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Anti-thymocyte Globulin and Cyclosporine as First-Line Therapy in Treating Patients With Severe Aplastic Anemia

Primary Purpose

Aplastic Anemia

Status

Completed

Phase

Phase 2

Locations

United States

Study Type

Interventional

Intervention

cyclosporine

anti-thymocyte globulin

About this trial

This is an interventional treatment trial for Aplastic Anemia

Eligibility Criteria

12 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

All patients with sAA as defined by Camitta who are candidates for IS therapy; these criteria include bone marrow cellularity < 25% or 25-50% with < 30% of hematopoietic cells; it should also have two of the following three parameters: peripheral blood neutrophils < 0.5 x 10^9/L, platelets < 20 x 10^9/L and reticulocytes < 60 x 10^9/L in anemic patients
If cytogenetic testing has been done, it should show normal karyotype or be not informative
Patients should be either unwilling or otherwise ineligible (age, comorbidities, lack of donor) for bone marrow transplantation as a therapeutic modality
Not previously treated with ATG for sAA
Patients must have ECOG performance status of 0, 1, or 2
Vitamin B12 and folic acid deficiency must be ruled out by measurement of serum levels
Patients must have had a bone marrow biopsy examination in the three months prior to enrolling in the study
Must be able to provide informed consent
Systemic and other hematologic causes of pancytopenia, based on clinical presentation, must have been ruled out

Exclusion Criteria:

Patients with clinically evident congestive heart failure, serious cardiac arrhythmias; symptoms of coronary artery disease must be cleared by cardiology prior to therapy
Patients who have had chemotherapy, radiotherapy, or immunotherapy or other investigational drug use within 3 weeks prior to study entry
Pregnant women
All females of childbearing potential must have a blood test or urine study within two weeks prior to induction registration to rule out pregnancy
Women of childbearing potential are strongly advised to use an accepted and effective method of contraception
Patients who have medical, psychological, or social conditions that may interfere with the patient's participation in the study or evaluation of the study results

Sites / Locations

Cleveland Clinic Taussig Cancer Institute, Case Comprehensive Cancer Center

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

rATG + Cyclosporine

Arm Description

Patients receive anti-thymocyte globulin IV daily over 4-24 hours on days 1-5. Beginning on day 6, patients receive oral cyclosporine twice daily for 6 months followed by a taper. Treatment continues in the absence of disease progression or unacceptable toxicity

Outcomes

Primary Outcome Measures

Patients Treated With Rabbit Antithymocyte Globulin (r-ATG/Thymoglobulin) and Cyclosporine (CsA) Achieving at Least a Partial Remission (PR) at 6 Months

Patients will be classified as responders if they have transfusion independence and meet two of the following three criteria: ANC greater than 500/mm3; platelet count greater than 20,000/mm3; and reticulocyte count greater than 40,000/mm3. Transfusion independence is defined as no need for transfusions for one month prior to response assessment.

Secondary Outcome Measures

Full Information

NCT ID

NCT01231841

First Posted

October 29, 2010

Last Updated

May 25, 2023

Sponsor

The Cleveland Clinic

1. Study Identification

Unique Protocol Identification Number

NCT01231841

Brief Title

Anti-thymocyte Globulin and Cyclosporine as First-Line Therapy in Treating Patients With Severe Aplastic Anemia

Official Title

Protocol for Prospective Phase II Study of Rabbit Antithymocyte Globulin (r-ATG/Thymoglobulin) and Cyclosporine (CsA) as a First Line Immunosuppressive (IS) Therapy for Severe Aplastic Anemia (sAA)

Study Type

Interventional

2. Study Status

Record Verification Date

January 2018

Overall Recruitment Status

Completed

Study Start Date

March 2005 (undefined)

Primary Completion Date

December 2010 (Actual)

Study Completion Date

December 2010 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

The Cleveland Clinic

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

RATIONALE: Immunosuppressive therapies, such as anti-thymocyte globulin and cyclosporine, may improve bone marrow function and increase blood cell counts. PURPOSE: This phase II trial is studying how well giving anti-thymocyte globulin together with cyclosporine as first-line therapy works in treating patients with severe aplastic anemia.

Detailed Description

PRIMARY OBJECTIVES: To determine the response rate of r-ATG and CsA in the first line setting. SECONDARY OBJECTIVES: To determine the level of IS as assessed by Immuknow assay in responders and compare it to non-responders. OUTLINE:Patients receive anti-thymocyte globulin IV over 4-24 hours daily on days 1-5. Beginning on day 6, patients receive oral cyclosporine twice daily for 6 months followed by a taper. Treatment continues in the absence of disease progression or unacceptable toxicity.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Aplastic Anemia

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

20 (Actual)

8. Arms, Groups, and Interventions

Arm Title

rATG + Cyclosporine

Arm Type

Experimental

Arm Description

Intervention Type

Drug

Intervention Name(s)

cyclosporine

Other Intervention Name(s)

27-400, ciclosporin, cyclosporin, cyclosporin A, CYSP, Sandimmune

Intervention Description

Given orally

Intervention Type

Biological

Intervention Name(s)

anti-thymocyte globulin

Other Intervention Name(s)

ATG, ATGAM, lymphocyte immune globulin, Thymoglobulin

Intervention Description

Given IV

Primary Outcome Measure Information:

Title

Patients Treated With Rabbit Antithymocyte Globulin (r-ATG/Thymoglobulin) and Cyclosporine (CsA) Achieving at Least a Partial Remission (PR) at 6 Months

Description

Time Frame

At 6 months

10. Eligibility

Sex

All

Minimum Age & Unit of Time

12 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: All patients with sAA as defined by Camitta who are candidates for IS therapy; these criteria include bone marrow cellularity < 25% or 25-50% with < 30% of hematopoietic cells; it should also have two of the following three parameters: peripheral blood neutrophils < 0.5 x 10^9/L, platelets < 20 x 10^9/L and reticulocytes < 60 x 10^9/L in anemic patients If cytogenetic testing has been done, it should show normal karyotype or be not informative Patients should be either unwilling or otherwise ineligible (age, comorbidities, lack of donor) for bone marrow transplantation as a therapeutic modality Not previously treated with ATG for sAA Patients must have ECOG performance status of 0, 1, or 2 Vitamin B12 and folic acid deficiency must be ruled out by measurement of serum levels Patients must have had a bone marrow biopsy examination in the three months prior to enrolling in the study Must be able to provide informed consent Systemic and other hematologic causes of pancytopenia, based on clinical presentation, must have been ruled out Exclusion Criteria: Patients with clinically evident congestive heart failure, serious cardiac arrhythmias; symptoms of coronary artery disease must be cleared by cardiology prior to therapy Patients who have had chemotherapy, radiotherapy, or immunotherapy or other investigational drug use within 3 weeks prior to study entry Pregnant women All females of childbearing potential must have a blood test or urine study within two weeks prior to induction registration to rule out pregnancy Women of childbearing potential are strongly advised to use an accepted and effective method of contraception Patients who have medical, psychological, or social conditions that may interfere with the patient's participation in the study or evaluation of the study results

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Jaroslaw Maciejewski

Organizational Affiliation

Cleveland Clinic Taussig Cancer Institute, Case Comprehensive Cancer Center

Official's Role

Principal Investigator

Facility Information:

Facility Name

Cleveland Clinic Taussig Cancer Institute, Case Comprehensive Cancer Center

City

Cleveland

State/Province

Ohio

ZIP/Postal Code

44195

Country

United States

12. IPD Sharing Statement

Citations:

PubMed Identifier

21606164

Citation

Afable MG 2nd, Shaik M, Sugimoto Y, Elson P, Clemente M, Makishima H, Sekeres MA, Lichtin A, Advani A, Kalaycio M, Tiu RV, O'Keefe CL, Maciejewski JP. Efficacy of rabbit anti-thymocyte globulin in severe aplastic anemia. Haematologica. 2011 Sep;96(9):1269-75. doi: 10.3324/haematol.2011.042622. Epub 2011 May 23.

Results Reference

derived

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Anti-thymocyte Globulin and Cyclosporine as First-Line Therapy in Treating Patients With Severe Aplastic Anemia

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