Using umbilical cord blood and stem cells to treat bone marrow failure disorders

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

This study is testing if a new treatment using umbilical cord blood and stem cells can help people with bone marrow failure disorders like aplastic anemia feel better.

Quick facts

• Phase: Phase1; Phase2
• Study type: Interventional
• Enrollment: 100 (estimated)
• Sex: All
• Sponsor: Shandong Qilu Stem Cells Engineering Co., Ltd. (Industry-sponsored)
• Locations: 8 sites (Jinan, Shandong and 7 other locations)
• Trial ID: NCT05794425 on ClinicalTrials.gov

What this trial studies

This clinical study evaluates the efficacy and safety of combining umbilical cord blood (UCB) with umbilical cord-derived mesenchymal stem cells (UC-MSCs) in treating various bone marrow failure disorders. It involves a multicenter approach, systematically observing patients with conditions such as aplastic anemia and myelodysplastic syndrome. The study will assess the clinical outcomes and potential benefits of this innovative treatment strategy. Participants will receive interventions including cyclosporine A alongside the stem cell therapies.

Who should consider this trial

Good fit: Ideal candidates include patients diagnosed with specific bone marrow failure disorders who meet the eligibility criteria outlined in the study.

Not a fit: Patients with severe liver or kidney impairment or those planning for stem cell transplantation may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for patients suffering from bone marrow failure disorders.

How similar studies have performed: While this approach is innovative, similar studies have shown promise in using stem cells for treating hematological conditions, suggesting potential for success.

Eligibility 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.

Where this trial is running

Jinan, Shandong and 7 other locations

• Shandong Provincial Third Hospital — Jinan, Shandong, China (Recruiting)
• Affiliated Hospital of Shandong University of Traditional Chinese Medicine — Jinan, Shandong, China (Recruiting)
• Jining first people's Hospital — Jining, Shandong, China (Recruiting)
• Linyi People's Hospital — Linyi, Shandong, China (Recruiting)
• Rizhao People's Hospital — Rizhao, Shandong, China (Recruiting)
• Tai'an Central Hospital — Tai'an, Shandong, China (Recruiting)
• Weihai Municipal Hospital — Weihai, Shandong, China (Recruiting)
• Yantai Ludong Hospital (Shandong Provincial Hospital Group) — Yantai, Shandong, China (Recruiting)

Study contacts

• Principal investigator: zhe Yu, MD — Shandong Province Third hospital
• Study coordinator: zhe Yu, MD

Conditions Bone Marrow Failure Disorders