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

NCT01231841 | Completed Phase 2 Results DMC

• 2 other identifiers: CCF7922NCI-2010-01365
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

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.

Conditions

Aplastic Anemia

Outcome Measures

Primary Outcomes (1)

• 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.

  At 6 months

Study Arms (1)

rATG + Cyclosporine

EXPERIMENTAL

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

Drug: cyclosporine; Biological: anti-thymocyte globulin

Interventions

cyclosporine DRUG

Given orally

Also known as: 27-400, ciclosporin, cyclosporin, cyclosporin A, CYSP, Sandimmune

rATG + Cyclosporine

anti-thymocyte globulin BIOLOGICAL

Given IV

Also known as: ATG, ATGAM, lymphocyte immune globulin, Thymoglobulin

rATG + Cyclosporine

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• The Cleveland Clinic: lead

Study Sites (1)

Cleveland Clinic Taussig Cancer Institute, Case Comprehensive Cancer Center

Cleveland, Ohio, 44195, United States

Location

Related Publications (1)

• 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.

  PMID: 21606164 DERIVED

MeSH Terms

Conditions

Anemia, Aplastic

Interventions

Cyclosporine; Cyclosporins; Antilymphocyte Serum; thymoglobulin

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Failure Disorders; Bone Marrow Diseases

Intervention Hierarchy (Ancestors)

Peptides, Cyclic; Macrocyclic Compounds; Polycyclic Compounds; Peptides; Amino Acids, Peptides, and Proteins; Immune Sera; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Serum Globulins; Globulins; Biological Products; Complex Mixtures

Results Point of Contact

• Title: Jaroslaw Maciejewski, MD PhD, Principal Investigator
• Organization: Cleveland Clinic Foundation

maciejj@ccf.org

216-445-5962

Study Officials

• Jaroslaw Maciejewski

  Cleveland Clinic Taussig Cancer Institute, Case Comprehensive Cancer Center

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 29, 2010
• First Posted: November 1, 2010
• Study Start: March 1, 2005
• Primary Completion: December 1, 2010
• Study Completion: December 1, 2010
• Last Updated: June 22, 2023
• Results First Posted: March 9, 2012

Record last verified: 2018-01

Locations

US (1)