Aplastic AnemiaPhase 2OSPNCT02203396
A Single-Arm Phase 2 Study With Optimized Standard Protocol for Severe Aplastic Anemia
A Phase 2 study testing rabbit ATG, Cyclosporine, Levamisole in people with Aplastic Anemia, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
Trial summary
Severe acquired aplastic anaemia (SAA) is a bone marrow failure disease characterized by pancytopenia and a hypocellular bone marrow. The corn pathophysiological mechanism is the destruction of hematopoietic stem/progenitor cells mediated by auto-reactive effector T cells. Read more about this study
- Who is it for?
- Ages 6 to 70 years
- Conditions the study is for
- Aplastic Anemia
- What is being tested?
- rabbit ATG, Cyclosporine, Levamisole
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- 1 siteChina
- Study dates
- Aug 2014to Sep 2017
- 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 6 to 70 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Newly diagnosed SAA (according to the standard criteria)
- Bone marrow cellularity less than 30% (excluding lymphocytes)
- At least two of the following: Absolute neutrophil count less than 500/ uL; Platelet count less than 20,000/ uL; Absolute reticulocyte count less than 20,000/ uL.
- Age greater than or equal to 6 years old
Who cannot join
- Serum creatinine greater than 2.5 mg/dL
- Underlying carcinoma (except local cervical, basal cell, squamous cell)
- Prior immunosuppressive therapy with ATG, antilymphocyte globulin (ALG), or high dose cyclophosphamide.
- Current pregnancy or lactation or unwillingness to take oral contraceptives or use an effective method of birth control.
- Diagnosis of Fanconi anemia or other congenital bone marrow failure syndromes
- Evidence of a clonal disorder on cytogenetics. Patients with super severe neutropenia (ANC less than 200/uL) will not be excluded if results of cytogenetics are not available or pending.
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- Underlying immunodeficiency state including seropositivity for HIV
- Inability to understand the investigational nature of the study or give informed consent
- Moribund status or concurrent hepatic, renal, cardiac, neurologic, pulmonary, infectious, or metabolic disease of such severity that it would preclude the patient s ability to tolerate protocol therapy, or that death within 7-10 days is likely.
Trial locations and contacts
1 site in 1 country.
China1 site
Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences
Tianjin, Tianjin Municipality, 300020
About this study
Severe acquired aplastic anaemia (SAA) is a bone marrow failure disease characterized by pancytopenia and a hypocellular bone marrow. The corn pathophysiological mechanism is the destruction of hematopoietic stem/progenitor cells mediated by auto-reactive effector T cells. Immunosuppressive therapy with horse antithymocyte globulin (ATG) plus cyclosporine (CSA) is currently the standard of treatment in patients with aplastic anaemia who are not eligible for bone marrow transplantation and with response rates from 40% to 70%. Previous studies showed that horse ATG (hATG) is apparently more effective than rabbit ATG (rATG) as the latter has higher treatment related mortality (TRM). Unfortunately hATG is unavailable in China, so we conduct a optimized standard treatment (9 days protocol) of rATG plus CSA and Levamisole (LMS) Sequential maintaining (termed Optimized Standard Protocol, OSP) for severe aplastic anemia. This prospective study is designed to evaluate the efficacy and safety of Optimized Standard Protocol as first line therapy in newly diagnosed severe aplastic anemia patients.
Read the detailed description
During the treatment period, rATG is administered at a dose of 1.97 mg/kg/day for 9 days by slow intravenous infusion. CSA is administered orally at a dose of 3 mg/kg qod, and Levamisole was administered orally at a dose of 2.5 mg/kg qod. The CSA and Levamisole (LMS) is designed to alternately every other day.
- Conditions
- Aplastic Anemia
- Being tested
- rabbit ATG, Cyclosporine, Levamisole
- Keywords
- SAA, Immunosuppressive treatment, ATG, Cyclosporine, Levamisole
- 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 ATG, Cyclosporine, LevamisoleDrug
Also called rATG, CSA, LMS
rATG is administered at a dose of 1.97 mg/kg/day for 9 days CSA is administered orally at a dose of 3 mg/kg qod Levamisole is administered orally at a dose of 2.5 mg/kg qod. The CSA and LMS is designed to alternately every other day.
| Study group | Role | What they receive |
|---|---|---|
| Severe Aplastic Anemia | Experimental | Drug: rabbit ATG, Cyclosporine, Levamisole Receives: Drug: rabbit ATG, Cyclosporine, Levamisole |
What is being measured
The primary outcome is the main question the study is designed to answer.
the response and complete remission rate with Optimized Standard Protocol.
Response will be evaluated at each clinic visit. Complete response (CR) was defined as achieving all three peripheral blood count criteria: (1) Hb level up to the normal range; (2) ANC≥1.5×109/L; (3) PLT≥100×109/L.
Partial response (PR) was defined as transfusion independent, no longer meeting criteria for severe disease. Persistence of transfusion requirement or death was evidence of no response (NR).
Measured: month +6
Secondary outcomes (1)
Relapse rate, sustained response (SR), survival, and clonal evolution to myelodysplasia and acute leukemia.
Relapse was defined as a responder who met criteria for SAA again after achieving response and keeping stable blood counts for at least 3 months.
Sustained response (SR) was defined as Hb > 10 g/dL at month +12 and +60, in the absence of any treatment.
Measured: month +12, month +60
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Jul 2014 (estimated)
- Study startAug 2014
- Primary completionSep 2016
- Study completionSep 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 40 participants (actual)
Sponsor and investigators
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, China
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Zheng Yizhou, M.D., Ph.D, Principal investigator, Anemia Therapeutic Center, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02203396
- Sponsor's protocol ID
- IIT2014006
- Acronym
- OSP
- Status last verified
- May 2024
- First submitted
- 27 Jul 2014
- Last updated
- 11 Feb 2026
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Rosenfeld S, Follmann D, Nunez O, Young NS. Antithymocyte globulin and cyclosporine for severe aplastic anemia: association between hematologic response and long-term outcome. JAMA. 2003 Mar 5;289(9):1130-5. doi: 10.1001/jama.289.9.1130.
PubMed 12622583Camitta 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 11 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single-Arm Phase 2 Study With Optimized Standard Protocol for Severe Aplastic Anemia
- ClinicalTrials.gov ID
- NCT02203396
- Acronym / sponsor ID
- OSP / IIT2014006
- Conditions
- Aplastic Anemia
- Keywords
- SAA, Immunosuppressive treatment, ATG, Cyclosporine, Levamisole
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 40 participants (actual)
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, ChinaUniversity, hospital or other
- Study start
- Aug 2014
- Primary completion
- Sep 2016 (actual)
- Study completion
- Sep 2017 (actual)
- First posted
- 29 Jul 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.
Severe acquired aplastic anaemia (SAA) is a bone marrow failure disease characterized by pancytopenia and a hypocellular bone marrow. The corn pathophysiological mechanism is the destruction of hematopoietic stem/progenitor cells mediated by auto-reactive effector T cells. Immunosuppressive therapy with horse antithymocyte globulin (ATG) plus cyclosporine (CSA) is currently the standard of treatment in patients with aplastic anaemia who are not eligible for bone marrow transplantation and with response rates from 40% to 70%. Previous studies showed that horse ATG (hATG) is apparently more effective than rabbit ATG (rATG) as the latter has higher treatment related mortality (TRM). Unfortunately hATG is unavailable in China, so we conduct a optimized standard treatment (9 days protocol) of rATG plus CSA and Levamisole (LMS) Sequential maintaining (termed Optimized Standard Protocol, OSP) for severe aplastic anemia. This prospective study is designed to evaluate the efficacy and safety of Optimized Standard Protocol as first line therapy in newly diagnosed severe aplastic anemia patients.
Detailed description
During the treatment period, rATG is administered at a dose of 1.97 mg/kg/day for 9 days by slow intravenous infusion. CSA is administered orally at a dose of 3 mg/kg qod, and Levamisole was administered orally at a dose of 2.5 mg/kg qod. The CSA and Levamisole (LMS) is designed to alternately every other day.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Severe Aplastic Anemia | Experimental | Drug: rabbit ATG, Cyclosporine, LevamisoleDrug: rabbit ATG, Cyclosporine, Levamisole |
Who may qualify
- Ages
- 6 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- Newly diagnosed SAA (according to the standard criteria)
- Age greater than or equal to 6 years old
Key exclusion criteria (9 listed)
- Serum creatinine greater than 2.5 mg/dL
- Underlying carcinoma (except local cervical, basal cell, squamous cell)
- Prior immunosuppressive therapy with ATG, antilymphocyte globulin (ALG), or high dose cyclophosphamide.
- Current pregnancy or lactation or unwillingness to take oral contraceptives or use an effective method of birth control.
- Diagnosis of Fanconi anemia or other congenital bone marrow failure syndromes
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 ATG, Cyclosporine, LevamisoleAlso: rATG, CSA, LMSrATG is administered at a dose of 1.97 mg/kg/day for 9 days CSA is administered orally at a dose of 3 mg/kg qod Levamisole is administered orally at a dose of 2.5 mg/kg qod. The CSA and LMS is designed to alternately every other day. | Drug | Severe Aplastic Anemia |
Primary outcome
the response and complete remission rate with Optimized Standard Protocol.
Time frame: month +6
Response will be evaluated at each clinic visit. Complete response (CR) was defined as achieving all three peripheral blood count criteria: (1) Hb level up to the normal range; (2) ANC≥1.5×109/L; (3) PLT≥100×109/L. Partial response (PR) was defined as transfusion independent, no longer meeting criteria for severe disease. Persistence of transfusion requirement or death was evidence of no response (NR).
1 secondary outcome
Relapse rate, sustained response (SR), survival, and clonal evolution to myelodysplasia and acute leukemia.
Time frame: month +12, month +60
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 11 Feb 2026.
- Results publication: PubMed 12622583Rosenfeld S, Follmann D, Nunez O, Young NS. Antithymocyte globulin and cyclosporine for severe aplastic anemia: association between hematologic response and long-term outcome. JAMA. 2003 Mar 5;289(9):1130-5. doi: 10.1001/jama.289.9.1130.
- 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.
Zheng Yizhou, M.D., Ph.D principal investigator
Anemia Therapeutic Center, Institute of Hematology and Blood Diseases Hospital, 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 2 study testing rabbit ATG, Cyclosporine, Levamisole in people with Aplastic Anemia, run by Institute of Hematology & Blood Diseases Hospital, China 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 9 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 11 Feb 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?
the response and complete remission rate with Optimized Standard Protocol. (time frame: month +6). 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 NCT02203396, which also lists 2 linked publications.
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 11 Feb 2026.