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

NCT ID: NCT05794425

Last Updated: 2023-11-29

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

100 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-08-01

Study Completion Date

2026-04-30

Brief Summary

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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.

Detailed Description

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Conditions

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Bone Marrow Failure Disorders

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Cyclosporine A

Group Type EXPERIMENTAL

Cyclosporine A

Intervention Type DRUG

3-5 mg per kilogram per day.

UCB+UC-MSCs

Group Type EXPERIMENTAL

Umbilical cord blood & Umbilical cord derived mesenchymal stem cells

Intervention Type BIOLOGICAL

Intravenous infusion of umbilical cord blood and umbilical cord derived mesenchymal stem cells (5-8×10\^7) at 1 week interval.

Interventions

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Cyclosporine A

3-5 mg per kilogram per day.

Intervention Type DRUG

Umbilical 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.

Intervention Type BIOLOGICAL

Eligibility Criteria

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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.
Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Shandong Province Third hospital

OTHER

Sponsor Role collaborator

Dezhou People's Hospital

OTHER

Sponsor Role collaborator

Children's Hospital Affiliated to Shandong University

UNKNOWN

Sponsor Role collaborator

The Second Affiliated Hospital of Shandong First Medical University

OTHER

Sponsor Role collaborator

Tai'an Central Hospital

UNKNOWN

Sponsor Role collaborator

Linyi People's Hospital

OTHER

Sponsor Role collaborator

Rizhao People's Hospital

OTHER

Sponsor Role collaborator

Lanling People's Hospital

UNKNOWN

Sponsor Role collaborator

Jining First People's Hospital

OTHER

Sponsor Role collaborator

Jining Medical University

OTHER

Sponsor Role collaborator

Zibo Municipal Hospital

OTHER

Sponsor Role collaborator

Binzhou People's Hospital

OTHER

Sponsor Role collaborator

The Affiliated Hospital of Binzhou Medical College

UNKNOWN

Sponsor Role collaborator

Shengli Oilfield Hospital

OTHER

Sponsor Role collaborator

Weihai Municipal Hospital

OTHER

Sponsor Role collaborator

Weihai Central Hospital

OTHER

Sponsor Role collaborator

Shandong University of Traditional Chinese Medicine

OTHER

Sponsor Role collaborator

Gansu Provincial Hospital of Traditional Chinese Medicine (TCM)

UNKNOWN

Sponsor Role collaborator

Wuwei People's Hospital

UNKNOWN

Sponsor Role collaborator

Gansu Wuwei Tumor Hospital

OTHER

Sponsor Role collaborator

The Second Affiliated Hospital of Harbin Medical University

OTHER

Sponsor Role collaborator

Yuncheng Institute of Hematology

UNKNOWN

Sponsor Role collaborator

Kaifeng Central Hospital

OTHER

Sponsor Role collaborator

Zhengzhou Central Hospital

OTHER

Sponsor Role collaborator

Air Force Hospital of Western War Zone

UNKNOWN

Sponsor Role collaborator

The First People' s Hospital of Yunnan Province

UNKNOWN

Sponsor Role collaborator

Zigong No.1 Peoples Hospital

OTHER

Sponsor Role collaborator

The First People's Hospital of Jingzhou

OTHER

Sponsor Role collaborator

Jiangxi Province Children's Hospital

OTHER

Sponsor Role collaborator

Second Affiliated Hospital of Zhengzhou University

OTHER

Sponsor Role collaborator

Yantai Ludong Hospital (Shandong Provincial Hospital Group)

UNKNOWN

Sponsor Role collaborator

Shandong Qilu Stem Cells Engineering Co., Ltd.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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zhe Yu, MD

Role: PRINCIPAL_INVESTIGATOR

Shandong Province Third hospital

Locations

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Shandong Provincial Third Hospital

Jinan, Shandong, China

Site Status RECRUITING

Affiliated Hospital of Shandong University of Traditional Chinese Medicine

Jinan, Shandong, China

Site Status RECRUITING

Jining first people's Hospital

Jining, Shandong, China

Site Status RECRUITING

Linyi People's Hospital

Linyi, Shandong, China

Site Status RECRUITING

Rizhao People's Hospital

Rizhao, Shandong, China

Site Status RECRUITING

Tai'an Central Hospital

Tai’an, Shandong, China

Site Status RECRUITING

Weihai Municipal Hospital

Weihai, Shandong, China

Site Status RECRUITING

Yantai Ludong Hospital (Shandong Provincial Hospital Group)

Yantai, Shandong, China

Site Status RECRUITING

Countries

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China

Central Contacts

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zhe Yu, MD

Role: CONTACT

Phone: 86-18753103739

Email: [email protected]

Facility Contacts

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zhe Yu, MD

Role: primary

yuan Fang, MD

Role: primary

haiguo Zhang

Role: primary

yu Xu

Role: primary

jun Guo

Role: primary

guanchen Bai

Role: primary

xiuzhi Deng

Role: primary

xiaolei Wang, MD

Role: primary

Other Identifiers

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UCB&MSCs-BMF-2023

Identifier Type: -

Identifier Source: org_study_id