Bone Marrow Failure DisordersPhase 1/2NCT05794425
Clinical Study of UCB Combined With UC-MSCs in the Treatment of Bone Marrow Failure Disorders
A Phase 1/2 study testing Cyclosporine A and Umbilical cord blood & Umbilical cord derived mesenchymal stem cells in people with Bone Marrow Failure Disorders, run by Shandong Qilu Stem Cells Engineering Co., Ltd. at 8 sites in China.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Bone Marrow Failure Disorders
- What is being tested?
- Cyclosporine A and Umbilical cord blood & Umbilical cord derived mesenchymal stem cells
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 100 participants
- Where
- 8 sitesChina
- Study dates
- 1 Aug 2023to Apr 2026
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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 .
Who cannot join
- 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;
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- 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.
Trial locations and contacts
8 sites in 1 country.
8 sites in 1 country
China8 sites, 8 recruiting
- Recruiting
Shandong Provincial Third Hospital
Jinan, Shandong, 250031
zhe Yu, MD, 86-18753103739, doctoryu1120@163.com
- Recruiting
Affiliated Hospital of Shandong University of Traditional Chinese Medicine
Jinan, Shandong
yuan Fang, MD
- Recruiting
Jining first people's Hospital
Jining, Shandong
haiguo Zhang, wfmchero@126.com
- Recruiting
Linyi People's Hospital
Linyi, Shandong
yu Xu, xuyumm@qq.com
- Recruiting
Rizhao People's Hospital
Rizhao, Shandong
jun Guo
- Recruiting
Tai'an Central Hospital
Tai’an, Shandong
guanchen Bai
- Recruiting
Weihai Municipal Hospital
Weihai, Shandong
xiuzhi Deng
- Recruiting
Yantai Ludong Hospital (Shandong Provincial Hospital Group)
Yantai, Shandong
xiaolei Wang, MD, wxl1978@126.com
About this study
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
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.
- Conditions
- Bone Marrow Failure Disorders
- Being tested
- Cyclosporine A, Umbilical cord blood & Umbilical cord derived mesenchymal stem cells
- Medical subject headings
- Bone Marrow Failure Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Cyclosporine ADrug
3-5 mg per kilogram per day.
Umbilical cord blood & Umbilical cord derived mesenchymal stem cellsBiological
Intravenous infusion of umbilical cord blood and umbilical cord derived mesenchymal stem cells (5-8×10^7) at 1 week interval.
| Study group | Role | What they receive |
|---|---|---|
| Cyclosporine A | Experimental | Not described Receives: Drug: Cyclosporine A |
| UCB+UC-MSCs | Experimental | Not described Receives: Biological: Umbilical cord blood & Umbilical cord derived mesenchymal stem cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
Objective Response Rate (ORR)
To evaluate efficacy of UCB in combination with UC-MSCs in the treatment of bone marrow failure disorders
Measured: Up to two years
Secondary outcomes (2)
Progression-free Survival (PFS)
To assess Progression-free Survival (PFS) of the combination
Measured: Up to two years
Overall Survival (OS)
To assess overall survival (OS) of the combination
Measured: Up to two years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Apr 2023
- Study start1 Aug 2023
- Primary completionApr 2025 (estimated)
- Study completionApr 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Bone Marrow Failure DisordersStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Shandong Qilu Stem Cells Engineering Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Shandong Province Third hospital, Dezhou People's Hospital, Children's Hospital Affiliated to Shandong University, The Second Affiliated Hospital of Shandong First Medical University, Taian City Central Hospital, Linyi People's Hospital, Rizhao People's Hospital, Lanling People's Hospital, Jining First People's Hospital, Affiliated Hospital of Jining Medical University, Zibo Municipal Hospital, Binzhou People's Hospital, The Affiliated Hospital of Binzhou Medical College, Shengli Oilfield Hospital, Weihai Municipal Hospital, Weihai Central Hospital, Shandong University of Traditional Chinese Medicine, Gansu Provincial Hospital of Traditional Chinese Medicine (TCM), Wuwei People's Hospital, Gansu Wuwei Tumor 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, Second Affiliated Hospital of Zhengzhou University, Yantai Ludong Hospital (Shandong Provincial Hospital Group)
- Responsible party
- Sponsor
- Investigators
- zhe Yu, MD, Principal investigator, Shandong Province Third hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05794425
- Sponsor's protocol ID
- UCB&MSCs-BMF-2023
- Status last verified
- Nov 2023
- First submitted
- 20 Mar 2023
- Last updated
- 29 Nov 2023
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Nov 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT05794425
- Acronym / sponsor ID
- UCB&MSCs-BMF-2023
- Conditions
- Bone Marrow Failure Disorders
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- Shandong Qilu Stem Cells Engineering Co., Ltd.Industry (pharma, biotech, devices)
- Collaborators
- Shandong Province Third hospital, Dezhou People's Hospital, Children's Hospital Affiliated to Shandong University, The Second Affiliated Hospital of Shandong First Medical University, Taian City Central Hospital, Linyi People's Hospital, Rizhao People's Hospital, Lanling People's Hospital, Jining First People's Hospital, Affiliated Hospital of Jining Medical University, Zibo Municipal Hospital, Binzhou People's Hospital, The Affiliated Hospital of Binzhou Medical College, Shengli Oilfield Hospital, Weihai Municipal Hospital, Weihai Central Hospital, Shandong University of Traditional Chinese Medicine, Gansu Provincial Hospital of Traditional Chinese Medicine (TCM), Wuwei People's Hospital, Gansu Wuwei Tumor 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, Second Affiliated Hospital of Zhengzhou University, Yantai Ludong Hospital (Shandong Provincial Hospital Group)
- Study start
- 1 Aug 2023 (actual)
- Primary completion
- Apr 2025 (estimated)
- Study completion
- Apr 2026 (estimated)
- First posted
- 3 Apr 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cyclosporine A | Experimental | Drug: Cyclosporine A |
| UCB+UC-MSCs | Experimental | Biological: Umbilical cord blood & Umbilical cord derived mesenchymal stem cells |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (8 listed)
- 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;
Key exclusion criteria (10 listed)
- 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;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Cyclosporine A3-5 mg per kilogram per day. | Drug | Cyclosporine A |
| Umbilical cord blood & Umbilical cord derived mesenchymal stem cellsIntravenous infusion of umbilical cord blood and umbilical cord derived mesenchymal stem cells (5-8×10^7) at 1 week interval. | Biological | UCB+UC-MSCs |
Primary outcome
Objective Response Rate (ORR)
Time frame: Up to two years
To evaluate efficacy of UCB in combination with UC-MSCs in the treatment of bone marrow failure disorders
2 secondary outcomes
Progression-free Survival (PFS)
Time frame: Up to two years
Overall Survival (OS)
Time frame: Up to two years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Nov 2023.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
zhe Yu, MD principal investigator
Shandong Province Third hospital
8 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Cyclosporine A and Umbilical cord blood & Umbilical cord derived mesenchymal stem cells in people with Bone Marrow Failure Disorders, run by Shandong Qilu Stem Cells Engineering Co., Ltd. at 8 sites in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 29 Nov 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact zhe Yu, MD (86-18753103739, doctoryu1120@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Objective Response Rate (ORR) (time frame: Up to two years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Nov 2023.