Hepatitis-associated Aplastic AnemiaNCT07850362
Clinical Study of UC-MSC Combined With Immunosuppressive Agents in the Treatment of Pediatric HAAA
A study testing UC-MSC in people with Hepatitis-associated Aplastic Anemia and Severe Aplastic Anemia(SAA), run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
Trial summary
The goal of this clinical trial is to evaluate the effectiveness of umbilical cord mesenchymal stem cells combined with standard immunosuppressive therapy in children with HAAA, and to provide a new therapeutic idea for children with HAAA. Read more about this study
- Who is it for?
- Ages 1 year to 18 years
- Conditions the study is for
- Hepatitis-associated Aplastic Anemia, Severe Aplastic Anemia(SAA), Umbilical Cord Blood
- What is being tested?
- UC-MSC
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteChina
- Study dates
- 1 Sep 2021to 31 Oct 2025
- Phase
- Interventional
- 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 1 year to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 1 Age < 18 years old, gender is not limited; 2 Diagnosis of hepatitis related aplastic anemia according to the criteria for hepatitis related aplastic anemia;
- The patient has a history of acute prehepatitis, and the course of hepatitis is generally less than 6 months. The main clinical manifestations are fever, jaundice, anemia, bleeding, generally no liver enlargement.
- Routine blood test: pancytopenia, HGB < 100 g/L; Absolute neutrophil count < 1.5×109/L; PLT < 100×109/L (PLT reduction must be included in case of two-series reduction). Serum aminotransferase levels were elevated at least 200 U/L and hepatitis autoantibodies were negative.
- Bone marrow aspiration and biopsy confirmed aplastic anemia.
- Jaundice caused by other reasons and other pancytopenia diseases (such as congenital AA, clonal diseases and autoimmune diseases, etc.) are excluded.
- 3 No hematopoietic stem cell transplantation (HSCT); 4 The guardian and the subject will participate voluntarily and sign the informed consent;
Who cannot join
- 1 Patients will not be enrolled in this study if they meet any of the following conditions: 2. Patients with a history of hematopoietic stem cell transplantation; HIV infection or carrier in the past or at the time of screening; Positive for syphilis; 4. The subject had developed malignant solid tumors of any organ system within 5 years prior to screening; 5. Participated in other clinical trials within 3 months;
Trial locations and contacts
1 site in 1 country.
China1 site
Institute of Hematology & Blood Diseases Hospital
Tianjin, Tianjin Municipality, 300020
About this study
Official title: Clinical Study of Umbilical Cord Mesenchymal Stem Cells Combined With Immunosuppressive Agents in the Treatment of Children With Hepatitis Associated Aplastic Anemia
The goal of this clinical trial is to evaluate the effectiveness of umbilical cord mesenchymal stem cells combined with standard immunosuppressive therapy in children with HAAA, and to provide a new therapeutic idea for children with HAAA.
Whether UC-MSC combined with immunosuppressants can improve outcomes in children with HAAA.
Read more
Whether UC-MSC is safe in the treatment of children with HAAA.
Participants will:
1 Age < 18 years old, gender is not limited; 2 Diagnosis of hepatitis related aplastic anemia according to the criteria for hepatitis related aplastic anemia;
- The patient has a history of acute prehepatitis, and the course of hepatitis is generally less than 6 months. The main clinical manifestations are fever, jaundice, anemia, bleeding, generally no liver enlargement.
- Routine blood test: pancytopenia, HGB < 100 g/L; Absolute neutrophil count < 1.5×109/L; PLT < 100×109/L (PLT reduction must be included in case of two-series reduction). Serum aminotransferase levels were elevated at least 200 U/L and hepatitis autoantibodies were negative.
- Bone marrow aspiration and biopsy confirmed aplastic anemia.
- Jaundice caused by other reasons and other pancytopenia diseases (such as congenital AA, clonal diseases and autoimmune diseases, etc.) are excluded.
3 No hematopoietic stem cell transplantation (HSCT); 4 The guardian and the subject will participate voluntarily and sign the informed consent;
- Conditions
- Hepatitis-associated Aplastic Anemia, Severe Aplastic Anemia(SAA), Umbilical Cord Blood, Mesenchymal Stem Cells, Pediatric
- Being tested
- UC-MSC
- Medical subject headings
- Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
UC-MSCOther
UC-MSC Combined With Immunosuppressive Agents
| Study group | Role | What they receive |
|---|---|---|
| UC-MSC + IST group | Experimental | UC-MSC Combined With Immunosuppressive Agents in the Treatment of Pediatric HAAA Receives: Other: UC-MSC |
What is being measured
The primary outcome is the main question the study is designed to answer.
Complete response rate at six months
Complete response rate at six months
Measured: Up to 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Sep 2026
- Study start1 Sep 2021
- Primary completion30 Aug 2025
- Study completion31 Oct 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Hepatitis-associated Aplastic AnemiaStudies of the same condition, open and under way.
- More trials run by Institute of Hematology & Blood Diseases Hospital, ChinaThe sponsor's other registered studies.
- 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 N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- lixian chang, MD, Principal investigator, State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, CAMS & PUMC
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07850362
- Sponsor's protocol ID
- IIT2020027
- Status last verified
- Sep 2021
- First submitted
- 13 Aug 2025
- Last updated
- 30 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: After the research is completed, it is shared in the form of published articles
Publications
Fang L, Meikuang L, Ye G, Xiaojuan C, Wenyu Y, Min R, Lixian C, Weiqiang W, Zhibo H, Zhongchao H, Xiaofan Z. Successful Treatment of a 19-Month-Old Boy with Hepatitis Associated Aplastic Anemia by Infusion of Umbilical Cord-Derived Mesenchymal Stromal Cells: A Case Report. Cell Transplant. 2021 Jan-Dec;30:963689720977144. doi: 10.1177/0963689720977144.
PubMed 33525921
Study at a glance
From the registry record, last updated by the sponsor on 30 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Study of Umbilical Cord Mesenchymal Stem Cells Combined With Immunosuppressive Agents in the Treatment of Children With Hepatitis Associated Aplastic Anemia
- ClinicalTrials.gov ID
- NCT07850362
- Acronym / sponsor ID
- IIT2020027
- Conditions
- Hepatitis-associated Aplastic Anemia, Severe Aplastic Anemia(SAA), Umbilical Cord Blood, Mesenchymal Stem Cells, Pediatric
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, ChinaUniversity, hospital or other
- Study start
- 1 Sep 2021 (actual)
- Primary completion
- 30 Aug 2025 (actual)
- Study completion
- 31 Oct 2025 (actual)
- First posted
- 30 Sep 2026 (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 goal of this clinical trial is to evaluate the effectiveness of umbilical cord mesenchymal stem cells combined with standard immunosuppressive therapy in children with HAAA, and to provide a new therapeutic idea for children with HAAA.
Whether UC-MSC combined with immunosuppressants can improve outcomes in children with HAAA.
Whether UC-MSC is safe in the treatment of children with HAAA.
Participants will:
1 Age < 18 years old, gender is not limited; 2 Diagnosis of hepatitis related aplastic anemia according to the criteria for hepatitis related aplastic anemia;
- The patient has a history of acute prehepatitis, and the course of hepatitis is generally less than 6 months. The main clinical manifestations are fever, jaundice, anemia, bleeding, generally no liver enlargement.
- Routine blood test: pancytopenia, HGB < 100 g/L; Absolute neutrophil count < 1.5×109/L; PLT < 100×109/L (PLT reduction must be included in case of two-series reduction). Serum aminotransferase levels were elevated at least 200 U/L and hepatitis autoantibodies were negative.
- Bone marrow aspiration and biopsy confirmed aplastic anemia.
- Jaundice caused by other reasons and other pancytopenia diseases (such as congenital AA, clonal diseases and autoimmune diseases, etc.) are excluded.
3 No hematopoietic stem cell transplantation (HSCT); 4 The guardian and the subject will participate voluntarily and sign the informed consent;
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| UC-MSC + IST group | Experimental | Other: UC-MSCUC-MSC Combined With Immunosuppressive Agents in the Treatment of Pediatric HAAA |
Who may qualify
- Ages
- 1 year to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (6 listed)
- 1 Age < 18 years old, gender is not limited; 2 Diagnosis of hepatitis related aplastic anemia according to the criteria for hepatitis related aplastic anemia;
- The patient has a history of acute prehepatitis, and the course of hepatitis is generally less than 6 months. The main clinical manifestations are fever, jaundice, anemia, bleeding, generally no liver enlargement.
- Routine blood test: pancytopenia, HGB < 100 g/L; Absolute neutrophil count < 1.5×109/L; PLT < 100×109/L (PLT reduction must be included in case of two-series reduction). Serum aminotransferase levels were elevated at least 200 U/L and hepatitis autoantibodies were negative.
- Bone marrow aspiration and biopsy confirmed aplastic anemia.
- Jaundice caused by other reasons and other pancytopenia diseases (such as congenital AA, clonal diseases and autoimmune diseases, etc.) are excluded.
Key exclusion criteria (1 listed)
- 1 Patients will not be enrolled in this study if they meet any of the following conditions: 2. Patients with a history of hematopoietic stem cell transplantation; HIV infection or carrier in the past or at the time of screening; Positive for syphilis; 4. The subject had developed malignant solid tumors of any organ system within 5 years prior to screening; 5. Participated in other clinical trials within 3 months;
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 |
|---|---|---|
| UC-MSCUC-MSC Combined With Immunosuppressive Agents | Other | UC-MSC + IST group |
Primary outcome
Complete response rate at six months
Time frame: Up to 6 months
Complete response rate at six months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Sep 2026.
- Background: PubMed 33525921Fang L, Meikuang L, Ye G, Xiaojuan C, Wenyu Y, Min R, Lixian C, Weiqiang W, Zhibo H, Zhongchao H, Xiaofan Z. Successful Treatment of a 19-Month-Old Boy with Hepatitis Associated Aplastic Anemia by Infusion of Umbilical Cord-Derived Mesenchymal Stromal Cells: A Case Report. Cell Transplant. 2021 Jan-Dec;30:963689720977144. doi: 10.1177/0963689720977144.
- Individual participant dataSharing planned: No. After the research is completed, it is shared in the form of published articles
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.
lixian chang, MD principal investigator
State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, CAMS & PUMC
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing UC-MSC in people with Hepatitis-associated Aplastic Anemia and Severe Aplastic Anemia(SAA), run by Institute of Hematology & Blood Diseases Hospital, China 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 6 inclusion and 1 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: finished, as of the sponsor's last update on 30 Sep 2026. 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.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Complete response rate at six months (time frame: Up to 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT07850362, which also lists 1 linked publication.
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 30 Sep 2026.