Trial Outcomes & Findings for Cyclophosphamide Plus Cyclosporine in Treatment-Naive Severe Aplastic Anemia (NCT NCT01193283)
NCT ID: NCT01193283
Last Updated: 2017-05-22
Results Overview
The safety endpoint will be toxicity profile after 6 months of treatment. The efficacy endpoint is complete response rate at 6 months, with complete response defined as blood counts no longer meeting the standard criteria for severe pancytopenia in severe aplastic anemia.
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
22 participants
Primary outcome timeframe
6 months
Results posted on
2017-05-22
Participant Flow
Participant milestones
| Measure |
SAA Hematologic Response
Treatment-naive severe aplastic anemia patients will receive a low dose of cyclophosphamide (120mg/kg) and low dose cyclosporine ( target therapeutic level of 100-200 micrograms per liter). Cyclophosphamide will be given once daily for 4 doses. Cyclosporine will be started after cyclophosphamide completion, cyclosporine will be given twice daily. The dosing will be modified to attain the therapeutic level.
Cyclophosphamide: 30 my/kg for 4 days
Cyclosporine: daily to a trough of 100 t0 200 ng/ml
|
|---|---|
|
Overall Study
STARTED
|
22
|
|
Overall Study
COMPLETED
|
21
|
|
Overall Study
NOT COMPLETED
|
1
|
Reasons for withdrawal
| Measure |
SAA Hematologic Response
Treatment-naive severe aplastic anemia patients will receive a low dose of cyclophosphamide (120mg/kg) and low dose cyclosporine ( target therapeutic level of 100-200 micrograms per liter). Cyclophosphamide will be given once daily for 4 doses. Cyclosporine will be started after cyclophosphamide completion, cyclosporine will be given twice daily. The dosing will be modified to attain the therapeutic level.
Cyclophosphamide: 30 my/kg for 4 days
Cyclosporine: daily to a trough of 100 t0 200 ng/ml
|
|---|---|
|
Overall Study
Death
|
1
|
Baseline Characteristics
Cyclophosphamide Plus Cyclosporine in Treatment-Naive Severe Aplastic Anemia
Baseline characteristics by cohort
| Measure |
SAA Hematologic Response
n=22 Participants
Treatment-naive severe aplastic anemia patients will receive a low dose of cyclophosphamide (120mg/kg) and low dose cyclosporine ( target therapeutic level of 100-200 micrograms per liter). Cyclophosphamide will be given once daily for 4 doses. Cyclosporine will be started after cyclophosphamide completion, cyclosporine will be given twice daily. The dosing will be modified to attain the therapeutic level.
Cyclophosphamide: 30 my/kg for 4 days
Cyclosporine: daily to a trough of 100 t0 200 ng/ml
|
|---|---|
|
Age, Categorical
<=18 years
|
4 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
16 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
2 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
7 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
15 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
8 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
14 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
22 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 6 monthsThe safety endpoint will be toxicity profile after 6 months of treatment. The efficacy endpoint is complete response rate at 6 months, with complete response defined as blood counts no longer meeting the standard criteria for severe pancytopenia in severe aplastic anemia.
Outcome measures
| Measure |
SAA Hematologic Response
n=21 Participants
Treatment-naive severe aplastic anemia patients will receive a low dose of cyclophosphamide (120mg/kg) and low dose cyclosporine ( target therapeutic level of 100-200 micrograms per liter). Cyclophosphamide will be given once daily for 4 doses. Cyclosporine will be started after cyclophosphamide completion, cyclosporine will be given twice daily. The dosing will be modified to attain the therapeutic level.
Cyclophosphamide: 30 my/kg for 4 days
Cyclosporine: daily to a trough of 100 t0 200 ng/ml
|
|---|---|
|
Blood Counts and Adverse Event Profile After 6 Months of Treatment.
Complete response
|
4 participants
|
|
Blood Counts and Adverse Event Profile After 6 Months of Treatment.
Partial Response
|
5 participants
|
|
Blood Counts and Adverse Event Profile After 6 Months of Treatment.
No Response
|
12 participants
|
Adverse Events
SAA Hematologic Response
Serious events: 16 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
SAA Hematologic Response
n=22 participants at risk
Treatment-naive severe aplastic anemia patients will receive a low dose of cyclophosphamide (120mg/kg) and low dose cyclosporine ( target therapeutic level of 100-200 micrograms per liter). Cyclophosphamide will be given once daily for 4 doses. Cyclosporine will be started after cyclophosphamide completion, cyclosporine will be given twice daily. The dosing will be modified to attain the therapeutic level.
Cyclophosphamide: 30 my/kg for 4 days
Cyclosporine: daily to a trough of 100 t0 200 ng/ml
|
|---|---|
|
Infections and infestations
Catheter-related infection
|
18.2%
4/22 • Number of events 4
|
|
Infections and infestations
Bacterial infection
|
27.3%
6/22 • Number of events 6
|
|
Immune system disorders
Fungal infection
|
13.6%
3/22 • Number of events 3
|
|
Infections and infestations
Pnuemonia
|
31.8%
7/22 • Number of events 7
|
|
Blood and lymphatic system disorders
Febril neutropenia
|
36.4%
8/22 • Number of events 8
|
|
Gastrointestinal disorders
Pancreatitis
|
4.5%
1/22 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Lung abscess
|
4.5%
1/22 • Number of events 1
|
|
Gastrointestinal disorders
Cholelithiasis
|
4.5%
1/22 • Number of events 1
|
|
Gastrointestinal disorders
Gastroenteritis
|
4.5%
1/22 • Number of events 1
|
|
Blood and lymphatic system disorders
Hemorrhage
|
4.5%
1/22 • Number of events 1
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
soft tissue neoplasm
|
4.5%
1/22 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
cellulitis
|
4.5%
1/22 • Number of events 1
|
|
Infections and infestations
sinus infection
|
4.5%
1/22 • Number of events 1
|
|
Blood and lymphatic system disorders
petechiae
|
4.5%
1/22 • Number of events 1
|
|
Infections and infestations
blood infection
|
22.7%
5/22 • Number of events 5
|
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place