MDACC Study Summary 2005-0115

Study Summary
No. 2005-0115:.......Blood And Marrow Transplantation; Leukemia......Farhad Ravandi-Kashani......Leukemia

Study Summary Title



Study Summary Number:	2005-0115

Study Title:	Phase II study of combination of thymoglobulin, cyclosporine, methylprednisone, and GCSF in patients with newly diagnosed aplastic anemia or with hypoplastic or low/intermediate-1 risk myelodysplastic syndrome

Physician

New Patient Referral



Name:	Farhad Ravandi-Kashani	Patients Call:	800-392-1611 (in U.S.A.) 713-792-6161 (outside U.S.A.)

Dept:	Leukemia	Referring MD Call:	800-392-1611 (in U.S.A.) 713-792-6161 (outside U.S.A.)

Phone:	713-745-0394 Contact us about clinical trials

General Information



Disease Group:	Blood And Marrow Transplantation Leukemia	Supported By:	Genzyme incorporated

Phase of Study:	Phase II	Return Visit:	once or twice weekly depending on blood counts

Treatment Agents:	Cyclosporin-A G-CSF Methylprednisolone Thymoglobulin	Home Care:	oral medications

Treatment Loc:	Only at MDACC

Estimated Length of Stay in Houston:	4 days


Description/ Intervention:	The goal of this clinical research study is to learn if combining the drugs thymoglobulin, methylprednisolone, cyclosporine, and G-CSF (NeupogenTM) can help to control severe AA or hypoplastic MDS. The safety of this combination therapy will also be studied.

Study Objectives / Outcomes

Primary Endpoint:
To assess efficacy of the combination of thymoglobulin, methylprednisone, cyclosporine and GCSF in achieving response (complete response, partial response and hematological improvement) in patients with severe aplastic anemia or hypoplastic myelodysplastic syndrome and also to assess the effect of the treatment on transfusion requirements and overall survival.

Secondary Endpoint:
To assess the safety of the above combination.

Study Status Information



Study Activation / Registration Date:	05/12/2005

IRB Review and Approval Date:	04/06/2005

Study Type:	Therapeutic

Recruitment Status:	Open

Projected Accrual:	N/A

Enrollment Eligibility

If you do not meet the enrollment eligibility, there may be other treatment options for you. Please Contact the Referral Office for more information.

Inclusion Criteria:

1) Diagnosis of severe aplastic anemia (bone marrow cellularity < 30%, with two of three peripheral counts at the time of initial presentation or currently low with ANC < 500/mL, pre-transfusion PLT< 20,000/mL, or pre-transfusion hemoglobin < 8 g/dL and presence of no other underlying disorder.

2) Diagnosis of MDS (WHO) with bone marrow cellularity < 30%, with two of three peripheral counts at the time of initial presentation or currently low with ANC < 500/mL, pre-transfusion PLT < 20,000/mL, or pre-transfusion hemoglobin < 8 g/dL.

3) Patients with MDS who have received prior biological therapy (not chemotherapy) are eligible. Hypomethylating agents and histone deacetylase inhibitors are considered as biological therapy.

4) Age 15 or greater

5) Adequate renal function (creatinine less than or equal to 2.0 mg/dL) unless related to the disease

6) Adequate hepatic function (bilirubin less than or equal to 3.5 mg/dL) unless related to the disease

7) No other investigational therapy in the past 14 days

8) Able to sign consent form

9) Able to comply with the need for contraception (abstinence, condom, birth control pill, or other acceptable form of contraception) during the entire study period

10) Diagnosis of MDS (WHO) with bone marrow cellularity greater than 30%, with low or intermediate-1 risk by the IPSS score, and requiring treatment (i.e. transfusion-dependent)

Exclusion Criteria:

1) Active and uncontrolled infection

2) HIV positive test

3) Pregnant or breast feeding

4) Active and uncontrolled medical illness (pulmonary, cardiac, neurological, or other) that in the opinion of treating physician would likely interfere with study treatment

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