RecruitingPhase 1Phase 2NCT05794425

Clinical Study of UCB Combined With UC-MSCs in the Treatment of 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


Sponsor

Shandong Qilu Stem Cells Engineering Co., Ltd.

Enrollment

100 participants

Start Date

Aug 1, 2023

Study Type

INTERVENTIONAL

Conditions

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.


Eligibility

Inclusion Criteria8

  • 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;
  • 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);
  • The prothrombin time (PT) or activated partial thrombin time (APTT) or international normalized ratio (INR): ≤ 1.5×ULN in the absence of anticoagulant therapy;
  • The left ventricular ejection fraction (LVEF): ≥50% by cardiac echocardiography
  • 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;
  • Patients with no plans for stem cell transplantation;
  • Patients with ECOG 0-2;
  • Those who voluntarily participate in this clinical study and have signed an informed consent .

Exclusion Criteria10

  • 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;
  • 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);
  • Patients with severe mental illness;
  • Patients with clinically significant infection should be recruited with delay;
  • 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;
  • Patients with test positive for HIV, HCV or syphilis;
  • Patients with severe allergies or allergic to the active ingredients, excipients of the drug, or blood products in this clinical trials;
  • Patients who are pregnant or breastfeeding, or have a childbirth plan in the near future;
  • Patients receive allogeneic/autologous hematopoietic stem cell transplantation by the assessment of investigators;
  • There are other conditions that the investigators consider inappropriate for inclusion.

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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.


Locations(8)

Shandong Provincial Third Hospital

Jinan, Shandong, China

Affiliated Hospital of Shandong University of Traditional Chinese Medicine

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, China

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NCT05794425