Complications of Organ Transplant Stem Cells Umbilical CordPhase 4MSC-SAANCT02218437
Treatment Protocol of Child SAA With the Injection of Mesenchymal Stem Cells（Umbilical Cord Derived）
A Phase 4 study testing MSC+ATG in people with Complications of Organ Transplant Stem Cells Umbilical Cord, run by Chinese Academy of Medical Sciences at 1 site in China.
Trial summary
Mesenchymal Stem Cells (MSC) is a non Hematopoietic Stem Cells (HSC) in adult bone marrow and takes part in the bone marrow microenvironment. The response rate of early treatment on Children's SAA application combined with anti-thymocyte globulin (ATG) (40-50 days after ATG treatment) is associated with long-term… Read more about this study
- Who is it for?
- Ages 1 month to 18 yearsHealthy volunteers can take part
- Conditions the study is for
- Complications of Organ Transplant Stem Cells Umbilical Cord
- What is being tested?
- MSC+ATG
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteChina
- Study dates
- Oct 2013to Oct 2015
- Phase
- Phase 4
- 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 month to 18 years
- All sexes
- Healthy volunteers accepted
- Child, Adult
Who may be able to join
- Non-Severe Aplastic Anemia (NSAA)atients
Who cannot join
- Severe Aplastic Anemia (SAA) patients
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Institute of Hematology and Blood Diseases Hospital Chinese Academy of Medical Sciences and Peking Union Medical College
Tianjin, 300020
Xiaofan Zhu, MD, +86-22-23909001, zhuxf6465@gmail.com
Xiaofan Zhu, MD
About this study
Official title: Child With Severe Aplastic Anemia (SAA) Therapy: the Injection of Umbilical Cord Derived Mesenchymal Stem Cells.
Mesenchymal Stem Cells (MSC) is a non Hematopoietic Stem Cells (HSC) in adult bone marrow and takes part in the bone marrow microenvironment. The response rate of early treatment on Children's SAA application combined with anti-thymocyte globulin (ATG) (40-50 days after ATG treatment) is associated with long-term effect. The injection of Umbilical Cord Derived Mesenchymal Stem Cells combined with ATG improves the efficacy of children with SAA.
Read the detailed description
- Mesenchymal Stem Cells were prepared by in vitro separation, screening, and culture from healthy human umbilical cord tissue; The " injection of mesenchymal stem cells (umbilical) manufacturing and verification regulation " was also formulated.
- The starting dose of Umbilical Cord Derived MSC was 0.5-1.0 * 106 cells /kg, based on the previous human studies; And the maximum tolerated dose was 1 * 107 cells /kg
- The response and complete remission rate, relapse rate of the injection of Umbilical Cord Derived Mesenchymal Stem Cells (or combined with ATG ) for Child with SAA were determined.
- Being tested
- MSC+ATG
- Keywords
- Mesenchymal Stem Cells (MSC), Umbilical Cord, thymocyte globulin (ATG), Severe Aplastic Anemia (SAA)
- Medical subject headings
- Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MSC+ATGDrug
Also called Mesenchymal Stem Cells (Umbilical Cord Derived)
MSC+ATG:Starting dose was 0.5-1.0 × 106 cells /kg; And the maximum tolerated dose was 1 ×107 cells /kg after ATG application.
| Study group | Role | What they receive |
|---|---|---|
| MSC+ATG | Experimental | The first agent MSC injection, began two weeks after ATG application; Each patient was injected three times, one time per week; Study on single dose tolerance and efficacy index; Each subjects received three dose groups of treatment. Receives: Drug: MSC+ATG |
What is being measured
The primary outcome is the main question the study is designed to answer.
The response and complete remission rate with different doses of Umbilical Cord Derived MSC to treat child with SAA
Complete response (CR) was defined as achieving normal levels of hemoglobin adjusted for age, platelet count >100*109/L, and absolute neutrophil count (ANC) >1.5*1.0 9/L. Partial response (PR) was defined as injection independence, reticulocyte count>30*109/L, platelet count >20*109/L, and ANC>0.5*1.0 9/L above the baseline. Persistence of injection requirement or death was evidence of no response (NR).
Measured: 1 year
Secondary outcomes (1)
The relapse rate with different doses of MSC to treat child with SAA
The relapse was defined as injection dependence again; or progressed or paroxysmal nocturnal hemoglobinuria (PNH)/acute myeloid leukemia/myelodysplasia syndrome(MDS); or cyclosporin A (CsA) dependence.
Measured: 3-10 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Aug 2014 (estimated)
- Study startOct 2013
- Primary completionOct 2015 (estimated)
- Study completionOct 2015 (estimated)
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 Complications of Organ Transplant Stem Cells Umbilical CordStudies 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 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Chinese Academy of Medical Sciences
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Xiaofan Zhu, Chinese Academy of Medical Sciences
- Investigators
- Xiaofan Zhu, MD, Principal investigator, Chinese Academy of Medical Sciences
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02218437
- Sponsor's protocol ID
- MSC-2014-SAA-1
- Other IDs
- H0806-MSC-2014
- Acronym
- MSC-SAA
- Status last verified
- Aug 2014
- First submitted
- 6 Aug 2014
- Last updated
- 18 Aug 2014
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Lan Y, Liu F, Chang L, Liu L, Zhang Y, Yi M, Cai Y, Feng J, Han Z, Han Z, Zhu X. Combination of umbilical cord mesenchymal stem cells and standard immunosuppressive regimen for pediatric patients with severe aplastic anemia. BMC Pediatr. 2021 Feb 27;21(1):102. doi: 10.1186/s12887-021-02562-x.
PubMed 33639900
Study at a glance
From the registry record, last updated by the sponsor on 18 Aug 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Child With Severe Aplastic Anemia (SAA) Therapy: the Injection of Umbilical Cord Derived Mesenchymal Stem Cells.
- ClinicalTrials.gov ID
- NCT02218437
- Acronym / sponsor ID
- MSC-SAA / MSC-2014-SAA-1
- Keywords
- Mesenchymal Stem Cells (MSC), Umbilical Cord, thymocyte globulin (ATG), Severe Aplastic Anemia (SAA)
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Chinese Academy of Medical SciencesUniversity, hospital or other
- Study start
- Oct 2013
- Primary completion
- Oct 2015 (estimated)
- Study completion
- Oct 2015 (estimated)
- First posted
- 18 Aug 2014 (estimated)
- 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.
Mesenchymal Stem Cells (MSC) is a non Hematopoietic Stem Cells (HSC) in adult bone marrow and takes part in the bone marrow microenvironment. The response rate of early treatment on Children's SAA application combined with anti-thymocyte globulin (ATG) (40-50 days after ATG treatment) is associated with long-term effect. The injection of Umbilical Cord Derived Mesenchymal Stem Cells combined with ATG improves the efficacy of children with SAA.
Detailed description
- Mesenchymal Stem Cells were prepared by in vitro separation, screening, and culture from healthy human umbilical cord tissue; The " injection of mesenchymal stem cells (umbilical) manufacturing and verification regulation " was also formulated.
- The starting dose of Umbilical Cord Derived MSC was 0.5-1.0 * 106 cells /kg, based on the previous human studies; And the maximum tolerated dose was 1 * 107 cells /kg
- The response and complete remission rate, relapse rate of the injection of Umbilical Cord Derived Mesenchymal Stem Cells (or combined with ATG ) for Child with SAA were determined.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| MSC+ATG | Experimental | Drug: MSC+ATGThe first agent MSC injection, began two weeks after ATG application; Each patient was injected three times, one time per week; Study on single dose tolerance and efficacy index; Each subjects received three dose groups of treatment. |
Who may qualify
- Ages
- 1 month to 18 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult
Key inclusion criteria (1 listed)
- Non-Severe Aplastic Anemia (NSAA)atients
Key exclusion criteria (1 listed)
- Severe Aplastic Anemia (SAA) patients
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 |
|---|---|---|
| MSC+ATGAlso: Mesenchymal Stem Cells (Umbilical Cord Derived)MSC+ATG:Starting dose was 0.5-1.0 × 106 cells /kg; And the maximum tolerated dose was 1 ×107 cells /kg after ATG application. | Drug | MSC+ATG |
Primary outcome
The response and complete remission rate with different doses of Umbilical Cord Derived MSC to treat child with SAA
Time frame: 1 year
Complete response (CR) was defined as achieving normal levels of hemoglobin adjusted for age, platelet count >100*109/L, and absolute neutrophil count (ANC) >1.5*1.0 9/L. Partial response (PR) was defined as injection independence, reticulocyte count>30*109/L, platelet count >20*109/L, and ANC>0.5*1.0 9/L above the baseline. Persistence of injection requirement or death was evidence of no response (NR).
1 secondary outcome
The relapse rate with different doses of MSC to treat child with SAA
Time frame: 3-10 year
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Aug 2014.
- Derived from this study: PubMed 33639900Lan Y, Liu F, Chang L, Liu L, Zhang Y, Yi M, Cai Y, Feng J, Han Z, Han Z, Zhu X. Combination of umbilical cord mesenchymal stem cells and standard immunosuppressive regimen for pediatric patients with severe aplastic anemia. BMC Pediatr. 2021 Feb 27;21(1):102. doi: 10.1186/s12887-021-02562-x.
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.
Xiaofan Zhu, MD principal investigator
Chinese Academy of Medical Sciences
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing MSC+ATG in people with Complications of Organ Transplant Stem Cells Umbilical Cord, run by Chinese Academy of Medical Sciences 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 1 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: status not confirmed, as of the sponsor's last update on 18 Aug 2014. 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
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 Xiaofan Zhu, MD (+86-22-23909001, zhuxf6465@gmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The response and complete remission rate with different doses of Umbilical Cord Derived MSC to treat child with SAA (time frame: 1 year). 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 NCT02218437, 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 18 Aug 2014.