Severe Aplastic AnemiaPhase 4NCT01997372
Different Doses of Anti-thymocyte Globin to Treat Child Severe Aplastic Anemia
A Phase 4 study testing ATG in people with Severe Aplastic Anemia, run by Chinese Academy of Medical Sciences at 1 site in China.
Trial summary
Severe acquired aplastic anaemia (SAA) is a life-threatening disease characterized by pancytopenia and hypoplastic bone marrow. Immunosuppressive treatment with antithymocyte globulin (ATG)and cyclosporine remain the standard regimen with response rates of 70% or more and excellent overall survival. Read more about this study
- Who is it for?
- Ages 1 year to 18 years
- Conditions the study is for
- Severe Aplastic Anemia
- What is being tested?
- ATG
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 100 participants
- Where
- 1 siteChina
- Study dates
- Dec 2010to Nov 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 year to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- clinical diagnosis of childhood acquired severe aplastic anemia(SAA)
Who cannot join
- clinical diagnosis of no childhood acquired severe aplastic anemia(SAA)
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Department of Pediatrics, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences
Tianjin, Tianjin Municipality, 300020
Xiaofan Zhu, MD, +86 22 23909001, zhuxiaof@yahoo.com.cn
About this study
Official title: Different Doses of Anti-thymocyte Globin With 2.5 or 3.75mg/kg to Treat Child Severe Aplastic Anemia
Severe acquired aplastic anaemia (SAA) is a life-threatening disease characterized by pancytopenia and hypoplastic bone marrow. Immunosuppressive treatment with antithymocyte globulin (ATG)and cyclosporine remain the standard regimen with response rates of 70% or more and excellent overall survival. However ,there are no clinical trials to illustrate the response and complete remission rate with different doses of ATG.And there are no data reported on children with SAA so far.
- Conditions
- Severe Aplastic Anemia
- Being tested
- ATG
- Keywords
- ATG, SAA, immunosuppressive treatment, cyclosporine
- Medical subject headings
- Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ATGDrug
Drug ATG:2.5mg/kg/d or 3.75mg/kg/d for 5 days; Drug Cyclosporine A (CSA):3-10mg/kg/d ,Adjust the dose to maintain drug levels between 150 and 300ng/ml; Drug prednisone:1mg/kg/d,d1-21 from the first dosage of ATG; Drug Granulocyte Colony-Stimulating Factor(G-CSF):5ug/kg/d until absolute neutrophil count (ANC) >1×109/L.
| Study group | Role | What they receive |
|---|---|---|
| high dose ATG,low dose ATG | Experimental | Not described Receives: Drug: 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 ATG to treat child severe aplastic anemia
Complete response (CR) was defined as achieving normal levels of hemoglobin adjusted for age, platelet count >100×109/L, and ANC>1.5×109/L. Partial response (PR) was defined as transfusion independence, reticulocyte count >30×109/L, platelet count >20×109/L, and ANC >0.5×109/L above the baseline. Persistence of transfusion requirement or death was evidence of no response (NR).
Measured: 1 years
Secondary outcomes (1)
the relapse rate with different doses of ATG to treat child severe aplastic anemia
relapse was defined as transfusion dependence again; or progressed to paroxysmal nocturnal hemoglobinuria (PNH) /acute myeloid leukemia/myelodysplasia syndrome (MDS); or CSA dependence
Measured: 4-10 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Nov 2013 (estimated)
- Study startDec 2010
- Primary completionDec 2013 (estimated)
- Study completionNov 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 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 4
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 100 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
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01997372
- Sponsor's protocol ID
- Xzhu
- Status last verified
- Dec 2013
- First submitted
- 1 Mar 2012
- Last updated
- 3 Dec 2013
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Camitta BM, Rappeport JM, Parkman R, Nathan DG. Selection of patients for bone marrow transplantation in severe aplastic anemia. Blood. 1975 Mar;45(3):355-63.
PubMed 1090310
Study at a glance
From the registry record, last updated by the sponsor on 3 Dec 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Different Doses of Anti-thymocyte Globin With 2.5 or 3.75mg/kg to Treat Child Severe Aplastic Anemia
- ClinicalTrials.gov ID
- NCT01997372
- Acronym / sponsor ID
- Xzhu
- Conditions
- Severe Aplastic Anemia
- Keywords
- ATG, SAA, immunosuppressive treatment, cyclosporine
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- Chinese Academy of Medical SciencesUniversity, hospital or other
- Study start
- Dec 2010
- Primary completion
- Dec 2013 (estimated)
- Study completion
- Nov 2015 (estimated)
- First posted
- 28 Nov 2013 (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.
Severe acquired aplastic anaemia (SAA) is a life-threatening disease characterized by pancytopenia and hypoplastic bone marrow. Immunosuppressive treatment with antithymocyte globulin (ATG)and cyclosporine remain the standard regimen with response rates of 70% or more and excellent overall survival. However ,there are no clinical trials to illustrate the response and complete remission rate with different doses of ATG.And there are no data reported on children with SAA so far.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| high dose ATG,low dose ATG | Experimental | Drug: ATG |
Who may qualify
- Ages
- 1 year to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (1 listed)
- clinical diagnosis of childhood acquired severe aplastic anemia(SAA)
Key exclusion criteria (1 listed)
- clinical diagnosis of no childhood acquired severe aplastic anemia(SAA)
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 |
|---|---|---|
| ATGDrug ATG:2.5mg/kg/d or 3.75mg/kg/d for 5 days; Drug Cyclosporine A (CSA):3-10mg/kg/d ,Adjust the dose to maintain drug levels between 150 and 300ng/ml; Drug prednisone:1mg/kg/d,d1-21 from the first dosage of ATG; Drug Granulocyte Colony-Stimulating Factor(G-CSF):5ug/kg/d until absolute neutrophil count (ANC) >1×109/L. | Drug | high dose ATG,low dose ATG |
Primary outcome
the response and complete remission rate with different doses of ATG to treat child severe aplastic anemia
Time frame: 1 years
Complete response (CR) was defined as achieving normal levels of hemoglobin adjusted for age, platelet count >100×109/L, and ANC>1.5×109/L. Partial response (PR) was defined as transfusion independence, reticulocyte count >30×109/L, platelet count >20×109/L, and ANC >0.5×109/L above the baseline. Persistence of transfusion requirement or death was evidence of no response (NR).
1 secondary outcome
the relapse rate with different doses of ATG to treat child severe aplastic anemia
Time frame: 4-10 years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Dec 2013.
- Results publication: PubMed 1090310Camitta BM, Rappeport JM, Parkman R, Nathan DG. Selection of patients for bone marrow transplantation in severe aplastic anemia. Blood. 1975 Mar;45(3):355-63.
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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.
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 ATG in people with Severe Aplastic Anemia, 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 3 Dec 2013. 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, zhuxiaof@yahoo.com.cn), 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 ATG to treat child severe aplastic anemia (time frame: 1 years). 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 NCT01997372, 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 3 Dec 2013.