Alkaline phosphatase: ≤ 3×ULN; Serum creatinine: ≤ 1.5×ULN); 3. The prothrombin time (PT) or activated partial thrombin time (APTT) or international normalized ratio (INR): ≤ 1.5×ULN in the absence of anticoagulant therapy; 4. The left ventricular ejection fraction (LVEF): ≥50% by cardiac echocardiography 5. Hepatitis B surface antigen (HBsAg) and Hepatitis B core antibody (HBcAb) were negative. If any of the above items is positive, the HBV DNA titer in peripheral blood must be lower than the detection limit or 1×10\^3 copys/ml; 6. Patients with no plans for stem cell transplantation; 7. Patients with ECOG 0-2; 8. Those who voluntarily participate in this clinical study and have signed an informed consent .
Exclusion criteria
1. Patients who have suffered malignant tumors other than squamous cell carcinoma of the skin, basal cell carcinoma of the skin, malignant melanoma cured by surgery, and carcinoma in situ of the cervix in the past 5 years; 2. Patient with severe cardiac insufficiency (e.g. grade Ⅲ、Ⅳ by NYHA classification NYHA), and medically uncontrolled hypertension (systolic blood pressure ≥ 160 mmHg or diastolic blood pressure ≥110 mmHg); 3. Patients with severe mental illness; 4. Patients with clinically significant infection should be recruited with delay; 5. AST or ALT: above 3 times the upper limit of normal, creatinine, total bilirubin, or alkaline: phosphatase (ALP): above 1.5 times the upper limit of normal; 6. Patients with test positive for HIV, HCV or syphilis; 7. Patients with severe allergies or allergic to the active ingredients, excipients of the drug, or blood products in this clinical trials; 8. Patients who are pregnant or breastfeeding, or have a childbirth plan in the near future; 9. Patients receive allogeneic/autologous hematopoietic stem cell transplantation by the assessment of investigators; 10. There are other conditions that the investigators consider inappropriate for inclusion.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Objective Response Rate (ORR) | Up to two years | To evaluate efficacy of UCB in combination with UC-MSCs in the treatment of bone marrow failure disorders |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Progression-free Survival (PFS) | Up to two years | To assess Progression-free Survival (PFS) of the combination |
| Overall Survival (OS) | Up to two years | To assess overall survival (OS) of the combination |
Countries
China
Contacts
Primary Contactzhe Yu, MD
Outcome results
None listed