Trial Outcomes & Findings for Thymoglobulin and Cyclosporine in Patients With Aplastic Anemia or Myelodysplastic Syndrome (NCT NCT00806598)
NCT ID: NCT00806598
Last Updated: 2013-03-12
Results Overview
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.
COMPLETED
PHASE2
53 participants
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.
2013-03-12
Participant Flow
Recruitment Period 5/12/2005 - 6/20/2012; all participants were registered at The University of Texas M.D. Anderson Cancer Center.
A total of 53 participants were enrolled on the study, with four patients choosing alternative treatment; one participant died before administration of study treatment.
Participant milestones
| Measure |
Thymoglobulin + Cyclosporin
Combination of Thymoglobulin + Methylprednisone + Cyclosporin + G-CSF
Cyclosporine : 5 mg/kg orally for 6 months; start after completing thymoglobulin.
G-CSF : G-CSF 5 microgram/kg subcutaneously daily up to 3 months, start after thymoglobulin.
Thymoglobulin : 3.5 or 2.5 mg/kg/day IV for 5 days
* Aplastic anemia patients receive 3.5 mg/kg/day for 5 days
* MDS patients \<55 years receive 3.5 mg/kg/day for 5 days
* MDS patients \>55 years receive 2.5 mg/kg/day for 5 days
Methylprednisolone : 1 mg/kg/day IV for 5 days, given before each dose of thymoglobulin.
|
|---|---|
|
Overall Study
STARTED
|
48
|
|
Overall Study
COMPLETED
|
48
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Thymoglobulin and Cyclosporine in Patients With Aplastic Anemia or Myelodysplastic Syndrome
Baseline characteristics by cohort
| Measure |
Thymoglobulin + Cyclosporin
n=48 Participants
Combination of Thymoglobulin + Methylprednisone + Cyclosporin + G-CSF
Cyclosporine : 5 mg/kg orally for 6 months; start after completing thymoglobulin.
G-CSF : G-CSF 5 microgram/kg subcutaneously daily up to 3 months, start after thymoglobulin.
Thymoglobulin : 3.5 or 2.5 mg/kg/day IV for 5 days
* Aplastic anemia patients receive 3.5 mg/kg/day for 5 days
* MDS patients \<55 years receive 3.5 mg/kg/day for 5 days
* MDS patients \>55 years receive 2.5 mg/kg/day for 5 days
Methylprednisolone : 1 mg/kg/day IV for 5 days, given before each dose of thymoglobulin.
|
|---|---|
|
Age Continuous
|
59 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
22 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
26 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
48 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 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.Population: Of the 48 participants who received treatment 46 were evaluable for response.
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.
Outcome measures
| Measure |
Thymoglobulin + Cyclosporin
n=46 Participants
Combination of Thymoglobulin + Methylprednisone + Cyclosporin + Granulocyte Colony stimulating factor (G-CSF
Cyclosporine : 5 mg/kg orally for 6 months; start after completing thymoglobulin.
G-CSF : G-CSF 5 microgram/kg subcutaneously daily up to 3 months, start after thymoglobulin.
Thymoglobulin : 3.5 or 2.5 mg/kg/day IV for 5 days
* Aplastic anemia patients receive 3.5 mg/kg/day for 5 days
* MDS patients \<55 years receive 3.5 mg/kg/day for 5 days
* MDS patients \>55 years receive 2.5 mg/kg/day for 5 days
Methylprednisolone : 1 mg/kg/day IV for 5 days, given before each dose of thymoglobulin.
|
|---|---|
|
Overall Response
Complete Response
|
8 participants
|
|
Overall Response
Partial Response
|
8 participants
|
|
Overall Response
Hematological Improvement
|
4 participants
|
Adverse Events
Thymoglobulin + Cyclosporin
Serious adverse events
| Measure |
Thymoglobulin + Cyclosporin
n=48 participants at risk
Combination of Thymoglobulin + Methylprednisone + Cyclosporin + G-CSF
Cyclosporine : 5 mg/kg orally for 6 months; start after completing thymoglobulin.
G-CSF : G-CSF 5 microgram/kg subcutaneously daily up to 3 months, start after thymoglobulin.
Thymoglobulin : 3.5 or 2.5 mg/kg/day IV for 5 days
* Aplastic anemia patients receive 3.5 mg/kg/day for 5 days
* MDS patients \<55 years receive 3.5 mg/kg/day for 5 days
* MDS patients \>55 years receive 2.5 mg/kg/day for 5 days
Methylprednisolone : 1 mg/kg/day IV for 5 days, given before each dose of thymoglobulin.
|
|---|---|
|
General disorders
Fever
|
2.1%
1/48 • Number of events 1 • Seven years, one month
|
|
Nervous system disorders
Syncope
|
2.1%
1/48 • Number of events 1 • Seven years, one month
|
|
Infections and infestations
Infection
|
6.2%
3/48 • Number of events 3 • Seven years, one month
|
|
Infections and infestations
Neutropenic Fever
|
2.1%
1/48 • Number of events 1 • Seven years, one month
|
|
Nervous system disorders
Confusion
|
2.1%
1/48 • Number of events 1 • Seven years, one month
|
|
General disorders
Death
|
4.2%
2/48 • Number of events 2 • Seven years, one month
|
|
Blood and lymphatic system disorders
Prolonged Thrombocytopenia
|
2.1%
1/48 • Number of events 1 • Seven years, one month
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory Alkalosis
|
2.1%
1/48 • Number of events 1 • Seven years, one month
|
|
General disorders
Abdominal Pain
|
2.1%
1/48 • Number of events 1 • Seven years, one month
|
Other adverse events
| Measure |
Thymoglobulin + Cyclosporin
n=48 participants at risk
Combination of Thymoglobulin + Methylprednisone + Cyclosporin + G-CSF
Cyclosporine : 5 mg/kg orally for 6 months; start after completing thymoglobulin.
G-CSF : G-CSF 5 microgram/kg subcutaneously daily up to 3 months, start after thymoglobulin.
Thymoglobulin : 3.5 or 2.5 mg/kg/day IV for 5 days
* Aplastic anemia patients receive 3.5 mg/kg/day for 5 days
* MDS patients \<55 years receive 3.5 mg/kg/day for 5 days
* MDS patients \>55 years receive 2.5 mg/kg/day for 5 days
Methylprednisolone : 1 mg/kg/day IV for 5 days, given before each dose of thymoglobulin.
|
|---|---|
|
Blood and lymphatic system disorders
Neutropenia
|
50.0%
24/48 • Number of events 24 • Seven years, one month
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
45.8%
22/48 • Number of events 22 • Seven years, one month
|
|
Renal and urinary disorders
Elevated Creatinine
|
39.6%
19/48 • Number of events 19 • Seven years, one month
|
|
Hepatobiliary disorders
Elevated Transaminases
|
33.3%
16/48 • Number of events 16 • Seven years, one month
|
|
Hepatobiliary disorders
Elevated Bilirubin
|
31.2%
15/48 • Number of events 15 • Seven years, one month
|
|
Blood and lymphatic system disorders
Anemia
|
27.1%
13/48 • Number of events 13 • Seven years, one month
|
|
Metabolism and nutrition disorders
Elevated Uric Acid
|
14.6%
7/48 • Number of events 7 • Seven years, one month
|
|
Skin and subcutaneous tissue disorders
Rash
|
14.6%
7/48 • Number of events 7 • Seven years, one month
|
|
General disorders
Fatigue
|
12.5%
6/48 • Number of events 6 • Seven years, one month
|
|
Metabolism and nutrition disorders
Low Magnesium
|
12.5%
6/48 • Number of events 6 • Seven years, one month
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
12.5%
6/48 • Number of events 6 • Seven years, one month
|
|
Gastrointestinal disorders
Nausea/Vomining
|
10.4%
5/48 • Number of events 5 • Seven years, one month
|
|
Metabolism and nutrition disorders
Low Potassium
|
10.4%
5/48 • Number of events 5 • Seven years, one month
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
10.4%
5/48 • Number of events 5 • Seven years, one month
|
|
Metabolism and nutrition disorders
Low Sodium
|
6.2%
3/48 • Number of events 3 • Seven years, one month
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
6.2%
3/48 • Number of events 3 • Seven years, one month
|
|
General disorders
Edema
|
6.2%
3/48 • Number of events 3 • Seven years, one month
|
|
Gastrointestinal disorders
Diarrhea
|
6.2%
3/48 • Number of events 3 • Seven years, one month
|
|
General disorders
Infusion Reaction
|
47.9%
23/48 • Number of events 23 • Seven years, one month
|
|
Infections and infestations
Infection
|
20.8%
10/48 • Number of events 10 • Seven years, one month
|
|
Infections and infestations
Neutropenic Fever
|
18.8%
9/48 • Number of events 9 • Seven years, one month
|
Additional Information
Tapan Kadia, MD/Assistant Professor
The University of Texas MD Anderson Cancer Center
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place