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

Primary Purpose

Bone Marrow Failure Disorders

Status
Not yet recruiting
Phase
Phase 1
Locations
China
Study Type
Interventional
Intervention
Cyclosporine A
Umbilical cord blood & Umbilical cord derived mesenchymal stem cells
Sponsored by
Shandong Qilu Stem Cells Engineering Co., Ltd.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Bone Marrow Failure Disorders

Eligibility Criteria

undefined - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria: 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 Criteria: 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.

Sites / Locations

  • Shandong Provincial Third Hospital

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Experimental

Arm Label

Cyclosporine A

UCB+UC-MSCs

Arm Description

Outcomes

Primary Outcome Measures

Objective Response Rate (ORR)
To evaluate efficacy of UCB in combination with UC-MSCs in the treatment of bone marrow failure disorders

Secondary Outcome Measures

Progression-free Survival (PFS)
To assess Progression-free Survival (PFS) of the combination
Overall Survival (OS)
To assess overall survival (OS) of the combination

Full Information

First Posted
March 20, 2023
Last Updated
March 20, 2023
Sponsor
Shandong Qilu Stem Cells Engineering Co., Ltd.
Collaborators
Shandong Third Provincial Hospital, Dezhou People's Hospital, Qilu Children's Hospital of Shandong University, The Second Affiliated Hospital of Shandong First Medical University, Tai'an Central Hospital, Linyi People's Hospital, Linyi Central Hospital, Rizhao People's Hospital, Lanling People's Hospital, Shandong Jining No.1 People's Hospital, The affiliated hospital of Jining medical college, Zibo municipal hospital, Binzhou People's Hospital, The Affiliated Hospital of Binzhou Medical College, Shengli Oilfield Central Hospital, Weihai Municipal Hospital, Weihai Central Hospital, The Second Affiliated Hospital of Shandong University of traditional Chinese Medicine, Gansu Provincial Hospital of Traditional Chinese Medicine (TCM), Wuwei People's Hospital, Gansu Wuwei Tumour Hospital, The Second Affiliated Hospital of Harbin Medical University, Yuncheng Institute of Hematology, Kaifeng Central Hospital, Zhengzhou Central Hospital, Air Force Hospital of Western War Zone, The First People' s Hospital of Yunnan Province, Zigong No.1 Peoples Hospital, The First People's Hospital of Jingzhou, Jiangxi Province Children's Hospital
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1. Study Identification

Unique Protocol Identification Number
NCT05794425
Brief Title
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

2. Study Status

Record Verification Date
March 2023
Overall Recruitment Status
Not yet recruiting
Study Start Date
April 2023 (Anticipated)
Primary Completion Date
April 2025 (Anticipated)
Study Completion Date
April 2026 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Shandong Qilu Stem Cells Engineering Co., Ltd.
Collaborators
Shandong Third Provincial Hospital, Dezhou People's Hospital, Qilu Children's Hospital of Shandong University, The Second Affiliated Hospital of Shandong First Medical University, Tai'an Central Hospital, Linyi People's Hospital, Linyi Central Hospital, Rizhao People's Hospital, Lanling People's Hospital, Shandong Jining No.1 People's Hospital, The affiliated hospital of Jining medical college, Zibo municipal hospital, Binzhou People's Hospital, The Affiliated Hospital of Binzhou Medical College, Shengli Oilfield Central Hospital, Weihai Municipal Hospital, Weihai Central Hospital, The Second Affiliated Hospital of Shandong University of traditional Chinese Medicine, Gansu Provincial Hospital of Traditional Chinese Medicine (TCM), Wuwei People's Hospital, Gansu Wuwei Tumour Hospital, The Second Affiliated Hospital of Harbin Medical University, Yuncheng Institute of Hematology, Kaifeng Central Hospital, Zhengzhou Central Hospital, Air Force Hospital of Western War Zone, The First People' s Hospital of Yunnan Province, Zigong No.1 Peoples Hospital, The First People's Hospital of Jingzhou, Jiangxi Province Children's Hospital

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Bone Marrow Failure Disorders

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
100 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Cyclosporine A
Arm Type
Experimental
Arm Title
UCB+UC-MSCs
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Cyclosporine A
Intervention Description
3-5 mg per kilogram per day.
Intervention Type
Biological
Intervention Name(s)
Umbilical cord blood & Umbilical cord derived mesenchymal stem cells
Intervention Description
Intravenous infusion of umbilical cord blood and umbilical cord derived mesenchymal stem cells (5-8×10^7) at 1 week interval.
Primary Outcome Measure Information:
Title
Objective Response Rate (ORR)
Description
To evaluate efficacy of UCB in combination with UC-MSCs in the treatment of bone marrow failure disorders
Time Frame
Up to two years
Secondary Outcome Measure Information:
Title
Progression-free Survival (PFS)
Description
To assess Progression-free Survival (PFS) of the combination
Time Frame
Up to two years
Title
Overall Survival (OS)
Description
To assess overall survival (OS) of the combination
Time Frame
Up to two years

10. Eligibility

Sex
All
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: 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 Criteria: 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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
zhe Yu, MD
Phone
86-18753103739
Email
doctoryu1120@163.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
zhe Yu, MD
Organizational Affiliation
Shandong Provincial Third Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
Shandong Provincial Third Hospital
City
Jinan
State/Province
Shandong
ZIP/Postal Code
250031
Country
China
Facility Contact:
First Name & Middle Initial & Last Name & Degree
zhe Yu, MD
Phone
86-18753103739
Email
doctoryu1120@163.com

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

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