Trial Outcomes & Findings for Safety Study of Danazol With Plasma Exchange and Steroids for the Treatment of Thrombotic Thrombocytopenic Purpura (TTP) (NCT NCT00953771)
NCT ID: NCT00953771
Last Updated: 2024-07-23
Results Overview
The total number of plasma exchanges performed after initiation of the first plasma exchange.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
9 participants
Primary outcome timeframe
up to 30 days
Results posted on
2024-07-23
Participant Flow
Study opened for patient recruitment on Oct 29, 2008 at Beth Israel Medical Center and St. Luke's-Roosevelt Hospital Center. The study was closed by the IRB on 10/12/2018 due to low enrollment. The last date of study data collection was 11/19/2015.
Subjects were not enrolled to the control group; data was retrospectively collected by viewing the charts of 20 patients who received Plasma Exchange
Participant milestones
| Measure |
Danazol, Plex, Steroids
Everyone will receive Danazol with plasma exchange and corticosteroids
Danazol: Danazol 600 mg PO will be initiated with plasma exchange and corticosteroids at the time of enrollment.
|
Historic Control
The subjects were not enrolled to the control group; data was retrospectively collected by viewing the charts of 20 patients who received Plasma Exchange
|
|---|---|---|
|
Overall Study
STARTED
|
9
|
20
|
|
Overall Study
COMPLETED
|
8
|
20
|
|
Overall Study
NOT COMPLETED
|
1
|
0
|
Reasons for withdrawal
| Measure |
Danazol, Plex, Steroids
Everyone will receive Danazol with plasma exchange and corticosteroids
Danazol: Danazol 600 mg PO will be initiated with plasma exchange and corticosteroids at the time of enrollment.
|
Historic Control
The subjects were not enrolled to the control group; data was retrospectively collected by viewing the charts of 20 patients who received Plasma Exchange
|
|---|---|---|
|
Overall Study
Death
|
1
|
0
|
Baseline Characteristics
Safety Study of Danazol With Plasma Exchange and Steroids for the Treatment of Thrombotic Thrombocytopenic Purpura (TTP)
Baseline characteristics by cohort
| Measure |
Danazol, Plex, Steroids
n=9 Participants
Everyone will receive Danazol with plasma exchange and corticosteroids
Danazol: Danazol 600 mg PO will be initiated with plasma exchange and corticosteroids at the time of enrollment.
|
Historic Control
The subjects were not enrolled to the control group; data was retrospectively collected by viewing the charts of 20 patients who received Plasma Exchange
|
Total
n=9 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
51.2 years
STANDARD_DEVIATION 13.8 • n=5 Participants
|
—
|
51.2 years
STANDARD_DEVIATION 13.8 • n=5 Participants
|
|
Sex: Female, Male
Female
|
6 Participants
n=5 Participants
|
—
|
6 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
3 Participants
n=5 Participants
|
—
|
3 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
3 Participants
n=5 Participants
|
—
|
3 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
6 Participants
n=5 Participants
|
—
|
6 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
—
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
—
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
—
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
—
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
5 Participants
n=5 Participants
|
—
|
5 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
4 Participants
n=5 Participants
|
—
|
4 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
—
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
—
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: up to 30 daysThe total number of plasma exchanges performed after initiation of the first plasma exchange.
Outcome measures
| Measure |
Danazol, Plex, Steroids
n=9 Participants
Everyone will receive Danazol with plasma exchange and corticosteroids
Danazol: Danazol 600 mg PO will be initiated with plasma exchange and corticosteroids at the time of enrollment.
|
Historic Control
n=20 Participants
The subjects were not enrolled to the control group; data was retrospectively collected by viewing the charts of 20 patients who received Plasma Exchange
|
|---|---|---|
|
Number of Plasma Exchanges
|
14.8 exchanges
Standard Deviation 9.64
|
16.1 exchanges
Standard Deviation 8.93
|
SECONDARY outcome
Timeframe: up to 30 daysOutcome measures
| Measure |
Danazol, Plex, Steroids
n=9 Participants
Everyone will receive Danazol with plasma exchange and corticosteroids
Danazol: Danazol 600 mg PO will be initiated with plasma exchange and corticosteroids at the time of enrollment.
|
Historic Control
n=20 Participants
The subjects were not enrolled to the control group; data was retrospectively collected by viewing the charts of 20 patients who received Plasma Exchange
|
|---|---|---|
|
Time to Remission
|
11.4 days
Standard Deviation 5.05
|
11.0 days
Standard Deviation 7.06
|
SECONDARY outcome
Timeframe: up to 30 daysOutcome measures
| Measure |
Danazol, Plex, Steroids
n=8 Participants
Everyone will receive Danazol with plasma exchange and corticosteroids
Danazol: Danazol 600 mg PO will be initiated with plasma exchange and corticosteroids at the time of enrollment.
|
Historic Control
n=20 Participants
The subjects were not enrolled to the control group; data was retrospectively collected by viewing the charts of 20 patients who received Plasma Exchange
|
|---|---|---|
|
Length of Stay
|
27.3 days
Standard Deviation 17.7
|
24.9 days
Standard Deviation 13.1
|
SECONDARY outcome
Timeframe: At 2 YearsPopulation: Two of the 20 historical controls did not have data for hospitalization days or time to remission, therefore computed complete response rates among the 18 patients for whom full data was available.
Outcome measures
| Measure |
Danazol, Plex, Steroids
n=9 Participants
Everyone will receive Danazol with plasma exchange and corticosteroids
Danazol: Danazol 600 mg PO will be initiated with plasma exchange and corticosteroids at the time of enrollment.
|
Historic Control
n=18 Participants
The subjects were not enrolled to the control group; data was retrospectively collected by viewing the charts of 20 patients who received Plasma Exchange
|
|---|---|---|
|
Number of Participants With Complete and Continuous Response Rate
Complete Response Rate
|
9 Participants
|
17 Participants
|
|
Number of Participants With Complete and Continuous Response Rate
Continuous Response Rate
|
8 Participants
|
16 Participants
|
SECONDARY outcome
Timeframe: up to 12 yearsOutcome measures
| Measure |
Danazol, Plex, Steroids
n=1 Participants
Everyone will receive Danazol with plasma exchange and corticosteroids
Danazol: Danazol 600 mg PO will be initiated with plasma exchange and corticosteroids at the time of enrollment.
|
Historic Control
n=8 Participants
The subjects were not enrolled to the control group; data was retrospectively collected by viewing the charts of 20 patients who received Plasma Exchange
|
|---|---|---|
|
Time to Relapse
|
43 months
Data for 1 participant, therefore no range
|
28.5 months
Interval 2.5 to 97.0
|
SECONDARY outcome
Timeframe: up to 12 yearsNumber of participants with relapses
Outcome measures
| Measure |
Danazol, Plex, Steroids
n=9 Participants
Everyone will receive Danazol with plasma exchange and corticosteroids
Danazol: Danazol 600 mg PO will be initiated with plasma exchange and corticosteroids at the time of enrollment.
|
Historic Control
n=20 Participants
The subjects were not enrolled to the control group; data was retrospectively collected by viewing the charts of 20 patients who received Plasma Exchange
|
|---|---|---|
|
Number of Participants With Relapses
|
1 Participants
|
4 Participants
|
SECONDARY outcome
Timeframe: up to 12 yearsNumber of relapses
Outcome measures
| Measure |
Danazol, Plex, Steroids
n=1 Participants
Everyone will receive Danazol with plasma exchange and corticosteroids
Danazol: Danazol 600 mg PO will be initiated with plasma exchange and corticosteroids at the time of enrollment.
|
Historic Control
n=4 Participants
The subjects were not enrolled to the control group; data was retrospectively collected by viewing the charts of 20 patients who received Plasma Exchange
|
|---|---|---|
|
Number of Relapses
|
1 relapses
|
6 relapses
|
Adverse Events
Danazol, Plex, Steroids
Serious events: 2 serious events
Other events: 0 other events
Deaths: 1 deaths
Historic Control
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Danazol, Plex, Steroids
n=9 participants at risk
Everyone will receive Danazol with plasma exchange and corticosteroids
Danazol: Danazol 600 mg PO will be initiated with plasma exchange and corticosteroids at the time of enrollment.
|
Historic Control
The subjects were not enrolled to the control group; data was retrospectively collected by viewing the charts of 20 patients who received Plasma Exchange
|
|---|---|---|
|
Respiratory, thoracic and mediastinal disorders
pulmonary edema
|
11.1%
1/9 • 12 years
Adverse events not collected for the historic control arm
|
—
0/0 • 12 years
Adverse events not collected for the historic control arm
|
|
Vascular disorders
hypotension
|
11.1%
1/9 • 12 years
Adverse events not collected for the historic control arm
|
—
0/0 • 12 years
Adverse events not collected for the historic control arm
|
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place