Trial Outcomes & Findings for Study of Cyclosporine or Corticosteroids as an Adjunct to Plasma Exchange in Thrombotic Thrombocytopenic Purpura (TTP) (NCT NCT00713193)
NCT ID: NCT00713193
Last Updated: 2023-05-03
Results Overview
Number of Participants with Exacerbations in the CSA/PEX Arm Compared to the Steroids/PEX Arm
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
26 participants
Primary outcome timeframe
From the start of treatment until 30 days after discharge from the last PEX procedure
Results posted on
2023-05-03
Participant Flow
Participant milestones
| Measure |
CSA Arm
Patients in this arm will receive cyclosporine (Neoral) at a dose of 2-3 mg/kg orally as an adjunct to plasma exchange.
Cyclosporine: 2-3 mg/kg orally in a twice day divided dose for 6 months
All patients initiated daily PEX (one plasma volume), using plasma as the replacement fluid. Patients randomized to the corticosteroid arm received prednisone at a dose of 1 mg/kg/d rounded to the nearest 20 mg increment. Patients randomized to the CSA arm received CSA at a dose of 2-3 mg/kg (rounded to the nearest 50 mg increment) divided into a twice daily dosing (Figure 1a). Patients randomized to CSA did not receive steroids, but were permitted to receive hydrocortisone as required for any hypersensitivity reactions to the infused plasma. Daily PEX was continued until a clinical response was achieved, then patients received PEX every other day for 2 additional procedures, with the first day that the platelet count and LDH were normal counting as the first of the 2 procedures.
|
Prednisone Arm
Patients in this arm will receive prednisone at a dose of 1 mg/kg as an adjunct to plasma exchange.
Prednisone: 1 mg/kg orally, daily for at least 30 days, then tapered over 30 days after achieving remission.
All patients initiated daily PEX (one plasma volume), using plasma as the replacement fluid. Patients randomized to the corticosteroid arm received prednisone at a dose of 1 mg/kg/d rounded to the nearest 20 mg increment. Patients randomized to the CSA arm received CSA at a dose of 2-3 mg/kg (rounded to the nearest 50 mg increment) divided into a twice daily dosing (Figure 1a). Patients randomized to CSA did not receive steroids, but were permitted to receive hydrocortisone as required for any hypersensitivity reactions to the infused plasma. Daily PEX was continued until a clinical response was achieved, then patients received PEX every other day for 2 additional procedures
|
|---|---|---|
|
Overall Study
STARTED
|
12
|
14
|
|
Overall Study
COMPLETED
|
12
|
14
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Study of Cyclosporine or Corticosteroids as an Adjunct to Plasma Exchange in Thrombotic Thrombocytopenic Purpura (TTP)
Baseline characteristics by cohort
| Measure |
CSA Arm
n=12 Participants
Patients in this arm will receive cyclosporine (Neoral) at a dose of 2-3 mg/kg orally as an adjunct to plasma exchange.
At the time of diagnosis, patients will be started on daily cyclosporine (Neoral) therapy at a dose of 2-3 mg/kg/day (rounded to the nearest 50 mg increment) concurrent with daily plasma exchange. Doses will be administered in the capsule form. The total dose will be divided into twice daily dosing. For consistency and standardization of cyclosporine levels throughout the study, doses of Neoral (cyclosporine) should be given at 8 AM and 8 PM each day. Neoral (cyclosporine) should be taken on an empty stomach, and patients should not eat for at least 30 minutes after their dose of Neoral (cyclosporine). Cyclosporine will be continued for a total of six months of therapy and then discontinued.
|
Prednisone Arm
n=14 Participants
Patients in this arm will receive prednisone at a dose of 1 mg/kg as an adjunct to plasma exchange.
Prednisone: 1 mg/kg orally, daily for at least 30 days, then tapered over 30 days after achieving remission.
At the time of diagnosis, patients will be started on daily prednisone therapy at a dose of 1 mg/kg/day (rounded off to the nearest 20 mg multiple) concurrently with daily PE. Patients unable to take oral corticosteroids will be switched to an equivalent intravenous corticosteroid until they are able to take oral medications. Patients may receive intravenous corticosteroids at any time to urgently treat reactions to the infused plasma and for prophylaxis against future reactions prior to the next TPE.
|
Total
n=26 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
12 Participants
n=5 Participants
|
14 Participants
n=7 Participants
|
26 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Continuous
|
40 years
n=5 Participants
|
37 years
n=7 Participants
|
37 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=5 Participants
|
14 Participants
n=7 Participants
|
16 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
10 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
10 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
12 Participants
n=5 Participants
|
14 Participants
n=7 Participants
|
26 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
2 Participants
n=5 Participants
|
14 Participants
n=7 Participants
|
16 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
10 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
10 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
12 participants
n=5 Participants
|
14 participants
n=7 Participants
|
26 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: From the start of treatment until 30 days after discharge from the last PEX procedureNumber of Participants with Exacerbations in the CSA/PEX Arm Compared to the Steroids/PEX Arm
Outcome measures
| Measure |
CSA Arm
n=12 Participants
Patients in this arm will receive cyclosporine (Neoral) at a dose of 2-3 mg/kg orally as an adjunct to plasma exchange.
All patients initiated daily PEX (one plasma volume), using plasma as the replacement fluid. Patients randomized to the CSA arm received CSA at a dose of 2-3 mg/kg (rounded to the nearest 50 mg increment) divided into a twice daily dosing (Figure 1a). Patients randomized to CSA did not receive steroids, but were permitted to receive hydrocortisone as required for any hypersensitivity reactions to the infused plasma. Daily PEX was continued until a clinical response was achieved, then patients received PEX every other day for 2 additional procedures, with the first day that the platelet count and LDH were normal counting as the first of the 2 procedures.
Cyclosporine: 2-3 mg/kg orally in a twice day divided dose for 6 months
|
Prednisone Arm
n=14 Participants
Patients in this arm will receive prednisone at a dose of 1 mg/kg as an adjunct to plasma exchange.
All patients initiated daily PEX (one plasma volume), using plasma as the replacement fluid. Patients randomized to the corticosteroid arm received prednisone at a dose of 1 mg/kg/d rounded to the nearest 20 mg increment. Daily PEX was continued until a clinical response was achieved, then patients received PEX every other day for 2 additional procedures, with the first day that the platelet count and LDH were normal counting as the first of the 2 procedures.
Prednisone: 1 mg/kg orally, daily for at least 30 days, then tapered over 30 days after achieving remission.
|
|---|---|---|
|
Number of Participants With Exacerbations in the CSA/PEX Arm Compared to the Steroids/PEX Arm
|
3 participants
|
1 participants
|
SECONDARY outcome
Timeframe: Time to starting treatment until 6 months after the last PEX procedureDays to achieve a clinical response, defined as a normal platelet count (\>150 x 109/L), normal LDH, and no new end organ injury.
Outcome measures
| Measure |
CSA Arm
n=12 Participants
Patients in this arm will receive cyclosporine (Neoral) at a dose of 2-3 mg/kg orally as an adjunct to plasma exchange.
All patients initiated daily PEX (one plasma volume), using plasma as the replacement fluid. Patients randomized to the CSA arm received CSA at a dose of 2-3 mg/kg (rounded to the nearest 50 mg increment) divided into a twice daily dosing (Figure 1a). Patients randomized to CSA did not receive steroids, but were permitted to receive hydrocortisone as required for any hypersensitivity reactions to the infused plasma. Daily PEX was continued until a clinical response was achieved, then patients received PEX every other day for 2 additional procedures, with the first day that the platelet count and LDH were normal counting as the first of the 2 procedures.
Cyclosporine: 2-3 mg/kg orally in a twice day divided dose for 6 months
|
Prednisone Arm
n=14 Participants
Patients in this arm will receive prednisone at a dose of 1 mg/kg as an adjunct to plasma exchange.
All patients initiated daily PEX (one plasma volume), using plasma as the replacement fluid. Patients randomized to the corticosteroid arm received prednisone at a dose of 1 mg/kg/d rounded to the nearest 20 mg increment. Daily PEX was continued until a clinical response was achieved, then patients received PEX every other day for 2 additional procedures, with the first day that the platelet count and LDH were normal counting as the first of the 2 procedures.
Prednisone: 1 mg/kg orally, daily for at least 30 days, then tapered over 30 days after achieving remission.
|
|---|---|---|
|
Time in Days to Achieve a Clinical Response, Comparing the CSA/PEX Arm to the Steroids/PEX Arm.
|
5 Days
Interval 4.0 to 19.0
|
5 Days
Interval 4.0 to 8.0
|
Adverse Events
CSA Arm
Serious events: 0 serious events
Other events: 0 other events
Deaths: 1 deaths
Prednisone Arm
Serious events: 0 serious events
Other events: 0 other events
Deaths: 1 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place