Thymoglobulin and Cyclosporine in Patients With Aplastic Anemia or Myelodysplastic Syndrome
This study has been completed.
Sponsor:
M.D. Anderson Cancer Center
Collaborator:
Genzyme
Information provided by (Responsible Party):
M.D. Anderson Cancer Center
ClinicalTrials.gov Identifier:
NCT00806598
First received: December 9, 2008
Last updated: March 8, 2013
Last verified: March 2013
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Purpose
The goal of this clinical research study is to learn if combining the drugs thymoglobulin, methylprednisolone, cyclosporine, and G-CSF (NeupogenTM or NeulastaTM ) can help to control severe aplastic anemia (AA) or hypoplastic myelodysplastic syndrome (MDS). The safety of this combination therapy will also be studied.
| Condition | Intervention | Phase |
|---|---|---|
|
Myelodysplastic Syndrome Aplastic Anemia |
Drug: Thymoglobulin Drug: Cyclosporine Drug: Methylprednisolone Drug: G-CSF |
Phase 2 |
| Study Type: | Interventional |
| Study Design: | Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | Phase II Study of Combination of Thymoglobulin, Cyclosporine, Methylprednisone, and Granulocyte Colony-stimulating Factor (GCSF) in Patients With Newly Diagnosed Aplastic Anemia or With Hypoplastic or Low/Intermediate-1 Risk Myelodysplastic Syndrome |
Resource links provided by NLM:
Drug Information available for:
Prednisolone
Prednisolone acetate
Methylprednisolone acetate
Methylprednisolone
Prednisolone sodium phosphate
Prednisolone phosphate
Prednisolone sodium succinate
Methylprednisolone sodium succinate
Cyclosporine
Filgrastim
Sargramostim
Lenograstim
Granulocyte colony-stimulating factor
Antilymphocyte Serum
U.S. FDA Resources
Further study details as provided by M.D. Anderson Cancer Center:
Primary Outcome Measures:
- Overall Response [ Time Frame: From date of randomization until the date of first documented progression or date of death from any cause, whichever came first. Assessed first at 3 months on study, continuing monthly up to 3 years. ] [ Designated as safety issue: No ]Complete response (CR) was defined as normalization of peripheral blood and bone marrow with <5% blasts, a peripheral absolute neutrophil count (ANC) >/= 1 * 10^9/l, hemoglobin >/= 100g/l, and a platelet count >/= * 10^9/l, Partial Response (PR) was defined as transfusion independence with a peripheral blood ANC >=/ 0.05 * 10^9/l, a platelet count >/= 20 * 10^9/l, and a hemoglobin >/= 40 g/l. Hematologic improvement was defined as a clinically relevant increase in hemoglobin, platelets or absolute neutrophil count.
| Enrollment: | 53 |
| Study Start Date: | May 2005 |
| Study Completion Date: | June 2012 |
| Primary Completion Date: | June 2012 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
Experimental: Thymoglobulin + Cyclosporin
Combination of Thymoglobulin 3.5 or 2.5 mg/kg/day intravenous (IV) for 5 days + Methylprednisone 1 mg/kg/day IV for 5 days, before each dose Thymoglobulin + Cyclosporin 5 mg/kg orally for 6 months following Thymoglobulin + Granulocyte - Colony Stimulating Factor (G-CSF) 5 microgram/kg subcutaneously daily up to 3 months
|
Drug: Thymoglobulin
3.5 or 2.5 mg/kg/day IV for 5 days
Other Names:
Drug: Cyclosporine
5 mg/kg orally for 6 months; start after completing thymoglobulin.
Other Names:
Drug: Methylprednisolone
1 mg/kg/day IV for 5 days, given before each dose of thymoglobulin.
Other Names:
Drug: G-CSF
5 microgram/kg subcutaneously daily up to 3 months, start after thymoglobulin.
Other Names:
|
Show Detailed Description Eligibility| Ages Eligible for Study: | 15 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- 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 absolute neutrophil count (ANC) < 500/mL, pre-transfusion platelet (PLT) < 20,000/mL, or pre-transfusion hemoglobin < 8 g/dL and presence of no other underlying disorder.
- Diagnosis of MDS (World Health Organization) 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.
- Patients with MDS who have received prior biological therapy (not chemotherapy) are eligible. Hypomethylating agents and histone deacetylase inhibitors are considered as biological therapy.
- Age 15 or greater
- Adequate renal function (creatinine less than or equal to 2.0 mg/dL) unless related to the disease
- Adequate hepatic function (bilirubin less than or equal to 3.5 mg/dL) unless related to the disease
- No other investigational therapy in the past 14 days
- Able to sign consent form
- Able to comply with the need for contraception (abstinence, condom, birth control pill, or other acceptable form of contraception) during the entire study period
- Diagnosis of MDS (WHO) with bone marrow cellularity greater than 30%, with low or intermediate-1 risk by the International Prognostic Scoring System (IPSS) score, and requiring treatment (i.e. transfusion-dependent)
Exclusion Criteria:
- Active and uncontrolled infection
- HIV positive test
- Pregnant or breast feeding
- Active and uncontrolled medical illness (pulmonary, cardiac, neurological, or other) that in the opinion of treating physician would likely interfere with study treatment
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00806598
Locations
| United States, Texas | |
| UT MD Anderson Cancer Center | |
| Houston, Texas, United States, 77030 | |
Sponsors and Collaborators
M.D. Anderson Cancer Center
Genzyme
Investigators
| Principal Investigator: | Tapan M. Kadia, M.D. | M.D. Anderson Cancer Center |
More Information
Additional Information:
No publications provided
| Responsible Party: | M.D. Anderson Cancer Center |
| ClinicalTrials.gov Identifier: | NCT00806598 History of Changes |
| Other Study ID Numbers: | 2005-0115 |
| Study First Received: | December 9, 2008 |
| Results First Received: | January 30, 2013 |
| Last Updated: | March 8, 2013 |
| Health Authority: | United States: Institutional Review Board |
Keywords provided by M.D. Anderson Cancer Center:
|
Myelodysplastic Syndrome Aplastic Anemia Thymoglobulin |
Cyclosporine Methylprednisolone G-CSF |
Additional relevant MeSH terms:
|
Anemia Anemia, Aplastic Myelodysplastic Syndromes Preleukemia Hematologic Diseases Bone Marrow Diseases Precancerous Conditions Neoplasms Antilymphocyte Serum Cyclosporins Cyclosporine Lenograstim Methylprednisolone acetate Prednisolone acetate Methylprednisolone |
Methylprednisolone Hemisuccinate Prednisolone Prednisolone hemisuccinate Prednisolone phosphate Immunosuppressive Agents Immunologic Factors Physiological Effects of Drugs Pharmacologic Actions Enzyme Inhibitors Molecular Mechanisms of Pharmacological Action Antifungal Agents Anti-Infective Agents Therapeutic Uses Dermatologic Agents Antirheumatic Agents |
ClinicalTrials.gov processed this record on May 23, 2013