Severe Aplastic AnemiaPhase 1/2NCT02407470
Safety and Efficacy of Patient's Own AD-MSC and AD-HSC Transplantation in Patients With Severe Aplastic Anemia
A Phase 1/2 study testing Rabbit antithymoglobulin (ATG), Adipose derived mesenchymal stem cells ( AD-MSCs) and AD-MSC transdifferentiated HSCs (AD-HSCs) in people with Severe Aplastic Anemia, run by Navy General Hospital, Beijing at 1 site in China.
Trial summary
RATIONALE: It has been shown that about 30% of patients do not respond to immunosuppressive therapy or experience recurrence, and graft rejection and graft-versus-host-disease (GVHD) decrease event-free survival to 30% to 50% in the alternative donor (matched unrelated, partially matched family member)… Read more about this study
- Who is it for?
- Ages 14 to 70 years
- Conditions the study is for
- Severe Aplastic Anemia
- What is being tested?
- Rabbit antithymoglobulin (ATG), Adipose derived mesenchymal stem cells ( AD-MSCs) and AD-MSC transdifferentiated HSCs (AD-HSCs)
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 90 participants
- Where
- 1 siteChina
- Study dates
- Jan 2015to Jul 2017
- Phase
- Phase 1/2
- 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 14 to 70 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Male or female recipients must have histopathologically confirmed diagnosis of SAA-I without or with more than 6 months after less than one treatment with ATG. Diagnostic Criteria for Server Aplastic Anemia will be based on the definitions set forth by the international Aplastic Anemia Study Group.
- At least two of the following:
- Absolute neutrophil count ≤ 0.5 X 109/l, Platelet count ≤ 20 X 109 /l, Anemia with corrected reticulocyte count ≤ 1%, and Bone marrow cellularity ≤ 25%, or bone marrow cellularity ≤ 50% with fewer than 30% hematopoietic cell, Hepatic: alanine aminotransferase (ALT)/ aspartate aminotransferase (AST) no greater than 4 times normal, Bilirubin: no greater than 2 mg/dl, Renal: Creatinine clearance at least 50 ml/min, Cardiovascular: Shortening fraction or ejection fraction at least 40% of normal for age by echocardiogram or radionuclide scan.
- No clinically significant comorbid illnesses (e.g., myocardial infarction or cerebrovascular accident).
Who cannot join
- Active and uncontrolled infection, Active bleeding, Severe allergic history of ATG, HIV-1 infection, Pregnancy or breastfeeding, Carbon monoxide lung diffusion capacity (DLCO) <40% predicted, SAA-II, Patients with severe psychological disorders, Recipients of other clinical trials.
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Navy General Hospital
Beijing, Beijing Municipality, 100048
Jianliang Shen, M.D.,Ph.D, 86-01-66957676, nghxyk@163.com
Qinwei Yin, M.D.,Ph.D., 86-01-84008003, jamesyin2010@126.com
About this study
Official title: A Multicenter, Randomized, Controlled Study of the Efficacy and Safety of the Combination of Adipose Tissue-derived Hematopoietic Stem Cells (AD-HSCs) and ATG in the Treatment of Severe Aplastic Anemia
RATIONALE: It has been shown that about 30% of patients do not respond to immunosuppressive therapy or experience recurrence, and graft rejection and graft-versus-host-disease (GVHD) decrease event-free survival to 30% to 50% in the alternative donor (matched unrelated, partially matched family member) transplantation. Although an overall and disease free survival of 85% to 100%, can be obtained in allogeneic blood or bone marrow stem cell transplantation using an human leukocyte antigen (HLA) matched sibling donor, only about 25% of patients have such a donor.
PURPOSE: In an attempt to avoid GVHD, reduce earlier infection rate and decrease regimen-related toxicity while maintaining better engraftment, this study is to evaluate the effectiveness and safety of patient's own adipose-derived mesenchymal stem cell (AD-MSC) or AD-MSC transdifferentiated HSC (AD-HSC) transplant after an immunosuppressive regimen in treating patients who have severe aplastic anemia.
Read more
The patient will be in the study for one year for observation and active monitoring. After treatment and active monitoring are over, the patient's medical condition will be followed indefinitely. The principle measures of safety and efficacy will be :
- Patient survival probability at 3 months, 6 months and 1 year.
- Engraftment at 3 months, 6 months and 1 year
- Incidence of graft versus host disease (GVHD), incidence of acute and chronic GVHD and Incidence of earlier infection rate as well as other complications within 6 months and 1 years.
Read the detailed description
Severe aplastic anemia is characterized by severe deficiencies in peripheral-blood platelets, white cells, and red cells. These defects in mature cells occur because aplastic bone marrow contains severely reduced numbers of hematopoietic stem cells. To date, Hematopoietic stem cell (HSC) transplants are routinely used to treat patients with many different diseases, including various cancers and blood disorders, such as aplastic anemia. The main sources of HSCs are bone marrow, cord blood and peripheral blood. However, challenges include obtaining enough functional HSCs to ensure optimal engraftment, and avoiding immune rejection and other complications associated with allogeneic transplantations. Novel abundant sources of clinical-grade HSCs are therefore being sought.
Our novel studies have demonstrated that adipose-derived mesenchymal stem cells (AD-MSCs) can be converted rapidly (in 4 days) into AD-HSCs on a large scale (2X108-9 cluster of differentiation 34（CD34）positive cells) by transfection of small RNAs to the the early region 1A (E1A)-like inhibitor of differentiation 1 (EID1) in the presence of specific cytokines. In vitro, AD-HSCs expanded efficiently and resembled cord-blood HSCs in phenotype, genotype, and colony-forming ability. In a mouse model, primary and secondary transplantation analysis and repopulating assays showed that AD-HSCs homed to the bone marrow, differentiated into functional blood cells, and showed a long-term ability to self-renew. we show that adipose-derived mesenchymal stem cells (AD-MSCs) can be converted into AD-HSCs by transfection of small RNAs to the E1A-like inhibitor of differentiation 1 (EID1) in the presence of specific cytokines. In vitro, AD-HSCs expanded efficiently and resembled cord-blood HSCs in phenotype, genotype, and colony-forming ability. In a mouse model, primary and secondary transplantation analysis and repopulating assays showed that AD-HSCs homed to the bone marrow, differentiated into functional blood cells, and showed a long-term ability to self-renew. In the safety aspect, we saw no evidence of leukemia, teratoma and other cancers in the blood, testes and subcutaneous tissues of transplanted mice. More importantly, our preliminary data have shown that AD-HSCs can reconstitute hematopoietic function in five patients with severe aplastic anemia. Based on these premilitary studies,, we have determined to conduct a further clinical investigation in multiple medical centers. In this study we plan to enroll up to 90 patients, to make a comprehensive assessment for this new treatment regimen and to show it is equal or superior to the current immunosuppressive regimen. Patients will be in the study for one years for treatment and active monitoring. All patients will be followed until death.
- Conditions
- Severe Aplastic Anemia
- Being tested
- Rabbit antithymoglobulin (ATG), Adipose derived mesenchymal stem cells ( AD-MSCs), AD-MSC transdifferentiated HSCs (AD-HSCs)
- Keywords
- Severe Aplastic Anemia (SAA), Hematopoietic Stem Cells, HSC Transplantation, ATG, Mesenchymal Stem Cells, Transdifferentiation,
- Medical subject headings
- Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Rabbit antithymoglobulin (ATG)Drug
Also called ATG
Rabbit ATG at 3.5 mg/kg/dose IV is given from day -6 to -2.
Adipose derived mesenchymal stem cells ( AD-MSCs)Procedure or surgery
Also called AD-MSCs
Participants will receive rabbit ATG at 3.5 mg/kg/dose IV from day -6 to -2, and then patient's own AD-MSCs at a dose of 3000000 cells/kg/d on day 1-3.
AD-MSC transdifferentiated HSCs (AD-HSCs)Procedure or surgery
Also called AD-HSCs
Participants will receive rabbit anti-thymocyte globulin at 3.5 mg/kg/dose IV from day -6 to -2, and then patient's own AD-HSCs at a dose of 3000000 cells/kg/d from day 1 to 4.
| Study group | Role | What they receive |
|---|---|---|
| Rabbit antithymoglobulin （ATG） | Active comparator | Patient in this arm will receive rabbit ATG at 3.5 mg/kg/dose IV from day -6 to -2 with the goals of ablating host repressive T cells. Receives: Drug: Rabbit antithymoglobulin (ATG) |
| Rabbit ATG & AD-MSCs | Experimental | Patient in this arm will receive rabbit ATG at 3.5 mg/kg/dose IV from day -6 to -2 and then patient's own adipose derived mesenchymal stem cells (AD-MSCs) at a dose of 3000000/kg/d on day 1 to 3. Receives: Drug: Rabbit antithymoglobulin (ATG); Procedure: Adipose derived mesenchymal stem cells ( AD-MSCs) |
| Rabbit ATG & AD-HSCs | Experimental | Patient in this arm will receive rabbit ATG at 3.5 mg/kg/dose IV from day -6 to -2 and then patient's own AD-MSC transdifferentiated HSCs (AD-HSCs) at a dose of 3000000/kg/d from day 1 to 4. Receives: Drug: Rabbit antithymoglobulin (ATG); Procedure: AD-MSC transdifferentiated HSCs (AD-HSCs) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Engraftment at 42 days post AD-HSC transplantation for patients with severe aplastic anemia.
Absolute neutrophil count > 0.5 X 109/l and Platelet count > 20 X 109 /l without infusion of platelet for 7 days.
Measured: 42 days posttransplant
Secondary outcomes (8)
To estimate the overall survival (OS) at 1 year following AD-HSC transplantation for Patients with Severe Aplastic Anemia
Number of Subjects Alive at 12 months Post Transplant
Measured: 1 year
Relapse
Return of SAA during the specified post-transplantation period.
Measured: 1 year post transplant
Incidence of chronic graft-versus-host disease
Number of patients with chronic graft-versus-host disease by 6 months and 1 year
Measured: 6 months
Evaluation of the occurrence of secondary malignancies
Occurring of any tumors during the specified post-transplantation period.
Measured: 6 months post transplant
Hematology labs
Association between AD-HSC transplantation and response in hemoglobin, platelet, total white blood cell count, and absolute neutrophil count to be evaluate by maximal hemoglobin, platelet, total white blood cell count, and absolute neutrophil counts achieved in patients with severe aplastic anemia
Measured: 12 weeks
Number of participants with adverse events as a measure of safety and tolerability of intravenous AD-HSC infusion in patients with severe aplastic anemia
Adverse events like allergic reactions, infectious diseases, organ dysfunction or other related to AD-HSC infusion will be assessed
Measured: weekly untill 12 months
Transfusional requirements
Units of blood or platelets transfused after AD-HSC infusion will be measured and compared to previously.
Measured: weekly untill 6 months
To assess treatment related mortality
Number of death after transplantation during the specified post-transplantation period.
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Apr 2015 (estimated)
- Study startJan 2015
- Primary completionJan 2017 (estimated)
- Study completionJul 2017 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Severe 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 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 90 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Navy General Hospital, Beijing
- Sponsor type
- University, hospital or other
- Collaborators
- Peking Union Medical College Hospital, General Hospital of Beijing PLA Military Region, Chinese Academy of Medical Sciences
- Responsible party
- Principal investigator, Qinwei Yin, General Hospital of Beijing PLA Military Region
- Investigators
- james Q Yin, M.D.,Ph.D., Principal investigator, The military general hospital of Beijing
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02407470
- Sponsor's protocol ID
- Ginkgocell-ADHSC-AA-001
- Status last verified
- Mar 2015
- First submitted
- 25 Mar 2015
- Last updated
- 3 Apr 2015
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 3 Apr 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Randomized, Controlled Study of the Efficacy and Safety of the Combination of Adipose Tissue-derived Hematopoietic Stem Cells (AD-HSCs) and ATG in the Treatment of Severe Aplastic Anemia
- ClinicalTrials.gov ID
- NCT02407470
- Acronym / sponsor ID
- Ginkgocell-ADHSC-AA-001
- Conditions
- Severe Aplastic Anemia
- Keywords
- Severe Aplastic Anemia (SAA), Hematopoietic Stem Cells, HSC Transplantation, ATG, Mesenchymal Stem Cells, Transdifferentiation,
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 90 participants (estimated)
- Lead sponsor
- Navy General Hospital, BeijingUniversity, hospital or other
- Collaborators
- Peking Union Medical College Hospital, General Hospital of Beijing PLA Military Region, Chinese Academy of Medical Sciences
- Study start
- Jan 2015
- Primary completion
- Jan 2017 (estimated)
- Study completion
- Jul 2017 (estimated)
- First posted
- 3 Apr 2015 (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.
RATIONALE: It has been shown that about 30% of patients do not respond to immunosuppressive therapy or experience recurrence, and graft rejection and graft-versus-host-disease (GVHD) decrease event-free survival to 30% to 50% in the alternative donor (matched unrelated, partially matched family member) transplantation. Although an overall and disease free survival of 85% to 100%, can be obtained in allogeneic blood or bone marrow stem cell transplantation using an human leukocyte antigen (HLA) matched sibling donor, only about 25% of patients have such a donor.
PURPOSE: In an attempt to avoid GVHD, reduce earlier infection rate and decrease regimen-related toxicity while maintaining better engraftment, this study is to evaluate the effectiveness and safety of patient's own adipose-derived mesenchymal stem cell (AD-MSC) or AD-MSC transdifferentiated HSC (AD-HSC) transplant after an immunosuppressive regimen in treating patients who have severe aplastic anemia.
The patient will be in the study for one year for observation and active monitoring. After treatment and active monitoring are over, the patient's medical condition will be followed indefinitely. The principle measures of safety and efficacy will be :
- Patient survival probability at 3 months, 6 months and 1 year.
- Engraftment at 3 months, 6 months and 1 year
- Incidence of graft versus host disease (GVHD), incidence of acute and chronic GVHD and Incidence of earlier infection rate as well as other complications within 6 months and 1 years.
Detailed description
Severe aplastic anemia is characterized by severe deficiencies in peripheral-blood platelets, white cells, and red cells. These defects in mature cells occur because aplastic bone marrow contains severely reduced numbers of hematopoietic stem cells. To date, Hematopoietic stem cell (HSC) transplants are routinely used to treat patients with many different diseases, including various cancers and blood disorders, such as aplastic anemia. The main sources of HSCs are bone marrow, cord blood and peripheral blood. However, challenges include obtaining enough functional HSCs to ensure optimal engraftment, and avoiding immune rejection and other complications associated with allogeneic transplantations. Novel abundant sources of clinical-grade HSCs are therefore being sought.
Our novel studies have demonstrated that adipose-derived mesenchymal stem cells (AD-MSCs) can be converted rapidly (in 4 days) into AD-HSCs on a large scale (2X108-9 cluster of differentiation 34（CD34）positive cells) by transfection of small RNAs to the the early region 1A (E1A)-like inhibitor of differentiation 1 (EID1) in the presence of specific cytokines. In vitro, AD-HSCs expanded efficiently and resembled cord-blood HSCs in phenotype, genotype, and colony-forming ability. In a mouse model, primary and secondary transplantation analysis and repopulating assays showed that AD-HSCs homed to the bone marrow, differentiated into functional blood cells, and showed a long-term ability to self-renew. we show that adipose-derived mesenchymal stem cells (AD-MSCs) can be converted into AD-HSCs by transfection of small RNAs to the E1A-like inhibitor of differentiation 1 (EID1) in the presence of specific cytokines. In vitro, AD-HSCs expanded efficiently and resembled cord-blood HSCs in phenotype, genotype, and colony-forming ability. In a mouse model, primary and secondary transplantation analysis and repopulating assays showed that AD-HSCs homed to the bone marrow, differentiated into functional blood cells, and showed a long-term ability to self-renew. In the safety aspect, we saw no evidence of leukemia, teratoma and other cancers in the blood, testes and subcutaneous tissues of transplanted mice. More importantly, our preliminary data have shown that AD-HSCs can reconstitute hematopoietic function in five patients with severe aplastic anemia. Based on these premilitary studies,, we have determined to conduct a further clinical investigation in multiple medical centers. In this study we plan to enroll up to 90 patients, to make a comprehensive assessment for this new treatment regimen and to show it is equal or superior to the current immunosuppressive regimen. Patients will be in the study for one years for treatment and active monitoring. All patients will be followed until death.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Rabbit antithymoglobulin （ATG） | Active comparator | Drug: Rabbit antithymoglobulin (ATG)Patient in this arm will receive rabbit ATG at 3.5 mg/kg/dose IV from day -6 to -2 with the goals of ablating host repressive T cells. |
| Rabbit ATG & AD-MSCs | Experimental | Drug: Rabbit antithymoglobulin (ATG), Procedure: Adipose derived mesenchymal stem cells ( AD-MSCs)Patient in this arm will receive rabbit ATG at 3.5 mg/kg/dose IV from day -6 to -2 and then patient's own adipose derived mesenchymal stem cells (AD-MSCs) at a dose of 3000000/kg/d on day 1 to 3. |
| Rabbit ATG & AD-HSCs | Experimental | Drug: Rabbit antithymoglobulin (ATG), Procedure: AD-MSC transdifferentiated HSCs (AD-HSCs)Patient in this arm will receive rabbit ATG at 3.5 mg/kg/dose IV from day -6 to -2 and then patient's own AD-MSC transdifferentiated HSCs (AD-HSCs) at a dose of 3000000/kg/d from day 1 to 4. |
Who may qualify
- Ages
- 14 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- Male or female recipients must have histopathologically confirmed diagnosis of SAA-I without or with more than 6 months after less than one treatment with ATG. Diagnostic Criteria for Server Aplastic Anemia will be based on the definitions set forth by the international Aplastic Anemia Study Group.
- Absolute neutrophil count ≤ 0.5 X 109/l, Platelet count ≤ 20 X 109 /l, Anemia with corrected reticulocyte count ≤ 1%, and Bone marrow cellularity ≤ 25%, or bone marrow cellularity ≤ 50% with fewer than 30% hematopoietic cell, Hepatic: alanine aminotransferase (ALT)/ aspartate aminotransferase (AST) no greater than 4 times normal, Bilirubin: no greater than 2 mg/dl, Renal: Creatinine clearance at least 50 ml/min, Cardiovascular: Shortening fraction or ejection fraction at least 40% of normal for age by echocardiogram or radionuclide scan.
- No clinically significant comorbid illnesses (e.g., myocardial infarction or cerebrovascular accident).
Key exclusion criteria (1 listed)
- Active and uncontrolled infection, Active bleeding, Severe allergic history of ATG, HIV-1 infection, Pregnancy or breastfeeding, Carbon monoxide lung diffusion capacity (DLCO) <40% predicted, SAA-II, Patients with severe psychological disorders, Recipients of other clinical trials.
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 |
|---|---|---|
| Rabbit antithymoglobulin (ATG)Also: ATGRabbit ATG at 3.5 mg/kg/dose IV is given from day -6 to -2. | Drug | Rabbit ATG & AD-HSCs, Rabbit ATG & AD-MSCs, Rabbit antithymoglobulin （ATG） |
| Adipose derived mesenchymal stem cells ( AD-MSCs)Also: AD-MSCsParticipants will receive rabbit ATG at 3.5 mg/kg/dose IV from day -6 to -2, and then patient's own AD-MSCs at a dose of 3000000 cells/kg/d on day 1-3. | Procedure or surgery | Rabbit ATG & AD-MSCs |
| AD-MSC transdifferentiated HSCs (AD-HSCs)Also: AD-HSCsParticipants will receive rabbit anti-thymocyte globulin at 3.5 mg/kg/dose IV from day -6 to -2, and then patient's own AD-HSCs at a dose of 3000000 cells/kg/d from day 1 to 4. | Procedure or surgery | Rabbit ATG & AD-HSCs |
Primary outcome
Engraftment at 42 days post AD-HSC transplantation for patients with severe aplastic anemia.
Time frame: 42 days posttransplant
Absolute neutrophil count > 0.5 X 109/l and Platelet count > 20 X 109 /l without infusion of platelet for 7 days.
8 secondary outcomes
To estimate the overall survival (OS) at 1 year following AD-HSC transplantation for Patients with Severe Aplastic Anemia
Time frame: 1 year
Relapse
Time frame: 1 year post transplant
Incidence of chronic graft-versus-host disease
Time frame: 6 months
Evaluation of the occurrence of secondary malignancies
Time frame: 6 months post transplant
Hematology labs
Time frame: 12 weeks
Number of participants with adverse events as a measure of safety and tolerability of intravenous AD-HSC infusion in patients with severe aplastic anemia
Time frame: weekly untill 12 months
Transfusional requirements
Time frame: weekly untill 6 months
To assess treatment related mortality
Time frame: 12 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Apr 2015.
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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.
james Q Yin, M.D.,Ph.D. principal investigator
The military general hospital of Beijing
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Rabbit antithymoglobulin (ATG), Adipose derived mesenchymal stem cells ( AD-MSCs) and AD-MSC transdifferentiated HSCs (AD-HSCs) in people with Severe Aplastic Anemia, run by Navy General Hospital, Beijing 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 4 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 3 Apr 2015. 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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact James Q Yin, M.D.,Ph.D. (86-01-84008003, Jamesyin2010@126.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Engraftment at 42 days post AD-HSC transplantation for patients with severe aplastic anemia. (time frame: 42 days posttransplant). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 3 Apr 2015.