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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)


Phase 2
12 Years
N/A
Not Enrolling
Both
Aplastic Anemia

Thank you

Trial Information

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)


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.


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

Type of Study:

Interventional

Study Design:

Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

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

Outcome Description:

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.

Outcome Time Frame:

At 6 months

Safety Issue:

No

Principal Investigator

Jaroslaw Maciejewski

Investigator Role:

Principal Investigator

Investigator Affiliation:

Cleveland Clinic Taussig Cancer Institute, Case Comprehensive Cancer Center

Authority:

United States: Institutional Review Board

Study ID:

CCF7922

NCT ID:

NCT01231841

Start Date:

March 2005

Completion Date:

December 2010

Related Keywords:

  • Aplastic Anemia
  • Anemia
  • Anemia, Aplastic

Name

Location

Cleveland Clinic Taussig Cancer Institute, Case Comprehensive Cancer Center Cleveland, Ohio  44195