Aplastic AnemiaPhase 1NCT03055078
Umbilical Cord Derived Mesenchymal Stem Cells Therapy in Aplastic Anemia
A Phase 1 study testing mesenchymal stem cells in people with Aplastic Anemia, run by Hebei Medical University at 1 site in China.
Trial summary
This study is to evaluate the safety and efficacy of Umbilical Cord Derived Mesenchymal Stem Cells transplantation in aplastic anemia. Read more about this study
- Who is it for?
- Ages 14 to 60 years
- Conditions the study is for
- Aplastic Anemia
- What is being tested?
- mesenchymal stem cells
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteChina
- Study dates
- Jan 2017to Dec 2019
- Phase
- Phase 1
- 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 14 to 60 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Diagnosis of AA according to established criteria in 2010
- Age from 14 to 60 years
- Suffering from AA within six months
- No serious infection or acute hemorrhage.
- Left ventricular ejection fraction (LVEF) ≥ 50%
- No acute infectious diseases.
- Understanding and willingness to sign a written informed consent document.
- Eastern Cooperative Oncology Group(ECOG) score of 0-2pionts.
Who cannot join
- Patients with AA have to be disqualified from this study if any of the following is applicable.
- Severe aplastic anemia(SAA) with severe infection.
- Severe aplastic anemia(SAA) with active hemorrhage.
- Severe heart attack, liver and kidney disease following serious complications
- Patients with allergic constitution.
- Pregnancy and lactation.
- Accompanied by malignant tumors and other clonal disease.
- Patients with active tuberculosis, acute sever hepatitis or infectious period of diseases.
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
the First Hospital of Hebei Medical University
Shijiazhuang, Hebei, 050031
Jun Zhang, Master, 86-311-85917384, june_luck@126.com
About this study
Official title: Evaluation of Safety and Efficacy in Patients With Aplastic Anemia by Transplantation of Umbilical Cord Derived Mesenchymal Stem Cells
This study is to evaluate the safety and efficacy of Umbilical Cord Derived Mesenchymal Stem Cells transplantation in aplastic anemia.
Read the detailed description
Aplastic anemia (AA) is a disorder thought to be caused by an immune-mediated bone marrow failure. Not all people with AA are eligible for today's standard treatments. One new treatment approach uses umbilical cord derived mesenchymal stem cells-specialized cells capable of developing into other types of cells-to provide the basis for clinical application. The aim of the present study is to investigate the safety and efficacy of vein infusion of allogeneic mesenchymal stem cells in patients with AA .
- Conditions
- Aplastic Anemia
- Being tested
- mesenchymal stem cells
- Medical subject headings
- Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
mesenchymal stem cellsBiological
Patients in the cell treated group were given umbilical cord derived mesenchymal stem cells by intravenous infusion besides conventional therapy. There was only conventional treatment in the control group. All patients after treatment for 1months, 3months, 6months and 9months were evaluated respectively the therapeutic effect.
| Study group | Role | What they receive |
|---|---|---|
| mesenchymal stem cells | Experimental | According to the inclusion and exclusion criteria, selected patients were divided into a cell therapy group and a control group. Umbilical cord derived mesenchymal stem cells at a dose of 100-300 million by intravenous infusion. Receives: Biological: mesenchymal stem cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
Hemoglobin levels in peripheral blood
The recovery of bone marrow erythrocyte is evaluated by hemoglobin levels (g/L) in peripheral blood.
Measured: Post cell transplantation: 9months
Secondary outcomes (5)
The number of red blood cell in peripheral blood
The improvement of bone marrow erythrocyte is evaluated by red blood cell count (×10^12/L) in peripheral blood.
Measured: Post cell transplantation: 1, 3, 6, 9months
The number of granulocyte in peripheral blood
The recovery of bone marrow granulocyte is evaluated by granulocyte count (×10^9/L) in peripheral blood.
Measured: Post cell transplantation: 1, 3, 6, 9months
The number of white blood cell in peripheral blood
The improvement of bone marrow granulocyte is evaluated by white blood cell count (×10^9/L) in peripheral blood.
Measured: Post cell transplantation: 1, 3, 6, 9months
The evaluation of bone marrow megakaryocytic series
The status of megakaryocytic series is evaluated by platelet count (×10^9/L) in peripheral blood.
Measured: Post cell transplantation: 1, 3, 6, 9months
adverse reaction
Adverse reaction includes temperature changes(℃), the change of blood pressure(mmHg) and allergic reaction.
Measured: Post cell transplantation: 1, 3, 6, 9months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Feb 2017
- Study startJan 2017
- Primary completionDec 2018 (estimated)
- Study completionDec 2019 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Aplastic AnemiaStudies 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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Hebei Medical University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Quanhai Li, Hebei Medical University
- Investigators
- Baoyong Yan, Doctor, Study chair, The First Hospital of Hebei Medical UniversityQingchi Liu, Doctor, Study director, The First Hospital of Hebei Medical UniversityQuanhai Li, Doctor, Study director, The First Hospital of Hebei Medical UniversityXiaohui Jia, Doctor, Principal investigator, The First Hospital of Hebei Medical UniversityXianyun Wang, Doctor, Principal investigator, The First Hospital of Hebei Medical UniversityFan Zhang, Bachelor, Principal investigator, The First Hospital of Hebei Medical UniversityYang Shen, Master, Principal investigator, The First Hospital of Hebei Medical UniversityBing Ma, Master, Principal investigator, The First Hospital of Hebei Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03055078
- Sponsor's protocol ID
- 17277787D-AA
- Status last verified
- Apr 2017
- First submitted
- 4 Feb 2017
- Last updated
- 7 Apr 2017
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Apr 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of Safety and Efficacy in Patients With Aplastic Anemia by Transplantation of Umbilical Cord Derived Mesenchymal Stem Cells
- ClinicalTrials.gov ID
- NCT03055078
- Acronym / sponsor ID
- 17277787D-AA
- Conditions
- Aplastic Anemia
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Hebei Medical UniversityUniversity, hospital or other
- Study start
- Jan 2017
- Primary completion
- Dec 2018 (estimated)
- Study completion
- Dec 2019 (estimated)
- First posted
- 16 Feb 2017 (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.
This study is to evaluate the safety and efficacy of Umbilical Cord Derived Mesenchymal Stem Cells transplantation in aplastic anemia.
Detailed description
Aplastic anemia (AA) is a disorder thought to be caused by an immune-mediated bone marrow failure. Not all people with AA are eligible for today's standard treatments. One new treatment approach uses umbilical cord derived mesenchymal stem cells-specialized cells capable of developing into other types of cells-to provide the basis for clinical application. The aim of the present study is to investigate the safety and efficacy of vein infusion of allogeneic mesenchymal stem cells in patients with AA .
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| mesenchymal stem cells | Experimental | Biological: mesenchymal stem cellsAccording to the inclusion and exclusion criteria, selected patients were divided into a cell therapy group and a control group. Umbilical cord derived mesenchymal stem cells at a dose of 100-300 million by intravenous infusion. |
Who may qualify
- Ages
- 14 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (8 listed)
- Diagnosis of AA according to established criteria in 2010
- Age from 14 to 60 years
- Suffering from AA within six months
- No serious infection or acute hemorrhage.
- Left ventricular ejection fraction (LVEF) ≥ 50%
Key exclusion criteria (8 listed)
- Patients with AA have to be disqualified from this study if any of the following is applicable.
- Severe aplastic anemia(SAA) with severe infection.
- Severe aplastic anemia(SAA) with active hemorrhage.
- Severe heart attack, liver and kidney disease following serious complications
- Patients with allergic constitution.
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 |
|---|---|---|
| mesenchymal stem cellsPatients in the cell treated group were given umbilical cord derived mesenchymal stem cells by intravenous infusion besides conventional therapy. There was only conventional treatment in the control group. All patients after treatment for 1months, 3months, 6months and 9months were evaluated respectively the therapeutic effect. | Biological | mesenchymal stem cells |
Primary outcome
Hemoglobin levels in peripheral blood
Time frame: Post cell transplantation: 9months
The recovery of bone marrow erythrocyte is evaluated by hemoglobin levels (g/L) in peripheral blood.
5 secondary outcomes
The number of red blood cell in peripheral blood
Time frame: Post cell transplantation: 1, 3, 6, 9months
The number of granulocyte in peripheral blood
Time frame: Post cell transplantation: 1, 3, 6, 9months
The number of white blood cell in peripheral blood
Time frame: Post cell transplantation: 1, 3, 6, 9months
The evaluation of bone marrow megakaryocytic series
Time frame: Post cell transplantation: 1, 3, 6, 9months
adverse reaction
Time frame: Post cell transplantation: 1, 3, 6, 9months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Apr 2017.
- 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.
Baoyong Yan, Doctor study chair
The First Hospital of Hebei Medical University
Qingchi Liu, Doctor study director
The First Hospital of Hebei Medical University
Quanhai Li, Doctor study director
The First Hospital of Hebei Medical University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing mesenchymal stem cells in people with Aplastic Anemia, run by Hebei Medical University at 1 site 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 8 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 7 Apr 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 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.
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 Jun Zhang, Master (86-311-85917384, june_luck@126.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Hemoglobin levels in peripheral blood (time frame: Post cell transplantation: 9months). 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 7 Apr 2017.