Bone Marrow Failure Disorders Clinical Trial
Official title:
A Multicenter Collaborative Clinical Study of Umbilical Cord Blood Combined With Umbilical Cord Derived Mesenchymal Stem Cells in the Treatment of Bone Marrow Failure Disorders
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.
| Status | Recruiting |
| Enrollment | 100 |
| Est. completion date | April 2026 |
| Est. primary completion date | April 2025 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | N/A and older |
| Eligibility | 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 ??? 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. |
| Country | Name | City | State |
|---|---|---|---|
| China | Affiliated Hospital of Shandong University of Traditional Chinese Medicine | Jinan | Shandong |
| China | Shandong Provincial Third Hospital | Jinan | Shandong |
| China | Jining first people's Hospital | Jining | Shandong |
| China | Linyi People's Hospital | Linyi | Shandong |
| China | Rizhao People's Hospital | Rizhao | Shandong |
| China | Tai'an Central Hospital | Tai'an | Shandong |
| China | Weihai Municipal Hospital | Weihai | Shandong |
| China | Yantai Ludong Hospital (Shandong Provincial Hospital Group) | Yantai | Shandong |
| Lead Sponsor | Collaborator |
|---|---|
| Shandong Qilu Stem Cells Engineering Co., Ltd. | Air Force Hospital of Western War Zone, Binzhou People's Hospital, Children's Hospital Affiliated to Shandong University, Dezhou People's Hospital, Gansu Provincial Hospital of Traditional Chinese Medicine (TCM), Gansu Wuwei Tumour Hospital, Jiangxi Province Children's Hospital, Kaifeng Central Hospital, Lanling People's Hospital, Linyi Central Hospital, Linyi People's Hospital, Rizhao People's Hospital, Second Affiliated Hospital of Zhengzhou University, Shandong Jining No.1 People's Hospital, Shandong Provincial Third Hospital, Shandong University of Traditional Chinese Medicine, Shengli Oilfield Central Hospital, Tai'an Central Hospital, The Affiliated Hospital of Binzhou Medical College, The affiliated hospital of Jining medical college, The First People' s Hospital of Yunnan Province, The First People's Hospital of Jingzhou, The Second Affiliated Hospital of Harbin Medical University, The Second Affiliated Hospital of Shandong First Medical University, The Second Affiliated Hospital of Shandong University of traditional Chinese Medicine, Weihai Central Hospital, Weihai Municipal Hospital, Wuwei People's Hospital, Yantai Ludong Hospital (Shandong Provincial Hospital Group), Yuncheng Institute of Hematology, Zhengzhou Central Hospital, Zibo municipal hospital, Zigong No.1 Peoples Hospital |
China,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Objective Response Rate (ORR) | To evaluate efficacy of UCB in combination with UC-MSCs in the treatment of bone marrow failure disorders | Up to two years | |
| Secondary | Progression-free Survival (PFS) | To assess Progression-free Survival (PFS) of the combination | Up to two years | |
| Secondary | Overall Survival (OS) | To assess overall survival (OS) of the combination | Up to two years |
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Active, not recruiting |
NCT04478227 -
TPO-Mimetic Use in Children for Hematopoietic Failure
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Early Phase 1 |