Clinical trial NCT05794425: Clinical Study of UCB Combined With UC-MSCs in the Treatment of Bone Marrow Failure Disorders
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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
PHASE2
Conditions: Bone Marrow Failure Disorders
Lead sponsor: Shandong Qilu Stem Cells Engineering Co., Ltd.
Sponsor class: INDUSTRY
Collaborators: name: Shandong Province Third hospital
name: Dezhou People's Hospital
name: Children's Hospital Affiliated to Shandong University
name: The Second Affiliated Hospital of Shandong First Medical University
name: Tai'an Central Hospital
name: Linyi People's Hospital
name: Rizhao People's Hospital
name: Lanling People's Hospital
name: Jining First People's Hospital
name: Jining Medical University
name: Zibo Central Hospital
name: Binzhou People's Hospital
name: The Affiliated Hospital of Binzhou Medical College
name: Shengli Oilfield Hospital
name: Weihai Municipal Hospital
name: Weihai Central Hospital
name: Shandong University of Traditional Chinese Medicine
name: Gansu Provincial Hospital of Traditional Chinese Medicine (TCM)
name: Wuwei People's Hospital
name: Gansu Wuwei Tumor Hospital
name: The Second Affiliated Hospital of Harbin Medical University
name: Yuncheng Institute of Hematology
name: Kaifeng Central Hospital
name: Zhengzhou Central Hospital
name: Air Force Hospital of Western War Zone
name: The First People' s Hospital of Yunnan Province
name: Zigong No.1 Peoples Hospital
name: The First People's Hospital of Jingzhou
name: Jiangxi Province Children's Hospital
name: Second Affiliated Hospital of Zhengzhou University
name: Yantai Ludong Hospital (Shandong Provincial Hospital Group)
First posted: 2023-04-03
Enrollment: 100
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


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.

Primary outcomes:
measure: Objective Response Rate (ORR)
timeFrame: Up to two years
Secondary outcomes:
measure: Progression-free Survival (PFS)
timeFrame: Up to two years
measure: Overall Survival (OS)
timeFrame: Up to two years
Other outcomes:
Locations:
city: Jinan
country: China
facility: Shandong Provincial Third Hospital
state: Shandong
city: Jinan
country: China
facility: Affiliated Hospital of Shandong University of Traditional Chinese Medicine
state: Shandong
city: Jining
country: China
facility: Jining first people's Hospital
state: Shandong
city: Linyi
country: China
facility: Linyi People's Hospital
state: Shandong
city: Rizhao
country: China
facility: Rizhao People's Hospital
state: Shandong
city: Tai'an
country: China
facility: Tai'an Central Hospital
state: Shandong
city: Weihai
country: China
facility: Weihai Municipal Hospital
state: Shandong
city: Yantai
country: China
facility: Yantai Ludong Hospital (Shandong Provincial Hospital Group)
state: Shandong
Study officials:
affiliation: Shandong Province Third hospital
name: zhe Yu, MD
role: PRINCIPAL_INVESTIGATOR
References: