Sever Aplastic AneiaPhase 2SAA MSCNCT07903649

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 study testing Umbilical Cord Mesenchymal Stem Cells with ATG/ALG and ATG/ALG in people with Sever Aplastic Aneia and Children, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

Search all trials

Trial 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 more about this study

Who is it for?: Ages 1 year to 16 years
Conditions the study is for: Sever Aplastic Aneia, Children, Umbilical Cord Mesenchymal Stem Cells
What is being tested?: Umbilical Cord Mesenchymal Stem Cells with ATG/ALG and ATG/ALG
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 64 participants
Where: 1 siteChina
Study dates: 25 Feb 2021to 20 Apr 2026
Phase: Phase 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 1 year to 16 years
All sexes
No healthy volunteers
Child

Who may be able to join

Age ≤ 16 years old>1 year old, gender not limited;
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; Platelet 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 (HSCT) directly; 4. The guardians voluntarily allow the ward to participate and sign an informed consent form;

Who cannot join

Individuals with a history of hematopoietic stem cell transplantation;
Patients with uncontrolled bleeding and/or infection after standardized treatment prior to enrollment;
Previously or screened as HIV infected or carrier, or positive for hepatitis C antibodies;
History of liver cirrhosis or portal hypertension in the past;
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;
Participated in other clinical trials within the first 3 months of randomization;
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.
Patients with congenital aplastic anemia -

Trial locations and contacts

1 site in 1 country.

China1 site

State key laboratory of experimental hematology,National clinical Research center for blood diseases
Tianjin, Tianjin Municipality, 300020

Other trials in China

About this study

Official title: Clinical Study of Umbilical Cord Mesenchymal Stem Cells Combined With Immunosuppressive Agents in the Treatment of Children With Sever Aplastic Anemia

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

Conditions: Sever Aplastic Aneia, Children, Umbilical Cord Mesenchymal Stem Cells
Being tested: Umbilical Cord Mesenchymal Stem Cells with ATG/ALG, ATG/ALG

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Umbilical Cord Mesenchymal Stem Cells with ATG/ALGOther
Evaluation of the effectiveness of umbilical cord mesenchymal stem cell combined with standard immunosuppressive therapy in children with acquired severe aplastic anemia
ATG/ALGDrug
standard immunosuppressants for the treatment of severe acquired aplastic anemia in children anemia

Study groups in NCT07903649
Study group	Role	What they receive
Group A	Experimental	Standard immunosuppressive （ATG/ALG）therapy for acquired severe aplastic anemia in children Receives: Drug: ATG/ALG
Group B	Experimental	Umbilical cord mesenchymal stem cells combined with standard immunosuppressants （UC-MSC+ATG/ALG） for the treatment of severe acquired aplastic anemia in children anemia Receives: Other: Umbilical Cord Mesenchymal Stem Cells with ATG/ALG

What is being measured

The primary outcome is the main question the study is designed to answer.

Complete response rate (CR) at 6 months of administration
Complete response rate (CR) at 6 months of administration
Measured: 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
Measured: From the start of treatment to the cessation of treatment for 3 and 6 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

25 Feb 202120 Apr 2026

First posted29 Oct 2026
Study start25 Feb 2021
Primary completion20 Apr 2026
Study completion20 Apr 2026

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 Sever Aplastic AneiaStudies 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 2
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: None (open label)
Enrolment: 64 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT07903649
Sponsor's protocol ID: SC2019003
Acronym: SAA MSC
Status last verified: Feb 2021
First submitted: 23 Oct 2026
Last updated: 29 Oct 2026
Results posted: No
Data monitoring committee: No
FDA-regulated drug: No
FDA-regulated device: No
Individual data sharing: No

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

Study at a glance

From the registry record, last updated by the sponsor on 29 Oct 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 Sever Aplastic Anemia
ClinicalTrials.gov ID: NCT07903649
Acronym / sponsor ID: SAA MSC / SC2019003
Conditions: Sever Aplastic Aneia, Children, Umbilical Cord Mesenchymal Stem Cells
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 64 participants (actual)
Lead sponsor: Institute of Hematology & Blood Diseases Hospital, ChinaUniversity, hospital or other
Study start: 25 Feb 2021 (actual)
Primary completion: 20 Apr 2026 (actual)
Study completion: 20 Apr 2026 (actual)
First posted: 29 Oct 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.

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

Detailed description

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: None (open label)
Number of arms: 2

Arm	Type	Receives
Group A	Experimental	Drug: ATG/ALGStandard immunosuppressive （ATG/ALG）therapy for acquired severe aplastic anemia in children
Group B	Experimental	Other: Umbilical Cord Mesenchymal Stem Cells with ATG/ALGUmbilical cord mesenchymal stem cells combined with standard immunosuppressants （UC-MSC+ATG/ALG） for the treatment of severe acquired aplastic anemia in children anemia

Who may qualify

Ages: 1 year to 16 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child

Key inclusion criteria (4 listed)

Age ≤ 16 years old>1 year old, gender not limited;
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%;
The absolute value of neutrophils is less than 0.5 × 109/L; Platelet 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 (HSCT) directly; 4. The guardians voluntarily allow the ward to participate and sign an informed consent form;

Key exclusion criteria (8 listed)

Individuals with a history of hematopoietic stem cell transplantation;
Patients with uncontrolled bleeding and/or infection after standardized treatment prior to enrollment;
Previously or screened as HIV infected or carrier, or positive for hepatitis C antibodies;
History of liver cirrhosis or portal hypertension in the past;
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;

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
Umbilical Cord Mesenchymal Stem Cells with ATG/ALGEvaluation of the effectiveness of umbilical cord mesenchymal stem cell combined with standard immunosuppressive therapy in children with acquired severe aplastic anemia	Other	Group B
ATG/ALGstandard immunosuppressants for the treatment of severe acquired aplastic anemia in children anemia	Drug	Group A

Primary outcome

Complete response rate (CR) at 6 months of administration
Time frame: From the start of treatment to the cessation of treatment for 6 months
Complete response rate (CR) at 6 months of administration

1 secondary outcome

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

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 29 Oct 2026.
Background: PubMed 25583265Zhang 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.
Individual participant dataSharing planned: No

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.

The record lists no contact. Use the official record to reach the sponsor.

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

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 4 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: finished, as of the sponsor's last update on 29 Oct 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.

What does this phase mean?

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?

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 (CR) at 6 months of administration (time frame: From the start of treatment to the cessation of treatment for 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 NCT07903649, 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 29 Oct 2026.