Clinical Study of UCB Combined With UC-MSCs in the Treatment of Bone Marrow Failure Disorders

• Status: Not yet recruiting
• Trial end date: 2026-04-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Shandong Qilu Stem Cells Engineering Co., Ltd.
• Collaborators: Air Force Hospital of Western War Zone; Binzhou People's Hospital; 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; Qilu Children's Hospital of Shandong University; Rizhao People's Hospital; Shandong Jining No.1 People's Hospital; Shandong Third Provincial Hospital; 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; Yuncheng Institute of Hematology; Zhengzhou Central Hospital; Zibo municipal hospital; Zigong No.1 Peoples Hospital
• Treatments: Cyclosporine; Cyclosporins

Criteria

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.