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
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.
- 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
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, China
- 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