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A Single-Arm Phase 2 Study With Optimized Standard Protocol for Severe Aplastic Anemia (OSP)

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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
Sponsor:
Information provided by (Responsible Party):
Yizhou Zheng, Institute of Hematology & Blood Diseases Hospital

Brief 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. 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.

Condition or disease Intervention/treatment Phase
Aplastic Anemia Drug: rabbit ATG, Cyclosporine, Levamisole Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 40 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
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.
Other Names:
  • rATG
  • CSA
  • LMS




Primary Outcome Measures :
  1. 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).



Secondary Outcome Measures :
  1. 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.




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Ages Eligible for Study:   6 Years to 70 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Newly diagnosed SAA (according to the standard criteria)

    1. Bone marrow cellularity less than 30% (excluding lymphocytes)
    2. 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

Exclusion Criteria:

  • 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.

Information from the National Library of Medicine

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


Contacts
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Contact: Nie Neng docnn@163.com

Locations
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China, Tianjin
Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences Recruiting
TianJin, Tianjin, China, 300020
Contact: Nie Neng    +86 22 23909023    docnn@163.com   
Principal Investigator: Zheng Yizhou, M.D., Ph.D         
Sponsors and Collaborators
Yizhou Zheng
Investigators
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Principal Investigator: Zheng Yizhou, M.D., Ph.D Anemia Therapeutic Center, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences
Publications of Results:
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Responsible Party: Yizhou Zheng, Yizhou Zheng, Vice director Institute of Hematology & Blood Diseases Hospital, Institute of Hematology & Blood Diseases Hospital
ClinicalTrials.gov Identifier: NCT02203396    
Other Study ID Numbers: ATGIIT201401
First Posted: July 29, 2014    Key Record Dates
Last Update Posted: May 4, 2016
Last Verified: May 2016
Keywords provided by Yizhou Zheng, Institute of Hematology & Blood Diseases Hospital:
SAA
Immunosuppressive treatment
ATG
Cyclosporine
Levamisole
Additional relevant MeSH terms:
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Anemia
Anemia, Aplastic
Hematologic Diseases
Bone Marrow Failure Disorders
Bone Marrow Diseases
Cyclosporine
Levamisole
Cyclosporins
Thymoglobulin
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antifungal Agents
Anti-Infective Agents
Dermatologic Agents
Antirheumatic Agents
Calcineurin Inhibitors
Adjuvants, Immunologic
Antinematodal Agents
Anthelmintics
Antiparasitic Agents