ClinicalTrials.gg
CompletedNCT07903649SAA MSCUpdated Oct 29, 2026

A Randomized, Controlled Clinical Study on the Efficacy and Safety of Umbilical Cord Mesenchymal Stem Cells Combined With Standard Immunosuppressants in the Treatment of Children With Severe Aplastic Anemia

A Phase 2 interventional study of Umbilical Cord Mesenchymal Stem Cells with ATG/ALG and ATG/ALG in Sever Aplastic Aneia, Children and Umbilical Cord Mesenchymal Stem Cells, sponsored by Institute of Hematology & Blood Diseases Hospital, China. Completed at 1 site in China. Open to participants aged 1 Year to 16 Years. Per ClinicalTrials.gov, last updated 2026-10-29.

Sponsored by Institute of Hematology & Blood Diseases Hospital, China · Phase 2, Interventional, and Treatment

From the registry’s dates

  • Registered 5 years 7 months after the study started (first participant enrolled Feb 2021, registered Oct 2026).
Updated Oct 29, 2026Newly registeredGo to Updates ↓

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Phase
Phase 2
Study type
Interventional
Enrollment
64
Allocation
Randomized
Ages
1 Year to 16 Years
Sex
All
01

Study summary

Study on the efficacy and safety of umbilical cord mesenchymal stem cells combined with standard immunosuppressive agents in the treatment of children with acquired severe aplastic anemia

Read the detailed description

A randomized controlled clinical study on the efficacy and safety of umbilical cord mesenchymal stem cells combined with standard immunosuppressive agents in the treatment of children with acquired severe aplastic anemia

02

Conditions studied

  • Sever Aplastic Aneia
  • Children
  • Umbilical Cord Mesenchymal Stem Cells
03

In context

Lead sponsor

Institute of Hematology & Blood Diseases Hospital, China is the lead sponsor of 413 studies on the registry; 292 are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
1 Year to 16 Years
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

  1. Age ≤ 16 years old>1 year old, gender not limited;
  2. Diagnosed as severe aplastic anemia according to the Camitta criteria; The degree of proliferation of bone marrow cells is less than 25% of normal; If ≥ 25% of normal but\<50%, the remaining hematopoietic cells should be\<30%;

Blood count should be specific to two of the following three items:

The absolute value of neutrophils is less than 0.5 × 109/L; count\<20 × 109/L; The absolute value of reticulocytes is less than 20 × 109/L. 3. Not suitable or willing to undergo hematopoietic stem cell transplantation () directly; 4. The guardians voluntarily allow the ward to participate and sign an form;

Exclusion criteria

Exclusion Criteria:

  1. Individuals with a history of hematopoietic stem cell transplantation;
  2. Patients with uncontrolled bleeding and/or infection after standardized treatment prior to enrollment;
  3. Previously or screened as infected or carrier, or positive for antibodies;
  4. History of liver cirrhosis or portal hypertension in the past;
  5. Screening for subjects who have developed any malignant solid tumors of any organ system within the past 5 years, regardless of whether or not Already treated, metastasized or recurrent; Subjects with blood tumors found in the past or during screening;
  6. Participated in other clinical trials within the first 3 months of randomization;
  7. The researcher believes that there is any possibility that the subject may not be able to complete the study or be given any guidance Other situations with obvious risks.
  8. Patients with congenital aplastic anemia -
Terms in these criteria, in plain language(5)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Platelets

Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.

Allogeneic and autologous transplant

Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.

Informed consent

Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.

You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.

HIV

The human immunodeficiency virus, which weakens the immune system.

Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.

Hepatitis C tests

Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.

After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.

05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
None (open label)
Enrollment
64 participants (actual)

Study arms

  • Experimental
    Group A

    Standard immunosuppressive (ATG/ALG)therapy for acquired severe aplastic anemia in children

    Drug: ATG/ALG

  • Experimental
    Group B

    Umbilical cord mesenchymal stem cells combined with standard immunosuppressants (UC-MSC+ATG/ALG) for the treatment of severe acquired aplastic anemia in children anemia

    Other: Umbilical Cord Mesenchymal Stem Cells with ATG/ALG

Interventions

  • OtherUmbilical Cord Mesenchymal Stem Cells with ATG/ALG

    Evaluation of the effectiveness of umbilical cord mesenchymal stem cell combined with standard immunosuppressive therapy in children with acquired severe aplastic anemia

  • DrugATG/ALG

    Drug page: Antilymphocyte Serum

    standard immunosuppressants for the treatment of severe acquired aplastic anemia in children anemia

06

What researchers measure

Primary outcomes

  1. Complete response rate (CR) at 6 months of administration

    Complete response rate (CR) at 6 months of administration

    Time frame: From the start of treatment to the cessation of treatment for 6 months

Secondary outcomes

  1. Partial hematological response rate (PR) and total hematological response rate (OR) at 6 months of administration

    Partial hematological response rate (PR) and total hematological response rate (OR) at 6 months of administration

    Time frame: From the start of treatment to the cessation of treatment for 3 and 6 months

07

Study locations

1 site
  • State key laboratory of experimental hematology,National clinical Research center for blood diseases
    Tianjin, Tianjin Municipality 300020, China
08

References and documents

Publications

  • Zhang L, Jing L, Zhou K, Wang H, Peng G, Li Y, Li Y, Li J, Ye L, Shi L, Fan H, Zhao X, Wang J, Zhang F. Rabbit antithymocyte globulin as first-line therapy for severe aplastic anemia. Exp Hematol. 2015 Apr;43(4):286-94. doi: 10.1016/j.exphem.2014.12.002. Epub 2015 Jan 9. PubMed 25583265 ↗

Individual participant data

Plan to share: No

09

Timeline

1 registry update since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reached

Ran from Feb 2021 to primary completion in Apr 2026, 61 months.

How these figures are worked out, and which studies are left out of them.

Registered
Oct 23, 2026
First posted
Oct 29, 2026
StartActual
Feb 25, 2021
Primary completionActual
Apr 20, 2026
CompletionActual
Apr 20, 2026
Last update
Oct 29, 2026
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
Registered
First appeared on the registry. No changes since
Oct 29, 2026
Show all 1 update
  1. Oct 29, 2026
    First appeared on the registry

From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗

10

Registry details

Key details

Study ID
NCT07903649
Responsible party
Sponsor
First posted
Oct 29, 2026
Start date
Feb 25, 2021
Primary completion
Apr 20, 2026
Completion
Apr 20, 2026
Last update
Oct 29, 2026

Oversight

Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Feb 2021. You cannot join it, but the record below documents what was studied.

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Discussion

Questions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.

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Studies of the drug tested here: Antilymphocyte Serum

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