A Single-Arm Phase 2 Study With Optimized Standard Protocol for Severe Aplastic Anemia (OSP)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT02203396|
Recruitment Status : Unknown
Verified May 2016 by Yizhou Zheng, Institute of Hematology & Blood Diseases Hospital.
Recruitment status was: Recruiting
First Posted : July 29, 2014
Last Update Posted : May 4, 2016
- Study Details
- Tabular View
- No Results Posted
- How to Read a Study Record
|Condition or disease||Intervention/treatment||Phase|
|Aplastic Anemia||Drug: rabbit ATG, Cyclosporine, Levamisole||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||40 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Single-Arm Phase 2 Study With Optimized Standard Protocol for Severe Aplastic Anemia|
|Study Start Date :||August 2014|
|Estimated Primary Completion Date :||September 2016|
|Estimated Study Completion Date :||September 2017|
Experimental: Severe Aplastic Anemia
Drug: rabbit ATG, Cyclosporine, Levamisole
Drug: rabbit ATG, Cyclosporine, Levamisole
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.
- 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).
- Relapse rate, sustained response (SR), survival, and clonal evolution to myelodysplasia and acute leukemia. [ Time Frame: month +12, month +60 ]
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.
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
|Ages Eligible for Study:||6 Years to 70 Years (Child, Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
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
- 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.
- 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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02203396
|Contact: Nie Nengfirstname.lastname@example.org|
|Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences||Recruiting|
|TianJin, Tianjin, China, 300020|
|Contact: Nie Neng +86 22 23909023 email@example.com|
|Principal Investigator: Zheng Yizhou, M.D., Ph.D|
|Principal Investigator:||Zheng Yizhou, M.D., Ph.D||Anemia Therapeutic Center, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences|
|Responsible Party:||Yizhou Zheng, Yizhou Zheng, Vice director Institute of Hematology & Blood Diseases Hospital, Institute of Hematology & Blood Diseases Hospital|
|Other Study ID Numbers:||
|First Posted:||July 29, 2014 Key Record Dates|
|Last Update Posted:||May 4, 2016|
|Last Verified:||May 2016|
Bone Marrow Failure Disorders
Bone Marrow Diseases
Molecular Mechanisms of Pharmacological Action
Physiological Effects of Drugs